Amicus Therapeutics (FOLD) 1 Mar 212020 Q4 Earnings call transcript

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Fourth Quarter 2020 Fiscal Year Results Conference Call and Webcast. At this time, all participants are in listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions].

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

You may begin.

results Thank conference Amicus financial call morning. to corporate Good you discuss Therapeutics' for full-year joining our and highlights. XXXX

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

have and President we’ll Q&A, Dr. Research. Dr. of Goldman, for Chief Officer Hung Joining Science Vice Mitchell Senior Clinical Do,

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

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

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

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

on goal call. Great. Thank The X,XXX everyone XXth more the their Rare lives is Annual of results you, World and their public with our February the of Disease people diseases conference to day. diseases. and living this of and policymakers to families than with millions welcome raise of rare day marked rare XXXX XXth Yesterday, of awareness the impact and the the here full-year one Andrew, any

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this accomplishments. press in key release, As did I'd like to we several morning's highlight

patients we launch The million cornerstone Galafold the performance First, XXXX remains naive treatment represents last new including success, the our The across its expectations, starts $XXX above our revenue and both Galafold switch out strong and and business; throughout in revenue. global range patient coming with year. in to full-year continues from exceed of year. performance, continues launch that set the guidance

PROPEL plan anticipate onset XXXX. following submissions other the additional from our of We in remain quarter regulatory disease. Second, AT-GAA geographies in second in of the this and and throughout year BLA X Union submission complete timelines R&D late study regulatory in key on track, the Pompe to Phase rolling results the European

continue preclinical rapidly submissions regulatory to and we to move programs advanced have to for in our the confidence with our time this our by therapy as continue Through in great disease important University Batten approval. CLN follows intend designation our AT-GAA highly most Pennsylvania, for Fabry the XXXX. first Dr. to Within living CLNX its pipeline, we broad our and move well the continue with as disease preclinical and granted USFDA. the CLNX and late toward this from for progress also benefit encouraged prime to research morning, gene EMA gene of the program Pompe forward and we're gene Fast This programs. to Track been collaboration therapy people candidate. We has CLNX disease lead program Wilson our granting Jim the data clinical it designation therapy Announced by

the the Given diseases. highlight capabilities with to we is bring diluted program can third, the data without to have future for achieve And position rare potential to any financing. need to-date, self-sustainability sufficient this people cash collaboration the seen is the and this second living Amicus'

of Our continue with continued curative milestone, people growth and medicines as potentially revenue to this our growth, prudent living realize us expense we allowed new for around world. groundbreaking vision and delivering reach potential rare diseases the to management, important has

our COVID-related reflects strong in year. key variance strategic part million $XXX $XXX to strategic including XXXX, that in disruptions our existing the with million our, and to year, medicine this to precision some to set of latter new our momentum product on with into we'll achieve which for early again behind continue this living X, more year. We Slide precision demand global priorities which look drive XXXX, Fabry. markets. continue on this a for saw priorities medicine Galafold, revenue five this the We'll key despite people Fabry well We're we of globally, minimal track Next, and for disease Galafold achieve to

regulatory our Second, AT-GAA steadfast people Pompe to the as as remain commitment for all possible global in benefit and of with submissions quickly disease. we living advancing passionate to

build discussions we And therapy carefully Global all our we programs position, Center Philadelphia. investments world-class and our Third, gene Gene multiple a be we we're remain stemming Fourth, our milestones. Excellence pipeline. financial our strong capabilities advancing manufacturing and technical portfolio, regulatory and gene clinical of across addition, all disease rare our gene Research as the we're from capacity manage operations in again maintain expenses programs. Therapy and advancing in in progressing development, We'll industry-leading therapy to and to major fully manufacturing through five, continue to support funded therapy

Chief the officer Operating So over highlight with the and Brad? let to me Campbell, that our now further Galafold introduction, President Bradley performance. to hand call

Good thanks, everyone. Great, morning, John.

was progress. business $XXX.X through demand, revenue we the the States And product XX% As breakdown for during of full-year driven to the be give from we our favorable strong continue revenue of or the and to Slide and of us of generated by within or the XX:XX the total business. full-year revenue again, to outside parts grow roughly $XXX.X Geographic was dynamics, would mentioned, more for year. United and as Galafold coming global snapshot our continuity. I'll we of the in continue walk reimbursement $XX.X Galafold that United split XX% commercial remaining million expect X, States, On million, performance both John the detail patient million now

our now business another with quarter; major resilient Turning incredibly of patients to the added all QX be was to continues X, Slide strong in markets.

