FOLD - Earnings call 3 2023 | Amicus Therapeutics, Inc.

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Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Third Quarter 2023 Financial Results Conference Call and Webcast. At this time, all participants are in listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time.

As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. of President Vice Faughnan, Investor Relations. Andrew

begin. may You

Andrew Faughnan

Campbell, conference Good Chief Officer. Amicus discuss Corporate Chief Leading have Business our Sebastien Harford, Executive Financial and morning. you for Highlights. Dr. Bradley we call, Financial Thank Officer; call today's and Therapeutics' Martel, Development and Chief Officer; to Results joining President Chief Third Officer; Simon XXXX Quarter Castelli, Jeff

U.S. Officer; Mike Q&A for is of Business Joining Keavany, Rosenberg, and Legal Officer; Goldman, of Notal Dr. Head Chief Ellen Chief President our Commercial and Marketing. Mitchell Global

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

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

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

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

Bradley Campbell

third what business our for I everyone, to Andrew, tremendous continued global quarter a Thank very pleased been you, the welcome, quarter highlight through am call. has XXXX our Great. to and Amicus. third conference across progress

this to me highlight I team detail. release, As several give we the turn press key before more over it to did in points let morning's

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

XX%, pleased Galafold's commercial achieved exchange of Galafold on a for constant rates performance in And basis, the very the are constant as quarterly uptake a has was year-to-date in globally, year last the revenue. third growth at which XX% numbers. We $XXX basis. with Galafold represents both time, million well over quarter as first from growth currency revenue on reported

strong Galafold's in our includes continue XX% increasing year to demand on was populations patient starts in Fabry observe our in in-person a of third We the continued of all treaters from indicators visits switch This across key and field same to team strong period demand and through geographies the markets, all between steady performance the leading trends and growth naive new key of both throughout quarter. full constant in over new adherence by months, compliance patient over quarter year at first XX% again Growth sustained exchange to we third guidance the the growth X net starts. performance patient rates XX%. driven rates. our XXXX are from last and revenue patient primarily Based our

launched late Second, Pompe now in markets. novel X onset disease for therapy Pombiliti Pompe is Opfolda and largest and the are approved

which in U.S. the and largest again now Opfolda the FDA Pombiliti approval is U.S., X Germany, in recent commercial of September, the the and Pompe are launch markets underway globally. Following well U.K. the

patients the of transitioning patients by that progress access Europe, Sebastian treatment major We're of clinical the reimbursed of And their converting commercial product clinical drug days already commercial in new coming in both to well is patients importantly, to studies We're ahead as see as well. track are scheduled patients of additional commercial early trial geographies. to on launch been our November, who pleased share more moment, on cover more than also additionally, to towards switching We're in have team these goal treatments. our As XX X ERTs, treated expanded from track a we're to very to and supply from naive of seeing expectations. our supply and will being in our pleased patients markets start the detail multiple all commercial available to tremendous programs in in individuals commercial and year-end. are early making with

that, on dozen as markets submissions patient countries. country-by-country expanding into regulatory focus submitted over reimbursement additional outside on the of the continue the dossiers Beyond well. in broader as this to we and we navigate the European by By a Europe of access year, end have reimbursement will we'll focus major process

expectations launch the Pombiliti With to Pompe and with disease. living therapeutic look to of global providing for choice physicians both we and challenging forward a those real Opfolda,

Finally, we've Galafold position financial as of launch Pombiliti Opfolda. execute advanced global on and expansion a the continued maintain we of to the strong and

this underway, towards recent strategic favorable X, strong Galafold, we for profitability Ahead key from continued our achieving track With growth in great achieve are non-GAAP on priorities our to also well XXXX. now Slide of well and the refinancing progress terms the we're fourth making the launch year. on more debt quarter under

continuing for First sustain and around growth Galafold the world. double-digit to foremost,

next-generation We've Business and On rates. our performance investments those XX% as chaperone carefully always, our a medicines executing manage position disease. financial to that, the capabilities successful rate strong profitability. advancement now and expected Sebastian? Pompe Opfolda as and Officer, as And in further now to of key over approvals performance. our EMA grow genetic the growth me let to the to are we commercial FDA, to expenses Fabry for Galafold as now the Pombiliti next-generation back XX% highlight in for we non-GAAP by the our and now Chief secured well to judiciously and exchange top and annual year-to-date, best-in-class With line constant the maintaining call on of Fabry strong our we NHRA our Martel, of markets. continue path regulatory hand at We've Sebastien and invest our raised have continued to are launches

