Amicus Therapeutics (FOLD) 5 Nov 202020 Q3 Earnings call transcript

Company Profile

Participants

Andrew Faughnan	Director of IR
John Crowley	Chairman & CEO
Bradley Campbell	President & COO
Mitch Goldman	SVP of Clinical Research
Jeff Castelli	Chief Development Officer
Daphne Quimi	CFO
Hung Do	Chief Science Officer
Anupam Rama	JPMorgan
Ritu Baral	Cowen
Joseph Schwartz	SVB Leerink
Salveen Richter	Goldman Sachs
Mike Ulz	Baird
Evan Wang	Guggenheim Securities

Call transcript

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Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Third 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

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

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

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

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

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

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

John Crowley

everyone and Andrew, you, welcome quarter to conference results third call. Thank our Great. XXXX

safe remain you and and we to your we families continue last hope quarter, healthy. did As

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

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

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

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

patient the and The quarter. third including from the naive global new switch business, quarter revenue patients represents throughout starts all across the performance both

key PROPEL late for R&D Pompe out to timelines in the the read in now our disease remain AT-GAA track. Second, study Phase X We quarter on expect first onset of XXXX.

third, program Pennsylvania, well to Dr. for much in has is than with with following has disease therapy research most cash Wilson, forward CLNX, been and Batten preclinical IND now quarter, moved the morning move Jim into advanced for that achieve lead and to therapy need major we collaboration a BLA the to-date, sufficient study schedule, diluted data Within without Amicus-sponsored planned on we'll rare rolling our submission strategic pipeline, expect remains diseases. continue this sharing the and for, and we the in submission this can financing Additionally, have this position natural Through as that disease to as self-sustainability move to to our financings. on University studies. CLNX the bring Based the living capabilities Fabry collaboration clinical also Today, third and potential this our faster we our both are the programs. gene And seen people programs any candidate end first of gene be selected Amicus future from history disease. year. that Pompe of therapy originally results gene pleased the highlights announce before enabling our we the the

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

a and are to we Galafold XXXX. gene include five planning our move X, strategic people year. Fabry Slide the Gene continue we therapy will look we this of achieve Two, leading disease to to to we to Therapy revenue increasing and to as in clinical We and Galafold, will of global research are key regulatory continue surrounding this Philadelphia, product achieve manufacturing therapy These in priorities are track and from minimal for to more million existing variance Stemming for CLNX regulatory launch million precision industry new portfolio. we $XXX to approval. manufacturing Fabry, Pompe program, well with our rare disease for we programs. Batten living Turning our both Center with development, clinical CLNX new geographies. the drive activities on the medicine our advance our $XXX global advancing discussions Excellence toward Three, and

and manufacture position towards And the and is remain declared our all Pompe of addition, fully funded manufacturing we an progressing expenses investments. of carefully partners that potential we major milestones. up again, work gene underway our to manage first IND, In our And Fabry gene we for A lot therapy financial our announced maintain program. we through for the scale continue as therapy the for gene time has and the IND therapy, candidates. strong our in candidate will IND are been

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

Bradley Campbell

Good John. morning, Thanks, everyone.

revenue where We quarter, detail more favorable third I’ll our through we continuity. XXXX. Galafold business revenue Galafold our through in dynamics, of gave walk the the in total go Galafold global for growth million, As the reimbursement snapshot and continued X, demand, you And the of performance by on progress. product was John patient quarter. third quarter of driven here $XX.X And strong Slide for mentioned, our I'll the commercial start

approvals the And within reimburse or access announce XX% world to global and now of the compliance the coming United $XX.X list of product. quarter States adherence generated from the XX%. Importantly Iceland, to the outside revenue growing rate and regulatory exceed of the geographic breakdown continues of or to revenue the around Poland, pleased remaining United today I'm $XX.X the million Argentina addition XX% during Luxembourg million countries and to The was States. with of

commercial countries the Galafold product XX expansion today. very As a AT-GAA it newest launch highly reminder, to continued additions patients, of of we those sales important had approvals XX but well. expanding as support laid over in before of these just global support course, footprint This the foundation for to in strong and not and potential is which access also Fabry of future a leverageable is to

patients all above now business major interactions and largely COVID in for resilient another mentioned, the markets. quarter impacting comes incredibly pandemic with and the was in see patients expectations due to new internal Slide The of Galafold, continues third Turning patient In geographies in X, the care. our some added and starts, the disruptions the for outside John States, patient the be rate us. in strong to quarter quarter between to bottlenecks select United we the provision physicians of of as did

