BioMarin Pharmaceutical (BMRN) 28 Oct 212021 Q3 Earnings call transcript

Company Profile

Participants

J.J. Bienaime	Chairman and Chief Executive Officer
Traci Mc Carty	Vice President of Investor Relations
Jeff Ajer	Executive Vice President and Chief Commercial Officer
Hank Fuchs	President worldwide research and development
Reg Guyer	Executive Vice President Chief Financial Officer
Brian Mueller	Executive Vice President, Chief Financial Officer
Cory Kasimov	JP Morgan
Phil Nadeau	Cowen and Company
Chris Raymond	Piper Sandler
Salveen Richter	Goldman Sachs
Akash Tewari	Jefferies
Gena Wang	Barclays
Paul Matteis	Stifel
Geoff Meacham	Bank of America
Kennen Mackay	RBC Capital Markets

Call transcript

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Operator

Welcome to BioMarin Third Quarter 2021 Financial Results Conference Call. Hosting the conference call today from BioMarin is Traci McCarty, Vice President of Investor Relations. Please go ahead, Traci.

Traci Mc Carty

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

management keep Commercial are hour, Chairman Executive to portion Executive Q&A research I so BioMarin Berberine hope to limit Fuchs. will the call mention, Executive the you that now and We you Chief so over J.J. from On Reg Vice request call worldwide and the JK to Officer. Officer, Officer. one Chief Financial BioMarin's Vice during Brian an Chairman yourself today Guyer, turn President Chief much. and development. call. CEO and Thank President Financial Officer. Mueller, to Ajer, President President, Bienaime. question Jeff Executive respectfully, and of Chief Hank Vice the this call

J.J. Bienaime

a demand our come number a were for on and up. children excited seventh expectations. seeking prescription X will today. approval Thank good for Germany, this I has be us would Thank organization ages of revenues afternoon, VOXZOGO year BioMarin Europe, in already and children commercial from of our to is where for patients 's years treatment you initial you, the say joining launching achondroplasia product, following commercial treatment therapy. of August VOXZOGO in all that of everyone. And Tracy. the the for VOXZOGO France level there Europe exceeded

you early Latin as access well countries as East, Middle in of and America. Africa. And selected Europe, also number As a

and of the first underlying targets approvement, growth achondroplasia and it's only children. that phone improves cause seen the As in

late-stage in In product pleased are U.S. discussions with the requirements early FDA. the indicators we labeling very with and of are demand. post-marketing We

in less potential remain encouraged the we month So, a about than PDUFA outcome now. action positive FDA

I think and for set US our for starting largest XXXX. product pave stage would way significant approval the -- revenue in launch global largest growth to-date, the the

as understand first financials, excluding to global while patients this in with course, bomber our year, in Kuvan this top it of therapies, growth Kuvan appreciate Despite is and as as the Kuvan revenues, strength. strength to of ordering products, the [Indiscernible], full-year the full-year our expected this, marketed And first net the important year, from underlying the anticipated to compared from we XXXX we is of half are half XXXX guidance. what the products year underlying and mostly the our dynamics generic generating in Turning to This than levels. to is increased second expense result anticipate business, management. revenues, having continued line of in despite has the of our for treated all revenues 'XX the product be uneven despite of actually, with last The BioMarin, Quarter we quarter bottom-line of which since by the which faced number it pleased competition record business. base in illustrates impact improving of

XXXX. up the finish the quarter, in Third We to with expect Quarter compared strong in as revenues to a rebound as end so Fourth to

to anticipated GAAP in sustainable significant anticipate knew XXXX, additional the the boxer beginning full-year for leading positive a revenues And we With U.S. next starting up. that expect in we income step-up next approval, year year.

Day, on meaningful driving drive increased profitability have expansion and the business, in to With with for on to in we VOXZOGO, that over reasons. are global our next six for many and layer planned. clinical our agenda full-year ROCTAVIAN products infrastructure pipeline. base for the and our years. products nearly discovery year-to-date our early-stage our fourth-quarter. of And operating February. plan, Suffice to share Quarreling will on from to guide upcoming progress out past it our is XXXX setbacks, on to capabilities activities, as expect preclinical we to technologies genetic potentially coming strategy to has us strong Harnessing million XX support assets. during R&D We our to XXth. that new etc. capabilities as the you provide cash $XXX rolling multiple say exponential initiate regulatory The are pandemic, months excited calls usual, global very of trials been leveraging to resulted BioMarin The November flows growth challenging In our manufacturing stage speed earlier an

have BioMarine helped before our our goals. to us. And of associates want level that for developing growth. focused achieve I many. are opportunities mission medicines their We to remained to has positioned thank BioMarin been on colleagues capitalize better continued essential and so many the commitment on next the that never to and plan the we

for continued call the to Jeff we'll business So, discuss all that to you commercial support your Jeff. update. over thank the now turn

Jeff Ajer

Thank are what with quarter. performance launching brands year, brand start to-date, are the improving biggest at our VOXZOGO. for only you, we maybe top-line the J.J. but of and all very the all pleased Not the regions guidance team's across I'm we during

