Amicus Therapeutics (FOLD) 28 Feb 182017 Q4 Earnings call transcript

Good day, ladies and gentlemen, and welcome to the Amicus Therapeutics 2017 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference is being recorded. I would like to introduce your host for today's conference, Ms. of Director Senior Pellegrino, Investor Relations. Sara may you begin. Ma'am,

our Good Financial discuss to you Results joining morning. for conference Amicus recent Highlights. call Corporate Thank and Therapeutics full-year XXXX

Slide this Chief have to Private Speaking may X, Portfolio call, and our participate Officer, Castelli, and of Chief Chip on the Officer. here and our John as in Medical Executive Financial as the Officer; Officer; Securities available Chief are Jay Crowley, Chief on the as also and Operating to business Act we On forward-looking of call, Bradley we within session. Reform Chief statements Chairman XXXX make relating prospects. Dr. today's plans well Baird, Officer; President referenced Campbell, Q&A Jeff and Barth, meaning Litigation and

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

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

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

Great. activities host of Today Day to is we everybody. day impact is held Day. why of people to every than know, as anniversary XX that of pleasure Disease XXXX Rare every Rare Sara, are many initiatives taken morning, vision It Disease in on call. throughout million our February to It variety Day. countries work an to day traditionally XXX benefits a beyond Thank year with to Rare and good our through of being disease world. it's diseases and awareness honor communities deliver extend At you, the the people a rare the a It’s day that Disease in fulfill the to come place living the to rare estimated XXth has day and diseases you broader of and more Amicus we on raise collectively significant today's medicines. of that a unique participating reminded each of are responsibility corporate rare last

discussion, for the in programs two to XXXX extraordinary and your we an begin was Amicus Fabry I'll Slide call So Pompe. year our leading for key turn four to today's across priorities achieved attention strategic and X.

$XX.X launch mutations amenable million than revenue at with our we First, treated exceeded Galafold with XXX more patients target Fabry in and global year-end.

established with balance million Japanese treatment we our the with Galafold. disorders sheet hand. for shareholders. value living significant of and laid lives focused with patients ended long-term novel for global Three, U.S. Throughout we creation we company proof-of-concept XXXX, cash metabolic on paradigm and people And submissions leading rare strong our definitive for Second, the to regulatory a continued the $XXX biotechnology for build fourth, disease. Pompe our we we for of year completed for improving and a groundwork patients, on

Turning priorities. strategic on five in XXXX key X, are focused see you can we to Slide

doubling XXX are this patients confident revenue and treatment range Galafold million full-year guidance. on today, First, to than we achieving XXXX highly in expect global in the of we more million with $XX $XX

Next, global a we Galafold that with in has PDUFA XXXX. NDA our regulatory Galafold are to target and continue under half accepted just Japan XX, first of broadening two a priority review FDA weeks year, six-month the access date committed the In to track U.S. announced to we through August additional we toward approvals. decision the for of ago

pipeline Pompe to that to are this approvals. we paradigm value our XXXX. later least was at milestones Third, further ATBXXX/ATXXXX the the early focused expanding data in medicine so highlight for to can call. we San Pompe refer Diego our we to I'll set which of regulatory regulatory, a novel clinic treatment manufacturing disorder today's believe and February. and has committed presented Symposium that towards by significant in program the more and create advance the preclinical into one validated by rare advancing and approach as most move potential recent on that at laser We World we are clinical, on achieving end key submissions metabolic new our Fourth, the

have to with so rare benefits and metabolic our our expand in As and our have Pompe sheet and our current $XXX technologies from are finally, potential for current that programs next-generation and metabolic significant recent capitalized patients. to deliver are we for strengthened well And our core rare we significantly for focused we maintained disorders on and offering disease additional pipeline, obsolete to therapies Fabry an invest balance very in pipeline. innovative the million the follow-on

