FOLD - Earnings call transcript Q1 2021 | Amicus Therapeutics, Inc.

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Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics First Quarter 2021 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Investor of Head Faughnan, Relations. Andrew

You may begin.

Andrew Faughnan

Mary. conference call you, financial and Good first morning. our joining results highlights. for you corporate to XXXX Thank quarter Thank discuss Therapeutics' Amicus

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

joining. will have Vice Clinical Research Senior Dr. President Goldman, we Mitch Q&A, for Joining of

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

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

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

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

John Crowley

beginning of and conference to XXXX call. that the to quarter I welcome quarter Thank Good our that first report the strategic we the across Great. everyone key first pleased at year. am Andrew. priorities morning, of the reflects you, execution XXXX results outlined

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

performance very First, uneven cornerstone the quarter are continued with our the success. and of continued remains strong globally, of first With Galafold on ordering Galafold patterns. end despite headwinds continued revenue, COVID-XX, momentum continues quarter as million of $XX.X our exceed pleased the we in number as at Galafold uptake its of launch well to internal expectations patients the from the the

our pipeline our continues timeline R&D Second, regulatory key remain therapies of to advance. gene for track, and on AT-GAA

with of pre-BLA Following and we in the written Union the and a anticipate rolling complete with successful plans AT-GAA BLA communication XXXX. geographies other in and meeting continue throughout submission of quarter submission this European year, second the our additional regulatory the U.S. in FDA, to

great in disease Wilson gene we preclinical disease Batten Dr. further and data to the University advanced our from programs. people most candidates. gene have with submissions to it as rapidly broad Fabry our benefit pipeline, move to Pompe Within progress our CLNX, and therapy and our research by therapy preclinical and we as Pennsylvania, collaboration Jim encouraged highly well Pompe clinical Through are of the both gene confidence onward AT-GAA to intend CLNX our regulatory for towards continue approval. living with [Ph] continue programs lead We disease, we therapy and through

both preclinical will this programs are ASGCT presented to that at share data We from be pleased new week.

future sufficient position without Amicus to financings. for Third, need the diluted the cash achieve self-sustainability is any

I'm Vice very of for development, delivering team. Head with a strategy, And the curative growth we Sanofi Amicus to mission allowed around ago, prudent management, as as growth, people Diseases to as world. Sébastien to new Strategy groundbreaking Senior potentially We our rare expense this just living realize Sébastien's look us most Genzyme, to continue pleased Global and at served revenue of welcome medicines company. and Development, Our announced potential milestone, as new Amicus's commercialization. to recently continued of finally, important reach has we the corporate expertise Business few global vision our Rare business in and to contributions forward days a & Martel, for and advancing Sébastien biotechnology President patients brings deep leading diseases

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

commitment as as all we'll world-class portfolio. funded as clinical disease again, gene gene passionate discussions manufacturing therapy finally Therapy and submissions addition, of strong we're our progressing to pipeline. global a advancing multiple and of steadfast capabilities five, to position be financial stemming and to programs programs. operations from carefully milestones. our we and our we're Pompe manage therapy regulatory remain our build to quickly advancing benefit continue capacity people Global and our support maintain And manufacturing, we Four, technical remain in we regulatory investments, and disease, major Three, across expenses Center in in advancing through in gene many Research Again, through the our with development, Second, the and Excellence therapy living Gene Philadelphia, possible. for as AT-GAA rare industry-leading fully all we

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

Bradley Campbell

morning and Great. everyone. Thanks, John, good

commercial a six, snapshot now As our I'll Galafold global have in On the the performance walk we Galafold you John quarter. mentioned, through detail more of for slide progress.

detail demand slightly exacerbated million, patient despite by revenue strong revenue to on that geographic For uneven impacted continuity, or United quarter-over-quarter COVID. were total that and of million, revenue And ordering QX more of driven States, and $XX.X $XX.X the from breakdown or by the XX% States. generated provide of the The XX% was was I'll a being United patterns million QX quarter bit by from the remaining business typical outside in moment. revenue a little $XX.X within the quarter, coming product during

continue we As to grow that of both roughly before, XX/XX business. as split we the continue we've to mentioned be to parts expect

quarter our internal business end And of patients major Turning in be added continues now to incredibly estimates. ahead quarter another the resilient of Galafold all of patients was was to of The the markets. seven, growth. slide on global number our at QX with of the

quarter irregular to first patient offset timing ordering increased was of As due revenue reflected United by I States, demand. in patterns some orders and the slightly in alluded It COVID. reauthorizations some ex-U.S. to, geographies,

