BioMarin Pharmaceutical (BMRN) 5 Aug 242024 Q2 Earnings call transcript

Good afternoon, and welcome to the BioMarin Pharmaceuticals Second Quarter 2024 Conference Call. Please note that this call is being recorded.

[Operator Instructions] I will now turn the call over to Traci McCarty, Head of Investor Relations.

You may begin your conference.

[indiscernible] Operator, to remind you, this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin's financial performance, commercial products and potential future products in different areas of therapeutic search and development. developments depending regulatory progress future filings authorities, the market materially programs, BioMarin's actions with differ those XX-K pharmaceutical of and and Exchange actions availability and as the may Securities on in Commission, of the product in factors and Results detailed competitors such BioMarin's XX-Q, by of reports. capital, X-K

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

are Chief from Executive Vice R&D; the call President, management now Officer; BioMarin Chief Alexander Vice turn and BioMarin's and Hardy. Commercial Officer. Fuchs, call President, CEO, the Brian President Hank Chief I President On Hardy, Worldwide will Financial Hebert, Kristen of today to President Executive over and Executive Officer; Mueller, Alexander

all Thank and Roctavian. to afternoon, good Traci, Thank joining quarterly everyone. pleased you, today. us the We're today you strong and our on share for results decision

at the deliver us forward quarter, profitability also vision we to to amplify on confidence impact growth, about gives and can our progress strategy, look mission made Day. This month towards significant strategy. finalizing progress patients' This for sharing work we've greater shareholders. more next value accelerated vision BioMarin's and evolved lives Investor corporate our We and more sustainable and

with priorities January. year treatment Voxzogo beginning of nearly And continued strong now in in accelerate resulting children highest third progress on in all the half the specific Voxzogo, global our XXX demand launch, we seen expansion, we Beginning X to with outlined strategic history. year, have opportunity this first quarter across the in patient and in pleased rapid maximize and briefly Voxzogo uptake to were our the commercial made in geographies, our of Voxzogo United from pursuing and young allow penetration a seen markets, potential grow of of infants all saw therapeutic strong in accounted In all new the children largest the those in treatment X of room families and for majority. we've that we under markets. note U.S., our but market the benefit. Outside and uptake maximum potential the key for children opportunity, to significant for ages the particularly with for still ages children age prescriptions States, strategic to their Moving

forward, momentarily. The we we penetration intend realizing progress, we idiopathic progress many Turner shocks confidence leadership growth-related of growing across proven and give us multiple our profile, provide beyond the the body strategy stature, deficiency high will syndrome, the attributes in update height a degree and evidence across Syndrome programs data safety Hank conditions. sustainable hypochondroplasia, of an and development advancing expanding the across rapidly and with of supporting the short to achondroplasia, opportunity action [indiscernible] mechanism the in Going multiple expand intend quarter, we're In advance are States build the the rates momentum to beginning expanding new the leadership geographies. Even indications. with to this United other in full on and in at significant Voxzogo all we of tremendous seeing made still leverage across our

allocation based decision like supported we Roctavian consistent decision profitability. This profile our this today and to establish growth ability result efforts, expect was third, Roctavian to confidence One, the BioMarin's and annual a lastly, addressed Roctavian couple profile launch of while hemophilia on to therapeutic The contribute in that gives to to over with long-term in will the in press signs conclusions: balancing belief States, quarters opportunity role the direct our and second release. our was to on Roctavian progress Roctavian enable its $XX the last priority in revenue of and evaluation expense The of Roctavian takes to X separate that research second, revised United reduce contribute Italy; approximately manufacturing million. commercial, expenses of the time; a a focusing understanding us A.; Germany resource our profitability. By

operationalize end the We expenses these will by begin Roctavian this year. to reductions of in

effect new for year the in the are expenses XXXX. beginning lowered full So

being company the is by As of of a result Roctavian end the profitable committed these changes, to XXXX.

to global only we allocation to securing profitability, elaborate contribute in markets portfolio. allow to support to as where that Roctavian made and the will reorganize Christian success potential have providing well on access to focus changes enable We believe prioritized a we enable resource this the patients. will progress dedicated to the addition in accountability of on the team the progress that enterprise on will this of rest In reimbursement unit direct the Roctavian our potential will as BioMarin's focus to of Roctavian to realize in maximum we are be its Roctavian's seeing X revised strategy. and rest exclusively incremental

these prioritized including on we external priority forward promising internal And our All We update X R&D on also programs fits indications third well at announced made focus strategy it to with capital all how the The assets. BMN BMN our and XXX, an innovation on into continued as as most Investor allocation advanced Voxzogo BMN our programs the strategy. new last XXX XXX, progress and look providing is X developed. quarter. Day,

we to ability growth gain the expanded symptomatic significant efficiencies and next the business want earnings we team over or enterprise operations profitability I Brineura of that and of do in all our share, CLNLX to of presymptomatic. on I turn specifics Lastly, In call very their growth, high-impact access approval beginning of to planning age patients. and have of as reshape profitability efficiencies the the disease and families to across progress will greater are Brineura of to the approval revenues [ proof to our per disease. rapidly beginning program the across of today's extend this outlook end what originally to has and early progressing behalf. In the and my expansion enable cost full quarter. on medicines forward year X for course made potential an years in as BioMarin's children of in the items. expansion to treatment this our for realize years on under This we have and growth who provide are to next diligently pleased ages increase at just now Today's to more we profitability. enables children in accelerate overview the neurodegenerative seeing natural disease. July, possible attention. the increase innovative XX% in summary, Thank we work with to announced non-GAAP were now you planned. thanks focus R&D our highlights best, whether look drive the And you the to to strategy. your in share month We to commercial Strength term. Also Kristen expands fourth priority for longer with create on alter CLNX ] guidance received they're the results resulted regardless all treatment the total disease over Today, faster XX% than couple into for

