BMRN - Earnings call transcript Q4 2017 | BioMarin Pharmaceutical Inc.

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Operator

Good afternoon. My name is Kathleen and I will be your conference operator today. At this time I would like to welcome everyone to the Investor Update Fourth Quarter and Full Year 2017 Results Conference Call. All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question-and-answer session. you. Thank Relations may Investor your BioMarin, at McCarty, begin you conference. Traci

Traci McCarty

Results business of to availability Kathleen. containing President, those the detailed Securities that the and in future potential Chief and Dan as products BioMarin's reports. actions regulatory authorities, development. product market Worldwide development capital, pharmaceutical different Pharmaceuticals, Today Research performance, materially and future Commission you, depending Chairman therapeutic BioMarin's Technical remind Head today expectations Bienaimé, forward-looking Exchange BioMarin research of by XX-Q, and regarding the Financial areas Commercial including competitors, programs, I'd with about and me and differ Officer; Jeff presentation and of Fuchs, Thank X-K commercial Jean-Jacques Spiegelman, on non-confidential With factors like Ajer, you Robert filings Chief financial XX-K Baffi, Operations. such BioMarin a this of statements of Chief actions prospects in and Executive in products Hank may Officer; the progress of Development; Officer; are is and

like I'd Bienaimé. and J. CEO, to our call turn J. to Now over Chairman the

Jean-Jacques Bienaimé

you afternoon Good you, us Thank Traci. for joining today. and thank

significant momentous for a was XXXX So and BioMarin financial, driven year clinical by regulatory achievements.

CLNX, our the For approval than product. EU less and being which is the the for compared with patient the $X.X in topped We from Brineura XXXX, full U.S. of received an treatment to year treated billion, revenues four increase XXXX. first of for years XX%

notionally data our separate for roxaparvovec product severe of we the health on past safety sold the gene with The of enthusiastic which a Not Phase for a the to joint patient community one-and-done with was other bleeds than valrox, in earlier As weekly patient multiple, PRIME X/X very even A we received inconceivable treatment launch will and early December and started to taking Brineura's the breakthrough which this Pediatric at for Hank valoctocogene frequent Momentum in supporting a who recombinant community stage will last half from hemophilia that always efficacy of two study a before dealt program, Priority designations. we our one definitely therapy. received authorities X be have year. in and Encouraging updates valrox we forefront. costs. potentially to VIII and are development only shortly. is about and infusions Factor face and of option medications or building are need the treatment resulted one of Phase Voucher, result more programs gene of the development approval, valrox in studies, thrilled therapy few the start access of of the of this is area in Review additional provide the hemophilia, both

over global anticipate other completion our with execution a last the demonstrating late and of safety KUVAN. XX seen May. The we both our program to subjects. product, in June, pegvaliase potential have PKU we with pegvaliase for opportunity BLA months late progressed At launch stage was forward U.S. will a the outcome October, The continues Jeff through us year. plans walk and Phase and PDUFA commercial pegvaliase, significant our to for vosoritide Turning R&D last plan shared development our consistent moment. commercial filed in And represent we next in the plans as durability as and expected X/X over of vosoritide, these look data markets achondroplasia X Phase to mid-year. Day as in in we enroll which The and program

million growth the between come, year. million we a a double-digit our income while midpoint. we In to to revenue $X.X non-GAAP for XX% to reached XX% to our between years total range the not XX% be $XX with to steadily productive line improvement was to continuing year, we in but revenues of expect In XXXX from invest XXXX, the in in $XXX billion, In to intend bottom non-GAAP to-date. anniversary expected XXXX results of representing income, increase results. one upon continue to XXXX, should those And summary, to XXth our of million XXXX, most pipeline. compared $XXX results $X.XX to growth only also of forward billion Looking to improve

productivity the the foundation I commercial will As execution call the our regulatory details. reviewed turn who business across even now in commercial I greater and laid Jeff review have I more the and in over for clinical, Jeff? have just will XXXX XXXX, to accomplishments in pipelines.

Jeffrey Robert Ajer

revenues with of in remain XXXX to Other in same quarter, areas the buying Last the for were anticipated on We fourth patients you, patterns. uneven ordering I'm communicated in patterns region record quarter with XXXX we as am in exceeding million particular, quarter billion the in due periods in in fourth increasing in significant XX% order we the of Latin grew Starting well and with only full Brazil. the sales $XXX Thank risks J.J. fourth strength identified compared growth previous that the X,XXX to new revenues quarter opportunity pleased of and total identify continue included $XXX the Turkey product half delivered commercial With there to of in quarter-to-quarter and year XXXX, identified the to XX% VIMIZIM results in to EUMEA we had but Brazil, VIMIZIM, XX% first XX% with North the continued commercial and report and cautious Brazil patients patients is year that America. fact XXXX. continued patients, time. net roughly positive for currently here. year from very for XXXX of as I on breaking $X.X for and full worldwide. rise over the therapy Colombia respectively associated the million and glad mark therapy on BioMarin by reflected increases America

growth continued between and year VIMIZIM revenue towards million we Looking million full full to XXXX in revenues. year expect $XXX guidance, $XXX

revenues Moving Brazilian NAGLAZYME, XX% NAGLAZYME full underlying commercial net million respectively on XXXX, by product uptick continued indicator periods in in to an million XX% and were the of the quarter $XXX increased $XX increases X% compared year, benefited same and an the patients Consistent growth fourth with in for for in in to quarter. from VIMIZIM sales number of in NAGLAZYME. the Significantly, the of the orders demand results, XXXX.

in Brazil buying patterns uneven result, to also steady will remain As anticipate optimistic quarter. XXXX cautiously we but sales a that quarter in be

Our million NAGLAZYME million. revenues and full-year is XXXX guidance product for net between $XXX $XXX

of to the very the KUVAN. PKU KUVAN KUVAN full of BioMarin net acquisition we on pleased year over line of the for results. fourth product year-over-year. global the by Merck of quarter, $XXX second XX% the in Now an sales since increase with revenue million franchise and international contributed are markets the XXXX represented in Serono for top

increase between us America, in guidance full million, increase $XXX North solid year, confidence about year KUVAN an net $XXX in product was XX% results. American over North uptake strong million full an gives year-over-year. the results of of in internationally, and combined commercial XXXX with XXXX over providing XX% patients of paced For revenue by The combination XXXX. were a X% $XXX sales of million, increase

