BioMarin Pharmaceutical (BMRN) 28 Apr 222022 Q1 Earnings call transcript

Company Profile

Participants

Traci McCarty	Vice President, Investor Relations
J.J. Bienaime	Chairman and Chief Executive Officer
Jeff Ajer	Executive Vice President and Chief Commercial Officer
Hank Fuchs	President, Worldwide Research and Development
Greg Guyer	Executive Vice President and Chief Technical Officer
Brian Mueller	Executive Vice President and Chief Financial Officer
Salveen Richter	Goldman Sachs
Chris Raymond	Piper Sandler
Robyn Karnauskas	Truist
Cory Kasimov	JPMorgan
Paul Matteis	Stifel
Gena Wang	Barclays
Geoff Meacham	Bank of America
Phil Nadeau	Cowen
Matthew Harrison	Morgan Stanley
Joseph Schwartz	SVB Securities
Debjit Chattopadhyay	Guggenheim
Joel Beatty	Baird
Luca Issi	RBC Capital

Call transcript

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Operator

Good day and thank you for standing by. Welcome to the BioMarin First Quarter 2022 Financial Results Conference Call. Hosting the conference call today from BioMarin is Traci McCarty, Group Vice President of Investor Relations. Please go ahead, Traci.

Traci McCarty

Thank you, Ashley. joining for everyone you us today. Thank you, the business remind this Inc. non-confidential about statements forward-looking contains prospects presentation To of Pharmaceutical BioMarin including of performance, products financial expectations and regarding future potential commercial in research therapeutic products areas and development. different BioMarin’s Exchange the and depending actions authorities, and such in report. factors developments Commission, capital, by differ of BioMarin’s in actions BioMarin’s regulatory of the detailed on competitors may XX-K product progress filings Securities XX-Q, Results the and availability future materially programs, of pharmaceutical and with as market those X-K

Technical Bienaime, turn and today and Executive Vice Executive On BioMarin the Chairman Officer; Research CEO, Vice now the call Chief Officer; from and Chief Bienaime. President our Chief Greg President, Executive management Hank Officer. call Ajer, and are Jeff Brian Development; I J.J. President, to J.J. will Worldwide Executive and Chairman Mueller, Officer; Fuchs, over Financial Commercial Chief Guyer, Vice President,

J.J. Bienaime

us you and Traci you, Thank joining good everyone. Thank for today. afternoon

So full profitability, interest guidance for we been as today’s GAAP a from achondroplasia family a the cause to VOXZOGO between treatment and in VOXZOGO of underscored significant the million. seeking transition million positive, underlying begin increase $XXX has growth that in year sustainable addresses and XXXX very XXXX option to by points $XXX

year We previously guidance. XXXX full metrics other our reaffirm also in released financial all guidance included for

growth year-over-year, including the by significant This marketed $XXX generated to revenues up first Kuvan. record representing revenues excluding we were XX%, products million XX% the the BioMarin for So, Actually, BioMarin’s start in marks growth. double-digit return Kuvan. of quarter,

years. over as start the to our next off growth really So hopefully double-digit strong few revenue

ex-U.S. capabilities that of ROCTAVIAN $XX the from States, the ahead markets. with of business as advantageous total in and and potential the were lies we It the for important of of second first This the that $XX a outside the this our and So, approval. prepare is our VOXZOGO footprint the be of million breadth the these our of from global sales U.S. for year, importance significant highlight potential note strength half commercial to ahead emphasizing will the launch a of quarter European results million VOXZOGO. base opportunity United

With robust VOXZOGO forward months. we regulatory to global the and coming financial to the steps outlook plan, of next ROCTAVIAN tracking launch the over look with important

treatment that over genomic we This actually mass. the words not BLA we gland believe findings data vector Phase efficacy evidence ROCTAVIAN integration have currently more a for of results evidence analysis study, patients did years hemophilia safety the part this provides review ROCTAVIAN and was inclusion the June. hemophilia the entire completed of late X from profile news pleased X the of as today the a identify for control seeking on analysis is with we gland the contributed in We share this a say to people our few and We prophylaxis, therapy And Europe great For were ago. also from of plan test our in gene treated salivary in to which news completed supportive Phase in A, salivary that the moment. will Hank AV for field. an the the for superior participants X planned – our observed studies. under marketing option and X in on that seeking resubmission think authorization ROCTAVIAN Phase to in based and application presents

exchange stable the of to the the year, quarter to United XXXX, States forward pipeline largest we the ROCTAVIAN the look GAAP in to way our with Europe are the we set broadest applications remainder progressing forth to-date health and and at also of profitability, authorities on pediatric in achieving goals history. turning opportunity ramping our As the start VOXZOGO the up addressing our with and

momentum beyond and will this commercial financial, XXXX in earnings on and story. growth build as continue transition we to We regulatory to make an the

support. over commercial your business now to for turn I you discuss And the call to the continued Jeff will update. thank So, Jeff?

Jeff Ajer

J.J. an of I in XXXX year first Thank revenues. $XXX in quarter million increased am million pleased with will growth million you, $XX in The $XXX story. very recording XXXX, performance the our $XXX first between and XXXX our to guidance important drove of full component quarter and contributions be million the total VOXZOGO VOXZOGO

estimated to treated XXX Emirates VOXZOGO XX Arab Saudi XX additional estimated this we quarter, launch, Republic, commercial pleased Arabia, March countries States in States XX Czech States, were and were that process of XX the in This specifics XXX children the Slovenia, children within in turning of sales United previously VOXZOGO. and as sales outside first markets the Now, in spread of United across being active with children United and XX. At the as were an of year, that end United in are new April children to markets includes reported the share Italy. VOXZOGO of including the

there the We of been from by has smaller and Upcoming and be combination driven region, pleased continue markets. very a to of the individually growth Germany outside collectively with EMEA uptake in in which we potential year. is to we expect Australia the expected and opportunity EU, this contributions to later expect Japan be significant, The in and begin later revenue year. Japan in approvals in

dynamics pick the prescription launch demand United we up seen to States, have quickly. in Turning

quarter, convert patient from the and prescriptions to been expected. referrals to In able have We pediatric patient as endocronologists starts. genetics we experienced rapidly

our look of ramp our our European are regions we we decision are are toward In aligned summary, the the the see year supportive. as of of uptake BioMarin ROCTAVIAN largely large to during also with was label policies the a that very location. commercial and ahead pace of for pleased with published We this CHMP potential particular opportunities Launching the in expectations. experienced VOXZOGO. more months and United market underscore coming and payer be into ability first teams is should tap criteria in launch States which the or of our clinical trials importance regardless This the for coverage EMEA consistent