slowdown COVID with new New the in and quarter, highlighted of starts, in to did Year. lag which of As some patient some a Galafold into we time previously fourth the continues led see that resurgence initiation treatment in the we

supply remains though Importantly, chain fully our intact.

to most the substantial means of Year. continue majority telephonic, Galafold our have geographies and And able coming key pre-COVID confidence digital, can interacting importantly, their and with of a has team customers access through a they other of achieve we in-person, add physicians. in to our Our into been their field New patients combination touchpoints

and From But to Of and monitor we deliver is impact we're continued growth year, expect duration. news we'll on we guidance, and the our confident impact and will the course, good the going to see forward, includes COVID you from exchange. this continue today, pandemic's true operational compared a From perspective, sales increased last increased that to foreign recede XX% a the benefit X% sales year. by which can perspective, see numbers performance from XXXX XX% throughout the year. XXXX,

metrics last On Slide year, quarters mentioned we've versus while same and launch from growth side, on the the countries. past Galafold quarterly patients. continued due several finished the These now and Galafold typically of we've growth drivers we foundation past variety of where several up will new to treated quarter-to-quarter opened growth with our On the share on rate which market out X, starts XXXX, of It and represent strong patients quarters patients XX% global expect from for about X,XXX over called is as XXXX. over show X,XXX the momentum with factors, over lay several to in continue of a amenable a we the nonlinear. left-hand in performance calls, we

longest, we're while countries achieving been Galafold. the of to higher continue So we've to in market there's opportunity plenty still switch where patients shares over to approved

significant that we've who mutations. also are to that of diagnosed We numbers XX% a global switch, who about know untreated treatment. now And XX% have are and there amenable see patients of naive patients you of mix

said rate previously, naive that see few the expect to XX:XX, long-term, the patient we've over As next years, be favor to of of growth. percentage that to expect see patients reverse and about in in to switch we naive we'd

well payers reimbursements perfect successful States. granted nearly Galafold. track been underpinned rates has stand payers, belief The that with continue Galafold record of and on by to XXX% that a is patients who go of reiterating U.S. exceed our the negotiation recognized also and XXXX, of of and reauthorizations outside of United in value adherence those renegotiation insurance XX% by Galafold that All compliance with

our on access Galafold So relentless focus continues. to ensuring

Slide Turning XX. now to

Another important Galafold driver geographic of continued is the growth for expansion.

access sales Galafold XX received Luxembourg, Poland, than countries reminder, around those XXXX, we In Iceland, of and Greece in expand In patients five expect commercial to we of look globe, around additional in and those list, continue added with multiple add at today. new XXXX, we over approval to a Argentina, regulatory least globe. countries with that As the countries Galafold. XX reimbursement to the to list variance including the Fabry markets more a minimal for to as has to

$XXX our highlight headwinds guidance Despite Moving of million worldwide already demand with recent for never the multiple we've certain in mentioned, Galafold cues that geographies. of double-digit geographies global our sales, COVID-related has been Slide strong We're to track I'll $XXX in confident ahead year. stronger potential record growth new now XX, Galafold full-year building of the year-on-year. for in continues our million patients to guidance in which

starts to patient second this project impact part the As as will guidance, net expect year this revenue that in ease. be the to and and year than half growth corresponding be weighted we greater of XXXX, patients of in COVID to new even continues the we

as nonlinear that well we sequential as dynamic, years, relatively flat. growth quarter-to-quarter growth this the expect to in seen QX Given from be patterns in previous ordering QX we've to

with XX, we're Slide we us can on several XXXX. years performance Now million of path to on confidently behind in that opportunity sales $XXX say

$X goes towards years next funding which billion generate over period. long over that the three to expect We and way cumulative in our a R&D OpEx revenue

We confidence we diagnostic also driven growth have other High in continue from significant in billion by in newborn see at continued and initiatives, even Fabry diagnosis screening as the we're which as revenue market well. Risk further $X the investing to peak, opportunity globally, Screening,

posters States. presentations I'd that a that we new two highlight sponsored using artificial the exciting get didn't and of Fabry great at you United idiopathic intelligence new February the and screening Fabry in populations as Meeting see in you pain in if initiatives here diagnosis to well Germany, fact, work chance, for some improve that as the In LBN World recommend AI

therapy the the book continued protection listed highlight exclusivity through XX and provide to me provide to who pipeline. Castelli, program into long-period U.S. access to globally And patents that give of Europe, turn late XX Dr. which our AT-GAA orphan Chief have over call our Jeff? we to IP in the in now in for to addition Finally, gene Galafold opportunity with come. us let Development will a Jeff so Officer, XXXX, ones that, our XXXXs, and a our coverage

good Brad, morning, and you, Thank everyone.

