Earnings call transcript Q4 2022

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Operator

Good morning ladies and gentlemen and welcome to the Amicus Therapeutics Full Year 2022 financial results conference call and webcast. At this time, all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will follow at that time.

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

Development today’s Thank Great. Officer; Dr. our joining year Chief financial highlights. Executive Chief everyone. President Quimi, Jeff Bradley Amicus Sébastien Thank operator. Good XXXX Martel, Leading corporate and Financial you Officer; you results we conference and and Officer. full have Campbell, for Therapeutics morning call Chief call Officer; Daphne discuss to Business Chief Castelli,

Chief Rosenberg, Officer. Joining Medical is Officer, for Dr. Chief Ellen Q&A Goldman, Mitchell and Legal

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

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

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

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

call my Bradley pleasure to Chief President At this Executive Campbell, over Officer. it’s turn and to the Bradley? time,

Bradley Campbell

we’ve conference of our into across welcome full start made the now highlight and call. our XXXX. progress the everyone results Andrew Thanks pleased throughout business and today Great. XXXX to I’m XXXX year to

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

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

We this revenue and people with we globally, medicine. a strong What treatment Galafold pleased X% currency for over continue last of currency XXXX with Fabry. to foreign living significant disease, only demand Galafold on taking or of for now throughout their Galafold an growth constant XXXX, Fabry reported year, operational XX% be amazing And very with despite precision growth in headwinds basis. or milestone for the experienced as an

Throughout and strong and performance visits populations, naive to continued last starts key this and and between world person trends year, compliance patient Fabry switch we over field across demand new throughout rates into from increasing and in sustain number growth and of including of year our continue the see through patients adherence our XX%. a indicators, the both traders teams

expect patients we by XXXX, physicians. to digit growth see and demand Galafold fueled continued In from significant double

the see by Galafold to There pleased a also year. XX of XX We’re through related estate Intellectual States, in total patents of the Property now are XX over which issuance We Orange new this late XXXX for And broad our of this XXXXs providing precision drug patents. orphan along five intellectual exclusivity, and protection long-term to importantly, novel, rights patent this composition provide matter beyond. the patents growing listed issued United last Book into property portfolio, medicine. the Galafold of well includes the with and

two commercial Pompe on planning disease. launch Second, and component we global filings for progress AT-GAA, continue to therapy make regulatory novel for our our

AT-GAA third Pending of during this will the the in launch Biologics States has successful year. approval U.S. announce pre-approval to WuXi quarter scheduled China a anticipate during of United the of required XXXX. second FDA today inspection, thrilled and regulatory the the quarter be site are completion we in We manufacturing inspection of of the the

AT-GAA. on MHRA track Europe, on by applications reviews we’re the with both and regulatory for EMA in marketing the Additionally, authorization

the opinion December, Commission positive Miglustat We be approval in a opinion of of this this expected the in marketed CHMP the with reminder, quarter. CHMP European anticipate alfa from a of year. cipaglucosidase as adopted quarter second a enzyme Pombiliti to As component the AT-GAA stabilizer

EMA WuXi indicated And for expected and a In third initiated an regulatory quarter on require the year. the the does in that December AT-GAA as for this it approval. reminder, site an condition of inspection of manufacturing U.K., their process approval not submission has as in of is the a the track was

in Kingdom, see now significant France, As are expanded throughout globally; we participating the growing XXXX, for with U.S., place and Japan, number we’ve observed United patients Germany participation mechanisms of a access our interest to each. programs continue in in AT-GAA in in

our of XXX X% which studies than world. we expanded today, believe patients on-going on we’re all of access Pompe around over more AT-GAA total and the programs treated patients clinical represents Across the now

We number and States and United expect grow we approvals to as Europe this to approach the in continue beyond.

getting applications this move another people Pompe disease with In to for finish these States, prelaunch months ahead, including inventory. across and capabilities reaching United and living the the personnel Europe anticipation in of our regulatory plans support to launch with further in additional planned our option milestone AT-GAA and the treatment investments ahead. anticipated much in Kingdom United regulatory activities, providing investments near to line the the and of launch launch closer approvals We're targeted on-going continue and building global the and term,

And position a global expansion third, the maintained of launch for we financial global as the continue to execute and Galafold AT-GAA. strong has prepare Amicus of in

presentations from remain And of latest projected Fabry programs finally, programs, and to oral half continue Pompe to just that the on our AT-GAA, two XXXX. of expenses the and which the we Annual launch Fabry assumptions and highlighting highlights second in profitability, disease. development progress Symposium in key on a and World out achieve Florida. based Galafold, February Amicus held for posters at in presence round and Importantly, our across expect once and track XX do data in to the significant we’ve careful our made we to plan want mention of again I disease approvals current operating Orlando, this non-GAAP we has growth strong has continued management Pompe XXth our

the Most from patients of notably, key our later showing years first for now Disease, And data Galafold. years. results III of in supporting the detail as we evidence registry, in the in up Phase extension consistency Also effect efforts provided encouraging first the of for and AT-GAA demonstrating in will Pathfinders three to build from -- population. to go the represented continue own study long-term Fabry call, we FollowME noteworthy, late safety and Pompe open-label out into time part we efficacy be two of EGFR more durability to presented onset stability and a that the will this our body to Jeff

