FOLD - Earnings call transcript Q4 2021 | Amicus Therapeutics, Inc.

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Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics’ Full Year 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, Executive Director of Investor Relations.

You may begin.

Andrew Faughnan

morning, good and you, everyone. Thank joining Thank Amicus call discuss conference full you year highlights. for Therapeutics’ corporate XXXX to and our financial results

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

Sébastien Business Officer. Joining for Mitchell Dr. Chief have Goldman, Chief Martel, Q&A, we’ll Medical Officer; and

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

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

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

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

John Crowley

throughout of results I our and reflection everyone, business. now Andrew, the Amicus year full of pleased highlight that start the into at we’ve conference you, across broad and to to a our Thank progress the XXXX, Great. XXXX call. made am welcome, XXXX execution

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

First, Galafold the cornerstone continues performance our and is of its strong success.

line with revenue full year Galafold the year For full XXXX, our million in revenue and guidance. was $XXX

pleased are patient which and from with globally, and Galafold as as sustained both adherence largely very by of patient We rates. driven the accruals the well switch compliance naïve new execution populations was

growth XXXX, peak and we be significant a Galafold the Galafold billion at for potential expect has We that in $X believe medicine revenue. to

the agencies. we regulatory filings the AT-GAA, extremely our Second, well, of standard on are Pompe global review next-generation Pompe AT-GAA’s reviews The very from with we level mid-cycle progressing to great novel become regulatory and remain disease. therapy the make continue in care we to completed its recently engagement and the approvability U.S. of and next our progress disease. EU FDA, for are with pleased We the in regulatory for and potential confident

of in the set the the enzyme reminder, in July a the XXXX, for fourth engaged As date In reviews, we action a CHMP we the XX, biologic. marketing the EMA XX, authorization AT-GAA opinion FDA were later Medicines PDUFA and stabilizer, to applications XXXX the are submitted the NDA quarter this European and May expect of the for and the year. Agency has of the to actively for BLA EU, XXXX, related

AT-GAA, global investments significant including to in launch and launch continue accelerate in to plans for support personnel launch Our preparations. the targeted additional investments inventory

and the global execute of of strong Pompe financial importantly, we expansion third having States our – maintained has applications global Galafold as for prepare position AT-GAA. and months people and planned United in living another with in both and to on to committed remains further treatment financial And closer to disease, now our potential ahead. in XXXX Amicus profitability achieving in regulatory the with Europe, the continue are the launch We for

to financings equity-linked or equity any for achieve need profitability. no have We

Slide Turning X. to

results As as remains and strongly and committed sector environment technologies. on current achieving Amicus priority without to Fabry in need market our our spin-off decision we an increasingly disease financings. the XXXX disease. will equity-related decision equity gene further in here the dilutive to and have Pompe well any Again, made our the as vast therapy standalone announced challenging In The majority companies. to for biotech and in therapy of franchises affecting growth gene profitability efforts order not programs broadly the this, this we today from highlighted slide, strategic unfavorable accomplish or investments the for concentrate conditions

savings expected therapy million cost similar is the XXXX. a that overall Pompe is two, launch global focused This for approvals judicious Galafold; team with to highly net in by on is We gene efficient continued designed approximately associated strategic R&D thirdly, next-generation delivering $XXX as the growth AT-GAA; and in advancing and result for prioritization technologies our this Fabry more and our a therapies to to in announced spin-off. in in decision our will commercialization strategic core platform the through global and pipeline pipeline medicine. in shareholders. activities is portfolio field by and savings This organization This prioritize well in and the amount of of science and through; of internal and the patients operations previously of streamline genetic to reorganization secure R&D for regulatory do one, as commercial create and making investments

actions savings We are respect in taking cost our able principal by million R&D. achieve with to three to ahead $XXX this

portfolio our decision including prioritizing our to the portfolio, are focus pipeline and platform we on core First, technologies. – enabling

clinic prioritization therapy significantly clinical operations, This expenses R&D-associated in programs moving ahead. reduce be will activities. manufacturing we and supporting in such, As not into the gene will years the multiple

strengthen XXX achieve launches will of workforce are XX for are Amicus while to count Amicus internal at by coming organization continue manufacturing all overall at XXX the next years. employees AT-GAA, the headcount mostly and indefinitely to keeping portfolio organization. of third, facility. And to about overall These employee we therapy changes sharpening about This today R&D employees suspend Galafold personnel maintaining our hire globally. and financial inflection for overall us will rapidly again, continued individuals, gene in and we by We in pivotal our very flat allow pipeline build Amicus judiciously approaching growth flat global about appropriately X%, strength. key ensuring two Second, and and Amicus we To organizational points. are our our plans reducing investing our the at several this, at focus, will an our

the of enter of the First, biotechnology. believe we echelon rare medicine to second in disease profitability. and feats these enable Amicus And upper truly profitability, that secondly, rare are launch AT-GAA. product A second a global will

to priorities XXXX. now strategic Slide key X, Turning for

one, revenue Here, the strong We will existing focusing double-digit more last this continue global to our product year. XX% precision key of variance living reflects drive Galafold revenue on we behind Fabry strategic to million This to look month, growth out million new to as in in momentum medicine achieve and with and laid for demand the These with markets. $XXX this growth of disease amenable $XXX we include: XX% priorities year. for to globally. people are with we achieving

team drug anticipate across another launch, launches seasoned AT-GAA of AT-GAA. remain our commercial Leveraging steadfast prepared areas and are launch experience and the our needed global United AT-GAA filing we fully the advancing to commitment regulatory successful anticipated we and an Second, all effective with initiating in States. in for

all our through position strategically remain are will best-in-class we we maintain we a our and major and strong genetic Four, continue Third, investments, financial we funded carefully fully next-generation again, medicines and manage advancing to capabilities. as expenses milestones.