Specifically, we these as orders in largely have did did see respectively. now Brexit QX, Both the in but some year-end worked to it that system of QX relates through accentuate larger ordering phenomenon. typical and COVID the

examples into QX couple expectation last non-linear fell our a days of of that had also orders growth. in consistent of are with These quarter-to-quarter large QX. the We

patient delays our quarter care of some into in full-year new in identification the the with did between and see we and treatment. of QX, at due call, fourth in point As patient we the of slowdown initiation to highlighted resurgence COVID starts

fully supply chain intact. Importantly, though our remains

is, with second the physicians. other to Our of pandemics in customers we've of in-person, good majority able latter into improvements that team the of And to of combination they the course beginning news first telephonic, the points the confidence Galafold. monitor has but We interacting achieve the means have digital, today substantial field the pre-COVID their can of continue our quarter touch part access and quarter. a been a of and observed impact, through

underlying months second-half we've confident in rolling last meeting full-year and now months, think increase about average momentum the from steady because And three Specifically, prescription pandemic. and and month demand. we're new the before a XXXX it recovery continuing our this Based we've the in requests, saying XX number to what months. the saw last reflects anticipate States, our in in a ex-U.S. really last on In guidance. business, starts of patient COVID, seen to is United since April six important, I a been of the largest the strong monthly this

starts launch non-linear metrics Although patients quarter-to-quarter Galafold be growth countries. again, called with really throughout with On we momentum for several in we year. finished and It XXXX, new the on slide lay X,XXX growth from opened continued which out will up and nine, we've the foundation drivers our expect do that over of XXXX. the where the several

market Now amenable at with about we're patients. share a over global X,XXX treated on patients XX% of Galafold,

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

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

that and XX-XX. to next to in see years, about to long-term, reverse to growth. of fuel to we'd patients before, few we see favor As said of over rate expect continue expect patients the percentage naive we've that the switch we And move naive as in market

on patients Galafold recognized to underpinned Galafold. U.S. Galafold payers track our and XXX% renegotiating, insurance agreements is on who think has by re-authorization been strong payers negotiating successfully that States. well United reiterating of the continue value those XX%, of a With rates and with of that that compliance on exceed is now being adherence of and that outside nearly All stay the similarly by XXXX. granted record We reimbursement belief go

a Galafold Our to relentless continues access on ensuring strength. major be to focus

to of world. look add is XXXX, Another geographic patients traction expand as we for access amenable Japan to And America, and expansion. to In Galafold five launch in continued least the rest important the continued newer Fabry variants we of U.S., driver growth in at with course, Latin to additional see Asia-Pacific, around our countries expect we the and of globe. markets expect

those a and received XX over XX in Galafold sales regulatory approval As has commercial over in countries reminder, of today.

related despite COVID we're geographies, guidance been geographies, Galafold of in headwinds in cues with Galafold recent patients building sales. $XXX never some in full-year of our for million some and certain now in confident the potential to So $XXX worldwide million has stronger demand global really, new

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

IP over initiatives, significant in market successful Finally, growth really in patents our that $XXX we investing orphan confidently in And will even one Europe, milestone listed to through the of on will confidence we turning me, performance funding a our next to return And milestone million generate opportunity are say record and Risk in that period. in next in quickly timing exact and driven us, Screening to from diagnosis provide track to billion $X we further the into continue here. drivers is years, newborn three of over have that addition give with expect XXXX. now peak other things nine, year have orphan path which finally, the And important behind the excuse as OpEx protection in Screening, strong to revenue we diagnostic Fabry several XXXXs, exclusivity contribute R&D cumulative ones we of and which the we're XX U.S., revenue post-COVID, those on which and us Galafold. see lay as in years well. and two globally, to the we by $X how book late that XX out significant continued which of for approximately we And slide can normal High towards years coverage as now billion amount a to be coverage, but dependent this global continue

our many Jeff call So who lots our Jeff? Development Dr. globally Castelli, highlight Officer. of program, to years let the over to will Chief me come. access hand therapy pipeline. provide our Jeff is opportunities gene to that, AT-GAA to Galafold continue and to now for With