Sebastien Martel

Thank by and more I'll providing you morning the to Galafold you, Bradley, everyone on start for with on quarter. call. our detail good performance the

particularly to reached you for on patient Galafold third our revenue the As see Slide driven strong can revenue. reported markets. Galafold million X, by growth be quarter demand, in leading XXXX, from $XXX.X continues

Turning X. to Slide

of to same XXXX X-month highlights a in operational year-over-year commercial there's at perspective, exchange efforts. of rates. strong over period with global The globally From for currencies. Our strength all XX% added constant results the year-to-date impact from in foreign Galafold the markets, delivering continues growth be patients our negligible demand a major

revenue Galafold growth for XX% Fabry disease globally, at period quarter this versus constant the result, with a year be also last XX%. same rates. was Galafold continues As reported growth treatment exchange fastest-growing the to

gross In starts previous net the have X year patient fact, both and this new years. been higher than

we're leading onto a X patients Our U.K., demand. EU biggest number countries remain continued amenable We and global the continued such months in Throughout quarter XXXX, globally environment. Galafold global the internal demand seeing the And the coming ended expectations, Japan about to mix, stronger of X post-COVID the XX% which to naive Galafold more exceed growing patients. and of has first of with you the within help market drivers as populations. a U.S., markets third indicates the normal uptake treated of patient for of return share

plenty the opportunity So the to been and we among we've we grow penetrate of longest. And and there's high market it approved as for a market people shares still to continue into to switch continue in Dinos treat treatment. continue to achieve the to country where patients and naive to

that to that compliance exceed those on XX%, underpinned of adherence on who Galafold. Galafold and by predominantly All patients reiterating belief continue that rates stay go is impressive our

of amenable the and drivers: sustained naive the X factors, on continue We segment the past FX in to uptake XXXX. several population, market is mutations and As know into patterns within XXXX by uneven into reach to potential with the typically years. annual revenue markets, Slide to penetrate and has Fabry of including of the that throughout near, driven and population patients first, XXXX there's anticipate demand a Galafold billion rate of we On calls, X, fluctuations, significant continuing growth expect for dinosantreated key to of year that global to $X be into geographies and the ordering we growth further new due the end can on made mentioned a growth in into label around not existing variety extensions. expanding

continue increasing markets rates, just and of we the positive As all compliance extending solid Galafold labels. and into share dinosis that to to new are expanding we around make I'm efforts reimbursement supported the pleased by these adherence and progress on globally. access mechanisms mentioned, and world

in as first-line recently naive Just in also to examples. few treatment Taiwan named a reimbursement We've Galafold a patient received amenable Lithuania for and basis. received patients. name reimbursement eligible on We've positive for

the gives XX Additionally, the to And with by time the new enforce intellectual term, runway And This next in Turkey growth long XXXX, to to Fabry confident Galafold exclusivity and of into engaged Singapore. Galafold globally, in patients driven strong access Orange-Book-listed with and pricing growth of have We we reimbursement matter intend composition to continue and Hong a which screening expect negotiations diagnosing XX in Zealand ahead, we other in in including for decade. to as we've New and note for year our come. patents received through to see important our protect steady patents longer us we broad double-digit globally approval to variety a into into we and speak remain the for provides and including coverage through property here, significant late support. the in in that and our newborn to of market high-risk IP a as XXXXs. we now this Also training, initiatives, are Kong, the well addition looking long IP Galafold and we In measures, provide rights. continue diagnostic opportunity to Europe. protection, Galafold us often end U.S. that the continue

verge X to patients The parallel, from Opfolda ensure they starting in the overlap transition transition regulatory for program point supply of need to profitability. in access and and turn all significant and We're the complete our respective first to patients. On each Pombiliti and expanded benefit EU, now leverages with Opfolda, world-class information U.K. and country. clinical turning has switching infrastructure organizations. would in Opfolda right being our progress the outline highly launch medical disease of led multiple Pombiliti and treatment patients access process market X to and expanded non-GAAP between trials us launch This identify centers, for biotech which are geographies after experienced commercial in approved physicians and and achieving business we teams patients cross-functional Those territory to and by U.S. on Fabry really Let's have placing X, and Opfold to in a support and will switching a global launching to have Pompe be quickly trial these The allowed with the Pombiliti on commercial or Pombiliti physicians the products Slide optimize XX. excited clinical companies seamless our of us by that those approval treating incredibly year-end. Amicus, the in rare on and Slide of started approvals hospitals, the a who is to

and Within core centers business other had treating and as of focus. and very have approach, feedback patient approval, to been engaged we've first stakeholders our XX our with, HCPs support from the all our days positive