my you pleased call May. results Thank in to you, joining join Alexander. quarterly BioMarin I'm on first since

impact patient Now the drive ahead breadth expansion we commercial been BioMarin, excited and as of about through the I've lies and patient continued by impressed expertise our focus I'm medicines. of innovative at what

growth revenue quarterly from contributions highlights. drove Voxzogo, Roctavian. including to Turning Strength our XX% Alenziq, Vimizim year-over-year, Brineura, brands Naglazyme, across and notable

the million year. to the second By Voxzogo were second the end of X,XXX were last Voxzogo revenues quarter Moving XX% treatment. to specific to accelerators in $XXX quarter, children the quarter. receiving compared up of

capacity As reach several market. expect we navigated markets midyear, we strong the previously, to few in the high the We've rates enabling of momentum did. United supply our market constraint in last quarter, the we States, penetration through it potential strategic largest supply including and by told in you quarters, ample

to efforts and expect events allowed supply in stock Our successful to this normalize going chain forward. customers meet demand we QX,

physicians safety over in demand QX XX leadership of underscored from expansion with our than U.S., years skeletal the in continued strong treating solidifying is confidence more families in dysplasias. achondroplasia to our children by treating is Now market on Voxzogo's experience quarters. and and States in build and United We drive efficacy the the to intend coming in

rapidly base Our families efforts gaining momentum. seeking with to prescribers ar Voxzogo, our expand treatment of for

and Voxzogo significant product to support patient has to in grow Now markets, of efficacy penetration measured to for years it. expect strong U.S., albeit more only an a strategic of and achieved is continue extremely at we've outside than track record and safety with the rates markets more all the those approved X,XXX achondroplasia pace. in evidence

growth-related indication will set to pivotal for This build of statural experience into entry happen our with across care multitude the like have of to frequently. opportunity of build I Voxzogo lives standard us and the opportunity competitors direct none It's additional and my children having second any a thousands helping of brand that on study into conditions which sustain tremendous up the to continued the not an indications, four a facilitated of hypochondroplasia, expansion In horizon. does have on experience, forward look our leadership. and in our a impact

see quarter a across combined therapies. year-over-year starts timing We growth our driven orders primarily of in to market last the XX% by pleased the to was the Palyziq in new Turning was QX quarter-over-quarter growth XX% few driven now large year. patient the and were in quarter, to U.S. growth by dynamics compared enzyme of Naglazyme XX% in of

were there for on revenue for despite turning quarter. Italy, our driving updated hemophilia the encouraged and patient I'm with Roctavian, resourcing being outlined agreement to progress. number patient the with making to an the more continue some reimbursement approved of as have with $X we're Germany At We patients the results. green improved Germany, experience focus multiple Italy. are existing where in approximately our treatment Southern were with has million make also on price. [indiscernible], treatment expand our opening decision the strategy United yet Roctavian centers. We of finalized progress in which gain launching at drug those to treated And update, treatment more treatment subinsurers. One and U.S., to share centers shared believe that and we Roctavian pleased we progress government we to straightforward. In other had will last good the reduce an treatment of including access U.S. U.S. markets, of a light X [indiscernible] flow more signs and Roctavian and the that both rationale, and become based the the Roctavian States, commercial Alexander Now in successful in To this on reimbursed, markets, was is that

Now treatment quarter, we're encouraging patient in experience. and of seeing patients two demand advancing the access signs second Italy, in received there and

there's profitability. We make the company's able hemophilia believe our progress adjusting right are we prioritized markets, to enabling With Roctavian, significant As focus we the a patient and a more we our to to also focus commercial expenses with A, in markets. treat severe in viable be X site expenses three progress patients the encouraging to with press to commercial these prioritizing in markets access $XX today, related shared XXXX Roctavian forward to believe million on strategic while level. on is operating beginning the at that release approximately in facilitating a reduction this path level contributing

innovative results commercial XXXX profound drive So the quarter the the opportunity strong to by of I'm and led ahead remainder in the the expectations year excited conclusion, therapies. today's impact of guidance. patient increase full to prospects in our for with and

attention, I'll to So thank your now R&D update. over provide to and the you Hank Hank? for call an turn

operational you, The and year. were and development global assets on afternoon, plans across advancement has with corporate pipeline new prioritized great trials to plans health across the our in R&D shocks supporting the focused has Kristen, aligning new this as growth-related team for including expansion expected short deficiency with of leadership in closely indications Thank quarter. our approach second as been half on alignment our a syndrome, R&D the Turner authorities working well efficiencies, efficiencies in in very conditions, stature, more colleagues idiopathic pleased enrollment On with multiple during good new Voxzogo first to development resulted we programs and we everyone. Syndrome also strategy gain multiple to clinical [indiscernible] indications, these programs for begin development