Jeff Castelli

everyone. good and morning Brad, you, Thank

to with is today. a slide AT-GAA, our XX. unmet novel R&D prevalent lysosomal Moving fatal disease. severe one our start on Pompe updates We and the wanted neuromuscular most next remains disorders on disease generation to therapy significant need that a with Pompe of and for

majority patients all that to human we've history initial of is tragedies, the benefit a addition a decline few by Pompe studies natural disease seen multiple years then and publications the that In followed for treatment on of of key individual of for patients. highlight measures long-term of

experienced population with in have treatment. ERT clinical control that summary a trial X Pompe in biomarker disease AT-GAA, efficacy unmet only studies a As Phase data. other high line onset top all of has in setting ever presents a Slide naive participants controlled reminder of to need late PROPEL been been XX and studied our the

randomized reminder, the naive treatment a was against adult patients efficacy enrolled in assessing therapy. of experienced As XX and those extension disease. PROPEL continue all and their have participants AT-GAA patients study as the currently the modifying a voluntarily disease approved only in double-blind enrolled and to of safety XX We countries. at for XXX ERT sites study in study on Pompe completed AT-GAA their treatment XXX the

not those superiority. reach on but primary distance, overall the walk the endpoint on XX between which quite alfa of did On population, AT-GAA difference patients significance groups outperformed statistical agalsidase the of for in six minute meters,

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

on for Here that only superiority the and of and patient be not superiority requires pulmonary reported strength, bar the product an approved populations. over second tested the we've overall see study approval the everyone typically data remind into these domains alglucosidase experienced of favor to that muscle both of not grouped PROs, totality AT-GAA motor slide We the or therapy, versus function, biomarkers. outcomes, PROPEL and placebo, for in can ERT generation for endpoints shown you approval. alfa And non-inferiority that function, this

highly the non-inferiority on of and our of call secondary shown key the these in primary post-hoc significant. year for six-minute As non-inferiority first were endpoints full FVC, endpoint analyses walk analyses

rolling have our surrounding the fourth completed to the the XXXX, quarter in regulatory as now our development, program; in on with we submission, was AT-GAA and which of the BLA slide first, for remains submission final FDA be with Moving of manufacturing here on strategy quarter track XX, the next module initiated second of mentioned, steps XXXX. the the John to

access that patients for use for Pompe patients response received disease with patients year both and adolescent infantile to forward in to expanded follow. study MAA XX Pompe patients. multiple this in expected our Additionally, Pompe disease our with access of this expect second-half the and open-label global to is ongoing we clinical infantile year completed submission the EU study And at year. our finally, children many additional expected is expanding with filings year onset compassionate to onset this the the look onset as adult we've disease, olds for or for to be requests expanded our programs begin and enrolled Pompe well have to infantile We XX living with

believe regulatory additional discussions Data of leading activities on history. and programs Data studies the Conference. WORLDSymposium given for our our Gene combining are gene protein potential on Therapy transfer be patients XX to of to expertise programs therapies February excited across to and Fabry disease year, Amicus new when our Ahead dosing the CLNX this focused to the preclinical compared material slide with were respectively. our Batten covering therapy gene to from Conference possible. programs at share the to Society and with from progression Cell XX, technologies. industry remain studies will clinical and at our to diseases, for gene validate to data now in gene gene presentations disease and we untapping GMP expertise slide by American programs that clinical power both gene the innovative CLNX natural Penn progress this the enable these the and lead several Penn's We We these in continue in to just beginning portfolio soon manufacturing rare further therapy therapy highlight briefly children continue make stabilize of Amicus's both of therapy as Moving Pompe from is this engineering as collaboration. we grade this on of show week approach presented of for

our rights over over Amicus review including Quimi to reminder, a to the As like preclinical that, guidance financial has to to I this results, diseases XX turn active collaboration, under currently call and in Daphne outlook. Daphne? programs. seven With would now