J.J. on balance Keep $XXX revenues, on revenues half marketed timed were patient by expectations order more first and by previously. key numbers BioMarin, excluding communicated year-over-year. quarter million than consistent a half of year and third U.S. more Vimizim remains than the demand Quarter in solid quarter. Kuvan the as a the was XX% As Despite impact mind, revenues Kuvan, of second gauge described, between already year, QX as the in our exclusivity year-over-year growth both enzyme half around net patient where timing ago orders revenues in of in market demand increased product the total and for the year-over-year replacement demand with we first increased Naglazyme XX% loss for well respectively in by Brineura to brands the product therapy therapy children unevenly our and Third third indicator notable large, reflecting dynamics, those in achieved

have continued Moving anticipated. net capacity. COVID impact to new last patients the with note rate quarter, dosing, PKU is our maintenance clinics patient year-over-year PKU U.S. This was resulting patients starts product opened partial up revenues that we important XX% Palynziq, to a operating many million achieving at to more translated achieved the $XX it the well Consistent quarter, in new of as where growth for clinics not at in despite in commentary to reflecting third initiating in therapy. growth combination revenues as

by was expected, U.S. U.S. increases As XX% the year-over-year. the quarter patient in contributor growth of driven of approximately the main

of at we increase in the PKU While starts new Africa U.S. erosion franchise, through Middle about primarily progress exclusivity country U.S. in XX% individual in the decreased in patient the than level Continuing XXXX expect contributed The reflecting clinics 's for PKU negotiations. to as year-over-year, quarter, region reimbursement third slower revenue optimistic Palynziq with October Revenues by the anticipated. loss originally generics market incremental prospects in Palynziq growth to continues BioMarin for Kuvan remain we pricing due to of and anticipated. US million $XX and the commercial East, to year, pace balance the the a Europe, of launch

treat in being opportunity can that pursuing patients be our for newest we in August. markets with and generating demand on Moving revenue, and date. quickly the patients on number treatment Europe on the approval begin of and processes markets largest the treating which been Voxzogo, level sales. gates generate able European We've lengthy. prescription likely and other to patients revenues. reminder A seeking and very reimbursement we processes to Voxzogo are These we receiving these commercial focused and France, markets launch can primary of patient are revenue pleased where of for set to since named include are the Germany, Therefore, a purposes, price

the demand in and treatment The commercial to patients prior treatment. quickly treating therapy to and begin shipped Switzerland Voxzogo is QX recorded patients, planning optimistic robust QX the to demand a the the France U.S. to the positive France seeing revenue And the started additional in EMEA following in do launches have team revenue. we prescription convert we're demand these small experience patient for respond end potential patients to Already with was in in and launch Importantly, approval. of to at our PDUFA first we're outcome the for launch, been U.S., November, markets. year, our Concurrent with assuming ability as has being already. have we action the they're able that of Germany prescription have and about from prospects from starting

to While medical facilitate payer have U.S. experienced and while are coverage numerous yielding navigating process process, early demand we prescription work we the payers policies. diverse, through the exception

the next with also potential of As Voxzogo on report We the will metrics forward for guidance XXXX place that the team for U.S., We in this Biomarin providing for we we February. launch for a when fourth-quarter brands is approval. to like in six quarterly full-year prepared quarters. and experienced expectations look report previous next results to would set

region. and to is Kuvan commercial launching product the largest help box quarter, I progress update to countries an will potential to expectations. our of the team for of you. the $XX to reported, the EMEA the status will now at are brands low for RND opportunity the preparing is of of with and the on for and evaluate raising color your as the the in tracking the inform launch by Palynziq and and end revenues, will for net commercial you be provided do launch where your over In Voxzogo million, the top-line algo we our robust number and our ranges well turn Thank energized markets attention, we we November. conclusion, active today the for in receive patients being the low Thank expectations we the XX Voxzogo you the should in report revenue in sales, the potentially as guidance total business in of of for U.S. the newest, end For approval are medicines established with that quarter total Demand guidance growing. Vimizim XXXX eagerly results product Hank end commercial other essential provide call The improved

Hank Fuchs

now endochondral ages Similarly, research available the experience over look of robust we'll achondroplasia. forward who to so you is contributed for the important the want Jeff. much. opportunity families European a ROCTAVIAN, also a plan. mark thank We're believe are growth medicine Phase step potentially tracking milestone option for two of for your the possible real been the and echo of while as access placebo-controlled next months should study we access option At for maximum of a through We the are to next as demonstrates two-year organization VOXZOGO, for to the treatment important period when a lifelong meaningful take the which have from study open III forward data Voxzogo to month. U.S., of treatment children time middle family’s we hearing VOXZOGO provides XXXX, growth potential end VOXZOGO. beginning in treatment. milestones to the waiting of early on in Europe, role the seeking expanding long a And pleased of two-year for growth children Voxzogo and Families of of and importance everyone our the regulatory as date treatment everyone In year follow-up clinical enthusiasm of for the achondroplasia. -- data of during dossier European mechanism Thank PDUFA I treatment to are approval children. coming newborn Thanks, the under XX-week development Briefly further and with as plates clinical and and this the bone on years. have treatment. children next age promotes this signals readout on of target that X November targeted that we benefits time impact to in forward period. look VOXZOGO for the approval supportive. the those behalf plates to observation look benefit be up provide reach amenable We around years review very toward to believe the clear their action

those application continue ROCTAVIAN months to for the of to supported continue U.S. in with supportive biologic second your treatment. reductions of XXXX We resubmission We validated be potential from year, followed quarter opinion [Indiscernible] In remain expect we demonstrating the clinical in will application available. an observed evidence, which authorization next severe to half U.S. CHMP in review, to in under review an Factor for bleeding with it when Factor by important hemophilia see marketing in procedure is and shared data, be confident the data, two-year of on expect based Europe, today treatment with expected assuming the EMA the eight first the assuming ROCTAVIAN option infusions six following A, dramatic a rates.

