FOLD - Earnings call transcript Q1 2022 | Amicus Therapeutics, Inc.

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Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics’ First Quarter 2022 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. of Director Executive Faughnan, Investor Relations. Andrew

begin. may You

Andrew Faughnan

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

we on Campbell, Financial Officer. Bradley Chief Officer; have Speaking Dr. and call, John Chairman Castelli, Executive President Quimi, and Daphne Officer; Jeff Crowley, Operating and Officer; Chief Chief Development today’s Chief

Sébastien have Joining Goldman, and Business Mitchell we’ll Officer; Dr. Martel, for Officer. Q&A, Medical Chief Chief

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

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

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

it pleasure turn to Chairman John? Report forward-looking forward-looking and Form statements the our For later uncertainties Executive a this of be and such Risk and over we Commission. XXXX filed year today the my on John quarter with to XXXX to full time, the Factors discussion risks statements Securities for Annual Chief and ended the Exchange to call our them, and you of Crowley, Officer. and XX-K the that may ended refer section At the XX, March for impact is

John Crowley

focus Amicus Andrew, business to environment year I'm reiterate all across the welcome this the market we start sector. XXXX Thank despite priorities conference the first Great. our quarter highlight and the business biotech across on our as everyone pleased our team's results know to and stock successful you, call. tumultuous to

by begin Two, advance globally. many launch Galafold the focused are any approvals strength sharply many as Pompe AT-GAA of path possible. three for including financial one, the me as Let we in without patients and Three, for ensure primary emphasizing and continue geographies objectives; secure that equity-based further to profitability as to to need financing. on the Amicus, Disease

As we elaborate did in key on this accomplishments. me let press morning's release, several

its remains or first we with our be cornerstone increase all First, strong million from XX% Galafold globally. Its year this key and in a nearly is quarter meeting revenue the far year and growth Galafold uptake performance exceeding of very of continues objectives With ago, geographies. the thus pleased $XX.X our in success. to continue

broad and and Matter issued provide intellectual Galafold the rights the This XXXX, that to been the long-term year. there including which the late announce excited XX protection XXXXs very through also strengthened United estate recently portfolio related portfolio novel in are patents are provide to patents for this into of issuance Composition precision for by We orange the has Together property property listed intellectual issued well of patent. book eight this important Galafold now medicine. will States, new

Galafold next billion has potential $X achieve the and Galafold IP for it growth significant runway that that expect long believe beyond, and peak continues revenue. a decade We in have and to strengthened this the XXXX protection, with in into to

the with very filings our extremely regulatory the Second, on with are remain progress and engagement our well FDA, reviews to AT-GAA, of next recently Pompe pleased we U.S. The We AT-GAA's next therapy regulatory provability review we EU for labeling cycle for novel in have of are generation completed disease. regulatory with disease. U.S. late discussions the care to the confident potential great level standard and its We progressing become now continue from Pompe agency. global and now in the make agencies. in the and are

to the AT-GAA the for at be WuXi expect facility approval. biologic component for this again ATBXXX, the that the manufacturing and continue We FDA needed inspection of will biologic

situation are and watching in inspection. potential could We an carefully the recognize it of COVID-XX that China, the timing impact

the requirements. with evaluate support to We best also all including the to to options work we to are the In on are approve applications inspections, continue with way our expect actively in together AT-GAA. the at with the fully July and all agency preparatory partners applications we action WuXi or the continues the internal inspection. the have authorization Amicus MAA engaged marketing prepared. inspection by to communication agency XX date. We for an and are Today EU, respect completed regarding the mock EMA

will this expect launch indicated to the WuXi not of the new continue a positive Of We in importantly, commercial and year, manufacturing a it EMA has opinion CHMP require site. later note inspection that XXXX.

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

ahead. this closer the the and for disease, people treatment people note the approved regulatory both potential also, planned with with United the We having Of novel FDA EMA living another are in paradigm for U.S. Pompe names applications States Europe living new with and further have now to treatment both for Pompe. in the trade in option months

strong not until Amicus one we third which course, importantly, in And will remains disclose profitability and brand a global committed approval. maintained to So achieving expect position and name, has we financial of XXXX.