now our to XXXX. and of is and Russia relative and our results concentration Egypt, we cause revenues, gratifying during business, the have with Vimizim demand expect some Turning record in orders first last This both from Consistent both XXXX of second year to first such Naglazyme enzyme fulfilled good to QX Arabia. Saudi our will for replacement our experience of large and quarterly last similar half quarter half we year achieved annual to as brands, Brazil, the therapy experience of for brands, XXXX. Turkey, markets our the

we and annual revenues. Naglazyme our for mentioned, reaffirm guidance J.J. As Vimizim

patients XX% therapy. million and XX% first quarter in Brineura, was revenue year-over-year For growth by in of new driven growth $XX starting the

in the in trending what to end the from impact to variety compared impacted the expect market care quarter QX now first the In loss Palynziq we grew of was experienced revenues the with of revenues further revenues million for in of factors. of Since ongoing first the and by in Palynziq, the U.S. to to coverage exclusivity quarter quarter. seasonality PKU in XXXX the the dip October Moving as step this XXXX the XX% dynamic a of first to experienced as in recover Kuvan the the down in XXXX. XXXX. lower limitations guidance to Palynziq as the QX quarter product in of XXXX, full of $XX of health franchise, PKU a as year compared U.S. XXXX, X% fourth Continuing a resulted similar the for of full Kuvan the revenues first were remainder down net U.S., compared And quarter to past in range while contributed clinic we U.S. with in year.

are market full of to the the provided its retain As Based conditions, we as from of Kuvan would we closer a we revenues in be small range market the the able and resulting we to in lower gratified life trend full previously drug, nears share guidance Kuvan expect year expect the end year to end are to XXXX experiencing. molecule cycle, revenues XXXX. on

Lastly, prepared regulatory approvals CHMP launch, year. in readiness near on to team progress. launch the The ROCTAVIAN with to opinion continue is this later assuming well activities and the expected future onboard

exception our just you anticipate with providing longer of value data severe proposition term treatment offer updates with results forward potentially as detailed brands for increased look our that attractive option a with launch. encouraged all to those XXXX, we more for Kuvan and In we are hemophilia the in We described. of at commercial A, and conclusion, demand

Our significantly year. to MBS are contribute revenue expected growth this products to

markets new an now VOXZOGO for your provide XXXX. represents expect R&D call this in to meaningful robust attention. believe continued turn year that VOXZOGO ramp revenue the Hank? We to I demand guidance. in in will increase foundation factor you Thank a to noted Hank including over And also update. growth, a the prescription We today’s for be throughout

Hank Fuchs

Thanks, Jeff you joining us well. all organization today. as had very quarter and a busy R&D thank The for

on Our our the currently regulatory under United license resubmission authority ROCTAVIAN June been biologics Agency the team focused the for health the Medicines and has application interactions European application on review with States. preparations in of

the with later putting collaboration on to and we procedure. for in to satisfy been as potential a information, the have the Thus We enter as scheduling CHMP far, their we degree us have high stages EMA. EMA request the subject of track for enjoyed a of able midyear precise review

with our license in plan – States Needless have the to pleased profile pleased the the review tumor We further associated in of observe are findings as it findings of that include February. the gland participants and as the was in with we completed growth X the one noted as this presentation the as with the were to as to June. date. tumor are for study past part to application application consistent say, of we analysis mentioned, market ongoing these integration with resubmission ROCTAVIAN data has J.J. the cells. optimization safety demonstrated EHA ROCTAVIAN did the ROCTAVIAN in we Phase identified of not biological our profile data As salivary our and of as well Results the integration benign the supports our EMA provide United

As Society Union and forward Hemophilia and the leaders field community, XXXX sharing the World of American in to upcoming in Congress Federation Therapy look the the therapy, scientific Gene the World at Annual we gene of Cell of addition. the of analysis of results

to the this Turning X. May we from his Meeting share Pediatric present genetic coming study Dr. natural conditions results Endocrine Dave Andrew in that pleased VOXZOGO, are will to Society Sunday, at

As R&D we our study genetic statural Day, the shared in different X last conditions. spans

the with forward we other VOXZOGO of conditions learning positively So, achondroplasia. about besides to potential to impact look children

analyze to of our data achondroplasia. they Next randomized we Phase of and the young year. infants in with expect the study in February, medical the unblinded Team them middle since X with VOXZOGO age results the milestone has to a in and update had placebo-controlled VOXZOGO opportunity meeting with children years from double-blind, the at share X the were

candidates Finally, development study X patients using that the or all stage under we to pipeline, advance. angioedema. in X/X is the turning earlier update of for Phase today to A therapy HARMONY the begun dosing hereditary BMN-XXX gene new have continue

this of and of severity that current are preclinical potentially of opportunity patients. suggests for standard therapy our reduce chronic by BMN-XXX We the encouraged burden new some and frequency reducing the eliminate data care. attacks patients, can significantly treatment and HAE for as HAE the

set. and potential data first based X/X the The to to inhibitor muscular of the this quarter Phase development protein first We year subset expect treating with we goal year. arm this which BMN-XXX humans dose of the boys chronic XXX of of half the the fourth restore Duchenne learn process addresses the to on IND file completed of single-ascending are begin analyzing disease, the in for BNN journey Concerning for excited BMN-XXX, the study the about encouraging in the the in the dystrophy, CX-esterase and preclinical we of in Duchenne are results.

this gene half to advance its And AYD, cardiac be in in for Our preclinical efficiency. the to health the by with second enthusiasm formerly build on of to caused case, XXX alpha-X is BMN-XXX people therapy potential candidate. to clinical as BMN-XXX mutations hypertrophic continue areas next the DYNA-XXX, studies our In dramatically our we continue to treatment and living of CX, INDs XXXX. track of referred towards antitrypsin XXX liver myosin for to in improve

in X Lastly, on of to BMN-XXX And results process. February, the clinical will we the be hold. await said gene remove clinical a we required studies PKU multi-quarter that this current we and therapy, as January believe

present quarter. applications, turn organization Brian move pipeline results program our the for So R&D the your work. Look will when forward as in we And financial at of ROCTAVIAN support. over progress the across you our call that update Brian? meetings I’ll to status the now to data available. Thanks advance products we update to earlier-stage medical keeping apprised upcoming to