Daphne Quimi

you, Thank everyone. morning Jeff, good and

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

$XX.X of XX% operating the at revenue $XXX.X period first of $XXX year-over-year were costs. year. a quarter, non-GAAP For of Net constant in of or XXXX net for last share share quarter revenue changes prior million the share-based compared as the to This per first excluding per the expenses contingent expenses depreciation. the the which as or in batches the reflects compared a as expense, specifically and and positive research growth over XXXX, timing we value million driven Total $X.XX operational by development and basis, the expense achieved a impact contract investments compensation Galafold X%. to in first and costs research of as of were year non-GAAP currency for to $XX.X first to XXXX. manufacturing incur $XXX.X our same define million, development decrease is quarter rates in million period. in exchange compared we currency and $XX.X AT-GAA On of loss in the We total decrease $X.XX $XX.X quarter, expect fair of X% the of the is research SG&A which manufacturing was of increase timing expenses, The research million a and consideration quarters. includes measured of pipeline, loss timing costs, in subsequent and in the quarter million of which primarily first quarter million operating operating

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

slide Turning XX, we now without on a to diluted future financing. are position, to for our cash current with path need self-sustainability the

driving continued achieved and well as growth with through efficiencies, Galafold have our management. savings milestone as cost careful this expense We revenue

$XXX.X therapy Wilson objectives. gene ahead the Penn. already all expect and that lab straight AT-GAA programs in XXXX XXXX associated relatively and $XXX.X and commercial few in the remain To the costs multiple high development we total cash, maintain products priority financial We our place A million For cash at about in programs year for pipeline. fully schedule, position meeting we to on the non-GAAP work continue securities March we the our to comments is and and XX, global therapy pipeline were launch in operating with flat are transitioned as to other XXXX with compared third our leverage expenses reiterate gene at financial while million to cash December research moving infrastructure support XX, AT-GAA discipline we marketable equivalents in XXXX. our guidance, at of

We let call are million reiterating expense our full that, $XXX million guidance closing million million. to for non-GAAP me $XXX in And John turn year Galafold to the with operating remarks. revenue to guidance $XXX of our back to $XXX addition of

John Crowley

Great. and and Thanks, Bradley Daphne, well. as Jeff

all will and take driven stronger. performance global us our happy we emerge team patient across mission. lead even confident by the focused that world to will of have so am across emerges this continue been led can relentlessly the business a from And on Amicus I who questions. as pandemic, we're focused passionate programs, Operator, on as have entrepreneurs, you see, global to

Operator

Instructions] you. [Operator Thank

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

Your line open. is

Ritu Baral

means of current Galafold, or full what thought by I conversion? to guys. Can also us kept Thanks question. drove either it's requirements? for certainly the post Good Thanks. me, Fabrazyme approval these track recent things. the that I promotion for through what you that wanted of I kind And know, ask FDA. morning, the that approval about was unexpected by It walk of you taking and

John Crowley

everybody has that full -- as we very of the experience from X much. was was meet its the Sanofi Sure, available Sanofi, drug, a a don't indicated, market than It continued Ritu. real-world know of conducted on approval. approval to given then their registry the -- not and product really the XXXX. was just data, in that from combination patients. endpoint, remind study think saw what you history under to it that I framework conversion Fabrazyme primary Phase largely the did the Other appropriately approval that but to for I'll led And be accelerated accelerated

studies down accelerated Galafold we confirmatory down X building evidence, path be the real-world the as a a we're continue think For to of could through of also study, also but Galafold, well registration full from as evidence body very Phase of I approval an a road likewise, approval. supportive that converting

no no on been with and be Galafold with for always who marketplace, patients. patients for disruption whatsoever that's strategy. impact. perspective treatment and impact zero no our part Today, So, in has said, that variance, the from would a of It it seen disruption we've Galafold with our minimal to suitable payers, getting