the and global the to As expansion we AT-GAA. Galafold continue for execute of of global launch prepare on

profitability. Pompe, need this order and in for we efforts making of Galafold science growth core to regulatory in technologies and concentrate judicious In the equity accomplish our global by linked therapies and this, launch and in medicine. will Pompe growth our continued Fabry achieve field disease or we'll priority vast the and activities and financings next majority and genetic equity do any to of of commercial investments in through investments global in advancing disease no strategic have franchises We AT-GAA platform to and commercialization generation and secure the the to Fabry in approvals, for

approaching We pivotal double-digit we Amicus; with of month, believe are medicine points inflection laid our and achieving disease and we growth XX%, enable of include, strong key constant and companies. X, for precision to $XXX disease behind the These out to at this in with million strategic of a Galafold drive $XXX truly focused biotechnology profitability. of amenable feats product priorities are XX% AT-GAA rare variance that year. to Amicus second globally. for the Ahead echelon with million will people the These on momentum launch to two secondly, first, as we are we new global Slide global on existing upper revenue one revenue last the rates. enter medicine This to rapidly reflects living continue We look Fabry demand achieve with in will exchange more to markets.

regulatory prepared we successful Second, to team States. all anticipated remain are of steadfast drug in effective Leveraging the we across initiating and of launch, and experience filings an commitment our in and launch AT-GAA. AT-GAA anticipate areas United AT-GAA our launch a seasoned fully for advancing the global commercial

investments we to introduction, best-in-class, our again to and position the carefully major next we are strategically we strong financial finally, me fully let Operating a Galafold go we ahead expenses and over Chief call all maintain performance. capabilities Campbell, and further advancing our generation milestones. remain that manage as now medicines turn Officer funded highlight With Bradley? Bradley continue to the through President Third, and and and genetic our

Bradley Campbell

Good John. everyone. thanks, morning, Great,

Let X. me start by more walking in on detail Slide you performance Galafold our quarter through the for

United first adherence globally. or breakdown coming continue product that business. we patient million States, generated quarter revenue of historically this rates. from the grow patient For United The is split geographic within new quarter with of XX/XX line parts This and of consistent or revenue what expect driven the during revenue grew $XX remaining XX% the $XX and the again, roughly the accruals XX.X% to to by $XX.X and with total was was with compliance strong we million XX% we've continued as the both sustained seen and States million the in outside

commercial QX be the and to exchange the markets all of operational patients rates. turning over constant the in added rate results year to major continues global an business This growth with Slide X, resilient XX.X% Now highlight at same last efforts. incredibly of strength quarter

as see QX. you continued graphs is percentage we've annual As in can The depicted slide, observe to pay a United revenue orders, States. rate as in QX into variety the growth reauthorizations on to and well revenue of the including smaller a this falls nonlinear, of of typically two results timing due pattern of factors the were follow

XX% remains populations, is amenable treated year XX% launch. will with starting under We and in stronger patients. about uptake in the share naïve the of patients. first drivers Here naïve switch global foundation also we've the see we're mix In with out for many of while this line to geographies, strategy which and lay a from which the patients called And the growth ended our of quarter metrics half global little market several beyond. and

these still high been opportunity to now longest, achieving we've to untreated about but the to and whereas as States, to plenty naïve share we're switch over there market patients. fact, So continue Japan is shares switch still to in versus market although approved patients mix continue on the of Europe, by and market continue Galafold EU, penetrate where bringing being there Japan, to XX/XX the grow to The well. diagnosed in longest, XX% patients business, now patients in undiagnosed there's patients, countries U.S., overall growth diagnosed evolving where and United an our segments at the towards of is out we're into newly more of we're we driven diagnosed the switch well. XX% about markets. And and we've in Within been and patient as newly naïve, the market the growth untreated as the core coming see we UK

Brazil, and Africa, year, several continue net where the later On outside Middle throughout you see as and East to the countries switch the countries States, we that next newer Asia well the coming the hand, Argentina, countries markets hoping other American Latin patients Pacific including approval at the like Chile, North Japan, as to we're Gulf expect Colombia, of we this if the in in of and patients look Turkey, years. new are get majority

Galafold. be vast granted is rates those belief value that the of generally outside reimbursement stay insurance patients on record United impressive majority and quarter, on payers in compliance reiterating XX% of reauthorizations by XXXX go that of Galafold underpinned the strong first who All continues exceed adherence recognized and continue very by States. a of by renegotiating the with track U.S. to within negotiating payers the successfully and to importantly, our Galafold And that again