intact. fully remains chain supply our though, Importantly

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

you by confident include say out impact exchange. to XX% very time as belt, from we third to geographies, in full ex-U.S. to quarter operational global certain performance guidance does while non-linear. we to of and perspective, we’re so of under expect in we next XX% X% half year, will quarter XXXX calls, we same compared left sales. we monitor the and true that but our quarter show past year, performance months the see quarter-to-quarter which side, a XXXX. the expect fact, sales growth a the do perspective, of time growth increased the our XXXX, from be million we we despite third duration, previous recent can factors come million from good at see increased hand guidance pandemic's we sales close news we And great typically in benefit will continue COVID-related the foreign From top quarterly several ten last quarter the to on variety in And confident is this continued $XXX growth, our $XXX year. over continue in continue course, headwinds strong deliver From range. into year that another growth to to Of the more today, can our quarters. on is again, And in to And mentioned as due the that

now XXXX. Turning we that performance on say with opportunity X, sales $XXX behind to us, Slide several we're confidently a in years with can million path

that the And in in we previously, coverage $XXX orange XX% to we growth from by investing annual from Fabry to patents expect I've multiple compound at expect screening, addition to Europe, generate XXXX, sales, exclusivity of revenue growth get to million five As peak, as rate our and and initiatives, global billion year to in. XXXX we have which significant funding long which XXXXs. continue about into risk outlined a of U.S. diagnostic give also in our and revenue in a accumulative a IP XXXX listed driven expenses high billion over gained $X R&D late towards period. market that We've also other and us we're $X alone, operating to continued between confidence further screening and see opportunity the XXXX we working book course, way goes the globally, newborn diagnosis the in

of lot Galafold globally to to for to provide a period long opportunity So come. access continue a to

the Dr. for call Vice Clinical Research of President highlight now hand AT-GAA let Senior review the of Mitch who's Goldman, our program. to the performance year, revenue to our me Mitch? that with So over

Mitch Goldman

Brad. Good morning, everyone. you, Thank

human the standard next updates or enzyme investigational the is be to through and on body enzyme, would as and in ATBXXX cells enzyme The enzyme recombinant our intended administered R&D its from and Migalastat, development to or therapy infusion, shortly of cells circulation, muscle an consisting begins the administered stabilize any combined on active to AT-GAA combination novel alfa, therapy bind differentiated Pompe with our to and our is administered lysosomes. an Moving highly cipaglucosidase the target enzyme in AT-GAA the of intravenous body, into therapy everyone of stabilizer. is XX, stabilizer and ATBXXX I infusion to like the into therapy Slide designed action. and throughout generation GAA The mechanism before more ATXXXX to orally replacement of ATXXXX from up care, is currently replacement taken Pompe. delivered major one treatment enzyme of allowing remind distinction

data therapy of has replacement here mature people in we're History target GAA. unique cells receptors of let's late the and the the study mind, Dr. to enzyme Today, long-term be Science improved onset the over showing next disease, enzyme with turn right treated form XX. past compared his Do, with processed Amicus literature. care The decade, side. POM-XXX Pompe the the is more in believe Natural ability the as standard that data been Pompe are slides well current and Slide have living uptake the carbohydrate advancement for we cells, hand profile Officer binding from slide, alglucosidase hand study on other that by develop working of literature into alfa. Chief left the consistent both efficient These shown distinction, with those the medical to With The as to impactful our itself. on medical data the team Hung of this POM-XXX POM-XXX to in

These need [Indiscernible] We which assumptions but on for data minute PROPEL. underpin on over declines. support average over and are therapies six generally treatment, design producing it where the if on see stabilizes, the walk benefit you’re test then a for patients new time

slide, Next please.

here the for and Importantly, than been timelines ongoing that on on of our X,XXX To-date shown assessments quarter have more track completed planned is in with of remain XXXX. the this from XX% PROPEL the PROPEL schedule. data first infusions studies on slide expected

for platform and manufacturing families, In XXXX to investigators the call team of Pompe all Castelli, continue participants, unwavering we time. enroll to review to to our staff, want With studies Dr. Amicus functional Officer, filing. the in their MAA patients the gene Chief during our this commitment collective our to my to site pediatric over our addition, express and We cross Pompe advanced thanks BLA a support Jeff? to and Development to Jeff we efforts unprecedented clinical that, and supportive therapies. hand and study the portfolio

Jeff Castelli

and good Mitch, you, morning, Thank everyone.