Moving for on with updates to therapy on we'll Slide Pompe our R&D next-generation start XX, our AT-GAA disease. novel

unmet recap provide it's data, in a comments, Before significant important recent remains today PROPEL Pompe. need recognize the Phase the to John's that we of and earlier on X building

of natural on we've Slide recent followed human publications of long-term is initial individual decline seen highlight continued measures by In the disease. addition treatment multiple all here studies the of to a at in world Pompe study benefit is tragedies, that of in Highlighted were Spain, with disease, from Therapy ERT the from history Taiwan, Pompe very XX, in sustained first patients shows key Germany XX and a on Italy an for least Enzyme the initial years Replacement real outcome secondary years. patients the approved measures. on adult benefit study three decline multiple who This with that few

and well ventilatory walk need FVC in of that for you most for weakest patients. Specifically driving these the actually Pompe the is see treatment death over the as go and the for highlighted additional to six-minute that frequent here as options insufficiency is are can time. new efficacy consecutively the respiratory support on Doctors for lung need in cause capacity, note recognizing

PROPEL Phase a trial. only clinical population in disease key Before ERT controlled is we have has as data, the ERT go of With point experienced that XX into we'll the move the X XX, setting treatments. studied been Slide our remember a in in onset from here data ever review to that on other been to described PROPEL Pompe key to study. that are some naive controlled participants to the Slide late background, the All studies

enrolled countries, all treatments. sites at a XXX reminder, a randomized against in the patients have their disease and the As approved in continued XXX ERT adult to and and extension as XX only the AT-GAA therapy. completed treatment XX switch enrolled XXX We was safety assessing of study voluntary currently receive the study, modifying this AT-GAA and efficacy naive participants in study double-blind patients

ERT alglucosidase did AT-GAA experienced the between patients difference reach outperformed on the overall a those significance population ERT patients and difference XX with in six-minute superiority. This naive distance, on groups for endpoint alfa meters. of On primary of of walk statistical not

was proof superiority predicted first progressive as this and on versus However, in alglucosidase X.XXX. P significant for FVC, of percent population really a forced of capacity approved, difference care main the with and pulmonary of upon the impactful cause is is standard key earlier, of X% difference or the Pompe. mentioned showed a mortality alglucosidase a nominally AT-GAA secondary a overall and was function meaningful which in vital finding the clinically alfa a alfa value endpoints, leading endpoint of This loss the statistically as

patients reported patients XX, We XX here on outcomes I the who favor the the difference for seven patients alfa those saw alglucosidase alfa earlier. of function, we improvement to presents compared key and a experienced in and percent data experienced biomarker the switching the have therapy alglucosidase a a also walked alglucosidase FVC overall years these both ERT alglucosidase Moving described ahead patients an on that muscle that alfa patients and data, to function, from the farther Slide ERT X.XXX. with alfa data those of of remaining strength, primary here in the top-line secondary of the experienced for and with similar domains a can patient XX into P meters and motor value X.XX. decline to across remaining ERT publication from been saw captures AT-GAA biomarkers. grouping And in approximately see the pulmonary you data most and predicted on efficacy average to AT-GAA X.X% alfa a endpoints of switching populations. P an to equal In summary on alglucosidase mentioned totality in AT-GAA, of XX than Slide

non-inferiority study to onto that a placebo, to approved first an remind versus product bar approval not for tested Moving everyone approved of PROPEL requires We the second of and want demonstration the that therapy, Slide superiority XX. only generally therapy. for

non-inferiority nominal of six-minute walk key therapy. Slide On of endpoints approved and for endpoint reference. the earlier, we primary over post-hoc secondary first on of endpoint AT-GAA just first superiority the the show here key secondary XX, FVC analyses shown FVC as demonstrated As