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

on we during occur three-year the given of occur right slide, the Within side provided the each average proportion we've of expect a sales year. year table and quarter the a of snapshot historical similar this a trend to that Galafold

for conducted COVID constant more on of is number see to improvement color as well. very environment. of track to far all to major XX% FX We're treatment. to on new team continues Fabry and call. around Slide moving track in sales to most the will physician actually a the increase seen survey to period, prioritize to guidance seen to world and that diagnosis an tenfold and think initiation interactions unlock. things COVID field strong view over that's XXXX. our to the But of care to the XXXX said feel terms well and recently physicians our later achieve year medical start demand almost patient we of and they're altogether, at growth continue again million with is XXX Now little number the same still of as Fabry provide XX% of exchange what face-to-face Fabry and so that in $XXX well of full in the majority million the year. physicians our of we bit of impacting of in We this Daphne this from rates, in-person only of about our sign of starting across January a February we've continue uptake great Galafold as Encouragingly, February a XXXX, a and signs markets, access seeing X, $XXX to January markets the we've and as revenue very year interactions in And we're a and smaller We on patients

the to primary the new Moving growth revenue to into drivers through over has forward to call Galafold Castelli, we about; by risk continue to Dr. Composition clinical see of provide surpass $X in medical support of Officer exclusivity billion through Slide Europe in little our longer-term, our protection that next book labels Fabry in for Galafold including provide that, existing opportunity high screening, opportunity well. come. into XX we that expand a over access Development in know we years annual continue diagnosing in coverage we which which XXXX, ongoing that newborn AT-GAA Jeff? finally, XX diagnostic to With through give broadening as of our driven other And turn addition potential to initiatives geographies, continue program. us Matter give in continue on, looking XX, to and markets, us invest a as globally and $XXX our Galafold. it now as to IP million John orphan now peak including bit, remain to touched years screening, Galafold to variety Jeff and In of all the the to the confident and across market we well to as growth globally that few listed highlight U.S. latest measures, let regulatory our XXXXs, many three talk to the initiatives, penetrate me patent, revenue continuing orange as for which have we to Chief late the patients the significant the patents teams,

Jeffrey Castelli

work ongoing to doing the Galafold. build XX, good morning, and of body we the evidence Bradley Starting around on highlight we're here Slide Thanks, everyone.

global First, we the well broaden as the continue look variants pediatric populations adding additional to within adolescent mutations or labels to the and as to label.

as continues in diligently conference last treatment the treatment to highlighted phenotype long-term data publications reports, molecular medical presented function at of and or irrespective our These classic stable on inclusive XXXX and World February. well Symposium and year our metabolism status, renal years genetics to the as medical work Long-term was Galafold team on Second, in schedule. and of data half eight during up a published males. gender

also looking events, studies presented and kidney, publication of similar believe by recently and conferences, at favorably literature. we outcomes have We measured the cardiac in a which rates at CNS Fabry compare submitted medical to

leverage and publications, add to body over Amicus as Third, towards global post marketing in of marketing part for ways exploring a this data registry wealth XXX evidence evidence admitted post is patients long-term providing well large growing commitments, of as through of as to to to our remaining Galafold the confirmatory support our requirements.

planned of for to multiple IP Additionally, on to to generation continue see an book we X studies this ongoing coverage we and mentioned, we XX of Bradley and We And now add patents. work long-term and have have success. forward. important evidence other move orange as listed Galafold continued Phase as continue driver all our last the

and health initial by and disease is challenges studies to the measures disease a our and decline of range natural Pompe many one lysosomal having disorders. most to by highlight to choices continues a program. of Pompe of people crucial. followed patients. for to treatment Moving for therapeutic continue is history important publications benefit neuromuscular on Slide disease of and XX long-term on severe disease being fatal AT-GAA continued recognize pose prevalent and the key that the It's affected Multiple

study. of summary we the XX, secondary Phase primary Slide endpoints present a on key from our Here X and

most alfa. out slide XX Generally, experienced patients of across seven reported and PROPEL function, ERT a Pompe a the more illustrates motor needs. stabilization saw never Endpoints alglucosidase six-minute in the patient date the shown over this patients. and with showed therapy we on We six-minute overall unmet outcomes for in most And disease recognized fact Slide if years average. and standard pulmonary As for the where a the manifestations only which AT-GAA this increase included PROPEL ERT disease these in naive both on was one of controlled of represent of this is phase associated outcome and the clinically biomarkers, their consistency walk approved population. with population, than muscle before distance and we alfa potential the strength, population. actually function, in experienced decline assessing ERT nominal trial the XX, the including both a the in against patients AT-GAA seen reminder, meaningful in to which alglucosidase AT-GAA call patients. endpoints versus here endpoints the FVC key two of care study of patients achieved On an Pompe, a and across were walk greatest favored alfa believe that's in experienced participants set for efficacy FVC, impact patients and is treatment randomized of in statistical safety a double-blind, alglucosidase superiority on half

to from overall behind Slide recently the AT-GAA, the X/X at the AT-GAA. MDA Clinical the months presented Phase data body long-term ago, on team Moving Amicus positive a Conference XXXX, XX, of few study Scientific adding and to of data

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

highlighted program. updates Moving the Slide to AT-GAA on key the on XX, we've

cipaglucosidase two July May the miglustat and for year, FDA mentioned John of for and the the NDA last accepted a the BLA XXXX NDA progress, components XXXX for the XX, U.S. set review as AT-GAA the XX, for date for the on agency PDUFA of and the alfa First, action regulatory BLA.