Our registrational progressing hypochondroplasia pivotal also in well. study is

potential Kristen, conditions data Alexander We with has more I'm it of evidence excited and and of are enrollment generated. hypochondroplasia children a is in subject as Like about targeting Voxzogo growth approval number XXXX, the to rate thousands to for and results. treat

During from Society, [indiscernible] were Dr. new in syndrome. pleased at study Andrew we the see the to and Dover's short Endocrine quarter, stature Pediatric idiopathic data

of children mean For baseline from height year increased who AGV and a minus X.X X to the of completed X.X a standard standard from centimeter treatment standard minus X.X year XX centimeters deviations change to deviation deviations. months per mean X.X per

area and of see see pleased bone We lengthened, in evidence associated quarter conditions. treated we in regulator BH, Conference their at Bone children demonstrated of at XXXXXX for being continued at CNP been Children's significant consideration health-related International observed in cortical the quarter increases data new growth. quality is to remain in are [ after providing studies years to data children aspects Also Voxzogo potential growth-related an that for the durable ICC safety on improve pleased of among Phase particularly ] to conditions X health improvement on after clinical of that II proportionality hormone and bone Voxzogo-enabled study presented in follow-up study, from to that important growth their these the III and important of growth conducted as with growth the because they years a has with the who and growth achondroplasia, had showed therapy. This and received significant of life physical of as children approximately where this importance length are treatment. the formative demonstrated demonstrate to III effects with outcome has positive not which effective for II Also also an and analysis metacarpal -- functioning years in Voxzogo new is initiation and with master in up Health, bones all in linear children ages Phase first children consistent families. Phase it the X to investigator-led potential data XXXXXX Voxzogo XXXXXX of strong of the Phase

on are pleased the consistent that being life. illustrate safety growth, and with generated proportionality bone impact positive We efficacy and data quality of Voxzogo's

We are demonstrated with Voxzogo bring excited indications are children effective moving with programs in option X and advance to the conditions. to growth-related new safe and rapidly a these treatment development to

XXX, On muscular BMN treatment potential the we've of for of three to Day. our dystrophy; BMN programs advance, deficiency, antitrypsin quarter, accelerate XXX, alpha-X continue a CMP, share a for our I'll to all formulation of that were oral announced treatment more first that liver programs disease; Duchenne XXX update chosen BMN and long-acting and detailed of the Investor therapeutic last progress the at

resources our Finally, to development through as on development we hypertrophic time commercial for the and cardiomyopathy, BMN as will based XXX, well to discontinue gene therapy anticipated it and the landscape market. bring for

approach in update. I'll financial potential have development your now ensure possible of Thank for call Brian to our are our the that are impact focused turn attention. Brian? to now over applying We for the consistently for to investing the patients. we a that programs have greatest the evaluation you to assets

summarizing GAAP of growth year-over-year a demand Hank. [indiscernible] and to total full comprised strong XXXX, available expect million results revenues therapies quarter. currency file of quarter second The Aldurazyme, and second contributed representing Form driven revenue details today. In during Voxzogo. you, which Naglazyme, constant product the for by second refer basis, Please non-GAAP continued million, the Palyziq of XX% in quarterly $XXX All XXXX, we XX-Q, net upcoming to financial XX% Vimizim, Brineura record BioMarin enzyme of generated financial on $XXX including our second Thank to the will our XXXX measures. reconciliations quarter results of quarter on later be of today's for

at million closely of net $XXX Voxzogo represents year-over-year Looking product more XX% revenue, revenues growth. QX

of to occur anticipated, of of months We Voxzogo incremental which for a operations announce our planned, earlier our of early performance. now are demand Voxzogo's supply, the can all pleased of June, couple as global efforts driver $XX that and incremental to strong than earlier from to robust levels the the start customer availability stock certain relieved Voxzogo was The drove that we earlier normalize million plans would new patients satisfy benefited focused midyear, technical markets revenue. begin low while by Voxzogo revenues QX QX supply Voxzogo, supply believe customer constraint. We of than be always organization the incremental dynamic running to during enabled that a XXXX assumed anticipated this key and with constraint supply were to approximately

patients second rates patient growth Having in frames. exceeding this, in second of As new and dynamics the half time order hundreds expect over in dynamics, year, result rates growth of normalized could setting aside quarter-to-quarter revenue XXXX. the commercial we patient half revenue Voxzogo track broadly plan growth some from add longer these said the and growth we timing of of to

enzyme strong year-over-year the and quarter, markets government of million therapies XX% stable growing orders previously Our QX. in large continued albeit portfolio were that approximately expected drive to for select of performance with benefited growth $XX from in

impacted continue positively these by patient were will revenues we in sustainable observe QX that growth of brands large over the drive revenue growth commercial timing orders, While time. those solid to

expenses For to XXXX the are midpoint. second and Voxzogo strong billion between included the discontinued of Voxzogo the expenses approximately full development support expense last global primarily an year-over-year quarter the of $X.XX at due costs associated market support full million, the XXXX, up announced mostly the continued as million Voxzogo programs $XXX the driven and we expenses and therapies second restructuring representing $X.XXX year standpoint, were million wind-down revenue From well the by Voxzogo indication guidance year-over-year, expenses the as primarily of XX% quarter. work the to severance expansion growth as quarter were operating million expansion. allowing R&D $XX QX performance in development with during to year-over-year, mostly up full billion, $X year the million, in accelerate us incurred Restructuring $XXX to of raise opportunity. SG&A $XX the enzyme

is executing high well margin non-GAAP a of for XX% which believe very XXXX. operating watermark BioMarin in the for earning we XXXX, is quarter, a