as announced leadership I taking Before Officer, term me transition partnership now become, Amicus transition the to effective previously time, long-term provide and call tremendous hand I to at that of now announced Again, our transition. continue the also will as in X, we defined I’d place XXXX. in August XXXX, like I for enable Campbell September expect Board This be to succeeding two-year the with very a will Chairman clearly over, an Brad Chief Amicus roles Executive Executive that Non-Executive to previously as thereafter. the Chairman ahead. update of will Bradley our and at continue full-time

in our mutual remain I’ve past there on and today one our goals for strategic here to Bradley is As ahead. role, Amicus for and highlighted in achieve out vision succeed this capable our and to more both laid I steadfast drive priorities calls, no and years

the to And over Bradley, you. turn I’ll call so with that,

Bradley Campbell

and John, and to amazing leading deliver morning, August to for look Amicus in now to good the really Thanks, Board to continue and and And building leading shareholders. and that value to you’ve Great. again work me thanks in at organization. for you forward everyone. our ongoing in your patients Amicus for beginning inspiring done here in the this mission confidence our I partnership

our X, progress. through, in you year. I’ll As Galafold Galafold snapshot global by for commercial of Slide detail, John walking give the On performance mentioned, start more our we

product largely the rates. breakdown from $XXX was As or was John or and generated $XXX mentioned, million States. sustained new of the strong the for adherence the of remaining $XX full million XXXX, and United XX% total coming revenue during driven revenue compliance revenue globally, outside accruals full United Geographic quarter patient XX% patient within and the million by year States

this for grow come. expect we previously, we in many the parts business as to is we’ve As of years with to mentioned that the XX/XX line split continue both roughly

business – of all And as exchange from highlight quarter our full be headwinds and to new increased revenue also efforts tailwinds benefit. continued global Turning excuse incredibly QX with added continues fourth and and reflected of well. execution. year the now commercial markets our me, patients benefited our to major demand in and Slide markets The many foreign patient X. results strengths resilient financial

including growth the has As growth third payer second past versus reauthorization quarter typically continued. and of variety as given year pattern and mentioned the first factors, quarter as due quarter-to-quarter in in quarter is a and the United nonlinear. to quarter, of Usually, timing a in fourth States, well year, see calls, stronger orders we within that on rate of

of will X,XXX which with for the share around little in And more XXXX, historical first snapshot of good a the several occur continued discussed world. time naïve for stronger remains the to markets the a so this On global of a for mix earlier right-hand as is patient year. so year the in is expect slightly for good budget, the the of similar of than geographies, beyond. this we by would slide, demand this and patients each of sales even globally, XXXX. XX, to quarter that off drivers occur patients, the we’ve and ended and while at the last during ahead amenable the is year. the a of the trend Galafold even under at in We the populations metrics called and year XXXX early we global year, midpoint dynamics COVID trending for provided the on We as news or side strong key half uptake the of starting we that And table Slide which treated market quarter, patients on have pass global in we’re of JPMorgan a now, XX% on lay out again, year anticipated. end we’re The to And given driven Galafold start percent many we’ve XX% part three-year foundation patients. just the patients year, this that into switch growth see our we’re with naïve in about the Galafold of many

the to XX% in an Japan, at of and been approved bringing coming countries to market so there’s the now newer the continue Chile, to United get the greater expect as countries seeing states switch Middle like we’re markets. and Within we countries to where and So, XX/XX segments. market to well patient still Colombia, hoping core at on of diagnosed switch in into Europe, and the Japan we’re on in we these as see Japan, but traction Brazil, the longest, diagnosed other is into to from launch. East terms Whereas if driven to and Galafold Pacific we continue to naïve where be as out newly where Asia and share about Latin continue opportunity on XX% hand, in of in largely our approved the continue countries this to throughout Africa, new like U.S. such fact, you coming like in over Galafold. Gulf achieving switch year, starts as years. that penetrate North major And States, growth Turkey, driver market higher later patients, we’re patients coming next several Argentina, to the plenty strong business, patients outside the been of the EUX, a untreated XXXX, we’ve we’ve the we’re by markets still And growth grow markets approval growth shares and now the at about American we’re longest, of look new

is on by that impressive reiterating and Galafold generally Galafold. the rates that of underpinned on who go patients continue to belief those our XX%, exceed All adherence compliance that stay

strong value States. the be world by the of continues payers of granted to XXXX negotiating around of record reimbursement Galafold also the vast insurance successfully renegotiating track and Importantly, with recognized the by reauthorizations payers United a very majority in and U.S. outside

ensuring Galafold focus relentless access remains on Our needs who anyone it. to for

$XXX and Galafold In know With in other significant years couple $X broadening diagnosis of into key surpass screening, the existing market the and which in performance from growth screening XX. continue continuing expansion penetrate to into initiatives, markets; of support we successful billion in term, several diagnostic revenue years as in the Fabry we to remain and next to annual Slide longer labels. through growth confident drivers: the over revenue the that high-risk well. driven to we record to continue a and geographies; million by see potential now as track new us, that we invest globally, behind newborn opportunity has several peak Moving

of us provide orphan Finally, protection to coverage late our Orange the Europe, XXXX. which United exclusivity Book-listed that in patents XXXXs, we into XX the give have through XX addition and States in IP

So provide to come. Galafold years plenty many to opportunity of access for to globally

on XX. that commercial organization rare disease experienced Slide just Galafold across Finally, This our world. highlights and medical supports the

very directly through the either We’re a is There present will additions launch. here And this to the is globally. AT-GAA the that team will outlined what’s to anticipated that FTEs really countries, dozen as on launch incremental team now support with new XX few same be or is distributors, in than globally the critical less slide.