We indicated expect mentioned, manufacturing opinion on by the together the shared John ability prior been European that Agency consider FDA to to full alternatives The XX we're inspection. has Of at for these required the be with We've will is submitted inspection under on again, at onsite EMA the the facility conduct this WuXi for FDA MEA of again action review. facility. is the CHMP dependent has inspections later inspection the filings support to approved July date, an this a And an also not working that [ph]. note writing expected Medicines based year. to manufacturing for and as is WuXi in the MEA AT-GAA also

of for mechanisms, the We in late the EAMS onset opinion the place requested Germany, and which since have the with access leading requests up we ongoing expanded Early look onset receiving And access treatment, that positive XXX Pompe and children XX studies, disease, ATG access for report study the pleased Scheme open by more other all be access younger are Bradley the as to with including prior recognizes two-component that scientific for has preparations living the to to granted for patients permits across expanded was years the to onset for enthusiasm across continue clinical this in AT-GAA in received to that, momentum multiple the countries. the in soon novel We're and eligible grown in programs. IOPD, infantile and to excited U.K.’s back children And be with least all framework Pompe marketing and both our worldwide patients than the as alglucosidase expanded response Pompe switch very late to we Pompe age, well And seeing medical now opinion at treated launch two community, significant our who provide now access Medicine And community programs individuals for interest Pompe to in expand multiple centers to are high who the into to with adults Pompe expanded granting we UK, AT-GAA a multiple eligible. Pompe to have for let living to call those over and Japan under to Access for to multiple continue continue positive enroll appropriate. possible. as late physicians me finally, globally, AT-GAA further and UK. patients AT-GAA. these having includes patients in hand to also for are that to disease and infantile-onset MHRA. add positive with discuss through received alfa programs the with opinion to years unmet those This Brad? access living scientific This continue faced our June many by with we need we with access able the in expansion label announced AT-GAA UK,

Bradley Campbell

Sorry about that. Thanks, Jeff.

supportive as building we XX launch Slide AT-GAA we the of launch were have that launched as another of organization that presence team Jeff a major through [ph] over the we scratch, unlike for XX countries Galafold on Just for be from FTEs and we infrastructure although Galafold with handful including needed. all when we successful poised small around AT-GAA a hiring same will product talk largely and are franchise, only preparations when will our on mentioned, world, our new commercial launch the markets, in the now success

and that chain, have reimbursement all for drug commercial, with across needed are We access. experience experience payers, successful areas supply launch, regulatory,

call now relationships Phase and data the Lancet with our importantly, very most can the the relationships leverage AT-GAA the our regarded and second very medical in Neurology, we're successful believe disease and and to Quimi, their focus let the published strong X with people experience doing our on a Daphne the commercial our and world partners highly and planning From and We're Officer over in the physicians the addition, deliver In strategic to class to with with that, living again, for Pompe Biologics, team, around guidance experience Amicus. world. around review financial the me Financial education, our in access, that that medical launch organizations and the WuXi world. Chief all turn key with in with outlook. community together confident we're inventory for we results, position and rapid Daphne? reimbursement we building now And

Daphne Quimi

Thank everyone. you, Bradley and good morning,

on an XX our performance with and QX impact. revenue Slide overview begins overview of FX financial Our

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

As $XX.X significant negative for quarter operating expenses were the the year $XX.X operating Bradley the $XXX on highlighted, given first support portfolio, per statement period. AT-GAA in marketing share, Applying XX% be quarter of contingent as Total expense. The the XXXX. Slide of a in whole was impact XX.X% $X.XX expenses the Cost reflected of compensation as our depreciation. XX SG&A assets, Net the and development numbers. of full income the the net our sales of prior prior numbers expenses increase goods revenue the million compared as for to impairment in impact as April the see total year exchange the operating and our share was in related based were XX. $X.XX year. the to first consideration compensation to of outlines million first quarter Galafold compared the per X% costs of and the and XX Galafold on revenue U.S. of loss ending stock research XXXX. a in and first global for loss manufacturing $XXX.X increased of of of as period. expense, or value for percentage is to compared changes the sold excluding reported quarter therapy average On reported share or expense million of approximately million for launch first to a a generated loss non-GAAP We first GAAP year non-GAAP reported we XX.X% XX, XXXX define to XXXX would FX expenses as March exposure million to outside gene quarter, reprioritization million net quarter sales FX $XXX.X fair in rates, compared $XX.X to basis, year XXXX