for Turning XXX BMN the the renal guidance steps. received Food that with from now place Phase clinical gene agency therapy, study we've next and by area, than communication therapy kidney XBX whole following Drug Administration, the on

the the agencies in of are for process addressing We request information. additional

therapy was findings a gene on might GLP needs, immune The we're update So, on to to patient’s when understanding committed we the recently have not serve safety identified lifted. be scientist pharmacology striving hold study, The non-clinical PKU on non - findings. deficient based for hold these our do study mice. an

As and with our on we've XXX to that specific issue other panel will view, safety gene therapy or treatments. during AAV unlikely corroborated this shared these AAV the was previously, FDA in it translate with and, uncertain BMN in it discussion remains findings a September, safety

the We process working FDA updates. relevant we'll are we with through have you keep posted as the and

to mechanisms XXth, capabilities to Turning updates disease stride, order s medicines. internal in. sharing to I'll will with new the Thank forward describe has the in its you underlying for and tools event for advance understand development excited new detail turn so results Brian? you quarter. to our Day organization hit The these assets to many your R&D earlier in planned all data melding the develop we of financial now genetic our you. now update stage November you share in over to to with targeted We greater discovery and support R&D under own much call the hope with We're and program tune in Brian pipeline. look

Brian Mueller

Thank you, Third details on our release for summarizing Quarter Hank, press full please refer today's to results financial of XXXX. the

comprehensive on As XX-Q, days. the over the few available report quarter to Form upcoming on usual, file track will our which our on next be are

timing select million QX As illustrate and total XXXX, timing and and for J.J created third the and Jeff the QX quarter the country mentioned, between half year. order last XXXX, meaningfully $XX To order the quarter the has that did previously impact excluding first East fluctuation. the revenue, XXXX Kuvan. there flow not Russia, revenue quarter, prior second anticipated From was of in individual uneven a on Brazil, Middle approximately more recur the revenue accounting quarter, over substantially of

in strong of in dynamics, Based key well point, are non-GAAP of including the year of guidance Palynziq, measure demand Vimizim guidance. as the previous the and GAAP line Vimizim timing our to revision the by in again pleased revenue as we guidance both improved today financial guidance, to to Jeff To to first patient XXXX. full-year our best we're Again that back upward of business however, that products. performance. on Palynziq these and start including of mentioned, metrics in as And end guidance raising July, all full-year our a lower for continues the driver full-year Importantly, the improving our increase the revenue XXXX the drove announce U.S. for we total top-line [Indiscernible] emphasize anticipate underlying as half bottom

full-year for our in by line expectations impact growth, beginning total at continued Kuvan generic of COVID-XX is mostly XXXX total As the better XXXX, be U.S. on of to our which the this competition is revenues impact noted the and year. than in with despite revenue in J.J. for expected of a

we the are year. early-stage by which the R&D third research XXXX, to R&D for on than Moving the our third a for trends the expense fourth $XX quarter in million guidance was expense $XXX range and of Based $XXX expenses, operating expense million expense million, quarter, $XX quarter for development. expectations QX for for and reflects increases which was 'XX, R&D our higher of lowering R&D full the million through

XXXX third to compared with XXXX. expense, SG&A essentially which of quarter million, $XXX Next, SG&A flat respect the to totaled was QX

preparing in VOXZOGO fourth are launch the We towards the in the of weighted expenses and the launching year. for in U.S., are XXXX the EMEA quarter SG&A region,

net performance nearly of discussed cash XXXX of operating of potential European our the XXXX XXXX in set One foundation leveraging VOXZOGO our cash call end revenue previously developed is Quarter position the is launch Operator? year. growth flow we at $XXX we billion primarily to XXXX. of net And to million operating next to products GAAP between to has an potential and Which the loss of dollars a next a that Net of bottom solid with timing transfer an Please income Turning now up lower from a And to a XXXX. increased through and entities, third XX ended operating guidance. non-GAAP lower guidance to very was Thank of quarters flows look Non-GAAP non-GAAP totaling performance in increase $XXX market in pleased as continued which level a the bottom on are of in XXXX. Third the XXXX non-GAAP in large will line We of progress $XX first coupled the of of potentially significant strong is non-GAAP and substantial midpoint XXXX, This XXXX generating of of full-year $XX our organization the same also of the QX GAAP our results, We've comment million the forward of chapter three quarter compared observe $XX programs three in range. that timing. the our late-stage That large Company's early-stage and for previously the at reduced guidance to million, the [Indiscernible] Fourth loss year the income year first income reported BioMarin GAAP to We're And positive the bottom-line the GAAP quarters of first as future benefit net that strong with the Launch GAAP impacts line improved cash brief full support the Quarter, the level million XXXX in million included representing delivered $XXX to end year. have which $X.XX $XX result revenues GAAP million story. between generate in million the we the of the continued an open to quarter cash business increase property GAAP to seven third the of million based the also a discussed investments portfolio expectations the quarter and helped this at due net $X.XX and your of tax and U.S. expenses for the through pipeline two billion BioMarin's income total XXXX, cash third to to million compared we of last in half of with between of you is of year-to-date. approved last R&D that drove million now income quarter rights recall the and of weighted third income certain year. exciting year. flows. million of we in and intellectual positive goal the $XX related $XXX than Income non-GAAP your quarter our questions. of $XXX

Operator

the [Operator At will sir. of And our while session. by Please speakers, we time, JP Kasimov first Cory roster. you, begin compile Q&A our this stand from Instructions] question we Morgan. Thank question-and-answer

now ask may your You question.