Brian Mueller

release Please results top Thank details commercial line XXXX. results refer financial and of the business, quarter. Jeff Hank. Since you, on to the full will expenses, for first summarizing other results on updates I many today’s this line touched primarily the focus financial from bottom press quarter key our operating on

results all Form upcoming usual, are to be which As will in our file few over track available we next on the XX-Q, days.

is highlighted BioMarin. February, an in we for XXXX exciting As year believe we that

of in business, a company strong the its GAAP profitability driven by contribution year. anticipates transitioning our from growth launch base continued As VOXZOGO to sustainable the plus significant

second XXXX on first XX% in company’s revenues XXXX, half of upon that that the markets, excluding important expect a of elaborate It’s our first contribute the even will for of goals will globally, Based ordering XXXX. we these drove ‘XX. to select We patients the that note on while provided quarter in just plan though we income of to in the mechanics XXXX of compared total XXXX. the of up observe on and comment even to our that to Naglazyme expect in second expect mean year the of nicely be to out over revenue trends of new daily XXXX, a on the Kuvan total of we full us to today. to of one results metered achieve we uptake orders our To to VOXZOGO, the weighted VOXZOGO between half slightly the Total the to our revenues of Also and for the VOXZOGO quarter brands growth first year, other GAAP full be are important early first are by as the XXXX. non-GAAP Jeff, for sets pleased roughly patient the Vimizim and while dosing increased revenue. be year the less first half quarter BioMarin expectations pleased therapy patterns and tracking remainder growth balanced be full which patients full the add continue will comment year portion to year for new based

SG&A million in to for million commercial last year since and global costs. year ROCTAVIAN’s in ROCTAVIAN VOXZOGO in R&D Moving period of approvals as the the XXXX first $XXX were for for the U.S. the SG&A year as expectations. line R&D to as last $XXX European of quarter half increase launch and the programs. the efforts on our our operating with quarter quarter were the first reflecting expenses $XXX expenses million, both XXXX, slight and launch increased commercial increased reflects same first first expense R&D development for fell early-stage the of a as compared to preparation XXXX, compared second well expenses efforts and

first for Moving quarter. to the line bottom results

shared QX XXXX, first The of sold the the United of we was during asset a sale primary gain driver in and of million. on our recognized States. the review approval income in the of of a $XXX.X was with priority voucher GAAP as February, transaction As of received net quarter VOXZOGO non-financial we operations on statement the

non-GAAP we QX from business a start and sale. PRV not profitability $XXX sale income million note to respect million related $XXX of of PRV upon for income, the from year the income relatively plan earnings $XXX gain XXXX the our of and XXXX dependent of gain and the the benefit on will year. non-GAAP XXXX excludes strong With to million in after-tax are profitability million the first GAAP While quarter that expectation to non-GAAP the to our $XXX the between income represents flat was of and non-GAAP this expectations sale

XXXX. flat total we cash to to Turning quarter the year-end first billion, investments, mostly $X.X with and XXXX ended

cash proceeds the total our differences sale QX PRV, While experienced XXXX. also working from capital during timing quarterly we with of increased the

the we Ukraine As on presence do briefly we year, cash footprint provided region. and flows commercial in XXXX expect said positive operating for this our full we when given global crisis the guidance,

Russia of approximately what X% X% in we be As the we to And we BioMarin’s to know shared, and today, based XXXX. previously total that expect markets on represent consistent revenue. Ukraine

for of which working nature disruptions no the treatment essential product to and our patients available, to Ukrainian the Given the serve for patients. underlying treats region are which conditions pharmaceutical are treatments minimize we alternative continue our in

fact, fulfilled business. have are growth supply to sustainable profitability to significant start, achieve launch In we we great first transition with and on a solid revenues, Russia. already base global in and closing, by In Ukraine off portion of quarter and VOXZOGO our of to is strong the XXXX expected to XXXX driven a record track a the

pipeline product same time continued your generating revenue, open up Our for will plan further continued and we call decade. both into to eye to your with towards you is support growth, the innovative flow growth sustainably an being approvals And profit and realized cash the now this operating increasing questions. while attention. Operator? Thank at

Operator

[Operator Sachs. Salveen from Instructions] line Your Richter question Goldman first of the comes of

Your now line open. is

Salveen Richter

Good afternoon.

me, us Thank then GAAP in VOXZOGO in endocrinologists. turning outreach Two on pediatric on how the progressing you the for and congratulations for out uptake how And questions anything discussions ROCTAVIAN, U.S., is in regulatory you. can U.S.? provide are First, from profitable. playing

Jeff Ajer

call example, seeing we our physician take really from well for specialties, noted, connections It establishing [indiscernible] we I question. outreach geneticists active, with new point would Hi, shutdown, from in Yes, a a emerging Salveen. doing Germany. endocrinologists. is we as this the the pediatric call I are where are are point the expect. as new are other from mainly as teams first coming with in will and to we markets and of but prescriptions in United we mix are States and And

on notable that’s describe So I going would kind with right of to we say expected. surprising it’s nothing track different than or

Hank Fuchs

Drug on discussions, and connection conversations gland pretty Agency and to recently have genomic our But which to we resubmit. and the including have Administration. Drug of the of they regulatory them have information the the completed plan Medicines already since between further been do And the advance want fact us pre-submission in we again, had provide European In tumor, even Food the the CRL will questions we to regard have feel scheduled in bulk that interaction good bit information do with and about about analysis quite have Food with them salivary discussions dialogue Administration also of with U.S., back favorable. when the we is a is they to provide we and the resubmission, to the remaining

pretty raised sufficiency about for We good in also satisfying of the the were feel in [indiscernible] concerns the we that the hand that letter. data have

feeling markets. pretty So, in both situation about good the regulatory

Salveen Richter

Thank Got it. you.

Operator

comes of question Chris Raymond next Your from Piper Sandler.