U.S. both approved Turning now and in the EU to which Brineura, was XXXX. in

said products. we for Brineura we commercial when ERT the As got than for is our strategy approval, different typical

and diagnosed while Our going to primary focus The will be that is early have facilitate screened good patients physicians treatment, on forward. focus The they as goal launch to and is function out among are good is initial awareness raising Brineura and to early rolling our for continue diagnosis. anticipated. candidates retain

gradual additional the had And ramp as receive At end sites prepared and countries result, of quarter fourth Brineura. worldwide seeing XXXX, countries steady a are over treatment. and commercial to sites. being treatment of new spanning the commercial of transitioned are We XX patients a patients we XX X to provide

to by this treatment expect XX sites with have provide to to about continue equipped Brineura mid year. We

is efforts number Brineura is early progressing raise driving physicians the in awareness experienced of nicely. importance and expanding the Our The in of diagnosis to community. interest of and Brineura

launch the multidisciplinary can within due quarters readiness of few preparation a things of site new learned complexity that have longer the to expected We over hospitals. than including take first two

very However, We're of we XXX given community. challenges growing we expect the track manage the to infusions. in to CLNX with this the on country of The Brineura we on with across institutions participated Europe in place to-date are resulting the program, gaining launch we part process through steady Behind momentum. awareness complexity reimbursement, as navigating In are any Seizure believe view of program. of the early the programs U.S. and are uniqueness several are CLNX have the expect surgery Brineura, that in related and our and and put being have pace the diagnosis for track and where physician throughout in today through progress country XXXX. by diagnosed we of children pleased planning

in expect resulting million. As guidance $XX a XXXX of $XX result, and growth full year revenue in between we solid million

expect Finally, turning which approval we now for status to learn pegvaliase to late May. of the U.S.

U.S. the a underway be second cautiously are We in launch of year. will commercial that half optimistic this

the a and As take launch. this pegvaliase, approval commercial approach we communicated, potential of will upon go have to we slow

but be choice to an many effort a transition the KUVAN and more interested been studies respond the at average expect to take and pegvaliase appreciate expect pegvaliase, we and knowing franchise. adult you it maintenance the of patient, our price The pricing to patients during and the working annually clinical have we're upfront phenylalanine. patients additional that access a specifics at then is resulting our excited pricing so market right on over phase component a opportunity once the that so approved. would expect get the do price keep currently there and learned the and about gotten full pegvaliase In adults, following. significant of lot and once I of will pegvaliase is revenue reductions U.S., therapy. We share maintenance the patients about of The it that are treatment. induction approval treatment achieved. in for thinking course will worth how initial time to PKU of to the feedback to significant over be proven it get treatment severe and of PKU XXX up titration approval, first as the of phase modest to be of have induction Further, phase We upon we is currently we premium fractional of for the will to of in the through We from gaining during be the to we in through be in that has developing pegvaliase Pegvaliase $XXX,XXX to treatment varying in is to is choice KUVAN, We're with about like plan dosing interest rates, therapy maintenance children. about believe of the net. pegvaliase available about pricing time efficacious very which is be phase treatment we highly has adults excited treatment, would approved complementing of continue of titration will of to

have with in running clinical and working clinics the product the U.S., the hitting sites We of of years U.S. PKU experience ground the directly therefore in anticipate XXX approval. in the trial total XX

look apprised an I'd case our to quarter to of results. revenue May you full fourth outcome forward we guidance provide to was detail turn the our year now, call Dan provide not PDUFA. and in year, Brineura, the financial over pegvaliase with we the so like keeping to on Dan? approval And progress the do with following of of launch As late more

Daniel K. Spiegelman

today's detailed quarter provided point Thanks, results will to Jeff. Since Jeff XXXX our items XXXX guidance, XXXX revenue Please already the full QX and see for press reviewed and Two provide financial year I revenue results key then financial and the guidance. cover summarizing other XXXX term release XXXX. our out. fourth items result and longer

as classified priority of on the sale $XXX the operating of expenses on gain a of The proceeds million. statement is voucher the sale for First an in intangible operations. asset are review consolidated

fourth is Act. Associated by expense quarter. results the with were the also in for impacted the Tax provision $XX also associated sale, Fourth with of a charges there quarter tax Reform approximately million

to million $XX to related the assets XX% revaluation federal on the our tax from tax XX%, additional due to compensation. charge is to in First rate due reduction and limitations deferred of a directly deductible

to valuation we conduct now the company R&D we Tax incentives for cash of income uses $XX million. the the established States, in utilize California plus notes California tax million buildout finished Shanbally from State results year and foreseeable need of of In VIMIZIM, driven generate commercial to We allowance $XXX loss our From cash against tax will included $XXX on future GAAP the line the the million sale million a investments. with San United office for project expense the gross therapy non-GAAP the than $XXX existing State Major the capital related in support Major expenditures for billion $XX full addition, the million allowance $XXX for Based credits. supply our and on the and of valuation basis, XXXX, year year construction bottom facility of million, Act hence Rafael. for the year manufacturing PRV. credits in ended inflows facilities in for the by Reform to and was our that some the the increased of credits. for a full gene quarter in new and cash, annual with facility, in more just convertible this from of an California included equivalents we of $X.XX manufacturing will standpoint, issuance of under of year and net the Novato year

the VIMIZIM remind out In new the by XXXX. and long-term we in you let plan reviewing inventory of approval to revenues million me of and $XXX and of addition, guidance increased Brineura investment due KUVAN financial XXXX that Before increased the at laid end guidance,

and level plus and XXXX reiterate valrox. that and from expect and profit and year, respect then that. rate absolute guided starting lower per products to grow an dollar generally the line revenues said and a revenue the above through of to to growth end With operating end basis, of creating we the growth, in today that expenses, approved margin we previously acceleration after grow expansion with that revenue incremental decade, but of have decade through for pegvaliase bottom we've then at on XX% our they than vosoritide will Specifically, operating the accelerate growth

our consistent In is our XXXX reiterating with plan. addition to guidance, today long-term that guidance