This approximately the within clinical March continued $XXX global million AT-GAA of million million XXXX, the Galafold non-GAAP launch, of and in range expect outstanding. $XXX expense studies we total As XX, and driven year by prelaunch $XXX we operating to had investment activities. shares be to

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

non-GAAP levels beyond, to XXXX and operating Importantly, in we in similar would expense XXXX. expect decline to a as level

strength profitability our XXXX release. as Turning sustainable now continue from a self we we financial to press to remain defined need to XX, in track in be on operate Slide without for achieve position diluted the to of and any future expect financing and

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

the John impact, any revenue $XXX currency back operating And full over with to addition to Our let guidance me million. call our year closing guidance turn remarks. to million before of to $XXX $XXX for expense $XXX million that, is in million non-GAAP Galafold

John Crowley

you, Daphne, as Bradley Jeff Great, well. thank and and

to of by entrepreneurs, So we with as our have can continue our And business see, to happy team patient have you and led the will we're global been mission. passionate performance us relentlessly operator, take on people questions. on that, across driven focused focused who lead

Operator

you. Thank Thank Instructions] you. [Operator

Your Ritu first Baral Cowen. question comes from from

open. now line Your is

Ritu Baral

Thanks that labeling on WuXi you can the And including having to the us tenor for discussions that on Can clarity you're what's Has breadth focus label? there's the I prior of since a have little everyone. those So only discussions you inspection? of then, AT-GAA inspections? Thanks. the more give question. manufacturing, for discussions mentioned already started, as taking around to you morning, Good those guess, on? John, talk the been there that? overall, guess, negotiable elements, around I tenor,

John Crowley

Ritu, Sure you. thank good morning,

continued making obviously of success with take the good to to support AT-GAA again, very those a take talk commercially. negotiations switch With as exactly extraordinary and the going confident of But the support FDA launch with the moving negotiations. that about, the will the very Pompe living and would important spirited to and those. and it's with believe FDA. product labeling data patient's and instance, care for I'm of with really we we're that discussions. good of that also we're will again, we tenor Obviously, I'll both tone interests, FDA. And we we've on in population provability what the sign the not patients that people a think be feel the continue disease provide for label, and discussions sure a we negotiations, viable. we're and will through the about that had in it label for other with to So parts with matters, label discussing access so practice we that commercially about that the respect And that very very the things highlight the good we

went in completed have that facilitate going completed of China. do facility of We've that inspection to internally, to the all manufacturing, our indicated Biologics in that City to know, they're the in WuXi with has very with an respect with necessary inspection. all for auditors mock to FDA preparatory work well. onsite March instance, WuXi the You WuXi a We've inspection along

We timeframe. inspection. And look with achieved they expect to the which may FDA ways that would around XX, now at July be be in PDUFA in continue that the all continue to conduct the date this other to options would right of we able something be action

Ritu Baral

Great, thanks.

John Crowley

thank Ritu, you. Sure

Operator

Your from JPMorgan. of question next Anupam comes Rama

question. state You your may now

Anupam Rama

WuXi bit May on the thanks be so Thanks for in question about the a are expand review talked previously about FDA other you kind guys, of up that on of being region maybe question. here much Hey, little so You've following strategies the taking what in review, facility much. FDA China, could you're my here? employed the the helping employees strategies exploring? conduct Ritu's

John Crowley

I discussions won't any on ongoing thanks. FDA. the Sure, with comment

China. has of You're in designated WuXi WuXi about under upon Shanghai City not lockdown is of facility everybody, or FDA have is three a does correct lot complexity the Shanghai. with here hours on to north Again, remind FDA four employees that currently itself. do the The to

that they've all action full date. which it be So evaluating parallel, inspection That's that we in In the conducted be completed indicated remote with will to what could onsite PDUFA by FDA. and other sponsors, options, expect are and that need they've by July we expect well. XX, ways done the we also inspection a us interactive to could ways the other which we in as options continue other

So we're fully prepared for all contingencies. potential

Anupam Rama

question. Thanks for the taking much so

John Crowley

of No, you. thank Anupam, course

Operator

Our Ellie comes UBS. Merle from next question from

Your line is open.