only let We now assembled call With our not to program. group passionate me successful, Chief great to grow eager Development Jeff? have to second anticipation AT-GAA. entrepreneurs a Officer, to we AT-GAA what that, highly be with continue Castelli, the of now approval over of our plan also our but to capable with Dr. who Jeff are Galafold, highlight hand

Jeff Castelli

disease. everyone. morning, therapy next-generation XX, we’ll Bradley, start AT-GAA, with novel On Thanks, and Slide good our for Pompe

it’s recognize of Pompe neuromuscular First, fatal and health and prevalent to that is range continues disorders. challenges Pompe a most important pose crucial. one by people a affected disease having for to of disease the therapeutic the choices and severe lysosomal is

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

Moving on to Slide XX.

our Phase of the secondary we endpoints from Here, summary present a biomarker X primary and key study.

adult patients only against of clinical patients treatment the approved efficacy AT-GAA of controlled in included of therapy, PROPEL date which alglucosidase is a one patients, randomized As to ERT-experienced and represent safety naïve the reminder, study greatest unmet a with the PROPEL the set assessing needs. alfa. was trial double-blind that and ERT-experienced

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

on Slide XX. to Moving

the AT-GAA highlighted on have We program. key updates

for has for the the Miglustat, has of FDA cipaglucosidase XXXX, components alfa The for accepted and the U.S. July BLA PDUFA on BLA. AT-GAA. May NDA FDA the and review two set XXXX of NDA First, the date for the action a progress, regulatory for XX, the XX,

open-label alpha we access to recognizes to the framework review, two across and positive positive mechanisms CHMP these across the AT-GAA with programs. to that the multiple by be disease the MHRA. June, are our Access high And U.K., opinion living XXXX. mentioned, early XXX have call in switch the disease into in are authorization many in submitted expect later for positive will soon Early have which with community, expanded are MAA are happy faced having younger the leading And and We to we and and including than that provide in we granted the Pompe receiving access to for for both late-onset to Medicines countries. through and years patients access with access Pompe received We’ve Pompe, grown, U.K. we’re to up multiple This shared the and physicians AT-GAA in our the discuss with centers was includes June, infusions. that to launch Pompe John to have also all early eligible from or disease XX patients access we multiple scientific With EAMS who pleased individuals. medical for to preparations scientific by to AT-GAA that expand now and now permits of together. opinion also for worldwide is been our over clinical place, as to report the extension Pompe with least This in for possible. and we As infantile-onset Agency adults able continue opinion let Scheme enthusiasm other Bradley and EAMS finally, children the community reviewed access look U.K.’s study filings under expand announced AT-GAA treated alglucosidase the we those requests in AT-GAA. unmet be multiple under prior to more the being the for European living mechanism eligible. received has late-onset living Pompe to a access significant Since Japan Germany, infantile-onset to living who Medicine need with for in response Pompe opinion me in seeing years these Pompe requested that, Brad? in at EAMS the age, this marketing this hand point, with At And of U.K. momentum to late-onset patients with enroll interest AT-GAA as and studies the back and children expanded infantile-onset ongoing has continue programs those

Bradley Campbell

scratch, And preparations Jeff. launch Galafold, XX, Thanks, from we another the team now product we’re Great. we launch again commercial infrastructure on largely major poised around have of mentioned for same in countries as the will successful Slide Unlike XX and I markets, including when hiring presence before, we the world, outline nearly AT-GAA for that that when we anticipated supportive as organization and were be all launch. a will the launched AT-GAA. the

experience We medical have all drug supply affairs. that successful for areas across chain, launch, are regulatory, needed commercial,

reimbursement most communities. disease experience with patient with the and and patient access, and and rare payers relationships have importantly, key We advocacy,

we’re with to to our strong very over world, inventory outlook. medical Phase strategic successful in our around WuXi planning Officer, and that a for for me living and organization to the Financial that, position disease world-class all with regarded we’re now experience access second partners building Amicus. the turn results, From with people highly in Chief Daphne around confident education, that believe for AT-GAA review financial the and with our let the to very Biologics, data rapid again, experience published team, We’re Quimi, Lancet and with the we and we guidance a Neurology, and the our together world. call launch deliver the Daphne? relationships And X doing our reimbursement in can lever their Pompe

Daphne Quimi

Thank and you, Bradley, good morning, everyone.

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

loss currency XX.X% XX% year, currency operational Cost a X%. GAAP year-over-year includes benefited the in For costs. by XXXX. compared share-based were over total compensation full was as SG&A Galafold of constant XX%, prior XX.X% in We goods in million million timing a a share prior sold $X.XX period. the compared consideration in operating loss expenses research of of million, compared the $XXX.X $XXX.X XXXX, compared operating net The of exchange per non-GAAP $XXX.X for and of value excluding to as which $XXX.X increase in or to sales full further as basis, as percent XXXX. and as reflected $XXX.X as stable expenses, revenue year development a impact pipeline, year to million Net XXXX On expenses changes million measured achieved for million were was decrease This a million of in revenue $XXX.X of depreciation. contingent fair our or and $X.XX positive we the the the relatively offset a at Total partially year to non-GAAP expense, share growth in rates operating is XXXX year $XXX.X XXXX. third-party period. investments per net expense in define to by

prelaunch As December had non-GAAP be outstanding. we investment XXXX, studies million of activities. the approximately XXX $XXX XX, year, continued This million, expense AT-GAA Galafold total within and launch, to range in driven shares million operating clinical $XXX we by the global expect to of

includes Let the the to launch me gene for to nonrecurring the also portfolio. manufacturing committed support obligations related guidance as million $XX of approximately costs therapy that well emphasize this as to global AT-GAA certain

to decline Importantly, a similar to in operating XXXX levels and beyond, non-GAAP XXXX expense XXXX. level from would expect in as we