Cory Kasimov

question. Great. there's if label years X, view of approved young either afternoon, patients the patients to point if equally access guys. pushback course? under you. you FDA Europe Thanks. pretty broad, of Good two potentially wondering any old. do covering Thanks The and in early as be the broad for expectations is physician from in Thank in taking or for Voxzogo I'm obviously as comfort have treating label

J.J. Bienaime

to want part? the of part you Jeff, the and answer first the question second Hank,

Jeff Ajer

happy Yes, to.

CHMP really a ages described safe and Europe, we're for both open-label And endorsement the we statement with what to but powerful it, of to early or up for up -- us effective X the to Europe. provide on we're turn the you payers for believe maybe So, on years for patients the and Europe, use and in payers as X- of one Hank part pushback anticipating not years and is that X in happy specific it prescribers including to I'll second too And It's patients. in X-year-old from over prescribers. question. color the

Hank Fuchs

Yes.

FDA FDA label. condition, And we're we're R&D no to the and and the course, of influenced, there's will as hopeful EMEA we're between encouraged the Day where a scientifically but context And the Obviously, conservative. EMEA the to independently. to more similarly Of of to know, you you that and know, FDA that of you connection direct that they really operate information. I'd be the the share understood going consideration the tend is in bit little as encourage some we're come vigorous

month see just where it through we'll so, to to lands. the muscle And have next

Cory Kasimov

guys. thanks, Great, Okay.

Operator

question next our and of Nadeau Company. Cowen Phil speakers, And from

may your question. You ask

Phil Nadeau

Thanks for taking our question.

one the that need Another two-year in was discussing VOXZOGO a for more the letter perhaps aspects the of the one on of acceptance is controversial data. language FDA VOXZOGO, placebo-controlled

for of some late-stage of negotiations, Now, at approval information us FDA dealt could that it's around. us time reviewers some Advisory or or data commentary you to VOXZOGO discussions, safe in two-year placebo-controlled and are the for around the you with whether assume give that since this won't be have how Committee's [Indiscernible] we're that necessary slimming idea

Jeff Ajer

But Phill throes say, that give I U.S. durability of to making and worth or can't and the you really feel or of natural the history FDA Yeah. unorthodox focuses I data say from that of open-label interest, was. so is placebo-controlled discussion. good for on transparent the question. I of that really sock’s you in the we've exact much the reminded too the We're of at as where is it say agencies Thanks, continue of edge time the get-go agencies what about And the fairly just to been would design, and its a your pretty them We bit I amounts essentially it's of on sources to in growth. about supplemented what add should years’ in we've on little to two study provided data. we good the years treatment comparison pretty with data X the effect linear some durability but

related is I clinical or to would or is question just of that that at very administration of about case predict clinical a deficient also to been you should Does to in think around be the evidence is a substantial going growth. that's say likely growth be, two-year us is in a hormone benefit that final And one suggests benefit? benefit lot there growth a final sort of I that constitute hormone reasonably the of to the that that concerned field, language site in has tied. all non-growth FDA that and disorders aware this adult

expected of the not is I to final what come think be of some going their what decision-making two-year sort say But about about in from I resolution weeks. this can related All So, does story. few form think to ultimately adult-type? next this durability of the to will be

tuned. stay So,

Phil Nadeau

Perfect. for taking our question. again That's very Thanks helpful.

Operator

our Chris And of is Sandler. next speaker's Piper question from Raymond

question. your ask may You

Chris Raymond

pipeline a XXX. maybe on ask Specifically could I if question One, Thanks.

but. And I but sense what like in haul near with issue I'm maybe to experiments? maybe -- with the you you on you was that. This I wondering additional and that quick -- comment met can could And can felt going can in don't be this something not as share on month answer mice. if you're you've met meet a Thanks. clinical maybe maybe just And have you were this them? agencies FDA this that I'm when or the might sure then comfort age? analyses FDA get an to the have to kind what's when did what I addressed term is be them or question. willing comment if lifted, you think started you will patients experiments said Hank the this you is only with to So, like know what Europe, average you a longer-term to if you on give related requested? to provide VOXZOGO could the Of

Hank Fuchs

take the while question. part first second [Indiscernible] your Jeff of the will part of Chris question, I your

It we finding a discussed with to was, You FDA, tactical that business. to cohort you it happened correspondence know, and evaluation observed dose the agency meetings with you we meeting sort correspondence were had from was know, we've Chris, because duty-bound the what the We had And with the of get pretty their start felt period out second report. turned patients we a in that. of nowadays.