To forecast XXXX through So billion. revenue $X.XX would excluding billion the and current that note represents guidance turning that key XXXX. XX%. $X.XX to growth help and between guidance to remain Sarepta this of revenue payment, revenues this our total XXXX is XXXX now the total analyze upfront XXXX one-time revenue revenue currencies between year. XX% at I rates the assumes compared exchange

in $XXX revenue our revenues million, we of pegvaliase. line addition, net non-GAAP over of guidance United million to benchmark our income perspective, $XXX half the bottom without even and assume we does the believe achieve million at a but In for this guidance range GAAP the improvement launch XXXX $XXX States is can XXXX, to is pegvaliase our loss an $XXX From guidance the results of non-GAAP second guidance million XXXX midpoint XX%. in in

importantly guidance year-over-year. is Shanbally G&A by pegvaliase, basically the XXXX volumes. production between but is in and main last XXXX spend driven swing are non-recurring million, In unchanged XX% cost of in with growth on and points and XX% facility SG&A to of specifically, XXXX. for of unit vosoritide the is increased With a basis full light XXXX to launch rate straight margin current is more of SG&A approval XXXX XXXX, valrox planned XXX of between year spending in More $XXX the million being of by expense of additional both revenue in between The of XXXX XXXX $XXX the the Phase cost online. increase of production XX%. for and infrastructure VIMIZIM continued revenues, relatively million sales Ireland in shows spend guidance to result. global driven area Shanbally, launch to $XXX compared R&D X being per increase compared R&D and related to guidance place, in between charges One our higher about of sales XXXX of is facility recognized coming million year's clinical XX% than efforts costs contraction and KUVAN. capacity from growth X% $XXX in and continued at results XX.X% Our Brineura between the total, drivers some and guidance an

levels. approved, products As line and more get capacity, as cost new revenues return we utilized and utilized we gets expect capacity increased historical fully the to sales to that that of

margins, historic valrox out, improved level below could have substantially Looking gross drive further our could down both our and long-term vosoritide of levels. cost which sales

and $XXX to we similar laboratory capital spending, overall and XXXX between in facilities. XXXX capital be investment and manufacturing primarily expect $XXX In terms to of million million, in

In addition, $XX inventory and support an growth. in to additional launches be approximately investment million there million revenue product and will new $XX to

intend continue that outlays, in we existing except won't or or prior XXXX three refinancing of the XXXX of XXXX per $XXX with to amount our financings Finally, or our they we've repay these do need million recall bonds the setback. equity-linked converts guidance, a believe please in not. in resources product circumstances capital equity mentioned, previously the we to potential that our major we whether financial cash mean Even for additional to principal cash money mature and our regardless maturing M&A,

update call to I'd Now like over to development an turn on program. to Hank? the Hank our provide

Henry J. Fuchs, M.D.

you, Thank Dan.

to call, about productivity proud On months. and and full results occasion others take the their potential. their helping to been reflect a what live the to on innovativeness life XX over last and year-end achieved all I'm moment BioMarin, alleviating suffering has at I of our the of it's

like to rely only people for rest their I'd treatments good valrox, of R&D approved where work. our So and the program my new to colleagues on was year thank who execution it to remarkable before come the a the I to ago. accomplishments mission continued of walk of BioMarin reflect numerous our the on through have Starting team year, is at for with the past get entire the

on You were in from going our talking hemophilia. hemophilia. to a may state we patients we our mild you were We milder the the we recall both our for efficacy severe plans launch dose, studies, GENErX-X and hoping that when kilo and are beyond program to and addition positive X/X achieving Phase well XeXX we proof-of-concept data and were convert of we vector here started of of the having which to genomes Phase GENErX-X with X safety of XXXX which to augmented Last are year, led today observed about the enrollment. per demonstrated program results open

Importantly, pivotal studies, achieved patients our within maintained of genomes that with level rates factor vector these Most At and were the update to very kilo zero be we median have VIII XeXX excited the the point. data in treatment to in with most increase the enthusiasm the start at from and seeing both last for ASH time valrox normal recent patient patients we're severe communities. results for hemophilia metrics of December, of an A. physician after and continue Factor dose. range per Further, bleeding at levels use annualized update

VIII near longest up normal with the results year or also for to the with patients factor in levels the genomes dose vector per We follow-up kilogram three range. one with shared XeXX

tremendous the XeXX within the accompanying lower VIII levels per as had Phase dose point the for vector of and bleeding, established Cure editorial preliminary of to of roadmap published of per patients a with of potentially for ASH, even the dose of in patients nature subjects kilo better ages, at titled Factor an in The or normal As The end The cost time prophylactic disease because time highlighted range with immunity ongoing the capsid, most circle Hemophilia annualized point. sustained England the including enable Concurrent previous possibly earlier vector whole, demanding Reach high ever transfer patients. and the results at an X/X also or interment genome the within the A kilo Journal larger XeXX also editorial of and Medicine demonstrated zero approaching that genomes to liver access results therapy for hemophilia patients that rates for and factor important median inhibitors. factor bleeding of with the treatment patients, of need valrox. to of replacement. risk residual use study therapy New of

we're participation possible enrollment as patients. committed X steps immediate mid-year, dose studies, to do consequence, provide by As the and in any don't are think Of study so to a enabling leader is sites the patients way, Phase We enrollment the in X/X for next for high. in as to the continue thereafter. much stay the valrox update this for Phase patient dose and and XeXX the program Enthusiasm around by very interest an the studies XeXX on field, year-end two tuned. will, limit in shortly

to capability we further It for gene to and developing it's X,XXX in the at believe annually. use ready vector material our other product therapy compared severe hemophilia this built gene have to between facility, scale company has of We completely the expect kilo genomes in to end the and lead capacity field Turning A. study therapy per BioMarin's commercial out pivotal April. XeXX any the increases for patients in supply X,XXX

showed throughout kilo treatment for XX, At set XX using October, vosoritide throughout to children sustained R&D microgram durable we Achondroplasia. Day last per velocity the on months growth of dose. Moving with XX

spinal shared of supporting we compression, lead apnea, take for as addition, that In from improvement and us activities for the potential sleep foramen indicators complications magnum and cord expectations growth condition on such based for compression, continued other daily many data granted. disproportionality in

mean approximately growth the over increase predicted. absolute patients experience periods, Specifically, X growth would their of what observation centimeters over have velocity baseline XX-month

the improvement a part as and update. UL continued of measurements XX-month the for of and over upper body demonstrated proportionality treatment as lower time ratio by measured addition, In with vosoritide ratio

community a The enroll mid-XXXX continues in baseline treatment, now Our in data enrolled in of to feeder second with to and internationally in topline U.S. anticipated and fully in the of degree completion prior the rate the patient treatment reflecting XXXX. half particular. which establishes growth study, study is high interest

half to in our our the the supporting in announced this we benefit expected to begins particular, in year, also derive. in study we anticipate the As first patients more indication treatment commence infant/toddler this of enrollment earlier belief are that

that of as on for us standards developing are in the you March the Register need hold leaders in in patients, XX let the and and goal Federal study those who unmet in know also on fact exists couple and given key to meeting those endpoints expected that to been have name We this we no participating therapy to interested will has high clinical meeting discuss standards planned January key to for is for a where design treatment indication. the just opportunity advocates community, matters. to look of the FDA pediatric a Achondroplasia. and well for in this opinion in The as the noticed of Achondroplasia development meeting approved of the this with establishing year, establishing We collaborate forward drug

direction. disease newest Turning the X program the range, liver subjects escalation share Development We're parts spleen XXX to ago, glycosaminoglycan In and the weeks with Quotient of especially neurodegenerative light from replacement or of data modality as of most study both Part on demonstrated dose well trending Brineura rapid program, DQ of two this young the which progressive in the of part. MPS included results the study reductions. a that therapy the size as we've based children. our levels X now enzyme patients. the the cerebrospinal excited to who We're our fluid IIIB, and intracerebroventricular patients pleased the were in recently Medical BMN very in At from our early treatment rolled WORLD and success the updated right study, is dose the of Meeting results updated both experienced X about we of portion of the escalation rolled subjects from Part Part delivery normal for over for over

we the track for XX Quotient portion enrollment the first to We in Development XXXX. where of of of the this expect enrollment (XX:XX:XX) and complete close months quarter study