Turning now to Slide XX.

release. and position financial to on to remain in financing, operate for dilutive profitability future from to as achieve continue the any in be our of need We XXXX we press without self-sustainable track expect a defined and strength

agreement cost and and the This disease AT-GAA savings continuing the we of deliver well management. driving will our investments careful portfolio disease. strategic alignment, as launching majority the Pompe growth global as franchises the in and on includes focus on approvals our also of efficiencies, Following value-driving securing to globally, Fabry Galafold, business and expense termination combination core of

financial the organization Caritas four our our $XXX.X our Importantly, R&D million position previous spinout. savings with the $XXX strategically next at associated same million XXXX, the XX, guidance. marketable cash OpEx at cash pipeline and XX, amount XXXX results XXXX. equivalents December Cash, in over A $XXX.X million net few approximately compared aligning securities to and R&D comments in were in internal about years, December and prioritizing

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

John Crowley

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

that to before the remain our and with a with believe, $X second me in So as Ukraine. you performance to and to by on relentlessly been disease billion our firmly yet of the that about rare at potential, business, people we we global turn can have thoughts focused meeting. in to passionate state on global and with have ability respond companies will the led changes lead team best, to who driven the our those of also, And again let our future call patient-focused XXXX. believe, have medicine we as I’ll boldly, events to launching are see, too, the just say we Amicus, look prayers thrive we operator, across us the we together And also forward decisively that and lives, questions, profitability enthusiastic change global believe incredibly also looking and that entrepreneurs again, revenue continue people’s also turn we in

happy any questions. take operator, to So

Operator

comes first Instructions] from [Operator of the you. question with line Ritu Our Thank Cowen. Baral

open. is line Your

Ritu Baral

Good going not for there the indications the about for additional expected potential morning. Hi, inspections, guys. any Day Letter? advisory of ask mid-cycle and wanted including I any XX so indications Were Hi in be the taking to question. there any review. Thanks new are about WuXi what committee? Were to topics just of

John Crowley

say Yes, to going much I to give in a really I’ll we’re want just Ritu. too be place. good I think color, don’t

it didn’t mid-cycle an excellent prepared. surprises. was team fully review think any has meeting. Again, the been I see We

at on information facility, that respect mid-cycle we’re always responded Very, WuXi to the FDA we’ve approvability need series to AT-GAA, of it’s be the well. significant for they meet the AT-GAA, will and will those. typical, I’ll work launch inspect to to requests be we inspections, what demand in the pleased to a we in has review. build reiterate the with to and up the again, – belief And significant of With of we’ve think yes, known including investments a engagement the leading our Again, inspections is make As those for very level as with inventory potential the continue engaged to extraordinary preparations to FDA. AT-GAA.

in I good kind the remind date we’ll a place. as no continue we’ve I PDUFA again, to time, different two So the said target be with the of everybody think PDUFA that. And date, dates, PDUFA yes, – would some unique for it’s really there’s for precedence

for regimen date people of those the XX novel in the approvability dates, of towards this July guide the continue to latter We full.

good we’re Great. place. think in I you. So Thank a

Ritu Baral

is quickly promise. I’ll and and I for inventory inventory just the a fielding WuXi you product situation how lot with us review Could supply have the stop, where been of the you calls Just then and about position? much

John Crowley

It’s a – excellent an question. okay. that’s it’s No, good

really good a place. in We’re

on We have very inventory. focused always building been

in we we the remember, into any you If clinical fact, pivotal year any study. of wouldn’t had in into patient for studies, go of a hand our until inventory at a clinical study, coming least

maybe through. Bradley, also Bradley, supplies, chain including but managing building the And you general the that globally, also Ireland want has ahead. philosophy the to facility. supply inventory maybe do go at carried builds, about So how we’re talk

Bradley Campbell

Yes, Ritu. Thanks, sure.

as of markets were able lot products product during we to for as what conducting intact John we was laser-focused the about been we So time different on yes, into move were the that China an a challenging supply PROPEL mentioned, ensuring talked study a world. have chain, we certainly moving and successfully around that out

come that that effectively. building WuXi John we’re in then to our inventory there as to the And mentioned, use support long this in WuXi and on invested then both is the chain can year we which supply the facility and year. part Ireland as ago do use The in we So next know expected of is here to sufficient U.S. commercial global and Europe. clinical facility, launches support plan the

supply like place the those to the a global both support feel that we’re all we we launches. and really So of in good have anticipated for demand

John Crowley

respect I’ll do and chain, Thank significant the we sure you, Europe very not various its out only out demand, United expecting as maintain further add for to building are then with to moves managing significant the just material inventory the supply launch, States. completed to the and points always it we It that inventory runs. make China also continues around including of to world, move bioreactor the Ritu.

of So outside also is significant being China. inventory held

Operator

Thank you.

next question comes with of from Anupam the Our Rama line JPMorgan.

is open. line Your

Anupam Rama

so last hard guys. it talked about COVID, conference on initiate given the Galafold patients for patient some but trends that building last the taking you’ve What for Hey or therapy. was of of seen Thanks to the much. month, weeks the like six get latter quarter, are over actually Thanks to much so part a so in fourth this? queue the At of kind you the question.

John Crowley

our budget color, I’ll than quarter trending above and we’re it Yes. we’re more but Brad this projections. more Again, to turn just for slightly halfway through

Bradley, ease I up, demand sure. see So that on you’re want comment still for compliance and that? some by fundamental COVID driven to you but headwinds think continued adherence, starting of again, do to

Bradley Campbell

Yes. Thanks, John.