were protocol be -clinical is in and this found the including, they've finding is that all seen that. We wrapped as much were the up not agency what really pre a informed to and we we us an as it, four as they consequence, But about so, And that straightforward soon that transparent anyway. were asked asked not they enrolling this how relatively moment with reflect minutes and patients yet. like at consent of things kinds pretty can of comprehensive and for in going they things package and

list So, considerations. things before obvious it's pretty and the prescribed they of have that types they've seen questions of they a -- these and

think off Zolgensma and agency meeting. thing FDA in presentation when the back bigger Committee XXXX, of spite that they in what of struck (ph). came is 's to approved your approved approved do XXXX genesis did I the that subsequently of drug fact a lot administration approved Zolgensma. think I and subsequent they to we XXX integration were us put the and the was perspective have and described we about search think and a beyond. goes where orthogenesis, Zolgensma. is importantly, Advisory that agencies And And importantly through to The was Zolgensma to for in clinical know the divided been about IV presentation it XXXX gene X,XXX of been that know to what a treated relevance I in -- into have to your it's and question treated the liver. or [Indiscernible] And of patients with patients was from lot in the and this therapy trials over fact, insertions The have with that mice. goes the vector

a think not about other the I experiments because not And says pre be In there a thinking on one that hand, so, a that information to as an what hand, of kind there's that breakthrough the in additional agencies yet, patients’ of for -- the don't on think issue. agencies information that benefit of -- as real has is issue providing. risk discussion, -clinical any is the I of the aware appear risk sort but it's was what a terms risk and not

seen I point. to-date. [Indiscernible] expect at think oncogenesis in mice rather the there that on little given scientific on that that and the is We of to what's understand this of humans in go that haven't a to humans, vulnerability more see any community aspire was going challenge to maybe a cancers for is ask insertional there speculation to question, fully we in assertions a

that would and should think in has about there a the point, interpret one and that probably to abundance aware was do, you be versus I set That think they fact of found this more aspect see but predicting cause-and-effect us the humans of delineating quite question that answer technical oncogenicity a terms more And relationship to uncertain. in all study clonal to to are findings or that of lot rather, the of issue mutations. been lot between and just of comfort indicated the field give postulated cells a translate for of highlight of of while done a I experiments and syndicated directly a insertion. had had specific of how I the specific dealing that risk humans, there with this had So won't these how to that a does details experiments And you're a cells insertion want

our on has and in X.X think not study is to mind I However, in characteristic. this the every see thing cell keep the that we sample

are in to get raises think in we the to first a the that or going better of about relevance question even humans. understanding And base terms mice. answer not in idea the in of mice to have whether I so, question. And causal Passengers on We're fact findings these it do

shortly. front a we Jeff therapy info me and let And the Jeff, get because gene agency -- after start can trial awesome we look of on better. in it problems this it pass season to So, the forward in scientists question handle are row providing and have part needs our who of at now see to going X.X the a are of to if in a to we lot it the all on your we lot

Jeff Ajer

historically might to to that patients you EU, but with in digesting about I'm still the say, we specific implementation the privacy that all we of Hank. have VOXZOGO had the our with had. Thanks Chris individual same don't respect have and data regulations GDPR very

your question. So, I'm around rounding kind of

say of the get product by early that of size don't coming work parallel program approved data we France with -- estimates, of We strength. some was age the might in patients separately mix the negotiations or years patient and under we've I That an ESP ATU yet treated is to up. treatment. have two are usage early from label access that. EMA patients ATU As about to able What led be the early Will there. through bile enough thus that the and got make on to data

up. interesting ATU the label indication patients So and for five-years enough, is

so, experiences of, we'll that keep you range get to color kind posted of as evaluate. more the France, the And XX with or say X we data patients years going - And forward in with let's X old. in early

Chris Raymond

Thanks guys.

Operator

Speakers of our next Richter question from Salveen Sachs. Goldman

You question. may ask your

Salveen Richter

Good countries demand. about XQ give afternoon. you and strong just in early talked how just in of you Germany has there Thanks should taking about access color for the on so expectations? us how question Can we VOXZOGO exceeded again, think the my the launch but far France, select not some

Jeff Ajer

never ends a Germany. seeing question. clinic later add color C of a B. end with a pleasure but we're A. patients the X, from brands this clusters. that one to characterize us through comment clinic clinics patient really through we're part, in seeing from in drives Hi that for we've still time, some What this here, straight about in and once. we're of in at one, patient of A this, of that patients that Thanks clinic coming We've ends one from I from patient also launches, time as second generally while A, our previous one experienced is it's around gives really Salveen. some a there's would of I in -- emerge coming sometime so come seeing It one answer -- maybe prescription later, clinics demand individual and from the prescriptions patient specifically had patient at little another strength from would patient X

which emerged of sales from to an that In sales lot demand named is not addition, there's that EMA patient mentioned I by a been markets. we Switzerland, prescription is market. have the way

experience, prescription that -- larger one demand. ends patients, EMA Switzerland We an And is comment J.J. Latin initial demand the than our for combination approval, two. patient sales, and seen so seen we've named as named East it And registration driving covered not and quick prescription So, groups outside from that in the mentioned. really market. That's about we've Middle of demand of a America. is or one of in markets, of cluster -- in markets also, in not by patient if prescription thus

J.J. Bienaime

definitely it anecdote when we in obviously is an for been revenues reimbursement kind But filed. government there. have significant However, the X [Indiscernible] only sales have I because limited the mean, boards having going the treated, to patient's or that's in treated are the growth we well to the and dollar demand XXXX X for be like more. compared QX Interesting anecdote. interesting federal got Russian on lot the we a there It’s Voxzigo that got XXXX, interest. in for even of Other in of is Russia, that not noise from we Federation

Okay.

Next question.