User one DQ We XXXX an end We're Act data finally, the year approval. for forward program immune to Drug tuned for expect in pegvaliase. hopeful action properties updates additional J.J. data pegvaliase, of progresses. of so have for stay are first look Fee date as lowering And of so the at in the we response quarter May the the confident mentioned, to the Prescription as Phe acceptable baseline that

current approval keeping U.S. trial clinical for questions. look thrilled the submit that, later marketing application XXXX. European for is in forward the experienced apprised the to year progress authorization We're in would pegvaliase. call Our and quarter are our this with And their open we benefits plan potential potential they application heard European to patients first Union the that the hoping we of with like reviewed operator, now be and approval, our with will for in voice you as with have in to

Operator

line ahead, first Salveen Your please. the Goldman Richter comes from of Go from question Sachs.

open. line Your is

Salveen Richter

for my Thanks taking questions.

just revenue XXXX your So clarify guidance. wanted to

without comment the in to secondly, how pegvaliase with you'll valrox And guidance progressing you've attributed can to what achieve pipeline, or wondering can in then the revenue, on is just you range you be but mentioned just You pivotal XXXX? pegvaliase in regard trials? Thanks. enrollment the

Daniel K. Spiegelman

is This Dan. Yeah.

I'll So honor take your the answering question. of not

So consistent enrollment? that we're continue guidance year and practice, don't XXXX. in going give launched to with to newly practice revenue first we our prior products specific And

Henry J. Fuchs, M.D.

year abundant complete that. of study cetera, essentially in have in is first prepared of sufficiently gating to identified end with enrollment more studies Similar et my The of context, IRBs, factor that's I remarks, the that after contracts, in of and two progressing a site phenomenal the the pool smoothly coming activation as as our as less patient patients in projection the enroll. by this in we or the the said predicted second will interest

patient pleased with track identification. and enrollment we're quite record So the although early, it's of

Salveen Richter

you. Thank

Operator

question JPMorgan. from next Cory of Go from Your the Kasimov comes please. line ahead,

Your is line open.

Cory W. Kasimov

Hey. for Good me. afternoon, A a guys. pipeline my Thanks commercial and questions. for taking one

hope do guess there move that need need and about of what potentially enough side that on data I or that the to on follow-up you pivotal wanted then Hank, And Is before I follow-up commercial. to program. you one, do much just on the DQ for BMN improvement, a So DQ forward? front, ask one then year Is to you XXX think you've have a I And stabilization mentioned? how to see pipeline too? moving you

Henry J. Fuchs, M.D.

little a It's about to talk early to study. Yeah. pivot a the subsequent

on triggered think context was on in I on time I they the data things reminder goes the now updated Brineura rapid United real of just data, A basis in review. actually approved inserts a to on – relatively able data, fillings the XX-week XX-week on States include what more and is for credit the the basis that Europe got the big package put basis of the to XX-week basis that being and authorities health in the to of data.

context – concern in of measuring history BMN treatment tool outcome patients. those we've as context the exact and was don't eliminated that a patients the what that's application. review on in using different XXX enough. was say itself same Development based we're We the in XX XX as of BMN improvements So the made I'd were weeks but the Brineura, about in efficacy context. patients to a know speculate, part to natural is of to for a substantial XXX the we Brineura, that not Those Quotient full cohort same We're specifically Brineura the in of measure have by the big weeks it's premature so improvements why programs, the that

to we've of approval finally, In where there's a size, already good the of you basis BMN systemic addition, there Brineura, activity biomarker remind no liver unlike in unlike manifestations I'll disease product. therapies. biological case of And XXX, of other which the for demonstrated been a has normalization follow, with were enzyme replacement for Brineura the very of

So it's time we're cautiously progress we Brineura. of BMN either another matter of what optimistic a where magnitude that things to trigger application, about made would of think kind stay or an of we've And as tuned. at I were study were a in of ahead case with the the improvement XXX, similar

Cory W. Kasimov

in the approved that we but this see I the on May, about movers should appreciate be how I what you thinking there should for biggest assuming launch can that and about? that's do pegvaliase of commercial thinking indeed and helpful. the mean, is front, we tailwinds the Okay, then slow be some curve, headwinds is product? needle initial steady And

Jeffrey Robert Ajer

Yeah.

So the first picture Cory, the simplified only we're United I next the in launching year. would I thing by is the is say think States that fact anticipating

XXX that's We around approval tailwind. assuming trials commercial be are able patients, to of of to over course they year, expect the would clinical to transition PDUFA, a an so which we the majority our the during have

For patients than naïve XX- phase. maintenance noting would to that weeks patients, That a induction reach some average, longer be titration the XX-week have get have treatment and through something on taken but also to we phase. to period to like a XX

will of patients time, pre-filled microgram of a a a doses be would period XX XX that During variety we X.X and microgram, taking syringe SKUs, microgram anticipate of three a dose.

would per phase, titration during fractional and XXXX headwind. of revenue dosing so year majority to up starting induction of syringe there calendar on naïve the so the fractional phase, schedule, induction a And for depending that revenue week could the and would in so And patients, during slowly be induction titration. eat the bit a to titration only you that treatment be at out one the consider

we clinics investigators is of the States off start XX United experienced gives know have new PKE in are base approved. trial those clinical to us a good and the when using new of of in trialing that enrollment, XXX In pegvaliase so (XX:XX:XX) pegvaliase terms program, experience that patient from, interested through patients the

We to induction titration them work through the guided that I something and approaching patients in of dosing next up would so starting and several-ish, small they initial patients know they've – batches, or and that maintenance think start phase relatively on. then several the also batch

the measured clinical program, So the patient don't a trial of And that'll in that the of then about to through kind clinics of pegvaliase. we States – the have through. education have the for introduction get – United PKU for experience usual XX be process new pace the

patients, initial they patients, to we about having start would pegvaliase good Before anticipate and be patients. fact those starting then would experiences and additional in very trial we guide with with would anticipate clinics deliberate several-ish

throughout measured be XXXX. a So it'll start

Cory W. Kasimov

All right.

