BMRN - Earnings call transcript Q4 2024 | BioMarin Pharmaceutical Inc.

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the BioMarin Pharmaceuticals Fourth Quarter and Full Year 2024 Conference Call. [Operator Instructions] As a reminder, today's call is being recorded. I will now hand today's call over to Traci McCarty, Group Vice President, Investor Relations. Please go ahead.

Traci McCarty

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

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

Alexander from Hubbard, Chief Hardy, call today Officer. Brian and R&D Commercial and BioMarin the Greg Officer; management Officer; Financial Chief Executive On Chief President Cristin are Mueller, Officer; Friberg, Chief

over call CEO, and President turn Alexander BioMarin's now to will the I Hardy.

Alexander Hardy

for for and joining Thank fourth today everyone. XXXX you us you, Traci, results good Thank afternoon, quarter our update.

most a impact BioMarin's we and value be amplifying patients performance realized, pleased making serve. creating to to profound see and shareholders, our We strategic new the starting vision, are on for financial importantly,

outlook, need a promising BioMarin's ambitious of mind, the to introduced prioritization give include the these In reorganization centered our positive units a growth model initiatives $XXX and are of initiatives now realization results. our already pipeline most we specific producing cost and achieve million program. ongoing long-term business around XXXX transformation us of the throughout operating The framework candidates,

growth Starting performance. with financial our XXXX record a year and profitability. of was

pleased our reinvestment top that over grew XXXX earnings innovation margin year the are further growth. points, in exceeded strength to items. puts Year-over-year, XX%. operating ongoing XX%. financial expectations and our in expanded share This Non-GAAP per path XXXX diluted double-digit non-GAAP increased across and We line full XXX revenue us and share market to on enables basis guided growth

skeletal data VOXZOGO is hypochondroplasia well to plans expansion expand supported which should across with therapies, BioMarin's as and CAGR as conditions us increasingly year-over-year confidence additional global Strengthened X% portfolio. XX% confidence unit. on business at be billion year-over-year now in Our outlook XXXX conditions supportive. $X into quarter high in anchored for annual our profitable revenues X approaching enzyme of this the by to commercial Leadership long-term achondroplasia is grew skeletal fourth single-digit gives from our growing

address. We strategic provide prioritization last year to CNP, the treatment results later conditions in that data this look highly long-acting The dystrophy clinical for forward XXX differentiated options XXX for they candidates X a BMN both muscular resulted Duchenne the advancement candidates. of may BMN for pipeline year early our

unrestricted even make an For fee response Phase PALYNZIQ progress and shareholders. our we're BioMarin. made by at be excited allowing us sustained potential well and the patients, The our greater tangible and operational diet, the provide to year, making our an yielding with for summary, we're employees for impact results and decisions plan significant data from opportunity to XX-year-olds our adolescents. In last supportive. strategic expansion study results should to year, III age we This XX- represents as share as to

world strategy, continue growth ahead of from our and double-digit revenue contributions We year. programs towards the business Thank at employees our last progress advancing you the journey around to Investor X attention. I execute global data share XXXX. Day in make like clinical for look in our would drive development your delivering in outlook we we XXXX, Looking forward XXXX. will thank long-term their on expect together mid- to provided to financial our the

overview I turn now Brian to provide the over to will call for our the financial highlights an of quarter.

Brian Mueller

available full Please results, XXXX to to file measures. XXXX release upcoming in non-GAAP fourth today's will financial in GAAP we Thank coming press Alexander. detailed you, XX-K, to Form which reconciliations results year of refer for including quarter be All expect the days. our and

significant in of transformation. are a especially XXXX, execution during pleased We with year very BioMarin's

fourth consistent quarter to Full record increases marketed growth revenues in Double-digit from full to for BioMarin's XXXX. revenues strong billion a the Also totaled the across results increased to with quarter year were XXXX contributing Fourth and $X.XX and year. stage XX% the year-over-year set in billion therapy million. brands. results the VOXZOGO $XXX XX% contributor grew versus year in increase growth total enzyme for to record full the full $X.X over XXXX, XXXX, business a year, XX%

Moving expenses. to

expense non-GAAP due development XXXX, of in X VOXZOGO was was the the discontinued spending compared full XXXX, program. new indications to for full as to the reduction slightly the fourth year to by programs during deprioritized spend quarter. on realized fourth lower non-GAAP quarter R&D higher the fully the in offset For year expense In XXXX and same quarter a was R&D XXXX, primarily compared million in of impact of $XXX

BMN increase in and our indication XXX as that XXXX expense up R&D expect We expansion we studies. ramp VOXZOGO XXX, BMN will

with a by lower primarily SG&A expense expenses For implementation, both year-over-year, full and due costs and partially offset year the our Roctavian spending. VOXZOGO XXXX, fourth reserve, of ERP associated commercialization quarter to higher bad debt non-GAAP increased

to Moving profitability.

impact positive BioMarin's ongoing cost of year. the last the performance, revenue of with outlined pleased are with program transformation We coupled strong our progress

operating by lower growth. from R&D Fourth operating related cost of of for non-GAAP increase benefit year leverage a margin non-GAAP programs points transformation XX.X% and full strong was full margin XXXX, to initiatives, the quarter an boosted percentage spend As of result the the reached our year this discontinued momentum, XX.X% profitability X.X versus XXXX. from the revenue