to precision turn Campbell, that in COO medicine Brad? highlight call Brad progress our to President will worldwide. in and to delivering now introduction, our people I over geographies the our with So more Galafold more

amenable Fabry their for on revenue Galafold we target exceeded Based million people in to $XX our for well million us in launch I'll we set Galafold as on sales Fabry initial of country-by-country processes in year-end new begin our of to XXX million disease recap patients $XX revenue XXXX we the with through is than By and growth XXX can and Europe. what were reimbursement on which more for in in securing who and year as focused our our in Slide success sales Galafold $XXX on pricing full-year thanks there we for double XXXX full-year physician opportunity global track totaled a revenue treatment markets was will peak adoption first full believe Great, Good and $XX.X million. you Galafold believe on XXXX with have patient guidance. morning X XXXX, final mutations. John. with of our everybody. revenue a XXXX know and which plus

as Turning of Slide to update progress February now so we've XXXX great an on X, I'll in XX. far today, the provide made all

Now revenue focus you the to track we'll guidance for introduced throughout somewhat for that our streamlined set on metrics we've commercial of year. and XXXX, regulatory

as diagnosed high of of in number to momentum were in mentioned months patients the press well. year reported diagnosed patients, who John include that a more into coming naïve with as with are introduction, patients the today. patients This As more a today's proportion continued we reflects XXX of the handful switch never two newly the of previously well in than year. than patients treated treated, mix new but growing The this strong release Galafold continues a being as XX first

this which to is year patient launch as these segments as spectrum seen day up in penetrate the markets very across among females adoption we and throughout strong rate we've we see well markets. And males continue continue to adoption the and our of with and mutations. in have we line late of existing the also including and oral regimen. as continue a Overall disease our compliance expect dosing open with other much classic to the We into new strategy high onset every

goal medicine quarterly payers generally Galafold As data EU five the as with already in industry for averages. a a numbers We've to an quarter, the strong move pricing we we we've away From XX of to and with resonate and a countries and of reimbursement as metric instead top-X secured that set revenue ahead final reimbursement in reminder, and year. towards from now well markets progress launched clinical proposition including all precision patient to in Europe, markets XXXX, the America. new a we throughout in looking reimbursement our ahead additional pricing year oral South and and highlight and and and pricing do or value to in of believe expect measure continue next Latin revenue pursue the continues will offers a countries Asia-Pacific, sales perspective, opportunities patient new

is up of approach highlights amenable well inclusion the mutations amenable identifying a in to a date. regulatory great you mutations X, do perspective, this we example mutations that regularly in XXX lookup have incorporate feature six EU listed of for our to target markets the precision total if mutations listed of and and this in the secured the Again, the of XXX turn proprietary is amenable to One time And table as Slide new table. label as we label slide from broaden in approval. a submit lookup the which is at our mutations to finally, to to unique we now have on new of original from continue medicine approvals

amenable the billion proprietary to what on With population But an to we opportunity Canada, well we still continues others, novel for next-generation Fabry deliver Pompe program. people Fabry also We with treating existing with there with U.S. in in believe preparations key that, I'll In mutations. potential underway as committed hand for to our including our update mutations well significant the on as for markets. year in those and we is to the enzyme mutations half our the XXXX are there be well focus with to expansion continue opportunity John call is and U.S. summary, as foundation that the we're patient approvals. a rest peak our therapies evaluate. Fabry and for technologies markets to guidance and We patients amenable of strategy believe new innovative with advancing Japan a patients migalastat the building in our approvals geographies drive and further of sales and Galafold timing remain back among adoption growth other to replacement therapy all Fabry to as launch continued our track this the Japan, non-amenable provide in co-formulated is in dollar global substantial

am Brad. and pleased clinical treatment Pompe substantial our some across highlight for ATBXXX/ATXXXX. regulatory novel paradigm now activities Thanks progress our manufacturing I to

latest a in Diego Pompe Just leading from weeks disease. with study our few the had world's the experts the the clinical during in to set share X/X data ongoing we Phase opportunity ago San World Symposium

key biomarkers in We data treatment results the to physician functional feedback on the are now XX patients. all durability magnitude, those key and of terms patient across extremely on the of on pleased months up consistency of and for three measures safety, data cohorts, with in

underway is Slide to few community I'd Pompe global the the that key to for with critical manufacturing now so our XX, activities future to a updates. like Turning success important and supply it highlight

