FOLD - Earnings call transcript Q2 2020 | Amicus Therapeutics, Inc.

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Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Second Quarter 2020 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 Director Faughnan, Relations. Andrew

You may begin.

Andrew Faughnan

Thank discuss Amicus morning. corporate you financial for to call highlights. conference our Therapeutics’ and joining results Good second XXXX quarter

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

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

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

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

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

John Crowley

to quarter Thank our Good results call. everyone welcome you, XXXX conference second morning and Andrew.

start we well are hope that and and safe. let last quarter I stating As their did by me everybody families

and initiatives of to to during continues pandemic. our global a workforce ongoing protect at team this leadership support emphasize Our and programs range Amicus

global clinical, While adapting been science, Amicus biotechnology to and as continue all commercial people poised one the around to first of the a of we the our great of build to execution see you excellent next across has of changes efforts and half period of growth our the about including pandemic, for by companies aspects with all business brought diseases. living the as regulatory global impact rare XXXX world

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

$XX.X launch in expectations. Galafold and Galafold million its launch remains The the our First continues revenue. strong second to quarter success continues performance cornerstone exceed with of

Second quarter all revenue the represents the global hardest and hit performance regions naive throughout treatment including in starts business including both switch patient major those patients across by COVID-XX. the new from quarter

Second, track. timelines on R&D our remain

late read AT-GAA study continue in X expect of out We XXXX. Phase PROPEL the Pompe to to first in half of disease onset

And third, financings. Additionally therapy. our we remains July, expect sufficient as CLNX and our Within is cash any move advanced the programs following Amicus rolling BLA need gene profitability for pipeline to our the achieve position pre-clinical future well to Batten on disease continue lead therapy and we year. forward schedule later submission the gene submission this for AT-GAA first programs financing as CLNX strategic most diluted without in for our in now

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

disease. precision medicine X, for we on Turning track Galafold, to key five our to slide XXXX are including first well achieve strategic for Fabry now priorities our

year to people million disease around the look revenue million. existing achieve new and more the of drive variance continue living this with to world. in Galafold to will We with Fabry We global $XXX $XXX markets minimal product to

are we planning and therapy AT-GAA this surrounding we Pompe our towards the important regulatory program as Second, for activities clinical move pre-commercial and approval. manufacturing increasing

and the Third, two in our gene candidates manufacturing programs. we IND therapy of as underway the our we scale well work disclose again center IND we research partners from are is manufacture regulatory lot and potential of Pompe Pompe our and therapy we candidates. advancing our our addition to progressing CLNX and of our towards up the industry-leading advancing gene Philadelphia for rare portfolio will year. therapy IND manufacturing additional A be therapy plan this disease excellence up in for clinical with CLNX gene both as global other new and to gene development, Fourth, discussions Batten sending are

So the and we collaboration remain later fully IND additional we this strong our continue our candidates financial again as PENN manage and look we to again we Fifth, carefully now our maintain investments from forward position profitability. year. a expenses to to will sharing funded

like I team quarter of So to strong as board wanted the board. patients. a our his take of friend call Love's and the thank nearly more Vice on leader, moment of on Ted to from section with Amicus very of and mine the board provide that Dr. many decade company position. and detail as to Amicus Bio I the the a for personally know great innovation a put to for helped fierce in much would you him closely remarkable of I I for to the acknowledge Chair our a will Ted directors. service before over has He to and advocate emerging work continue just stronger Chair on to a Ted turn retirement together is

Campbell that Galafold president officer further highlight Bradley the you call to to turn let With performance. the thank and me our over strong operating chief now Ted. Bradley? So

Bradley Campbell

Great. Thanks John. morning Good everyone.

for growth this start will we Galafold revenue is John detail quarter continued quarter in here now the I and commercial give XXXX let on our slide and of progress. X the performance snapshot second Galafold As through me the where the mentioned walk more our on of Galafold in you of global

of as even quarter worst QX was quarter basis decline generated to strong global total geographic rates and from did the or of $XX.X and United United million outside demand, product and the from far revenue COVID coming XX%. driven by States. timing to orders. was so second patient of our the the business the John the The million the favorable of breakdown during compliance revenue million quarterly XX% mentioned QX sequential and within XX% adherence ex-U.S. States quarter continue remaining that On $XX.X to revenue due dynamics importantly a reimbursement $XX.X during revenue For exceed you'll slightly note or continuity

growth patients added second into now quarter strong first QX. see to strength has and during the that continue in we However, of continued month

year expect finish we U.S. So the to XX/XX sales. ex-U.S. to around

XXX over important support Galafold now the and leverageable patients lays future sales One around to for as not the regulatory with access a support really As now XX Pompe other to a of of growth and but expanding countries potential commercial world that global in AT-GAA, our is of to over aspect just XX footprint it reminder highly this approvals launch and have strong with foundation products interesting the now and slide countries that is of all access we the additional mutations those which X,XXX market continued well. were the we gene those with mis-sense all the in have are amenable European by patients been associated highlighted mutations. expanding of identified our is represent added who'd mutations Galafold this to new on These specific possible mutations amenable mutations as unique that amenability assay. with to determined nearly to quite GLA The over this Galafold the is previous is label.