XXXX, in of growth achieved its meaningful BioMarin and revenue objective significant profitability XXXX to levels. addition at growth In sustainable accelerating

achieve non-GAAP share increasing our BioMarin's our priority top Our is positive XX% million our $XXX development diluted XXX% operating of increase XXXX flow flows year non-GAAP earnings full and internal BioMarin's of QX share full further cash earnings investing the $X.XX, the and to development cash research diluted puts allocation on XXXX. billion enabler $X.XX target $X.XX. than flow flow of per to cash generating increased a through of through over business long-term year significant key and capital to full growth, in with of increased both generate and external track also per profitability innovation cash BioMarin year a XX% operating levels ability XXXX. of meaningful to strategy. greater in revenue our investments operating our is for

outlook to our full Turning XXXX year total XXXX and revenues.

the and We business year-over-year expect And to between BioMarin VIMIZIM, strong second of translates XX% billion XXXX. In as in business the to third growth demonstrated growth majority contributions to the unusually from order quarter. the in $X.X in in expect total enzyme increased as year-over-year and base benefited growth rates for the unique dynamics, PALYNZIQ which strong is compared growth NAGLAZYME year billion the high additions from $X.X ALDURAZYME XXXX, of BRINEURA. new expect of of year revenue, the growth revenue expected revenue half and very in first we the year. we half timing. in half In Similarly, NAGLAZYME and of growth the patient well represents an second Due from ALDURAZYME therapies as VOXZOGO, benefited XXXX, lower the these XXXX.

total for target in keep $X VOXZOGO strong of of XXXX the and $XXX performance will our total XXXX. growth million Importantly, contribute track on $XXX estimate expected achieving in it is BioMarin's contributor XXXX be to revenue growth, million total the revenue revenues we year between XXXX full and and to revenue. billion a expected XXXX us

only overall expectations provide not these insights align with are we the framework total to to your guidance changing structure revenue, of directional ours. a are guiding meant While our

operating non-GAAP for margin the full XXXX. to Moving year

expansion XX.X%. operating are between and to represents points the XX%, We the XXXX guiding of of at percentage X.X versus non-GAAP margin XX% which midpoint

Our guidance with transformation the along initiatives. revenue continued by ongoing impact cost the growth, of strong is supported

important While important our critical XXXX year long-term an enable to on marketing non-GAAP profile. activities and also XX% with next target year. sales in achievement on noting an the of activities represents the for our of Based investment operating this pipeline these in that stepping stone is implementation our growth margin prioritized continued XXXX,

benefit which rate Xx driving per $X.XX, year Finally, expect we top XXXX, for the XXXX BioMarin. and full expect Building for non-GAAP for share, high growth XXXX, growth share expected corporate BioMarin's from at continued $X.XX execution the strategy revamped towards another and as beyond. per year performance earnings our of over XXXX, profitability in represents a model in leverage we midpoint, on we strong diluted line between operating

pass now the of Cristin Cristin? discuss the to commercial call to our will drivers performance. I

Cristin Hubbard

build strong of XXXX team momentum. and increasing growth global led XXXX, this continued PALYNZIQ for by to Thank VOXZOGO, and in delivered expect The products, on enzyme . we you, demand Brian. expansion penetration the replacement

actual and results States, X-year-old adoption in international the prescriber significant and age and commercial children. eligible increased U.S. global Germany, to significant age. infants X XXXX. VOXZOGO cohort In In we today, we strong leveraging BioMarin's provide prior to and from the a by Consistent as providing markets throughout U.S. for significant growth expansion quarters, total insight into in of infants or for in years the XX% of on were starts driven potential, deeply young VOXZOGO globally, In drove focused U.S. young To were OUS. with with the and demand in new such the strong the United new robust in families expanding contributions awareness early infrastructure the year between dynamics, of demand the and patient children starts Record global of patient footprint revenues from with these penetrated growth drive patient year-over-year children and the we under saw with outside X- base XXXX. diagnosis also full we're number percentage of the split population. markets, growth combined VOXZOGO are majority In population. the markets the and treatment incident year-over-year

of revenue from total XXXX, For and million, U.S. VOXZOGO from U.S. $XXX outside countries of full all the XX% was the XX% year was

from of million, U.S. XX% fourth total was XXXX and XX% the was quarter VOXZOGO from For $XXX of OUS. revenue

we Japan, on and growth and efficacy based Now the of and continue on moving in strategies our and In and the we from of and to population from VOXZOGO's our benefit. infancy States, diagnosis forward building look U.S. in realizing infants growing treatment strategic demand children ahead and momentum base prescriber younger focus to markets, Outside opportunity on incident proven of maximum the families XXXX. treatment therapeutic continued to VOXZOGO such to the expansion United the with to Germany will larger drive profile. with as provide early safety

infants countries other access treatment. penetrate not than turning and already expansion to Now countries growth these to treatment VOXZOGO is improvement intend for we X published untreated field arm initiatives confidence the achondroplasia. in young to where introduction issue we an recommend efforts, are the awareness We additional commercialization by of countries. early patient efforts, focused deeply diagnosis, but highlighting achondroplasia VOXZOGO to in referral year. we platforms see VOXZOGO a VOXZOGO raise pleased as investing VOXZOGO were publications and bases, early significant international and importance proportionality treatment. independently personnel starts growth follow-up. after early and of XXXX, of limited Also, new and is to to strategies our with new the in only By prescriber December benefit choosing the reach increasing XX to that added Further To children facilitate to are VOXZOGO more enhance published X Recently with provide to Med, in children facilitate for and more demonstrated families to, plans, years to control markets. treatment. of clinical maximum increased this followed guidelines adoption are for includes on are These guidelines achondroplasia international the of very versus created supporting treatment statistically broaden VOXZOGO in when including, available have

