BioMarin Pharmaceutical (BMRN) 22 Feb 242023 Q4 Earnings call transcript

Thank you for standing by, and welcome to the BioMarin Pharmaceutical Fourth Quarter and Full Year 2023 Conference Call. I would now like to welcome Traci McCarty, Head of Investor Relations to begin the call. Traci, over to you.

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

isolation in financial during GAAP. superior or accordance as our and financial are addition, reconciliations section will non-GAAP Investor find to substitutes earnings the measures presentation, not Relations today. These to GAAP In prepared can available defined both related in you in release should in measures of which the use the the from, earnings call with U.S. And Regulation on website. U.S. G non-GAAP for measures we considered as be of

the call the Greg Executive portion the the BioMarin's Q&A to Hank On during Worldwide us of and turn Officer; President management Vice from answer Officer; Chief Financial and Hardy, are Executive President President, President, to Technical will President Jeff R&D; Vice and Mueller, questions Chief Fuchs, Brian Guyer, Executive of Alexander Executive Chief Ajer, call President over and Hardy. Commercial with Alexander Officer, are and Vice here call. I BioMarin CEO, Officer; today Chief now

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

XXXX evident. Touching BioMarin's positively patients' profitability therapies through value transformative revenue the revenue while reiterate more operating has bottom BioMarin by equally on results. quarter basis, and as As more and results guidance revenue I position expectations, a compared today, XXXX Non-GAAP financial self-sustaining a performance. On XX% XX% the opportunity fourth provided to XXXX earnings for the business This line lives today's full in financial story increased the year. as Total at a to And per margins XXXX. said in Brian our and will XX% share. the provide our XX% of of expand the our is per year, year-over-year. These grew of earnings as shareholders implied been important by constant full that results. growth impact XX% financially XXXX, was full total creating quarter never financials of fourth the growth XXXX fourth delivering solidifies the growth increase an very fourth to to and the accelerate total compared January in currency grow share for growth quarter revenue moment. grew in XX% year prior fourth along revenue, to today, with the on strong quarter in of can quarter versus a details

priorities January. now will outlined on on I you I the progress turn So update in our to

accelerate opportunity to and Voxzogo first, maximize The front and remains the center.

results QX. financial pleased were status. with sets last driver. XXX XX% October. a added growth close in multiyear patients be new growth new The from children FDA's the that last prescriptions today, in Voxzogo and by As infancy approval of U.S. expansion treatment QX, Voxzogo The of quarter, with blockbuster We under to allowing up age X in on approvals XXX% is the following age the demonstrated EU U.S. to path major achondroplasia year-over-year, a launch to

to and for this an potential just of pursue as enable treatment encourage having expanded and record as families therapeutic height. profile benefits years track Voxzogo's age safety believe clinical beyond to efficacy possible We X,XXX benefit. early from Beyond label, will than Voxzogo patient maximum substantial more data as long-term long

As an of seeing breadth scope data, in our a growing long-term of we're the extensive result for the clinical the demand increase and global treatment our Voxzogo of prescriber base.

endocrinologists on to engaging achondroplasia expand fourth program children run-in fast conditions. In the of build Our quarter, outcomes pivotal into observational in extremely as of plan of BioMarin prescribing over With our in are Voxzogo launch United of programs our well the X-month the pathway pediatric X, world and in health and as later work children the with Voxzogo statural confidence uptake under regarding received. uptake intervention, to goal greater earlier leadership begin with stature for States year. to to -- established multiple being relationships actively conditions, has plans with treatment been the genetic idiopathic with our multiple hypochondroplasia, and on recent portion treatment the stature therapeutic short plan studies authorities in in began other development longer and we to drive pivotal enrollment achondroplasia the We for global is build children short twice X track. with this

will of As achondroplasia. discuss that believe indications there Hank is proof we beyond and strong later, concept

hope empower these to of we patients their to lives live As families across will conditions into we Voxzogo statural fullest. the indications, treatment spectrum and expand a

For and other the this reason, top remains acceleration, priority achondroplasia expansion of into indications Voxzogo BioMarin.