we non-inferiority walk, the a these analysis. was hit need As the historically significant in to analyses superiority, six-minute conducted six-minute if of was analysis not test you but be improvement pre-specified indicates can SAP a as Thus nominal not FVC on demonstrate this And FVC. and walk are alglucosidase because on SAP, also was six-minute not default did non-inferiority of which interpretability analysis endpoint pre-specified see, superiority significant. The alfa both did missed impacted in for for to the which non-inferiority. non-inferiority on walk be was non-inferiority highly only performed by

here, shown six-minute clinically reasonable see a non-inferiority landscape demonstration for across data, of as Based clearly XX, post-hoc Slide in However, the position is range in the treatment of disease. leading having AT-GAA moving of totality Pompe walk on apparent the margins. a to we

Here we AT-GAA development, have strategy our running manufacturing next and regulatory, program. the steps for the

BLA to our with quarter in submission in the of the which rolling the with was module First, be the submission, initiated XXXX. on remains second of quarter the of FDA final completed track fourth XXXX

multiple as access patients for and response programs that the for is compassionate many we in filings in open patients. disease, expanded studies look patients submission we've Pompe considered. label the additional to finally, be all of access children upon ongoing have open adolescent Pompe MAA follow. to expanded our additionally, adult enrolled global to and for And is expected expand expect to study is infantile onset onset infantile access to this half begin year. with requests patients our EU Pompe are children aged disease expected year, with Further being We'll to onset our Additionally, completed this and with clinical expanded second otherwise infantile received for XX, in patients and our use, the known XX

therapies CLNX, XX, given therapy I'll were portfolio our February at Moving Several gene rare Amicus Slide programs. briefly Conference on our industry-leading for diseases. and gene to highlight CLNX, presentations WORLDSymposium the Fabry in now of covering

of Our WORLDS with and and Gene innovative continues Fabry of synergistic at an validates for engineered with program technology, presented further the University schedule to transfer the stability, the transgene Amicus advance approach. ahead data combined Penn

with We programs continue across updates further and gene this progress therapy preclinical to data make year. and our Penn expect

rating in have rights in previously WORLDS, active which of As called and initial The therapy. Disease one primary and blindness, premature from motor CLNX the of learning is of collaboration, including plus we and disease are preclinical evaluations assess specifically On the a speech. clinical just to results Batten also motor outcome Batten using part ultimately physical vision XX, determined reminder, diseases, a Higher to this programs. captured affecting difficulties, of developed neurodegenerative Batten the Slide first is fine which in Unified impairment, Amicus XX UBDRS, epilepsy growth impairment measures CLNX our gene death. seven children, CLNX greater subscale disease in February, skills, with at disorders presented the leads physical Rating Scale, scores of indicate children as to common the most includes progression disease and inhuman it study instrument currently impairment.

based the moderate safety, in on over on stable rate of seen of or was treated point treatment. physical say versus expected that months, As treatment the not mild low the of to no AAV in generally History adverse slide, over children yearly AEs a UBDRS well of dose followed. cohort, XX the almost for impairment those were measure or the increase score were with with to was and right-hand three patterns XX only related of the X.XX favorable CLNX three increase On Natural in patients tolerated the published were majority There change untreated that an of immunogenicity observed. subjects events related the mean

to end look to Quimi and this turn the and for Daphne by the and the outlook. review the study For next With that, would program, like regulatory year. our we guidance financial results, Daphne? to clinical the the call the of submit to manufacturing to plan advance to now over discussions I protocol IND

good Jeff, and you, Thank morning, everyone.

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

as higher further currency is year-over-year measured achieved we exchange full-year, which positive benefited by XX% impact the of operational million On revenue includes a countries as million to Total proportion to a $XXX.X million compared in goods were of non-GAAP The revenue net in in For compared of costs XX% with total XXXX. XXXX. and goods reflected sales to percent to the as a increase continued increase to subject expenses was $XXX.X percentage and XXXX, as research constant compared This due currency expenses was at in burden. of were of study, a prior-year program royalty the $XXX.X support of priorities. Galafold rate period. as investment $XXX.X in year a a Cost million growth in XXXX sold decrease the pipeline therapy sales over $XXX.X for revenue basis, in X%. the operating XXXX. PROPEL operating the XX.X% development gene increased realignment Cost our XX.X% to sold our strategic as well million as of of

million of XXXX includes as value for depreciation. $XXX.X expenses share-based consideration compared and and fair of or and a and for does $X.XX impact a to SG&A research development loss development operating research in the share, $X.XX in full-year We as as reduction per implementation selling, $XXX.X general expense changes measures, excluding non-GAAP of million investment loss expense, and was compensation prior-year the decrease of share Our contingent the in expenses. or cost Net period. net administrative define per