for same in to medical will and those should be the the increase that ready and achondroplasia. for strong will of approved so growth approval. are upon we raise time, record in are team III confident experience VOXZOGO treatment prepare hypochondroplasia The awareness treatment for the this are children execution we to supportive. the of interested be of another with achondroplasia VOXZOGO, for launch treatment We be the VOXZOGO and to data year Phase XXXX potential creating using commercial diagnosis only infants of provide programs and our hypochondroplasia, At to

over moving Global ongoing Double-digit products. to the Now marketed BioMarin's substantial Japan. strong and result growth therapies. in expansion drove results demand in of uptake quarter was our enzyme in the U.S. PALYNZIQ all across of the revenue patient

therapy. we the NAGLAZYME, quarter, and saw growth continuity efforts new in and during significant year-over-year addition, our to for In enabled VIMIZIM by maintain find BRINEURA those on ongoing patients treatment

make and new starting our patients We in enzyme finding to replacement continue progress with therapies.

programs where expanded to have in diagnose certain we we and panel testing MPS seen help considerable success with regions gene instance, For patients have our CLNX.

expect an into we to over coming the resulted have initiatives example, quarters. increase and countries in multiple out Brazil, For new diagnoses, programs roll these these in in

highly PALYNZIQ across and treatment PKU new that by has reinitiation and all Now of with works it profile phenotypes, adults this been moving patient to PKU. growth differentiated driven from with starts

deliver this We fee that the are midyear, data also With potentially in year. the we in diet. adolescents an forward of with that enzyme applications our Phase can have and normal half the to coming access look may in only III U.S. to submitting second soon PKU substitute Europe therapy unrestricted excited

higher pleased conclusion, the already to during accountability of In resulting very across transition we of the a benefits am The execution performance, are I business and organization. in the unit model. this and XXXX is operating seeing transformation in defined year with team's focus

XXXX set the performance of in target have on greater achieve total We our of billion are for revenues in course and stage to $X XXXX.

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

Gregory Friberg

across than Recall nonhuman pipeline. Thank that higher great Starting those pre-CNP you, with demonstrated exposures for in fold our making we BMN long-acting Cristin. achieved sustained We're progress CNP several primates, other XXX, agents.

therefore, greater deliver acceptable XXX goal an is, to leverage while for superior Our this potential maintaining profile. BMN for efficacy exposure to safety

well presented look half PK in As line year, data at underway, is and with forward our today, the of study the in to from study volunteers congress XXXX. data first a second the top of be detailed healthy in to the half sharing of we scientific

For kilogram dosing Duchenne's dystrophy, BMN in with Phase study enrollment in our XXX the X-milligram I/II per and muscular is cohort. advancing

for the us are rapidly this kilogram potential in pivotal treated as complete shared, data of we give believe data clear With eagerly cohort. could per that We CANOPY boys half XX% be sight available XX-week VOXZOGO proof-of-concept additional at to we achievable hypochondroplasia from cohort, X which data second scientific state year. continue our in approval X-milligram X-milligram the these be in we skeletal to per XX-week levels study half our previously in the As steady program this were the will assuming awaiting to whether conditions, will track the in level, and expect the XXXX at is data programs, kilogram clinical arms of of will treatment the biopsy the we of present our in of in this advance supportive. to on dystrophin enrollment to line XXXX, recruiting, a target are III Phase remain study and to first Pivotal come the XXXX. and congress

idiopathic SHOX Noonan another in one enrolling deficiency II studies, encompassing Phase short X and Turner syndrome, patients. syndrome Our and CANOPY stature screening and are

PALYNZIQ. to Moving

Our even second XX reduction the this PKU early afford across filings and an Phase data patients XX U.S. for PALYNZIQ believe half potential restrictions to readout unrestricted in filing and adolescents dietary We to a phenotypes, transition thereby diet. for Recall, and patients for their supporting that living. independent European supplemental year. to is of support in on study potent and aged delivering all an families allow phenylalanine PKU some of manage could challenge for the works track can age, smoother from III the to adult the adolescents,

oral of we volunteer portion are dose liver well, XXX the alpha-X first for with healthy in having ascending the and cohort antitrypsin BMN deficiency-associated dosed study. therapeutic disease, multiple Finally, the progressing

Following to last we progress organization look pipeline, strategic most its prioritization year's in and hitting advancing quarters. stride forward the you updated keeping coming the the high-impact really is our on R&D our of candidates,

now your for support, to call your Thank you questions. continued and the open we Operator?