Operator

next question of Ahmad from America. Your with Tazeen Bank is

Your is open. line

Tazeen Ahmad

for Hi, taking question. my thanks good morning,

little impact over COVID. quarter And level for COVID impacted what seeing question wanted to the confidence able of the what about Thank you. so quarter range of a specifics this the meet guidance you're how year? quarter? guidance Brad being any of you improved current on get in far, Just on that the Galafold have if to color maybe of has seen from was for your And get a bit the and first guys is and this that for you to how you've

John Crowley

Tazeen. Sure. feel Thanks, those. let I'll Bradley, you

Bradley Campbell

Yes, thanks, Tazeen.

the United a metrics of thought that, that from new sort six of the following in and color color provided as know, month good perspective, patient we month, well XX think provided I And was in on since call. are we we're been the some pandemic, the we highest it's the kind importantly, where number month this the the of better sort months COVID six to rolling relates than it starts as of So, prescriptions you I which months. now States, the of and three on month that then is the than any better than that before was

things what where as still demand. see know that tells And stable, XX%. patchy but And I just not all first-half. us there confident I that that starts that be in places but adherence the we right that is new your we is out So better. up the we're And would then it's we here. throughout we starting as said, supported that that's how steady steady quickly we second-half look demand guidance be underlying look increasing. dependent over year rates And into think I think But anticipate to very in the know, it's we've is That that are to to being reside XXX. all field, certainly the the we XXX of where will think continue the the And with kind why color of year. continue compliance provided think now higher growth, patient will by end that between and I on and again in of question range to see

very well. as market it's durable So, a

today. where we guidance to good confident to about and we that the we So, here reiterated patients, are, get feel we're ability our really very product in

John Crowley

as throughout patient reemphasize excellent and I'll States continues be add, reimbursement just Bradley the starts, the add. compliance continued to growth highlighted, to just all price the I'll markets. Tazeen, you, new well that the with in I'll demand, in global just demand, and holding addition that United the reimbursement opportunities, Thank near reauthorizations XXX% Brad. patient the

Operator

Your Kluska Fitzgerald. next question Cantor with Kristen from is

open. is line Your

Kristen Kluska

intrathecal gene to And selection, everybody. you outside via I which how of what your and data to taking question. Batten, considering Fabry, when you the route and candidates like items have going ask capsid clinical the is pipeline, morning, transgene, show for recently how on considering criteria the the Good of place especially the is wanted Thanks collaborators one you you're administration? then few are into prioritize administration, one of and engineering since an to franchises? disease Pompe therapy

John Crowley

Great. Thank you, Kristen.

portfolio, much how on select of very a based our portfolio, on the building I'll was back decisions initial go we to approach. terms our In that within and disease

the as Looking feasibility a at brought X,XXX each of years again, technical a down nearly be of gene we rare therapy that Pompe those Amicus, therapy the there under with are specifically looked science ahead Amicus at at we engineering, Technologies. projections. to the strategic the of look Technologies announced the competitive to those XX selection, for moving dynamics you combining of combining our with fast portfolio and recall, programs, you of to technologies in again I Penn key our all that fit look allowing. the Fabry XX when patient back that instance, Amicus diseases; drug disease, November, And And of, analytical our we well. the Transfer at as of was history, and then list internal Penn if unmet technical today, months we think take, availability priorities the and them candidate continue broadly, of XXX, manufacturing if at need, we look Gene then And protein of as Fabry size looked and gene for populations, ago we're moving began course, forward. at number to We're original the we and natural

the financial we the our capital, sure did capital, available, and we human priority. to the So, earlier which have of have parts reordering some we capacity by make that manufacturing

about to our those we of if administration? that, within it Jeff based leapfrogged pieces on the effect on feasibility of think you has different of Pompe the Fabry technology, in all ability maybe and how route gene technical disciplines. With So advance including therapy can comment