Slide Moving now XX. therapies portfolio for briefly leading highlight gene I'll of rare here to our diseases. industry

living During after able progression as continue critical and PENN. therapy at positive and gene suggest programs to Batten our in this franchise XX has a month effect Society interim Fabry our demonstrated disease. Natural X/X been gene to therapy our science meaningful ongoing compared data The potential at months, last reported Starting time disease Pompe slowing well we've and CLNX disease Virtual Annual that including programs lead the CLNX The data to in study therapy History. as Child a in treatment gene that results disease, Slide in be to data function, portfolio, motor Batten maintain an XX, in clinical in ultra-rare Batten the leads Phase on with condition death of option to Meeting, CLNX COVID, a childhood. progressive Neurology for cognitive we've debilitating and our across CLNX with declines Batten, early and children often

at regulatory continue CLNX. for update initial to path CLNX through read the conference. early form an neurodegeneration. results a advance most plan believe to XXXX We CLNX X/X finalize expect clinical important our year the childhood for of in to Batten provide Phase and to medical regulatory in We study the discussions clinical CLNX disease, the initial common provide data next study also report in from and ongoing We

on studies material. on for to CLNX Additionally, year commercial great progress make programs remain we track using for and continue these process manufacturing to our commercial this programs, initiate next both CLNX and

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

collaboration, including Amicus this of to part diseases, preclinical rights in As currently has programs. plus five active XX

candidate a a Initial data transgene. and proprietary to Slide ubiquitous now an on we're GL-X announce that are IND selected and knockout for GLA to reduction therapy therapy, clinical XX, has Fabry stability mice, collaboration, part been also gene We're Moving then GLA for in to as that to target we wild one very therapy transgenes gene of promoter, now did for our that AAV into we very excited candidate, includes Amicus and our disease. transgene IND protein pleased transgenes better AAV as which uptake from engineered to cells, demonstrated improved show containing for Pompe improved the activity. engineer addition type able engineering gene significantly

therapy Fabry With as and the over initial medical in therapy like data preclinical full and I the gene takeaways in the make to programs, this our continue turn Daphne here on that, of presented the have across Quimi, for excited progress XX. XXXX. to our data highlighted call would Daphne? We're by conference very review to early this financial for now results, outlook. guidance Fabry preclinical gene Slide will We to set at

Daphne Quimi

good everyone. morning, and Jeff, Thank you,

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

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

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

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

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

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

the $XXX.X flat place relatively for from launch, of is programs high XXXX multiple in the with to PENN. the and A again forward, to at Cash, reiterate, commercial XXXX, research we Pompe AT-GAA gene Fabry. all to the transitioned expect AT-GAA September in marketable XXXX, CLNX, continue meeting pipeline. fully and Lab therapy securities we XXXX. and million as To and financial total And objectives. other remain cash maintained therapy development products emphasize, support schedule, our the gene about our And the expenses XX, are while few to in Going in guidance. XXXX financial a work at our that CLNX, leverage we equivalents ahead infrastructure were operating global compared non-GAAP December priority XX, our our in $XXX.X cash Wilson especially already at on comments moving position pipeline. associated costs We programs we discipline to with million

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

John Crowley

Daphne. Thanks, Great.

So, resiliency by to be the all and as see of shown, focused performance on business. have the we the proud that team I'm continued brought pandemic, everybody challenges it. relentlessly global great this despite our and they've can incredibly across about

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

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

Operator

[Operator comes Your the Instructions] Rama JP Morgan. of you. Anupam from Thank line from first question

line Your open. is

Anupam Rama

much so question. for Thanks guys. Hey, the taking

John Crowley

morning. Sure, Anupam. Good

Anupam Rama

John? you are how Hey,

John Crowley

I'm great. Thank you.

Anupam Rama

say, about me. Thanks Slide the X deeper seeing, some emerging penetration think do new ads just Thinking we of So, in the operational look that growth versus the one how quick core sort you're of regions? into so regions on in patient particular, a into when much. Galafold, market newer you about deeper for

John Crowley

question? there provide do more want sure. you Anupam's to Bradley, color Yes, to some

Bradley Campbell

Yes. Thanks, Anupam.

are U.S., mix. still pretty their it's and I launch of early sort we cycle. Japan have the in So, mean, who still

starting call what well. rate, a And in naive geographies. curve, we on important in launch large, getting those out patients the And some although new few on there. would of markets, of they're maybe you're so third as from markets, along together, seeing bit both part None are patients growth switching naive almost those which an patients and of are of an you're we a of is well. different further see I the of are In is themselves little their so the today. there taken course an of highlighted growth evolution in and particularly those the coming equal large kind there over Europe, switch really to the then expect as the story come with launch on strong but a when

is countries that for next small launch So bringing mix. in and to to new ones, of think the continue be we're U.S., a it out as the kind midsized I three years, still also, the two and of to we can. penetrate big continue the but Japan in going mature in And markets, the to many much push as very

Anupam Rama

taking our Great. Thanks for question.

