FOLD - Earnings call transcript Q2 2021 | Amicus Therapeutics, Inc.

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Good morning, ladies and gentlemen and welcome to the Amicus Therapeutics Second Quarter 2021 Financial Results Conference Call and Webcast. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Head of Investor Relations.

You may begin.

Andrew Faughnan

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

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

will we have Chief Q&A, Mitchell for Goldman, Officer Medical Joining Dr. as well.

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

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

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

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

living And among the I to him across to giant as deepest at his pleased death contributions, that of his University our I our many Dr. the industry. consultancies priorities we I is Jim with of in advocate a Yamada. with he family. you people passionate honor a Amicus know, rare execution to serve at beginning the had the sudden know quarter where to note the Center Disease for he strategic of the diseases. and I that Orphan to was year. want of for yesterday well with chaired XXXX Tachi, our report the second Before begin, we And outlined for Tachi just Tachi Wilson reflects of am Pennsylvania, as many research came

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

the the have the a that XX announced Commission European amenable first above, an number quarter continues treatment with of With community The Galafold to and on older the the Galafold $XX remains from continue years. and for expanded mutation. now very globally. second treatment at pleased we patients our as of end First, and label for a disease Fabry our both exceed pediatric continues for quarter XX with Galafold we the add patients EU European strong is moment for Fabry week, over momentum This this aged throughout long-term time we the patients and XX in naive adolescents to transformative Monday external Galafold success. approved on of our in switch uptake expectations Galafold, in has And for where cornerstone exciting the quarter. populations revenue, aged news of new are in – the internal continued new million performance the the the its Union, of option

the today for have track, gene I therapies NDA BLA pleased of continues AT-GAA, to to submissions completed. our initiatives regulatory share and for remain novel disease, and am Pompe next-generation key two-component for advance. therapy the pipeline on our been Second, AT-GAA R&D our rolling that

repertoire submissions the a pleased following share AT-GAA. and co-repertoire that Application, meeting, or MAA, for to EU also of regulators are We positive Marketing are Authorization supportive

We in year. of these EMA submissions second the this complete half expect to

with programs. in lead We our move confidence these broad continue well intend AT-GAA with through as to to to our advanced research regulatory towards seen CLNX gene have we achieve clinical and for have cash position we the the Dr. current benefit great and the and sufficient highly most people programs CLNX pipeline, continue disease, self-sustainability Pompe for our gene as our third, without Within collaboration submissions candidates. financings. And to approval. data through living rapidly any preclinical it far the and is Jim therapy we to disease further therapy preclinical dilutive of therapy University Pennsylvania, and Pompe need And under the future the continued from Fabry plan thus Wilson we progress are Batten encouraged gene by Amicus

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

we X, key well our on are to track strategic achieve priorities our include medicine for Fabry disease. These five for Slide to precision Galafold, XXXX. Turning

Fabry behind global which Galafold’s to to variance $XXX demand million revenue drive markets. medicine new achieve more amenable and to this existing strong We product this will reflects people in $XXX precision with of in with globally. momentum continue disease to and million look We availability living year,

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

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

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

Bradley Campbell

good Thanks, everyone. morning John and

walk the I Slide you in mentioned, for give will our John our On commercial As detail we more now X, of progress. quarter. through Galafold global performance snapshot the Galafold

of million revenue the remaining and quarter United by States, rates XX% the million the United XX%. total continuity. generated the outside $XX.X product continue starts patient was demand, patient $XX.X of XX% from geographic adherence with million was within exceed revenue new For $XX.X during driven compliance business Global and the breakdown States. to quarter, strong or The revenue or coming

XX-XX the with as As before, this we’ve business. we to to both grow global see is split expect in we that the mentioned parts continue line roughly of

efforts. quarter X, to patients global Now, was our major business all commercial The another great incredibly QX for be resilient, with markets. our Slide turning to continues added in of

year. the into the patient the greater June second since best in starts in patient momentum fact, XX-month of rolling going the launch. average And which the starts than should new is month include average, the X-month respectively, net and globally, X-month our half In was of month second building international market

and from demand and orders increased a Second FX of quarter timing tailwind. slightly also U.S. reflected continuing in benefited revenue patient geographies the ex

in patient point continue we due at delays care of in QX highlighted to new call, treatment. COVID-related our identification some see we starts sporadic between to and of As patient initiation slowdowns the