Roctavian is establishing priority The the opportunity. second

XXXX believe long-term inform I potential. XXXX uptake As we and will Roctavian's said January, in curve and

covered. Furthermore, in well with confident been payer strong profile We is have lives product, value of with been warranty with positive it to clinical the marketplace. published this the and into be and policies proposition associated response pleased which and how the received by the we translated Roctavian continue evidenced equally to the has payer the very has agreement,

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a The transformational be the in profitability in spend third fourth benefits each terms by the of under Investor XXXX. In promising priority our in prioritized of faster candidates we we A midst. To to which the Day is undertaking with than on planned. I've complete our and and focus with in those financial shareholders update potential pipeline strategic with patients later to the provide will potential creation advance lastly, priority lot assets, a end, high that commercial high highest of transformational most the keeping for portfolio a Hank asset profile potential. review value moment. And are update development. impressed will and highest our communicated But most ambitious meantime, on patient our with we intend a not. on is advancing, the very an will the prioritization bar currently level disciplined will a increasing R&D at team I've determine time have of and those those innovation R&D originally assets been understand developing in to pipeline unique R&D for hold which assets, and impact number and therapies. been spending of expertise of impactful goals, productive

and significant our demonstrate Our operating XXXX our XXXX for growing profitability results guidance leverage. full and year both transition to

focus we our year Voxzogo streamlined total includes growth progress business you margin item, double-digit all of reflects the expansion achieve Our anticipated a reflected you will guidance, primarily XXXX regardless year P&L guidance, priorities well our with our commercial share to full products. Non-GAAP line year, X expected just to the strategic in year-over-year full into a is items per clear and guidance and track across major as earnings this We of margin non-GAAP because sight revenue allow of described. on BioMarin I full than XXXX financial revenues. guidance which strive performance. faster driver our providing leverage streamline believe it BioMarin's the guidance year as as into a reflects growth, item contributions, be new put and line revenues operating is to Roctavian is growing

financial So in I at which will exciting entire include and achieve shareholders. for hard and sense team as with views see organization of on patients is entire setting it's this for as strategy, value The time at corporate BioMarin, truly and The R&D targets. steps shaping is summary, work create capital future allocation, mobilized tremendous approaching an taking long-term on and opportunity leadership ambitious a to urgency. work and to well

could That updates. Thank Day, for attention. provide R&D we highlights. to the Please will progress to turn that the significant Hank? on We over specific externally timely to will an at we update tuned be evaluating you incrementally on share I for want basis. call a additional key now And for Hank will the stay Investor and which are timing. your making be

and benefit productivity those therapies. R&D The who behalf gratified afternoon, good Alexander, achieved you, on everyone. is Thank the organization by from of innovative our

the which drug financial advance. we level to with patients. spend the assets our What medicines the most the of goals, and BioMarin align our As capabilities will rigor to of and will development high-impact ambitious change prioritize future take used determine is we into for continue the criteria

assessment study assessment look from we results Phase briefly this of the a oral effectiveness. the and Day hypochondroplasia is and with in time to patients. open website treatment at competitive the with the of -- expedite Investor Dr. of forward few As and proof development, trial cost represent with in our of highest Alexander Dauber's updates, pivotal as next in prioritizing undertaking the at II ranking, each to to determine of advancing We amount and recently value to success presentation probability clinical XXXX. Touching mentioned, higher required assessment strategic strategic through study hypochondroplasia the investment investment XX-week his month. the opportunity The portfolio global Toronto Voxzogo those middle then on Results Voxzogo as to our were we of meeting a of increase landscape market to sharing the intend improve of the designed for only posted ACMG the will assets approvals ahead evaluate well for the concept that in are of bar later the asset. goal The year.

XX-month initiate plans of mid-'XX. X.XX provide the pivotal We sustained X and months. treatment program observed treated and hypochondroplasia children support hypochondroplasia, XX in an in begin increase are velocity data These durability expected for see in growth Voxzogo-treated imminent our in at our and with to a of safety in months encouraged to further achondroplasia the phase centimeters annualized profile to

Meeting conditions, pathway begin stature Regarding are with studies our genetic with We we position development programs programs just and at May. forward idiopathic across later the securing in is pilot plans short to stature multiple the authorities the Dauber's treating to update our ISS look to health for genetic in and BioMarin look our on in forward pivotal Endocrine year. X we leadership statural discussions Society multiple global you conditions, progress updating described. Pediatric Dr. this

molecule that as with XX the small other briefly functional designed proof-of-concept Duchenne in measures. BMN boys stage designed to both liver We're expand enrollment study the to share potential of BMN-XXX XX-week of XXX treatment II the with the Phase assess of assets and The is for study alpha-X and will levels for Turning is muscular pipeline. early enrollment. dystrophy enroll is early an the treatment dystrophin Duchenne muscular disease. antitrypsin dystrophy, open deficiency to pleased bioavailable, needed for to

our of over prevents to which healthy updates. protein following And financial indicate if is progressing forward thus drives on volunteers assessment Our pipeline to for safety mutant assets and I disease. look nonclinical the the ascending far. cells the for will strategic Thanks your turn Alexander. Thanks update other its for the polymerization Brian studies described with We liver observed The providing by your call support. study Brian? attention. signals in now secretion single progression no an in of unchecked, dose liver human enables