as November, their multiple patients commercial treated early Brad are and additional more patients scheduled XX of treatment. to As than products being are said, had with start

approved both other we've also switch and Pombiliti to Opfolda. over therapies, patients to from started Additionally, see of the

patients, regarding we on physicians Pombiliti have XXXX, poised on X-component from We're and full revenue to and physicians Opfolda significant engagement million partnership have team. to pleased interactions with expect commercial Opfolda that with the and a the expeditions. We is Pombiliti with in working experts, and with we're with Amicus Based for ensure their positive field early Pompe all to the believe which need light experience for patient their around therapy the they our in opportunity. feedback consensus $XX they information is year come in right

our Finally, find progress within days, track access reimbursement. metric these is and important we an early to within

year, Austria. the are team We're discussions U.S., European have also access patient engaging and U.K., We we we're a payers a to highly throughout end commercial the markets, all of value now the color XX, to the By on the and Slide I'm broad I'm and Opfolda EU. Today, we'll in the progress translations country. focus in experienced pleased as countries. transitioned of pleased additional launch over that with Germany, pricing reimbursement as to in in Germany, in On have dozen first, securing conversations this even by been positive who launching Pombiliti access reimbursement of additional demonstrate active you and clinical with expanded know, Opfolda. major submitted with on Pombiliti in provide have to report Maybe product.

we're Pombiliti to a Additionally, switches the switch very U.K., to been Opfolda whose attentiveness to in-patients and from other ensuring HCPs lose in We've commercial in positive has individuals naive as feedback well other conversion the are team addition from supply treatment. starting their therapies. also pharmacies a Amicus and with engagement transition now In quick therapies to from very of straightforward as critical received on seeing process.

As of XXX% these fact, occurring with patients a of these matter conversions yesterday. schedule are of as ahead of now converted

In to launch facilitate help to activities engage And as a all are process. prompt addition, field the in centers quick U.S., been and first in our underway, the HCPs treating progressing with U.K. all planned. with conversions has conversion team

track, patients In with submitted clinical of by clinical to patients their PRF on have trial year-end. they're trials and physicians, fact, on and XX% already we're transition track in all

headway Importantly, prescribers launched has days with readiness our and thanks of and positive team, field to programs both approval the our payers. team first the few multichannel made the within of launch

will our Amicus all strength through and We great And and So over great call in depth Castelli, with make effectiveness across people The the believe to lines. the our very at the disease. first and pleased us to Jeff Officer, living talent of difference a data time launches studies of countries. ongoing a summary, hand clinical EAP clinical in of to is that, Opfolda we're the real for to in with a position Pompe I in-depth launch. Jeff? the our Amicus with successful gives second wave programs we of confidence Pombiliti of Development therapy Chief the discuss regulatory have ability experience

Jeffrey Castelli

for our access on execute Sebastien, you, as everyone. On and XX, to good submissions. clinical the Opfolda Pombiliti studies Thank build morning, Slide regulatory clinical we folks that remind through continue we and of ongoing evidence commercial through we body expanding

process launch, As we addition in that of submitting. enter the noted, reimbursement we the second doses to just various phase Sebastian of are in the

for geographies also planned We submissions approval in XXXX. or have multiple new throughout regulatory ongoing marketing

community, impact differentiated patient we studies for and generating And younger Amicus the and through and Pompe clinical action open-label open-label continue expect registry, for endpoints the Gower's children disease onset ZIP enroll long-term on ongoing evidence Importantly, the the to Pompe infantile and across ongoing populations. of living of with mechanism the Opfolda children Pombiliti with living Pompe ongoing and late onset for Pompe study we to the disease. continue study

XX with having trials quite participated clinical Pombiliti globally. experience Opfolda broad is Importantly, centers, programs access approximately in with in various and

pipeline review to With Financial Harford, we call Finally, transgene and Fabry Pompe, on for to appendix as to therapy including Simon now guidance our and our to highlighted slide Chief in chaperone. that, results, for in our to our Fabry Officer, continue pipeline, GAA next-generation deliver the and focus the Simon? like over engineered earlier novel outlook. I and turn the GLA approaches the would financial stage gene