Importantly, though, remains fully intact. supply our chain

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

to our second so ability field monitor the observed to personnel continue many of with in impact, but news the we We saw an is in pandemic’s interact that physicians improvements the person. quarter, good increased

we line the first The quarter-to-quarter XXXX to of versus while been have a continue in have of expectations. quarters our factors, year, mentioned and previous year rate we typically last of growth nonlinear. see growth the with calls, is variety the same within due to as on two quarter expect from And

second far XQ stronger and XQ quarter, performance a now quarters year, first given so great here year quarter. in a than confident in of that second anticipate revenue. global in continuing guidance full the year momentum $XXX a So this and COVID, half and XXXX we’re meeting continued from this our in to recovery and Based usually $XXX third pattern million on has million are quarter with to

we XXXX and Slide Now the which out X, have and beyond. on laid in drivers of foundation the the metrics, growth called for several

ended at a about we’ve global and amenable are XXXX market of treated X,XXX-plus with now we globally Galafold we mentioned, on share patients XX% patients. As

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

remain also amenable We and the significant Galafold, uptake patients markets, patients numbers And we’re variants. XX% that in switch naive stronger – on diagnosed have in XX% there see know untreated mix patients. many who global of while to naive are starting remains about who

see reflecting As XX-XX. favor term, see we’ve naive switch patients, in long about expect to further reverse And rate Galafold. percentage the growth few that to to we the for that said years, patients would over in be of of market previously, we naive next in expect

of of insurance a in to by outside that The reiterating underpinned recognized and negotiating reauthorizations now stay who been and go that payers payers reimbursement strong renegotiating also those is belief adherence the to continue that XXX% patients has XXXX of and Galafold Galafold. compliance granted tend record very our with nearly U.S. track United Galafold of XX%, value exceed States. successfully All by with of rates on on

label the focus upon, in Commission a John to on And briefly relentless population. ensuring to strength. European Galafold approved continues that touched adolescent thrilled Galafold European announce expanded the as just Our access the to I’m of be

further with for Europe, XX% for to to have only long-term and new old treatment is, mutation treatment eligible secure treatment individuals amenable to which will approval So geographies over allow of the of in with X% course, possible. us a as aged therapy patients first approved the as in quickly regulatory the time and authorities closely with of work we’ll for XX XX to the XX continue This other expand to eligible disease. Galafold Galafold, oral years, access an to option population Fabry very now, years

at we In to to Fabry to markets to Asia and patients least reimbursement Finally, for another amenable elsewhere. globe, Galafold we is to we XXXX, X traction expansion. around countries Pacific driver expect expect secure newer access and new look and Latin variants expand important see continued continued the with as our America the reiterate in growth geographic of in

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

multiple in demand in the queues building some worldwide geographies, recent headwinds geographies. never of has for certain Galafold COVID-related potential So new with stronger, Galafold been despite patients

$XXX global We of confident million sales. in in are full our guidance year to million $XXX

continues continue path next we part and as than As that and to to the generate that next even several X to with post we on of the us, expect X, significant patient Slide confidently milestone the Galafold. annual reminder, the quickly number be OpEx starts in second timing to growth as return which X will years weighted will approximately over $XXX can continue of to record a will funding of in in guidance, the do over we patients – million for in $X of be a greater corresponding contribute dependent important of this XXXX, our of and towards normal net cumulative COVID will The R&D on in to but COVID, years, to we global a track ease. behind On milestone the a this things say year period. the how and half year which revenue exact expect we’re impact be new project performance revenue billion amount years revenue

over in We screening, diagnostic provide Galafold to reminder, the orange other a which we $X addition diagnosis XXXX. coverage our peak in continue newborn continue in to the and orphan which for opportunity we the we into late globally, And and initiatives, from driven the Fabry give us Europe, also market have even IP revenue XX further exclusivity billion screening of patents high-risk as invest have confidence growth that well. as in as U.S. through protection see significant to support book-listed XXXXs, in and continued finally, by XX

to continue to of globally to access a for lot long opportunities a Galafold to time So provide come.

now turn me Development will therapy Jeff with that, Dr. let our program So and to our call Chief highlight Jeff over Officer. our the AT-GAA pipeline. Castelli, gene Jeff?