Chris Raymond

the taking for thanks question. Hey,

on the there. Just just dynamic on maybe questions a of VOXZOGO, couple

high then quarter. on to of or sort the the seems doing some number and of the third U.S. I but patients gave maybe last this the a just so parity there revenue indicative also, in just guys the I be the you number of and then dynamic? quarter more was know patient a than math relatively a early, think it’s for revenue or with of is a sort And pricing third of add about little less patients. is that

no where – just you a there I mention ask dynamic? I of of program anything in And you. Thank heard no there add is terms sits? the BMN-XXX. sort pipeline can that question, you of mention can give Can Just think of sense a I us if of pricing

Jeff Ajer

on I’ll the off Chris, question. VOXZOGO start

And did a we enough confirming reporting that pharmacy also was huge about event. in the kind say, were of but to and and mentioned a which have our That’s said, balance a we on no inventory that as specialty pretty pharmacy from there the U.S. the network. resting our would you in I is specialty by States would United late stocking, in you there a essentially the demand. one-time – the a inventory SPs so buying United QX, revenues specialty was Not conclude numbers I event. of further of enough that of March, third that number, network that States. Early reorders ex patient to seeing pharmacy consistent ordering we in ties which our one-time some we’re a were up the U.S., and So that down that stated closely basis

bump towards you that little a discussion. be and pricing our QX along And a of could followed So the tilted for U.S. bit in

have particularly price, in a As robust the usual, U.S. we

pretty And cover a that other so far, your Does we’re tight price in managing for corridor VOXZOGO question? markets.

J.J. Bienaime

market I so two of add, may basically, If in that in is is and bit of ahead numbers very mean, is of the and always the well see spending revenues a difference the so the launch going commensurate that. But U.S. actually in little And October the different U.S. started the we outside size. the Europe I the a really sides have was you to largest actually, are the because said in aspects, and Atlantic the both the opportunity January. selling little started Germany shares, what’s we confirming year, that last U.S. are in happening

the again, the I which anxieties launching read product we of in is U.S., be that And that, I’ve in a is some you launching here Europe issues can first and show news U.S., before Europe for in good we the do happen product about likely think were there ahead analyst time that. the that’s So first a very report to successful updates. some

pretty good. answer want Hank, this to all the... you So is

Hank Fuchs

findings are that with results now. hopeful you the additional we But Chris, were Yes, to of studies, remove we several updates going to to which game from for we’ve to this unfortunately, some to that any requirement quicker. We that been the got some studies, complete agency that the be conduct had preclinical on interactions whole agency. based we the probably that quarters the have additional with reported be is on those have plan would able previously the pertain to studies until early won’t

looking they on updates unfortunately, say, be for I’d near-term So will XXX.

Chris Raymond

Great. Thanks, guys.

Operator

comes Karnauskas Your next Truist. from of question Robyn

Robyn Karnauskas

my taking for question. Thanks guys. Hi,

a quick know just salivary you’re it the that on I docket, go gland it So discussion, into U.S. adding the discussion could that guess you’re we not the in for data. EMA I Is one. given additional July? possible for April And like is for longer on looks August? could think EMEA. do middle the of second July, year, could for then same little you the the if question a take filing like And be this U.S., that VOXZOGO.

So, of you addition patients same And did have it a then and process comment compliance. you maybe lot, you is more that can comment – Do like on – seems it’s but this the quarter. do maybe think can number last to patients Or a you of of on bolus consistent? in upfront? you expect you the this be

Given early – daily compliance with and early, subcu the it’s but dosing. Thanks.

Hank Fuchs

the in a them in label, then information and June. decent I’ll EMA commitments have they will on Medicines question, that that. also that last This decision risk part is information line a – review able questions the we’re the based We to of to the your that Agency. a has subscriber of But European and chunk plans, information the decent terms management the as asked of to the make things question in we like and amount it’s leg a of feeling said time is will provide Hank. review pretty of all be to the take enable EMA first shot at optimistic we

with July, to we into lined to were could So drip them to come but and yes, enable them opinion. it the positive up getting be

the right will they question appreciate team the objective course, And my of water of carrying are which same regard the that. the at ask all that the on is Of to the U.S., application that’s certainly to in time. of to nature lot you’re European the a question,

finalizing U.S. the application. We’re

review track. on We But and So an that facilitate will sure, will and June, take that to U.S. longer. a Yes, want really application submit the make effective sure that we thing, that’s done in hopeful the target, with are really again, we as may execution remaining things an by little is that execution.

J.J. Bienaime

your the bolus elaborate answered of I growth compliance. but be, believe whether it on just is And Jeff it’s to on mean, I’ll whether I going patients can VOXZOGO, let question sustainable. the will be or question under of

compliance. we’ve patient So good lost the patient. patients only I think so one Jeff? we have saw, one we that Is All far. correct? lost extremely

Jeff Ajer

for Robyn, of which the one in the process. question. we’re in patient in launch a track world the United trajectory It’s able is the actually numbers sign positive place to Thanks, the States, that

approved So getting a of holding in ago the then enrolled patients the ability quarter pretty patients fact work was fulfilled. of steady of through indicative products that those process is out them to it steady compared also where having both a is stream to treatment, new our for being get get and rapidly shipped and prescription

very at high far. this compliance number as have drop so compliance, figure, noted small so We drop-off small – supportive a compliance other number I of rate have which don’t J.J. a to a is stage, of off a that estimate positive far. I the view than related So quantitative don’t of

patients we’re we’re that Europe both proactive this for kind very about And thing feedback and injection. with training daily their of being the we’ve is other super families and The in United doing States. gotten

compliance really been I for think forward. routine. that’s going helpful giving So for families, and out confidence well bodes daily Thanks. injection the that to the begin them go And

J.J. Bienaime

it’s I right. mean

the they We this in to and on because be that said have the very to that treated first basically in want with be VOXZOGO. they good family roll they visibility want is kids signify kind of as U.S. Jeff

that this this in good in to So a average I basically and how patients long And – when for the U.S. treated time think and in we on that we have when to wanting start those you be we is it visibility terms shipping signed mention the patient is have slowing of and accretion at the takes rate, between want new up down. is it Jeff,

Jeff Ajer

Yes.

So, so far, it’s pretty rapid.

Our means. around average patient a some complete prescription the of to to from first enrollment is that prescription that with days shipment variability XX with

Robyn Karnauskas

Great. Thank you.