and and to XXXX. file for results be XX-K, next non-GAAP press XX% generated you, will updates comment Thank XXXX quarter for I release refer financial a comments few quarter summarizing guidance financial Kuvan, our basis $XXX results measures. full of revenue expect GAAP which Form growth, and to my full We available XXXX, today's in XXXX, strong on on XXXX to upcoming financial our the the Kuvan. excluding BioMarin and including provide total fourth the year million, we for All details representing GAAP financial year-over-year including on key growth, within XX% of days. In will reconciliations Hank. fourth will results of

expected, generated XXXX, than revenues net year-over-year. $X.X X% strength for full more increase As of of our billion enzyme of products year product an the the

Voxzogo XXXX. in closely million We've quarter-over-quarter performance more $XXX previously Looking constraint on at growth. XXX% supply the revenue in the represented discussed revenues fourth quarter, Voxzogo of

increased supply middle as expectations. R&D activity $XXX $XX corporate well $XX of fourth expenses we discontinuation to XXXX the supply over items for Palynziq offset our the SG&A million, primarily the Voxzogo guidance full XXXX. impairment in intact, related in combined these research quarter peso and revenue available by to to increased plans in Argentinian were exceeding our million devaluation up the of important last first-generation during stage fourth pen. to as quarter incremental transitions. expenses partially fourth satisfy million, including incremental revenue year-over-year, an leadership from $X.X clinical that Together, million governance expense $XXX The up year of Our activities commercial G&A a this lower unique quarter became expense the higher along with number in due Vimizim year. Please and in fourth of for revenues, the our quarter the QX revenues contributor were $XX program. the to than by quarter some was also late Kuvan of remain note, billion had with being in million and and total anticipated driven result line total around to year and early charges growth was of both matters in XXXX, our forecasted demand the quarter. Double-digit fourth was related

share. quarter, an on the GAAP the recognition line. net income $XX.X income impacted the of of bottom income to fourth representing similar for lowered that other third on a GAAP per share of quarter the recognized fourth for $X.XX incremental effective diluted full for GAAP rate that the by year, the and income earnings some benefits net we was quarter will net GAAP contributing the quarter per $XXX to full for taxes, Moving in loss line. I also the million, expense was impairment our tax in the recorded tax year. note discrete fourth to million

was over low and the the for an XXs XXXX effective share, continue tax global and full year, in We full Non-GAAP $XX consolidated to growth and diluted million project, $XXX for year rate representing on beyond. a level, for income the over EPS million fourth XXXX. per XX% quarter non-GAAP of $X.XX

please we As into effective becoming XXXX. in there that in move are select changes geography XXXX, P&L note BioMarin's

bottom impact changes, clarity to being industry Details classified our expense, and expense. These better plant of foreign foreign operating previously the on our and costs as line prior idle expecting prior instead revised plant presentation peers. forward and results that use going to only currency conform on other idle XXXX income losses align the available which of and made within reported presentation gains to and from are we'll are core of are forward SG&A cost do the in be its periods with to are going their We well provide website. will SG&A with and forward, revaluation as classification XXXX of exchange expense in classification within of costs greater not sales time-related instead going classification again classify

we operating for non-GAAP share. earnings mentioned, XXXX, Alexander total As revenues, to per are and diluted margin non-GAAP guiding

with a the continued exclusivity. starts, of strong are guiding Kuvan and is at decline continued XXXX since our additional full not $X.X being portfolio, revenues, expansion patient that growth represents our For of provide key growth expect by the brand this driver growth specific first anticipated XX% approximately younger in loss of brands XXXX. To single-digit year XXXX, time to high the across which driven to by market the $X.X Voxzogo, we revenues, to its to billion offset revenue into age ERT we total is X context, new due guidance the groups in some between compared and midpoint mid- billion, XXXX

definitively revenue based the experience. our modest is in from our on recent risk-adjusted relatively Roctavian included revenue and Roctavian is contribution total While forecast,

be the QX stand forward, XXXX, Roctavian first revenue we I half year. With comment QX the some the versus with reclassifications between quarter expect to of behind year results guidance will operating volumes, second inclusive we to expansion is for respect revenue speak XXXX, we to and provided our X we full Going a specific non-GAAP and represents will actual of revenue that most performance. Also, dynamic the total the the guiding timing margin XX% included This to today, XXXX of XXXX and XX.X%. margin are QX of percentage operating expecting to to We the growth which of total let QX respect XX%, G&A the growth. to weighted full of XXXX are mentioned. one non-GAAP points that to modest incremental year impact order whereby just that