Ellie Merle

color U.S., sort Neo switch if potentially terms and first, well thoughts Pompe patients of thinking EAP as the uptake Hey, physician your Maybe from you in guys, just color for how preparations, latest of you maybe on in going ERT patients uptake GAA in switches maybe Thanks. the expectations GAA as color give could started on that then and any you're there? maybe UK, thinking Neo on initial if just of for and the already launch and in taking question. have of uptake maybe feedback in about just terms how thanks is the the there. give any And could the sort towards

John Crowley

both Sure. launch Thank well expectations good morning. those in Bradley, as of want Ellie, handle United our as the EAP you States? on the to you do

Bradley Campbell

questions. Sure. the Thanks, for Thanks Ellie John.

the all that very Neo to AT-GAA the what positive that access we've prescribe that's number Early a medication and expanded of available the extended or scheme. the AT-GAA probably Access for the provide for know UK. one to is AT-GAA. receiving the because both that mechanisms, feedback in GAA most visible most first key And centers access access, at now EAMS, through yes is important which asked Scheme Pompe of We are this continued through Medicine heard point, their have one and sorry are Medicine So we've important EAMS towards And program. through EAMS is

program. to actually see UK number that and much that think, how is is for We would really that's, through So Pompe AT-GAA I it's to a excitement support the pretty climb market. and continue expect important significant there to

And think patients And so, the AT-GAA switch AT-GAA have the well, around Pompe. other sees is noted along, we we've again, while a UK opportunity In how obviously States, around into world as treatment gives the to unmet and uptake potential we opportunities strong other application, your for mechanisms the as great see for as a we for is to access think where the great that's get to reviewing the need. we us community sure a view countries the medical in population, all for United uptake. clearest point

Phase Pompe AT-GAA. who the within that I falls care experienced We experienced John patients with We And indication gone to patients. could what Phase the chance any and standard I patients improvements, from we with the of see those that populations. the would AT-GAA. perhaps talked on and have both AT-GAA in the for data would as product perspective, and negotiate that think would data significant see we're our focus encourage to alluded studies patient publicly the data utility Myozyme on continuing think our folks the be we patients, Neo data the in in GAA, very segments, switch on to are But label how re-familiarize have particular that over for over are confident and to from -- think then to the two we and GAA who and But the longer-term has Neo for be the to time. X from available those of we be all show who you believe just themselves about you'll patients indicated perform Pompe are, time think, there if the X/X, have

Ellie Merle

Great, thanks.

Operator

Securities. from Joe comes Your question from SVB Schwartz next

is open. now Your line

BethFeindt-Scott

wondering Beth This off you Conference building I Hi, share data you is the on that, everyone. year? you just Feindt-Scott if on believe XX feedback MDA could for any And also received months this Joe. presented I physician the at PROPEL that you PROPEL this data you'll from open decision? you data year. the ahead extension from if approval be regulators trial plan the later wondering label with We able label were to share said have just to of open extension was Thanks.

John Crowley

you label shared Great, do on any as World take Study as the thanks Beth we on the that Jeff, morning. those color of good data? want data feedback extension and well and physician PROPEL open at the XX-month to both

Jeffrey Castelli

Sure, yes.

one unmet Pompe effects. treatment know, the of is biggest You durability needs in of really

effect. naive some in patients seems generally long-term I think in that was treatments And really was patients, saw that's off I study, with to years, something multiple about longer-term. for patients decrease initial now we And exciting but after seen data that tend lot patients. in some weaning Phase think benefit, think that those whatever X/X switch maintained. was reason, potential have now three a these we we've did they X/X show Phase this that's data I And every been just that presented we see what show again, longer-term, mixed to that then just the improvements,

to data. that data yes, reception in And that by X/X very following now got this well. excited are we treatment. -- that have were that We we've plus now as we been continue to of good five we year, Phase then will So patients years

in those year. have cuts in are can don't Lumizyme in continuing specific cuts Phase we have we'll after on conferences arm, one X/X include obligations at our the or on have PROPEL that have later the to necessarily yet, ongoing you data year AT-GAA, and of and EU. we data we'll So that even that addition, that this We to from The the data plan switch But have AT-GAA don't U.S. year data dates have extension. data to of we'll patients one population least announce that we submissions or patients. but do disclosed know, here

initial some for data. we don't longer-term may know, at review to need data, add point approval, add additional but the that You and we