The GMP are reached comparable. right agreed studies. comment From analytical of on the GMP successfully for the and ATBXXX material this the three with I'll completing as our and scale on under comparability commercial to quarter FDA the to the and the that way processes we the First today the very with using in the XXXXL that there between comparability report testing analytical happy used XXXL reviewed we at have GMP campaign important additional also conditions. of batches, well are time campaigns agreed and substance XXXX can strategy we our we're GMP comparability to in for that vivo completed materials studies of call and establish drug the all process have that the only registration FDA engineering as purposes. to test that meeting GMP of out for set I'm agreed methods end XXXXL analytical XXXXL batches to and number XXXL comparability Two, the at methods. establish agreement the FDA of we our fourth same and the first experiments the the one, time on we in engineering supply this our scale first our FDA materials comparability for repeat study and our directed

approvals. for Slide control foundation to within of and fastest to all XXXX toward that lay I'll best in focus are the activities ensure Turning on the to and the our success XX path

data clinical several robust assemble have a clinical We activities to package. underway high-quality now and

patients a patients treated X on Pompe our XXX as are Phase ATBXXX refer retrospective additional POM-XXX treated X/X clinical planned in includes First, ERT study the XX Pompe clinical natural and X/X to XX our Pompe that in results context to of X to months ongoing Phase up had to for help the we've study enrollment what provide current of our studies. for we Study patients to history conducting secondly with up we disease

Our as assess registration to POM-XXX prospective to serve also a potential treated This running or observational also to safety and a third, functional study ERT. second we as is run-in observational STRIDE patients refer are the in half outcomes in we study standard-of-care may of for start which with study XXXX. the study expected currently

our XXXL work we've steps and XXXXL FDA towards GMP final material clinical are campaigns mentioned, between to I've on progress GMP next As made to manufacturing the and batches. our already and with significant comparability XXXXL the agreement manufacturing continue

potential States. likely facility based potential pathways facility China our additional here at XX and believe the United in the well Slide expand to next as the summarized XXXX. we few beginning supplier And Wuxi in the the that source regulatory at second existing own our the capacity of for same years We Amicus also on we we've in manufacturing a Biologics as plan for outlined

this We and provide to with of discussions expect continue EMA we year. to an the second update and in collaborative in FDA engage quarter

soon Before ahead over living turn can. that, reiterate providing important treatment like we more to with could the the to pleased over that of be with I as as and to remain many With go to Pompe fully to Chip, hand results. the We I'd committed this call access people discuss program. as financial ahead XXXX as Chip not call go our me please. this paradigm advancement possibly Chip, let to we possible Pompe to

good everyone. and John morning Thanks

Our on slide begins financial statement. our with overview XX income

goods dozen XX.X% costs goods in had in reimbursement in as For our why of sales to associated a net for sold sales Galafold more of XXXX. of included in although percentage full as six we place uptake continued of see countries year the recorded at have recorded the cost products, sales first sales a our we Cost is for XXXX $XX.X the which XXXX year XXXX we countries only with to the of million full expect compared revenue royalties year-end sold was of trajectory. launch well we as beginning XX.X% as for Europe, than we manufacturing where year-end

the investment in $XX.X recording R&D costs the XXXX. the and The Epidermolysis During XXXX full of expense to was of expense increase we for $XXX.X the increase of significantly clinical year costs the million increased in to related costs SD-XXX down development the compared to our million million as cost in the $X.X R&D recognition program XXXX Bullosa. then SD-XXX to in manufacturing included R&D $XXX.X wind study. increase accelerated and for of ESSENCE ATBXXX million in The due increase and