Operator

[Operator the Instructions] Your with question first from is Philip TD line Nadeau Cowen. of

Philip Nadeau

reimbursement decelerating. million that We was you want up healthy, they're be will pushes to to which to generally $XXX is in where expected year? but on year-over-year, or pulls what coming and guidance, better maybe to territories come where it you to on And currently performance and the new short? X expect guidance on from? fall the could more cause a be VOXZOGO the talk bit growth And what it on and more be reimbursed certainly particularly could this cause Can that's

Brian Mueller

Phil, this I'll color. for is provide question. start Thanks and then ask to more some Brian. Cristin the

that you scale at terms VOXZOGO sustainable pointed to absolute global at on in as to here. billion growth. this what but which So some here revenue numbers growth. was observing grew gets increasing play close $XXX close large to we're overall strong $X.XX not base. million there's dollar of revenue in guide, still out VOXZOGO XX% the accurately of XXXX, impressive, that It's

are on XX% the And XX% $X growth the the the year, be plus and that decrease growth the time track may again, through billion by XXXX CAGR. compound in with XX% low over the will greater that rate for growth base both we and of implied growth today in as provided what we going last the forward, we're that targeting -- as increases, continued guidance multiyear greater than for XX%. range is Just a reminder long-term XX% conditions annual rate skeletal than VOXZOGO

Cristin Hubbard

so Phil. that. add Thanks to maybe for Yes, the much I'll question,

our So growth growth. from, anticipate sense And drive dig and there that a markets, to you doing as largest seen to bit where strong geographies continued of growth our coming single of and I'll we're give international beyond. standout a in it, call the certainly growth highly around do into I is and we're our XXXX well XXXX seeing we globe. continued would much into Germany what the We've U.S. of also in the a being in markets penetrated clear and I'll say, opportunity, the market little

One of XXXX. the bigger drivers into being again, continue which will Brazil,

geographies currently, by ongoing have to over expect and But into of where in commercialization a to So you we're a we bit U.S. expand give efforts sense the XX XX XXXX. of little

as to So given contribution opportunity that's revenue it lot we of putting we such is had a and about that mentioned, grow XX% are market large us. for contribution, and currently the as effort we it that anticipate into of a energy

So of to as delay there in was a in infants just terms timing of when reminder, bit the a had the of U.S., access drugs.

it older. XXXX, X the So indicated to in younger X and expansion get And X the years was did population. into for initially only of we end the

early upon a X patient population. the that treat line that new younger to years excited so So out where all that growth in have the diagnosis should to X starts for is immediately recently where we're guidelines diagnose to come X exciting well VOXZOGO, in really goal really because with growth with consensus in seeing and that which see out then of be in that's we're international really lot coming age is cohort,

our on definitely prescriber base. working we're expanding In addition to that,

the that is we're So growth for us. exactly in is in terms area expansion target the the of biggest of endocrinologists, seeing which pediatric prescribers

well ensure out continue we We're to getting on where we're back platforms treaters. the the to commercialization, breadth depth. out personnel the in to reach types build so only is populations we're outside U.S. And and broader this in we're we're in continued more doing where also investing then leveraging footprint driving of can want ensure those we information to as And depth there in that we namely patterns pediatrician drive trajectory. referral our community can that there growth both investing -- as efforts field our what to

see mentioned, of We're say our pushes -- already able only markets, in new I'd accountability we're pulls windy and and and importantly, in international really of guidelines really but a in drive our and course, model. do we focused the that. to our in level of right both with not lot BU of as the I've that the focus particular and some So being we see U.S., international sales,

Operator

Your next with question is from Fye JPMorgan. Jessica

Jessica Fye

about international spend I business global of in assets was on you for type wondering development, to bringing follow-up. a And the business, call to quick assets there? your focused patterns footprint? example, would And versus commercial your out think for maybe you the talking BioMarin a for clinical what's if or could year-over-year leverage appetite your ordering Anything as quarter time risk? little enzyme for in priorities more we should quarter-over-quarter more think like about just first any

Alexander Hardy

start to question, last want you with and do business development? the Brian, then the handle I'll

Brian Mueller

and Yes, I of known here time our order as the to wouldn't point enzyme time. diversified midway subject the to large pattern is know, dynamic some payer of granted. question, especially course. single to We're the business, quarter just Jess. you from ordering specific across different brands that bolus therapy global for patterns through Thanks

point to prepared our weighted you to out, as the growth the in year. the quarters, be half I I But XXXX So nothing there. second remarks, point will in specifically to since do of the mentioned expect mentioned to we

just be for about the enzyme Cristin VOXZOGO, So talked we're in plan here or up whether ambitious the the year. of first on that the therapies it XXXX part ramping just

be we weighted and to So expect QX to our QX. growth

Alexander Hardy

then what in strong where in in wanted we've is the know, Thanks, space. yes, the sure that coming Brian. making regard and terms question. play for business we to business the to development outlook department you the add Last company team was place, year, the business compelling of as And about setting got place. a the obviously, to very the clarifying thanks capabilities With strategy we're right already pretty over And manufacturing, financial we development for development the the from the JPMorgan. -- we're flexibility us. more at recognition strengths reflection of what business excited commercialize for and we're strategic the on meetings We results globe. to what about of from very our the ability now development, XXX in of of the And assets. commercialization and are we're a is across a BioMarin had the the specifically these looking standpoint. a producing hear potential we It's development, various about Jess, seeing research, years. very excited from commercialization JPMorgan completed process at business partners the to meeting, role much

these term. asked assets growth assets. of what also range the You We're to outlook looking to longer preclinical stage for stages. assets a looking our are We're Again, at strengthen at be clinical expect into and at. we able

Operator

Your from of Richter next Sachs. line question Salveen is with Goldman the

Unknown Analyst

Congrats on And can on a Wondering on XXX. get hormone you the and interested with Salveen. may durability we progress. CMP that and if follow-up safety thoughts This of for be just on the Is is growth XXX. your in this the [indiscernible] something combo.