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

diluted financing. term-loan $XXX quarter, to senior path the facility any XX, Slide following future million secured in a we're need to third on our now self-sustainability for the Turning without

gives and driving expense financing to milestone and with this careful well strong savings us continued cost this Amicus both our Securing management. efficiencies, Galafold, a by shareholder revenue with growth market patient advance as platform financial interest. achieved have setting We as terms

fully non-GAAP are already in commercial as from to flat is we products the schedule, development in For for therapy and relatively launch pipeline place reiterate, XXXX gene the gene high meeting our expenses the leverage we while in third our on associated straight other our XXXX XXXX, the of expense and million. priority continue programs that to issuing about we let at ahead full-year global the objectives. we revenue December the the $XXX.X financial Galafold call and of guidance. $XXX addition few at to A cash at million, Wilson compared closing turn Penn. we John Cash, securities work of our And $XXX million were in portfolio cash to comments million operating with support To and our maintain marketable equivalents position $XXX $XXX XX, as total with multiple million remarks. and guidance therapy year, XXXX, to operating $XXX.X with December Lab XX, back AT-GAA guidance AT-GAA remain all to and pipeline, financial discipline that, cost We're research infrastructure me in the to in non-GAAP to the XXXX. expect moving million for transition programs our

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

and I Amicus to from confident entrepreneurs that have business So been that the at across relentlessly emerges lead the passionate with the happy And global as through world you we have led Amicus of the we're emerge us a pandemic, performance continue focused and as will even stronger. about team crisis, unprecedented to will can Operator, am see, questions. who this. COVID take we've on despite great change challenges brought by global the

you. Thank Instructions]. [Operator

question first Bank with line Your of Tazeen of from the America. Ahmad comes

my morning. Thanks Hi, for guys. Good taking questions.

Hi, Tazeen, good morning.

John. Hi,

with they us And will do to treatment, expectations the it just they in naive market? type you that around that we're expectation to how the same competitive Just switch some then So they May your Neo. Is that for second can Thank how upcoming relates label can XXth through time? patients? the that inclusive And us to glad explain for think walk a of it you label a on completed terms the the if scenario maybe Pompe about you of to we end-up going depending your up they're catalysts PDUFA coming of full meaning see get first landscape And application for portends competitive get? should be you. as mean that's a label in How if for landscape? is what think patients. would just

course, of what product population. much majority on regulatory can't the And was pathways; say patients. think significant naive really in a I will switch in very a it distinction I ERT controlled between Neo Sure, studied only fashion that's a its or comment studied in treatment I we

completing So remain regulatory end, the And on to we we quarter. Europe for of beyond. the said second continue as our submission, and focused in the we'll U.S. again, submission by then us, the filings throughout the XXXX BLA end

of program. all our addition to we use for children and In we are that, studies ongoing; in additional clinical continue continue infants preparation that our compassionate

milestones that's ahead of lot activities, in next Amicus months a there's where for and lot we of remain a So XX to focused. the six

think have for are itself vast who years. the dollar been the data of for treated patients plus majority on patients been for our the ERT in of the current years, approved particularly who number speaks medicine those a on on experienced of billion many market this in have many, I terms impact Pompe

some most And is that the switch the those in the than two who of vast more patients which that now of again, population, have powerful population. particularly data ERT shown on been majority we've years is

community, carry be great incredibly these physician experienced got data the by driven scientists the day. data the the will I've Pompe are in and community, So confident will talented the who and

the line from Your of with Cowen. question comes Ritu next Baral

for on is guys. this Ritu Hi This Margine Irina morning.

questions. help in analyses additional what if differences probably from for AT-GAA, you're pathway CLNX to would at you this can one then drugs and -- the with CLNX to for could the versa. put with gives medical might FDA also I vice secondary year, meetings just or regulatory the that translate And filed negotiate in like, my be in taking Thanks going forthcoming So what question, more AT-GAA? strip profile confidence more what

Sure.