I think key the points. hit of some you

placed of is work those the of those which system, to specific through the of year, question, last the your terms we end great. that Anupam, orders larger to are some at Just in starting saw year,

last even to diagnostics patients, end physicians able see, slowed said, us that in in of help year. disruptions QX. little think or therapy and trends all in major some things we those patients a potentially in add year, of in the markets. It’s in will John towards saw particular, our but as baseline we February, that their January, again, we see particular, doing just, similar patients I a start started those So ease. bit we’ve of as pretty did towards end new identifying year, just of still between environment to But the the And the

We work some are starting to those of through queues.

reflects see, to normalcy, more that and the between at over interactions medical bellwether. think were commercial there, and in-person for a what great a anticipating more We return to we Galafold we’re was patients, with and XXX and Conference Pompe. great meeting. and all had which an attendees physicians think is And World presence increasing the organization. I starting think both There for our live I

So, And so to slightly look we’re looking forward we’re growing the much Galafold. budget, of great. far, year for said, another and trends as successful very John ahead

Anupam Rama

for much so taking Thanks questions. Great. my

John Crowley

Anupam. thank you, Great,

Operator

Thank you.

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

is Your open. line

Joseph Schwartz

Hi, thanks much. very

with waiting just dynamics the that AT-GAA Neo-GAA use patients? of to are because could walk think make incident you contrast until thoughts through? in usage switches minimal limited expect that approved, for you physicians where Neo-GAA, that your AT-GAA, could seen in that think there patients us available? I is we’ve wondering Given your other you to greatest give uptake expectations Do now just you and was is on that the Or do from uptake? if it’s And minimal us get are you of

John Crowley

Yes. Thank you, Joe.

majority community, parents again, I the think all patients, patients, I Pompe sophisticated treat physician and are expectations they’re looking data. who of incredibly at are And our the as these them scientists are you the children, think built Pompe, the who with and on, therapy. Lumizyme. thousands patients diagnosed are at an of on the world developed are today approved They’re on if in vast Myozyme the look of

living know, – children, universally, a of years couple of begin they looking benefit, as we and Pompe. they’re after almost with again, adults, all decline to So patients

in study, markers, secondary So significance study that capacity in all statistical and where nearly population. patients. were data that and six-minute in walk, And vital our the switch achieved switch again, patients when forced on and improvements virtually were we most the PROPEL both again, of compelling the did XX% we

have we to compelling. to think switch with been we’ve physicians, data patients, I as I be is contrasts the we’ve in at data, looked given and as So in done. virtually that all think, other the our going are quite first think think several quite And studies that years talked robust the that patients switch I

think and our on I that’s as experience. gives what So well based also us enthusiasm

from see AT-GAA. from AT-GAA. vast of And what are infants on patients we patients As standard stay them through AT-GAA, than on to The is switch they had of XXX indicated, when Jeff more ranging majority adults care. switched

and that build. experience significant to people also caused world all has inventory investments, both infrastructure around think I our so in And in launch, make us the but to the the of significant support

to else well? dynamics So Bradley, you the do want as on anything in comment market

Bradley Campbell

you think I No, Thank captured you. John. it,

John Crowley

Joe. you, Thank Great.

Operator

you. Thank

of Our from line the next comes question with Dae Gon Stifel. Ha

Your open. line is

Dae Gon Ha

get Good focus Pompe, terms Thanks and Fabry I the with of morning. gene about If gene totally that. ballooning one. Great. we I’ll in I has being therapy therapy endeavors, for Fabry guess core the think arena. taking in mentioned been our stick questions. you programs also

sort talk know I through maybe about talked of previously targeting. can cardiomyocyte you So your you strategies? us

or there? it or something that we also We AGA much. if you still vector. should think how should there for – differentiation also cardiomyocyte about gene we should on? focus can Fabry levels, know therapy key the Thank very comes we about has And we that when How is think think targeting be about XD else endpoint to the

John Crowley

of that but challenges therapy, to about only of regulatory talked let targeting, expect at we’ve that broadly don’t What next United environment be other needs gene persistent No. programs to biotech not gene these challenging, before, of Yes. core Thanks, – clinic. diseases Dae environment, in again moving therapy Amicus there’s the States. more think an Gon. years and against Pompe of solved. in in reiterate durability, manufacturability me We into Fabry, several Again safety, we the in incredibly particularly science much the be

Fabry would It’s Fabry for based core We to therapy on believe studies. science. very need we accelerated a many biomarker going clinical and you’ll road work. with think what’s you developing on many, is it long gene drugs gene full think instance, So see that We our as approval of approved R&D Again, support is there all to together really franchises core of in be led sharply for think to Pompe. But even us that it’s for effort our Fabry, focus focused very anybody therapy. don’t be comparators. will years We a approval endpoints. or

benefit programs So the been stopped. we’ve therapy, served approved reality recognizing you recently the There population there the is but that have we with broadly therapies. specifically that I challenges very therapy think And the again, for were amenable different. very is risk clinic. that Galafold. going programs in of have Fabry, We has the that very gene specifically been that’s think think for in to as assessment very And gene have in well clinic have the be been of seen very,

be we focus field, for needs before having We of see the the for and I Again, challenges we think don’t Jeff, care, the AAV really we do again, know think done ahead. go become broadly. a think work these science We work technology So that more core ahead. to that still of transgene. are to lot there’s terrific on if on standard us, potential transgene. we’ve with you want delivery But Jeff, there comment developed differentiation. to a

Jeff Castelli

Yes. for Thanks, John. the Thanks question.

to actually engineered enzyme. possible enzyme. kidney two alpha-Gal kidney, importantly, only gene to we So the in our differentiated. not aspects produce therapy heart, more is Fabry a skeletal promoter, capsid more but and there’s many transduce is John as in stable, transgene the main trying heart, transduce places then active to in only which muscle alpha-Gal really And the secrete so trying a of be or our program ubiquitous One as use to as and mentioned,

look importantly, is you cells I just – the a of really in approach levels then important enzyme our very to substrate and the and of produced looks in We not studies that’s and And translate we’re towards. the requires should it be substrate mouse by producing reduction enzyme in in it’s look – effective. from with but then plasma, resulting When deeper the AAV, clinic, therapy important of the the ultimately as get blood much the given key whether at what enzyme, to see assess our animal dose working at on that of know and tissues gene and to quite it’s we to translate a parameters. tissues enzyme good those in clinical is So it substrate one bit clearance more reduction to blood something in but robust think that doing to we studies, transgene. way

Dae Gon Ha

Great. Thank you much. very

John Crowley

Great. Thank Gon. you, Dae

Operator

Thank you.