Jeff Castelli

good and everyone. Brad Thanks, morning

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

a approved most we and with for despite need one unmet to and recognize disease is fatal therapy First, believe it’s disorders. market. always Pompe, living lysosomal Pompe on disease prevalent having the the remains important one the significant people neuromuscular that of severe

individual being decline followed In in on patients highlight addition we’ve patients. to of for treatment of natural the history studies seen human of that publications benefit numerous multiple the by measures Pompe key tragedies, continued disease long-term initial many

As ERT-experienced X setting a been PROPEL studies controlled patients need All in a for our reminder, studied have sustaining to unmet only in treatment. Phase been the high Pompe clinical other controlled late-onset disease ever in participants naive trial. has

now presented Slide endpoints at on in International XX, the key we of secondary May. a present biomarker recently primary summary Diseases Moving the to Congress Neuromuscular and

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

consistency in the disease the believe potential of for ERT-experienced address We this patients Pompe overall unmet endpoints of see to all can function, Pompe. of outcomes across favor AT-GAA of domains pulmonary that the motor needs vast AT-GAA biomarkers, alglucosidase into muscle key both the the function, majority over slide, grouping alfa you with on across manifestations impact domains populations. patient-reported Here living and of highlights strength, endpoints these of and these effects

reach As for reminder, superiority. alglucosidase meters, XX population, primary groups patients a not endpoint in outperformed the of walk X-minute those alfa with between in on distance, the statistical on a overall AT-GAA difference which significance of did

therapy. nominally this first for However, alglucosidase endpoint a population, capacity, of loss PROPEL meaningful cause leading versus only was for approved the Pompe, percent and the a impressive p-value a superiority upon a and of was We is clinically in X.XXX. difference statistically is the product generally everyone placebo, that second-generation approved therapy, X% This superiority AT-GAA of or key alfa showed function approved. and remind initially the alfa progressive FVC, finding as study of not significant on versus secondary in overall that requires bar mortality demonstration a predicted difference endpoint, of pulmonary for versus approval which tested forced alglucosidase an main and a with of the vital non-inferiority

these the As highly of primary prior in shown key post on secondary hoc analysis non-inferiority of significant. calls, for the endpoint analyses were X-minute FVC, first walk endpoint non-inferiority and

parts quarter. BLA AT-GAA, into and the FDA. up two last BLA both we CMC Moving have John the completed submitted first, and completed the surrounding of for the on make in new manufacturing FDA. in The the application to Slide regulatory July, rolling miglustat, FDA now been steps to the development, was next clinical and for as drug second modules strategy XX submission for submitted The that module mentioned, completed and ATBXXX AT-GAA, were to the components which have submitted our rolling

the opinion pleased through to granted received regulators This repertoire In prior supportive disease opinion following need filings expect are and positive we UK. marketing Medicines or have the the marketing a for years by to switch least was We EMA that adults eligible with to the to alglucosidase a scientific alfa access EU AT-GAA living MHRA. who’ve the announced are and high authorization application AT-GAA community unmet second half this Scheme, positive at authorization Pompe in positive of in EAMS, share Access follow. by global UK’s to late-onset AT-GAA co-repertoire of and Pompe of to June, year, faced medical submissions Early permits the and X recognizes additional we also meeting,

of this put expected expand patients Pompe that the to is adult for For response onset, point, Pompe onset is study those to this we’ve requests XX-year-olds, our And report open-label disease this we access that younger are both At with AT-GAA. begin year. being living community, now this children to our and perspective, And close adolescent treated our to look are over pleased study to for today and the to we worldwide many in to with programs treatment. patients expanded with infantile-onset Pompe, received into infantile-onset in ongoing XX individuals. with with Pompe finally, access currently on disease we expand clinical figure XXX X% people continue to will for multiple infantile expanded for for

Moving on compared continue and in participants and rare continue where industry in first potential for participants Phase from natural the X/X disease CLNX follow the progression of studies, the history. when therapies these XX data show portfolio we CLNX now to highlight to stabilize to to our XX studies children to our diseases, gene Slide to leading X briefly both

result patients focused with diseases We’re for Slide We clinical To neurodegenerative really in excited we programs combining engineering progress therapy on across this and with regulatory as Amicus’ innovative GMP additional to dosing XX, discussions by devastating Data programs on gene both enable activities to preclinical studies. manufacturing about the material grade our therapy approach our and technologies. to Fabry these gene registration-directed Penn’s early are protein mortality. head future expertise our gene with particularly gene Pompe make therapy continue transfer validate regarding with programs further these from remain programs. Penn,

build programs We believe capabilities the to is of out ahead studies. beginning. both the IND-enabling for continue this We manufacturing just expected