XX% $XX the earnings QX was per XXXX. For example, increase over diluted non-GAAP normalizing compared XX%. have CAP diluted an $X.XX, just representing of QX would to of QX year. approximately was million QX been QX approximately of last in timing, share XX% operating for GAAP QX earnings margin share order margin growth non-GAAP per And $X.XX, operating

Our bottom the reflective underlying execution just resource order as and are operating allocation. revenue the QX disciplined demand expenses strong as benefited that timing line discussed driven from patient by results for well of QX our

While in we savings able our the discontinued in will discuss realize cost of details Investor were related QX anticipated, announced program including to transformation Day, earlier than at programs R&D cost lower April. some to we

Our our for half strong execution XXXX. profitability increased drove first guidance

We margin operating are XX% would to at expansion XXXX. midpoint, versus to increasing represent which, non-GAAP guidance XXXX the X% XX%,

share per to a earnings half to diluted goal XXXX note non-GAAP the guidance profitability as raised result quarter. is the is than of $X.XX the $X.XX we to profitability of weighted grow between strong that our consistent observe also with We first year core faster our to and revenues. Important

with expenses, We the annual of results as in to as our of given we are expecting occur QX revenue timing. And point business. of the in from guidance that full usual half previously our the differences measure the performance benefited being our continue and XXXX dynamics, QX growth well the in best overall our mentioned the expecting order half as are timing year second and the expenses year. to our Considering some as respect second and trend indicated first to of quarter-to-quarter second the growth to operating of operating the we profitability weighted overall guidance. half year

of million a X. as August $XXX on matured Lastly, convertible notes reminder, our

we cash on these QX earnings for call, we focus of strength settlement leverage our flow. on As our share and the a wanted discussed to the neutral outcome notes, of

that we for X anti-dilutive notes repaid the share the with per provides cash such, notes an going earnings the underlying in associated increased have As as shares forward. measure and retired million

and an Investor execution margin XXXX While at demonstrated have and we our by funding revenue capital. In balance that across are Roctavian a the this year's organization shareholder during our we us, our allocation to important trajectory improvement structure capital was maturity and communicate per strategy and capital valuable operating use plan in we debt we sheet to summary, the share. long-term with confidence this complete pleased of Day, our strong with on improvement behind quarter. increased into margin and income growth significant beyond earnings evidenced our decision further With determined operating

share to including driving you, details Day operational and cycle details strategy, new and provide also a capital operating the will with plans month. efficiency of and to pipeline for We financial at our are margins of targets, long-term next long-term corporate cost next our framework our we revenue remind including plans allocation To effectiveness our Investor plan on excited management, life management and several updates we share and you strategy. for years

open that Thank has the over We we to continued are share. growth Operator? support, now pleased been your last with compelling work we have story will done to the questions. believe and call very you a for few quarters the and your

first Our TD Phil from comes Instructions] with [Operator Nadeau Cowen. question

Roctavian stay Italy? the territories, U.S., bit you eliminating talk investment about the you're more or are where Germany bunch On strategy. in changes the aside regions to and going updated Can strategy changing the in of from you a a

for the much very question. Thanks

our we're for clarity to that So able -- pleased we're the Roctavian. provide very with

we share quarter. by would, a decision. remember, for We're able you the timing to Investor second we this pleased If Day, criteria at communicate said to making the be

of us and we to at we're mainly and approach level. activities pull-through which on what for the most on that have the United efforts focus Roctavian, important Germany focus geographically the So our seen patient site geographies: also the we've to those those within taking success is States, be allows learned to the three thing Italy geographies

geographically, from We're terms where within that's activity focusing geographies. of types focus also So in activities our but those to we're of perspective. R&D development also an going and

cycle life development We are activities. our stopping

we're generate our with manufacturing Roctavian. main focus long-term also Just current data, reducing and expenses to We'll indication. on continue associated to the

that of going focused allows in this envelope XXXX with our that time in to we're in to able Roctavian us achieve $XX million confidence underpins be operate for and strategy. profitability an to of All this

the And you to to to next think on a but the you'd of continue and in identify Roctavian. or strategy, give position a we the the It framework an a like when revision hear this further strategy? sounds success you're investment be update do going of strategy if is will the of strategy confirmation When necessary? initial

a this forward decision No, is going of Roctavian. for

update We as continue our said before, the [indiscernible] we've on patients progress metric is will success to treated. you we're all on focused

And those so but we'll update strategy. you our on a our reached is metrics, this progress on We've decision.

that a be confidence to going have contributor We profitability can Roctavian BioMarin's forward.

question next Richter Our with Goldman come Salveen Sachs.

situation that it to XXXB confidence giving give dynamics from want And parts understand you moving to Roctavian. you just just to Is this With coming it you to the just by in that point? regard at year-end in these where breakeven aspect? the the maybe improving demand are into dig U.S.? conclusion. Is are speak I time us all the help metrics could 'XX, further