with assay; one previously no mutation and So take they'll the longer could process for which patient physician characterize us of amenability the several new to run through these months. a to a now wait have it diagnoses mutations, the

simply can Galafold. apply the to geographies Now and can refer and the medicine the that website looking the a and pharmacogenetic of to and other application of to exciting amenability physician a get the check really really we're patient as application immediate mutation one this the Again unique to aspects well. of access

the of We've strong COVID-XX Turning now pandemic. a even very the epidemic in face had slide to quarter X.

Our intact. chain supply global remains fully

has touch of customers confidence they great means physicians. Galafold telephonic other and their field a can Our able significant have pre-COVID digital, with team been our through and achieve interacting portion their points of that access to

continued accounts. headwinds important key will naive youth All in physicians their pandemic the tailwinds continue treatment dynamics a it's evolving visit with to out in believe back to monitor as switch new go full the of does which has provide on to the hit and patients. to able which country to the on starts country uptake these organization local of has patient Spain, some led inevitably field exchange. that carefully as perspective, caused to From basis a many increased some that to now countries allow closely and see even Italy, We global negative remains all and Even UK, continue quarter hardest impact in U.S. pandemic impact the allowing are from both as unknown. our that to quarter a second XXXX quarter currently France, can from this we you foreign in include XX% we're and numbers the second the sales crisis throughout others the manner, prescribed X% may a like note Japan, Galafold patients very conditions throughout bring by

year. perspective operational compared from last sales true XX% a So by increased to

several past as left-hand our variety growth show calls due and of the On a we to quarterly we've quarters factors non-linear. on quarter-to-quarter performance to is the over rate we growth previous the typically while side expect strong continue mentioned of

year past in expect to compared million will very half of in which higher just be in we've year means in revenue quarter-on-quarter Looking QX year to the larger fall global to the the of portion the month the seen we've our in many as July into we $XXX half million we years QX in the we But year quarter. the finished full seventh guidance second overall confident back of sales. $XXX the to continue the of expect of as as growth here a fourth

with are the markets world several a to on and opportunity behind $XXX years say most performance of in we X, in slide on launched that sales us path million we Now confidently XXXX. major can now around

XXXX $X period. previously five-year XXXX our funding goes about from R&D between XX% XXXX XXXX million we OpEx of which and generate a in alone that that average expect cumulative to a outlined and expect get long to CAGR we and I've revenue towards way over As to to billion $XXX

peak have by untreated $X at billion population. see newborn We in the in to confidence driven Fabry diagnosed other and to in the revenue further diagnosis screening, globally continued penetration even also from high-risk opportunity initiatives addition as we screening market continue diagnostic the now growth into significant

IT us coverage have multiple access me for orphan to the of so orange period and we lots development in listed U.S. chief provide lead our to late that programs. exclusivity XXXXs As into long Dr. let further our Galafold have over to pipeline the to opportunity our addition book will hand give a the Castelli we officer Jeff? now highlight in who come. that patents With to Jeff call Europe

Jeff Castelli

good Thanks morning Yes. Bradley and everyone.

in Moving throughout our novel consisting care next to XX, its distinction administered on to the stabilizer. updates bind and treatments everyone an target GAA R&D delivered differentiated to administered of of lysosomes. intended orally taken cipaglucosidase human investigational of to AT-GAA and action. major or is and into designed IV allowing shortly remind in our infusion stabilizer via muscle more mechanism ERT generation slide therapy circulation the administered is combined enzyme before ATXXXX, active ATBXXX therapy cells to enzymes Pompe. ATBXXX we up be is is wanted our and cells the to of enzyme of with Pompe alfa from ATXXXX on other of body combination an one recombinant highly for begins The ATBXXX development and infusion At-GAA standard stabilize enzyme and