impairment compared gain XXXX the and study scope full-year to discusses the the will we that additional see fact stemming on statement we and SD-XXX. Moving benefit million with shareholders geographic milestones million $XXX we related $XXX.X the charges. XXXX impact The value million reduction share. as to We income Net of the statement, $X.XX I'll to the the the our $XXX.X to loss related a to tax items increase per the tax down ESSENCE stemming commercial for share evidence X were $X.XX compared per loss $XXX here expanded to recorded from R&D or the XXXX discontinue of non-cash again income ESSENCE there about for net and continued million administrative Galafold the Net investment of note you full-year year SD-XXX are million $X.XX charges former of several in-process deferred excluding and $XX.X was study XXXX. reported all and non-cash selling, and of will $XX.X finally are I tied consideration Phase development million or was impacted related recorded the loss any for results to the in was or the am not owe this of from further to [indiscernible] non-cash SD-XXX $XXX $XXX fair the that the general that XXXX. million decision million represents income those to per ongoing expense launch. for of total liability share, for loss

our As shares of approximately had February equity outstanding financing. February which XX, we million XXXX conjunction in follow-on XXX.X with shares the issued immediately included XXXX

$XXX XXXX end a of $XXX.X totaled the XX, our about cash to the on cash, and Slide comments at and current cash XXXX. equivalent financial to compared end Moving million few million XXXX guidance, securities marketable of position at

Our a million in net public offering. cash February in line with $XXX million. strengthened million Total our spend with of to $XXX.X was spend million cash for guidance further of was completion of balance the sheet XXXX successful $XXX XXXX $XXX follow-on

to of to fund cash, summarizes capabilities revenues, ongoing continue we Pompe and operations equity volumes Potential program net [ph]. today manufacturing key our development Galafold recent business cash least XXXX collaborations could our future million. on investment our offering the For of financials into capital year XXXX. we between and and and million capital proceeds in our full $XXX With $XXX future Biologics XXXX. for at This impact expect Fabry sufficient [indiscernible] spend year have expected

but Additional back details happy on filed which I'm to for during annual we'll it found now can questions X. be any address Q&A our John. report March in to turn be XX-K will the

Amicus. was Thank certainly a you, XXXX Chip. for year tremendous

priorities to with our key strategic as five we XXXX. We begin are on track respect

and create are to the even lives that meaningful part vision to XXXX living and highlighted treated long-term focused reiterate Amicus patients In patients Slide our As to on we more more the with Slide XX, patients again impact goal value with call more the of of maximize year this in diseases. in rare the billion just of and revenue closing we beginning in a I'll XXXX. shareholders with look of than make for as I an on importantly by $X XX on difference sharply here our medicine than

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

is now line open. Your

clinic the Fabry some throughout so program the in that have some ongoing there year? this advancing in on how for Germany of two for countries of get then the talked you me, and could guys, you - co-formulation to therapy, give reimbursement bit Thanks added questions getting also factors activities some what a on here patients the beyond that it wondering us XXXX, the front, And are XX Hey in the breaks gating on new given maybe down just of about guys there’s on progress much. maybe just the little into XXXX?