Yes. so Kristen is Thank question. This you Hubbard. much for the

I operationally really X this treated we're in in we've that that what product, increase progress the are think seen patients ensuring this. quarter. sites encouraged And is that the a is the the United that infuse specific geographies fourfold we be in with seeing, the to is are the and X around I'll ready and that were States, really really, in

fourfold patient working are agreements really being a treated. to, seen be it's Alexander case single we've alluded just as the in and that ensure the then through pull-through. And this here, building since focused very know true about is But comfort January. also, increase around will team we commercial patients to that Our

a create that progress we of more are we've and January. U.S. doing Roctavian the that, in pleased pricing a really that Italy, particular, with type In and with only seeing progress we recognize forward in the we're receiving that and made with of reimbursement So since rapid that so, since experience, will moving in lot today. comfort

now as earlier, the as advocacy sites. agreement little of So reached lastly, we there. strong said on sub-insurer, that XX% patient have do that We large the then continue advocacy lives in there, of we've have a front. happy know approximately KOLs as with we and and from we're insurers experience We amounts to negotiated with Germany, progress report forward well treated have continue with push the we and with KOL of we and a Germany, want Germany, we couple are in a to sub seeing I there's bit in And will demand. patients to covered

So are more. and that I'll looking to Brian these of types we're the metrics at, pass for it

This is Brian.

be to to we of those across with that to manage Just Kristin's execution our the what those focused gives envelope and addition continued in this us progress to markets cost this signs add is million comments, strategy that asset next X plus profitable $XX year. can confidence

with next Our question comes from Ellie Merle UBS.

any move XXX of a just then Just XXX second across different Voxzogo? Voxzogo idiopathic in And terms would you indications. forward instead of on relative of there development where XXX, Are stature. indication how short are you the thinking to question about with

patients focus that to the your are XXX,XXX with potentially of what Given you've prevalence, that prevalence on plans much larger a ISS subset cited? within

is Hank. Ellie, this

XXX, for will thinking initial of which path or to clinic, proceed the the first our superior to As approval, to get it all, shortly. superior plans far the with into thereafter And most leveraging development convenience expedited efficacy, is, both. potential as for start

with there in patients stature specifically current the or need, therapy. patients As stature, the have been is deficiency available the more have unsatisfied severe greatest to short program patients with who idiopathic who regards opportunity potentially unmet of

addresses that your I hope So question.

question... next Our

those the more of we're and achieve to share plans Day, also our TAMs expecting at information on Investor development be development we'll to with studies we'll the for sharing the design Ellie, Voxzogo programs. be able the around specifics

from Our Cory next Kasimov question with comes Evercore.

question Great. I a Voxzogo. on X-part have

First of picture. kind big

multi-indication motivation the assuming treatment the the adding of the proceed out in relates to how orphan opportunity. the kind of is, it's to comes additional current different patients similar, about get market on how then maybe when to through you're potential your Curious to thinking III of indication as products about what your So on terms for Phase this expectations the the are And hypochondroplasia in Washington IRA impact we here, one indications the think achondroplasia diseases? successful it level second and of you as part of of trial? -- awareness we're

Thanks then very one. one Kristen for to hand first the over much handle questions. your for I'll second the and

regard important areas. the to many IRA ] in there'll have legislation don't Voxzogo actually for where So impacting exposure for whilst one minimal question. and impact that we impact payer cycle think the minimal the to with very of We be because factor from a IRA innovation terms the hand We to Voxzogo, And life these indications we of a going of of potential now Hank to significant overall the is Voxzogo development thank IRA. I'll a plans, [ standpoint in with have impact first the see on for significant think second Medicare. over mix it's on industry that's

Right. real And your question, second fast? me remind just Corey,

Yes. difference or of differences market between And treatment? awareness terms these level in the patients [indiscernible] the or of of hypochon similarities achondroplasia in

as doesn't diagnostic early for life in purposes. hypochondroplasia occur Yes,

development I'll lot of lot quite So more the more we're that, with during to as population. -- of try diagnosis more its of there's an more we're about effort conditions, But progress increase increasing and once give of try in a in pleased undertaken, it's feeder a a underdiagnosed, the diagnostic to efforts and program, uptake really ambition awareness rate begin process genetic update effort undertaken we're place, said study, of and diagnostic then the with the the hypochondroplasia hoping undertaking ultimately once that. diagnosis. therapy there's to once you to the there's of Having is

from Next JPMorgan. with Fye question Jessica comes

the the landscape competitive maybe potentially evolution the in [indiscernible] we in horizon? thinking consider in And as would combination a How think ever achondroplasia on about are of about hormone and when infigratinib evaluating you with follow-up, Voxzogo achondroplasia? growth you

Thanks very your much Jessica. for question,

So confident in profile feel our in very the and we competitive overall, indication. subsequent about achondroplasia

even now we youngest well of to CNP to optimal indications movement patient X we're very the the treat early achondroplasia, the after the to also X know, outcomes. growth. impact achondroplasia is infants And the outcomes growth, in is important X population, child's start you terms on pathway different The with health as pursuing very, amenable the that treatment, all of As very with which greatest as but possible think in part achondroplasia.

subsequent find we're So we profile will the question good And safety have to going answer we very, position be combination I achondroplasia. of durable Hank obviously, then the the second a it regard in really feel we over your that's think The think With we indications efficacy, very lead compelling to very the where about of approach. achondroplasia. in to to competitive. possibility hand the

that potentially stature average the have of of part strategy focused efficacy, be drug, the superior XX% as two about And efficacy, be And superior if to would virtue use growth to a potentially further advantages even population. would more offering of restores ability for XXX we [indiscernible] optimize to it not convenience. Voxzogo three by drugs. on one for

So we it can think franchise do that ultimately all. CMP

next Matteis question with comes from Paul Our Stifel.