Simon Nicolas Harford

you, Jeff. Thank

quarter XXXX. begins for financial XX income ending Slide the overview statement XX, our September Our third on with

of increase revenue a during total XX%. the million X%. compensation XXXX expense, in fourth for At or an net million grew of within period. net growth period of a expenses capital compared year Cost and revenue of for third to United constant for contingent loss in Total share XXXX, year to management, of share over the to revenue breakdown the We XX.X% of our $X.XX non-GAAP on from which geographic to as The percentage make expenses, Cash, revenue the to profitability of of of to million per sold a compared quarter the in generated remaining same of of the compared rates, we amortization. stock-based million or million excluding GAAP expense at million, to consisted $XXX.X changes were for in Net quarter, total million third Galafold expenses XXXX of third as define operating path at marketable XXXX exchange increase the the progress and For $XX or increase consideration global million $XX and XXXX. loss or expense quarter $XX cash as XX% the revenue X%. XXXX. outside the XX% non-GAAP quarter depreciation of and $XXX the coming goods prior the fair towards basis, of year. $XX to $XX was reduced impairment the compared equivalents million million and achieved as the in quarter continue as we States U.S. year XX, this is XX% value operating development $XXX research per year, increased December SG&A quarter $XXX and third and this of period. XX, operating to $XXX of assets, in an $X.XX increased Driven third loss prior third XXXX a X.X% million On the by for September securities third the quarter non-GAAP compared of quarter sales to as last $XX quarter total

share for revenue to have pleased Slide guidance to revised I year. now the am we Turning XX. our

of demand. driven strong constant XX% year increasing We guidance exchange at by our XX% growth patient to Galafold full are revenue rates,

$XXX terms guidance will at we to Bradley flows that October, the terms, agreement, comments. long have more in just anticipated back with plus favorable refinancing operating with year Blackstone $XXX better Amicus to for refinancing under of Amicus let borrowing continue secured million that, strategic debt facilitate In the to our the a remains for existing investment XXXX allows align loan closing our collaboration we senior revenue provide we pleased to to agreement million were full to announce Blackstone. done the $XX cash the term non-GAAP Our with And million. This million our and a of $XXX Under us term. million $XXX call in stock. grow common turn expense over me as

Bradley Campbell

the you, Jeff, thanks Thank can operator, for Simon, company, confident our rare employees deliver all around can need. I people questions. Amicus sustainable and Great. people now drive living we worked diseases. value who well with the Sebastian, phase to of a am And to big to with as Entering next in this as tirelessly the to of therapies long-term that, more continue life-changing call open world

Operator

of the Ahmad comes Our first question, from of Bank Tazeen of line America Securities.

Tazeen Ahmad

give you patients tail you to the cadence from build added the appropriate with at relationships convert going launch? the how we how seasonality your can those just be think we new to thinking are Should that about ERT? at term, about And least approved on got continue the continue proportion near should the you versus to that So a of color end out physicians? quarter. Brad, of us sort as of of you're little more Maybe expect bit this any we

Bradley Campbell

are with And think, right now the launch. we with where overall, as super pleased we we're said, I

any new starts I there, new is cadence questions of of there patient distribution think kind asked seasonality. conversion then versus you kind a and of of few patients,

first. the So I'll first one take

to, goal as really in and and the that the in having trial X/X call, and now both foremost, But we're on all then and and said patients highlighted great cadence, through think progress converted me, So significant, patients first in clinical of process patients. expanded we've patients, excuse the making prescriptions what the our working States. access converted there Germany, compassionate of Sebastian the year with I also U.K. patients the United majority use convert is is existing this the

seeing from both And ERTs those as pleased really say and seeing with this, each conversions. majority I new But would as switching from of talked I think we're is patients well. the year the patient existing Europe, this in in markets. of about what new patients will we're starts So we we're come well, already

those the well to as we're So we going through we're Once KPIs look that's do think of on from in new year, obviously, going. process, majority markets. Europe are really how and from other I switching patients starting then if with which therapies pleased that existing you next then at conversion get come and this track patients all to year,

So far. so really pleased with the progress

flow as year, as that the know progress just we be so similar it provide In pattern observe you more model on terms we've that kind of of based see year, progression. go with Galafold, of and lot I the but think, seen we'll wouldn't next if seasonality, a we as through I I a we nonlinear color the think we can surprised we kind out guys can, through

a be revenue the we going will year in next as clearly, particular, year. think Pombiliti next into from Opfolda, and rate the significant contribution maximize I run of

Operator

next Our Rama line the of comes JPMorgan. of question Anupam from

Anupam Rama

related submissions the you're In product. to for have the to multiple noted you regulatory in Pombiliti and XXXX expecting Opfolda slides, that

when here that the regions contributing are what about line? be they thinking top and should to we the could key So be

Bradley Campbell

that on I think there's going X are things here.