I’ll and let Anupam. Sure, Brad the me have second Galafold take take one the the one uptake first

Excellent. Anupam. Good morning,

still. the of how within countries through then the can distributed of bucket portion you distribution assume terms markets, in of So largest and I largest new the EUX the patients patients that the and patients that good on But where represent in countries markets safely of will we've a reimbursement Galafold Europe are gotten think and other increasingly question the and represent throughout of in Europe. outside largest say I getting doing pool of great job of launching to now we and approvals that markets patients the are and other patients

the opportunities So, very see with that continue progress grow markets U.K., existing continue there, France great and we as hope launch to would feel we we new markets the in but be to comfortable fact others to Germany, expect to and see well. great

Anupam on things just here, I'll we're co-form of comment couple the working on.

the are the necessary all to clinical in to XXXX. regulatory First, that manufacturing clinic allow plans, the enter

for treatment, Fabry other gating patients you therapy particularly that options mutations become and into with the moving whom the are continued now in forward what our that diligence, for completing suitable with people developing co-form? therapies now we're non- newer Secondly, know we're for there providing be amenable potentially for important in there are and part part that factor of and new beyond those gene the not for living Amicus step will editing potential Fabry. even the disease, And living diligence gene right is the an that with Galafold our a technologies technologies of better patients patients portfolio evaluating particularly even space, is other could

thanks Great, the taking for guys. question

you. Thank

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

Your line is open. now

be on be the - are for first, Thanks that so Japan mutations you on relative therapy? taking to number can you would first will one Maybe still talk then about what terms have be so good what I finding identify have patients, for orphan secondly, morning to that Brad even all the my people early case? put pricing done generous you for is questions. in think of U.S., to you amenable drugs think much of far launch the historically, Hi And to your Japanese and of opportunity in in us tends be guys. more

There Japan, questions, Japan before in in Good second thanks replacement proportion Tazeen. an of Yes, significant as today a largest in Fabry slightly our little so treatment terms we've and the number we've patients. done high Fabry are approval or that bit launch in there. individual things unique said so diagnosed there's treated of terms launch getting is patients enzyme XXX sales. patients anticipated therapy of all their country It's markets a in patients, of the and of so for penetration we're lower historically doing in Japan, but there's ready a other a untreated so that

a similar sales suggest in that job, also So there so team and our mutations there. great great patients we physicians markets as side. and great have estimates building some around the believe We’ve it's our of people medical side a amenable and we're well working now with done we've on hired done the the leaders far amenability and putting in and of field the is to very XX% job other XX% between which somewhere

technology, science terms that team Japan. we that of came and other migalastat chaperone thing the attributes technology So of great ready we've Japanese believe first we've the only out from of the we're for highlighted is in got to go. the a And unique market, perhaps before

significant as of treatment a So oral of so also bit a the give there's that well. a -- mentality a little enzyme the burden every us of delivery could that day infusions extent sense home are there migalastat it's in home with there bit versus the other replacement little grown no and there advantage unique therapy the perhaps in Japan,

we're of underway opportunity of there, much and your and lots So question launch very Tazeen? well in preparation terms great then second remind me

On pricing..?

thank you. pricing Oh yes,

so remember than therapy, provide the enzyme yes, replacement infusion of There our believe slightly forego in higher average for pricing right. at we've bring you're it's means year pricing is a some a while as within perspective pricing it patient markets, costs unique weight but to than in from parity are perspective, perspective, an be a therapy associated then pricing the Galafold, which strategy again that about that on price with average Japan likely we we same that patients So historically. sits we'll markets, and that tend continue and to corridor we value patients our talked the which lighter Japanese basis proposition mechanisms still premium well unit the with per a other $XXX,XXX but part the per from from other $XXX,XXX

Okay track? timelines path accelerated an to terms similar in there and Japan in fast of U.S. is

closer you Well, two what can for get process, reiterating reiterating receive first for that to of we so three and timing. obviously of then track in approval but to for are as After then launch, actually year on sense we track we'll the anticipated are an we're on we've and said we're guidance. better reimbursement again this about have month half anticipated a get we a is we when that there's the the approval approval course

year or Japan? Okay lastly, this sales and your guidance then for not for sales does does include

are markets overestimate including and for this would portion a and not be year. markets, the isn't in contribution even the largest year, two sales we of the States, a United Japan having year and those U.S., careful of although launch the That country to though the remember individual Japan those

that. an there's to will certainly we if believe opportunity that go revise do As forward, we guidance we

thanks. Okay,

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

Your open. line is now

numbers, mean and to guidance, patient barely keeping of any are for want be and the morning Europe how to persistence Galafold compliance, and you're as guys. to range top get seeing about question. in pricing terms should and your guidance of pull of the should what growth as rest about I mind? for in that need half about compliance Thanks taking persistence asked you there the things you far we out if Good just world. think in I XXXX we

