FOLD - Earnings call transcript Q3 2024 | Amicus Therapeutics, Inc.

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Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Third Quarter 2024 Financial Results Conference Call and Webcast.

[Operator Instructions]

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

begin. now may You

Andrew Faughnan

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

Rosenberg, is for Ellen Chief Legal Q&A Joining Officer.

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

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

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

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

Bradley Campbell

on global across pleased months you, to a the quarter call. QX. strong year successful Thank third another particular, build full what top X rare our momentum, of very XXXX and individuals way us to of this of putting well first our welcome, non-GAAP in by our our has business profitability highlight advancing families our very growth and revenue line to everybody, and hope our affected year I'm in bringing quarter time, to diseases. today Andrew, Great. we've on while also mission been continued In to conference

let As key highlight release, points we in press me did several morning's this here. just

This revenue we its performance last XX% or which excellent product this basis. on to growth in constant with and the guidance commercial from the our high and First, continued currency our exchange the to growth us XX% of XX% XX%, previously. performance level, a outstanding increase continues growth in $XXX year-over-year was in basis, represents year. quarter at in the strong for our revenue at to XX% total to end a million On rates from basis. XX% a led coming year-to-date total execution global quarter Galafold XX% revenue At million constant year Galafold revenue year-over-year $XXX full representing delivered has on currency XX% year of constant for the guidance

adherence markets trends and demand continued both rates We indicators through from all all XX%. performance leading new the starts across continue over sustained to strong key and quarter, populations compliance geographies observe of Galafold our switch in including for our of in patient and key naive and

the diagnosed of growth treated is significant course, in with against Fabry of disease. this, backdrop and of for All people

for in We guidance serving make living with our with Galafold's XX%. the against An Teva started range in XX, the Based This lead to Teva our continue with estate. XX% forward to Galafold increasing This are the high in year a come. also this depth States overall of XX% as difference confidence and range and to of this on announced performance, as medicine agreement as this XX% the strength a we pleased important litigation. growth many decade revenue our growth we for by of one projecting business now Amicus year. disease countries the and around growth full major people our version a Galafold It market Fabry on remaining foundation world, agreement ongoing we're is represents And year our the was time sustained strength, IP breadth update XXXX license the support the we're huge for allows can to United for Amicus. litigants during for our amenable well as in We and with of IP in significant next community it beyond. extremely quarter with reinforces settlement third years beginning of XXXX. the variance generic of ensuring Galafold continues the the case a to the step a the well milestone parties Fabry January see to of

the by course, on maximizing Second, patients great be Pompe year to Pombiliti therapy rate and as Opfolda of highlight in huge of coming driver and mentioned, XXXX a us be Pombiliti disease. well. has our late-onset growth let to it's number continued me report novel to and Opfolda, focus and the patients for of Pombili the Therefore, of global commercial launch continued to this therapy for we've continues progress strong our Opfolda, year-end. #X continues of that exceptionally commercial new to on

health their the of demand we increasing the for depth which our beyond. of to fueling pleased this October, markets. We're Pompe and professionals new and the hear prescribers anecdotes launch will the and see care Opfolda, consistently keep responding of this therapy throughout people for inspiring to from help globally with are the As onset how year Pombiliti were breadth and or the remainder around late across world patients and and scheduled very there of treated continue end living XXX with treatment, disease

strong moving to also globally. and process outside well switch United we've XXXX, quickly reimbursement gain on in United launch additional includes And continuing countries as through Europe remainder and year States. We're significant increasing more in the in the U.S., Europe. progress in in front throughout of the the making patients the U.K. the the as And as launch the will in uptake seeing focus anticipated. States the we're we of This great build dynamics we patients into momentum reimbursement access as insurance for the with our naive patient on and market countries

access front, multiple our Opfolda and continuing regulatory to great to we're regulatory the progress Pombiliti expanding as through well. commercial on On make submissions

incredibly full contributor global sales standard disease strong XXXX, our raising million on in the course the for this for and to will to their $XX Opfolda, sets we We're growth. million to for achieve care full devastating global now physicians and our both year This to be are year patients. our expectations Pombiliti disease. to new of for significant to us providing ambition for with choice the a Given a become great a Pom challenging real be therapeutic which guidance and launch pleased off Pompe living people $XX

reducing as our we're I expectations our pleased to we've our Finally, excellent the year, therapies, expenses. commercial Sebastian? I of are share Fabry And both XX% strong XX%, leadership the across we and traditionally continue Pompe me as that guidance to we've we've to X as advancing Again, mentioned studies exceeded Pombiliti we in we rate and guidance position year. to and and hand year Opfolda. performance. priorities commercial our scientific and the expenses call of we multiple strategic our which, for now And goal further disease expected -- the as execution finally, support our highlight can months profitability again, Throughout manage raised financial a on Galafold in as our first that, non-GAAP continuing the our of sustaining non-GAAP achieve With key year. press carefully continue manage launches operating our Galafold expenses of growth. the me touch finally, as has And achieving third the to resulted profitability let for release, revenue Sebastian, full this successfully investments close as our maintaining morning's medical annual to to quarter. full non-GAAP execute commercial X, mentioned, there raised let over full Slide disease in double-digit well, growth who ongoing we to financial