Jean-Jacques Bienaimé

into currently therapy, add second in the to that on not commercial might patients are half you Jeff, go use year. of need they vast the majority that XXX full of But the can They this will titration.

Cory W. Kasimov

Okay. Thanks, Very helpful. guys.

Jean-Jacques Bienaimé

Yeah.

Operator

of Your line Go ahead, please. the Jaffray. Chris comes from Piper next from question Raymond

open. is line Your

Christopher J. Raymond

Hey, thanks. I've got actually two questions on pegvaliase if that's okay.

first, curious program adults. decision the approval on Just in I'm there's until to starting wait on pediatric a an

some was direction pegvaliase? you you your decision? pricing per you for of on fractional. would on striking comments apply infant this reason side, of or struck would that you guess thought to for sort the that not I the think or just same revenue color? I'm of of vosoritide, by a commercial driven I have clarify, be by said guys the rather earlier maybe I asking, starting this give there study then, this not maybe Should guys think I with maybe be Can is a logic SKUs. kind that a titration why it by heard announcement and milligram you're Can an patients you say about today's your the And little terms is And basis maybe measure? we drug amount FDA's other for is more while. I bit it's Thanks. that in

Jean-Jacques Bienaimé

Hank, you pediatric? go want to to

Henry J. Fuchs, M.D.

– form – from there that's with safety and pegvaliase elicited protein a going children, foreign the And that's has build in an sort Sure. along important business. is Thanks, a of of wanted another for Chris. an consideration in The different therapy. into which considerations alternative from therapy is blunted ongoing replacement been that's We of damage with other children, pegylated our is response. aspect especially therapy one indications. really our all that's and good in accrual before compared as an context And database and to with other that medical namely the as enzyme PKU nutritional different therefore immune

build of than the as children combined, strong the to adults in as well the safety children. So those is more database, approach is nature therapy it availability into medical things really nutritional wanting of a to I think to measured product and take suggests which two us medical/nutritional, a in the higher with compliance

be Now, clearly action, can that revisited. upon regulatory

Jeffrey Robert Ajer

the syringe a case. the milligram a X.X use to milligrams milligram in the would basis. out setting, Chris, titration on of expectations a of And, XX a once likely subject syringe or phase, revenue XX be of on milligram in and daily dose per maintenance and one that in receiving would receiving we induction then basis fractional to either one and patients dosing, thinking milligram milligram about maintenance pricing XX same day maintenance setting imagine patient why switching the week, they during on pegvaliase this day where way per how syringe patient syringe, point the SKUs, principally be XX dose syringe dosing. getting the and milligram pricing of we're or to likely per induction either syringe. of most mentioned In day; phase, XX at In syringe, price pegvaliase milligram syringe increasing method a XX the and maintenance titration that the milligram pre-filled States, we would United earlier of frequency anticipating different that to have per a milligram milligram phase a pricing XX X.X be up three for per all pricing be on would I syringe would an starting (XX:XX:XX) XX

on during very – titration initially be a during titration higher get would and that through phase that getting would and in range in whether patients to shifting induction fractional induction syringe piece So from basis the price or the fractional the low a a the per range the we basis, further as price period, they process. per essentially milligram be

Christopher J. Raymond

you. Thank

Operator

comes please. line question Go Robyn of the next Karnauskas Your Citi. from from ahead,

is Your open. line

Unknown Speaker

progress. will all couple the you beginning, patients on take how expect about on before place centers is several-ish about to actually you already in One so start question you these questions. these only to for take Krippa them, participate said new when that patients had just titration training through Do too on on And This talk them the a (XX:XX:XX) Hey, clinical expect is a clarify, the do online? did you to that plan pegvaliase and then in expect the they you trials. have take and centers guys. then I of maintenance maybe centers you not for timeline treat Robyn. and patients? the the in can that to go online period them a Congratulations for the induction trial are train

Henry J. Fuchs, M.D.

in. Maybe I'll start and chime Jeff can

we've about had what are to but current broadening and we XX bring other that yes, the clearly is need learning the with the can't plans, final. have the until of we that XX speak really product have apply moment a to quite is the XX the intend we to lot a of what we detailed for And learned plans in captured to about I to as a from the those And online. pegvaliase that learning detailed who want far say hands-on lot. have lot XXX, experience As the centers that label

in for on to plans detailed the the to moment leave And how maybe XX to team from extend Jeff the And to you how I build take So over. the with operationalize learnings that's that. and commercial we've you think context to and is serious wants going better the the got believe XXX. is

Jeffrey Robert Ajer

Yeah.

and I non-experienced the some trial investigators think be think end what shortly things – of with couple impactful document I peer-to-peer that are, through leverage receive experienced would pegvaliase one, anticipate of the approval. one be be in would to of thank of A to tips And you, on guidance opportunities second the to we experience of That the piece May. kind clinical their to share be Hank. working one the is guidelines patients. clinics year for would after peers their to kind a will We find it initial then naïve published piece. the

be the phase, most for and terms that clinical it's we've notion that likely XX goes additional investigators exactly get the Once is say to potential patients receptive dose the at trial the significantly. that occur, we that induction them patients adjustments, to heard XX starting patients titration patients, period events for and normal is intensive them week are it's phase would would to that adjustments, period clinics titration is of the again. adverse the induction where that's to the dose the adverse patients, of down And what sort and before into events, maintenance workload that managing several through anticipate those getting those titration be point starting through thing. the In and the they period on then exactly of several-ish induction titration additional starting and most induction and – phase

Unknown Speaker

one currently sneak question. on can Okay, great. more patients, in if I pegvaliase? any And in adult KUVAN, you. interest Thank seen patients Have from you

Jeffrey Robert Ajer

we for pegvaliase. Recall promotionally We approved. it's until entirely out pegvaliase speak patients in interest their that existing about and go can't have polled not

some I interest, but anticipate can't to I would that. specifically be there So would speak more

Unknown Speaker

Great. Thank you my so much for taking question.