For for could help frame understand the to to here any and data, looking us the you're studies? you later translation and guide AGV just what metrics

Gregory Friberg

you, Thank Friberg. Greg is This Salveen.

the first. take question me second Let

study question I that and was volunteer XXX is we're your a So healthy expecting study. the Again, to what believe about see. PK with

believe their a regard of to sustained that well what in greater see cynomolgus repeat With stay And BMN purely we're it's were above of to several animal in the referring increases the to models, species regard. similar time threshold, see to So first the you native hoping chance, see the to to. We free missed in that, in reaching first or levels that we me? that that a in an My growth the AUC looking as question. just I several will which again, XXX what for to We'll is terms give at model try whether to we're fold increases, CNP. of apologies, bones. monkey is the question we significantly can us PK hoping nuance dynamic as be long fold question, recapitulate the

Traci McCarty

growth hormone. It's

Gregory Friberg

but the hormone to growth that the single to and is goal combination, we demands. agent for of regard growth a only also kind think CMP With our delivers develop that convenience the patients, best-in-class safety not drug market

high-priced think our data the challenges the us goal single agents a to now, optimize to X have we achondroplasia. and convenient is for available But to therapy of our we we addition therapies, we that as agent goal. reevaluate again, trying have patients as can Certainly, -- that's In as or the strategy. always both that evolves, to of efficacious do with

Operator

question next Evercore of ISI. Kasimov Your the from Cory is line with

Cory Kasimov

Greg, at about maybe steady-state another mark. attained one program. in dystrophin believe patients levels DMD that treated I for are you've X-year mentioned you the on the

mentioned So for they that this when below you Is somewhere that? should XX% XX biopsies? be goal. Or your expectation the week

Gregory Friberg

us in to -- it's which the challenging been therapeutic backbone point space. What make. Yes. course Thanks a opportunity again a is to open picked affords an for window, Morpholino efficacy of delivery. the has question, and slower We, that we've slower the important chemical and on a backbone has that

the again, a of We The dystrophin that. other significantly for I expression have once would higher will also week engineered studies. the to cause word us That's use will sight. factors XX some data give we line molecule reach think we

of have looks We to we're out again, able is that, that we clear idea a success something that what a model of data of have that what seeing clear year. moment we'll very time And be once pretty to think the like. points from XX-week the at have we be multiple going

give a very going XX-week level, course, point on is there's here. which we're dose we is ultimately with other read able of per to the us kilogram well. we're X that time and add solid going model just to demonstrate dose nuance our the as that. want But to would I'll talking the headed X-milligram data. actual where Of in we're and up that reach we about are whether

of to right now. higher. again, go the forward. to us potentially This is for we're course, whether the clear where, of is opportunities milligrams X-milligram to that able cohort there dose So we track And to give that we're indication be a in X want field a And efficacious we're a are hopeful, be X [indiscernible] we going lowest XX%. that again, bring on very

Operator

is from with next question Raymond Your Sandler. the of Piper Christopher line

Christopher Raymond

Two questions.

Just ERT on business. the

this being you, are back-end You some of something terms time mix slide of the say, and for this heard you commentary inning on in I a was then that diagnostic XXX the think, sort being this can diagnostic terms which would data are you'll terms there data maybe maybe I And second in timing, in your guys from I lining we, press presenting where of great. to a then the but top change? on says effect color -- the half, full mid-XX. conference at of being able now give by bit, sort of sort protocols, in Is hairs be later? splitting that's maybe be release on indicating the harmonizing as guess, print But know data have, I of data Any mid-XX be growth. 'XX would Brian, sort along color this geographies. of select any able I about helped contribution you all the to demand and some government across harmonization. loaded buy-ins to talked think, higher of on Is

Gregory Friberg

Thanks. this Yes, Greg Friberg. is

quarters it, clarify, of X to middle of year. the Just course, the is

again, the read statements. into that, you both correct So can

presenting it the year. totality a in be of We early the will at publicly half in second congress scientific

Cristin Hubbard

question diagnostic kind of bit this Cristin little the And a component. answer to Hubbard. about Chris, your the is

most think enzyme countries CLNX in being into the BU MPS run certainly, tools looking can help our we CLNX. and particular, across that therapies, we the with world. around across new MPS and a are at really when are both in particular, certain in for of broadly different what about lot out model, of diagnosis really in put can activities put So speaking, efforts various that and the we understanding really countries diagnostic we

So family we're to we've Tree progress using Family is there's if in can seen MPS help we're at that so which identified looking then both great screening, gene the therapy. looking we at basically tree We're that patients them CLNX seeing or to on getting and Cascade and get the already. start panels right a ensuring

know Brazil be of anticipate and select in continue and very countries we this meaningful. that done subgroup to a we've will this on so we where instance, for push And really these successfully, could efforts

So bit growth coming also that's PKU because like amount of a to we're I'd But a B of a out little significant the talk part PKU. about certainly of strategy. expecting

keep our with the on of patients put we in day, with importantly, there U.S. double-digit and the XX% in year-over-year at new and driven therapy. alone, by Japan PALYNZIQ XXXX. then into that help growth the end efforts and really was largely saw to up get the you And expect As them growth and to this identified continue growth

really finding so keep So the importantly, that patients And adherence they have on dose. right that restart. them programs then can that have discontinued

hearing that and this. we're what we're that it differentiated potential is phenotypes this so potential of in diet. the works the great across really, PALYNZIQ of fee profile with the all levels And have for seeing is part importantly, And an for progress large because normal unrestricted PKU

continue. expect and think we'll we've So to progress there, it seen great I

Operator

Gena of line the from is question Wang next with Barclays. Your

Huidong Wang

some the IP Maybe for the XXX that provide I and And want regarding is know if can strategy there regarding U.S.? question already also mentioned one I the you processing update franchise. PAT. your Europe. your to you in CMP you the started ask second

that also regarding and us data you started you end fat Maybe are of looking a can completed last with share what the year. for You update.