Sebastien Martel

third continue you, prescribers Thank patient the with patients more call. Fabry with global to patients We're everyone morning news reported quarter on quarter see share demand, mutations. We on many potential driven The of amenable details for with the our that I markets. still leading the the strong the Bradley. to our performance providing by great Slide Good by more the treated Galafold revenue third for position $XXX XX% more million, choice XXXX, for for market treatment very than will variants. of there's amenable X, Galafold eligible you ended therapy. Galafold as pleased quarter. patients with of from particularly On our reached to is that start Fabry

X. to Slide Turning

delivering highlight strength in with rates. same major our continued in demand at patients operational strong to exchange of efforts. over Our results added be the markets, in the the of period third commercial The all for XXXX quarter globally global growth XX% constant Galafold

has demand great the drivers leading U.S., the confidence countries gives top such and this EU product term. remain in biggest over the the long the as growth Our U.K., markets patient Japan of and

Within XX% mix, in seeing about is populations. the switch and stronger naive, we're uptake naive which global XX%

opportunity shares to achieve the the untreated over diagnosed to plenty we of penetrate longest. newly diagnosed in the switch a as been and to There's and to continue we've where segments. market continue to keep still we growing approved patients high market Galafold, So country

market XX%, potential on key by significant that decade. that All of XXXX the several there anticipate amenable who and of in is demand billion predominantly patients we that Cataford with in On go adherence and Fabry by that the end underpinned the belief to know Galafold. the by be reiterating made sustained have to revenue on sustained of up that segment of a our Galafold stay continued reach compliance exceed to drivers. $X patients Slide those beyond rates for X, annual growth patient the We to and variants global driven is

going to and a coming finding from many the new rare patient increased the new First, growing of treatment ongoing see of screening, through initiatives. identification with seen education is remains to support screening most growth and We've patients portion novel population. through disease. and patients we of robustly treated diagnosed family Fabry and the continue medical significant one Fabry reaching market underdiagnosed

the the especially females, in So people becomes as that benefit many can Galafold. We're the to be diagnosed earlier appreciated. transition also better untreated may people treatment, seeing treatment more patients identified, for more from need

The of treated execution. Galafold's share patients other piece market continued is continuing through to drive amenable commercial

market, amenable reach our global were the of XX% that markets seeing mature share. than more we in about we up can market has XX%, currently Galafold to noted, As most of XX%

share in that we the through reach support and market again, those efforts potential programs. So these education know the levels as rates are supported all patient And by well. solid there's adherence and to and physician compliance global

progress markets Galafold expanding Additionally, in we'll make on to new the extending labels. continue and

IP its protection property of our mentioned, announced XXXX. agreement generic October the we January license Teva recently a of entered agreement license settlement on we Amicus in them in with Galafold to Bradley Teva U.S. a U.S. granting on intellectual market in Galafold. front, ongoing Importantly, XX, reached for beginning Pharmaceuticals into as version a with that the the

remaining of patents, time and in IP us outcome come, now orphan Galafold our now drug -- it confidence We're overall rights. strength intellectual XX estate. including as to the the of orphan by protection well Europe, listed a to we to We Orange and property long this for in XX depth litigants the which pleased exclusivity into as opportunity provide of and -- globally access continue enforce breadth, intend our have drug this addition gives very protect XXXX the and our to reinforces as Book case broad beyond, provide the have U.S. we XX our against to our to strength, sorry, in

XXXX, that long a throughout we runway with Looking Galafold double-digit is remain our next decade. growth steady Galafold into strong protection, ahead, the we and IP confident well for expect

to Turning launch our disease we Pombiliti Pompe now progress outlined global Opfolda. with and

Pombiliti a XXXX remainder provides the online second of and switching and to patients in sites [ and multiple the reached from XXXX, prescriptions continue from a XX% For for ] about we're switching more physicians This million. coming foundation an products. quarter with the of Nexviazyme, compared strong quarter We're QX. and from the This increase deepening remaining [ year. from In ] broadening of coming U.S., the patients means new also Lumizyme. on revenue $XX reported see seeing both over represents proportionately prescriptions XX% we to and of the Opfolda a third majority