Operator

Nadeau Your Cowen of and ahead, next Company. Go question from Philip from the line comes please.

is line open. Your

Phil Nadeau

you from reactions. specifically one Good mentioned FDA my remarks, XXX. with also mentioned questions, on one for you comments The more optimism pegvaliase immune are the like simply erring conservatism? bit or reason pegvaliase interactions then seemed (XX:XX:XX) afternoon. prepared for was on just in with Is a BMN Thanks heard what the that your of I'm the on your past, and side maybe comments cautious you and than and taking are your coming the cautious curious that. in what today It comfortable we've

Daniel K. Spiegelman

latter. The

current management long worked away experiences But enthusiastic. is of what own with chance patients you of what patients worth adverse that really drug We we're is process. and And their PDUFA until is that kind the division color about in are. more we've review for in, and positive. disadvantages adverse got date of had it of This development the Phe positive experiences providing therapy the on a of think in like, in benefit meeting what people is of opportunity very real detail the hear a over they to they lowering Just what requested came Medical a the where months The The I from the patient-oriented are to terms think interaction looking interaction for agency what haul. Therapy an – review on like, lot. closely little a Nutritional over. had is, away isn't three a from it's a the dig it's are tone I

which you the setting. review, validation, post-approval from planning, process, of the in activities consideration include of specification parallel requirements, FDA disclosures stages As the many FDA control in undertakes risk late labeling during know process public management

of All track activities quite these on are proceeding nicely.

the And not final. so we've the message been the really, where is from process is is change that and no until final process

Phil Nadeau

Day or R&D access you package then is Got data MPS is patients. it robust it. have about natural IIIB? how on the you that larger? history robust the natural that have the you on curious It I'm natural important like had database full And you presented to in history BMN so second that question XXX. that data helpful. think That's very getting XX history that held seems I'm curious, approved Dr. year, At was in last Brineura was I (XX:XX:XX) the that

Jeffrey Robert Ajer

that patients control the the fact going are And reasons FDA's to – academic, go the an different same. in for natural from but tools tools if were of contrast, appropriate the of the those robust. were be about the and were the that review literally treatment. history case are treatment before tools where more that same of is more we after focus, that not will assessed patients that in be substantially using use comparability a industry treatment patients natural for the will control setting similar driven patients, roughly In back tools if you're same set this the comparability and patients patients – comparably by The and XXX, the evaluations – endpoints said was drug generating the were a and conceptually to whereas sized be and But with history Brineura you and for in package we're we've is the in setting. using we treatment, developed the tools, BMN by dataset. talking And

on benefit actually benefit, signals. database that natural actual robust So and be we of – magnitude again, the building think will the depends it that treatment quite for we're history of interpretation treatment

Phil Nadeau

Got my it. Thanks questions. for taking

Operator

comes Deutsche Andrew from Go the question next of line Your ahead Bank. Peters please. from

open. Your is line

Unknown Speaker

for on Yes. the let our me taking for my all add Hello, you congratulations questions. and And is thank Andrew. This Carlos in progress.

BMN these for therapy emerge early the has Following gene of some of XXX, in therapies. up some replacement on to enzyme begun data

how So reposition about direction? compete position think the to your you landscape how competitive And this can franchise as well the would portfolio current you R&D going as forward? in

Henry J. Fuchs, M.D.

Yeah.

drug obviously mucopolysaccharidosis it's mean, worth a competition. bit back been development. relatively have general in of a of little So The for area step taking fertile terms I in

However, disease difficult CNS, disease, important principally to of little been more are given barrier a the nut and family most has bit of a challenging brain the in III manifestations a MPS is or the disorders crack. because been it's some blood-brain the the

system initially As be hold MPS on tried delivery. in you unfortunately that then Alexion evidence unfortunately intrathecal approaches some didn't after similar for delivery you sort and companies having recent had in appear central program know have real. lumbar doses, IIIA had conference or at activity just up device-related Shire the biological world low most effects of a demonstrated It program of like the of of relatively nervous with to have intravenous hold potential know, but those delivery, complications. Also, IV if been the Synageva in approach after gone clinical

easily and blood-brain take navigated. And there not are the think are so home challenges I messages significant barrier

blood-brain company to as in some an GAGs. signals therapy come and we reduction intravenous barrier another initial activity is using CSF and approach taking Now more of to gene address undertakings recent the delivery

the the the blood-brain We can remains need normal slide that correct GAGs, be blood of to of really of achieved accumulation and it barrier. to that the pathological be you have to published species whether seen from

it Our strategy – actually the a right brain. the has enough can is has been proved approach make is into deliver in already. central the nervous the that the system nervous proteins action to And directly We disease into disease. system central where successful

chances. of the competitors So We're we aware will there big the our tell. are competition. to like some time We think challenges but

Jean-Jacques Bienaimé

none normalization of mean, I in heparan that the think only me correct companies if wrong, we Hank, far have other able levels, are levels all heparan so company but sulfate shown normalization. to the report I sulfate reduction a has And of but them been I'm prove

Henry J. Fuchs, M.D.

for important this the disease because Yeah. histopathological rats J.J. reductions in is collect subtotal phenotypes showed And that species Thanks, do of the actually (XX:XX:XX) we animals. experimental in not especially

think you all yet that us. and way knock except pathology really nobody's done we the have to So to normal, for the down

Unknown Speaker

the And At if just side on data Class that follow R&D could BMN effects of X Day I program. the been the on is XXX the if inhibitors. this HDACX how understood a selective Okay. the we Thank may, overcome you correctly, other X you. over up some how HDAC seen with discussed pipeline have XXX and X for and guys BMN

might So the the body expect would in my specifically effects be BMN and and in question what where's you with HDAC side brain, XXX? expressed

Henry J. Fuchs, M.D.

and it our From right Well, different classes HDACs. throughout preclinical the dose there XXX limiting doses able of out histone inducing than are are every – different to achieve up frataxin toxicity, to because of cause studies, of that and degree point design myelosuppression XXX, most commonly that are tissue of cell BMN deacetylases isoforms regulation relevant BMN at to are we're used body, but which the the a lower substantial levels of be that the appears you're appears concentrations to myelosuppression. which of

essential we we will clinical that, pre-clinical clinical based We're Now be to up can required myelosuppression. accompanying experiments establish is will pre-clinical therapeutic cautiously to data positive regulation that. can index, optimistic question on that be a do without work all frataxin the that and say our obviously, demonstrate whether that's but human

patients the make frataxin this the initial is regulation in can only allele, to population will Now that of important an other our to specific be and so up the get one because done patients, of of very is we'll target points the disease investigations here answer diseased be quickly.

Unknown Speaker

you. Thank Okay.

Operator

from the of comes of Bank question Ying line from Merrill Go please. Lynch. next Huang America ahead, Your

Your line open. is

Aspen Mori

pivotal gating expanding for Real factors XXX, do nice. color What into to little Hi. And there moving our BMN Aspen factors on on for Thanks. the guys on are quick securing be reimbursement are It's some getting taking what are for where a Brineura you you ex-U.S.? maybe to development, would there of guys questions. ex-U.S.? think Thanks the Ying.