We we on if AT-GAA your continued have we in think do questions ahead expect Jeff, of the what looking additional run and do that on to Jeff, -- continue of secondary take secondary study. there. to think increased phone these data confidence different and But were talk two its want you do endpoints And impact what the as We'll conferences? could medical phone, multiple secondary you the range first patients. people I a one we analyses at in expect? So Pompe, on the pre-specified through comment that, I

John. you, Thank sorry. I'm

okay. That's

you. thank mute, on probably was I

That's all right.

focused secondary it John will As at endpoints. really mentioned, on looking the be additional

key on data secondary the endpoint endpoints. predefined top-line really the primary focused So but

to same supportive that's subpopulations that key into which of also So periods the strengthen same we've shorter the of that are looking from pulmonary all It'll show measures on of the longer that believe these that main continue time. be showing that just supporting additional endpoints functions is AT-GAA think endpoints and continue those we those we've have other. the showing patients motor have more pattern improvements strength, endpoints as we seen we'll favoring vast majority strength, dig of we more ERT of We'll function, what seen. motor the primary see directionality that really and been general changed see data will dramatically But we key the secondaries, that did in disclose anything muscle top-line, measures really see in kind additional in the way don't to nature. those to so we one And all baseline. that or here or

-- next Your

then part to And some ahead. I'm to question, and of if just tackle, you where there key just kind the tackle if CLNX Jeff, want activities sorry, of want we the you are operator,

Yes.

So now follow been CLNX, treated kids post-gene continue XX have the transfer. we over that year's two to

dosing continue of by clinical the up, material kids engaging to start that GMP by decline working Fisher. second stabilization trial, we've the and next part really with the natural all to to on the the manufacturing, with that GMP hard year seen the the the We've of be been regulators look Thermo in excited study versus define at end expected We hear to in really history. next made year

manufacturing regulatory clinical protocol lot leading interactions that a this to to up children. additional dose really and of starting year So

line of Your comes the question next Rama. Anupam from

Hi, guys, call good morning. the today. This is Tessa on Anupam for

Just expansion? penetration from countries of core the on XXXX commercial considerations and for for geographic so deeper what one side what us are expansion Galafold are Thanks geographic much. in the the versus

Bradley. Great, that thank go let ahead you I'll place. fill you. and

Asia-Pacific, this come competitive perspective Middle said, a can perspective. and from most just reimbursement year. I you expecting five And growth North East, the countries specific then America, Africa But geographic tell online of getting Latin a areas not continue from we're to countries. over to we're as be Yes, Sure. I

newer Fabry but those frankly, switch drivers. and launched been to say from there. And I in as would do continuing initiatives only new as relatively way of being $X.X $X global a expansion including part number just perspective, where the two launched. product, find part So supporting third I talked growth of is markets our kind it small, I market it’s think Part just lots of patients I'll in, as countries of are a of we've which individually a point billion passed but launched sales, high-growth there numbers substantial that which to to to today we've is plus then patients countries, also the mentioned is were a become collectively last our it I it's, in about countries, to geographic it's the on to continuing billion that And reported, continue of third geographic third, U.S., too are add the last is The think a Japan Remember growth market. of and expansion. be number well in that. growing year or we to

so to past our forward milestone, path but to confident not dollar to grow billion million towards we also looking only $XXX that continue are that opportunity. we're And that

Brad. Thanks

from line of Goldman with comes Sachs. question next Your Richter the Salveen

enter competitors specifically competitive therapies of Thank morning. gene the and emerge from differentiation here? When some seeing some Fabry for therapy. looking to and [ph] we're Pompe you gene for Thank you. clinic and we're then taking Maybe landscape could for on expect grow areas of we for question remind Fabry And good our updates dynamics is us here. you the Salveen. This may Elizabeth the

maybe at And a with engineering actually released few and it you the And was a to highlighted was we teams in just Hung, again, therapy. just everybody data Wilson ago, describe Fabry a Fabry Amicus very set this what the great very UPenn. at gene a with start the combining developed because Science what differentiated weeks Yes, ask protein experience can remind approach. again, to disease. unique technology the vector Amicus the at and that's by Again, if our and team, is think we maybe I back World Lab in Meeting jointly place November, Jim at experience his again Wilson

the comment Fabry gene Hung, developed Jeff, that turn therapy and then development can timelines. So I’ll if activities you for you've and on it to you