I we're $XX that confident of let Brad on persistence, reaching Ritu. very to in but again million guidance compliance, the I adherence, comment think million $XX

what the As we starts, $XX we'll for $XX seen high reaffirm and seeing want will the at need do market we comment to strong but of on compliance? that, you revisit Brad, guidance trends million in confidence to million. we're we with United continued see now as the the whether we’ve hard developing, take have new in Japan hopefully and approvals look we States, patient a to as certainly

remember if rate rate, last compliance. were get patients your range of and growth the the increasing of to which essentially you'd Sure in starts, to at so middle you sort increasing of point of year of Ritu is to take from terms that an new

good to of that's can we start potentially it higher. drive think So and a we assumption I with think baseline course

patients So million that we $XX to expect certainly new $XX revenue. million contribute of to to

see as As very rate certainly persistence, above continue of a would rate therapies averages and in perhaps said as with I well. the and we well infused high it chronic to expect relates see oral of with persistence my high compliance to a compliance that even therapies to you as and remarks

are So with we're we very pleased with the those the having physicians and patients rates that of course product. anecdotally think that and experience reflects

how first your on seen then into you that on is yields process that insight ATBXXX? first gone, you've can And GMP of goods does the batch, talk give about you potential it cost

were very good. Yields

sufficient of for campaigns. I manufacturing other potential still the and directed underway. It is next begin get again registration is series the think there campaigns us study already have to improvement further we we into for as

the term We for with added into near studies will that in additional material have Wuxi be available for other our and purposes. now capacity for commercial planning

think it’s a of of the goods premature on we'll of what cost be. place sold will I very too to our both and speculate quality terms good the be commercial material So and quantity now in in

you what enrollment also when a might question, is mention, start last that retrospective just for POM-XXX detail we on analysis? more and might enrollment you STRIDE and when Great target study did see the POM-XXX your and that did I the study, little miss study

let historical doing, that Yes, to and then the Jay me the study let XXX. both comment so those, Jay study the observational Ritu, ask Jay XXX of Barth on we're is add here me

Hi Ritu.

for about We regarding at observational question point planning and are XXX patients prospective treated POM-XXX POM-XXX the a was? this for your ERT study the patients the in study,

and Oh, analysis? might see when we the data

Thanks.

the period We’re of process I we but for of have right don't be results. of we'll quarters yet that months, when date data the in gathering have over specific the next It will the would a next now. say, knowing couple

I half the of share in would think we've externally I it said likely that think this we before. second year

the what's of XXX And patients for plan criteria a or you will…? entry it. the for type Got that STRIDE, for

functional criteria we full And the entry the standard-of-care on criteria. entry are to the an [ph] These a reflective of trained measures, a want patients ERT who treatment standard pretty Yes, been have their that's a for representative sampling with why broad capture into study. meet who that's of they population treatment really heterogeneous LOPD.