[ Outside of see what and from seeing some respective to ] the rate and population at ] launch. X some from all the exactly shares U.S., from stage a in segments, patients the Nexviazyme Myozyme switching this we're from naive you at proportional [ want to the market

Given of $XX we're year in XXXX, our our pleased increase first million million, and solid from for execution guidance full X to $XX million to Pombiliti to million revenue commercial $XX months the $XX to Opfolda.

launch Slide X We're months momentum year. XX. to first the through of Moving the pleased very with the

therapy. very are this with been That's mentioned, that late about patients Bradley there scheduled been patients We satisfied treatment have remain As October. treated XXX have demand who or ongoing for of are for as about XXX treated.

had highly all seeing indicated in to experiences leaders. physicians and positive breadth of outreach increase to the gaining greater and launch has our again depth leveraged and patients. cross-functional key with across confidence are prescribers teams, use in an with us are opinion Our it having we've great therapy experienced the We're that markets

and and other stakeholders treating HCPs feedback support we as to our receive our are engaged All approach, very centers to positive core business focus. and patient continue from our

an Finally, to progress is with access track and metric our reimbursement. important

launched by formularies seen Medicaid. in their highly with now U.S., payers discussions days to to European Czech with conversations down also and have believe positive the we U.K., team country. pricing X-month the have our The We Pombiliti have to U.S., approvals will active acceptance Germany, Medicare, interacting largest Republic is strong time we've of the the Opfolda. pricing to in overall we're that in XX happening on We Pombiliti In days. to additional from Opfolda pleased this experienced announce players and we're Today, and major in We reimbursement in demonstrate XX the a around to Spain, put prescription continue Australia value remain the improve. reimbursement the now respective in and infusion and pay and and markets. with

X Opfolda further across broad key pleased to the we're countries access thus securing X wave be a continue countries of Europe, We far we commercial on will the and to Pombiliti months. the in agreements next of uptake part throughout reimbursement over patient XXXX. with in very first launch focused next This in anticipate new driving Overall, be year. and the

highlight the experience number our confidence by talent ability in data, over the our maximizing patients of therapy on that, is clinical us The very progress. let gives Jeff living With year-end. Amicus focus Our and have difference on to real make strength Pompe with disease. clinical of regulatory the now to call me great a of our to and at hand people depth we for

Jeffrey Castelli

we Thank on how expanding registry commercial evidence as regulatory morning. body to you, access outline and our to continue good Sebastian, ongoing Slide of clinical through XX, grow the and submissions. On for we and Opfolda through we Pombiliti studies continue execute

under dossiers and Pombiliti Canada New the a that JNDA, are announce Opfolda review Welfare Japan. Labor to of Health, to for Japan we pleased submitted addition Ministry in we regulatory Australia, the and to and recently Application, Drug already in In or

with as expansions children. We Pompe For into study the onset open-label in to important these unmet in and Rosella for for see importantly, infantile open-label community, to label continue years significant disease. needs children the children opportunity disease this younger segments an living late Pompe ZIP with ahead patient study address the support these enroll living Pompe the to the in we ongoing onset

long-term on continue impact ongoing patient Opfolda and to evidence mechanism of endpoints generate action Pombiliti registry, of across differentiated the and Pompe we populations. studies the and Through clinical and Amicus

scientific Our medical part publications be an continue conference our presence important to efforts. and of education

now pipeline, results, in Finally, our I in and like we in and focus financial side Officer; Chief review as earlier-stage to over call to with to the our turn outlook. the appendix, Harford, would on Financial next-generation diseases. the therapies that, novel highlighted to pipeline to continue Simon? Simon Fabry approaches Pompe for our And guidance

Simon Nicolas Harford

Jeff. you, Thank

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

revenue QX, the over outside generated was in sales percentage of million revenue quarter revenue the XX%. we U.S. geographic total staying achieved constant from or is goods consisted $XXX as as exchange compared sold and Cost grew which At XX% X.X% in of within breakdown relatively year XXXX QX U.S. $XX the The the $XX global million during a XXXX. For remaining million flat. total of of a XX% period and rates, revenue increase same of prior the or of XX% X.X% also period, coming for net to the

quarter of a for XXXX a $X.XX profit of to quarter In income Total the $X.XX last of share X%. was net compared year, of development XXXX. net million to for expenses depreciation expenses, define to decreased or operating of quarter impairment compared amortization. changes million the $XXX reduced XXXX in basis, the value or in non-GAAP last as $X.XX third for to and to for expenses, $XX operating to the decrease third $X third We of non-GAAP million for consideration, compared decrease or a basis, the SG&A third decreased $X.XX third last loss GAAP a expense, the in $XXX restructuring year, X%. and share On total million compensation compared million XXXX contingent loss a non-GAAP year. On of third per loss assets and a non-GAAP of share quarter as stock-based share per expenses excluding research quarter fair per quarter million third of operating on million of $X of $XX net to or GAAP loss loss the net of a quarter QX $XX per charges $XX and million

cash at were Cash, marketable at September $XXX and million XXXX, XX, $XXX December XX, to equivalents million securities XXXX. compared