Bradley Campbell

Thanks, Anupam.

Operator

Thank you.

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

Your line is now open.

Ritu Baral

the Glad taking question. hear for I Thanks well. focus guys. morning to want to you Good on… guys

John Crowley

You too.

Ritu Baral

if [indiscernible] to PROPEL so that how much the the second Phase Pompe, sorry your study not or natural new -- change, data. think how new all? Just know the staying to of study, housekeeping arm we the GAA placebo first PROPEL you wondering around the I change POM-XXX powering of history of Hi. was PROPEL. saw? contributed on powering data the assumptions potential X XXX for And one about that do

visits managed that? timing of you've about like number retain primary endpoint If of and could to the you talk

John Crowley

Good. PROPEL, okay.

Ritu, So, we been have notes. furiously taking

questions, only three that's good. that's quite think but I actually

Ritu Baral

One question.

John Crowley

parts. multiple in But

to we yes, So, answer. happy are

for So I'll again, competitor, before window potential let weigh Jeff to an filing. as Mitch a I had just there originally and a the study emphasize the me, ask done been in early was for POM-XXX

Lumizyme then we but it time. and body decline adds once adds been over looks it benefit with our known the be about entirely consistent field, to any Here, I think of the completed everything to to for that's knowledge, it,

want one questions you any can't you colors comments Jeff, we add to Jeff, Jeff? those then hear Mitch capture Give begin we you still and Jeff. that, maybe Ritu, with ask Did us second, on No, Ritu. it. and or can are to from So, mute. you, to

Ritu Baral

Sure.

John Crowley

so him video, on our him and talk ahead can we'll and Then questions. happy see Mitch I'll have to muted but. with trying system, take up go be to I the video I

do Mitch, about we powering talk and want the first what terms to So, of assumed. you part in

I the think it But consistent plan. with the prior of made to takeaway assumptions did all inform partly any had this seeing data. entirely we is building

Mitch, So about you let I'll talk the powering.

Mitch Goldman

Sure.

it have right, along us house really the to data we think our We to I minute that I and walk understand in study. use John. the sets the Phase point, mean, outside moving really largely the the data with against time variable helped with see. variability six it's that X would the variability in of

study end So XXX how on at deliver the the versus landed PROPEL we looking of six or XX a competitor so the XX that's meter study with minute patients improvement walk alglucosidase. and to to in

John Crowley

thank Mitch. you, No,

the second patients see to essentially think This you a this, did worse we does one believe we of meter arm, flat so to XX the studies, than That XX our Lumizyme, powered. XX that that at they based significance study further patient is delta this control we those where is X about that to PROPEL think us assumption. around question, our for looking was meters But superiority. It’s naïve. XX% assumed study know our they switch, while it so we about Ritu, data. or well we there. their to we the you is gave this expected part once to so, improvement neo patient look neo we in know, to I had arm, just that was what statistical just show And I I XX% respect and was would extremely began. your a speak won’t on saw or more again, year, somewhere naive the meter population. naive like what confirmation in with all Again, again, interesting And XX need on referenced

the So study Lumizyme arm add what further just whatever saw to be would were our if, of to in it in of study to to closer as the the the well. reason and we our PROPEL study, patients randomized neo, powering for control

again, of even getting By off The we’re around lately. third infusions, but and aggregate of see patients of in pandemic getting your more all in assessments their of the late spring, April assessments than their we did assessments still have to XX% course, And some not receiving and early all seeing, the their third only part infusions, we the back of May, their the then your timeframe. the patients, of look were, question patients part of assessments. were in and the March, disruptions question of the were the the as We sites gone we’re planned. height of and were we're at vast X,XXX majority well still infusions those mid

the we pandemic. we I would that's of the integrity assessments. think again, And here. real very in missed for be a plan of And place to course, without even high. of team a testament study assumed, infusions, So remains you And with than the the we're actually missed study, any the pandemic, effort better in a

Ritu Baral

making Great. rising an cases right adjustments now are logistics, And are you any as increase for in guys before? pandemic-related and

John Crowley

And patient now for again, blinded master PROPEL is every patients of the to of of study. have have course virtually majority tracker We patient. week. every We patient December. got scheduled follow of every activities all that every completed. in out of set Last that, vast but remain completed all We've a patients

Ritu Baral

questions. taking Thank you so the for Thanks all Perfect. much.

John Crowley

Ritu. you, Thank

Of course.