we XX% constant as month, achieving key disease markets. while Galafold to minimal last focused strategic Finally, at in laid out with Galafold our delivering on a revenue we variance for XX% more XXXX priorities growth double-digit existing on new and with sustaining slide are exchange one, of Fabry including four, to number living rates, people

preparations Number well regulatory this two; AT-GAA year, laser launches. EMA we MHRA to remain continuing FDA, anticipated focused on as and the respective for advance the approvals as securing later by

fully global and our we a launch anticipate Galafold commercial, AT-GAA. access successful prepared experience and for market seasoned Given are of medical our and success with teams, and

on next advancement our And and path medicines three, we and chaperone our Fabry to of our best-in-class manage Pompe capabilities, expenses and financial position our next investments Number genetic well generation we will a for four, as will as we profitability. in invest as Fabry continue as always, maintain number the generation to judiciously non-GAAP carefully disease. strong

Chief our With Sébastien that, Officer highlight now further Galafold hand let the me Martel, the over Business call to to performance.

Sébastien Martel

million and for more of as the U.S. Bradley, we morning walking The of in you XX% year. the of geographic a patient revenue is good $XXX accruals that $XXX offset business. breakdown Galafold performance X/X see the by generated U.S. driven currency thirds, with revenue new both parts reported as continue strong from we reached to of everyone revenue, within year the you, full I’ll on of significant XX% by the This year of the XXXX, the outside On or Thank or slide the grow headwinds. line start through you can million, our for to Galafold by foreign in full details call. million X split expect and coming remaining during two consisted $XXX

countries are continued in patient Italy We Japan, U.K., just like growth U.S., of the the to a name our key few pleased and Germany, see to markets.

foreign at efforts, rates. added of in currencies impact to full X, strong, period XX% globally in all results for growth our strengths on be demand highlight professional over a Turning commercial the major global Galafold for continues the XXXX in The negative exchange markets, incredibly patients was our delivering rate X% same with constant the to XXXX. of the year slide

of treatment three full was and year. pleased period of result to X% All new market truest globally, revenue two last continues Galafold Pompe is the continued patient be the the at XX% versus the and trend Galafold greater and reported to fastest the saw I’m measure to look from a In Galafold the perhaps XXXX, at trends month contributor the of monthly and remarkable. growing disease growth. in underlying demands that the growth continued in for to of report Galafold. patients Fabry on net end than same the the growing greatest highest we net global As And our business, That’s globally, growth XXXX, growth which for Galafold the years. XXXX. indications the if was patients increase you the

seeing more ended mix year in share over Brad patients, and of about the global which while naïve As we with of we’re mentioned many is XXXX on earlier, populations. in uptake and the XX% switch geographies, XX% patients amenable treated than the naïve global half remained Galafold, stronger market

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

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

of Galafold to Importantly, track continue be continued and reimbursements the on value outside have successfully our strong by U.S. recognized the payers we and of negotiating renegotiating of record

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

well. very through continues track XXXX months those our we’ve is markets. What of we’re two in year seen uptake key growth first full XXXX, across the with trends And continue Galafold that all to seeing the

the to growth in $XX Slide strong quarter. with Galafold remains on Turning million X, fourth

fluctuations mentioned As calls, of a FX including typically factors ordering growth uneven due and non-linear. rate year patterns the within is the to past on of variety

XXXX. We continue that through expect to

year. given right the side to historical that this year Galafold during In five hand a We year. each work with similar the expect quarter of you we’ve would sales of trends snapshot through provided of table a the on the slide, percent altered see

And good so don’t guidance, water. we quarterly very while like benchmark for provide distribution is historical breakdown the revenue

in in an last QX example, that the years sales year current As year. with the tends generally two QX prior also aligned sales to observed the be in you’ve

patient X, into significant through years. XXXX it $XXX few and We annual potential Moving anticipate continuing a know on be to existing the drivers; first in over demand there’s to driven growth key for to million we by growth next Galafold, surpass slide penetrate that to that revenue market. several has sustained

label And and into into geographies third, and uptake further Second, the is new population. treated diagnosed expanding expansion.

to throughout and all the we and on markets, We to and a into efforts these as compliance mentioned, rates that are new market world. and in received supported policy mechanism year Turkey are reimbursement process authorization pricing continued pleased adherence As in by solid and reimbursement last we’re name we speak. making examples. I’m the few progress expanding just access share of

future. hope Hong as We New countries the Galafolds applications Zealand authorization countries those have for marketing the submitted in near new to and see in Kong and

diagnostics agreements and patients well. continue as Spain, growth risk have of testing Galafold initiatives, including diagnosing continue and variety We newborn to and in Italy, like screening will through high we the Switzerland, longer-term see successfully reimbursement and market globally which countries driven Iceland. for by in screening, support other measures renegotiated to a Poland, significant pricing With Fabry

to Europe. exclusivity orphan note important and Also we in the have U.S. in

who into to it opportunity to the addition coverage now come. globally to which IT patients Orange XXXXs, Book a the our XX listed to time provides us Galafold that long In patterns give late access to need for gives us

launch launch. largely at site will And needed. preparations for AT-GAA be That are for new the that today same On as over as major XX handful all product another Pompe we AT-GAA of launch world, countries a that the we is outlined around including presence team in Fabry successful XX, with SGS poised and it the markets. a only

launch, experience are areas most regulatory, with physicians. across successful with reimbursement commercial, key We access. and importantly, chain drug in have payers needed addition perhaps And supply all relationships and for experience that

we world We’re experience AT-GAA and confident our their to in Pompe Disease very deliver relationships with class the world. living and people can organization that around leverage

education, medical been without successful all the we our experience with WuXi access world. we’ve team, are inventory partners the And at doing position publish the around Amicus. III From building with biologics, Phase strong for reimbursement the planning the a strategic in very together again, believe we and for second launch data,

me call to Officer. Jeff? over Castelli, Dr. With hand our the let Chief that, now Jeff Development