XX. Turning now to Slide

raising range total from XXXX most XX% XX% to to year XX% the XX% at to XX% and This increase XX% at narrowed constant of to are We growth previously. beginning to revenue revenue guidance our now to XXXX recently to rates. XX% exchange to XXXX guidance full Galafold year full the is of driven XX% our from by XX% XX%

to are we the Opfolda for for addition, Pombiliti and sales to In $XX the million raising million guidance year. $XX

$XXX guidance commitment from at full our With expense year guidance. launch the XXXX year non-GAAP million first million keeping we $XXX $XXX Opfolda, profitability single year-over-year full growth expense digits been lowered to of $XXX full low million to operating million. during to non-GAAP Our has and of are down year of midpoint to Pombiliti in the operating

a R&D yet As to next-generation and as study reminder, continue Phase studies have Fabry for reimbursed both registry countries in Pombiliti. we ongoing including III The in not well Pompe commitments, manufacturing Pompe. as

growth, our we XXXX track guidance With full of on our comfortably year non-GAAP of total remain XX% first to revenue profitability. for XX% for

up, XX%. a Pombiliti first be work expensed inventory the year hybrid months think approximately part for As through heads of during a to year XXXX, the to previously we for COGS Total as year were and the of XXXX. to margins anticipate expect XXXX we as about we Opfolda begin next gross the first X

for let the call me Bradley to with remarks. that, we year expect the recognize We gross in for the mid-XXs back and turn our P&L over COGS to the XXXX year. through as And next begin Opfolda Pombiliti margins full closing

Bradley Campbell

Thanks, Sebastian, Simon. Great. Jeff.

commercial us focused relentlessly by team been patient-focused lead of have we continue see, employees our business, global execution to all and our across can the you mission. on performance on who As passionate driven

end the the near a in I therapies With and continue for laid that, XXXX, deliver open now transformative we the Amicus foundation to am operator, can solid we of call and have questions. years to confident beyond. we As XXXX

Operator

[Operator Instructions]

of Ritu from comes Cowen. question first TD Our Baral

Ritu Baral

Pombiliti I wanted that your about to prescribing you ask trends mentioned.

-- seeing a improving patients in U.S. Japanese describe quantify which You about coming trends what and more and had mentioned the U.S. now Could And us at prescribers. experiencing? that's switch that are opportunity and both Pompe point you the what that like sites help elect you on since go a could market bit to prescribers the into you're little you specifically then up. patients just little

Bradley Campbell

Sure.

Jeff a and was and little X of on with question then it breadth and Sebastian and the switch around we'll to hearing framing bit So we're provide opportunity. and well-articulated a the color decision to Breadth color then very comment one little what first on Japan I'll maybe depth start bit, then more depth, can Japan. parts. one, seeing a and kind ask

and says, breadth what exactly So kind in right? of depth, of terms it's it

had United the in on So But key the U.K. markets, where a and we markets prescribers. other States, a continue basis. to it's prescribe EMS some program prescribing Spain, more market, centers the all very have through like concentrated Germany, commercial most

have prescribers to they breadth, want you what to seeing things. new prescriptions, meaning want prescribe of repeating and we're depth, the so you continuing both are And see medicine and is both to see meaning to those

Pombiliti prescribers more So for and new Opfolda then more time using and prescriptions. multiple prescribing prescribers the first

see think this a to tracker to we'll product. great that's I use So spread the how continue of

when -- the of for patient are ongoing the to of talked and terms their course want of an that is see Broadly part think lot decision looking when heard important months. -- Once switch. a about I think XX they're community. that this the is made, And we've what of some to evidence patients. before switches, in over sort physicians of XX is we think ready I to year. physicians with seen speaking, the a decline I are the In discussion the

update that the And then specifically just Jeff opportunity. up next on and symptoms so be year. kind what for people follow Japan maybe and sets typical well I are about a give the would think of us But that Sebastian quick for to talk signs

Jeffrey Castelli

Brad thanks Thanks, for question, Ritu. the and Yes.