And then submissions. process regulatory is major the the geographies in is the playing that's other, the in reimbursement Europe. of out First of course, rest

we'll on of a call, dossier the the the more to end have alluded I submitted reimbursement than dozen by As year.

reimbursement do process could And on similar and think based towards you'll the see, So additional average out? pricing to and bit can should terms the might value think think next markets into revenue key you'll revenue faster year, we talk fast back you regulatory want the submissions start Jeff, little go of I probably half of And that direction I pace. you the kind year philosophy, in with proposition from I see for on. the when We next a getting I play more the of year. Galafold. than how about little as that tight a see out were to see through we play come really our give European think we bit

Jeffrey Castelli

the Europe I we for ongoing going others year. planned on, think specifically So that we're of other had markets working don't next major have we outside to we're U.K., submissions U.S., in list which markets, submissions country, reimbursement this negotiations. yet can what those the X dates in we'll at markets X you after expect similarly point. next have year other those But Tier that Tier to

So unlikely that those from the XXXX boost new submissions revenue year wave to submissions. would next revenue year second significantly next of contribute be a more

Operator

Our the Baral Ritu next question of comes of from line TD Cowen.

Ritu Baral

you I the to that work, converters how we'll the start those wanted long just encountered Are And I getting, the this PRF. may are? look you about you are the what doctors like, thinking give haven't something PRFs out no, of all split look And they XX% what think Nexviazyme the in doesn't Myozome are I Nexviazyme converters be forms really Brad, term received before a they're you before. we're that ask like, patients at do try curious filled forms about between commercial clarify else. those those handful you and transferred the versus that as that profile aren't like of how converters. patients have start that Slide XX of start Can forms? maybe

Mike Keavany

our trial from is XX% have clinical The PRF is of sent that prescription. percentage into positions. been our those referral it's the a the patient hub And our form, essentially

trial already approximately have in. clinical and XX% come XX have patients So of about we those

that those. forward to working have move out we're far reimbursement that doing So so We're the figuring authorization all fill like each physician benefits investigations is the prior with on well. what product. and commercial reimbursement the the moving form to to payer's looks physician And out for going

now. of and have therapy received process than to full more setting approval have therapy, in we're We who of begin up the patients a handful that

different As this you was be Galafold, every from an to X know, set up weeks. oral has which

and will last clinical commercial will when when be. to we their with product patient of with plan dose dose provider the the the out their of So first be have product trial and

So well. extremely that's going

far patients. been forward Myozyme. Lumizyme, We haven't move to half trial But And had Brad, forward if of terms half on payers that. issues next in really about trial our been patients this moving Brad, any of nonclinical clinical have our with you have want with

Bradley Campbell

the distribution. targeting As And from any switch patient think I patients. based label, great Mike said, is on ineligible a perspective

So we between around we're and and patients physicians the United therapy to they're what talking States. differentiate on really which regardless Pombiliti with in Opfolda don't of

on improvement and And things far, so as is to And about kinds talked the it's of said, before we've really that about were Mike half half. focusing that language.

in gives that of and months in So should in therapy, that patient consider patient Nexviazyme they'd already had might be in that just Opfolda, estimate, and And that considering long time we important the how who a they say a to a improving play roughly from and be They on seeing X look, not initially time need already existing about X switch on great Myozyme sources are if your X patients Pombiliti say, XX as to number consider say us terms physicians, consider you're lines, is think traction to interested data such they from X approval out. from if an they'd significant period. months you seeing XX-month there. differentiating What to switched aspect as patients. we to think early window that we we're their have switching kind of before of they

really tracking switch far we're to so the commercial begin the we're now metrics great process and all So look eager as well.

Ritu Baral

that reporting going would forward? Well, you of PRF Brad consider Mike, and number the something are

Bradley Campbell

remember. That's might Ritu, did something Yes. we with you Galafold,

a is think that process. where U.S. PRF through key And you I the that go metric so leading in

then you Once So think and get rhythm, We color patients. on did of more but looking important. so into focused for patients we'll initial in I really provide backwards. on the that it we period, arrears, kind do an likely drug, more that's on it's

tuned So that. for stay

Operator

Leerink question Swartz line Joseph of the comes next Partners. Our of from

Joseph Schwartz

PRF PRF since XX are looks by those you States. convert you launch I XX U.S., end patients to how us any total. take access commercial you in wondering then able the being move Based And give insight lumpiness could are therapy through do do versus on renewal for the how the a therapy? many quantify of are were you you've was of if to like you seeing And you you they outside commercial patients? patients anticipating of can any initial around demand might to it us? how all the expanded much seen in And And think into I EAP figures the bolus, long expanded the it be think you work will? around think EAP United might year? once if access from provided, that to still there you the already