Jeff Castelli

of neuromuscular publications treatment, followed disease, for and crucial. health On We natural having for individuals. Pompe initial and is key the lysosomal Slide continue AT-GAA recognize is morning generally disorders, and continues affected multiple and pose being that long-term by disease severe most fatal range continued good challenges the to history of and a Sébastien, we’ll to Pompe program. you, and choices everyone. disease Thank our of many disease XX, benefits with the people therapeutic decline studies prevalent start on a measures by highlight one of

onto be the of XX in are Bradley year. we third mentioned, Pompe very three now largest the to as this markets launches potential and Slide approvals regulatory Moving of excited quarter into anticipating

regulatory the or market. our on by highlighted Here this we’ve the status year slide anticipated milestones

progress final EC CHMP later on this Commission for in the testing expected First this approval for is to and validated in quarter those third followed great we’re track EMA the by European snip be an first pumpability in confirmatory miglustat the making data the on would expected EU, the And then month. quarter. approval quarter are second in opinion which

approval for both components site was of inspection. And in FDA required the quarter of the been then WuXi AT-GAA second scheduled the of in quarter, China the inspection the successful [Indiscernible] Biologics a pending we the manufactured, has U.S. pre anticipate with typically third and manufacturing approval

Finally, the procedure had also track in a we’re was the quarter. December. U.K. and the MHRA for an based regulatory opinion, expected in third They submission process CHMP AT-GAA for in initiated on the on approval recognition

expanded to Slide highlight our providing through Moving mechanisms demand on AT-GAA. XX, clinical that multiple on-going to studies, the and support of access

to Disease into label LOPD and younger on-going year. for we Pompe in of children the XX living Pompe First onset the years open community, study continue throughout infantile age, enroll with to with up this expanding patients on-going are

children Importantly, expanded the for and Pompe to in this the multiple the physicians our place we the leading AT-GAA the having in which including of we Germany, of the across in centers includes access have others. under was to response granted a treatment patients by now both expanded previously living requested EAMS for programs Japan, and for to IOPD mechanism, the now EAMS the announced many increase. that see framework that France, a world opinion with and requests programs two significant This continue continue access countries to number U.K., in around Scheme positive access of We receive U.S., for Medicine the multiple Access ATJ treatment. U.K.’s U.K., dozens And scientific receiving novel we continue component to MHRA. enthusiasm Early with LOPD

programs. are extension approximately trials centers to access grown scientific these our report patients access experience programs. to in clinical growing, our pleased And and worldwide Bradley the noted, in now being opinion to access is be able eligible. studies AT-GAA AT-GAA are and With and to XXX approximately momentum in are expanded Since treated those participating December for has with currently growth XX AT-GAA we now in as we that with provide worldwide with this are pleased positive interest access programs who across

of presented Moving growing of onset label meeting onto from slide study long-term the Pompe late extension and of open as XX, efficacy the body the positive as evidence Bradley supporting III Disease. safety just mentioned, of global propel a part we last Phase world AT-GAA AT-GAA week in at data

results clinical on these key here two together longer-term people presented distance and treatment and I/II the seen a LOPD. of year these on and studies the with importantly side, long-term for latest after six-minute seen data in four effect As consistency years data patients endpoints the with and these and of demonstrate FVC, durability benefit even consistent are Phase support really we’ve living our the from walk

continued were ERT- On the we results glycogen particularly [Ph] AT-GAA for label We naïve as on switched slide well. and effect reduction of ERT-experienced Abecassis who pleased also here, we’re the very large open galactosidase-alpha participants suggesting positive damage, storage results tissue, for extension. showing and group the the key in by the And subset from the muscle a the muscle in similar biomarkers observed to patients. patient of

body our growing confidence care studies new Pompe to gives living This long-term that AT-GAA Disease. further for people us clinical has of the potential of all evidence of the from with standard become global

With would Daphne to that, guidance call Chief to Daphne? our financial to Quimi, review Financial like turn over our the now results, and outlook. Officer I

Daphne Quimi

Jeff. you, morning, Thank And everyone. good

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

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

XX.X% Cost the increased as expenses full GAAP $XXX.X a of goods prior as to the percentage was for year net XXXX in compared compared in year sold of $XXX.X million operating XXXX. Total year period. XX.X% the to to as sales million in

and therapy Gene expenses On year reflecting as million the reprioritization to portfolio offset fair a changes expenses share based value, compensation expense, were of operating depreciation. XXXX and research by in We define non-GAAP XXXX decreased impairment compared million loss contingent for non-recurring of and non-GAAP $XXX.X to SG&A program in full $XXX.X basis, excluding consideration on assets, operating development as the total related expenses expense spent.