longer AT-GAA journal a work With development showed successful where the improvements mice. other of ability the team term impactful be decade with therapy Health Institute publication reversing over including pre-clinical restoring the in mind Pompe in clinical impacts like of accumulation believe those and muscle treatment to potentially past of in at binding ERT to efficient in the XX, that The published we Ravens] markers that up receptors carbohydrate as Hung ERT itself. would the while damage highlight previous our his working improved upon a previously in and or it's AT-GAA Dr. have Nina we completely after and the turning by to the [Dr. the GAA. of methods is the These from in science develop which to secondary to findings study to The building an slide uptake molecular cells significantly officer more for been study now retaining the had studies. additional of target team are Do, with showed and profile independent enzyme not distinction taken that was mature National cascade active self-signaling. improving more fibers, unique disclosed and the in of a importantly findings lysosomal cells exciting excess recent processed particularly form built-up abnormalities observed elimination strength improvements reduction and muscle autophagic of glycogen and these knockout been chief

more in plus We To-date first assessments of study XX% that Phase X,XXX Finally, remind for study ongoing have completed infusions on on the to than all reiterate track PROPEL data the our on expected slide the of and everyone XX planned been we of remain the schedule. integrity half clinical want timelines PROPEL XXXX. Pompe trial. of X with study

patients and to Additionally, their manufacturing and a unprecedented families staff pediatric in and to to to investigators to our cross-functional our Amicus our all MAA we express their for all XXXX and efforts continue Pompe unwavering thanks study we and support this time. enroll collective studies their and supportive participants commitment our in want to team clinical Pompe BLA advanced

to therapies briefly slides rare will now of on and Moving portfolio our XX, highlight diseases. industry-leading XX I here for gene

CLNX with our across Batten and able gene franchise well with including time Pennsylvania. our Fabry as programs portfolio CLNX, therapy and been and of of gene Starting maintain lead disease as we've this CLNX University programs Pompe critical During that neurology we're our the disease virtual meeting at COVID In AAV therapy from we to of science the CLNX and the data demonstrates disease. X extremely expecting Study. previously annual reported X, positive impact in interim meaningful data child therapy of this devastating October study gene in clinical Batten our Phase society form additional the Batten

initial ongoing data the provide neurodegeneration. to paths conferences. discussions we clinical and for regulatory Phase path common XXXX. plan Batten CLNX CLNX scheduling regulatory this We in advance report to initial data most important which the update and CLNX next to provides childhood for on of gene CLNX provide clinical in form early continue We to expect finalize disease through a believe the is of an read year early therapy the the Concurrent study to data with changes major X based year some from for the early next regulatory study program. will the We expected

manufacturing make We commercial and continue great CLNX process begin both CLNX both programs to on of progress next in remain additional these patients for of all and year. dosing to on our track

of gene for This and therapies be optimal cellular collaboration would Amicus protein Amicus' research novel of ability. targeting, Moving expertise driver slide at safety to Pennsylvania remind and of develop the engineering I transfer an are manufacture PENN important and with to XX, University that with the dosing, combines with the uptake, Lab the like expertise on which will technologies Wilson everyone PENN's to growth of future. gene glycobiology for in collaboration designed

different Amicus has engineered As meeting glycogen gene over ongoing the part mice. data disease was pre-clinical to were GAA the Cell results showed active Society Gene improved clearance which programs. targeting given back rights in of storage This this Several had at quarter. diseases highlighted Amicus the annual addition Pompe Pompe by Therapy of in therapy the and our second in anecdotes protein of American presentations collaboration in XX to

data to levels and you a suggested to towards therapy by is continued primates organs gene non-human and move very target relevant We data encouraged our larger species. expression factor key this remained an Pompe progress in IND. therapeutically preliminary as Additionally, which

year. make results, across to outlook. guidance additional to candidates Daphne continue over our would program also up With therapy look We pre-clinical progress and the this to that now our financial IND like review gene and turn to I two Daphne? disclose call to to later

Daphne Quimi

good Thank Jeff and morning you everyone.

XX June quarter XX, statement overview Our our financial with for on the income slide ended begins XXXX.

the over quarter we was a This second we Total we of impact of constant operating year compared where $XX.X negative million non-GAAP $XX.X of X%. the Cost the of XXXX. quarter XX.X% of second royalties. well to in in which XX% a million study second growth reflects in revenue second prior were achieved offset PROPEL expenses quarter as rates XXXX. gene decreased as quarter owe expenses measured XX% of the offset revenue at the countries total of in royalties pipeline. XXXX other our to do of continued net a for sold currency to development and as goods as basis cost Galafold currency quarter million the grow costs percentage by $XXX.X second the period. the were as The not sales a travel of which United decreased in is On increase where third-party exchange operational the of research XXXX $XXX in of in subject For lower quarter as and year-over-year to in by decrease million investments goods $XXX.X revenue the are to sold as second XX.X% percent includes compared Cost favorable compared to second in and was therapy XXXX. operating support quarter continues a States Galafold revenue as of million

third compared the administrative Our compensation, decrease quarter fair as operating as and share quarter measures prior of value expense the of Net was $XX.X expense announced of consideration the timing and general changes share-based the second or as $X.XX of of is non-GAAP as $X.XX to includes were depreciation. the loss in in to in year for expected per XXXX. does net for of million and SG&A expenses. per reduction Due development to expenses to quarter operating and previously research impacts cost million the and research the the implementation of investment increase non-GAAP the that expenses the second XXXX contingent selling, development excluding period. $XX.X or We define loss compared in share