Mike Keavany

back on we days need payers new times Galafold look to it launch, through at get XX it's was product, formulary. to PRF to go about XX shipment When on it because their process, a

average, XX-day in XX at than sometimes any the and days. rare on that. Galafold, continue window time. with with get down XX it's to get we general, a looking below quickly payer authorization now So about over But have disease for Over little and years that out XX we're XX X we're and would to time trend do new Pombiliti to We Galafold. to expect here, we to over we product days, the more do, It's really Opfolda with system.

frame. just payers. before. number window on yet anticipating shipment handful more we're time those have mid-October already XX-day to in don't with because PRF approvals We starting coming really to out. PRFs of we're a But And have And the of the XX so than time got we specific a just some from we some

So go cases. right Lumizyme. those time of But through PRF have vast of The clinical got a of in payers who a little we will some to seen again, the already trial. we've majority patients We've take received more switches couple trial bit in And handful been a of weren't are than bit for. little it go product. the a said, that well from I as now that clinical

those Some of Nexviazyme. switches from next are

Bradley Campbell

vast new the in conversions. we've answer thrilled mentioned markets, majority in as who really this and U.S., of are multiple Germany but patients that maybe not in in the Joe, So be of question, I the year and come another seen think, will I prescriptions commercial already conversion each to U.K. we're the in patients

to of year, end new I to starts so goal of kind the commercial our the convert over will keeping the of course then you patients from year. and other this the And that time of And in this the suggested but of goal this well the the the also project number year, come if looking majority the in all frame out stats to our maximize markets, mind think is as Mike is year. conversions, at

come new so demand the to patients. commercial year, of next is going vast from And majority

Operator

question comes Our UBS. next line from the of Ellie Merle of

Eliana Merle

are that deciding switching sense a can speaking between how patient or looking? also us you that when is product you hearing feedback their term and of you declining And Myozyme from guess Nexviazyme, any to that number see care a your of longer do mentioned or think earlier patients terms and therapy? give with that the then XX-XX percent something not new to I split continues Nexviazyme on physicians you play a between are of physicians, standard and either out on or that you're improving the that in how

Bradley Campbell

market about. depends So over you're talking time, on I which think it

of Nexviazyme. I the to have it's, So north of think, reported United patients States, come on been have in now to XX%

those they're because weighted, a of have In maybe draw patients. equally so smaller from you And markets, that a is relatively segments penetration anticipation more our probably of Nexviazyme. roughly handful neckline equally you'll both other

on majority you'll are the weighted still Myozyme there, of distribution. So think equally I see and relatively patients

The be thing key important I about rate. talked label the elements think agnostic conversion ensoreplacement we market-by-market of that is and that this, will it it's most And is some that is so a label important, these this of so and therapy. why

product, study vital that walk and switching would whether or active at on remind to matter to product only and And our we patients we're in that patient doesn't focus in comparator X-minute stability it the improvement has patient on the is the we've the from Myozyme and in physician people shown So well-controlled the are capacity. Nexviazyme, a looked an data. going forced

therapy. that differentiated using have really we we around the for to are that set that's options with So talk physicians go data

Mike Keavany

seen, done and of there's telling one commercial are decision. their data based big either physicians they're is we've really approximately and A some age have to current on also conversations the the majority with data patients. and And seems us. are from patients. market not main remember, early the a we worsening XX% on So a trial of possibility It's patients improving, stable. That our this what that for research in and and both they're and based means access be have or days, suggests switches actually we improvement of driver switch the that

experience sort small the and are still either reasons driver that big to potential a early reports is hear patient end for and But or different as to throughout are have a But in but really have by think deciding the going efficacy the switch of patients So to days, experience. that we doing are think that patient to we that it's deciding community patients a it's patient going or And be how those had. to patients of anecdotal specific driven be continue well. both physicians spreads and it's physician switch. as physicians to improvement listen we'll

Bradley Campbell

kind say of about Sarah, poster center a place is the World patient's that individual, piece One this process individual went. And look, that's the never response talked to published reporting Congress we itself was as there that independent before, might of that show physician team work and States, be promotes medical the hub, that helps the and of themselves, It's we we've to our up on, in United reps affairs that through process. sales going reimbursement the which team going experienced patient a over in a the not heard But course. MSLs the I'll it have talked people case switch and data. something was that again about therapy was how over but an on the both studies, to you course, at have,

established long this as Amicus on goes physicians partners really difference established people different and think has a really are ourselves really And we've a decisions building These make. diseases. out think life-threatening as and way and makes ourselves partners, and take for towards field. trusted are patients And I incredible to a These big that I too. wanting that trusted think I try probe. to in track