trials, They a covered or of been XX- follow still period the think to they well switching patients. life of of doing day-to-day measures, treatment very terms that activities, the starting of XX-month measuring lot sort after X treatments of a has they're So months lot patient there we in I measure quality every do or how it in their of what the in mobility, assessment holistic function, see to each ways at how assess really trial. the in that But different doing. recommended seen physician we're of respiratory measures after least before not workup first the is of XX certainly has the it's vision switch. quite seeing some that guidance it's certainly different XX at things sort kind Brian initiating you've that see typically patient But switch physicians wait -- so, of that them second their are even to months a

it wait they And they is if see if of they they patient stable, were a or showing currently, of improvement. treatment, new start sort declining If remain treatment was to declining new wait want they are the to and see stable to again. sort they on

it's the definitely we're are majority sort somewhat in that by that seeing XX- switches But to of patient XX-month patient. period. of coming So

Bradley Campbell

real just the Japan quick on Sebastian, opportunity.

Sebastien Martel

Yes. question. Thanks, Ritu, for the

Pompe there Fabry the speaking, sizable a XXX So This The market opportunity, not It about It's is currently that smaller we're in relatively actually is We being Japan. treated second disease to case we very prevalent Japan. is also market for fragmented. for patients for Pompe estimate -- in used largest Fabry Pompe a very in are less the market. disease. disease. the world,

Operator

of Our comes JPMorgan. next Rama Anupam question from

Anupam Rama

patients in that sort just wanted bit the now I of previously Galafold. on to a untreated are on dig little

patients untreated regions? of I core market more regions the versus That's the Can my of XX%. slides emerging some regions you speak previously around in what your to these some about one legacy think you're or question. at like seeing

Bradley Campbell

Anupam. Yes. Thanks a lot,

just one. take I'll So that

still untreated also, in So Europe, numbers I significant U.S., of finding key new example, we're pool an diagnosed there's so a think significant our patients. And Japan, of remember, markets, as patients.

about about into came X,XXX year sorry, in -- treated diagnosed estimating XX,XXX diagnosed and patients, with XX,XXX figures patients, we XX,XXX XX,XXX the updated diagnosed So untreated.

of untreated part that the diagnosed phenomenon market. is So into penetrating

the with we think, countries to as some diagnosis. family a find there. diagnosing well I to think in index family members as led significant X advances screening I you're numbers find that's levels Fabry JPMorgan, give you is you patients new of I updated developed whole when typically genetic adoption seeing we low-cost significant of an think, where overall, X or testing with, come in, But patient, what host more But of

The there switching launch some our shares, But opportunity is switch other regions we which are Latin still thing East, well. a market to regions, We're is patients. very as North Asia, sort newer significant of high percent. be at XX-plus still Middle America, in tend Africa Southeast

X there. But I So market solid underpinning diagnosis I again, think think growth drivers growth. of that and

Operator

Our next Securities. of of question Tazeen comes Bank from Ahmad America

Tazeen Ahmad

any all relates announced of tell just with? as then for filers you a front? rates are Galafold, point at On you it those And of the settlement dropout clarification can if rates IP, us chance, it much the recently? a if there as Galafold, relates to just have remain that same changed or left I'm there outcome could Is on you've wondering any the still that to get kind better than one

Bradley Campbell

very much, Yes. Tazeen. Thanks

So on those. both of

to against However, and of I the outcome. with litigation we're ] IP long-term course, still will comment reinforces remain the in that on your remaining and the vigorously Teva on said call, particular, we in are the we really X with the as litigation say force pleased this our [ and of confident So the the very based strategy. confidence continue, will prosecute settlement Lupem with very in because the case on can't or And Bindo we we're Orbendo strength there to question, it litigants. in potential

we're are. us ultimately, a won't than settlement a majority that And But we more these many very very pleased compliance smaller parties. types interesting an of actually comment gives Galafold rate question. for given come. pointed other this regularly. numbers which the discontinuation years is excuse not is date broader And cases, the to to fantastic there's a statistics, point confident in these I where on than and which later the that's Galafold, of that What's an effectively on opportunity everybody again, with for of of stand come your us -- sustained to patients would is the continue to place review I people On me, in on I this base. continued to and quite we've we're to think smaller earlier we your when they that be. second say has a to support gotten tend expect Galafold, adherence growth patient And think what's Galafold, course, I -- or actually,

Operator

UBS. next question comes of from Our Ellie Merle

Eliana Merle

guess on then of development? looking your as just what's Are how internal stages about with overall? you there terms pipeline out you as specific here? focused I where development work? And about clinical I guess, business pipeline, And your In you development, thinking more you're focused? you're your are building thinking think externally focus are business you strategy about or from of Or that think most modalities about development