to help $X.XX we year. And share, per earnings last over XXXX, this growth year. working cost are commitment the through years, as This approximately of Over to our the non-GAAP last increase points, XX percentage the midpoint including guidance. X set year it's for financial and however, XXXX operating of targets and XX% projected XXXX end for the at full be allocation efficiency in the share to we XX.X% between is represents story growing per XXXX, our which margin our not the guidance, diluted to through from profitability, demonstrates non-GAAP ambitious by approximately resource our expect our priorities XX% to communicated $X.XX midpoint long-term

new chapter our Voxzogo to strategy business call products and value achondroplasia, you we questions. As your driven growth executing and of enzyme up the of impressive potential and into now a with performance realize the durably to the move by your on in we'll continued we growing pursuit BioMarin, support, for Roctavian. Thank indications, of are open the Operator? next

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

and I baked guidance of do help OpEx strategic there that understand R&D is some for R&D just in And advancement? XXXX portfolio you EPS better your that investment the assumes? also the the understand us there? criteria and that Are basically wanted into assumptions to review what further what your cuts program, overall for are

question. the is Yes. Appreciate Thanks, Salveen. This Brian.

non-GAAP this noted, a XXXX. So first the as is over year. the items over pleased -- some over of year, growth I in non-GAAP those XX% mentioned was XX% XXXX. There And One that that all, this we're analysis. growth implies guidance impact EPS that few midpoint are of tax benefits those year-over-year

that beginning non-GAAP. is to in extent if now since benefits a you 'XX tax There's recur, So that be of non-GAAP headwind, the those to don't income our will, the XXXX would some We're system -- got also SG&A I 'XX. some some a unique incremental is 'XX. G&A in resources SG&A implementing mentioned unique We've commanding in new 'XX. in the charges ERP which

of some modest opportunities strategic just cost our about were are dynamics. And much items of included, question are the a efficiency to your amount known those savings the that on. already we of how working the of So this part or point at

to for on more model cost the be to embarking of the efficiency later we communicated setting this is going exploration the as operating be are We speak, target year. this financial ambitious future and part efficiency

guide. that of that amount related that not But a in stay there's part. this say on ask we you substantial savings So will I to tuned to

about the to of second portfolio. response prioritization R&D question the in Salveen,

access to ranking, concept well approvals having include competitive that subsequent are our the opportunity those goal the bar quickly. highest portfolio a important be, a forward, as and value When then may assets of things of of to more advance consideration with happen global foster impressive are case We development, shareholders. The of landscape. market involved time to strategic and the concept advancing money will prove happens only very as patients undertaking expedited much high to considerations registration assessment development. that to and going early and assessment for as market represent and the And and of or is prioritizing

in of also improving cost programs. We the can we investments bars, the have success higher see events look those of with forward subsequent to and a so strategic contribute higher later that also sharing the XXXX. of effectiveness to probability And results

of question line comes Our from Akash with next the Jefferies. Tewari

that past, resources Alexander, capabilities, but value. really BioMarin translate in of spent shareholder therapy and to building we lot time gene up a the its haven't seen

platform almost question that For looks the different or your spending with cut like amounts in Roctavian, of Roctavian mind? is focus Voxzogo to costs and story on in you're your option out on example, divesting it your results. potentially therapy thoughts or but What identical order are the even gene

Thanks question. very much the for

in We mentioned opportunity I very our stage we're establish the really terms the early As of Roctavian. to with still priorities, at want much there.

seeing We market terms access, in are progress market of activation.

XXXX, run also in spending that time, that So the we're think today meantime, review. supporting But is strategic currently wanted determine. In launch. we the wisely the XXXX be seeing same through probably of on we we're the referenced we'll to when and this to see we we're really conversation return mindful a from able Roctavian. At into investment cautiously very

that, world it the pipeline. we thinking outside course So to stay right also gene of take the looking uptake the lower gene therapies the scientific what's therapies. with strategy to future looking down of Is on, at we portfolio. regulatory We're we're about as double note do in assess our own happening in exposure we'll We're exposure taking or BioMarin's to R&D market challenges, therapy, market BioMarin. part walls. And of want have something our and the gene a access, at also experience the

So Investor year. you to later at Day keep we'll provide updated. information We'll this our continue more

JPMorgan. Our comes Fye of the next question from line Jessica with

A couple on Voxzogo.