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

Daphne Quimi

and Thank everyone. good morning you, Jeff

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

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

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

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

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

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

and year $XXX to of turn our million with full non-GAAP comments. million our operating John guidance revenue guidance closing call that, for to And reiterating Galafold to of over expense $XXX are $XXX will back million. $XXX We I million the

John Crowley

also Great. and Thank you, Bradley. Jeff Daphne and

this As across even mission. been world emerge you continue Amicus can on us global to have Amicus execution led by COVID that I relentlessly as of performance will the at focused lead and business on a patient-focused have our stronger. the will see, who passionate am and pandemic, from we team confident driven entrepreneurs emerges

so And to we happy to operator, over call are that, the questions. turn with

Operator

[Operator line comes Ritu the from first of question Baral Instructions] Your Cowen. from

Ritu Baral

Good morning guys.

period a wanted anything quick review guess, option. And BLA ask expectations that AT-GAA about accepted. are I So or to But the then priority review review review about trigger how is should I very is standard or once the necessitates I we and NDA follow-up would priority think there neoGAA? either review? have for priority an on

John Crowley

Yes. That’s Ritu. fine,

from So into submissions hear to their the back FDA, expect the acceptance and would complete on this now with quarter. hear that. We we’ll FDA back, and of to BLA hopefully, expect NDA

uniqueness of here. you as for the review. in we As as the of product did we request significant priority that breakthrough community part have need well also the this designation know, part the unmet to a in recognizes therapy data submission, Reminder as do everybody, the

hopeful hopefully the once that, of filing we’ll but that the are they FDA favorably we the So upon find look submission. will accept out

the the review, regulators. timeline. a With trigger review course, would that of don’t X-month to That’s priority that yet. But up know we

will So see. we

Ritu Baral

And that’s And remind is out EMA who the sort it. how I’m assuming to of within sort Europe, chemical believe, for bid for Can has, you application a of moving sorted chemical novelty EMA? Got novel competitor your repertoires for sort repetitioned status on the then I same us are? the exclusivity. of neoGAA.

John Crowley

biological either a discussions to it for order everybody, of EMA’s and/or per a in – its definition, in want first previously the previously or but it’s of a to I’ll or speculate in qualify differences Yes, a active or significantly combination but just biological due nature as differing competitor, material the authorized new molecular to to substance the efficacy the a safety all, don’t one process. the of has for be remind I structure, a substance, not on source substance product authorized, manufacturing

differentiated. differences. its everybody It’s to And we on know So component a I remind respect on We efficacy. with it’s will dual differentiated believe AT-GAA, ust differentiated it therapy. structural ll differentiated key clinical it’s has

So denial comment again, I on for not going we’re the the product, certainly very data product the am confident of uniqueness and our in but the that of to of neo have. that we

Ritu Baral

Thanks. it. Got

John Crowley

or you have follow-up, quick Did a Ritu capture it? did we

Ritu Baral

was on follow-up actually the... That the

John Crowley

It was good.

good. Very Okay.

Ritu Baral

Yes.

John Crowley

sure. Yes, yes, dynamics so for very interesting play, at

Operator

Your the Merle Ellie next question from comes of from line UBS.

Ellie Merle

Hi, guys. Thanks taking the for question.

the Just one the BLA of discussions the in – seen of have and data U.S. also the regulatory then versus another is interactions, in how how of curious EU endpoints? viewing the submission? sort Any differences viewing on filing, the differences you EMA they’re any terms or clinical the just there in has with terms

John Crowley

we the everybody, remarkably remind been again, And company program, the Pompe the scientific to were this joint I’ll similar, advice. program, Pompe actually It’s first Amicus Ellie. Yes. utilize

that began study. proposing we fall the PROPEL as were So XXXX the of in

regulatory to we design and and had received, on the path on plan. also similar FDA forward. the EMA EMA prior statistical analysis feedback from from And now feedback seen the So both start, FDA very, very trial study together harmonized we’ve and

we hope – So that we bodes think well.

Ellie Merle

thanks. Okay,

Operator

comes question your the line from from Cantor of And Kluska Kristen Fitzgerald.