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

$XXX mentioned are secured profitability XX facility need a term any John of on now path to clear to million our the salute without slide for now loan we following Turning financing. as future senior

year last as initially interests. have at we driving us patient expense revenue laid savings this continued we strong financial with cost day and shareholder as setting a Amicus efficiencies, and with well Securing milestone gives financing our our terms growth Galafold analyst the management. out As advance by careful both achieved platform to market

again expect for operating remain expenses flat that forward emphasize XXXX commercial our is non-GAAP AT-GAA to the we Going first the total XXXX infrastructure we from products launch other in as to in in already and pipeline. relatively place leverage

the with securities Secondly, at in and PENN. of cash moving gene we to position transition therapy continue marketable priority Jim our guidance. to cash financial our XX, compared XXXX costs Pompe our Fabry and research and especially equivalents associated work XXXX and comments XX, CLNX, fully programs December Wilson were $XXX.X about therapy $XXX.X support objectives. discipline CLNX, Cash, we at million million the on June few XXXX. A development reiterate and and maintain with ahead in third to To high pipeline all AT-GAA gene the are schedule programs financial multiple meeting in while

the facility of the XX. proceeds a prior term of of $XXX position July $XXX retiring loan over Amicus and from has receipt as July million debt Following million the cash of net

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

John Crowley

Bradley. Great. Thank Jeff and you, Daphne,

So on of see the all we about have the challenges change as the pandemic. been you brought unprecedented performance focused global and have relentlessly across been can despite business that by

will as that Amicus have this passionate global crisis lead to a emerges even led this. and who at team continue emerge have from confident We I am Amicus stronger. great the of us will entrepreneurs through world

we're operator take that to happy any questions. with So

Operator

line Your first of Rama Instructions] Anupam [Operator the from JPMorgan. comes question from

is open. Your now line

Anupam Rama

Thanks the much guys. so Hey question. for taking

question Just on study. quick PROPEL a the

skip For those so outside patients got scheduled who did one? get infusion, small patients just of was get these on-time infusion of ultimately did infusion, a it they the infusion and Or number much. window? an the Thanks then not an next

John Crowley

analysis Jeff study just any preparing state Anupam be plan question. to will of in with more common certainly expected for that's would you studies. X% in the infusions missed clinical study disease the kind of that I Sure. that [field] most and to studies maybe ask as statistical Again rare than in we even the

assessment So than very-very infusions. better even with infusion Jeff few could maybe COVID-XX to speak but an from we're an standpoint and expected performing we the missed

Jeff Castelli

number subjects and Thanks an to or fits but primary as what Sure. have some patients could study importantly Anupam. to in the seen still better of to that analyses what for individual slightly infusions we potentially the for entirely John and everything was two than having refer design had. had study we either It thanks planned initially we've within small pre-COVID even it the had infusion. allowed number be out remain window was in missed and and missed X% infusions part study infusion every initial could That of we the week of

John Crowley

medicine, to patient-by-patient, remember to Again desire with a study. just study, site-by-site by to we even April, back team. had March days efforts very so and the the think in of then plan efforts you maintain on the participate few integrity infusions Pompe and testament a through to remarkable That’s patient that the Yes. crisis motivation, height early was came some really access missed desire our that of these of I the real investigator that remarkable we in if up

today. we really we're where pleased So are

Operator

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

now is line open. Your

Ritu Baral

how been final love out timing completed? the COVID have the time to in how question. Can QX? assessment and might taking of that for primary necessary ask to I'd so to them of those and many about them the been about of to the guys. you we morning, spec at the specifically have side question know Anupam's Good the speak and how of, go, timing How Since are for danger. of I know the number are planned of point endpoint. for don't many talk many flip going those is take will specifically walk six Thanks minute

John Crowley

test level assessments probably were that within Virtually or window the lengths can't who's provide the six done We that terms which to those but of just a or then all in assessments. comment ask were and time timeframe quarter those due went real in granularity and that assessments completed Jeff make Yes. particularly assessments I'll sure We two we here. March of occurred. of on which April XX-month in was the extraordinary which date to those to again where on I'll for minute walk concern

I few, two out or of very-very So may it have one patients that think was fallen maybe window.