year for to management, loss net was share in by for share, loss $XXX.X Net of half compared Galafold continue this revenue our the growth full careful and $XXX.X we year. of million per a of the the progress $X.XX or expense path Driven make year to as period. profitability $X.XX the per prior second XXXX million to or towards

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

defined half on to the timing launch we financial including, operate our and continue to factors, approvals course, profitability is press a Turning position XXXX of our to as of slide strength, of release. non-GAAP the Profitability second dependent now number from goal of remains XX, achieve a and of in in of AT-GAA. the

XXXX operating expense in and $XXX Our continuing for compared and to operating achieved expense by of to prudent full year in is million million. non-GAAP activities to as global management efficiencies investment for guidance expected The expense and be XXXX the Galafold XXXX growth continued will $XXX decrease drive AT-GAA. launch offset by prelaunch

for costs We to also of certain non-recurring will manufacturing the support launch incur AT-GAA. global

at We support our execute to Galafold. be announced to and G&A look market maintain of of our a expect or under portion fashion, fund of AT-GAA sales as launch the continued look balance to the operating to growth we to may strategic year, to program and cash XXXX, previously as strong reallocate see a our we expense operations. allocated ATM our resources In larger we equity appropriately a in this judicious

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

is our of million revenue addition Galafold rates, $XXX growth to to guidance million. $XXX year at constant XX% exchange to operating revenue expense guidance XX% of full non-GAAP Our in

me turn let closing back our call over remarks. Bradley the for to With that,

Bradley Campbell

thanks, Great, Jeff, Sébastien. Daphne,

relentlessly can the on across been you business. focused have As execution see, all we

value from believe of in continue growth operate enable to disease of we a to us the position remain changing the on half of and in we profitability which Fabry people And path We therapies franchises and continue Pompe and our deliver the to advance create to life this second year. financial strength sustainable in to long-term two need. Disease, can

open can we the to now with call that, questions. Operator

Operator

[Operator from JPMorgan. first our question Rama Instructions] comes And Anupam of

open. is line Your

Anupam Rama

the taking Hey so much for guys, questions. thanks

the regulatory here, Sanofi XQ, are Thanks launch the decisions you launch some as coming about at similarities AT-GAA highlight next key the here the few that think we in differences so and you as much. years firm? With learning look what would

Bradley Campbell

were comes propel question. the Yes, long-term down studied. be I/II back really able thanks, it Phase and for to III just well present study to us excited extension and Anupam the the the for as as Look, to our data population on-going data both the really from there. we the from We study Phase

continue physicians discussed that and for just and line key are replacement differentiator excited and showing a patients impact timing as launch be new that to approvals to be for particular in to the we to as enzyme those expanded a to from of have that is we And the demand the going think of we We can able look forward options. thrilled that call switching treatment AT-GAA. sight therapy really to we and And access, U.K., mechanisms clear as patients now move all the at we’re believe have in in quickly then on to to the to

Anupam Rama

for so Thanks taking the much question.

Operator

our for next question And Ritu moment our next Cowen. comes from Baral question. of One

open. is line Your

Ritu Baral

the in Is how all booking access about to course, your we model of one, morning, And it world, the Thanks. inspection? guys. label approval the The is, my fall how taking after kind Of a fast itself after these inspection? question know, thinking has and commercial extended we I we how of start Thanks will are patients should Good we thing? the week expect guess? the approved be the programs question revenues three fast XXX under changeovers that from finally? across to them parts question. should for

Bradley Campbell

try Ritu, those. to three hit all thanks of I’ll Yes,

is through that two takes to team’s months from process. inspection that the our timing What one approval is to U.S. go to in about first relates The estimate

and we’ve why that’s QX, so inspection the in And expect QX. approvals is set we in

we XX has that execute trial, saw we able within that clinical to, been And timelines on were or commercial product, to expanded with Galafold. converting In to those days. our same from terms And goal of access have we patients here. the goals do to

within studies, In terms patients. again, continue that of that But to I a them of I of a Phase say the majority is be grow, think there might very think expanded be portion, to would the who would that XXX, labels. programs III of kind between will onset then the access fall small I/II portion are onset and small study, anticipated numbers infantile patients of vast number Phase we be the patients within late who going

the of all So certainly those and of three the I not think majority patients and would Germany. first be eligible. are countries, course, happened. U.K. the them large in U.S., And, But of majority

to Europe, a balances distribution in Kingdom, the XX of United of in about United the and patients XX States, Just between the those is give rest you about are in the world. XX in flavor regions, about of the

gives kind of you the a So flavor hopefully of that numbers of patients.

revenue And point, But job this this the patients as I and patients important well. a then in then contribution course, hit extension with people our as year terms cautioned of is are appropriate many you really as is onto of think who on-going think the that on will to year. possible course, of be expanded modest which I revenue. with Look, access is new studies, commercial AT-GAA, we’ve but the to put what

patients you of run on going is focus maximize number into the a strong rate And that really gives year. next So therapy. our

Ritu Baral

Great Thanks for questions. the taking

Bradley Campbell

Thanks Ritu.