Operator

next Akash from question speakers, And Jefferies. Tewari of our

You question. now your may ask

Akash Tewari

so much. thanks Hey,

of one's The stocks it's in been the patients? And used this study. safety kind commercial did the venues unlock that about Vosoritide, in the seriously for time a Vosoritide, your these don't rock approaching so value, higher what largest, being to Vosoritide to where So, guys that explore much. important population. there they for bit far point see two are How you a are bit last for strategic talk from use end, the younger or a more you of launches. of range-bound adjust two cape Is years, the fact one FDA you level. can dosing And despite XXX either perspective particularly, planned? as the in [Indiscernible] would a approval get most you so Thanks

Jeff Ajer

of I'll first the the field in study read part put start the The out. was with question. trial before phase your of

and evidence So, available safety in the time to in the older of study. wasn't comprehensive efficacy children

having dose months there and hypotheses to underlying of So, bringing designed authorities, terms core think in to three reason in about age, response exposure cohorts from months. that by health maybe ages that in patients were exposure with relationships. zero, the was I'm study consultation two And the differences I the I've or five ages the up two in was knelt six enroll three upwards to talked six for

to or using And response opening to children older microgram to PD found before dose and kilo X- And cohort concept so, was the the of there safety the was move that randomization actually up that we those an the that dose to we who efficacy PD under limited appropriate appropriate safely that. enroll the response application. children are then the evaluate. to with a their to two, same that XX dose children only active five. will such We the with relationship appropriate had was to turning increase are dosed can gave we the R&D dose than that - per dose Day find to as PK/ that did on of older I Sentinels exposure months to Then off of an in do contemporaneous X-year-old in information my in we've rest was no And infants dose. X-year-old that head, to to patients XX been from informs also that European medication that review because the microgram the data that top placebo. we to of number age response exposure forward tells informal were zero short on I data exposure us that the the is we're what and that six are for the had age. that those dosing and And had since drugs, because months, small amount control. a of And study in to enrolled health think means to X- be that long-acting benefited easier think cohort, authorities of from in short than that drugs what although X-X other acting on to we trial the basis is that actually months having think drug years. very years humans sense the from X acting by especially I relatively the accommodate did and X X direction X to adjust

exposure, seen significant And haven't From relatively perspective, so, profile additionally, it's adverse of in safety safety well-described information. Voxzogo, going with really to clinical now dose safety the much responses. to be this of we all a important have terms

and just a your shape I dose in get good relationship understanding and you'll dose feel month just month therapeutic And of own pretty say. I over we're under like response. of that safety And so, or should the a

me pause Let there.

J.J. Bienaime

question, I think part your of store the I your second because different.

J.J. Bienaime

that's U.S. think I about that the believe XX% don't but few it. survey. you, will there investors That VOXZOGO alley think so good be forgot Thank Obviously, you quite in is on that was apparently weeks recent like past only so of another approved a stepped I news. the weaker in

approved it's the on stock. And the moving -- that that's resulted be advantage I weeks in should about significant think away. three

QX then year. date in So, and conversation next VOXZOGO after CHMP again European opinion also let's PDUFA the of the start on the potentially

Akash Tewari

Thank you so much.

Operator

question your question. next from you ask Our Gena Barclays, Wang may from

Gena Wang

my for taking Thank you questions.

So, first is regarding the ROCTAVIAN.

of data is or type also related the regarding ask for what PKU on similar durability. is is ROCTAVIAN? preclinical for ESP wondering question And asking that then filing FDA expect anymore latest you interaction specifically for data the ROCTAVIAN do for program the Just data type of FDA

Hank Fuchs

complicated, a get can't see it's -- of Well, the I let's chase if to bit it. little cut but

years’ really no communications URL, worth some of timelessly our We've trying had things that reveal at really analysis, the data. they two-year than in deferring We there [Indiscernible]. of terms years’ terms news of is really worth In two data two resetting of of lower-level which In hard, FDA want statistical and or other there that correspondence want in they like interaction new spite about by expectations. want data. they said nothing

division the the with interacting division. and review are we So,

experts was maintained make conveying Sorry, tell the what we've collegial year. we had Phase on this first and good us an telling we're very on and discussion. that they additional they two-year situation to based us, through And optimistic two-year trial us of efficacy for a the much [Indiscernible] for which what two this to clinical believe FDA demonstrated were year, like have that's may far, as seen so XX be buys reported They're the might patients think the data. to be of asked we've in we not but be years out in earlier process in follow-up the and the And experts that second because that for decision basis data. III as patients treated going and will the hemostatic the in reads for

made pre the we one durability, believe for the second of a for or that maintenance VIII XXX question. actual based data, PKU adequately of that expression around the and measure in seen specific controlled answer confident The and durability we a to of more measure PKU VIII credits we that year so And little in expression, -clinical pattern remaining ROCTAVIAN. is that year. is whereas to from indirectly product. with like bit control therapy have difficult what a the that will So, patients we Factor on you there to itself, you gene through the will committing supporting But the reason be PKU, are stronger to Factor gene was looks be see expression, issue where want before than the argument simple transferring there

and we done immune not the do do to of long-term in the others nature long-term one don't for of and studies that Just did and lot recall them. of a ROCTAVIAN, studies typically Surface response non-human have week. or because primates, people

able immune You been durability can sustained protein. and species demonstrate to to species. in of the PKU the In lower effect we've human preclinical response see the

Phase So, is that, news yet data patients hope to things changed of prevails none trials. ROCTAVIAN we with where that is from we the good argument and are one the those that the trial three the with this regimen, steroid from unlike to Phase inform. all we be to had in The a material discussion to but agency, happening that in detail made actual in upon tweak sufficient the XXX of of

patients provide long-term earliest these will relevant So, information for exposure.