any So like no again Jeff color study expected please? you'd like a study in it this. should impact as have and on the

Jeff Castelli

covered even XX also plan are just is everyone minute together, you to before and off not just and missing track. data study had we particularly enrolled with well. visits of on is I'll the over well sure but that walk from John and month clinical you that those as some you study we six implies planned assume any going infusions to trial statistics XX% the really it XXX to the are we John trial within and what make we due assessments Yes. everyone primary amount the as endpoint pitching only that XXX do scheduled very missed and dropouts, remind said plus as

final in we're and So date great place patients in the analysis. of a for terms PROPEL available

John Crowley

Yes.

typically in plan virtually or for drop in participants study we've study Importantly and the more of to will plan had out. statistical up XX% dropouts again Ritu that you no to

missed a great So have those us as also that very patients and a for will us certainly assessments. gives few great who margin well deal help may

Operator

question And from line Debjit H.C. your of from next Wainwright. Chattopadhyay comes the

now Your line is open.

Debjit Chattopadhyay

morning my you question. good taking Hey for thank and

and program experience? sort therapy consider a a relative the any the when of on through in glycosylation migalastat with for program patients goes of you. Pompe, of clinic think as pH bis-MXP keeping the the therapy dose thoughts you as based you the gene and on migalastat So versus on PROPEL gene stabilization background stable the Thank contributions would

John Crowley

again had we cardiac, contributions levels enzyme we And so improved enhanced the is involvement be is Hung the exhaustive internalized great carefully developed phosphate move diaphragmatic, improved to be studies the with in the XX%-XX% for Chaperone the benefit looked target the the targeting the and Debjit, Yes with that as of very what new and pre-clinically well. so what's we cardiac at glycosylation majority I and absorbed into here would of move blood plasmid that that of to absorption would relative be to look all and high the key muscle in together did patients tissues that and It's receptors characterized that muscle do all think studies of absorption glycosylation saw would of is the levels phosphate skeletal, with to and think pretty from think of the are and nano-six to from remember nano-six of glycogen. breakdown the enzyme nature high the enzyme. necessary enhanced the the the plays important enhanced mechanism the Chaperone due some the the to those pre-clinical that with of benefit is we of our function glycobiology. the be an We on

active better but a protein contributes also plasma when it function more enhance form again it stability to So make to lysosome to to it which breakdown. tolerated the we even is the in think it the the glycogen the potentially deliver Chaperone of in greater does of it delivers

novelty is the the part great for again but glycosylation benefit the of that's we benefit again here patients, the in of of the the think preponderance combination So the therapy enzyme. of

Jeff Castelli

therapy basically just therapy gene the I the there which asking is to was replacement add is enzyme might think with sort as giving that majority also out the the think the into a difference this gene of the engineer that dose constantly of enzyme Jeff. A enzyme GAA. gene cells two transduced secreted to is of towards with question AT-GAA weeks therapy rather that well-targeted apply transgene benefit Hey the been than I'll only therapy is the every similarly from you blood. we have versus about

to a might So the as how simple it's not gene with Chaperone therapy. quite help think

with now a not the use just as gene potential but that's vision combined but our gene right therapy is plan. some where look you stabilizer So is therapy right could Pompe stabilizer for to theoretical now a there not a our

John Crowley

Yes.

are looking unstable You also we're exploring that's could other engineer transgenes in you into some that could enzymes aspect the plasma, where even diseases potentially we're of disorders. technology the at more and those for in that some one stability inherently

Operator

the Citigroup. your And Mohit next from line comes question from Bansal of

now Your line open. is

Mohit Bansal

Thanks my now taking Maybe which the question treatments all especially despite disease, Lumizyme question the Pompe is progress pandemic. my one is thing on control and are the least existing how for on neo GAA mind for from side program, one that have was Sanofi because of Great. the clear inadequate congrats in -- at we data that getting here.

experienced are assumptions mind here PROPEL relatively to here. arm control and be in that you this updated any powering Is assuming patients study with not because So have the if treatment at I'm for for mistaken do fair you that stable the thoughts control point? about

John Crowley

going the neo I'm Thank you, data. of any on not Mohit. to comment

think data will that itself. speak I for

the that arm the study study, naive showed. finding improvement that treatment any if that which less maybe the was it onset the recall studies, XX surprising LOT in previous I than two is seen the has just to been study control a meter the was that Lumizyme or month study Lumizyme in endpoint arm approval a patients showed again control reference at did You in late

I assumptions for improvement what treatment built we into or that think assumed in cohort than that's powering patients, naive XX Lumizyme was XX better it the our in it meter did common much patients the study. perform study. of our would We

into stats. the enhance or it Jeff that So comment? only and our color ability worse and of will our add that's course If distinction any with show built does to what's AT-GAA