Alexander Hardy

questions, Greg. it hand then Alexander. the much, I'll to for should your Gena, and over Thanks handle very

Munich. in said initiated So in we -- against as in court I we we've unified the essence, mean, in January [indiscernible] stay action that app

European to at provide on to to of update, that course, And next to process. due we moment the receive an no It's there in further in the we XX action decision underway. provide will, you We IP expect course. XX Should months. a have updates be in it's

if and As we the intellectual time States, to elsewhere, regards we or our U.S. against we infringement. take that when to United defend in production the property will see infringes conduct, believe

commitment those to the we're intellectual our unfolds. you, and property keep basically But to we fund updates Over updated go, So and continue on innovation. us our as to are following our as we'll through Greg. this

Gregory Friberg

XXX, aren't for antitrypsin those a who about Thanks, deficiency-related molecule Yes. familiar liver talking love I Gena. BMN alpha-X for disease.

Z used because a alpha-X who of it By are be XXX-fold potential have antitrypsin in phenotypes healthy as patients, the they deficiency. but ability heterozygous to in And may homozygous who to failure. about the studies protein's therapy very duration and will PK, versus fibrosis these native pharmacodynamics -- But also are the The created usual safety well which to protein circulating Z not course, the handful with is it has ZZ because genotypes It here liver, and Z and the It to And patients small looked some in as as one into protein just ascending lead though, going this and molecule. liver XXX, has profile to -- well. implication where potential titratable. misfold as that is, binds patients both protein. limited circulating The between of that doing how oral we MZ will ultimately, Z are to -- in closely be a are polymers is is it's in patients there of excuse decreases to me, antitrypsin of insights ZZ the be the It these get volunteer titratable alpha-X a doses. it's differential protein along polymers going be enrolled. Z at that. it the to potentially the so, nuance running to be has bind So for as some we're from able well. study, again, course, multiple with a protocols, a

both the can as open forward, genotypes volunteers we're around that right Again, with is for for well study plowing working I for molecule forward to a as accomplish for That clarify regulators And crossed. enrollment. now. the would healthy patients ZZ what NZ as globe well be fingers the handful described. for So of path also as what

Operator

Your the question from Paul Mattias with is Stifel. next of line

Julian Pino

on Julian is for This Paul.

what a if were relatively early study. at be time XX% you share the DMD that and assumptions confident did point, to bit you just you'd color this more curious that since what were and And you looking for goals laid for from to goal little exactly they be you've out? biopsies in your curious a in dystrophin order learned able in the X on Just

Gregory Friberg

you who Julian. X-milligram nicely. at of I have weeks, Yes. Thank These think cohort This the done XX the kilogram said is just it biopsies again. per were muscles been -- And boys Friberg, you, X of for Greg treated weeks. XX, XX

an yet. expression, so have of steep the were in not the in And protein we would in you if what part of regard, S-shape time in terms at that expect curve we of over curve terms

amount quantify translate and our would So mathematically, with muscle will that could questions. able the a the get really we into Are couple limited not drug can directionally, to the ask but there I one -- wanted was answer whether you of as there and the assumptions key we or And that is say well? to tell a modeling was we into our humans. yes. it One

The splice are second is variant. here goal we dystrophin. the produce near question but targeting be full-length a would not novel Again, to microdystrophin,

boys. was that in these end the cells at near so produced the with models. of dystrophin. being product would to end able were our that And course, the we measure measure that to were story the gene And consistent able full-length least we of biomarker from And to standpoint, be see of that finally,

though XX or that in going be the And in were XX% of off patients that when achievable. is going just so been be this tested useful pertinent X before. I in be line target very again, regard, a hopeful, this a humans it's hit table gives early. and to us That that sight that we now a weeks, taken say to good had it's never information. giving line is of negatives sight going is to we're would us helpful And that data XX-week not that for whether

Operator

is Akash with Jefferies. of from The the question line Tewari next

Akash Tewari

think is this their on you mechanism lot just therapy why of big an like and strategic think of I there infigratinib, for kind there kind approach is you one to the that's a that infant assets? a in efficacy Relay, same have in-license value are mean, which options. similar own only looking difficulty And if that of CNP could potential a terminal than has the for BD, better to companies about there they're is Or forward? way of I What's assets think VOXZOGO. have that that infragratinib-like not have VOXZOGO your these potential the the as confident they reason Maybe achondroplasia. appetite for be on if of oral very team Is I have investors, BioMarin looking modeling

Alexander Hardy

Thank you Akash. question, the for

skeletal conditions. enzyme around interested that defined conditions in really is have in genetically therapies. that focus we we're leadership in development positions establishing We're business and Our certainly already

right CNP of is for So and the FGFR. any we're know, And and a very But much hurdle that reassure that position safety treating both indications exciting of think, is important, those as establish that's leadership -- overlay which of to to a is FGFR [indiscernible] an sorts you hurdle, a with for possibility, which is development so safety in somewhat comfortable our to VOXZOGO, like CNP is activities. ISS a in now high states, caregivers. but profile is indications evidence amount development very that of produce have our going not with And in really absolutely and the and really, can business SHOX, and in reach disease these it's from physicians I and to efficacy critical. so infancy is significant Turners,

a path is it's high CNP. at bar. moment We on focused leadership So Right think that we're conditions. now, the in sustained skeletal to the

Operator

question next the line from Eli Your of Merle is with UBS.