Operator

JPMorgan. Your next Kasimov question comes from of Cory

Cory Kasimov

taking for my as Hank. well. questions. Thanks guys. afternoon, good probably for Both for Hi, me Two

First, to this pre-BLA with follow-up just Is situation. ROCTAVIAN curious there a issues be if on that U.S. specific is for standard anything I needs are guess hasn’t been already? if resubmission the just or addressed there the dealt in that a then to wondering, operating procedure of a Thank could in or just weekend be second you them? you Hank, compelling thing, if when an set What still this And investigator’s I the would stature short was speak VOXZOGO. the ahead discuss. stage your with for update view view you. meeting the

Hank Fuchs

FDA revenue with ROCTAVIAN interaction more than particularly think new the of necessarily the is information. pre-submission I No, Yes. SOP

are the to sure these think What looking is to make do to likely that eye important – is clear view information for for. be I are all that – of to they have the the that we we a what’s review. be resolved during going issues are agency us stance

have We all data. these

So the it’s in really content of looking it’s sure form just a matter what of of they for. making are

FGFR by But be the in SOP children of types improvement themselves is, be biology itself that believes a I So far mutation. measured he he’s that’s patients mutations case. to got positive think is lot an underlying side, he’s to [indiscernible] have range Dover will I in are more mutations across that in mutations, ass the who achondroplasia as the course, why that of hypothesis. think this the of that the of accrued he’s to think can different that I a amalgam that have from as that different with And for there of data six on be reminder stature presentation, sort as the expectation a indicated

across a great the the what or So good more think the in that different seretide. included. to we’ve And responsive AGB similar And I of I would achondroplasia, on even mutation-specific seen it might – be focus in be the baseline similar outcome than be are mutations some would better that he’s from change types outcome. to a mutations might

stage the think that’s set So for data. I the to

Cory Kasimov

Great. Thank you.

Operator

question from Stifel. at next Your Paul comes Matteis

J.J. Bienaime

hear. Sorry, we couldn’t

Paul Matteis

my talk said questions. she Matteis, Paul to taking Stifel. think I Good Thanks you. to for

you patients anything are natural VOXZOGO you benefiting. whether question determine commercial into do physicians of Obviously, patient are – physicians hypotheticals. I at growth the not but can you you every one on is Just look will seeing do guess different, think kind get or history, to side. or

the sort mind the reasons, So you efficacy just And what you so to you. comment clarifying? with on this you’ve seen drug. Thank if any far, how maybe then wouldn’t verify extent are of discontinuations

Jeff Ajer

Thanks your question, Paul. for

So certainly newly we in expect but not benefit, progress – that’s expect is patients. be There of tracking of to something physicians that would terms we – guide that we common would prescribers velocity along specifically achondroplasia, a the following I their certainly guidance helpful in published think kind guidance the denominator do on growth management and of be would that providing a of is which to in those expected. height lowest be the could to

of U.S. back And of for on publication enough. which future benefit the are renewal the to of for requiring management evidence guidelines. prescriptions, insurers Some reasonable seems example,

establish and with kids opportunity to to you’ve system, to it’s going there think heard be for for up with As use part scheme. me say that our I patients. in achondroplasia as VOXZOGO not I and medical for a was good home, and their kids home here to really, the but overall And no U.S. home the be part including, of that’s of is in great medical of management no and the before, particularly medical to families. for the limited a think going achondroplasia VOXZOGO achondroplasia And BioMarin

don’t have discontinuation, one. that terms stated have one have J.J. data, we we a as enough really I and discontinuation reason think of for mentioned, I In

Paul Matteis

ahead. sorry, go one Okay, – thanks. And maybe just

J.J. Bienaime

that or efficacy, to of our years the patient European X reactions now. for, young the of that’s But we advertising. – patients way my a is to tested a healthcare And using take-on – close be been this the your from picture If the and have X be for an with you go a inoperative other we [indiscernible] doctors is had it, campaign, to obviously we with patients. a have is families to professionals patients I and questions. who anecdote she scientific should really do treated back on VOXZOGO that way over using We should answer advertising actually we just chondroplastic tracking and she when but not think,

Paul Matteis

the approval? Thanks, to J. J. quick on path thing. again. last, you, one Any full And update Thank just

J.J. Bienaime

from the overall package good as trial. that Not is core to excellent. pivotal news program going the generate you very in particular, patients clinical the just be is we’re And – to that’s the the the compliance data these follow-up heard, going and you is

to So to date. able be we a expect do a we’ve fashion I specific don’t to provide that filing in know guided submission that

Paul Matteis

again. Thanks, Understood.

Operator

comes Gena next from Wang question Barclays. Your of

Gena Wang

questions. I sets two you Thank of have taking questions. my for

ROCTAVIAN salivary first one regarding is mass analysis. the gland the integration So,

up for So similar requires you related that any sites of event, ask analysis? for unwanted quarters but did your question you still integration several program, the your work. also not that And data a saw see but in TTU showed this show a XXX analysis tumor-initiating FDA pattern,

you makes have confident won’t here that what on to similar quickly ROCTAVIAN. VOXZOGO situation And happen I question. So

is negotiating what countries? here? listing since the price different the wondering, the be France, $XXX,XXX. have price lower, expect will like for of revenue U.S. Just you But you ex range would for future book now prices And how you

Hank Fuchs

the has described there was pattern one the about been in adjacent relatively tissue. integration. case Gena, in And And is noteworthy block between propensity vectors previously think recombinant think integration. regulatory tumor wild-type has And the perspective. compare that regards low to and AAV the tissue particularly as the foundational of Hi, a for the the so integration I have of of of pattern as of been pattern integration, really the the I then healthy to nothing

referred AAV guidance FDA You is an having vectors low there and remember to might document for this on was a integration. they propensity

frequencies far patterns, to in it terms is and the what that binding I preferential think about size is integration wild-type not similar particular saving of great vector of appears that similarly so is we’ve of the that to of ROCTAVIAN relevance. distribution say in one the found spots integration things hot

question And been to imagine so for for how And that requirements undershot the to as read be they going the XXX. XXX does XXX of for are does be similar it the they the effectively appear meet was XXX, not why that part evaluation? how have – next on your as there

of product unique answer talked its individual. suspicious satisfaction the signal only studies I about, to health XXX tumor, cleared of is arisen program authority been which has there in think the for is There sort that’s have is state. the in that the adequate vector because the particularly XXX safety in nothing just was what short we the the this findings

preclinical very, studied the think is I similar us to for not is with species. even Octavia the that with period study time, construction of so a a and similarly and similar oncogenic longer And having whose ROCTAVIAN or neither ROCTAVIAN very not vector that itself seen vector preclinical

ROCTAVIAN no for all namely oncogenic I So of of evaluation signal think vector studies in particular take overall together, things quite the these preclinical in humans, risk-benefit potential bode taken the has existed. and together well confirming that

J.J. Bienaime

ours differently is FDA clinical hold, the hold. products seems two Clearly, ROCTAVIAN we respected on is not some as although clinical

issue We these are see. rolling as we studies

Hank Fuchs

is that And enrolling. this the to reacting More is it’s decision. also indication-specific therefore, BMN-XXX, I either a think agency as vector-specific