Kristen Kluska

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

overlap disease. could the footprint into that Fabry of team often Galafold? to are for then commercial benefit also do ask, Pompe Thank AT-GAA more believe centers you And for wanted if treat is you treatment I this there were the territories existing your diseases, of is for for expand as for you. multiple to Galafold, Pompe also both approved, that specialists involved what So and the how

John Crowley

think for I we commercial We Yes. from necessary And so that AT-GAA. footprint comment answer global the great Kris. would I’ll built all we for there there. the is appropriate let Thank leverage But you, you highlight that and expanding I’ll think we the can great will we’ve that AT-GAA, this. be are further. synergies already for infrastructure that lever have to Bradley, Bradley geographies significantly Galafold just Galafold. again,

Bradley Campbell

highlights. you And hit the definitely John. Thanks, Yes.

commercial further the the what commercial intention AT-GAA the So leverage in launch of lots no field add which is And now, between of good and organization. organization AT-GAA, support fact, Galafold right great. news for will the to is be

the these up some details sort more that treat and to of Fabry typically the are few that, patients, so both of overlap, and a is some in other and Pompe particular, there explain specialties end help metabolic that patients. in terms there treating Just specialists, in specialties genetics

a high these follow terms overlap that the of in centers degree there’s However, of patients.

And an key of really gives LSD academic and multiple taken And able be Fabry so or to different hiring programs. we including the typically, will one center center approach to organization also lysosomal leverage treating you more Pompe, be really that’s both following disorders, have managers. account storage with patients use and the what commercial to support

of an to not of products. and physician who’s center, academic terms specific just supporting somebody how understanding So these they one used help support utilization entire the in

support and, will increase the So again, that launch AT-GAA. in we feel see really good about leverage to need the we commercial infrastructure of very that little

Operator

question your Anupam And have the coming from of Rama line we from next JPMorgan.

Anupam Rama

much. question. the so new that into really Hi guys. quick deeper XXXX? for by Thanks Galafold A taking or being penetration June first Thanks you of the in launch, being driven much patient second half so is since region one maybe adds country saw on on best the the international new in the adds this

John Crowley

turn Bradley. Yes. question. it I’ll to the Anupam, thanks for

new Bradley, actually number starts, it But that’s a product launch. of we’re little mentioned It you the patient bit second that? Just second had this you that want was had shortly the was most to most – highlight, dynamics the in further comment June. significant through to quarter significant on since seeing seen that driving we do

Bradley Campbell

Yes.

So question. Anupam, good

things. a few So I it’s think

for is all, we’re Europe, those and of countries on the it Central cycle, see example, reimbursement on. have Eastern been later some First countries. come new and starting to

on about opportunity it’s great Germany, our in patients a there, I patients think markets, etcetera. but which come for is Poland as also an we UK, new the larger example, talked existing seeing

were here their During were into in COVID, some that the and small to queues ability there, patients get there disrupted hospital.

think that of is it’s also growth. so But on. And naive penetration I so into increasing patients, there it’s going some of reflective market

half So and well. great factors underlying this of a combination it’s we we the into think a again, are of go we as the beyond that, signs as year, all those of think but back should number continue business, of

Operator

And we Stifel. Dae Gon next have from your Ha from question

Dae Gon Ha

Good morning. my Thanks question. for taking

alglucosidase Jeff. follow-up just Can familiar you. the this we And to guess, for the the then to profile press produce an secondly, going better back you in earlier me, the overlap the not even to if think AT-GAA, for follow-up maybe and so of whether you one think Thank with I the modification, release, in the ATBXXX, MXP Can speak I’m alfa I too biologic to wondering for about looking and should the there UK, another to modification EU Early between the Scheme the was biology a to exposure modification question MXP you and just Medicine is Just but I clinical on an having will side how better years Access guess, regarding speak a of X maybe the or on much, question degree language at more? outcome? FDA. regulatory