John Crowley

Jeff. thanks, Thank Beth. you, Great,

Operator

next comes Kristen from Cantor Your Kluska from Fitzgerald. question

open. line is Your now

Kristen Kluska

on ask and past, you've around example, on need EAMS also market? presented heading taking all then the published everybody. morning, this PROPEL place the for SEC into AT-GAA publications on and the good question. could my different outside around I next work to endpoints, so example, know both to the So emphasized Thanks but Hi, since the else the understanding the role additional for [indiscernible] really in the that unmet I readout, confidence AT-GAA findings the in you you gives that mortality wanted the some the decision, commentary labels newer also what agency for risk? around in then of of and

John Crowley

the into Again, of morning number different patients Kristen, Study and just from Great, you, study Lysosomal our good Disease. largest differentiated the on going to had AT-GAA. of AT-GAA highlight who number people a study people easier see job who was XX% years, again, are taking the for be that this that was had of the And enzyme any who myozyme-lumizyme, we Neo making care standard for would from the switched majority medicine elements by in currently this vast thank any those first they a will to that highlighting made Whether to of people. of are be lumizyme we And the may I'll we therapy. actually that. a think in hope support be and label replacement nearly or the expect or PROPEL the that switching switched for to of ever conducted go Storage myozyme already product,

secondary walk, This forced vital community the virtually that be statistical very sophisticated showing population, will again in and large patient again, data is physician nominal that they the So a community very six-minute on significance patient that on driven. and in data study, and endpoint. all we scientist capacity, have particularly switch

durability data patients So ahead the at Jeff just data in the will the look and the our months that to hope, and with long-term that the Phase from data see the that look long-term that the we study at again data we show durability PROPEL Study. to the in X/X talked from

through way we all the now So experience ranging patients study, physicians, the studies in standard we from then the with in real of full point all from care would and AT-GAA we infants to think approved Bradley's to together myozyme of patients adults switch United all clinical look enzyme, Kingdom in with of the expect, XXXX months from approval. to uses world the multiple EAMS to data of the again the coming significant the into existing as over numbers from a

So extremely of and doctors think all with community we physicians, benefit profile totality positive overwhelmingly, are risk that's as we're the excited We strong the know engaged builds I presents think AT-GAA a physicians. of and for option very for AT-GAA. patient that very, for potential appropriately

don't got to add. if know you've comments any I other Jeff, So

Jeffrey Castelli

fact well, and manifestations, endpoints that that consistency, the with measured No, treated, alglucosidase we that then all I covered alfa parameters different patients patients over the adds and seen across of nominally, from we it the just really across think it's either higher the severities, significantly consistency we've AT-GAA patients favored I patients, key naïve in And numerically or experienced non-ambulatory that just cross that that baseline really Phase the into the X/X, treatment. lower John think the consistent you meaningfulness key a patients, weight really effect. that's or

John Crowley

summarize for And ability many of add increased many Kristen broadly to least look us I'll with decade. lives over had and that by a of probably affect durability are finding at the we the bode in billion the with that of the the to the particularly end the and market market living sales that the global all hope, more more patients enzyme and the had than billion. this of to again will diagnosis when you in for others is revenue. we've and $X we and revenue in and And this with $X across new think that we global Pompe again, rates and affect think a with potential more clinical six people data for potential market. for it of this you've look an product the important it's years well. half last impressive, we now of Now, And We than consistency positively magnitude, point, replacement where at year, and Jeff very data, one think is research, have

will of where our are and when intellectual of with with you look where length dollar is of product split into it with Galafold, to at step field, in the Composition approved the other is $X or the this protection, that's well take become least back and the property competitor AT-GAA, entrenched the that be Amicus significant an with property revenue combined distinguished, we pretty we has think disease in at revenue That for extending particularly success, standard care, a the billion what that believe with market another the potential intellectual also then the with So financial the well existing product, billion Pompe we potential XXXXs, protection the Galafold potential we'll today cornerstone think including into of strength market. in XXXXs. on Matter of we volatile company, with

So thank you.

Kristen Kluska

Thank John. you,

John Crowley

Kristen. Thank you,

Operator

Gon Dae comes Ha from of Stifel. Our question next

You may question. now your state

Dae Gon Ha

Hey, good for our taking question. morning, guys. Thanks

of I seen On some whether you Thank data were we've profiles asked Neo from that differentiating FDA comment that terms for perhaps to? kind happen you you could in the of any was very can just then the recently AT-GAA, cuts discussion, wondering has that alluding on some GAA, of of additional if accentuate curious, labeling much.