Operator

you. Thank

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

Unidentified Analyst

any steps data? dialing remaining remind to taking BLA question is are on there just you Julie you submission My complete key Could in I'm once question. if Phase for Joe. AT-GAA. us rolling Thanks our X your Hi, for have your

John Crowley

Sure.

activities of quarter, submitted those the preclinical and by first have have module, all end of module, this the will We completed been the completed.

rolling the begin will BLA that process, So, Julie.

all We both and are complete We've manufacturing parts completed the the PPQ downstream. of the of with the upstream nearly it, activities.

data. final stability the and to on the the data, that expect all we the all some work And once module. the reports clinical update already important PROPEL module And of So, we're for we necessary with final have that CMC time and would studies last course, drafting clinical module. of

are So receiving other no, for the no there PROPEL us than data. barriers left

Unidentified Analyst

Thank great. in hop back queue. I'll Okay, the you.

John Crowley

Great. you, Julie. Thank

Operator

Thank you.

Sachs. Richter Next, have Salveen we Goldman from

Salveen Richter

morning, my for thanks Good taking question.

as around target And differentiated need the competitors Battens your moving a here. order see really of two their expand as few kind the then into construct around on program, forward or it in you versus protein forward So, an and candidate what discuss or regulatory understand approach move to we how the out gene portfolio. others Fabry do One And therapy we've that's had a pivotal to wise stage? well there?

John Crowley

science can that therapy the speak we in and to Thanks, Fabry the gene differentiation, unique come maybe into to tech Battens Great. what and Hung, went then moment. that Salveen's took, you question our work protein about can a you and Salveen. the was the

Hung, ahead go Let's please.

Hung Do

Hi, differentiating Salveen. we approach to Yes, the else gene it for our John. how actually question is approaching Fabry everyone therapy, is that. develop and between quite your Thanks, Sure. about us how

and and expressed to I that of foremost, it's intact stays functional. subunits. is as it stress dimer I that two a for First together imperative think protein importance the And of be that should dimer identical

while blood targeting were you activity showing that that And as active to panel the produces transgene, various so able it protein important, with to tissues. slide, that secreted we middle is dimer do to remains expressed the a its to a that our of in into it so that's And it's prior been that to it maintains we've mind, maintains formation, its and what the ground. engineer

of huge increase been and the essentially, a where that activity potency, is protein. have we've that so advantage to that we able seen, And

specific So, is be effective cells, substrates. it much actually for that potent when to actually more and able the targets to clearing

that the the maybe through more far as this as enzyme, again, is gene So transgene. potent we approach only therapy know, is

John Crowley

just the effort A amount focused comment and gone Children's believe quality. potency producing the commercial, spent this I'll of commercial and all questions They to the has development, and briefly transfer from Brammer. enormous successfully Fisher assay scale the Thermo tightening will be lot what into of the now related we an at year, analytics. to commercial the We've and the Salveen, are work regulatory. of then analytical Again, around And the parts technical your these time operations we've completed on programs. CMC Nationwide tech of

both terms comfortable to dose time key And figure with CMC interactions scale of what of of And operations going interactions, components you and these side, patients. the CLNX the bodies make then sure in on material out of follow-up to and from our So, amount the Fisher. of technical regulatory of the quality those patients are clinical additional commercial for to regulatory good Thermo as both all for right regulatory the and we're that with a additional going regulators CLNX. know, are the diseases in now, part work with we're is to right

look to the earlier in and first CLNX, And patients the be of four time, the provide number to material. CLNX, point part For with the only what patients For amount of then Jeff's of dosed is data early clinical nationwide at well? particular, that XXXX. able and as we've we'll in

And is that And so significant in So numbers fatal will patients we’re diseases dozen know just then the again, that brain the Batten largest two among disease. that, We very genetic diseases largest study forward and be much in our of is, a need regulators. move children, so of children into and guess to there. of around with the with somewhere or suffering the looking the CLNX. pivotal CLNX we to horrible

programs neurologic our moving Batten as preclinical through in in that we're continue possibly pipeline. as programs fast So can other we those and programs,

those ahead the more next lots in So, of couple on quarters. programs

Operator

Thank you.

Citi. the next Your from comes of question Mohit line from Bansal

Your open. line is now

Unidentified Analyst

and for there, on Thanks congratulations Keith quarter. questions, Hey the is our on Mohit. taking this for

looking be the Thanks. just the you they decline very of from be to avoid no to CLNX long-term looking we're than data, towards at seems rather would about the expectations more efficacy So, over pushed moved sort in at obviously seem out but cure It’s therapeutic. what for are gene and a then mid very bar it expectations away early, general, How entirely. therapy have and thinking there for of the this therapy? And probability the long-term for time?