J.J. Bienaime

Brian, we Jeff then And you a VOXZOGO of the recognition, on revenue explain? in and start revenue question you with range and will price had Europe

Jeff Ajer

Okay.

expectation that price realizable call, patient. we’re price the of guided Regarding to anchoring approval United per net or the WAC of to, range, Gena, price WAC our States got price kind we and gross the we’ve on to our the in prices

it’s year introduction I kind take the about as from in of prices you to list full and think got solid Germany We’ve And expectation price, to for product negotiated France. that’s note, our a pretty reimbursement. a going estimate. to get is that

U.S. will for And our basis. in pretty let establishing happen consistent And patient German sales markets meantime, I’ll where the cover later So right year. named corridor and Brian are tight with the we’re pricing in pricing. the French this that a discount

Brian Mueller

into nothing Gena. to net about are experience and launches the as speak well our net to get Thanks our gross prior Jeff. our similar respect for to expectations materially This will unique with gross products the be question, price record our is those cases, Thanks, a you could estimates VOXZOGO to get overall But initial Yes. start is not that and to Brian. as other to we’re price of get There the if dynamics. GAAP and in negotiated each do European clawback lower to required that reserves, an you when those some be make of under final you country-by-country will. to to then with there but

you’re revenues seeing the what net-net revenue. be So our in would reported

Gena Wang

Thank you. Very Great. helpful.

Operator

of comes of next Geoff Meacham question Your America. from Bank

Geoff Meacham

trying launch in terms or you I maybe it’s be the And different who weren’t the it’s Hey, the some the as the has Thank parsing analysis. on from just couple theme in patient was awareness. you but themes the that there along CHMP. could the was, you. then mid-XXXX guys, language for just of asking are VOXZOGO, for I’m and early look question. similar I in is on know XQ in – tumor working to but get or normal there just a launch? review Is everyone if much the thanks an in think and to what that commercially, there U.S. at a had clock sense bit, know flows a when fluctuation been impact when ROCTAVIAN early, a clinical I kind so fact, the starts look the new or same of to shift patient Europe studies this from I to

Hank Fuchs

is earlier, I that the piece maybe information is the there June, about was of on being concrete Geoff, minor to shift talking shifting the no the underneath summer.

news is that time they receive want of not when – was is But procedure And sort just up accelerated we’ve even only even beginning line you know we’ve how to met lines new I seen that’s and the been at the information. off as their never the procedure think the anticipated. of here just with assessment.

towards giving pretty caution a of we’re big what’s anything, do in control here not coming. the projecting abundant end a so tail obviously teams being data as we’re little of are in And think as because of a that they I review. of as fact our the them entirely terms just the slug much They result

questions procedure why summer, they benefit we done So that on going stores. they pigment. terms also to the that information an And the opinion it lead can have to with us. follow their but positive by going the that in that they the that’s we in believe to confident staying those means opinion those their of the we’re and risk but we has addresses questions, come, issued is thing have it on through happens exactly And take have important believe got most based when that to are we’re like the after that a

we’re So what that’s to support. working

J.J. Bienaime

business. VOXZOGO patients European

Jeff Ajer

Yes.

seeing also important orthopedics So approval, full up here. so themes and about Diversity, essentially there, specialty of that say, were age we way. genetics operating kind but which kind that diversity Geoff, I a we’re therapy, Germany, price we’re of of mainly of from behaves. one with in common say would though seeing experience points away an approach, themes here starting pediatric showing pediatricians of price and of market even And what we diversity final a I group prescriber is would some diversity in example, negotiated of of kind how under pediatric also endocrinologists, reimbursement the haven’t diversity

very a access in uptake And contrast, in France, structured there. so protocol pretty operating we’re rapid we’re seeing expanded currently under

fourth, and treated, in or been to name we has patients the say down that. sales working physicians kind there get working more else. And in starting I’ve of a and that off before, want with are of the window are noted under to as opportunity kind seen to And on older do their a that approach have treatment structured that to is That’s I France, a theme the from kids. in the patients, our of clinics. anywhere age approvals patients fifth named it’s eighth the something initially sales the take market hard of cases, the not patient full started third, they and older for actually are the as advantage of there would So there approved that, network or patients smaller one relatively some we in build getting logic patients really small we getting couple and the

actually So, a we lot of have diversity experienced.

Geoff Meacham

it. Got

Okay. guys. Thanks

Operator

Nadeau The we with Phil next Cowen. have question,

Phil Nadeau

afternoon. Good

Let add me my productive on a quarter. congratulations

couple us ROCTAVIAN. and from VOXZOGO a Just on of follow-ups

wasn’t towards with is to in call, there a expert patients is Hank, are on the availability flux you that second, identified first there launch the in what centers. a are one the I you Are ROCTAVIAN. briefly, review. that on think approval or likely Jeff reporting of And Do patients on review VOXZOGO, treated, patients VOXZOGO? I were your lot the expert expectations about centers ROCTAVIAN challenges FDA one just First, Thanks. to U.S. centers of know of inquiring of the then AdCom? general expert think resubmission? AdCom an of the hold for being the the the

Jeff Ajer

I will States. So, start in the on VOXZOGO United experience the

create term, in I I a patients medical network You pediatric ago, right. which physician, referral our of to that’s genetics approval on kind get of to a achondroplasia the were have home either a a to that out for longer pretty time. was of that – And call, moments are rate clinic to and opportunity a or treatment random home endocrinologists. big noted get that When earlier, and coming the task got for an we establish is in-process referrals referred we over steady for off we indicating not the good interested a patients patients them an is just that’s United didn’t that, achondroplasia, of and States in, have in said, achondroplasia. down and slowing a that’s I bolus also signal a mentioned group And

patients and coming of been from lot were However, able on patients up, U.S. lot A from and endocrinologists. underneath and in had being that that, a to patients that expert network random get our that clinics, achondroplasia couple interest referrals I have seen we last dysplasia months, they are of channel. so think to established you referral start by driving early skeletal that the the pediatric are centers lined of physicians genetics and from right. And early the in

that’s think of So, going that’s market. driver longer picking up now. And term the the growth I for to U.S. be

Hank Fuchs

One and they is called really I would were the a mean want more feels they lot when there to what profile purposes. comes profile tell an they good, the won’t review. decide is would it and it. two AdCom that good. they want the not the personally, I It to for tell use know efficacy like so a of say, asking to you AdCom safety agency you information they think headcount, show info something the around others since when I dialogue of then what I could to like want it that issue they generally rally and want something have that decision until they is if be kill because are that something like make add troops in to to agency The meaningfully and approve to the to AdCom the is don’t

would to what modus be for to, you so killing And the point it.