Operator

And Ahmad question. next Bank question next our of America. comes our Tazeen of moment from One for

open. line Your is

Tazeen Ahmad

Perhaps a Good taking you simple question. for for thank my me. morning. just Hi, And one guys.

that team the Department has to who So infection has just that the for includes the Or it, when of Thanks. technicalities to they that? kind some clear that does over, be State wondering mean scheduled, just goes? that there you been on needing example, does process send decides want pre just are and say to that FDA what

Bradley Campbell

for as Yes, understand point thanks from is usual, FDA, this business, kind the the this what it, relates to At the of inspection. Tazeen question. we

feel So execute what they is they’ll members we the inspection for going inspection. number upon reasons to, To an our need, we’ve have underway. From visit of of course, be things. understand, process site give scheduled who now. are have But been kinds those we pieces that you that a that very able example, who haven’t confident we’ll to and we there China the of now for own now years an able get to discussed and to team all to

business as in it’s [Ph] are usual. requirements by in very that we’ve know is where seeing own understanding special But them should point, it’s what’s And what is be we’re there’s days, at you we think going those we course, to competent of to this from usual, our happen. So caveat never and business team, I no for our seen, able that as execute what order be to inspection.

Tazeen Ahmad

thank you. Okay,

Bradley Campbell

Tazeen. Thanks

Operator

our Merle moment next question. And will from One our come for question next Ellie of UBS.

is Your line open.

Ellie Merle

the get Lumizyme your say like their they [Indiscernible] guess be whether I get I the physicians [Indiscernible] Hey how relative in how be should or switches work, guys And on from thanks from switches so to potential given see or we guess might to then U.S. pre AT-GAA maybe much about in of think the for whether uptake taking the has some question. Jeff, commercial the say called in in just Thanks. what’s it Pompe and often to from whether been feedback patients it patients approval? Myozyme

Bradley Campbell

can interactions Ellie with Jeff And I’ll Maybe Sure. the to the of physicians frequency stab one. kind patients. then the take a and Thanks, first then at and talk of for questions.

like anticipate think patients label or for United therapy. experienced I in feel So there’s need where of an we for I any think, our, a patients. new in targeting, opportunity the target be we physicians launch our try would who would States, a or We to terms segment where

the the that as again, is I enzyme is both have and mean in physicians could would studied that highly population. the portion, that are that are we replacement from we And controlled we be unique us, the data the across data on most for highly so differentiated the And and Nexviazyme that a compelling And only to patients well patients focused on will those patients. just, for who switching study thing lumizyme the controlled with patients think therapy therapy phase. during another as

to want talk about a kind little the you interactions physicians of do Jeff, between and bit patients? frequency of

Jeff Castelli

Yes, Ellie. thanks thanks, Brad and

patients they’re will physicians, when patients kind that months get But not are if how infusions their or they exam of see getting typically depends workout may may they’re they infusions. them the clinic, every or So more had. see months, larger six the really ones the comprehensive doing six every the sense from comprehensive assessment. doing is last a of at the atleast a regularly, if Obviously, pretty it they

is Brad So call new XX treatment, are about consider or they’re first started of enough if or make whether the new doing call mentioned changes, treatment would go a well about kind you they and patient might a treatment. on they before probably they’ve as patients and know, months final thinking physicians

in next now seen, treatment from the a patients at But, we’ve as that least design have studies the U.S. for the on been that over switched many to of year. much

we switches available of a I to need an So there’s from are have from think perspective, the perspective expect currently. products labeling from potential a both certainly opportunity that

Ellie Merle

product. distribution just of some Sébastien, of maybe give And the for of the us recorded a sales color

Sébastien Martel

the last of Yes, at add Nexviazyme, XX% within from U.S. the XX% was higher Ellie, to myozyme, that the XX%. of continue looking the miles just the the a be that actually sales also basis. speed global reported Sanofi, on by to, we coming majority of than quarter fourth for on Outside on in to the at were myozyme. look And to on miles quarterly is U.S., world as rest sales XX% this vast a sales of coming lot feel from

myozyme. still of likely be on patients time we the will So launch the majority by

I that switch quality have switch weeks. the think well bodes for data we

Ellie Merle

Thanks, Sébastien.

Sébastien Martel

Ellie. Thank Anything you, else?

Ellie Merle

for that. Thanks Perfect.

Operator

next our for comes And from our of Schwartz question next moment SVB question. Securities. One Joseph

Your is line open.

Joseph Schwartz

for you’re thanks supply I the of wondering Hi, accruing? are the could strategy much on Pombiliti continuing any And of building used? in for any supply? that How should launch build there your And readiness was be supply? what’s your terms manufacturing is update. launch to reason of the you you comment not if ahead

Bradley Campbell

thanks for Yes, question. the

last noted and an to AT-GAA year financials, investment in there. continued bit our prudently I think, we highlighted I have and we manufacture Daphne of think a quite So

launches, supply products So that. our into, We country advance. for move track feel in about significant we have we’re all of really on good to typically anticipated

to the testing in is there. here well. with about being we’ve And Pombiliti, as now of then products And able around likewise, start Europe, process so release progress States, which ERT to think move begun talked the I positive actually United the we’ve in

we So good about inventory. feel really launch

launch. described Our of which overall supply the of have think before strategy and or supply support we two coming we’ve to plenty China year first, is of capacity I out

partners the that coming XX And sort our the standing to the already well. have would in facility with in of that I before, to come we, in mentioned as timeline. see WuXi of started world the really on Ireland. up new expect the see rest of XX, capacity you’re think, starting and meat countries balance online, And in of be starting of to process Europe we’ve However, when we board as some

the long-term think, manufacturer I the become the will of that primary so for source product. of And capacity

feel and we good today to globalization support clear about are really product of of to continue So grow really how with the where launch. the plan a