Eliana Merle

by -- prescribers? that the do care think it for in for do less see penetration right the And is I are patients from U.S. increasing the U.S., currently stable term you then you Or ex achondroplasia in older do and you of And the VOXZOGO, this do or for you population? why the and contribution guess, patients? aren't longer to see the what with remain ex under this sort not 'XX So currently expect is eligible guess, guess do uptake revenues into proportion over of what's I and as the you I U.S. of potential penetration patients those mix patients, who 'XX? the U.S. treated. expect

Cristin Hubbard

question. the for much so you thank So

that increasing terms contribution first U.S. the the that definitely to today, question roughly time. think over the -- XX% first of at of we being in I see

U.S. As U.S. know, from split X/X contribution ex and X/X, in our portfolio, about the X/X overall it's you the is

U.S. time specifically. growth certainly as contribution we so United our anticipate And States in increasing the the over we continue trajectory

is was patients your to terms the I in the the We launched X of we to majority want around question course, patient That, to X case not were mean, cohort. come launched. are population in of we today. the in of first back not that starts where year-old the do bulk treated, seeing new X-plus Now I when fact

our where And so of was the that bulk were. patients of course,

patients So just X on split it treatment at split, in skews the many that's are rough the look if timing today, a component. X, towards how the you rough to more but

treated targeting starts terms that why. patient there's new patients. not we mean say patients were could myriad really really reasons that the in conclude. I'd probably the of So I of youngest And a

Some of be awareness. it would

of They that there might treatment was seeing the and first VOXZOGO where the the we option ensuring in being the actually options, or really prescriber on treater focused pediatric really, knowing be that would broad is only and it treatment of treated the is offices be. for are today. achodroplasia. Some why would be precisely that awareness

I why it, and awareness them using something in United the commercialization that's add. comfortable we and to working So States increasing to then with efforts the prescribers. we're we're comfort precisely and ensure might treaters you to that want very and is that. on, We our know, what want be Greg, have

Gregory Friberg

is could X-, X-, data with that organization want the this provide on treat. Again, year a not R&D What the just it there. I decision families through we have average about physicians, or mean, add is on complex go X patients velocity. data and to I X-year set their focused data. to If out just continuing build that treat course, to of story well. publish growth we are in a as whether not their our continuing We to is,

We again, these have years at this -- patient of infants. safety point. And X,XXX data are over years

we've by Frame The all craniofacial at means that AGV comfort all start proportionality of evolve this of these identify recently infants published put adult level the together, the isn't we with and seeing these the published of community height to the tip significant And in Magnum. about to that's final starts compared patients story finally, wellness them published And course, again, the that just making a something quality or when patients. story, and is then, life their you possible. and of independent this parents about treaters. balance statistically itself. control, So data and even the as those X our course, measures, years these and Again, as with guidelines, hoping early to these the the guidelines office seeing that were We're physicians that, these are of decisions.

Operator

next Your Capital of question is BMO Markets. the with line Biliouris Kostas from

Konstantinos Biliouris

In liver that clinical Great the the BP. on follow-up U.S. go from on early of address and the the on to both the ex lung one question numbers. manifestation disease. [indiscernible] approaches in clinical Akash's see Congrats validity. question already And these have Maybe sales can validation, the coming strong

that you in that modalities disease. complement consider Given one prepare modalities to your only with modality different interest in go to your or would space, you pipeline

Gregory Friberg

broke does were about the I I'm believe patients. alpha-X that? little the it a talking antitrypsin and sorry, And liver we out disparity between and lung, bit

Konstantinos Biliouris

complement to your editing RNA in your about asking have alpha-X approaches interest in efficiency was in approaches I and would you whether pipeline? such approach

Gregory Friberg

regard both on And trying that solve it's a challenges lung the the to of to mechanism, we've problems XXX, the We've across and liver-focused. again, is With the again, liver. you're seen focused the industry. liver focused on BMN correct,

of between and well, Now would replacement enzyme therapy selectivity protein, the this conjunction difference given long be which that address could with a challenges. in as M presumably for Z the because of some would be

of therapies other replacement said anything nicely Alexander, others. again, combination it a from with It's action potentially As might R&D a days, that to we're in very from that approach early differentiated of think work that's have the well. to mechanism and point, this quite hopeful, add our we're I standpoint. you strategic want going that And you like enzyme

Operator

line next Your the from is Mohit Fargo. Wells of with Bansal question

Unknown Analyst

Mohit. for on Rehman Sadia is This

Another question on DMC.

early that the to data that kg analysis up go X your So at you to And could presented about other initiate help levels if slicing how year be agents. biomarkers Wondering the to data? cohort. those that X are that like are in that talk on can understand you also decision there based And per how was this on mg compared tracking would to that biomarkers XX-week did any us mgs?

Gregory Friberg

question. Yes. Thanks for the

step that the to Let independent was to another could and from potentially a second And again, step data they were again, the seeing. question that trigger one The committee. speaks open, The and and that be was as planned that approved first. DMC. that, me benefit to simply well. there take again, X-milligram they, know cohort The the safety monitoring nice the

I've was biopsy you Your to question the those. in other those patients at what only with and other at biomarkers. weeks, muscle shared at regard We look with in XX looked we've

our addition in to when will that, to that's is of represented assays measure will Of we which we're we the make data can as assay. normalized say, data Suffice multiple present again, sure length clinical to the seen different. dystrophin values, that at we've full you're again want data and for levels, possible transparent and course, actually that, dystrophin at whether different And totality PK how as available. you be looking looking be

our a these patients course, and be looking to them ultimate more of of is measuring to we variety dystrophin not else in to increase that hope functional. nothing well. make boys as biomarkers, not but have absolutely are we're at functional lives So but but just treating only we're now, levers share better these And

this So nothing more share to to else at come, but point.