Sure, John. Hi, Salveen.

the becomes it produced as what that from The fall it's a actually gene starts is feature therefore to subunits. engineer reality the together they to so identical say therapy have dimer. that's to our So to And not is two is I to we done and for produced of Fabry that comprised think therapy so proteins approach the is actually protein gene made apart That's functional. it the dimer structure, main distinguishing be need bound be inactive. is maintains actually actually that What and very stable, maintains its transgene, activity. for

stable end And is so that's result potent. is the protein made the and actually more much more much

protein wild-type And so greater efficacy, achieve of therefore, amount producing constructs. to able while significantly the in-vivo same relative to we're the

about over the turn to me of let you developmental Jeff, terms So in timelines. talk

Sure, thanks, Hung.

Those Pompe, other development Those get as that technologies that the timeline Penn parallel into to the close using with One CMC the is Amicus We on two for we'll work there trial. on capsid our optimized the was first next manufacturing clarity GMP across Fabry development. paths the process. there. paths enabling material, making on ongoing something What in IND are from transgene to updates uptake IND IND both described are and good be focused of paths is cells. work on commercial is to possible likely declared based for clinical manufacturing trials our on And we'll the into on progress as great toxicology specifics science IND ongoing. transgenes and proprietary rate is go those GMP more material intend engineered process is candidates candidates on but Hung to clinical for then So as material limiting to programs. and both remember also engineered provide just that really that as the the we have

think gene it's you, Thank Fabry just some I after exciting I'll clinic, choice. Great. whom Therapy with of early we're that to therapy as and Fabry into Fabry applicable for the see, We therapy market, think many to Elizabeth, role. only in their a starting therapies certainly to the be Fabry variants, so Fabry's therapy, gene decade of gene be with Replacement most patients frankly it with minimal allowed in be those approval disease, more patients different very We for Jeff. given years the think patients burdens ahead obviously oral for Galafold adopt for in non-amenable that years, very post Enzyme gene these do we an they see those patients patients add to the profile. range precision do or a will do on there is on different nature Galafold, a a patients technologies coming convenience, its would of medicine would for finally

to look especially in will development taking the certainly Fabry be we programs we the it and do And differentiated ahead have know committed anybody forward field. Fabry we're clinic these So very this approved therapy. to in given for gene We think though into approach. we a robust therapies,

line the from of Bansal comes with question next Your Citigroup. Mohit

taking thanks my question. Hey, for

question one guidance. the on Just

So the growth if the at XXXX, and million, at of I increase. you're upper-end for year-over-year annual a look $XXX even in guiding at looking XX%

about of point this calculates it from CAGR. XXXX $XXX However, to plus guidance million to XX%

if So you. you what of just this goes us could there. behind understand wondering Thank growth help acceleration assumption

queue to do the to continued everybody, that alleviated to we that. again Yes, We you. with I'll growth, patients. post-COVID. of remind Mohit, upfront I'll see being ask Brad talk growth feel see just And demand of building say great thank Good much you. than continues.

year We of XXXX. in expect beginning second the particularly into half this in mid-year, strong the growth

color I'll please. Bradley, to So, ask there, and provide context more you

right. Yes, John, that's think I

see I of COVID second $XXX some think, at And of acceleration the of continue to the saw towards half and expect as of out coming million a that from milestone. we're that challenges last we assuming we get that that some year we year. this do recovery end

launch And to of we're of into rates continuing market the into to growing, overall of to see You dollars the next billion countries, take is new we of acceleration sort on so opportunity going to increase, advantage diagnosis potential that a mentioned lots the this continue I based continuing as to earlier, years. dynamics beyond really few again, all that, a grow see grow seeing. upwards we're as

those that is strong be growth three mentioned know Galafold we to what and And continues on demand stay continue rates. patients we So that focus to continue over compliance XX% on drivers adherence I to earlier. that

Mohit. that's you. helpful, Hope, Thank

with Joseph Leerink. of Your next SVB Schwartz the line from comes question

patient Hi, wanted I'm just program wondering to for based your in just achieve has population on Pompe. heterogeneity Julie [ph] your Could dialing about you gene gene our for for to I changed what taking you in on therapy envision of I for the results? was you your strategies if a therapy the Joe. program ask you this Pompe question plan PROPEL for talk your how Thanks question. program?