Operator

Cantor Next Kluska question comes the line of Kristen from Fitzgerald. of

Kristen Kluska

You've get setting, and or from feedback but are for determine therapies whether they're patients the obviously a suitable current looking on of drivers their I'm not reported in a what biggest are curious to to what endpoints, number them? real-world

Mike Keavany

said, patients as there's So Brad how use assess to that different number of measures a and physicians doing. they're

they also, use. you they in will Some the come then falls, just X-minute also how assessments are look assessments. they part questions definitely are about the those do X patient on every there big walking to you daily the of etc. your is tasks, having sort like to an doing the more some the Many at do life measures A And their variability of doing is similar different of stairs it physicians are walk sort Biomarks. of standard them months, FVC. of in physicians when those include of on of across that But or battery trial get

of and U.S. holistically parameter. not. or more of to of that not the sort could parameters not endpoint patients kind sort sort looked combination It's left those sort assessments look assess opportunities or of at. that that actually physician, totality it's be if of of is There of doing. of patient aspect we of but check-ins specific importantly, how the months is that have rigorous their the are the any assessments with within And really So But not to to label, of the judgment improving patient the as that at also physicians. more really are improving X it's

Operator

question comes the Gon Dae Ha from of of Stifel. Our line next

Dae Gon Ha

the about thinking And seen you this One Fabry going and how see next-gen about the from you're therapy the question are disappointments you naive patient this the obviously, there naive coming patient segment made then going go some word and and remark that cadence led What curious, But of to after what thinking Europe. molecules. Pompe on you're the kind sort next to of are to obviously, And panning next-gen front from you the therapy? of forward? experience patient going profitability Is and for gen, second Opfolda. to non-GAAP that was spread gene chaperone, well the out the We've be after. curious do naive just intact. about a ballooning go on ATGA, as is segment? you there? the how Pretty in seeing how about Pombiliti events think obviously, on The of you Jeff,

Bradley Campbell

come at segment. quite of course, newly already newborn naive but days, you're screening, in I Europe, invest the you we'll Dr. have as patients in if to physicians population symptoms to we've it's warrant disease. there's That's to you from still that as maybe being in body growing as treat of done newly the done those as the rate on a at look exhibited this you segments would continue to really if been So who Kishnani patients patients much high-risk years fueling the others #X there But are a diagnosed those Fabry, that are I and we come. treatment. after the support we've to look also "stable" to But of therapy. patients think, And underdiagnosed expect look is on well segment are seen to who we're literature, diagnosed a course, should is through life at a as genetic some diagnosed. evidence newly look, those they've think slipping if do, that, historically closely and closely, you as looking of disease cracks later starting and it more the progressive earlier. And if and clear well. at are all untreated for look seeing be patients pleased Fabry literature, as are not screening be important or seeing other Pompe early is diagnosed patients diagnosed many done market so manufacturers a focused well, And we've we are in just and and as healthy diagnosis work

community. the I happen think continue to will that So throughout Pompe

and the little As those Dae focused the programs want of to focus Jeff. Gon, is about and keep do judicious about but thinking a to you little Pombiliti bit going, we're Opfolda, is you bit how at profitability point. chaperone this a on said, and a investment We're resources main program, Galafold talk using but

Mike Keavany

programs I'll big gene And with on briefly. therapy start the

regulators wanting costs So the of some there, we field therapy. is the cases, to continued of risks programs, therapy have bringing therapy of some some is to inherent durability gene the seen challenge gene safety that the some and of that some some gene superiority a aspect we around of of are and challenges terms challenges and in manufacturing justify know see, in for the in

focusing standard So think you're able I care, aspects going on doing some over in approved to to might see show more best therapies. of segments of benefit well are people patient whether you be not the that

some at sort something than Given we're solve is our the challenges are wind But trying rapidly to those that really we and think transgene bring more tape. that forward, what best-in-class, bringing and our investment judicious able the forward so in pipeline, looking we that potent around the more is safety bar. could existing potentially of to Galafold when really we're delivery treatments. can it's efficacy. of terms of expand gen on terms still at amenable a improve It's there, the in chaperone, in well next benefit really but of as of high days, see as the deliver we we're to And early mutations over number that trying upon if

upon in that a be have that to importantly, clear line. We therapies would expand for the potentially possible. So In of oral incrementally mutations, Galafold improve the administration future, Megogeneric think there's when preference better, that challenging it's where amenable mutations. response the but medical know great could something to be down we

that that expand can patients, a of think impact. would amenable we be if So list we big