Operator

with line Your next Olivia is Cantor from the Fitzgerald. of Brayer question

Olivia Brayer

level you are about years, see a some expansion about Just the of And guys you considering then or 'XX. 'XX, next what and could what for look any patients franchise? next but margin enzyme are How over 'XX, that older over like comments medicines business these around wheat-based. that especially for about from the years? of enzyme single-digit might know I we CAGR couple so these the of talked thinking the growth high getting

Brian Mueller

is for that question. Thanks, I'll Olivia, Yes. Brian. one. the handle This

of growth don't the unit consolidated That's any XXXX, in 'XX not and margin in $X business corporate disclose the profitability this on double-digit substantially business the to PALYNZIQ. supply year, There's driver got single-digit above PALYNZIQ includes for billion term. the operating continued directly our CAGR XXXX. the both on flat be patient ALDURAZYME, that operating the enzyme of individual flattens some the healthy going more global unallocated more to over based Cristin kind high which at for talked then growth had brand, I business profitable part next high they're both. sustained goal. which But of is the dynamics for on but of commented we've which share that I'll buying We on example, said for think than year. the some to it well from you year that right, business. of you're units tied margin, pipeline. in touch are the couple and healthy penetration to years, franchise additional rate long there, digit in And BioMarin just as demand, touched mentioned, revenue midterm but the a margins and that, the Likewise, to patient the different with on you across based over to is brand Likewise, supports margin, these over reminder, single some unit that costs particular well expect operating of goal what I'll of go within it R&D we XXXX. exactly out So end be also primary level, Sanofi. and earlier, dynamics. then key 'XX. that of NAGLAZYME kind growth is We about market in And but

margin. mentioned generating doing well. part sustainability durability It's dosing. These are weekly that weight-based So the patient patients infusion substantial both you life, enzyme And the and enzyme for the therapies. of a is the And of are dynamics. business franchises

is franchise. means by that durability part the that So of of

Operator

Your of Alex Hammond Wolfe is line from question with the next Research.

Unknown Analyst

you on how competitor may the groups? following just VOXZOGO. launches? on dynamics well degree those dimensionalize Can across geographies provide market quickly expected follow-up Can any on as patient you of potential switch well. kind a Just differ of And as that as dynamics color age

Cristin Hubbard

Yes.

the for you question. very thank much So

on any there. So our always numbers, into very of been assumptions clear we just has VOXZOGO to mean be competition I the modeled put out

to mention, make to competitive just so And thing. I is clear. that Not bad a want think landscape not I

the specifically it and is we raising our about awareness speak I multiple a we I continue think, there's around think dynamics that think But honestly, around in the like good will factors where this particular, always thing. disease to here. leadership in treatments position, achondroplasia,

You are We and patients new VOXZOGO, almost that the can market the looking higher potential HCPs compound. experience switch a when know there's much the and in switching available? it's in it's to notion to a And seeing paradigm kind because VOXZOGO to positive world to stickiness caregivers talk become is really we're that trusted being believe it some another going that about that their certainly the real for what of achieve. we hear like on research becomes barrier from be compound do a that to start-and-stay so we of should

trusted. so compound very You choose -- very Not a adds know time. VOXZOGO not a and this in to is risk that that element It will to will an take. patient in it's at mention, this switching what's trusted some working of that point is

years thinking to and overall rate. really high mention have hype, goes have just experience, Not a health well growing, data talking building X,XXX about that body these we efficacy we're We compliance and of the safety the of evidence that is over beyond but of patients. patient of that on

as and So which well on continue leverage as both that build the XX% that rates U.S., outside we see, the is on compliance that globe, really across in about the we U.S. that. to average, important that is we

lot but of patient XX% you, outside addressable Now happening what's lives about remind total the I've thinking in of the a U.S., U.S. the -- talked to population

of is entrenched we those the global have lot that a a going we advantage so know in really footprint in experience in strategic understanding has this This global And where we we and dynamics are markets BioMarin these where much footprint have area be. an to an those so think a as where local lot of patients markets.

time, really as our do So United stickiness States well leadership this as see ex to position. really over of the business we both building a look on lot and in to U.S., we forward

Operator

to today's conclusion call call. of the now thank We Q&A portion does I the you This closing for This today's for also joining. conclude comes today's any over presentation. you. of Thank remarks. today's to would hand CEO

Alexander Hardy

and your for thank Thank operator, us BioMarin. for thank today, you interest and joining you, all you in

decisions results our investment the stakeholders. that with and make made last and As you've heard, and greater in ongoing impact tangible enabling strategic to operational even innovation growth year and yielding

and model continued we in benefit as BioMarin's and a beyond revamped expect you high all of our from XXXX keeping strategy great and much, We performance look operating apprised forward have day. corporate you and to progress. so Thank

Operator

joining. today's call. you concludes for Thank This

your lines. may You disconnect now

BioMarin Pharmaceutical (BMRN) 19 Feb 252024 Q4 Earnings call transcript