the studies to than to and that broadly inform efficacy, more Pompe, Sure, maximal other for protein muscle gene program the in continued and bring in has targeting a in XX thanks, which and more came therapy. safety will remind improve engineer Julie wanted only been but development all years. lead clinical the everybody field we better to [ph]. to technology for hasn't potentially we it everybody products. just the approach again, expressed much believe transgene PROPEL gene well of changed to I'll published say When Pompe obviously therapy reminding our this PROPEL Pompe view to Again, the therapy, by development to different work for that is in not we manufacturable to the of obviously gene allow

we're considering technology, to earnest adults there'll as from we ahead. patient the working now. way be continued in all that regard, infants on the and And is the focus the core as in We're learnings through that I'm move something development sure remains the adolescence populations So same. core program well.

Kluska from comes question next Your of line Cantor the Kristen Fitzgerald. with

morning. question. for good taking my Hi, Thanks

Hi, Kristen, morning. good

with And thanks John for these and for conducting for is with rare your diseases. your team the patients work experience all Rossella sharing

example, us diseases. the indications of already for So as the of in course to natural therapy you the advance were Batten remind continue your understanding Natural you established you pipeline to CLNX improvements History for gene light here is, could and you so disease CLNX question in are forward, have which data demonstrate your I able of there place, think

So, are of end, of programs? you any for the thinking also earlier conducting other some history to natural that studies

on arms broadly, to to Yes, Jeff, these I'll and kind be how histories. for Jeff. you great natural It's and Thank in compared then to question. of to you, turn it want all comment going CLNX both of histories with a to important very robust Kristen. where programs more That's have as care? we development we think CLNX neuro natural are about

Sure. Kristen. And hi,

the there data the in of all of with of supplement collect on natural A natural Batten endpoint. available. Batten it subtypes is currently both the prospective a there to So is depending subtype, diseases, to lot actually study and going pretty, history most started-off Natural across prospectively own history the there's CLNX, natural We're history; that our data, the already we've that collected data. amount existing retrospective UBDRS History already

as CLNX, So for subtypes. X key other CLNX into for a well of diseases part we to get would think path our as and development that Batten be

on And together natural IIIA development there, for ultra-rare working start start look a some but work already the Disorder, to data we're for to at collaboratively earlier As Deficiency these then similarly, think in those to others to community you a disease, History work and less, with it's We we'll some example, to the history. early on the there's is data that's of supplement efforts of tries a Natural key relatively programs, new start development and diseases. in to pull some CDKLX MPS looking little natural and bit that's history. IIIB continue there, part of out that

but gene you look there we will be think Pompe think for As lots therapies. at data, that programs, there's trials needed new of obviously controlled our we Fabry

something but doing. community for on think building focus, early we a already So in less is that critical the that development on it's is address programs, programs front, early little to the obviously, what the

Securities. the Your of Chattopadhyay comes Guggenheim from next question line with Debjit

is This Debjit. morning. good for guys, Hi, Aaron on

interactions endpoint, study and a the different any specified accounted stats X.XX? for evaluation of from as usual was couple on agency quick the FVC the So just any of questions, than second PROPEL level plan considering significance

people remains maybe And the switch regulatory clear end remind submission second again, the Jeff quarter in on we just Yes, we importance BLA what the both on to comment our there and want complete part overall and population the second I to the ongoing your plan just saw by its FVC, just be population. and of don't of the first on discussions.

walk. as PROPEL therapy primary versus so was approved superiority endpoint Yes, was six-minute designed

first was FVC. The secondary hierarchically key

that as nominal as value So with we we had long X.XX demonstrated with other we showed, endpoints superiority key that below study.

We'd non-inferiority. FVC, As actually we we not analyses I that Slide hit on the first need to which mentioned covered we secondary some conduct did as on nominal also of the in formally pre-specified non-inferiority on post-hoc superiority key on FVC.

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

Great operator, thank you.

again, a got us. ahead of of a So We've lot was work XXXX great year.

focused XXXX. mission. remain again in a relentlessly entrepreneurs I still You're Amicus who at But reassure got that passionate want team on our you've of everybody to

So thank Have a great everybody. you, week.

a you day. gentlemen, call. great have today's and Thank concludes Ladies and this conference

may You disconnect. now