sign international Voxzogo of inventory from seeing whether the would talk or modest revenue some basket is development XQ How you indications growth. the channel the Can potential any for you're Voxzogo ordinary? And regulators specifically financial FDA likely the sort to broadly type trials these XQ of reflected revenue of side, can with on then lumpy mentioned approach? what revenue, of number impact benefited so, I or just that you incremental areas. something XQ side, out do First, other XQ the changes quantify on off additional was that, on like think And that you orders? multiple to pipeline if

Jess, question. across And to the I'd your take of respond excessive the nothing the and Good marketplaces. channel I'll Brian. first broad I the made way nothing respect that it's question. is, part with comment because when full first end guide, about we the back $XXX November, million, constrained. we end of how the year on and that was gave guide our high talked was supply high QX essentially

And so here we are reporting for close the to $XXX quarter. million

I were explain additional So to became want that that circumstances supply some commercial the there available.

that to And can there. dynamic So that that used expect necessarily guarantee it we QX, with the we respect meet out demand same recur. that, or was point is don't to

So that's comment overall is not second see our in strongest of year. quarter, that an QX dynamic, to the is Kuvan that in to in going the and QX half decrease the we're plus going QX, the QX of just be most growth

Jessica. Thanks,

is subsets actually benefits of concept could a great the known of one really of study Dr. things short formerly Dauber already. his his feedback you that good as we of hypochondroplasia. on see that the of And that might question be basket idiopathic in fact, or study stature having to regards have do basket, As is itself regulatory

decisions authorities baseline we are when with the our in finalizing gained already for enrolling acceptance. interactions and because we'll those studies additional game though, said, that stages concept so can that, that these a communicate any of and of front we're the after for baskets of prepared the comments, not are the health theater these So last has FDA expedite of around registration we're online. All said run-in the And studies little studies essential been final having come of start already plan we that basketing as bit of our reflected that in world, and also completion of studies, design has implementing our the in waiting activities. our

world around sum, be studies the in think health trying working the as regulators. that be to they getting with So and underway meet cover authorities about to we as ensure demands I we're of can

Phil Cowen. next the TD Our Nadeau of from question comes line with

at question already? changing Voxzogo's to full, about approval any the what the potential Is to update potentially studies be any requirements follow-up would process converting that's the A on talked accelerated the regulatory necessary on Voxogo. in past, BioMarin approval from competition be point, and impact and In had Voxogo's this competition the it converting there approved. of to have for would that pivotal for on full

through Phil, Voxzogo's no particular read-through regulatory really every update one that's about is often so the or from we assess studies can diagnosis a at the a their that growth, has safely it's patients that way to in bar through this going all that key resume future rate status drugs features growth ongoing we benefit the Voxzogo entire earliest plate. of of think the important an be for growth-promoting period patients. And to and innovation sustaining look on to of report to we ensure of of other the its the of this But space, and been durability be effect, of of effect. of growth And on can types that's I unlike continue the starting strategy update or the things closure benefit competitors. remarkable physiologically normal the that reassured one really exciting Voxzogo, any --

So far. so good on Voxzogo

with comes Our question from Bank next of America. Geoff Meacham

growth a terms about of pace that the does pipeline so of I evaluate look of you, figure new the of in over to the beyond. couple of centerpiece what I'm the into year like been looking this second you kind investments commercial for base can XXXX? the half know of Voxzogo. starts what's Voxzogo. success impact the out of and to we do last then And broaden what for just trying ask of year? investment major metrics course and your think Another a far the one prescriber wanted or on it's year for I

Geoff, it's Jeff here.

me a at that. shot Let take

we've think had on our I to So date. some returns good commercial investments

the starts, United good mentioned the noted progress. And on more revenue. the from as last endocrinologists label area these is remarks. say following year the of expansion, would Expanding year. back for and last in in particular the into I prepared now accelerated revenue best base this recommend post-launch were States, particularly terms and We're is new You towards of we're making metrics, I starting Voxzogo starts in turning year in I launches specific prescriber late focus, both new line. would the pediatric revenue quantitative suggest dialing a going particular, that metric to forward metrics and was a might base,

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

in as mentioned What in coming patient to At will you're from Update, and that short indications prioritization and across in the any thinking many terms about we And be not? efficacy? And the ISS terms Data are should then you you stature move just of conditions, expect May. versus which looking look what threshold the numbers forward the you you'd see? Voxzogo of how different across

Okay.