John Crowley

their reverse doesn't on that, tools expectations. better outcome based high these very technologies. they're diseases. the continue it that that for as you whether awful in just and Yes, have seem you today's children patents treat can be. you unclear progression, Right neurodegeneration, And you ever it's can now, disease the it will really programs the Keith, know These candidly we seems earlier to devastating

we life. We important add something and be and guys but that think children therapy, targeted of it's can some quality with over So after the of gene but or curative so, a potentially a to soon really children Mitch, be the children gene therapy, as brain you want more damage, administer therapy? safe of to optimistic still these fundamentally have identify lines time. birth for seen remain the in properly without if cure still much to to as you Jeff, their significant can you color? changing that for difference Will younger it profound in

Jeff Castelli

hear Hey is you John, this Can me? Jeff.

John Crowley

paid bill. your You’re Good. phone you back. Yes,

Jeff Castelli

Yes. you think the close treated severity normal some whether as impacted other I what if the for with can the cure One largely neurological already that's addressing really factor be versus it's to normal, these disconnected and can they're at before. apologies life, the live not therapy. like impact can you're wrong baseline there comment John we done, that underlying thing getting remain really gene time pretty that's that genetic progression call My with on, so due kids still gene to will therapies a damage would there's mentioned,

that's So, that big a factor here. we think

blood therapies, In for of the therapies the terms we gene of some issue durability liver with of disorders, and that's gene an of example. some know directed

a should hopeful could turn are data delivery, therapies expressing not be over, we so really expect therapies of lot of turning cells CNS least that suggesting the for off sort that that With of these durable. there's or really quite CNS There's durability gene directed do gene be good. really any at we

Operator

from next Thank Ulz would from one you. Baird. The be Mike

open. is line Your now

Mike Ulz

guys for the taking question. Hey thanks

John Crowley

morning. Hi Mike, good

Mike Ulz

morning. Good

follow-up curious around by quick you sort a December, John, created timeframe Just unique the topline through on the just any results? challenges that you so And in the timeline extend bit PROPEL potentially about that get last talk Thanks. can there? maybe see provide little a could and of mentioned if I'm the to kind of patient turn dataset like COVID study. if you and the

John Crowley

We've be Thanks, Mike. would always said timeline. first-half tightened of it we've the Yes again, XXXX.

first through cusp out, ourselves But into data to clinical all some in of confident final quarter. the lock we're but cushion data patient would data scrubbing and integrity procedures also should we if that some is that point, the in by last time, that. just sites go very operations, theXX it anything that as that patient infusion so all confident database confident respect protocol report We the early I last place of weeks or XXXX, QX. for the through assembly. with some that, receive challenges the we’ll have protocols to narrowing Now be are ample there if data part we'll into. teams the we're of why go at we to get as on think see to that out don't that foresee That the the QX the able At will the assessment we're that that and to in be in clinical of delay and the COVID And that in gave our once even we'll research operating shortly database we have our of all of standard thereafter we'll we're December. that's

again, we the expect we'll the that place to in file really that So, report be to out submission. in United a QX, think I good complete in by States QX. And BLA

Operator

next Evan Thank you. from from be Securities. Wang will Guggenheim The

open. now is line Your

Evan Wang

with question. it grade And broadly. had between respect to sort therapy guidelines gene utilizing Hi. is seen question out especially impact difficulty work agency with this if And some of compared respect Thanks comparability, towards taking been Thanks. for I to its commercial a with we've with the to on Has building any growing the requirements change industry? FDA, CMC for communication house CMO? grade. with regulatory respect there gene to clinical therapy capabilities pain some the in or to CMC? Or

John Crowley

process world's product. Yes. gene therapy, one process companies, got the is of In if the going broadly, to Evan, leading therapy world's be leading respects many science. I gene gene just we've is also the think therapy companies the one Amicus in state I'll in of become to

at actually So excellence enzyme we biologics commercial among have here, produced. succeed program, the complicated experience in Fisher Amicus is quality, a on have Biologics, then we in That it. And for Thermo sophisticated CMC, glycosylated Brammer team. scaled partners partners operations, AT-GAA. suited with with a these it, therapy all been hyper again, technical uniquely WuXi our in applying Wilson and I UPenn team his together have most with to we with But also just it all focused and Pompe And that Jim lead regulatory successfully we the ever based on at in other programs. is think, our that have of CMOs, we're produced at experienced With scale. really and Batten

continued CMC around months been gene in tightened So it'll and requirements, XX much of think a for has have about the environment a evolve. thing. advantage without the much therapy. analytics manufacturing lot framework I the question, I a at strengthened your actually the good for standards Amicus. actually and of analytical work To regulatory great think that's be CMC, strategic manufacturing to FDA the on There the the requirements And the part components, last side. question to XX regulatory

so well. but with most assess gene I think shot important it drug get patients based to developers necessary, can for was and one technologies as so patients. we for different, to is innovators have at on one AAVs we've also it efficacy shot. are understand it's what fundamentally the think right in And particular, least today's we're as that patients I important exactly safety, therapy patient providing Again, we going got