Henry J. Fuchs, M.D.

per about updates been evaluation year, is going I cohort of to data think won't tuned effectiveness factor gating from for further said patients stay I the be probably first the XXXX, which treated key have an until be the of so that there.

the followed and the the treatment roll enrollment you, of of the Just of year over of activity to going history the be first will Those patients treatment first of why cohort be first Those to the a phase end through in – a data starting quarter will to we're first comprehensive patients take initial the XXXX a signal complete I of will of and treatment for XXXX. onto for natural sort first got signal remind the enrollment XXXX. the quarter the – in XXXX. quarter They'll year treatment from cohort. in that's in

So stay tuned.

Jeffrey Robert Ajer

reimbursement, think XX the to I that [Technical so... ex-U.S. about or Brineura Difficulty] before question noted (XX:XX:XX) your of Relative we've

Jeffrey Robert Ajer

four in those located America, named in reimbursement each a and Italy those at price in commercial set coming of that on Germany, be Europe. markets, we need would of approximately of process sponsors obtain reimbursement Europe prescribed or therapy And through the were we get reimbursements very navigate patients in on XX six commercial approval And through approvals named have The end activities a XXXX, there's the the and the France, to and whole like commercial get of Of patients price to most more from while or Latin basis patient level. ex-U.S. in markets, each to many is that patient markets. for in the two of those patients UK.

getting through are think process that navigated during other navigating of the system of And right markets we We key markets having course in those that anticipate in XXXX. midst and and we the now status have attained that or price that patients system patient to far. to attaining in Brineura commercial reimbursement approvals markets named relative access therapy certain named allow been and patient-like so will easier full that and the

Aspen Mori

Thanks my for taking questions.

Operator

ahead, from of from comes question Chico Laura line next Go please. James. Your the Raymond

Your line is open.

Unknown Speaker

for (XX:XX:XX) One on for Thanks Hi. gene question. Hamza therapy. our strategy Chico. This on Laura taking in pricing is

than about others models various within that you're there As sense therapy payment gene you. space? Thank hemophilia strategies, pricing thinking more certain the make are

Jean-Jacques Bienaimé

Jeff?

Jeffrey Robert Ajer

take J.J., did one? you to want that

Jean-Jacques Bienaimé

-- it. Yeah. Why go don't Yeah. Yeah, Jeff. yeah, for you

Jeffrey Robert Ajer

Yeah. Okay.

results, as on the of discussed positive until capture possible been basis, calculations clinical may think may for and BioMarin's have models product. interest those to United number implement to for So lifelong of to terms how number implementing that that in going perspective, gene to Those initial reimbursement a United is We're impossible the of playing models today. implement. quality be how to States, for very navigate therapies. cost Europe kind those the and number model, United seeing basis, of already government reimbursement kind in able of patients of that's value of to are but that for for. on three in from like as rebate programs don't is payment life probably the principal be challenging Outside States. And value valrox payment a initial be through two provide, of a time, as to the models single models might models adequately. a the what are expensive variable much account and value something some States that easier There that's offsets pricing one in our a treatment I is price are regimen a the for with to U.S. payer-like key solutions In include out respond which solved payment in approvals barrier the over problem

capture we're and of capture much dimensions, to all across will to as that different allow want us to as we value reimbursement three models those value. that possible So open

Unknown Speaker

was That Okay. you. Thank helpful.

Operator

from from question ahead Capital. of Your Go the comes next BMO Ian line Somaiya please.

is line open. Your

M. Ian Somaiya

Thank you questions, for two. taking my I have

and products or the on about you've valrox First spoken enthusiastically excitedly vosoritide, past. which two about in

of can second structural or whether development. uniqueness manufacturing the that the to And required, given those might question from be you curious there commercial was a investments be if sort required to are just products business type of going the marketplace, in standpoint? what speak Just changes investments, are the on of

products, a both influence might of pursue? growth your these high how a at of growth clearly You're sort associated into does stable type you and of that of the assets period with with visibility BD opportunities and decisions

Daniel K. Spiegelman

is stab want maybe So neither vosoritide you and the with what question. be BD I'll the than dealt we've J.J., different structure wise, a Dan, this valrox take question, Cost at structure past. would cost then materially in maybe to frankly, the nor do

out vosoritide. you to We've would either be and manufacturing to can different that, even we a for past, manufacturing resources we for demand know, force, elsewhere. similar of from a X,XXX different be standpoint, is and patients Commercial here There and we've is the but construct being are sales a in about in place and or additional that done. utilized year all same things to call X,XXX structures commercial what point capacity do the actually infrastructure to expand potentially a able initial the As scale facility. talked plant handle could

J.J., you nothing up business dramatically that take different to development? lined we're ready not materially for. So want or and

Jean-Jacques Bienaimé

say significant of would projects stage be transformational relatively would however, I In pretty ongoing – basis, be now, move year, products we products fact considering it's size continuing business Yeah. are, to projects, for the on to our deal there for very develop or will will peptides, I would of add have strategy continue in as combination very, a (XX:XX:XX). disorders. of genetic with this large very I to or within the that space, our time the significant we or large because end stick first-in-class course, analyze plenty planning low revenue overall billion or molecules, for molecules, develop this would know going gene front, mean, rare be opportunities and interested ultra that this announce for the will appetite say the earlier which new new mean, pipeline you orphan of two coherent probably space. we we're projects. best-in-class that orphan unlikely or potential But the it we of we, the obviously, on is always I are small an one would to opportunities or of and likely ultra-orphan year, always population. we of opportunities this before are development small a we I at company and much (XX:XX:XX) are patient shooting are $X.X say But at therapy. time, the strategically

stand where of kind today. that's we So

M. Ian Somaiya

J.J., is the mortality? or to likely comments you additional just what are speak that? your to unveil, they or to regarding address therapeutic And, based Can areas for on what the target you programs plan the trigger

Jean-Jacques Bienaimé

too gene I we want said but will therapy one a But could mean, one the for them. before there think is at much, another I some doing R&D are – that think have – work the on mean, I them I PKU. year. of comfortable end mean, announce we that say we I of I the day that at probably project don't of critical we last other feel to one be least potential to

move that the us have is the trials. comfortable what's work towards move towards and makes when activities are for that done clinical all to we pre-IND analysis, willing initiating later preclinical all So we trigger the that us and

M. Ian Somaiya

Okay.

Jean-Jacques Bienaimé

a for will will but likely the getting XXXX, year. be end them of point early INDs or two stay it or tuned to INDs, not of going on one now And be between likely we're that and XXXX XXXX to the it's They happen that. so

M. Ian Somaiya

for the J.J. Thanks Sure. and additional color, Dan.