Jeff Castelli

Kristen. hi, Sure,

collaboration to where approach our out, expertise that pointed secrete on can cross you we as and So treat cells transduce that cells rely really can then the we our on cells. optimize help to engineering other side, the with protein therapy Penn protein gene is the neighboring those correction

solvent We both. we're on the have address capsid really Penn bit that tissues amount versus into delivery, rely a whole host systemic of CNS, we the little on to of of the technologies trying a it's whether administration and depends sometimes get trying can from for to

across or So in it's therapies. this really the a toolbox transgene, to first right going to in intrathecal so best right that class really administration, get case is get that find really we capsid, gene goal a Penn, can right with

we've far, the key across programs we've Pompe is ongoing shown and studies right as So, we in Fabry great think combination have with finding right those And we now. had what those attributes. of preclinical success,

those You're of looking of at all factors. different combinations

that ability the So to we having that think strength that so great collaboration. do for is a

Operator

from with is JPMorgan. Anupam question Rama next Your

Your line is open.

Tessa Romero

the Good is taking for Tessa question. on morning call Thanks for guys, this our Anupam.

us us one remind Just clinical so additional with where the and patients respective the disease material on programs, Where CLNX on much. you from you for with CLNX CLNX dosing Thank you GMP for program? program? manufacturing see timelines the of similarly also can Batten are you grade

John Crowley

of Yes, course, Thank Tessa. you.

all That's So been can FDA, the it and grade and really, CLNX negotiations the analytical with place, both BLA. then commercial been with up could the technical so levels, production in as not patients to place. and analytical then manufacturing in ensure assays scale both components but to they not as necessary to in going that have CLNX into of and on helpful CLNX, the with gating and we're From for well only all assays discussions to go commenced also the of CLNX all place feedback. factor the for properly the and part both we appropriate we provided commercial characterize scale, material very but quality course patients that work into tightened the two standpoint, the GMP all be key material, have going have And as manufacturing scale-up manufacturing support activities grade GMP itself. analytical only having to having good a is ongoing really well so support a we the of of course those

it's us. Now, again, not just and

the I standards. of therapy a coming think the you've broadly across in this this We seen saw XXXX. tightening gene of field, fall the

been think the I So quite ahead in we've regards, of many curve.

has been the technical the as for very Fisher Obviously, tech the remained capacity GMP we've we at on, And some all Thermo that as potency with transfer tech place, really Brammer, feedback planning for a any instance, all in same a we already partnership the and that transfer again, in the had completed. team. Fisher agency success been assay we're they've good to There to good were manufacturer. Before well Thermo was ensuring we've scale. for the reserved, the with with have manufacturing But place process. the importantly, the the improvements our received

Operator

Your Goldman question with is Richter Sachs. from next Salveen

Your line open. is

Salveen Richter

my for Good morning. question. Thanks taking

build manufacturing, as from look bit just broader to based Just building the here? talk comment not maybe to more the internal portfolio, looking out but Could you your manufacturing with elaborate external to on little therapy about you you're of here. an past just the gene out whether the build a work Brammer, platform

John Crowley

our Yes, to manufacturing? for want do you strategy talk thank Bradley, gene therapy you, about Salveen.

Bradley Campbell

Yes, Salveen its great question.

multiple years out a our As capital supply the flexible world-class was first into we And delivered to manufacturing platforms. next external of step this think, with the Fisher towards actually we're is CNS But studies have to and space progress with work for perspective, to great made other design mentioned manufacturing CLNX. both will on Brammer. our secure, manufacturing facility support partners lot continue work previous we CNS number process building in support platform. we is first manufacturing, based And development very that with our platform as Fisher our to of medium-term GMP did and for and arrangement. that state-of-the-art Thermo working John, moved question, PENN I which for we're secured as the also generation suspension to to the we've own think in support our We Thermo the but good a the portfolio, programs, support part partners for CLNX a that well we portfolio, collaborators that From at have able facility. doing underway the to be next flexibility news internal of and clinical adherent to is there a

going asset. delivered systemically That'll other of to As building we could our in a be programs. a commercial really and manufacturing capabilities we Pompe the therapy think long-term, our support strategic our be forward. part important gene towards and pipeline own And look capacity

of support ongoing to therapy And that we the to portfolio. our to continue so growth gene look do would