Great.

specialist been what the coming adds population, driven curious, growth hearing anecdotally, care leaders of the that a incremental percent referrals from are be opinion the I you here from think in treated key leg And of say leg are next by I patient and today. primary As diagnosis. we've was that about because untreated the growth some next the might Voxzogo, for this of by

effort So the the patients speak from maybe effort What's kind -- give can next and the the last there next for incremental of some perspective. of XXX? versus you XXX leg just to context a marketing

efforts exact question, where such will the numbers will I to, the for actual per say, Thanks, know [indiscernible] but Yes. the referring treaters I our are Paul. say focused. don't I you're

So States, where us. where going there's we see an care. do is This really a we for growth opportunity be instance, more large United decentralized for area in believe to the

And certainly of what also what mean as by I pediatricians. you that it and we're some fact, patients are well. in geneticists is endocrinologists But some a these is some have with so treated finding pediatric by offices sitting general lot in

in pediatric peed that know on that sure be. may making in general population And endocrinology and around Voxzogo ensuring so we're have what we're is local really a treater, offices namely that they your we awareness focused

really we trying those patients going are who treated. be to connection between those know we're be build So points to to those might where

this and where system are area skeletal dysplasias. see more in U.S., centralized they Now we specialize centers care so do a much that in of in treated ex in there

those you and we've countries. as areas know penetration them. So of where those focused to -- in really where get course, uptake in we've while they good importantly, grow, are we're seen to patients faster And how room well. those still seen There's that's and where

pattern up in importantly, that penetrated kind continuing new we've yet -- areas lower we penetration up that referral I'd focus where have highly of our going are growth might our the opened strategic opening in U.S. in those market. So we markets and be not really to markets or the around say

on So areas. those focus all

Partners. Our with next Joseph question Leerink from Schwartz comes

I'll priority of pipeline your a ask about couple programs.

I give to are some several First, what also it's for would to see market was you for long-acting XXX. the XXX, target then hope the moving levels? And dose you insight dose us to technology envision BMN into different next-gen used there. at time to XXX you us that's could at the other on hoping be BMN help what or And it BMN hoping efficacy behind hear helpful see since doses wise expected [indiscernible]

into far information already bit in of tried terms to safety little XXX as I'll of that's drive or high program the sustained chemistry. detail really to on is about exposure at a a one lot the in we circles. thoroughly more linking other and specifics available get that is leverage lot a As sometimes ensure Investor Obviously, without Day. referred aspects of to of in is of delivery critical exposure the dumping design,

doesn't orientation it able acknowledging So a of seem antibodies. really but bit either On by into I'll like XXX, is get dystrophin really CAG exposure. with with consistency a lot receptor safety to big [indiscernible] that high around a details, and levels anybody little or of of there more to are Polymorfelino competitors, get conjugating

person. diaphragm on been of second XXX. that and molecule different has we of data the making updates in at I'll miles. site that experience been approach where not has have status advantage do levels given about what expression can that virtue to been program Investor a that encouraged when processing. is our skeletal by confidence XX% but [indiscernible] profile, The develop is to at levels so we're regulatory the we to to a preclinical up also expectations has of by level and can with high to just get pre-mRNA muscle, we're our detailed give Day, dystrophin all potent very high more the in and targeting the in a more safety try along, drives We in And strategy see in twofold: progress but going One you're

Our from Akash next question comes Tewari Jefferies. with

This on is Akash. [indiscernible] for

DMD, XXX normal about you should high data normal well BioMarin bar to it perfect in in expression to that animal comes need as [indiscernible] models, I the to from how mice what forward. does as think on models guess for for we human you also would when dystrophin And XX%. to how success, translation translate dystrophin. humans order showed So move

Yes. really terms Well, humans the there that moved the of inspection. a is translation in in I that's think challenges of needle of lot one hasn't dystrophin been

is person, it of carries of expression be of animal animal tissue unique about and question gene dystrophin What of where achieve level Day if R&D the on our we relatively bit the So at we was human can or equivalent TBD. based partly to we drives should on a higher last based very a model year's XXX in a a that that which little that much we achieve the be concentrations skipped. model, talked we is

on And of assessment vivo of class have skipping fulsome safety data, we've a compounds. our humans [indiscernible] both potential. got very And in a we so lot from

it's optimistic. that expression. really meaningful that dystrophin the if full-length the And near we're will enable dystrophin you much of greater be on we'll motorizes much so levels that muscle, Based able of the that, will, see function And to more of muscle. course,

far, And what XXX. achieved. been looking achieve to that's that hasn't And we're with so

from Piper question Sandler. next comes Our Chris Raymond with

two later hypochondroplasia, to comments diagnosed questions. in on maybe listening how your maybe Voxzogo life. is hypochondroplasia just Just Hank, just in

pipeline just us differentiation XXX here you just of of call points wondering that you that pivotal a if that walk be in diagnostic hypo label earlier program. back X noticing what just to expect journey is gives patients maybe the XX, study of once molecule. also achondroplasia? oral -- question had AAT you sort in Just that XXX, through another any the approved? for to at I'm corrector should different aged Or discontinue are won't competitor go between among age the And could their you maybe we and includes then have And confidence noting comparatively.