about we they therapy that they they So, I they come and a of regulate going like ROCTAVIAN could use what establish robust are hope I hope But guess I will... all gene to package didn’t fairly doing serious how is to because review. be products standards say, for

Phil Nadeau

That’s helpful. you. Thank

Operator

question next is Your from Matthew Morgan Stanley. Harrison of

Matthew Harrison

that I inventory the about or some how dynamics that want each things. done been Great. engagement helped or Good Could commentary there. just willing VOXZOGO you have secondly, up initial much and this much sense just afternoon. sort with how had Thanks then pharmacy. in give to to so and around One, as on for for are the the can of you And us of if any, far taking or just around some questions. give countries think maybe talked two are I just much preparing of helped? work you Thanks. ROCTAVIAN you Europe, price quantify at how us sense be engagement has Jeff, you some about specialty follow you quarter

Jeff Ajer

for Okay. the question. Matt Thanks

inventory, VOXZOGO and So, resting back from revenue on mix judge the pharmacy of in kind and ex-U.S. specialty the of could U.S. U.S. you

BioMarin And I revenue that were was to said March, also so, not resting that that be by specialty seeing inventory. $X drawing U.S. that indicated is orders we well, of were million down guiding the pharmacies

specialty know don’t good really, hold dynamics a to like also have And pharmacies to lot for really We they got don’t have because of inventory. reorder we them.

to inventory Europe, have launches VOXZOGO guide one ROCTAVIAN teams leveraging launches. experience opportunity then in of respect that So, are a market and And launch from that what is in United past States. company gotten we the the is several is and doing in to particular, resting million the have in the on I the all experienced with opportunity where that a an know that they there to doing, Europe that you things have in EU, capitalize couple successful large dollars think we I might of of are can market the with experienced we validating

hemophilia As business. with ROCTAVIAN in prepare in the seeding specialized formed teams United States, we significant with teams to for including experience our people the

get essentially ready rapid to particularly places would are basis. uptake we Italy past you and places experience expect first we in that a in We sales where in be where expecting And first might and France markets the relatively patient and in, revenues. are from like the current Germany go on

been Yes, have we So, research. we those got in price people have places. doing

VOXZOGO. for practice, followed active with example, with working advisors an access program that have Europe. We We market in

doing are ROCTAVIAN. opinion for get ready same I say positive really here. excited thing we We if with this are would are EC approval we CHMP it about an the launch and we and a

J.J. Bienaime

me Let a add things. few

and launch, have outcome-based around we driven or reimbursement is be close lot U.S. we in will we major by Fed are least a a at at So, agreements the research. There million. price price of because few interests years. Germany, $X therapy not either of about available also payers being maybe and And prophylactic are to of data well great go patients back a assuring do after now or low-single few to we the them, those ROCTAVIAN that very VIII making respond have digits responding majority Factor done

to So, them. interested very we success considering payers. that they they something species offering are very products know costing launch are that’s and they the basically understand well how guarantee can that by maximize a And at the in of the much

So, plan. that’s kind of the

Operator

Securities. with question comes SVB Schwartz Your next from Joseph

Joseph Schwartz

of Hi. been answered. A questions lot my have

ask BMN-XXX. So, your it’s over the since I IND filed about been some was mid-stage will for I pipeline. a guess things year

program. I mantra SAP maps. to the I Do on think was signal completed you be if to And us you could see this see you a we work you see portion? some will when analyzing it wondering the than an advancing healthy volunteers? are can these expect Is and MAD data the update to possible from give are there? So,

Hank Fuchs

of oftentimes part the early Hi, are Joe. the of All of good small The stages twistiest sort questions. the is molecule development road.

that have single can in which ascending the share the think is and portion I all we that. of is analyzing the with we you you is what So, results I put completed now question, study of are your dose

a I its genetic excitement or lot namely And be that key we oxalate of there corners recurrent who there stone renal enablement, XXX of molecular regulating that’s think is disease. is people the understand pathway excretion. chronic to will oxalate for a have excretion

way really of it be that, I should tuned as say, for tristiase particular if So, a the data we the But excretion, it figured exciting tap should this patients. should or to should oxalate, development it open in close out we gather with program. stay on be be,

Joseph Schwartz

Okay. Thanks.

Operator

Guggenheim. Your Debjit with Chattopadhyay is from next question

Debjit Chattopadhyay

for Hey. Thanks letting in.

launch the far, to are on long-acting in the zero Thanks age advance patients apnea number on X-year-old And So, much. limit of noted the into are two, so clinic. who in you prescribed. signal study? And so just younger getting upper finally, the VOXZOGO, sleep subjects any on version the currently the to clarity commercial planning

Jeff Ajer

Debjit. age question with will the Hi range. on start I the

quite younger enrolled in of including lot teenagers we data it seen earlier, have noted here we diversity well to the present being for kids. a have age as range, As measure as

that age, a their we what plates that will ceiling is benefit. kids have don’t close So, know growth have but not

was second limit to plus expect or to your or or So, And upper minus. would X-year-old. minus question XX-years-old something to zero the think related plus be we that I

Hank Fuchs

Yes.

overall picture that package So, that the launched, about of the first were possible to we to where in during the application data X-year-old seminal by zero have that on them data is set we talked Europeans in that. the we compelled biggest provided Europe,

in FDA a about to results population. that States, and I a X-year-old can bit the XXX, we sell we the label the we United we In with product the to have of wanted think the little talked going – that. information inform and have X-year-old are more

need about were the trends Food We step, pursue labeling the provide that come at And of world. decision. treatment health different rest to positive throughout may going they are think Administration conversations is believe support, information I the data families to with think giving working age treatment children pattern X stores decision geographies a with and achondroplasia option next similar announced the are emerging the come and for with points we data. children, different And the Drug to have therefore, these of them observed. time who And in that to based I under years young have requirements. to on of We that

say overall addressing... from plans for as would updates for tuned plans regulatory I us So, stay as on further both well

J.J. Bienaime

filing in is late are the for zero Japan commit for our to big of review birth we in for that stage any patients four. And application the from

– you be and want So, data when also do the to say few so a presented. is about will

Hank Fuchs

Yes.

also acting, the long-acting, far, of asked efficacy a most not I we an on thought think safety think then event. data or call much you it we I children, said long summer And of the much about think not so presented, advantage further. So, about in as I

have It X question that. We all pertains are the about is all an the as talked who behind interesting numbers under it to children years.

in You Phase who enrolls X might has their under study know X years. that Ascendis patients the are

again, about key about a far So, overall us. how But growth. there of data that is some effect could behind the on long-acting be reminder

Ascendis development study. this pathway. small relatively in terms really a of And X Phase is is

iterate of in the our nothing this global further and on children to we to option So, a there both brand. I else is as think the offerings well essence strength room time because young continue VOXZOGO as is have building to basis for product some offer

Debjit Chattopadhyay

Thank you. Good luck.