Jeff Castelli

that you John perfectly. No, covered

Operator

Fitzgerald. line And your Ellie from next question comes from the Merle of Cantor

open. now is line Your

Ellie Merle

on congrats taking much all the progress. Hey guys for and thanks question so the

Thanks. on Galafold the diagnosis treatment as seeing treatment you the just addressable you're one rate seeing in long-term sort of you right current what based realistic so given in sort I'm landscape to light a of now, market and of and for about Just how under-treated terms kind well as mentioned disease, guess potential for a under-diagnosed disease and broadly seeing you're therapy on you're in that currently I peak markets think in we diagnosis entrance evolving? this rate, I gene Fabry think competitors just about assumption of recent curious, penetration rate sort this trends improvements and of of your the in know see how pretty

John Crowley

Galafold people Again being with with and a help had is remains identified. of and disease are Fabry think experience as I great a lot Yes. living opportunity and to it patients more being more importantly more more

if We the or continue to under believe most that of Fabry existence. the human diseases one in is most not misdiagnosed genetic

we're what and growth about the So Bradley, here let you specifically potential planning for. I'll comment

Bradley Campbell

Yes. Ellie question. thanks the for

sort two there's of think your pieces of question. I

of of who the first I'll There little see penetration one into a of diagnosed diagnosed. penetration population. patients the are is it's bit the diagnosed percentage the then we'll is high take first, I I easier. population treatment second think potential into and First Fabry think treatment

if the to year were since on been on the grow molecule XXX introduction markets even we've diagnosed you introduce we've and shared think treated look market see end we Galafold further MS I the been treated at patients that we've PAH the looking able of the of of diagnosed, other see you know of a I We've we've analogy, to from at market even the been lot plus space other we'll that previously the if you you the question and time as but molecules MS of small into space think of spend growth about Separate of pain patients risk at think population. but population lot in also and of manufacturers screening class before that both talked in if seen growing. ask we in that potential other as diagnosed the ourselves space newborn last populations, them be out will significant look Galafold populations that thousand we cardiac a is number in patients well. screening clinics. think small introduction that molecules, the Fabry as and the at back [indiscernible] with of at and And the with the of Separate in TECFIDERA looking percentage you high kidney what a in of I play well untreated. see able

I That X,XXX XXX,XXX come a incidence this efforts disease see a see one years look an of if a continue would it going will to screening that that incidents. in unfortunately that you be under newborn that in said, a patients. could being Fabry and of diagnosed live that. global one are potentially lot at imply you into birth suspect So for to is X,XXX we

that on chip a do all great diagnosis rate So we at a to continue over us the I I would so. away but next think that would to be journey go that think job or of decade

Operator

the question And line from Baird, Ulz is from now your your Mike of next comes line open.

Mike Ulz

thanks guys question. Hey taking for the

John Crowley

Good morning Mike.

Speaker

morning. Good

quick I'm year? to BLA, track a Wondering if you're to plan majority prior in you to AT-GAA of on you'll a this X. and be that just you this curious of the think BLA position Thanks. the kind year. you can Phase a the have filed the Just that what of us if to rolling second sense give in on you're still half of start question file

John Crowley

Yes.

biologic know are three the we key As sections application really or to license BLA. there

module. pre-clinical the is first The

and Second be CMC module is third the clinical. the will full

part completed. That form the toxicology first will expect full been work the year. including foundation the filing has this all for We that of the pre-clinical the

and CMC the PPQ successfully. all then filing for section expect of the again We the completed we've runs second

part the frankly risk work So of significant a significant part the a that. taken and of of out been has

manufacturing a here. at feel partners about Wuxi our Biologics commercial up and just and we scale at quality really So with teams is our job remarkable the really done tech-ops good have where

of half in year to first section half the expect to go full that be the of then would we first the So in clinical. next year next last and also in go the then

that hopefully your Mike. question answers So

beginning in in hope in full this completed Europe. in XXXX very So BLA BLA quickly first year good that by followed the with and really we'll with be the thereafter rolling half the filing we MAA shape

Operator

of the Gupta [Operator from Securities. Instructions] next Your question from Anvita line called Guggenheim

line Your now is open.