John Crowley

for approval patients majority the more reality process, Kingdom That the the be for patients we Gon. more who X through on approved X years. Thank treatment-experienced remind would on the initial as than Sure, ERT United granted were on in patients had that comment been that the announced MHRA switch for patients back was who in just that had well we will And to everybody, again, AT-GAA or to Dae you. naive is I been indication. May, years. The as current as vast have all for expect well. of the for early to

of So standard we United approval offer many significant and full Kingdom to many, prior current think requested patients AT-GAA, there’s the care. final on as to to in the opportunity

get muscle, is that see into – occurring studies, including carbohydrate would product. our vitro now glycosylation, and through current of to That a into in of optimal data, we muscle has since skeletal is enzyme it’s with the proactive have unmet to smooth from that higher the that seen our again, I protein. as Jeff preclinical mannose-X-phosphate, for ERT stabilizer, be protein we data, And requests a significant that structure have the structure we’ve that, WuXi ATBXXX. patients from to partners the known the for There switched With to of proper Biologics, glycosylation, We It ability see in we multiple months physicians, and with Amicus naturally that only commercial at a novel all of now announcement biologic is seen well. uptake with authorities. carbohydrate developed with months think now clinical from working cardiac we MXP we now maintaining think certainly, that chaperone product. very differentiated scale, pre-clinically, the into occurring enthusiasm health naturally I’ll ago. to high at continues not significantly But the scale cultivated, muscle, really great a and local the we And modification, to in just way see that to ahead have very product, and levels particular tissue, cells. occurring to based AT-GAA the phosphorylated Pompe numbers continue diaphragm. requests it momentum we to And get significant think with phosphorylated. of expect is And be couple with and comment line clinical feedback again, highly ask need again, on what the cell and studies that the in what we’ve respect consistent a its population. selected, And naturally is a seen as

missed So anything, but feel with cell cultivated. free Pompe. And please that’s together, to again, very optimal not and level it post-translationally the I modified, we of living add. selected novel Jeff, line think product we think we’ve MXP, has if for an a people with that

Jeff Castelli

MXP. Yes, sure. Dae significantly phosphorylation said, we and John that improved know ATBXXX as Gon, has

the on an average. molecules bis-MXP molecule. vast ATBXXX the majority least protein fact, at have for every all There In of single one MXP is

uptake. sort We is MXP We optimally out. really that our think to we additional pretty create change or having of don’t that something So meaningfully notably targeted. think we’ve would maxed managed that

Dae Gon Ha

Alright.

Operator

Sachs. next your And question Salveen is from Richter Goldman from

Andrea Tan

Andrea your Thanks we Maybe what when program, would for for on from This Salveen. understand can you the data one next just on is disease that to question. Batten and the taking release? wondering expect look data

John Crowley

on Yes. Jeff, do Batten to the you comment program? want

Jeff Castelli

Yes.

X and at That with Meeting World with of CLNX, in the an natural That and X data who years included we CLNX in last domain the to Motor in So on with encouraging. it patients reached UBDRS Disease Lysosomal show CLNX CLNX the the was update was stabilization was first had the X for earlier really what we’ve dataset X and treated the the provided follow-up this Hamburg history, data and year. kids physical on compared XX in disease X-year the of treated time CLNX treated continued kids to Language seen which CLNX.

do we continue to and kids. continue will We follow those

year. new additional have of – in could key World fall do significant the are at typically, updates the meeting we have but the provide either we That said or happen venues the haven’t will Neurology Meeting as updates when, data. early We in where we specifically Society at Child amount

places those are are data So what potential share, those in updates we excited really we’ve expect to more to can continue we as two see you by have and but where kids. seen

Andrea Tan

much. so Thank you

Operator

Zhong of from we a Yun from question the line And coming BTIG. have

Yun Zhong

and Thank taking Thanks you my morning. question. for good

very with you is think start decline, not still well So the some treatment that losing And understood. I long-term on I they reason believe Lumizyme, patients, AT-GAA, mentioned response. after to

tell, by not entered early suggest I your receiving too you, any in have evidence might that the observation wonder did know is study, this AT-GAA? to happen it’s who declining So I phase treatment? to enroll Lumizyme to there from clinical Pompe but that patients And in – study any any chance, might

John Crowley

Jeff, field to if have you please. add, any Yes. Thank color want Yun. you Mitch to if you, Jeff, after, that, and Maybe,