Operator

is next from with Citigroup. Bansal question Your Mohit

Your line open. is

Mohit Bansal

question your congrats last of talk so, therapy, or AT-GAA. Great. challenges of on efficacy the point Thanks even regarding programs year, seen question, lot picture gene gene last we within from to taking FDA a and so And my on a for big XX safety the therapy like Maybe months have of our regulatory with view. or

more clinics that given safety drugs terms from what is a and which into out It your like go the bar And regulatory that existing there at are seems FDA of little you to is entering drug into with of you. there. Thank So, diseases that an deciding updated what's the requirement? of you how after? and Fabry, diseases view, and Pompe understanding in about in cautious bit is or cusp does mind, are efficacy on also, point the that

John Crowley

the the on broad could Wilson global his not an picture fact, came really not very we biologic patients. world with of Galafold I dose We were delivery developments regulatory in paradigm. to right for with AAV building appropriate, in that experience make very only the actually Mohit, complex the evolution, of the our we like a then important into manufacturing quite think bear. for experience it's for to diseases, Amicus, big dangerous AT-GAA, we Yes, wanted was with biologic our Jim thanks we appropriate brought selected strongly one. sure but systemic was therapy is that it medicine high technologies view be What also and in question and When genetic gene

It being the again collaboration UPenn we can mindful of Amicus. these of the of that's vectors in be gene technologies. part need and protein that So, -- thesis by expressed therapy was between to the we

an that at potentially evolve. where And could and we safe, of were very see I'll us advantage in of very investment it the as cells is reasonably efficacious, So, be to instance, expression transgenes, needed Fabry, there. just unique that be I do distinct appropriate second-half could of up. of targeting, focus from and of potentially to and so technologies we think at the we molecule data to enhance of will in transecting example change the lots lots plasma not on you're we in preclinical world. may that actually get the focus Amicus and I manufacturability. efficacious scale, course which each targeting is regulatory an paradigm just And the two could bring to we can enhance as liver potentially engineer market that small excellent the very taken have on a to is different a we price efficacy terms for stability a on each a again manufactured area to so be with this as think to Pompe, disease well customized comment molecule, very and hoping to not you We organizations Fabry a safety to therapy. all way deliver to gives eventually mindful very flexibility that have the unsafe ultimately briefly started where through continues gene XXXX. combine gets points think wanted mid-XXXX while the What It to to being tell most in

guidance to to, did the significantly think the provide by course, preparing the agency XXXX. CBRE but, COVID to throughout extent developers of they capacity overwhelmed I They gene therapy to had was was substantial that crisis.

forward-looking core the approach, longer were with technologies and strategic taken that really that's think develop. We regulatory paradigm our I to So was I to and think changing.

seen then safety that very, challenges cells the that of you'll lot targeting both on that the Fabry think in and Pompe maybe unique manufacturing. respect to with a And delivery delivery place on and see and and other in have of just CNS with we development. programs, we'll systemic delivery conclude very I'll be I programs a that we've

we I are both think, CLNX terms success programs we've CLNX feasibility of have, inherently, the they CNS and And the for technical that's each addressing including advantaged seen I of that those. in the think of better the in With our programs. reflected safety

question, big a big you asked you got answer. So so a

Operator

question Ha from Stifel. Gon Your next with Dae is

Your line is open.

Dae Gon Ha

and Good guys. quick on two AT-GAA side, the from taking clarification I John. my question questions quarter. morning for congrats Thanks us on the guess

outcome? now migalastat. the an using approved are that a somewhat -- AT-GAA, you're the to and you. approach recognized the that basic can very too employing remind manufacturing that AT-GAA basic, where it's guess for Does a for question drug secondly, that's And, modified So if you're and dealing combination but an and forgive with us Thank as drug, ERT regards you me the with warrants you're I material? sites it's also

John Crowley

of we're place perspective The previously increasingly in year BLA. AT-GAA is that an thanks, CMC been currently FDA parts of very think They the in part Gon. Great, constructing that With that Dublin also undergone Dae of be inspected next important and online outside will PAI inspection. one are the commercial of be by respect good a again, to we WuXi facility that's WuXi the and Biologics, a the in will in final from facility stages a a supply. commercial already the of manufacturing Ireland, large will final come in

and will require and along both this that we course, component, that stated an have and of way our a see to With providing we the Ireland this an does outcome. and a NDA, early having that that we've necessarily supply, both no ERT lead product. would an China on, BLA So a and dual indication respect to both Chaperone whatsoever