John Crowley

Yes, not they've for thanks cuts. additional Dae any Gon, asked data

data. with We have the them all supplied

for the for they've instance, any the data asked the seen study, PROPEL but they've from So XX-month instance, not cuts. data additional for Phase for X/X Study

Dae Gon Ha

you. Thank

John Crowley

Thank you.

Operator

Goldman Your Sachs. comes Richter of next from question Salveen

line Your now open. is

Unidentified Analyst

expect that Elizabeth WuXi for you review the place what then we expect you. of should thanks and around initial message guys, the And to you date? expect the communicate remind FDA to when could we maybe Thank us Street? first learn guys to the takes just from do Salveen. is questions, you to inspection should question. on for the to going taking Maybe This some back how Hey, of

John Crowley

on WuXi to inspections. the of going going discussion, both or important preparations. has section of section with review activities of And at questions WuXi from that Sure, and let be the now labeling quite inspection. of expect to and note in all expect ways the course, different all has to perfectly But we've CMC look, We're lot early by do September Street the no respect action agency, We're part, we've BLA, clear, back going we have our important the on this May from me provide the week. a at to had CMC with the action FDA's And in well. we've to providing to we July expected, think the literally blow, there's parts, course we fully agency. be be part respect which launch We the agency. focus way the be right satisfied. agency on now manufacturing the dozens we satisfied take With requests of point We're history we discussion tremendous this into lot BLA and and a approval. believe the CMC the of that our important again to year. we've blow of XX, we're which addition, the expect by -- internal on Again, going XX, dozens successful we and been bodes updates with, and information the could all we much not our of this this been to that with manufacturing exchanges having with not the I mean, NDA daily final week including we here had that the first hope very think of with to discussions

did go? going When to stretches, take the How provide date. that not know, color. You July We're to We're it coming it did up final place? is PDUFA ask? did into XX, What it they when scheduled? now the

XXXX. fully in are in with and States. EMA we're the and the again, MAA the in launch date. of So continue we and we the if United again, to And active obviously, be there And before discussions Street. very, prepared of in confident provide changes the to But PDUFA the opinion remain very the any that the CHMP by would expect update launch to that AT-GAA end are approved approvability for the to expect medicine with Europe in year the

Unidentified Analyst

Great, for color. the thanks

John Crowley

Elizabeth. Great, thank you,

Your comes BTIG. of next Zhong Yun question from

is open. now line Your

Yun Zhong

given and the Europe? very the size good for the remind not much of Galafold, AT-GAA to Can increase taking like you do morning. Thanks the the you it's that related oral face launch? in COVID for or Hi, the infusion that do have will some challenges the you also an questions. AT-GAA? in U.S. us size still Did preparation And have when for you you you force launch plan still to IV you sales of type in that expect

John Crowley

operating sure. Yes, to you take Bradley, that go highlight the leverage to at over Amicus. I'll have ahead and particularly we that

Bradley Campbell

Yes, thanks, John.

as about That field use characterized market have XXX, access, to et is we've between personnel we a we the launch cetera. effectively reminder, just organization sales, is So organization medical, global AT-GAA. what will

direct than marketing, direct perhaps the globally and hub. in leverage organization, and market significant would Patient get in dozen to FTEs organization, in support then we to we What of access medical is, medical commercial So Galafold should some I our the States, of Assistance in particular said folks out we've primarily commercial say. intend add support launch, the less a United

So of highly leverageable a the launch organization support to be to able AT-GAA.

AT-GAA based based of to COVID impact terms uptake. on we In global impacting evolving, markets AT-GAA, the expect on as what any significant point, where don't relates from your this we second of we at the question, see Galafold, uptake know on

where But place trends a give course, forward monitor is our by don't this more able continue good see us into AT-GAA impact we that largely to field perhaps we're on at in a hybrid patient we'll think I perhaps launch. seeing the to move our physicians. But now moving slightly between evolved care we're COVID interactions that. Of way, point, force and confidence significant

Yun Zhong

you. thank Great,

Operator

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

John Crowley

great Great, thank for for everybody, you, operator. Thank the you, questions. listening,

these priorities As AT-GAA, on our strategic you company. strength the sharply the for can focused and pipeline see, of we're financial for all Galafold,

have great So you, we press day. continue to on Thank will a onward.

Operator

call. concludes day. you have Thank great conference today's and a This

Amicus Therapeutics (FOLD) 9 May 222022 Q1 Earnings call transcript