Tazeen next of the of Ahmad Bank Our question comes line from of America.

Your line open. is

Tazeen Ahmad

guys. good Hi, my Thanks question. morning for taking

ask meaningful come Can Or And is For is me, would and there. a guess, is upside benefits maturing, sorry, just of adds want the peak most penetration I you for target us for Brad. be to now the questions Brad, any has kind patient pandemic, quite do within about as you maybe those like doctors? you through in to, sure of of one or where it time. increase where $X launches that And where I already to it share? trying new slowdown market that if of more before doctors? are billion the from, about making to sounds going than is are sales aware for think expanding that prescribing more talked this it in lot from Galafold, just appropriate to are walk just the some you pickup of relative you thought pandemic had you doctors most

Bradley Campbell

address No, I’m to all happy good questions, you, Tazeen. them. Thank

really couple opportunity Galafold. I a think So again, which growth And said of as before, we in key remain for drivers, embedded confident question. your there’s in we overall are the

all, just a first $XXX So roughly million passed XX% and under this share of treated little global amenable. a year, in of we sales, that’s

So anticipated if we’re business, double as from penetrate continue current XX%-plus just markets. we markets we the as launch markets just the we revenue potential our share, in expansion, continue our geographic that’s to within market current and to the assume can already get you in, almost

to lots opportunity to So of just patients. continue switch

driver. growth key one it’s So

the to point, diagnosed diagnosed see one, untreated we continue in second newly your to and the uptake, will markets. The both

oral talked And significantly with market the comparables that’s grown before into treatment other an see penetrating There there. about already you we’ve space. that diagnosed same as diagnosed the hand, And was that one into the we JPMorgan we launched, shared think untreated, are On I diagnosed – something launched that amount when by patients. roughly space. sorry, estimated treated was lots there patients the I effective an in untreated on anticipated of injectable that there of think we’ve we

many, a more out this market that’s who that many there from reality one diagnosis we But believe undiagnosed the focus. key are So growing robustly there. is perspective, is and are a patients

Fabry and And that population amenability with continue those to to well And the terms are rate. partnering have screening pain, patients talk late-onset patients, MS patients to so see space in particular, tend many, undiagnosed, manufacturers looking a we’ve populations for efforts come. as many that I Fabry think patients populations, to towards diagnosed high-risk about more have those those new we’ll years tend of higher the finding important and who efforts in what’s Beta, Fabry be the much that to many like in started put about in

of question, Galafold think you the business your And remains a come can maybe do waypoint come so a underlying to I passing patients we shifting that and strong I business seeing to opportunity. growth and drivers confident sustain that we to think Fabry large previously timing, that bit the opportunity, growth growing get us untreated see is market, actually strong. what but our think – you’re very or little upwards from part saw market how, rate do the will to your latter of maybe we’ll on a strong. we’re year point continue to years from million The opportunity last $XXX evolves, as I just $X bringing to now recovery, you So that will original talked that a confident billion of before. Galafold terms to is treatment. from about The in that remains this anticipation $X many a double-digit those what But billion on for

Tazeen Ahmad

percent ultimately, amenable for Galafold? And up deeming you what did end Brad. mutations then Thanks the Okay. of

Bradley Campbell

Yes.

even example, is the tend XX% to amenable We’ve than we And patients. estimated terms one-third of mutations, that’s in oftentimes, greater. by, the launched, never But between the amenability we driven market amenability where or was we patients, one-half which of found So at diagnosed be and Galafold. do a rate many really one-third. where have markets many first when cardiac less to for

mutations particular, studies you showed amenable some that’s the newborn the found diagnosed in said, That well epidemiology, that in look newborn over were market. the if so current Galafold. of of the to that or of And the being XX% screening studies range those at XX% screening

think lots I we there’s have the Galafold where opportunity in of long patients. to help those think and see, those for term, a higher will patients of you percentage amenability, that’s

Tazeen Ahmad

Okay, thank you.

John Crowley

Great, thank you Tazeen.

Operator

you. Thank

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

Your line is open.

Kristen Kluska

World Hi, analyses recently question. PROPEL saw good morning shared at everybody. Thanks my some I subgroup you for that taking the from trial.

data I switch with could outside the some already of ERT and in sets in aid if the experience strategy naïve your if discuss this So ask of to commercialized and particular these having both wanted advantage of you you might how having conversations help clinical patients. the

John Crowley

you Thanks, from Great. you. launches. maybe, supporting hear to And then the on we had want for how the some Good you’d data Jeff, that we do Kristen. if shown? Bradley, PROPEL subgroup to maybe see that summarize just everybody comment of