is that wide scientists really I the be that of fundamentally corroboration are I looking the indications. Dr. reporting hypochondroplasia his that update, than he's to as and regulator I ISS, enrolled things of is patients he his unless believe, what, reported, is as much study, there he's think to growth he CNP is think, a other was a very As determination understanding to work more useful Dauber's think indications. be felt My so this which master to bone under for most it's information But of anticipated to regards be across to what variety update. going going

think effect we basically of see down or for types gets are be to really expect has different process global to think seeing shareholders. that to second metrics described, question, in and created stature around I of concept fairly FDA as this conventional the patients the we and predicated X-month to regard first cost III proof of it's seen Phase on PES mark the program, we the And lot go at I ISS at had of therefore, with into cohort could the hypochondroplasia the to biology. outcome that's half so And enabled sort the same presentation time value prioritization been actually going of important is registration, so that directly a your as we gain confidence that and the to for the boils that disorders. And of to

only of With like will of something the higher hope expedite bar the shareholders idea bring those, high winners. value focusing to by faster setting And we then development patients on and concept, really of and your proof that by a advance and undertaking. you for

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

Welcome, Alexander.

operating heard Day I that the communicated outcomes say in be strategic the and as at think I Investor quarter. could of the the you review incrementally as committee third well

So sense more was a updates sooner might wondering have of which if formulate? updates could I to emerge time which and you require

guidance well. question. Thanks frequently and We've we're strategy. Joe. financial long-term the the for guidance, met on is The Thanks, extremely progress and much proceeding very making SORC financial the allocation capital

right also with into world a cost cetera, together Voxogo, a going At have cetera. And we're keen et to us can et BioMarin work the information of the time, we we're the the is, progress this of future. all can out external the Investor soon We of our the prioritization, get put optimization, as underway, faster share acceleration where as sense the timing to is from on a now, best benefit the we strategic figuring we're on other progress as same context we're on Obviously, lot these the you the to take even to R&D what making. Day of points. update allows

as more for be sooner. will day So If by for information. possible we'll of all information it makes sense, stand my communicate The information. prepared said please Investor that latest in those other I Day remarks,

Truist Karnauskas of with comes question Robyn next line Our from the Securities.

on One I'm on Nishant. for Robyn. is This Voxzogo.

us So Can gets it how Is [indiscernible] about both give or any competitors' if spoken for think prescribers on I about there approved? room have color, the Voxzogo you orals. patients achondroplasia. overall treatment know on any you for

trials. significant effects quite shared at improvements from growth more informative. And robust we've positive ACMG, on our and of in clinical Voxzogo as long-term data Yes, accumulating just also referenced, And beyond data see really is positive on importantly, we height demonstrating on height. gain highly these that I the and growth,

domains of physical life of the For of example, types in scores. quality improvements

see building lives. that and And patients' to an in statural leadership where entire of so possible through we're growing we the impact position really Voxzogo, in conditions again, growth for focused having incredible the normalize to the greatest potential us growth normalization on our genetic period sustain

line comes from the Bansal Fargo. with Mohit Our next question Wells of

puts I'm portion investing question would is a therapeutic Maybe you. modality? there? love what what for welcome. And is in pipeline think one gene thought and therapy how for the Alexander, gene And general congrats as general, takes therapy rare to because on a of and asking and significant diseases? In you general your your in to understand gene about are therapy

very much your for question. you Thank

Gene transformative is but broad bit a potential for significant a detail. We've bit, very modalities, little a go exciting little in got obviously including patients, referenced me let therapies. gene I therapy pipeline, offered benefits multiple more and benefits. As

therapies have that set capabilities That ability an commercialize develop, is geographies. manufacture has. now We and to unique gene of in BioMarin and multiple a expertise

going look the Hank potential? the really, What the asking where competitive a to at we're together we're at really same form transformative exposure also And answering the to the and pipeline, as therapy, question include about, we potential overall with our our also have will a are was And of the portfolio. prioritization assessing will of program-by-program we're going What program, BioMarin. to the going forward. which at? our looking is gene and we talking confident of As At at commercial time, be about that advantage we're strategy good basis. sustainable

in Day clearer hold and more later So it this. Investor your at this, come on share to We'll our of have idea Please question. we'll a XXXX.

comes Wang next Barclays. from Our with question Gena

I questions. very X have quick

dynamics in you second, pricing There total X,XXX Voxzogo. said opportunity market terms regarding is treated already for achondroplasia press there? And is And regarding of Roctavian. XX these numbers quickly also achondroplasia one patients very in across your the What then active what first active is you markets. The XX the markets? release,

that be patients the be dosed to was in the patients to think 'XX? X earnings X ready I quarter were expect 'XX. first dosed last mentioned we Should XQ remaining in

first I'm it's to that here. patient numbers. Gena, Voxzogo Jeff question on going take

or commercial total where the XX which on eligible months essentially that now patients quote in use group and of the usually X the to essentially, core of that's that months age eligible, for X, potential. would are the different that depending world. the patients, we represent And in markets footprint of kind So are those patient numbers you limit X lower matching about XX,XXX market labels, and