Joseph Schwartz

feedback Thanks Brad. it was general And previously, that The saw you done what who all, from in reported hear you the [Technical world? and change? at could all Difficulty] it physicians interpretation, if that data does did how

Bradley Campbell

present question data, of the you as -- the response question world? value, baseline. I excuse perfect the And way what think in second from part was, from it Yes, bit, predict the stated was we the question me, was, little at first and a the the we what displayed audience change presented predicted present Joe, then versus the

and the one. to the then on comment ask And I’ll Jeff first take then first. one I’ll second

as was Pompe I in to can overall, and at of activities you be So person with world it patients, great think in both physicians, excuse me, tons with imagine, Fabry.

World into present makes And to specifically you activities Joe’s data Conference really just program. in the change that was receptivity I having baseline from continue Jeff, think teases interact around the really the to sense. pre-launch coming third up see quarter. predicted want the our chance versus again, why overall was to exciting person think the and energizing. really fantastic. out well it was to And headed from I and at continued talk being question for new And a about

Jeff Castelli

Joe thanks for the Yes, and question.

minutes. age. So how you six kind to that standard height, they account you and walk of of the as percent years for account importantly, And go over durations six far minute predicted aging fact is, percent like for that patient's accounted be in pretty in follow two walk over certainly the should minute it’s six things walk on patients calculation four years, predicted use should but takes up, of into are to longer

data, meters So standard the very report over minute in I But long-term sense. world, longer-term did predicted, walk. percent up. propel, supplement year one we looks see meters it’s more programs, you’ll that people go like a follow predicted the kind way that’s usually use the up, of as just follow to six percent across it think using makes the comparable and We study, for

we as positive reception And Brad think really had I a said, to data. that

very represented, had a year throughout in kind years. We I/II and over years four two Phase four durability in showing propel of the of also here was the the consistent data our effect which

Joseph Schwartz

Thank you.

Operator

one Dae moment Stifel. comes from Ha question next question. for of And And our next our Gon

line open. Your is

Dae Gon Ha

congrats taking on on questions. Good the the question guys. may. progress.Two I for Thanks my [Ph] And morning, if HVGA part

they Nexviazyme different generated is. if that’s as post process have a of based as view your about through interactions, is been that your if physician take the CRE on Phase there fee PROPEL your -- the market evolution I/II, can thinking what wanting or was us you to well Just and and kind of curious maybe walk data research just see next what and

a been What’s Second naïve AT-GAA of little for digging feedback been part presented OLV segment? the you World, sort what's the on willingness Joe’s is, deeper try the or to patients? that of maybe naïve on now data that receptivity at question, physician the have sort

of I AT-GAA, separate how be the contribution much guess, on kind then understanding the variability year. sense we also from I that non-GAAP and question modest revenue half makes really second think this for of given wise hinges that about about hope pushes pulls that throughout if And But Daphne, progress? profitability we thinking or should

Bradley Campbell

of Okay, the for first questions, all. so thanks

post, And at, about profitability impacts both patients we’ve launch talking then and So non-GAAP AT-GAA showed patients. the year. guess the of the at one of pre different switch I impressions around naïve for goals Jeff, last second a the our half segments question revenue Daphne I data of we to how for guess, then next Lyzyme. world, little of more and got And bit sort

first one, again So, in it I I as then I’ll the call and relates said the honestly, to here. highlight

a that’s regardless continue a only of next of of the that AT-GAA. I from is physicians. patients. think those kind presenting critical data what that to the I in study, on replacement difference. have anticipate, control were arm just as of, the information think we in that the be highlight we And think as the that’s happens experience we’re that Lyzyme we differentiating will our therapy to most a side. switch as that’s, labels the one company hallmark in pivotal have is and to I to And, have switching Again, sponsored patients And of and well frankly,

course, consistency those of the as Phase And long durability see were that study. to two data, the Jeff year thing, we from really PROPEL. the other was the and I/II four of pleased The reprise as the data then the highlighted see of data now from we year

is durability really think I and So also that consistency important.

about long-term to the talk Daphne saw get naïve we the and can segments in do also patient Jeff, you extension, question. impact from quickly financial to then the want the

Jeff Castelli

Yes. And Dae question. the Thanks, Brad. thanks for Gon

and the the than ATZ and better XX% experience So other studies, the, show as even least gains, Lumizyme quite up patients we naïve benefit arms similar perform in very basis they follow your naïve stability think and we patients in only they to by were in a saw. about naïve. on large about other where arm [Ph] encouraged arm any continue of then we've the seen and reminder, the had. saw XX% increase we was We that year significant patients PROPEL were of kind naïve from is as at were additional And increases we saw both

combining AT-GAA sort quite and naïve year patients, together remarkable results, of two of increases. or and AT-GAA Lumizyme in those are the really the years two So at terms