Operator

Your with Leerink. is Schwartz next from question Joseph SVB

is Your open. line

Joseph Schwartz

wondering will team. AT-GAA? to that you others, based you meeting the think thanks comments might just than seemed de-risked any meeting. that bit Great, components I Amicus be made topics And a any you -- issues have pre-NDA with? both Hello, it need then last be of to carve the review on grapple about that a the still FDA expand just very the there were that Were more you on could pre-BLA particular much. to was discussed if little in at

John Crowley

Joe. Thanks Sure.

the Just will suffice known to is pre-NDA sure strongest on solid large issues, be. just small itself One meeting. will It we have a am didn't feedback provided and the BLA submission. no, have It was in excellent With never was answer there a be that, I there tone. we I have meeting review that review molecule. a comment the the that respect collaborative. cover meeting need both issues. to I'll needed don't that we to the ensure

was to together the we that And therapy the being important into end I data package that bring pre-BLA June. designation, we And, quickly think, BLA this FDA submission. on with we're this But submission breakthrough need the really patients So, FDA go product yes. fully as formal by I many unmet written aligned prepared as And very having that minutes the possible. with the will forward the have think the in again as feel of a to community. confident step the receiving meeting, certainly

Operator

next Guggenheim. question Chattopadhyay is with from Debjit Your

Unidentified Analyst

on Hi, Debjit. any Thank morning My Thanks dataset. MAA question approval you. team. if This today EMA, you have is confident the for interesting on Robert for team around the had AT-GAA? is good call. first-half feedback is for potential submission taking my from how would And be

John Crowley

MAA, second-half with are And to of timeline continue with for confident the Yes. be on submission in And MAA, Thanks, our say we we highly already I'll Robert. on to XXXX. authorities. just engaged AT-GAA's that track in are approvability. the the we respect the

Operator

follow-up Baral Cowen. Ritu with a question from have We

Your line is open.

Ritu Baral

one, cycle generate am Hi. just might this Thanks. generate make first study about And I pretty you you study perspective this if review I I'll thinking you adolescent constitute then, when that infantile I year? data and during BLA, you, study a the the might of data quick. efficacy if the design guess, to wondering the the submission? are data? efficacy it guess of John, start give for it on supplemental might ongoing. original And the that as it us adolescent mentioned is soon. plan on guys. that starting update Thanks infantile Can

John Crowley

some the deferring year The course. adolescent was in program we of last did study effect of Amicus where Ritu, begun clinical delaying enrolling Sure, or enrollments. an in see one we COVID

that pickup from reasonably data a the during It's a period. enrollment. we'll all in see small get will review study, So, that unlikely we the

the So, to of AT-GAA be just approval come population. the late that onset disease we what in will expect would Pompe after

-- forward But supplemental expansion will not would that controlled a not of So disclose think infantile the we're study. likely full part prepared of small. it yet straight nature I be relatively study, reasonably It the BLA be be label as the the of will study. again -- to In that submission.

under that of very infants. already also Pompe don't to have we to So, to expect product. months. of but And effect a the will But it's nobody evaluate the the it. here expect in a start do will everybody people a just And some we basis that relatively to a have more of that we the the two. important on we protocol. part it very certainly next we part medicine that not access rolling or I'll to of we of access very, a compassionate population, will small to have or very or protocol the over start to placebo population. our expanded believe -- continue the go expanded competitive color study under study then, that to a couple use provide remind And of next just too important treated remind data of And I'll It's number quarter the Pompe on infants actually also

Operator

There for no like to questions. Chairman now would closing further the Crowley, John Mr. remarks. time, turn this are and back I CEO, At to conference

John Crowley

QX, mission in as on us, Thanks analysts we quarter listening. for important year. all to everybody us you, we QX important Again, move this questions. Thank Great. for forward excellent an equally very our see for the for quarter the and

So, great everybody. day. Have to thanks a

Operator

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

Amicus Therapeutics (FOLD) 10 May 212021 Q1 Earnings call transcript