Jeff Castelli

pretty is the FVC Yun, uptake the have and we’ve much cases, there’s tissues clear it decline, and John. is and benefit like see the almost Thanks, blood glycogen many that the X-minute could even standard what Sure. also somewhat might first about continued And in what’s cause seen with But patients muscle was variable. studies with And looking, response that better after and care. MXP cleared, stabilizer pretreatment. seen that X the with walk, initial here on the function improved on stability We addressing believe in improvements then that that, do current AT-GAA deeper We years. continued help to be and for other is a now not really that into improve hopefully be that X in penetrate treatment the have of been on progression. will can of binding years and multiple

encouraged patients. in here We are patients lead results in their well ERT-experienced seen the X our on X-minute into and actually nearly treatment FVC our now patients, declining. we that both be walk really those to on and that years by we’ve improvements average, Phase X/X been they’re see in where in stabilize and to expected PROPEL, stage And where can we for, had in those study over all

see do offer patients trajectory reads those to switch for of we bodes in the We patients. only hopefully their them think naïve disease. to that to that that well that, those a become patient. ERT-experienced when continued really term something longer through akin patients they not change But could benefit will sustained hopefully, So more

Mitchell Goldman

is I’ll Yes. The add what This the patients to majority, reiterate. think during said went XXX study. call on I important was to Jeff the just extension that Mitch.

look them, and that’s and report trial at And that We we to an will on at doing. find that are venues. that’s to patient And And to point data appropriate heterogeneity, follow important. every ongoing in look also basis. how think they again, your ongoing cuts about continue data those at release look regular individual we again, will we

Operator

a Blum’s & from And line Company. Needham we have next, question from Gil

Gil Blum

which question Thank discuss Batten guys disease of you congratulations Maybe and have therapeutics? any program, you. upcoming a the execution. on about for taking a this AAVX question on bit September, AdCoM the will safety thoughts my in Thank do on you

John Crowley

I them those post meeting. an everybody Yes, of for be dosing. dosed remind XX years and then concern, one children no events with for great. of CLNX any safety will dose. Thank X of CLNX, And whatsoever we higher we’ve Gil. that at also a seen important all just think children have even X now Batten’s you, will the more again, with than and our We that them children, program

looks that low premise particularly with considerations. of therapy of capsids. our gene intrathecal the from with gene remind safety part with too together that AAVX So just involvement seen technologies, dose protein we’re the and delivery the that notion for to of problem the will Jim next-generation address the we’ve exactly using not with our there, Wilson’s everybody technologies any was AAV therapy safety, lab, coming with engineering I of these the Amicus’ that collaboration

think we we actually, very favorably be will So positioned.

does to present think complications concerns systemic have broadly that high-dose in continue We patients. AAV. We field and the around dangers for

this But engaged FDA and differentiated be expect I taking to free well. here. So review, to that as feel we from think, is Amicus or we would approach in Mitch very actively those Jeff the on to comment think again, the it’s discussions. terrific

Jeff Castelli

it to is covered have you John, This specific add. pretty I anything don’t well. Jeff.

Mitchell Goldman

I’ll into just And can output that color into is us look and we the to say Yes. welcome things safety these the Advisory it provide field we our evolves of FDA clinical the Committee to and to thinking, incorporate as patients gives that paramount.

really meeting. the So forward the output to we look of

John Crowley

Pompe systemic therapy we ability Great. AAV gene published deliver again, and lower ability some the with can the we’ve And work what well, that’s have those transgenes in now much you respect programs manufacture that dose, publicly to as think, Fabry utilizing discussed that our with technologies and we to delivery. to actually and safer better engineer and much We of therapies, again, better Gil, gene direct not I’ll efficacy our unique to we have that a and only pre-clinically. them demonstrated may potentially dose hope we just

Operator

Leerink. next have from Joseph from your question We Schwartz SVB

Joori Park

for Joe. am in Joori I dialing

do would to – Who patients? alternative patients be treatments? are adopters helpful? these several is be you Any could are on And how So abundant that of the therapy therapy there how switch the believe currently are assuming to for for AT-GAA that opportunities switch these AT-GAA? the patients looking available for therapies early treatment on switch approved, to color – now options eager

John Crowley

all the well to perspective. ask But of I’ll way through Bradley everybody, late disease, patient as comment had are years demographic adulthood, only option enzyme kind and therapy. people for the have approved XX with whether replacement market from to infants remind of a Pompe – living they Sure. to one just

think for therapies. potentially for new has AT-GAA it’s ahead potential these overwhelmingly have meaningful options we think clinically that shortly we the Again, demonstrated significant to patients. next-generation they’ll and multiple benefits have terrific So very

for great So you’ll see a novel as in hunger and need community a patient physicians well the new with therapies. next-generation the and