Jeff Castelli

thanks, Sure, for and question. John, Kristen, the

or levels So the preserved across we’re really switching seeing on FVC, imperative across those on of severity. versus AT-GAA. we we the AT-GAA It’s of that, and of are or that more different important six-minute going severity baseline a main alglucosidase saw patients alfa ranges walk different baseline, benefit new had whether subgroup to see improvement to presented more analysis showed on

of report some we’ve outcomes, really presented out. in of But the anywhere. implications for fully And coming yet on which patient-reported impactful particular, specific not to you use we’ll new in to the going look to So, to have and our X/X we of the Brad this terms think we’re that that look we from at data course, other continue besides we’ll will of both PROPEL into key – extension, PROPEL, Brad, how from data more analysis in Phase to that, launch. data and be secondary subgroup long-term that continue maybe analysis, year, we those over data continue

Bradley Campbell

for and be have which Sure. And to Look, is switch has mentioned, the well-controlled patients. study, their appropriate to we most an will be are John company incredibly to believe data I and And naïve and both may John we’re product think, patients. robust community, sophisticated the AT-GAA mentioned, only as continue set. a patient they data-driven sponsored, in understand, most physician this – first, they as

those is mentioned, baseline get think AT-GAA and as I want look down you different we that subsets to et Jeff As we in strong patients, as you what into are seeing a impact age, study see severity, cetera. that of across, has

data so of are would life. mobility to will I also think impact population. non-ambulatory both AT-GAA secondary further as critical think course, And the but for strong are different quality – of patients across on that important for would area support into what is we robust unmet is believe, or significant that of other strong factors they’re and naïve patients, Jeff that those more Phase we said, AT-GAA interest are and think which set levels continue to this we I be that and is need. really the patients, mine for as on number switch endpoints, And what I mention a all support showed we physicians, that points, PROs, or a other X/X impact a means of function the have which end data, impact Pompe as can pulmonary I severity. in And for of get think I have patients just from we their those there point whether

Kristen Kluska

you. Thank

Bradley Campbell

Kristen. Thanks,

Operator

Thank you.

of Yun with from comes question next line the Zhong Our BTIG.

open. is line Your

Yun Zhong

very Thanks questions. the taking morning. much for Good

AT-GAA. on Pompe ERT I follow-up patients question So, for have a switch

keep is the would patients on input that of are piece patients we that ERT, one on like if their So those physicians therapy. they from heard have to stable current existing

do those of starting to believe that maybe loss good that you be show physicians patients patient population is on don’t for target? be are existing or think do And would to I stable those what response Myozyme who probably could Lumizyme know switch evidence – idea? to strongest patients in a ERT, So the to those convince so easier you are who existing the

John Crowley

think several the those the think that’s Yun. the population, think shown dramatically, And be already ones that we decline, to those Thank is we would certainly expect. people what absolutely, have Yes, sometimes greatest We Yes. population. next years switch eager switch. you, most ERT and within opportunity in within the who we

to think to who those just into to the would currently declining, patients or for we next of plateau stable Of want hundreds we on And think people patients. to get go when those subtly of switches beginning be start the switch. going I they’re even how see open course, tier are kind most ERT, of those to many

why switch in we’ve only of I again, key the disease, patients drive improve patients. what’s that we physicians And is stabilize scientists, what and again, And strength, going pulmonary not on day them. but measures them, can for it data-driven think, muscle I that have many a was patients seen that’s should. world in actually switching. be into I mindset people too, to as and our the say, could clinic, initially this for And to before a think just of care wait goal on to I stop decline is think data would you Again, very our well, decline where patients stabilize we the when standard where to helpful. went would

So, time, standard to over ultimately patients of build switch I body all think of we think as evidence, ERT continue the a on will to care I consider AT-GAA.

Yun Zhong

you. Thank

John Crowley

Sure. Thank you.

Operator

you. Thank

line next Ellie from UBS. the comes of Our Merle question with

open. Your line is

Unidentified Analyst

of loss what are taking to constructs? your of disease-related Johnny are On you is the for the therapy on Thanks Hey guys question. this that kind Is or had for aspects from learnings the durability? gene Ellie. theories this? what CLNX, And the led it of

John Crowley

we but Again, We at dose a that that the disease in didn’t we the these know small stabilization, had that previously to over very a time, everybody we children, we it Yes. effect younger tragic initial durable was that very, looked children, patients given, data. have CLNX, announced was again, with saw remind in the first. an disease in long-term when saw that for while that, know XXXX, exactly why. decline. We all end of particularly Toward low don’t

done the disease, intrathecally could also follow-on show delivering be the optimal, given route into to of ability Secondly, that cross the probably so of a we’ve it lack it’s And with the think again, nature see that I delivery. is also it correction. of a magna yes, some experiments it more intra-cisterna was

in CLNX, programs And acquired several from Given we we the challenging. when biology ago. incredibly makes knew the that this years Nationwide it

incredibly So dose, a and administration, some it’s probably route to of of CLNX. difficult of biology unique just combination that, all

Unidentified Analyst

you. it. Got Thank

John Crowley

Sure. Thank John. you,

Operator

you. Thank

Company. line Our & next question Gil comes Blum the of from with Needham

Your line open. is

Gil Blum

taking well would for little fact Considering fits you. some the core you thank around have Amicus and Fabry more everyone that morning and flexibility, that you bit our Thank consider program a with Good Pompe? in-licensing the might questions. new strategy

John Crowley

Yes. Thank you, Gil.