No baselines, true small original much that both or patients groups FEC, decline. see whether of see that sort decline of had a because was to not in higher we stabilized, did that But these good that six-minute kind improvements and were It six Phase that the [Indiscernible]. had I/II naïve and and four And years. and robust walk for FEC for minute maintained saw walk four and improvements patients. both really citations seeing and stability in we FEC years, his we’re after certainly was

said, XXs. I that as our in I also physicians net, net the over think would to of naïve to certainly the then is data, the patients. treat carry the the more data So benefits difficult And as year the bulk there at our assume data supportive. And he is think looked they experienced, Brad observed

from in believe of but could physicians, think positive proposition geographies. naïve. And have play different obviously, part terms offer naïve. I So feedback in good value we a for value We prescribing the generated a we for we that experienced for we’ll proposition and naïves, both are a see labels where great

Dae Gon Ha

Jeff. thanks, Yes,

will, product revenue region are AT-GAA just would positioned points do I contribution look important. quickly how Of on the this labels profitability the and is remind final course, the folks all which But depend of want what like. I very Daphne, speak that to to to goal. course, of from think

Daphne Quimi

Sure.

a revenues profitability, just from also have just profitability costs do a approved, want definitely that everyone remind the to So to the contributor P&L. that reminder impact, remind was production that AT-GAA but I will to help the the metrics, will once modest to just revenues. cost, actually everyone biggest that the move Galafold

So from sheet. OpEx and on the move balance to

that. of I of it’s all a combination think So

P&L approval a different of p, the So impact has just in the not yes, it’s, it couple but ways, will from…

Dae Gon Ha

thanks taking question. for the Great,

Operator

for next question. our moment One

Cantor will me, excuse question from next Our of Kluska Kristen Fitzgerald. come

Your line is open.

Unidentified Analyst

taking our everyone. morning got on We’ve Kristen. Rick question. is for Good this Thanks This morning. one [Ph] for you for

preparation? the scheduled of clarity previously you now calendar? doing is mock from inspections mentioned WuXi at what And agency the there inspections. inspections the any up based expect been that on the part that you are facility or inspection from in You mock on what it on real planning Have the lead heard any the is to Thanks. to

Bradley Campbell

Yes, great questions.

Last China, very United then mock a to the successful was ground, inspection. the and supported was in States. able a we were our had from who team year, actually, we So here consultant who by conduct on

other So we that points of feel I outcome confidence manufacturing. status that add in us really about And the WuXi inspection. would of few in the a good give just

this before we’ve for that, is WuXi, The approved the agencies, both and from multiple multiple China inspected products. in first WuXi, hybrid, facility is mentioned well facility a person in

very mock positive a of pre-approval as of history So they whole inspection Previously, mentioned, inspections done and a we’ve host you have inspections. successful, activities.

we’re up, on As up. the in are we I call, from follow buttoned to help and the a team weeks, said, the coming prepare we continue will and sending or actually actually sending over our make everything’s question sure answer

chunk on talked agency’s as this about during also a chunk, of was a the review big big we We manufacturing. the and a the review, reminder,

of with done to are of We’ve manufacturing part runs under there dozens pointed product finally, at control agency. already the self. rich So we’ve then we’ve manufacturing scale, the answered our quality are satisfactorily the commercial the quality of process parameters, overall a successful product. of attributes key host quality quality for history critical that And the questions that whole that of

reasons, what scheduled those And sight feel So prepared approval. the thrilled for we hope to now the we’re inspection. we one have be line that and really and all we for will of eventual have excited kind of

Operator

Morgan our from of Stanley. our Jeff And for Hung next question. moment question next One comes

Your line open. is

Unidentified Analyst

a was [Ph] taking Hi, Thank you what you. analytical just by for I miglustat? for one. morning. Can risk requested question. And could you thank kind of opinion? Jeff, Catherine EU remind for regulatory there testing that what have is of good just the follow take a any, We longer CHMP confirmatory quick up authorities is us had if for on This it our

Bradley Campbell

Yes, we’ve Jeff, spoken color bit a what do than before? give you to little want to just more

Jeff Castelli

Catherine. Yes, thanks,

in we testing the silico, is was requested data, of something was. had provided all sort What the We said modeling during haven’t we show it have disclosed exactly that to we what to doc submission sorts was CSC we of there for got – these molecules. requirements to have for regulations, a that confirmatory like needed address all was, test testing of actually this measure what we’d the

that track, call on risk we validated clock CHMP be as are initially, that quarter. the delay, to said, second assay. don’t that currently we’re track are, on validated opinion the for believe with during timeline, on for we any month provide app to track the to there And well here we is we So four data in a the

Operator

One question Salveen moment Goldman question. next Richter of our will from And next our Sachs. come for

Your line is open.

Unidentified Analyst

ones. the now of Hi, the have WuXi it’s you Will PDUFA the color quick [Indiscernible] post the on Two question. that pricing any site provide And timeline? the our taking on for for we inspection you Salveen. be street of AT-GAA you Thank updating can by FDA?

Bradley Campbell

thanks for questions. the Yes,

relates to manufacturing. updates it and the So

outcome impact inspection material course. anticipated we there’s won’t impact, our provide there’s as then change long material no timelines, a any As manufacturing, if of that course of in or to the on updates due the would provide we

which and think promise of previously is that pricing follow continue we’ve pricing, discount to As it or to modest strategy relates described would I parody care. our we and access is standard

to to therapy. of receive who best Galafold strategy patients maximize eligible is And that with focus execute Our that number access the on and that’s to to against way we've to found are maximize the therapy.