Bradley Campbell

Sure. strategy start and our a framing bit the internal talk then program. Jeff I'll about little maybe with overall

to months, we've will we of that I we think a and in course, stage of looking later infrastructure overall, over So And kind continue for delivered assets quarter or another to think have XX [indiscernible] business, an support assets I we on great believe continue the focus great. And is there do of and the Galafold to built into because to strong be bring is next portfolio. in. first be de-risked commercial XX to bring would opportunity We the Amicus. here, growing which the we've focus to said

pipeline? have really beyond, in an do Jeff, a out to our think and then, I again. little to opportunity talk investing our might come free positive about we'll we're cash the of for what hope also call flows, it, And you want pipeline when bit strong XX internal begin generating months

Jeffrey Castelli

the Yes. question. Thanks, for Ellie, Brad, and thanks,

and proteins. with our proteins know, you potent to wild-type Fabry really Pompe disease much more the exciting we've In are transgenes developed terms various of And we've research for been efforts, as deliver those active, stable internal than through that some looking approaches.

Our constructs. focus original AAV with was DNA

or that So lipid our we're those approaches. other looking, but looking other that proteins to still or nanoparticles lead whether at is we're area transgenes, also be through that deliver ways

not are treatment, is in replacement we main Fabry people for continuing therapy. But secondarily. Fabry So focus to focus next-generation for there are on is opportunity well amenable look there's as absolutely Pompe currently think Fabry that some to the a that real especially at as

turn approach to that influence attention In the we're Pompe. product Fabry pay BD doing I the to and say how maybe for medicine out and pipeline. to on every internal I there will future, what close I'll terms of it about single Sebastian talk over we think might

that very something also that we of interest. of So areas broader a obviously we're but approach is keen disease on, have

got we Sebastian, you. to So turn to

Sebastien Martel

Yes.

a neuromuscular we've at interest, broad therapeutic we're areas. a rare of think specific We're indications therapeutic at metabolic looking renal, looking but looking areas rare reology, I at rare cardio, framework also adjacencies. and developed

Operator

next from Schwartz Leerink comes Our of question Partners. Joe

Joseph Schwartz

were added prior end It around end the October from to looks like around quarters. patients versus XX% in XX of July the of

$XXX a in and sales be driving million, of should the the cadence doubling quarter? for are how to into patient is Street trending around we things So and about thinking like we what wondering of about you I'm started new expectations about this that's it sales the assumes as of get how And looks fourth year-over-year. think 'XX? adds 'XX, Have

what So of forward? quarter of activity in make and demand you patient this going terms the do

Bradley Campbell

Joe. Yes. Thanks,

few things. a So

strong have which did we from great QX, is a perspective. a very So revenue

that the I your new call of at have think number been quoted numbers the would you treated, also you up patients I XXX. is if you that to think I of which to look line top who prescriptions, right. number

the is think which Sebastian that So kind of we're process, part gap of talked to insurance the I on earlier. closing

launch what you're QX, and Spanish a about number happened a switching of few the had QX, think trial we As still you patients. clinical things, in both

was the so there. are that just in out ongoing And really the various of QX launch kind countries

QX. more next to to outcomes, lead going do any we to some expected, which reimbursement would in although expect have launches countries We have get of We'll want into year. to new don't that launch

In terms It also the of sequence next on that Sebastian patients year, of depends that course, the on it depends we talked launch to. with. year finished this of rate

a have on half number are of next coming countries the of that back We year. in

a to over We you have sustainable is discussions. those playing out also of period, make while And to kind course, speed X- also X-month that have launch ongoing are but sure you to of want the profit. goal a reimbursement

very but and So the raising then what year more on like, to get we'll launch of this looks far pleased next the we year. the as more we be for super that year about know get excited clarity so with a end and to lot guidance

Operator

Ha Our next Stifel. of Dae comes Gon from question

Dae Gon Ha

the the on I And story. circle to congrats wanted back the BD on of progress. side

lot we're done and Now STC that There's up come chatter with a next. around election. of the who might

significant So on profitability BD GAAP pulling and driver over precommercial hurdle on here you the I a cash about or assets, the on guess once already about flow the that radar trigger, something there getting ultimately? think the Are front, to for non-GAAP before clarification focused profitability? just Jeff. what's pulling when trigger more you late-stage think get you you the the a is of your that's And these might

seen these that from these guys you same pipeline, what approaches ones we are you the do are have the separate that are from [ guys had retaining Terrata?] -- or versions Caritas for evolve that On genetic these