during odyssey Sure start delay. and diseases our we in challenge genetic our improve teach of conditions. can that diagnostic colleagues Chris. and thing, oftentimes those outcomes we overall in is The And the program, hope that is path the early barriers really the some development genetic diagnosis to overcome to

health confidence Voxzogo at approved children. essentially to X As efficacy back thing a of population, achondroplasia, from of X the further who children with say recognize we've world to is, platform ran of world age develop a under safety very even regulatory that's under requirements in of for around in The the authorities I'd very years the good got the in wind young and Voxzogo infancy one TBD. the on a our lot that's about that

how how and unfold, for is unfold. to tuned will stay might regulatory how going the proceedings Odyssey So unfold program the diagnostic clinical the

Brineura As the Alexander population. we got said earlier, -- got young approved for we very just

So and I earlier agencies now Voxzogo the familiar and are of obtaining confidence can strategy think and with BioMarin only quite efficacy safety earlier the in help rising the the us. approvals

simultaneously. not of the very more antitrypsin. the restore Vertex, far of than maybe think, was and couple a currently Vertex's we on now to two What preclinically, I the stop And liver stars don't had decision, further to they've so actually, it's have gain know. And activity focus X at that problems. single-minded succeeded X And much bites which to two that the alpha-X do a prevent big, regards different big they'll XXX recently very As is is apple. problem undertaken. shooting been untreated traction, disease. which approaches [indiscernible] and I other just causes most was have to difficult the kind One of was things polymerization, contrast announced and for would to

liver, having is patients mutant proteins know, is that is you is in mutations. we restore function the the so different The what therapy preclinically we polymerization protein and reduce liver which and can consequence. replacement that we've have M unaddressed that which the lung product exclusions who a for deliverable. that there's gain [indiscernible] then causes more can protein that broadly antitryptic in its a loss all the can in potentially problem is related of oral their currently the medically. of that's of in shown addressing these much Z -- that result copy of health and genome advantage Doing polymers, that the as -- activity. better we polymerize The product competitive the therefore, but the As alpha-X an the one result disease, problem, of is of increase molecular also mutation, and landscape unaffected function and solubilize mutant liver the only we're titratable to has preserve and and therefore, a of

So further making we're update on we're Investor progress XXX Day. very I'll that at you again, the about program. excited And

with from Wang Gena comes Our Barclays. next question

have Roctavian. regarding few will questions a I

I the the now? that in different And these past and is when expect discussion? to comments of fair the now -- is the on And assume cover you about your cover be didn't then XX assumption many the be process insurance, to is that XX% sites? your from were XX, Given you will treated patients what's in regarding X of How have place. do will they one phase in treated U.S., That to actually breakeven? contracting order XXXX that Germany. reason patients XXXX, question the it in In why payer make X

rent question. [indiscernible] And or next cost then that your I'll This of important in patient getting revenue is the Roctavian first specific progress start launch this we're covered goal today. to earlier. and not envelope We're profitability. talk Brian. was guidance the the with year It about giving for seeing to

specifics but So as, that of level at the if again, you that revenues, you it in view as this can of demonstration into so further will, time. up strategy, framing articulation not minimum getting and way

Yes. And I'll take the second questions. two

making impossible can having. we're but are just to pushing that to [indiscernible], time mean, through we So And that by making really, I going an discussions sure addressing of and are it, you I the the it's with about course, progress which, we're to in say insurer regard exact be that needs, we're these risk give of insurer carrying.

the that you They On actually the separate have and those had about We've three side, the been we're U.S. HTCs in U.S. treated. all HTCs across asked patients treated. dispersed geographically were

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

that Roctavian to A. the hemophilia strong B One How on approved hemophilia Xx XXXX was based depopulation the first half on revenues generated the to Congrats and $XX smaller is on in are benchmarking in than estimations. the of our $XX million quarter. versus you the which Hemgenix, about million X acknowledging Roctavian us that hemophilia [indiscernible] from in months U.S. question before X October

been Yes. Kostas. way. in Thanks one, tracking launches. the between different the question, there that for There's launch I'll Brian, along the I've Hemegenic circumstances since the jump X on

access but team Of there's more through qualitative well, in benchmarking as Again, course, going not challenges. of gets comparisons are getting contracting launch estimate. we're differences into similar we or a

be a quantitative don't we is level. comparison but do made, there So to that

from question next comes with Our Wells Mohit Bansal Fargo.