Gena Wang

is a pre was A of Sorry, to FD like data Hank, the I do sets data FD the also the similar ask additional type anymore, data asking, will data wasn't clear. for ROCTAVIAN more expect support program? for safety referring was for you whatever program asking safety I the they A PKU was -clinical

Hank Fuchs

studies look the post-approval have if label, either oncogenicity pr submission they requiring carcinogenicity at studies. mean, far, or So studies -clinical in to been the prior Zolgensma no there not studies. are you I

expect to be those prior well. safety additional to required to approvals, be held discussions are don't and as perspective a studies again, So from submissions but

Gena Wang

much. you Thank very

Operator

question from the of question. Matteis next you may ask Paul our line your And of Stifel,

Paul Matteis

understand did guess thanks full you heard need you confirmed? Do is I you either approval, as that so potentially if it if It two-year Thanks I for study think do accelerated approval this though of a point taking how at placebo-controlled that business as deemed to review, think you when a And of like an opposed marketing earlier. or about is Hey, will in study right. questions. I accelerated the be matter requirement, feel you'd ultimately do placebo-controlled two-year approval, much? the benefit issue talked sounded the you correctly this

Hank Fuchs

decision, the The tailwind the is approval a is of -- a agencies is decisions. pathway consideration specifically specifically

was much issue to have required, more studied it interval question promoting would Nexus been come velocities read patients to that was this landscape that study all the of just I characterizing. anything confirmatory of or a landscape don't we could what it group the And from were growth have then final, a that products growth main pass be other would an question. a If for longer-term the I say too I want than perspective, constitute of main already we to FDA that -- into should follow-up hope have So, the that, in evaluation.

of close on treated those but know, open-label. had And the patients of a provided patients I the agency lot have them. final not patterns, were height. data for is to interested years them agency, at treatment Several in as four think all of We you first we patients the in seemed X-plus

an accelerated tight So, different. hopeful they requirement whether come post-approval even full and approval it some all it's a a we're in, when to a accumulated commitment or that post-marketing characterizations [Indiscernible] it's of that benefit approval on And will whether a document to will come conditional VOXZOGO. powerful level for finally to in in, be the

Paul Matteis

you. it. Got Thank

Operator

next our Meacham line speakers, from of America. question And Geoff of the Bank of

ask may You question. your

Geoff Meacham

for question. the couple just main a Thanks one. guys. the Hey had of I

BD forward? on of are something could thinking it, you technology recovery to starts? the do play just maybe get about things, in bit in going little asset clinics potentially past mean, PKU or that's Palynziq not of helpful talked that on more or That'd new still kind you're maybe think Thank wanted bigger have you scrutiny a diversity, started of and in about technology opportunities reopened out NYU by the indication in looking be see is to guys you diversity the be there that. You just is I are for J.J. light how a business, And you. what then a fully AAV about of thinking how how commercial side going in you for update picture on Have to that on terms XXXX? an prefer

J.J. Bienaime

to PKU maybe part the go Jeff, over think [Indiscernible], you PD I want and question? the

Jeff Ajer

Sure. to. be I'd happy

the we're clinics capacity. remarks, are more U.S. in continuing and but in Europe, as is full PKU quarter what both to seeing importantly less the So at noted operate previous in than this our quarter,

do of access patients needs we're in to, so facilitate trying to have XXX the course, clinics hasn't clinics, think to a while that little there of across needs we in less optimistic we've that do patient those for staffing that that, live. but just of largest about virtually are there really getting and working to capacity is the VOXZOGO. there's U.S. conclusion been are year, we other usually are at patients are a comment referrals that the frankly, There that on terrible through a we're as is acute the about patients think if variability I I for were back genetics the And that they pre group a optimistic year PKU we've seen in And continue pandemic. therapy clinics might new clinics during those clinics we're previous patients getting and beginning the capacity, tactical reset issues balance would steady solutions bit to their conditions staff for to for in what a we're as what pandemic. are PKU for drawing been, there's of of their priority for -pandemic supporting there, the of we've now, expectations we reassigned children about Of been acute optimistic genetics happened not treating, that particular for while I basis. some PKU steady operating the here Having to are lot say it certain capacity. and continuing start. PKU And is the overall new the see for on but clinics PKU for through said first-in-line read kind growth.

In and relevant of really fact, other health are care as both providers to that pediatric, orthopedists pediatric such we VOXZOGO. on endocrinologists, call which

part restrictions For in back and protocols -- in specialties the and are place, with they're business. those most

your clinics, seeing through VOXZOGO. say capacity of constraints PKU expectations would kind the to in I don't let the we're about read that So,

J.J. Bienaime

So --

Geoff Meacham

[Indiscernible]

J.J. Bienaime

Sorry.

question. BD Regarding your

good So, it's I mean, question. a

comments. Just want few a

Company but was we a you in therapeutics, being protein with We [Indiscernible] BioMarin. started Kuvan an mainly modalities As have several on exception. based a small as local know,