Operator

our And question. next next question our BTIG. for will from of moment Yun come Zhong One

open. is line Your

Yun Zhong

for question. morning. Thanks much good Hi, the taking very

patients, versus for I versus strategy any XX terms differences prevalence, the that or versus is could AT-GAA? you given potentially few number Galafold? in driven for implication be Do what by approval? did wondering geographic regions of on both ex-U.S. what U.S. can AT-GAA a years launch the U.S. it’s that big sales, impact on there been And XX mainly, was after depending that be So the of think can depending your on disease Galafold, you could for

Unidentified Company Representative

the maybe for sales the current revenue on talk what of Disease. and patients Yes we Brad and distribution Fabry. might for expect thanks about and and bit based and little Pompe Hi, And, a question. distribution of

Bradley Campbell

fourth strong eight the U.S. switches than markets patients seen or We also strong. within of talked France, still trends gradually of year, general Yes, over these like probably some about about, there’s last longest see the talked we’ve population. in for a like, the of, of share of Last market was We’ve markets months amount been we a fair ads. performance the where XX.X%. the U.K. growth, U.S. amenable we’ve the growth in example. where the is, one contributing six very so the had terms the in patient the And more proportion to quarter to we in of new about, but greater naive terms a

from split the basis, a U.S. revenues bit if at a a pricing look you there’s the you, consider in standpoint, we between price on and of Europe. if difference global price in As a of Pompe,

So north overall, the north Europe around XX%, patients U.S. market. standpoint, of and actual of XX% We is in about treated see market world. of XXX largest from rest market greater another the Continental XX%, the slightly XXXX from coming coming coming a of We the the with Pompe from patients. and of made Pompe compares Europe number sales patients of estimate XXX U.K, Europe, we being From treated number for the estimate in U.S. U.S., is that within XX%, patient the the U.S. from than

approvals the that, also gives highlights approval you So European us. that very opportunities are that split some think the geographical overall for significant I U.K. the and

Operator

our for question. moment next Our

Blum from Company. come Needham Our next question Gil of will

open. Your line is

Gil Blum

Hey, and Lumizyme good of a squeezing on up question me follow you Nexviazyme about morning, A dynamics. and in. earlier thank for bit

how patients You Nexviazyme new many guys those all? naïve are you. have any detail Thank of if at on switch versus

Bradley Campbell

on Little hard as I’m have to Sanofi great you believe well we on behalf of I obviously. patients patients therapy And their on United look know patients choice public be to as that speak to naïve for switch based out we for States it’s want a sure and that, there. tease putting that there But don’t commentary. that to new And out.

treatment and the And a to just provide eager so were I options, third option patients. think that that there it’s are for community fantastic multiple for treatment

that to I’m there’s component distribution exactly some tough is, little on. what a going sure of tell So the but

keep also mind something stronger space, Gil in you you seen disease for market more public a under the And overall and unfortunately, significantly diseases we’ve very are to diagnosed is, market. rare need the undertreated. growth those that much in as see entering see We players

you support to raise therapies. to will see get awareness, see of diagnostics coming this hopefully as Amicus So choices initiatives, efforts having market, more patients more you and Pompe to the overall,

Gil Blum

you. Thank

Operator

next One comes moment our our question. for of from And next question Zhiqiang Shu Berenberg.

of open. is line next comes Your our Again, Zhiqiang from Berenberg. question Shu

Your line is open.

Zhiqiang Shu

taking question. the of the wondering that very half can discovery maybe status was that investment significance was Congrats in and your comment continued going the early second I programs. mute. much. on your on And related in on achieving of for you profitability Sorry, stage if maintaining confidence, wondering to Thank thoughts the progress. Thanks I on you forward? the

Bradley Campbell

company, milestone important a Yes. as to who think a a for from really I I Thank that's any perspective, has second you started questions. for now launching it Look, the our company phase product. made preclinical profitability think research

think I really investors, Amicus important our a for employees, for it’s So just for our think milestone. I

the to of certainly means the now We addition be also haven’t top long-term, a revenue of line see our grow. with what prudent And hopefully to, but to second that financial given product. continuing in yet. guidance continue ourselves but And seeing use we our resources,

of So is, looks second we’re really for year. half closer like about course, to the excited in kind as the XXXX. But getting there, what step we’ll which more talk we that of of get first future which, this at the

the sorry, I in colleagues Yes. yes, then, on asking around And second was question my programs. just early stage investment

right amount judicial revenues. we outcome expenses, a between of the it’s this we So to is the now, support goal is have have bring of in of there, we so what then said revenues, those profitability, investment. And the difference that an very that year,

answering I However, each think across three we key programs. of questions, those are

of of Fabry the of the goal of be sort We year what over Our vision and important sight excited I line trans you’ll a of effectively better develop for we very again, this begins think very still have the our end to much there in year Fabry opportunity to deliver really medicines is make next those and in us therapies. to believe hear we our Pompe in is the those continue year, programs. those and give to the about would to commitment terms towards course to also disease. next generation differentiated of and progress genes are see continue for part And chaperone

but think a appropriate there right So of more I to now, very come our judicious again, portion investment. it’s but

Zhiqiang Shu

you Thank very much.

Operator

last your question. and have This conference Thank you was today’s Thank concludes you. a call. great That day.

Bradley Campbell

Thank Appreciate the Good questions. you, luck. everybody. Good day.

Amicus Therapeutics (FOLD) 1 Mar 232022 Q4 Earnings call transcript