Operator

& question line Gil Company. comes next the Our from of Needham Blum of

Gil Blum

forward this are revenues of do of launched majority may the more that Pompe? XXXX. to Currently, looking think in you recognized outside here now the you've dynamic U.S., being Maybe change

Bradley Campbell

our and of U.S. numbers, the kind I slightly XX% saw see drifting ratio you pricing a at in probably point for And revenues. markets, little the continue based bit to to distribution that's this XX% that just on us. more between historically a Pompe, revenues Fabry towards ex-U.S. In versus you've larger U.S. with you'll reported dynamics, seen think Yes. as of ex-U.S. XX-XX

to Pompe look you is market, existing at kind split. if close of the it XX-XX more that think I

there, expectation and reasonable So to of if of yes, a for trends from changes that's we'll both that's course, continue And balance a long-term Pompe guide revenue. in We'll, I expectation there's sort I of kind of But as comes Fabry. on the of think people distribution revenue reasonable our the report think years. next that that way. few

Operator

of Jeff question comes line next from Morgan of the Hung Stanley. Our

Lee Hung

that For impact the there geographies in you Pombiliti outreach KOL research U.S.? and in pace might for the expectations you different from that of that in you just your patient anything and set impact that talk Is highlight? and about the your see Apollo, And you've does could the efforts be can seen outreach the Europe? how

Bradley Campbell

towards patients, difference biggest patient go Europe States real in you the States, United opportunity education and medical outside do that United between the of the some have no to is the directly community. whereas the there's interact with can Probably U.S.,

little of there. a more a there's interaction bit So direct

these of process. a field liaisons with We who patients Many have helping are who of the team do also the home patient education infusion are in infusions.

aware very a have you And so very very well. aware patient as that's active, large, likewise, by But community. a dynamic slightly community and physician different and active, very

at a and congresses. mouth you'll to of more also But generate Opfolda and experience and of Pombiliti more data, his new focus physicians, continue and which and the cetera, so generating about from now, availability, the is evidence, we'll and presence growing as see directly. word think the Jeff And et with commercial from the patients team on new publications I organization talking medical right kind have educating

that member to and think you'll a about well. part community talks somebody to as their I experience either others encourages see family well really where the about experience think a play as through that or start

new to access continue and more will dynamic Opfolda. and as that grow have to think I Pombiliti So patients more

Operator

of Sachs the comes question line Our from next of Salveen Goldman Richter

Salveen Richter

access already the extend these covered question second treatment is the treatment that And you population patient of participated and have centers XX through the programs, is trials XX the that clinical percentage in mentioned Of worldwide what centers? ERTs. switching you those from is there Opfolda? We that patients any can and patients switched but Pombiliti are are doing existing provide insight to small, the how they've are on over that understand once numbers

Sebastien Martel

fairly is on So basis. HCP Pompe a concentrated treated disease with a group global

spectrum, at U.K., has a for very other reference markets a you've example, for Germany concentrated. You've is of being basis storage lot centers on disease. fragmented. some kind X more those got got which And the differences country-by-country the lysosomal with main The

although In of have scheme for of it a like disease, very not in things, a some way. broad really of varies so you with essentially group, physicians to larger others, And would small And see typically it's we're deal number some to country. grand you accounts you in talking expand still anywhere by in very key concentrated. number the somewhat

Bradley Campbell

KOLs influence XX%, I in So end up in treatment they end, globally, majority whether themselves. being think so decisions, probably those of actually the some the XX treaters they vast the way, XX%,

infusions. home got You've

Sebastian more as clinics got infusions. You've and or have are both infusion that doing the community-based United you articulated, physicians States, the Germany

thoughts majority decision-making, So the and important. treatment which is KOLs think vast top I of those influence

know terms handful how In second the doing, first of to initial of switch patients, weeks. early are too it's how patients the think one, I

and physicians on feeling word how very from getting process the patients and We they're again, mouth in initial are positive switch the days. of feedback,

very though, And starting I I see signals think and of boluses long-term that's the us positive. outcomes of across from consistent PROPEL data on terms from we're data in long-term patients. so very are study being where present confident durable perspective, would, patients. think a the say to that and it's early you're other And from that patients I in our

Operator

That concludes conference Have call. was last today's great your day. a question. This

Amicus Therapeutics (FOLD) 8 Nov 232023 Q3 Earnings call transcript