If today, you look have at we therapeutics. our additional pipeline protein

about small some in molecules, oligo moving get second-generation, to and we into we We options oligonucleotide, have have we (ph) there DMD. or back with are beyond

beyond go going always one are to we modality. so, And

gene ROCTAVIAN continue Besides indicating And the pictures therapy clinic pre we in no So, programs be future, if at is hypertrophic [Indiscernible] potentially is about important will programs program But say, gene our years in the to talk all and development. will and therapy right in is and to Day the about Company. which in gene new we very I two start. would pure R&D now. terms therapy is some have be The -clinical us we the new got about when therapy, but in year-and-a-half, have see in on to disclose to for you a cardiomyopathy. only of therapy intents

go based billion end we huge that with I giving reminds we're know, it's to time, a same shouldn't the know many at that have monoclonal other those. a from I track first the we losing oh, some todays been major on the and considering antibodies aggregate will a sales the Company antibodies, not that the are we would so. Monoclonal gene forward were XXXX of when timing, that's are And that Then don't up gene say monoclonal died. guess case I the we don't patients not somewhat So, therapy, ago be got I I'm doing of still mistake with on over programs. believe year. continue XX far with of That have would antibodies $XXX many, potential. mean, programs I but years treated And me, therapy only it of We the to

So,

J.J. Bienaime

we at learning adventure the a therapy. the with beginning here of are So gene lot. We're

to [Indiscernible] better lot. constructs. continue illustrating, understanding learn in companies the design respect gene leads response as web Ginnie believe in of happens of so we expression, our immune and think to ahead durability, we or believe the can after therapy and this are most better was I We Hank insertion sales what a of we we response in cemetery

at treatments. We got reached to also look

that a hear Day, You'll don't therapy gene non-viral about at bit opportunities. look we little R&D

drives what the hope terms what So, that of middle to this maybe your answers the of And fully calc BD. looking give at Hank stuff of drives and basically question. framework I his too is in this. research perspective outside internal our of kind in here that can

Hank Fuchs

many genomic And this essentially unearthing and environment. -- I'm is so opportunities. just it seizures creates target epilepsy. children unexplained for really have that the have Well, I just screening genetic behind always evolution think who rich the done we program

numbers for to we history. used specific we're can be to to identify enough of start with accumulate concrete to CLNX patients genetic who starting of details now things We're we and concretely runs but specific that genes like mutations large medical find pedigrees indicated they that there's sort evidence in have limitation then -- increasingly etiologies. actually families, associated it's And linked actually starting find treatment family of trees so, because Brineura also are because in with that get but

time amazing molecules, leverage an that nucleic we small really it's acids, do proteins, peptides, and acid to be So, to where and invested we've mixtures. complex to are in nucleic technology-base we be diverse able can

together fundamental itself. presenting BioMarin thing So, science coming it's therapeutic the and of that really of in orientation and a about nice terms research are our to opportunities

is So that for early that that's that being, we've academic the are. in and think partnerships. way a we all of time successful I And where saying been

Geoff Meacham

Great.

J.J. Bienaime

indirectly One to more related comment this.

know VOXZOGO, of data for major presentations. in trial Day beyond going give achondroplasia. on part and to opportunity a not got VOXZOGO you type update we I -- with you, As any would to Day, attend indications but I'm disclose some, we're our that open on clinical up going could you but going you first R&D here, our [Indiscernible] the highly on recommend additional a We or so R&D

Geoff Meacham

Thanks.

Operator

And our Mackay Markets. next Capital from question -- from Kennen RBC RC

question. ask your may You

Kennen Mackay

the Maybe me in Hank, to that's BMN ROCTAVIAN knock associated can vector or might seen you. safety to the versus maybe integration some in. seeing questions you breakthrough on prior BMN Squeezing in the the XXX going in of levels of that adeno viral address used adenoviral XXX. of to Thank with - ROCTAVIAN. concern that back concerns we're

Hank Fuchs

across that It's not really levels can in integration the of are things of but, be facile to do head-to-head species kinds found general relatively low.

move have contrary, quite a designed lower and a that suspected more integrating designed guidance not be XXXX otherwise less the designed we do they could things therapy we And else any facilitating any referred and ROCTAVIAN integration. And integration that that so, to to there's know, meaningful of integrate. to don't than or vectors are in in I think that to anything a agency necessarily you likelihood early that is their integrate unless on to gene to propensity we Visa's fact,

fairly for So, side stay delivered and that's low the true believe be that propensity on likely integration we've to all? been to

Hank Fuchs

from longer-term a done both for that of sequence. through coding studies other its No. HAE lack the another. felt than the doctor and one to We've is about otherwise to vector already, vector read We Ractivian identical for good pretty

we read the nice to species. of lack about through pretty from fact, feel other good In

Kennen Mackay

you. Thank

Operator

are time. And this -- at speakers no there these questions further

closing You the may comments. continue for changing [Indiscernible] now

J.J. Bienaime

So, today. us again joining thank for you

based year as with and foundational in convinced well we growths region early-stage So, we sharing on to on during the business we are just therapy in on poised base are the I hope EMEA later virtual anticipated VOXZOGO ROCTAVIAN November in our forward -- pipeline launch described. we gene late on for a look mid significant on next [Indiscernible] XXXX, Also, U.S. you That's you month. our XXth that of R&D that top potentially as as to next Day.

by the important approval Voxzogo we end US are month. we continuing of including as our focused on achieve catalysts next So, in of potential the the strong the momentum next

you today attention your to we you soon. forward with look speaking thank So, again and for

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