Bradley Campbell

I Yes. that an of for business, And and/or that by Dae bringing are from on lot objective we built that the question. products thanks to is at can based in now therefore, an think perspective, derisked we've that already current our think part goal. launched. the was Amicus a is Gon, add the infrastructure which self-sustainable we And point

line maintaining, focus really the on while line. financial we around So bottom think, I top our discipline growth

you years, products I profitability So non-GAAP over deposit and think I period to of time. on along free few cash we GAAP the we to as think way mentioned, as the about next sequence the that's think that playing of focusing later-stage flows And out the then profitability, over of first. can go

able funding to start pipeline be as our I then a really own to we'll have resources well. think

it. just focused specifically think that's technologies. with Jeff, And to how component we're maybe the So of our generally kind clarify we on about current what financial

Jeffrey Castelli

Yes.

still we're ceratosopy in in of of and our and doing, R&D been as it even core have go on those AI is incrementally refined of early and Fabry, been one with but on the some And efforts also at Amicus we constructs what new approach work resources to the ] internal looking those terms even still technologies. businesses better the in focusing still an I'd focused to ways deliver part going most then made was on we've So we programs. constructs, worked resources into alternative is as them AAV them with constructs lead our [ say similar to later-stage previously Galafold we've and Palma,

Bradley Campbell

primarily the our focus course, So. delivering which business, the year to in gene would core on. therapy slim and some moved X/X can't -- that Jeff to again, to about those clinic. X of or as because, might down of we and we're important move Yes. We're in kind the with of particular Pompe Fabry the took half population when efforts mutations then allow be said, data who Galafold. decision, commercial away from of the proof-of-concept goal Again, on on don't And treat us Fabry really, focused closer to we to have course, really we next amenable generate

can more scenes that we forward. an of behind the we're be significant fund in now, could priority just along and right low kind is So But exciting until going things that approach again, all moving way. a them

Operator

Ding Our call from Dennis of next Jefferies. comes

Unknown Analyst

I if And out into terms anything QX? conservatism QX would for question a assumes of Pompe there's guidance? in flattish timing have worth just is had or much and around benefited shipments inventory how generally guidance be baked pombility. may XXXX drug calling your this wondering I of normal new that

Bradley Campbell

new come recently, Dennis. year. an over do The There incremental Yes. is of growth kind aren't launches some next We until although be that get breakthroughs. patients quarter. those we going think this Again, to Again, reimbursement any did [indiscernible] example, reimbursement see course there's to there on won't we for some the add quarter? question, patients of the question the much in is how there. Thanks new baked

we on tell rate course new of really of depends incremental it is the of we of some where adds guidance. growth as the range. us there into and that go within patient that are kind kind that just baked through quarter, So will the But

Unknown Analyst

maybe of adds? new see the -- the Pompe the as any growth? patient terms about And follow-up, do this more launch, as in kind and like of point think it. messaging is Or of acceleration of study you around Got a steady you trajectory the

Bradley Campbell

we of factors Yes. don't Thanks Again, yet, will the for but out. I determine plays guidance question. think a give that couple how do

So into of of have markets. patients the as starts fairly you've in next year. first And kind the example, patient -- you're new a are X-year the to that of do in sizable number rate zone new an patient X- U.S., moving all, think existing who we of now to got going

year been then talked the great dictated of And ] be to part rate the start by So or who've then a [ those have in Nexviazyme process. process, which new the mean And patients. it reimbursement and regulatory that's you And a opportunity countries could I switch about, we patients launching. by to for X. already on part also dictated

in back example, some year. next submissions think and we've online Japan, Canada of the half will As come an of made, the Australia, we

rest you still next the year kind think go as those we color -- see build to momentum do of have more out see to this of the new moving But on into year. this start -- of plays rate I time, draw more for the So will you again, we'll how the pieces. and patients. launches as over some see more ability you'll markets and

Operator

Kristen of comes Our Cantor Kluska next question Fitzgerald. from

Kristen Kluska

all on I updates have the a not to guidance of great on that of quarter, as operational the side, the from hoping the comment providing Congrats you just some more operating revenue today? of well. was from tightening but the led can expense some drivers you're

Bradley Campbell

to Kristen. Thanks, you that? take do Simon, want

Simon Nicolas Harford

year-on-year. Yes. operating that we wanted we continue our to Really, say expense, as extremely manage we to to do I said, fair think tightly it's

would infrastructure additional we additions launch as to was I place say that because have support to we've we I as IOPD add the importantly an from the didn't would Fabry, open-label the things headcount the some to we to in need to registry on numbers limited, that of however, deliver really LPD, we do perspective, talked continue, studies, and some adolescent remaining significant Opfolda. OpEx activities about such we of all expenses sort and of the related say, in extension. that So need built Pombiliti have we the

we're also all activity result really in an what guidance, but OpEx year, managing seeing as the of non-GAAP obviously know, because profitability million expense from of profitability. line this $XX reality of non-GAAP place with we work taking team of perspective, look of a is we'd and full to is that's ensure to you that that which management first year you're employees talked of lowering X all as the about So helping And bottom overall tightly. months non-GAAP operating achieved the already as we've opportunities the that at

going on So currently. that's what's

Operator

Our next question comes of from Hung Morgan Jeffrey Stanley.