Bradley, the So population we see adopters speak if early patient about how to to the want be talk helpful. that demographics you very would

Bradley Campbell

I John, so market the I we today had a can Sure. thing, – you about hit patient population. the that, $X.X is points patients for alternative X,XXX for important think treatments avoid over we you John, estimate, that’s so switching course, other globally highlights is one of of for important about revenue make, echo that Lumizyme. sorry Yes, from billion the the – there. need and or couple today, to A treated think the

well. high huge So to so potential single-digit rates, It is patients the of a some continuing population bring on as naive at it’s growing market, patients. switch that’s

we too, through Importantly even today AT-GAA. have depending we expanded clinical and number over on expect, And could patients further utilization that the on development as programs, would grow. XXX – access we various

treating from successful the present many covering coming when I team gives was that disease. major momentum we product commercial mentioned you’ll the global Galafold, who the And patients touched are and the many a launch, same will as earlier. think a launched before, already, world. something product, clinical around when of last trial commercial at you that here, which a the a this and launch. centers very building launching thing of John are is same in anticipate organization this to we were physicians the that Pompe but over, we so have markets Unlike well-experienced base our of we lot scratch I significant And we launch time, convert of And into offer we or we have

getting commercial so and of entrepreneurs medical-passionate experienced products And patients. team on out a to great focused our have we

great So we a the got launch. have start think from base to and into should great we’ve going momentum

Operator

comes question from line from Baral Ritu Cowen. the of our And last

Ritu Baral

Hi guys. Thanks for taking my follow-up.

CLN believe pursuing? to CLNX, back can the you round just around proposed you recently meeting patents Just soon. trials for back program on FDA wanted have I on interactions. And in general, going about pivotal address the and current thinking I Batten’s other were the a on or CLNX to you’re strategy that the

John Crowley

course, people of last expect focused of we the really large focused both have CLNX in the all fall the of field been particular, indication in lot course, the CMC. in interactions analytical the as a make Ritu. that leading tools. Yes, of tech for the on the manufacturing, scale enter assays, to series the that pivotal programs regulators, that trending Again, lead a direction saw we continue have It’s on toward both studies to We and been as of our transfer including on to including our that the effort we now Again, lot GMP Fisher, X.X Brammer-Thermo the years of Batten’s very put work from for activities we’ve CLNX. assays, at and, and university great into manufacture potency with the a to completed up, sure early partners XXXX.

lead how we had and some studies. ongoing. programs, Those regulator’s interactions With feedback idea Batten the that, pivotal on the to on about we’re we’ve were data for curve clinical continue But to Jeff thinking if and want think those our ahead there. also I our be give studies please. So you on Mitch maybe the and broadly future of thinking

Jeff Castelli

first, I’ll start Sure. Mitch.

topics. John regulatory mentioned, and on different CMC we as all to our focused work the across be enabling interactions continue So

GMP current on the be for done would next with process commercial they first, studies, the Thermo the thinking As clinical material.

to of general Hamburg focused and would history, we For similar study versus X/X single-arm a Phase did Motor on terms CLNX, natural in we believe study it be in what Language. size,

really that ability design to of succeed Phase based study in terms So feel we’ve about our our we confident in power what X/X. on already seen on

add only of X/X a believe our material For natural from was We course, data That the had CLNX, of the next believe of key We study with again, history study study physical to perspective? endpoint is all compared would level We larger it single-arm X patients. anything Phase expect a from course, exists domain that from there’s of available. endpoints, should with to your UBDRS we else think size patients. CLNX. Again, a that But robust the very of study, several endpoint. all in control was data dozens those Nationwide. agencies the discussions likely components, we arm, Mitch, the be about primary the initial high much a etcetera. in lot

Mitchell Goldman

No, Jeff. Thanks.

I think you covered it well.

John Crowley

all show operator, in So the I we think correct? the that’s questions queue,

Operator

Yes. to back turn I for John now would Chairman CEO, That questions. Mr. all is and closing Crowley, like conference the to the remarks.

John Crowley

half good listening programs. And Great. to the everybody. of and questions. It we of full across exciting and second you, incredibly you for for look you Amicus, Thanks quarter Thank strong the execution a can our forward Thank all an very Have again, year. to operator. see the was second of a day. all and great

Operator

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

Amicus Therapeutics (FOLD) 6 Aug 212021 Q2 Earnings call transcript