So focus that's just lot a lot a the focused we and of why are resources of our hyper attention CMC side. into

And also early of able expect world we're art clinical commercial describe of class very a of busy in we'll gene thinking that stages facility fully. more in at as scale that designing would the the early We a state a lot be work facility. well. XXXX, to larger are We therapy manufacturing through

I and key for lot working think next a manufacturing programs, behind develop therapy work of in So really, the generation invent, some technologies our to and gene ahead. of scenes technologies all the including of CMC investigating on

forefront So gene to manufacturing. need to the of be be we really, really want we at therapy

Operator

you. Thank

line comes from the go Please Your Gorski ahead. Cantor next Kristin of from Fitzgerald. question [ph]

Unidentified Analyst

congrats and taking Hi, the progress my great the made on over everyone. you've Thanks for last quarter. questions

I you looking including your higher amenable of what label are of for the detail U.S. disease, therapy you key in really your as program, advantages understanding then safe remind the some identifying versus peers, on importance us mutations Amicus the targeting and to on wanted past discussed and know trends the in of at So, And gene to And of differentiators that how Fabry would the view a Europe? here, transduction you as ask your the more approach could the as well heart to organs. in I course, of other Galafold? market, through you've the key and

John Crowley

to you. the in hear may do Great. to who first suitable part from Galafold? Nice Kristin. patients want take more Thanks, you about for Bradley, be identifying

Bradley Campbell

yes. Sure,

really of pieces two that. there's think I So,

we announce GLA saw you One we of the last in the the that include did as gene. them, sure of expanded European potential mutations all Kristin, I'm to quarter, label

label have Europeans imitability. European the with that the And label, a X,XXX been European that again, the have label. in really took In the with the internal the able with that who great to the do new we preclinical of diagnosed we XXX creative And United mutations mutations that to update order from sort update of different. is label. in ability little bit or science to for have. team we actually so were There, to that determining process approach So, patient we went the essay a the mutation States, with just a to has

with and is it's ones growing mutations. United kind twos patients but in growing those in So, as you the of that number States, find we see

that's piece So, of the one puzzle.

of amenable as use. to genetic patient with and newborn at that. the before interesting last the promote find studies is, minimal in MS data, particular identifying talked you talked under And some five can look other in four done also helps looked at a Fabry maybe the And diagnostic exciting piece talk new some doing all course, use patients. then, artificial and a with of testing. clinics. Fabry course, include the in and through that in Fabry the at in GI looking the the We've screening piece with really looking for mutations we're a then think the family is more first the going And puzzle, There, we findings more every guess on about we're both of the they that investigator improving in of We've And I work an of clinics. to bit you undiagnosed intelligence, typically United [ph], company finally, is around them going Fabry exciting because initiatives diagnosed ton there. then work. with disease the Fabry States doing initiated about closely year, about the the we've diagnostics. world. of members an hope the there we've we're disease, of I some NBK things well. panel, of And some panel which we've at find PENN excellent And the part to around little it's we next called And partnered Fabry

going So, work on of a ton there.

a significant everybody also medical more think in see identifying with think I the in continue focused to I and on you really field increases diagnosis that. is that's why And for patients Fabry, mutations.

John Crowley

question. Hung and few maybe want to guess therapy I go and gene Bradley. tackle ahead the Thanks,

Kristin more the important bit gene such be again, thinking approach ahead? in Salveen's doing, it I you level, provided but specific I complements explanation around we're a an for is of how continue a going therapy. part why the think Fabry, high questions this reminder Jim just to what very And to an what doing. about excellent question holistically Wilson again, overall Hung is think business Amicus of is