Operator

Joel is next Baird. Beatty with question from Your

Joel Beatty

the questions. taking for Thanks

The is of learned starting first level families? of study XXX also year? for awareness this from their patients the for initial What’s one will for drug VOXZOGO. DMD, be the and with And then among the later achondroplasia what

Hank Fuchs

awareness. the answer researching launch yet one would start to market at on haven’t that gone a question. question is will something that estimate typically quantitative That’s following we the and of level be with I we out research, that market to Maybe get

an coming We work would of we high patients will, say see have until estimate. will at we we the some point treatment. for do wait Qualitatively, that and pretty level on to I year, be based during that have to it the in to looks

J.J. Bienaime

which to this essential is And question by the that high compound said, based of what less the similar that between course, what that shortened be of or really concentrations between biology that, – assuming range regards as treatment Becker XX%, the of to and far as be from would goal to of a review the dystrophy that to plan of increase year. subject of the what initial should drives an can always I with the profile of produce And the have our achieve tissue tissue quickly we now. dystrophin we relative IND. the will its our is DMD learn dose, dystrophy, the boys is person nature with that’s protein, And in end But XXX the to you into concentrations, compound pretty the as to called ambulatory on what’s patients study, achieve. condition as XX% patients can we the would we I should skipping severe level regulatory that’s think safety and of because that that dystrophin muscular relationship plan, we delivered meaning have muscle of XX% being XX% the and XXX the that use the so able a chemistry developed, of believe we get

give meaningful that the started. as to demonstrating itself getting of muscle. timeline improvement available So, in concept specifically a safe proven Can’t will gear as towards on just be will as quickly one dystrophin you possible, we quantity initial selling protein are

Joel Beatty

you. Thank

Operator

Issi RBC Your next with question is from Luca Capital.

Luca Issi

know their you cell mentioned integrations past taking that but Well, you case. frequency carcinoma questions. know cause the their paracellular integration Great. piece. on cancer, I the is Thanks have one rolling of share actually as on the potential for the gland are so salivary evidence much X.XXX% surgical showing of of ValRox. in for my AAV uniQure you can observed I Quick AAV out the

number wondering that asked or tonsil maybe So, also hemophilia And how B the just your program. reported cancer in by to number. compares I related, think

strategy you then thinking your So, pricing it between ValRox, answer to get for so and and should that feel are And what’s latest much. wondering how the the on the us remind approved, program? assuming thinking EU, sense? still does Thanks U.S. dichotomize the about make free read-through on

Jeff Ajer

to numbers, with coming far we – that the WFH as and around is is integration. The ASGCT. way both it I seen described think been what consistent propensity think specifically regards as corner coming what’s low about that I are have the to presentations so previously at for

take that’s So view the those into qualitatively, your anyway, frame presentations. of

particularly regards seen really to-date tumors, that going to there I are of a occurrence cancer. mean that’s that think I other over And types. we As the of doesn’t fact that, just there do getting have mean clustering I in suggest be been I patient what think older time, tumor has developing life. of

consider at XXX kind agency really to what sort has one are and with question it’s learned enrolling raised. kind the only also our of time. have of to that XXX which talking seven is is proceeding of in we about of think vector three drains in is say, I we That’s this trials out earlier, clinical a XXX

moving agency of think instead is I audits each everything the on in being. clinical face of sort sort for So, situation each factor taking the to of time

hepatitis have think running about It’s or today what’s around to genome run I hepatitis from. about along that about AAV that we been fundamentally all reaffirming the about C relatively particular integration relatively regards are is the people low AAV. reasonably encouraging the B, AAV, contain for talking risk that risk spots cancer density not cancer around the with talking observed and people data known systemic low of – not specificity integration, in pretty events are in talking are what as for we

really we that corroboration integration of J.J, finding. are about is propensity talking a you low that think do powerful I want So, to… today

J.J. Bienaime

covered in already U.S. the previous in the pricing We Europe. question, or

to want So, need word I about we over again. the that But don’t you Jeff? U.S. a see think Pricing, to go

Jeff Ajer

Yes.

that years a dominant million say up. to probably So, of $X WACC, first ICER what was couple you kind of review ROCTAVIAN showed worked at presumed probably the less But more that’s that J.J. $X.X $X million a heard not than of not than million. ago range the publicly a be price choice at

so the the are administration effect of risk concerned over far very, of following the non-performance and administration the The to and not crucial when of have of capture not addition financially following we study average. agreement have about price. other the to low Payers patients and non-performance would Payers, those outcomes-based in suggest a about announce the ability durability that’s well one that think a including ranges durability an out study time. to do ability get seen launch. and very risk capture But bleed, data XX have approval risk have will just mentioned to X.Xx of our is over infuse pricing to we week the we encouraging premium J.J. GEN-X our and solid both data value effect really Maybe on X final at comment to earlier to durability look we is to for I time. We to pretty years our wanting on XXX generally, of that price ROCTAVIAN. me. that the patients

of largely And the we to the that’s So, off take factors us a high table can that we that allow Thank think initially. those value will capture for one risks you. payers.

Operator

J.J. Q&A now to Bienaime This it hand will session. concludes the remarks. for closing over I

J.J. Bienaime

all us joining today. operator, you and thank you, Thank for

important our profitability, We beginning year. are an component story first GAAP a portfolio with is forward for record paves the GAAP VOXZOGO again of pleased it addition to and sustainable quarter begin profitability of XXXX commercial results. growth definitely to this going way The a our

focus to months. transitioned and We of the U.S. over have of larger the be the hoping therapies year we for that you that you our apprised sorry coming our progress to look in conditions, and Europe. be innovative – And XXXX to the development commercialization ROCTAVIAN are keep of approved will successfully and hopeful weeks genetic will we forward

for your continued we you thank and all you seeing to support look So, forward soon.

Operator

This Thank participating. concludes conference you today’s for call.

disconnect. now may You

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