in of the And Alexander. back to Roctavian terms question,

Yes. Gena. Thanks,

correlated your at so tracking question. tests. far QX product take readiness uptake. CDxs to clearly, to second call revenues me treatment completed We're provide April. Let with have We're But hemophilia not on on positive details centers patient payers, progress including these going conference at metrics directly in yet our site

patient our no be updates we're just about again, and with we're revenue. quarter the commercial this infusions going and year with expectations longer on at report to the our future So metrics. let reiterate, providing the end specific experience we'll going actual Once infusions form to these to of actual progress

comes question Stifel. Matteis next line with Our Paul from the of

any helpful. capital I of on commercial after This and you for you're But thinking perhaps programs bit of bar Paul. just is for just capital high more do Voxzogo? provide can reviewing advancement? that in about on the could XXXX guess of business color on for moving in speak allocation provide growing that forward. cooling reinvesting just future allocation beyond, of super you you I -- you Julian would are on color Obviously, for meet guess anticipate you sort the pipeline, pipeline. little terms the don't that be Or focusing a

now we flow strategic allocation and that mean, be a only a innovation, record total BioMarin to the question. today, to positive. you but to going value continue recognize bit outcomes cash created capital given amount business for our the to can not track substantial investments Yes. in strategy. current details ask cash This Thanks and a priority. I carries But Brian. of also internal We'll give is say you are that we we wait for is the on of review longer our our

So is we'll we developing the you strategy work That we in to it ask you, work. much we committed to allocation interest forward look the that's tuned will are and shareholder but of until with stay maximizing process, value. sharing finish to capital a very in just

Purohit Stanley. Our Morgan from next question with line of comes Vikram the

bit follow-up broader had We one. question a of a on Roctavian, a

control areas different mentioned experience as that support you're So but commercialization you uptake just or patient that of over with XXXX? you X what to through your of release here, that the from a you have most couple and here mentions readiness, work leverage the do that you could X now specific in education months process the think a you've the few help near-term see factors Roctavian, had for and of of was trajectory wondering, to also drivers or going I site progress within

about I'll much reimbursement a site market demand, signals patient and about nice funnel priority we've of gate. important seen having we've talk clear good that where of in the importance uptake for reimbursement gotten a buildup Jeff bit talked of United of last year, talked that. this is very of of What that here. also in States, that about Vikram, strategic patient we approvals we We've kind example, of had market Germany is readiness the and testing. the CDx little the said

seen As Alexander we a correlation. haven't noted, direct

this leading to and uptakes. been good revenue indicators indicators are of that, patient say, predictive or So haven't or these hey, able we are infusion

we're from model to instead that And trying I better going is the speaking do revenue for here of think actual let so the Roctavian. the tune idea to

next William Our from with question the of Blair. comes line Tim Lugo

and operating a margin in other companies? margins whether expand like will and the have step-up initial to be to You your on what impressions nice north are continue reach able guidance eventually some the disease-focused of introduced of BioMarin you year-over-year, rare XX%, operating

appreciate It's one. Thanks a I question. great a Tim. lot, the Yes.

one we've that BioMarin the mention actually to year-on-year hand, revenue and of into growth and strategic for that. I'll upon there's question. know years We've the of while, are few the talked type a it So on we to is outcomes On about work able not lot been revenue your has, pleased over of answer a to -- that potential opportunity the a today, impact lot last the with future. pleased of improvement. it be deliver again, able and be to to talking about kind operating a X-part we to margin report deliver we're give

this for deliver that the to in by R&D, entire enterprise, the and reached guidance way of We've infrastructure model efficiency leveraging deliberate operating really manufacturing, we're the behind. And doing we've means these rallying that design that way, financial ask question example, can together share been Voxzogo opportunities. initiatives and the we'll picture will work and more you we'll enterprise-wide the got appropriate by long-term efficient. and them. business those is opportunity strategy to commercial, for means, focused now across company. rationale, structure stay for years. leadership part years. launch of got our is, have to the now numerically, the lot very doing tuned. scale. we're optimize just capabilities plans a And reason, cost what a second organic the we've but We strong the and be That's we to the have that you purposes targets business that growing my recognize the support And be support. But over the optimize with that We've what make company also of not work deliverable We team a to global And cost we're the to scaling is built real the for and are and sure upon SORC. world-class to for and But model all on

question. So bit the a you, more that's take just going though, for Thank time. to

question Cantor Fitzgerald. Our Olivia Brayer comes from of next the line with

have about But for the as as to dosing opportunities lot we optimize avenues maybe to any competitive your you could ask franchise. life management possible? see within schedule to product. that Obviously, Voxzogo any other expansion cycle efforts product Wanted keep better a maybe or of that

final, is parallel to alignment. again, good a in optimization. our dose That's after that Right authority to communicated undertake now, regulatory in yes, be designs thanks. thinking But try details some fact, with do do Yes, are when plan registration-enabling we to And trials. further question. study to

with from question comes line the of Kostas next Capital Our Biliouris Markets. BMO

the on Congrats progress.