Hung Do

a you years stability importance that, community interaction can help John. the and stabilized those Maybe we're therapy can with the combine us our add for that but Galafold our and ERTs our learned and lot of in development Sure, know has for. and improve we I the gene that with profile that I'll Chaperone think there. then And with Fabry and about over in that We've do you helped experience just start obviously to for Galafold Chaperone also looking proteins, proteins. not along,

that market, therapy. definitely oral Galafold safety switch are is burden treatments in one an ERT an don't time high molecule, very because Fabry do think So, there's a at issues there there's that today. you clearly a gene in the obviously address But convenient to when really, of patients to of the think on important terms lot in do have do we for pretty We therapy, a opportunity the existing look that a disease patients an option of gene therapy. are

like we the you approaches need out but is, matter approaches to our cardio the when the liver main at therapy, targeting So, directed or given either are no address, tropic clear gene a approaches, and looked vasculature. the specific what, organ kidney, Fabry is what's skin, there multisystemic heart,

at when and them. transgene, enzyme that and really Neutral that travel through so focused optimizing blood, cross some stabilized to going where is some You need some that the secrete to have the you it's protein correction pH. to And into tissues very cells. treat into And on need main to then transduction to also stable correction of out cells the put those we've are in blood. the But element now need cross the gets to other going get cells

stays believe. As more in gene therapy And gets it very potent and tissues active. it made into we we've cells a essence,

that blood, whatever have into small of with put efficacy. get that it amounts out optimal the will that enzyme So does

to that don't doses go start need concerns. you to to So have safety

think we to correction. construct add? about approach excited learned we cross anything improving the here lot about Hung, a this stabilized have are So and really for

Hung Do

add one there. last just Yes, from I on explanation Jeff's thing would this,

cognizant efficiency is down. in preclinical just in practical very way the view point primates, humans, ultimately goes people. a particularly I of that that studies know We efficiency in of the we between think transduction then too, from are and transduction

we dose were to And achieve actually develop, be going we are effective. potent certain that, so that enzyme with to are that we very efficiency drops of able in that's level to and the the in a trying produced, mind, when so humans, what in it's protein,

right and all hope quite again, weigh are differentiated, factors is be of this terms we in So improved these kind methodologies how over believe current quite to of now. we developing which we the approach,

John Crowley

guys. you, Thank Great.

Operator

you. Thank

question Cowen. from You have Please your Baral from next go Ritu ahead.

Ritu Baral

for given the the that little capsid? when XX, guys. taking Pompe clarity proprietary [Indiscernible] when both you back gene a talking clarity MD what the Hi, follow-up. question running you're you're these liver thinking with Jeff programs? this a the about studies, what potential talk is, get Slide Fabry And more and seen therapy, away on give issues might excellent no on truly Thanks we've with how we these the proprietary? for safety proprietary And could about TM makes little capsid on Can the guys Fabry activation, in about second My time. you bullet, for seen AAV more

John Crowley

speak you to want me to Hung, Jeff. Yes. Sure. or

Jeff Castelli

in. chime Hung then start I'll John, please I guess, and

me you this hope avoiding not I can hear Ritu, time. I'm So you.

or one these this those seems transgene have effect necessarily more we try capsids very proteins. think So of broad of would it's our little damage, gene risen on liver proprietary specific still this the the to DRG and AAV. from that's on the These specific broad. the a specific broadly a address point I challenges UPenn. it the AAV high well collaborators to AAVX, concerns safety the transgenes differences. But capsids coming of dosing, aware this think and It causing this to and having some could or concerns are systemic specific We by in various not of type at have might Generally, be people likely impacted capsids, trying to know approach field slight some concerns. we're like toxicity. be that AAVX, We're well, up it AAV potent therapy toxicity. by manufacturing processes. some those And have maybe overall, are

on we a level. toxicity, like to we in help DRG through So collaboration dose, push There's types need technologies things at well. working collaborators to technologies have to as reduce the access we those that early the are also keep of on our don't safe and PENN can

but not transgene. disclosing itself, of having main when potent though, on concerns So it in we're Hung, terms and add It of capsid provide anything those one through is we common to yet front? addressing really the we'll that AAVs, a what cleaves can. we're of is. the But -- specifics the

Hung Do

so think, last one that capsid this actually that, one of broad that organ point specific just here pretty we're The And is I transduction on has kind and any efficiency, not using on touched tissue. or reliant earlier. Jeff, you we're

transduction And for allows so and broad protein this much correction. we think more of so expression get that that effective having the we secretion a can cross

again, to this effective a very more going very think, say capsid that it's we lot we in terms is think I So, to but gene AAV what I the are have of for future, in particular a deliver therapy. confident, now,

Ritu Baral

guys. Thanks, Very helpful.

John Crowley

Thank Great. you, Ritu.

Operator

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

John Crowley

Thank you, day. Have a for Great. great you, operator. listening. Thank everybody,

Operator

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

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