Got it. questions. Thanks for taking all the

you. Thank

Thank you.

comes from next W. Robert of line question Mike our And Ulz from Baird. the

line Your open. is

for question. thanks taking the Hey guys,

there? discussions that EMEA you with comment, the sort there respect of that you required Phase in might of are the in approval and of is are FDA your with design? program I'm curious with emerging path terms can trial Pompe difference to conditional also regulators, then the generally Or to or are to for on thinking their ongoing extent notable X respect just an sort finding aligned what accelerated Just be and

ongoing we'll goal purchase just globally, we – on need the any not comment acknowledged Michael of this world, medical that's unmet that the certainly unmet say nature Pompe say of to is in and can to you perspectives, I'm Yes, discussions that questions. extent have have tell on that beyond going the our need uniform the except the to regulators and the

respect to not to potentially accelerated POM-XXX is limiting then or and think earlier those could question maybe we it? the an should to an about it POM-XXX the rate way up just studies, the follow necessarily assuming sort about on of and that Got you Thanks. think path with get

I POM-XXX I is very important. think think, yes

perspectives the understand published controls. the Phase Phase of in understand It of what's nature about any seeing our did remember X/X label control could know, we we And that a X/X the was having the traditional study provide clinical while Phase consistency magnitude, in You the improvements, literature, of what rigorous while experts information X/X have in the not study. think sample patients we further the we're a open study.

provide for see the that further way long very in no providing POM-XXX rate limiting. is drug months. on will us we people data We of next on couple be the do development a any this set At of think a at potential registration over to how for plans so study, potential we purpose standard directed don't period for but of importantly for as if run-in is patients very an care, approval. study or important all earlier of to a the see that So the that term evidence as related that rate to into limiting any ERT

thanks guys. Got Great, it.

Thank you.

Instructions] line [Operator question the from Partners. next of Joseph Schwartz from And Leerink the comes

Your line is now open.

area. relative insight much. undertake provide you the guys. with with presented POM-XXX been how FDA your you Disease treated all patients the for in Happy study that Rare Can to able any feel from strength us this Day into you've the analyses leadership very about and the Thanks you Thanks the you've to that of to ATBXXX?

and on X/X whether response we're collecting it’s the that those experience are or Yes, we that the ERT of you, published that analyses whether Joe. across we that of But information experts the received what that functional our on the experience to underway shared build study study. before measures not. remarkably terms is approximately from going the patients Thank on in again have and and and of have whether study POM-XXX the we history literature or they think cohorts comment ambulatory magnitude ongoing patients not discussions in comment the patients non-ambulatory, they've us Phase on with to different is natural is of XXX or ongoing can and clinical still know been

natural of ongoing. damage And it that all both extraordinary the will set but only of and study together is for we there support an we for, to-date. and measures still that's so biomarkers, And strengthen think measured so muscle data POM-XXX those we're, on glycogen hope with the a history enhanced that, the range

that think need scenarios longer then yes for X some from the to FDA? or just track could you your with you do where two of run file one file you ground outcomes result patients file discussions Okay, can Is middle on an you these likely collaborative no this say either before could you or data help can Phase they us envision maybe there's and instance? could the

of range sure I for Joe. could be think there outcomes a

on approval can path and fastest is patients, and the in for to so we to more provide front job have do for second that in Our to best we'll control quarter. that update do these the everything for our we allow work

is greatest ultimately that any in Okay subset there addressing and that vein quick be is you on possibility follow a indication think settle who FDA you known start a to just Pompe the the then similar or a in all patients or you the I those indication granted just at guess are do need? as on, could patients

our patients. ERT ERT naïve in drug approved and Pompe We to to be all determined, the get non-ambulatory. Still ambulatory goal is for treatment, experience have now experience,

experience we got So we've think date. great to

to the relatively to in extension We that and switch this additional patients additional with will X/X population. populations build be add patients in the added paediatric data such as to able we'll we study, short Phase order further other believe

on approval on think be, would come extent more that. to And of so really speculate to what to certainly but the any I

luck. Good questions. my taking for thanks Okay,

Yes, course of Thank Joe. you.

Thank you. At this questions. time, I'm showing no further

all you Have world living Great, for million thank rare the great diseases. in thank the recognition everybody, with no and for a you further for everybody day. and listening you. questions, XXX if there's people Thank

program a today’s gentlemen, the your have in does conference. you Everyone day. thanks and Ladies great participation for you all conclude may disconnect. and This