Anvita Gupta

could the hope Batten at on CNSA both many able about the and follow-up CLNX for quarter CLNX? on congrats want in your FDA how data in the that My guys, is worth in to question maybe additional from a you. how extent we you and see patients the October be and you path see and is if What CLNX programs. the Hi of discussions all talk are of forward the does will the of data long regulatory Thank doing to well. the

John Crowley

will comment that if FDA. a Anvita are child I the to Jeff CLNX this fall engaged both we and neurology ask discussions in in on with Yes. data we'll see in CLNX for moment the and U.S. again

to to XXXX. discussions. we we not expect be on treat commercial want year comment scale material. to year I'm said available first able That will the additional half to, very material in to with our that. ongoing next We of going know be this complete We've now that the share early we those need patients and always to

would it expect then yet great would that living for BLA be this people to filing. answer a good duration be want have in relatively will the a and number on updated we with for and of have that data both a we the really see? CLNX for will I sufficient path don't we But what an those So continue patients data of program be small treatment period gene two CLNX that therapies. respective comment to is the with Jeff, CLNX, confidence do being with on you

Jeff Castelli

Sure. Thanks John.

presented the year Meeting been eight from a anywhere and as reminder Annual two that just the of we first data with that patients was at So years on to had one follow-up. CNS last treated

we'll that this two one XX majority have in year treated of of follow-up. of two patients to year be data the and all So years follow-up will

we'll kids natural context data history. to In comparisons would have put to these as more history natural data into the also expected the in XX addition, from been on have based what

Operator

Gon from the comes next BTIG. question line of Ha Dae your And from

Your line open. is now

Dae Gon Ha

Great. Thanks very questions. the much for taking

[AAVX] to looking was but this the can you press therapy John for percentage toward Sorry we to, maybe about mutations second Galafold, us as maybe understand a question And your everyone's label XX%. the you when help Pompe I that label talk included can And if bit is of the or I in in EU patients that you little look what X,XXX missed now number gene plus internal release [XX genomes mutations. your encompasses, wondering think forward some provide to that context of such insights updates say XX% the I to of goals the outright, the than discussions to as thanks. per as a concurrently the as any Fabry was vector to previous KIG] pertains to it then less regards eXX number or vector FDA with trying avoid as

John Crowley

Sure. All right.

you squeezed in there. questions think I three

apart. me let those break So

the mutations. accelerate with help they size are the identified. relative of market to this the amenable mutations for here, or On the part us patients once It first the those addition treatment will not that just increases I would expect percentage patients

living to amenable with an of are So living half Pompe with we people still believe up disease about mutation.

to when that again the want FDA the So on Bradley any we comment expect necessary where completes those label. would work you with do expand label mutations? there this to additional on Jeff's update

Bradley Campbell

slightly Yes The different John. is a FDA process.

as essentially essentially in are of we So that additional batches doing been mutations adding Europe, identify. had we we're we

but a different send batch FDA large So that the with been this into patients year a have identified it's we'll new in regulatory process. mutations quite of

updates. we as those continue So have that we'll to share

as as this into these think similar access next and a is we other geographies John really the I We States facilitated did thing said for But course kind are that'll place update with the of year updates United some do of and most well. over Europe provide patients. the in to looking global as take important outside

allows the So for faster the rather and process much this patients individual through having website onto to the getting of physicians. or of and than new go into it to batch mutation really the label that be characterization then kind

John Crowley

very needed to around said the gene also all question with and of much our Pompe one in nature work our disease therapy of and your Pompe it's this that if properly to three around but the Jim and collaboration gene that complicated relative that that targeted needed to two then formed it We was disease. gene a with incredibly the like remember We've the delivery Wilson systemic it well. gene you And make therapy nature the given original collaboration not about years, the Pompe was key in therapy an continues our of of ultimately compromised back and built that therapies, we along progress is disease for likely AAV. of Pompe, only needed Particularly our collaboration patients initial of deliver Dr. One programs element sure safely. belief given disease foundation need do Wilson. the we effective could to we going

we by low the we've always gene enhancing a targeting can the through targeting been enzyme the that have dose enhancing therapy of so focused on the produced that's lower or that as So as have novel transgenes we the possible. vector

lot So difficult Obviously you believe this? I with is what to want field been that this therapy and from of in targeting in the space of comment think gene only news safety. the there Jim highlight that we a importance around others and around Jeff proper maybe

So Jeff I'll turn it to you.

Jeff Castelli

the we that's as beginning a that keep the possible. targeting as been based the existing well noted We our all guidance kilogram more therapies PENN in clinically. XeXX uptake systemic as in still think at a for on previously to our been based on went into had range an or things approach low that's design upper We've either better ceiling. gene gene John John. based of has colleagues ceiling per doses Pre-clinically are reported data. from on as to from likely range said as stabilization that potent the in therapy Thanks Sure. that we

one five that will committed remain keep high potent in weights through to that and gene process think we trying people study from we established dosing we out more as really systemic learning there has to therapy but so you and make more more hopefully think very range eXX some scale how the recent can across ages kids to data be therapy the figure dose authentic the to systemic of safety. I kig per gene

Operator

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

Your now open. line is

Ritu Baral

to outstanding but reading manufacturing where guys, completed follow-up. CMC much presentation Hey think there too thanks portion be your you CMC wanted filing? maybe you I'm it additional in the are I taking also And are did for to still sources into ask activities I John, -- the the that on of outside imply before China? BLA Thanks.