is and likely, steam you you whether on your third that potentially about details. to pipeline the took when you the the this third planned fate? programs, over, prioritization quarter, day for is progression which will pipeline until programs until share is wondering Alex decide year their going of full after I'm progress pause are XXXX, a some all this continue R&D of of Given you will to or quarter

the around of early Kostas. way big portfolio -- conversations Brian of having part The ties that about that can that that the The great thing developing generate through we And of that to longer-term a the medicines. of was Alexander think to year is be that cash prioritizing our R&D -- company Thanks, it's was purpose earn just it's outlook. having a in can the new I assets real have value generate financial a reinvested part portfolio. to have that allocation, our think around R&D to capital reapply then I

So we a have in to business context. do that

go going invest that forward. that spend things make and go soon to not are on money things going going expediting want sure to as forward, we want what's decisions about to so we that go not we we also that to don't make are And can to in forward, as

over of So course the that's of Day to it's, the informed very and intention, Alexander's the I the statement Investor year. much could how incrementally progress potentially be through our to of process ties think,

with comes of line next Our Jack Allen question from the Baird.

And all one of course the congratulations on the to I Voxzogo. on progress ask made the quarter. over wanted

is guess, label penetrated are? the growth the the about Voxzogo will think in could context Is there uptake of XX% younger patients? come U.S. well? from new patients about how as that Or the expansion. it more from expected older were we expand mentioned You and in patients older opportunity still moving quarter to in mainly of U.S. provide some the that the And you should around starts forward? the younger patients fourth it how I

the that the an heard question, you quote going we're quick Gena the on not X,XXX XX,XXX, In penetration of metric reported of arithmetic rates we disclosed eligible population that about States, you for United Thanks do numbers question of yourself. Jack. can earlier introduce the haven't me with to patients, patient and and to overall,

those approval younger and ages also from the patients. new one States, up value X encouraged uptake by the United older our the is the the from highest from both children, which of largest really and original continued in and Specifically the markets, demand we're

I opportunity pretty that an for uptake confident So increased forward. we're we've think going got there

Our next with question Lebowitz the David comes Citigroup. line of from

that, on? year? new maintained as to up from goes of on scripts on being that, the patients it number Voxzogo that following patients. Is young Just dial Or with year be assume weak should back that ] [ XX% would next

that's think on. how good younger expansion. and got in on one key going good little me think looking you age we But let I one, data signal, that to a color don't an to are label things that it's maintain give onetime David, market I specifically of going have But take intended the mix. a enough since forward. metric say it's we're a we encouraging that to bit that and important

Our final with of RBC Luca Capital question comes from Markets. line Issi the

appreciate Voxzogo, is given the which make cycle inhibitors, Alexander, a to driven VIII patients -- any here, really younger Or patients before at just antibodies time, by testing that including prioritize I relatively BioMarin [indiscernible], the with color neutralizing was R&D so slow to launch, [indiscernible], Day Factor primarily you efficiency for and year, which last AAVX about your capital, there, extensively there still talked on. patients quite invest with life is plan? much a don't operational management the that appreciated. story scenario

our in assessment review studies intend do and portfolio We include those to prioritization. of a

plans the overall our did portfolio Day we well Roctavian as so that communicate each outline additional prioritization at R&D of the corporate complete as just And as we'll process strategy. we for and did our you that potential list, indications

then. And so we'll follow up

I looking potential. impact of potential together would with we've is add to terms medical commercial in as said that the that, at the just prioritization And that R&D benefit the

So those commercial developments. the of cycle around we'll life be taking business experience with uptake to Roctavian into inform the our mind case

the CEO to remarks. President I would Alexander now of for like Hardy, closing to BioMarin, turn call over and

joining Thank today. you for much us very

future here As you've the heard, as we as well are shaping corporate really hard ambitious on and financial strategy at work long-term R&D setting goals.

and We're significant you are progress. we're sense We're urgency, these a on basis. a work with this with accelerating making about profitability. revenue looking approaching sharing of on heard, timely to forward -- and that you've As also

heard, you please for additional you updates. Thank tuned goodbye. and So as stay very for attention your your questions, much and

call. today's concludes This

You now disconnect. may