Unknown Analyst

be like start next for This strong previously How inventory. expect could far next-gen expensed Congrats year? that potential quarter. for XXXX the for carry Jeff. any the on on Opfolda can How that would inventory manufacturing you to would for gross far And margins? like Pombiliti able is Michael to on impact when that the into Reed like, provide you commentary

Bradley Campbell

second to I'll the the take Simon, have you first, talk one. one and then I'll first

the we of in, kind impact. kind can and decade about goods cost We talked of we've although working investing that a through by So next-gen manufacturing to back half process. something still probably think maybe of is That's XX% that we're we're before. reduce XX%, of

formal the but to hit a year. talk commercial few near-term guidance to out supply drivers maybe before has there, given haven't that we starts years it chain that's to and a the next real So that still impact starting

Simon Nicolas Harford

the therefore, COGS I hybrid fact next run should the in I range call year. year. say driven will gross it why the on the sort by margin mean heads year. the up a earlier part And in mid- in really expensed in that, terms the next of we mid-XX% Yes. that of or inventory that's out of That's

but you We to hence, previously, XXXX Opfolda make in rolling in sort XXXX that and has Pombiliti least as at we in will we've gross this of we wanted see XXXX, a margin year aware difference at point through compared of full of statement. said impact time what to see,

Operator

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

Gil Blum

genetic pretty genetic faster interesting. relates as guys FDA program a could for current approval wonder expected. what Fabry, kind So in was whether to and just from and think medicine the accelerated little potential than recent for dynamics feedback regarding I it medicine you of a push Fabry

Bradley Campbell

a it's good Yes, question.

in are We data based we've that out I patients really seen. think on as the the other therapies those it nonamenable there as with just mutations relates that to see impacting development.

for something we And continue other so population. love would we're part the grow really but patients, have for excited amenable to about to Galafold that of

thing finish think think From we a which Hard therapies, same read I as other just flexibility to know seeing broadly, you is the regulatory happen what's the some perspective, of in you're we agency, the the do. exciting. to part something But address going again, any that certainly those nonamenable coming the for on do get see I we our think they're population if of patients, the across to can almost line, in able to and if are so years as to own eager well.

Operator

Sachs. next Richter comes from of Salveen Goldman Our question

Salveen Richter

ex ex-U.S. some to Just Sanofi's and U.S. Vizyme dynamics further the ] for growth share for the it be in given competitive that next the possible asset last the just in us would [ earnings. versus commentary appreciable U.S., on give

Bradley Campbell

markets. switch Yes. the X proportionality to Pombiliti sort terms Sebastian, in of talk things what we're a Opfolda little those patients seeing maybe how of kind of evolve to getting to as in we're bit and

Sebastien Martel

Thanks, Brad.

in question. So the for

mentioned terms and we're much in shares U.S. I Lumizyme what seeing of of dynamics the the current reflection a as switch respective is the earlier, pretty Nexviazyme So in in of U.S. is

XX% proportion Accounts we more for that say which switch sales of XX% Lumizyme. there's [indiscernible] on got least us there's is our over did So Nexviazyme said at a potential numbers, switches that QX as still to great we've from slightly at time. this point, that than a I based reported large indication X/X and for Sanofi from all U.S., the patients along, in of of coming Nexviazyme, about

it's seeing We're that actually also the ex situation. reverse U.S.,

XX% Myozyme next is XX% roughly there Nexviazyme still and more So [indiscernible] ex U.S. align.

And a proportion so greater switches from coming here, tend to see of we Myozyme.

surprised. be wouldn't You

seeing order. of patients benefit on naive the have moving straight also some to We

Operator

I for you. to was would last like pass Bradley to it question. Thank remarks. our closing That now

Bradley Campbell

and great a the call. for everybody. conference listening day, Thanks, Have to thanks Great.

Operator

conclude today's for program. you This your the participation Thank conference. does in

You may now disconnect.

Amicus Therapeutics (FOLD) 6 Nov 242024 Q3 Earnings call transcript