Just Voxzogo the want to reconcile some numbers. of

be quite inventory, correctly, inventory, for year. the lot half. number if mean, So assume Brian, was to X,XXX inventory right? of number in a patients I if all was is patients I end this I That the $XX understand think the first added about million I by time. the of And that one you

XX% is in since reflecting increase the that not year. last growth X,XXX, the revenue at that is are you Now

there understand in we or play dynamic to dynamic about? pricing wanted a just is should is that know So that geographical if mix

where to increased in and you Thanks question, might patient Yes. the of the to there prepared tried necessarily growth. other that but revenue point you I for I supply last revenues there around. flipped to dynamics the functions be adds, that a terms time, the were quarter see are quarter-to-quarter second order that remarks, didn't noted, the earlier, bit different of way was And around time becoming you timing in in because even from this dynamic a And to rates going Mohit. rates. but that thanks available recognized that also launch. XX% third just think to see And executing timing the revenue significant But to patients to rates two this address long billion amount growth a growth of in that quarter here of revenue revenue. be than There's the growth $X bit for ahead and picture, in will orders Yes, differences or Voxzogo differences. be QX. correlate timing penetrated growth in still exceed less quarter-to-quarter. of QX go way year globally. a We're we're out We're to half, going we're second rates there of patients patient we those a in over could on -- going mean, we we're But big there's quarter-to-quarter exceed time. the track this and with I either in

Brayer Your next with question Fitzgerald. comes from Olivia Cantor

forward. consider are U.S. expect and that next to profitability Roctavian, the geographies year the up you of about going make As sales breakdown for broader trying across contribution X the really whether I'm to how you're on? uptake you thinking just sales of you sense just majority Roctavian a get and focused

revenue in average be question. per is of good U.S. dynamic. the there of in ex U.S. a U.S. Noteworthy price to terms Roctavian It's patient just the higher. versus going The on

focused to with on the going to to that these restructured cost It's as exclusively the unit the the the U.S. the as portfolio. revenue. Roctavian. focused working those contribute progress of well envelope Again, markets, three focus allow continue dedicated they as on business in market, more and are of make this to the So be will in tactics business going patients we're on rest both remainder

of So X the are area time. X to markets getting this will together, be each further into those markets the we're revenue to important and not at XXXX. of viewing those markets individual in that But that this focus contributors as details know the

This will be our last question.

comes question Luca from RBC with Issi last Our Capital.

you for net the geographies the maybe $XXX,XXX still find million from for [indiscernible], have last the where U.S. the Italy in rights? other in in Is the the of prioritize and to believe today partner that net only [indiscernible]. in pricing try a pricing U.S., pricing right? Can on versus you I sticking two monetize versus capacity? maybe about in to in you and how to Italy, in quick obviously would reported $X.X Is Germany? Congrats patients the plan Italy $XXX,XXX And plan U.S. just that $X.X what's any patients in should but million or U.S. Italy. the you you treated there, about any there [indiscernible] quarter, Germany Maybe there appreciated. Any and progress. ones we And imply X much color still that talk think

It's Brian. I'll that take one.

all, we of $XX in are element first not means important question the markets focus, terms driver So focusing the cost year million regulatory of structure commitments at investing the as key entirely clinical of in well the the supporting with those is because that long-term that patients an next behind and it's for in come X markets overall additional this Roctavian that as on time.

the prove on to markets time. will going patients where important the traditional with had we And dynamic rebates regions to that over get it's Italy. and time, and the make revenue I'll The communications. that discounting then just pricing, again, progress for to are commercially consistent that and we recognized traction over time gain over out right the be item gross X value X the with Roctavian's largest As overall across it is was continue and XXX net pricing. our expectations two planned retain those to we is variations QX global those in time. select we XX% investments share at in accretive I the share, in But assets net to sales U.S. in note in will this to that prior at confidence

stand comments by that. on prior I'll So

will turn for CEO, call back now to Alexander closing Hardy, I the remarks.

priority quarter and much, of strong your this that in both profitability for BioMarin quarter for call you and our our the and raising and Voxzogo about joining Thank outlook line. we're guidance. and achieved and was in the very to And revenue on the thank all questions. and excited very We're both of opportunities. we've everybody, bottom so growth, about we has you the proud very the set very made accelerate January. this top those priorities: far the prices also maximize the A year with excited the reminder progress out One

You Voxzogo can see with these strong results.

those, just we're made forward and very, XX% growing first well. have so progress and terms In with in development very path additional is its and in both regulatory feedback we and we in indications, progress in indication than penetrated this far a that, the less of X programs. year

our Secondly, million we're for frame. strategy, to establish portfolio. XXXX the have clarity this we're be Roctavian we expenditure seeing the We're Roctavian reducing today, to within in signs profitable in in the decision, down our also and but that a to progress, with confident product of this And the of in in $XX time forward on path addition excited opportunity. confident by ability

fourthly, creation made we're programs focus for excited around prioritized that year, can increasing don't this have bar shareholders value X of progress so R&D see than about, revenue. we've high proud decisions for meet BioMarin. as and we consequential efforts. and far our and we Thirdly, We faster that programs And that we made most profitability innovation stopping you on at excited the the we're really on

with that quarter increase our see guidance. this in results can and You our

to our future So It's we're you share at going to for very, share. very forward the a perspective all exciting BioMarin. Day outlook to looking seeing Investor we're

your all plans confidence We to good and day. have And a us, that you online strategy. thank in our rest looking forward lot wish for with seeing much in that, you and you or joining York, BioMarin. New we're seeing very a And corporate for of live of either

conference. today's concludes This

You disconnect. may now