the with point important savings cost here. we an on hit You in have that R&D

to profitability. $XXX million next over over will again, the us four that Now, years, take

continue will will research advancing have Again, a core of our development focus We and Pompe. core programs be in franchises our and Fabry pipeline. to in our

will We medicine, technologies, in do in continue also technologies. to and development therapy research genetic the particularly next-generation including broadly and looking platform core fields of gene in at

our Amicus. think, been at address With targeting promising labs looking more have and core like and technologies. Those at here in to developing are very have we will We some, developed refocused we team with need safety. durability that problems mature science and that, technologies a

look year next complementary over think technologies. I at we the two, would or

For genetic immune delivery instance, of medicines. maybe in the in of the field modulation

to us of of genetic in the applicable. an at here it we’ve again, be important that Fabry, the bring we restructure and pipeline, the the focused potentially outside of gives at ahead by So, technologies, that, could in we’ll areas done material, organizational non-viral AAV but potentially that but think it well Pompe, other in look bandwidth the in refocusing mediated point the look financial field, as and you delivery other years up our prioritization where

build now We think, Amicus with world-class we by we invested first, medicines we’ve teams half delivered we’re Amicus, product around a world. at advantage a profitable. I last global other So and the fully technologies have at look commercial over will when think organization. second, the years great All the to six another Amicus are that yes, substantially area

moved have AT-GAA the substantial to leverage launch. operating we’ve We as

in a launch can execution. $X operating just with That’s So further and billion investment for leverage a tremendous minimal medicine globally, potential. we commercial

organization full achieve other very can once again, profitability, think, deliver commercial that we we So products at we around the could through world. carefully we look our

next of foresee I in wouldn’t So, year beyond, but could or that two, be a partner choice. think the we potentially I

ahead to this again, build and years financially of both the gives flexibility pipeline. us So a organizationally our lot in

Gil Blum

Thank for our question. taking you

John Crowley

you, Thank Gil.

Operator

you. Thank

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

Your open. line is

Robert Finke

terms would And for naïve team This and you of addressable is in question. would And Sanofi? this? Robert how thanks the label AT-GAA of good the expect likely for Thanks. in to commercial that, patients? indicate in opportunity, Finke Hi our of morning taking the tied patients for share the plausible you characterize both into underperformance from and what What on Debjit. terms is explanation market

John Crowley

Yes.

Robert. lot a questions there’s of So there,

unpack Again the to study, study it. everybody it about me Let PROPEL randomized remind XXX was our patients, was pivotal X:X. and

on About on study one again, significance went often statistical in statistical AT-GAA, remained two coming superiority patients standard our we six-minute vital of every the endpoints. or we to plan, ERT prespecified care, patients almost of forced superiority to walk, we patients. XX% in capacity, for Myozyme/Lumizyme. on were of showed So statistical secondary care, standard other that And in showed with switch on analysis that to in – superiority multiple showed population,

data seven in anywhere, who in remind go patients, the care, but time pulmonary across in don’t So treatment-naïve there subgroup, they the to an they confounding Myozyme/Lumizyme. on of population, know And the We those expectations. What overperformed patients, strong significant to everybody, first the ERT in a patients on did. were We standard any randomized improvements. we for why, study, that much smaller only to were study study switch for six-minute function, was we In saw treatment-naïve population. incredibly AT-GAA, board. improvements set coming again saw saw again, walk

again, But to didn’t study we when everything of known look favorable seven published, the comparison standard patients, you there. relative for it’s working very group particular in patients. pivotal the just the If this strongly, we believe look in very Neo, COMET very in, Again, also frankly, that’s well population, more as look of is difficult-to-treat a as in care, everything look of The reminder, works treatment-naïve favorable biology some patients control just where switch the end, to five five care. at were at about and five of you – straightforward. strength. was small of in it AT-GAA showed treatment-naïve, our having to So X that standard a saw who the we that very comparison we study, Phase all dramatic in improvements out cohort patients been put on

the has strategy for well population. our think seek we several respect So in the of switch opportunity years, market, the a been, With in all as both a approval as in the to to is treatment-naïve geographies, population. XX%-plus switch population next

revenue standpoint, think commercial a from run, important Pompe newly several and to is we the desperately of be here. with long diagnosed they have also and have the people But percentage that for commercial the diagnosed a important we treatment-naïve full small So who yet in think treatment relatively years, opportunity next options. population patients, the in

So, that points. of your hope addresses all I

Robert Finke

Yes. Thank you.

John Crowley

Great. Robert. Thank you,

Operator

Thank you.

Our next question comes from Berenberg. with the line of Shu Zhi

line is open. Your

Zhi Shu

to I much. my just taking very Thanks Thanks want ones. quick Great. ask questions. two for

about your in And rationalization, there much. committee was indication given Thanks around a advisory should is, therapy review any we the workforce on FDA thoughts convened? very be any Batten will the with potential the franchise? How there? think disease mid-cycle that secondly, your gene AT-GAA, meeting First

John Crowley

Yes, those address of questions. both Sure. I’ll

we States. now not us review. So we path that that we they Batten, hasn’t told the CLNX very the remain we’ve the expect to said, data had and the changed. given to in FDA unfortunately, program. forward again, be seen committee to respect do previously confident advisory in excellent we mid-cycle convene in We discontinuing writing that an an are, again, The United that would With approvability had where and

of way to to We’re help the program. those outside that looking population with find to the small and of number CLNX development help formal to at ways a learnings take other children

January, on dose, had to There, need young a in everybody, patients we we – at four back So also with we low science. have a one and treated more work then CLNX, remind there that dose. we children, have – said, at three four higher the

for scientific we’re until the not So, be all path into we the forward the then to clinic best decide program. forward that going and that data, get we’ll moving

us. key the also discussions. regulatory to work particular, then toward There’s So And science longer-term in look more the in data children. for at year we CLNX, four there’s more for those of want this ahead end

this No stands did So clinical to it program January. that’s – in further immediate studies. exactly as move

Zhi Shu

very much. Thanks

John Crowley

Great. you. Thank

Operator

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

John Crowley

of all day. you for great Great. was That we Operator, that’s Everybody, questions. have. Thank a set a great have listening.

Operator

concludes today’s conference gentlemen, and Ladies this Thank participation. your for call. you

disconnect. now may You

Amicus Therapeutics (FOLD) 24 Feb 222021 Q4 Earnings call transcript