John Crowley

of will let we're comment. Bradley both do really get I but Yes. on Brad good let, in those? I'll these place to, do you a you want

Bradley Campbell

sure. Yes

much we one, our and to agreements. straightforward should PPQ the had manufacturing On have process of all upstream said that, according do product. PPQ and analytical spec sections We was lyophilization more and that first and complete that's portion that's the step. a here were It's at the kind compatibility a That's of previously released shortly. the manufacturing drug last the process. to all be was complete that much product runs underway drug process faster the

the and Of for hope we And we protocols per track you and agreed and package to submission I previously to with John course the question agencies. together that's pull to have when all on complete the that the timing rough section. a on think answered that

of out well invested discussed that you material around years into the the the our of similar facility and in facility first in previously coming material we launch China licensed complemented of of outside then that of as to intention WuXi, manufacturing by facility come of Dundalk, manufacturing first couple a is as that In the would, WuXi is Ireland time and capabilities the China, underway Irish, and right the plant. so the approval, the we facility terms the of the of anticipated commercialization be WuXi in for would manufacturer get be that has out would Ireland

a China hubs down appropriate you we've to of manufactured the already though you'd to then product the we in very two that times product outside process really done sources sure also our into the that of perspective even maintain for of but where So worst that's important have supply also to impact. part and quickly the through COVID we've various goods. regions was of would continuity from begun challenging able in the of make of chain even been level supply uninterrupted remind site COVID had moving been I very has and how various regional

John Crowley

to at we've capacity in provide on to have all that that make plant. as of timeline remains and track planning activities next to commercial so plant and and up to that the that Ireland the on we're everything we'll medicine be remains laid well able build support dual BLA hopefully you moving continuing as again for now everything well. sure both is to sources visit noted the to part that the the previously track clear scale the out with week section as actually far manufacture was the CMC that remains that's we in Thank to Bradley, Brad launch hopefully patients of Ireland need forward as the that inventory of I supposed and on so be

Operator

question of line Gupta next Anvita from your Guggenheim comes from Securities. And the

Your line is open. now

Anvita Gupta

guys Hi follow-up. the for thanks

and have, in-house? in-house on capabilities on you. you the model if earlier the but on we manufacturing if up your side manufacturing a I if hybrid building a could on therapy Thank in missed question manufacturing efforts Ritu’s see probably and Just then gene and comment therapy following I completely gene apologies first might this

John Crowley

Pompe Thanks space, therapy Battens Anvita work their the for gene the a will CLNX, instance other review on our including with know, working growing on manufacturing. materials capabilities materials Brammer on We've impressed Batten's as ago their gone They closely the with therapy in commercial been We're the exactly the ultimately exhaustive initial our years now two and including we chain contract we Thermal very nationwide enormously as have supply our programs plasmid supply evolving gene capacity. producers through began partners children's. at working of manufacturers. we're us them for for relied also that's you to and by programs. Fisher. with an in material I and entire the We basis what in supplied CLNX for then

facility. sites While manufacturing. we're manufacturing own we actually doing a evaluating X Phase as our for in-house We're that in now designing X, are parallel

uniquely and for manufactured, suited than seven operations, manufacturing quality most done glycosylated for working those AT-GAA now through of and one at XX again having in gone at the Amicus ever something more has technical of we've scale successfully. years, up that as people to this ahead, to We a beginning design in commercial believe well say more of months will have continue facility. that about scale forward I as the for basis looking one the field if the leading to of gene you're in going to with world's you the genetic therapies, need gene manufacture commercial and scale for and been medicine Amicus companies our therapies the design and the that be also the experts be last the manufacturing of complicated of one quality world's was in larger proteins

think many and we of really now Amicus become world's to translate those for manufacturers the So we therapy. can skills one of leading at gene capabilities

So strategy remain through been carefully it's you. a Thank for. on thought track very that one we and

Operator

at for Mr. and to question remarks. the this to conference no And turn closing there like time. are Crowley back I further John CEO would Chairman his

John Crowley

really Great. strong a Have everybody. strong Everybody remain everybody Thanks finish questions. and and second second to quarter the great I a your and day. out the had great thank look hope half again healthy we a of family year. for really you

Operator

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

Amicus Therapeutics (FOLD) 10 Aug 202020 Q2 Earnings call transcript