Operator

comes next William from Blair the ahead, line question Company. & Lugo please. from Go of Your Tim

Your line is open.

Unknown Speaker

a a have, that we was just able confirm full the dataset that (XX:XX:XX) Hi. you (XX:XX:XX) were very on larger Lugo. for and Very the Tim or see hold (XX:XX:XX). issues dose BMN filter a concern XXX with fix you reassuring did WORLD, out? at the in delivery deny to the though, nice to line that that or straight One was, once clinical the can Sanfilippo was core potential on rumor study

Jean-Jacques Bienaimé

to Hank, do you want this? talk about

Henry J. Fuchs, M.D.

ahead. lot with study, world. no moment do we anywhere, Yeah, regulators regulatory of around impediments pleased to In sure. have enrollment we're we the work Global steaming a about the so

Unknown Speaker

It's Thanks. deny to it. good Beautiful.

you product dosing? dosing that an The would estimates of the for if you second versus envisage pegvaliase regimen you pursue in And you what terms how just in-office at-home dosing, think and determine an are, peg the be? sales do are question, to looking would

Henry J. Fuchs, M.D.

at-home me pegvaliase. we of just aspect and was Well, anticipated that would be absolutely let uptake conducted say a was that critical that so of that study, the study an

so option than we no study at-home really other And for is that did. there that's the anything because

Unknown Speaker

and Beautiful. year. Thanks questions, very the much great for

Operator

next ahead, Stifel. Go question of from Stephen from the please. The comes Willey line

open. is line Your

Stephen D. Willey

that more the was could are Yeah. proportion on receiving I I XX mg access, comment Thanks XXX wondering under either on the patients are expanded of amongst Thanks. me squeezing for what specifically the trial the mg drug maybe the versus XX pegvaliase distribution in. and patients just you doses maintenance guess of dose? if those

Jean-Jacques Bienaimé

Jeff?

Jeffrey Robert Ajer

going just label. any to it give read I'd the is on of far all, is information that as product we're to a first say to as trial how be by information. I speculative other XX and we confidential majority marketplace, that, with than remind And you And the satisfied milligrams more milligrams. until the really are not going FDA, that would say the What patients distribution vast the projecting this XX piece finalize of give of important to the you actual the of is

have in bit of we I more the when the hand, product think the little time be of we can approval concrete. label the a actual

Stephen D. Willey

Appreciate it. Thanks.

Operator

comes question from of Jing Gabelli. the line please. from Go next Your He ahead,

Your line open. is

Jing He

Okay. for Thank questions. you taking my

and I'm the decrease function. obvious stabilizing volume liver ventricle and XXX, in trend wondering BMN on First than less volume are brain size just cognitive

I and these is guess important how think also mg So do enough? are measures clinically, you XXX

Henry J. Fuchs, M.D.

ambulation effects parameters, efficacy parameters important the milligrams volume. a Brineura, of want part. example, CSF GAG, little case surrogate that on far as less and parameters. of normalize XXX the We ask efficacy. milligrams saw parameters we the too of in XXX don't me and more much. progression to let the And others I on other of with efficacy underdosing second you start by so suppose example, and dramatic effects whether a for on the quite dramatic stabilization brain be little of ambulation, little mistake for are But main have the loss maybe reading clinical could therefore too make totally the question made CSF clearly receiving loss then of Well, of might

carry in good to we're today, our BMN is they that mature. when XXX variable and news ones what were the So program outcome the is cohort that's hoping observe fully clinical the important the

Jing He

your help high driver you in us mainly take guidance? end Is guidance? low countries? diagnosis Also, versus achieve could of Brineura end the or What ex-U.S. launch U.S. understand it to would the your your XXXX in

Jeffrey Robert Ajer

a really It's of combination those variables.

of of in And across access to inside this reimbursement registrations time. noted and approval of so then we we're also as States, and a the regulatory pursuing number the markets have We're we're outside States of United early I've those and We EU United price systems pursuing additional now, markets. identification the navigating at have treatment.

we're a So multivariate it's midst in and model the really it. of

Jing He

Great. Thank you so much.

Operator

next from Vincent from line the Your is Chen Go please. question ahead, Bernstein. comes

line open. Your is

Vincent Chen

follow-ups for questions. couple taking and reimbursement. my pricing of you on A Thank

key capture you for the pricing. to The of multi-year be earlier, as is, longer-term potential alluded therapy can first one value-based for would gene you aspect a value

to to comes get path What's is from to and more of optimism where arguably U.S. gene when a bringing in therapy further able level respect of indication. the we what pricing a relatively ahead You're market some today? with these are it structures to large for being to your achieve the creative there

Jeffrey Robert Ajer

Yeah.

strategies much in there's general provided thought pursue of I earlier, quality us of terms basis that's that noted value clinical valrox. the that think I of of allows the as and program to we outcomes. outside of in interested and potentially the creative So – life United highly offsets, to States, so more and on Value cost is motivated but typically value by better we're capture offers this that

happen. before do capture allowing vehicle the be right, So absolutely programs solution can reports effect, creative In some for like of to all logic by on the projects there calculations been in pricing advancements would that implemented be us CMS like these on that gene (XX:XX:XX) Medicaid are in based a could to That be fixes has on that original some was solved for that manufacturers on BioMarin. could place an would or by that this approving and we sponsor States, want of for United that. submitted to needs XX pricing a first all legislation those A There therapy could these Medicaid the States. but be the system in so-called that more durability pricing across implications the innovative value be for years programs put potentially overtaken three legislative United practical healthcare of must based to that today. time play based a And be ago demonstration example, you're over potential excellent dimensions. do

cautiously we're us overnight. that, to for So but optimistic, I solve happen think allow needs some to there that won't be happen to and they things probably

Vincent Chen

And there? forward between something how plans? change. sounds issue about the that's portability the legislative moving that What's It with folks would take like path

Jeffrey Robert Ajer

of gotten that have can payers I subject. from the feedbacks on that tell some we you

for a therapy wanting that in for heard a problem. and The free problem problem, to doesn't, to view have the example, upfront with for problem they're relatively that major interested cover free they're a flux and sorry, gene pay we initial is a the got would as of them. the four something subject creating It's model. I over portability So of the it's their but primary time to – about barrier or proceed model problem control payers payment patients out of being, patients, not they're ability be to then or valrox the they rider thinking one expect in that same of to has rider primary flux rider for another insurers free the and so

Vincent Chen

pegvaliase. I then, guess, And closer question see. a a I just little second – like getting see. I on

Daniel K. Spiegelman

me. the Excuse get if the Can you others back you in to line and questions, let ask don't mind?

Vincent Chen

do. I Yeah, Yeah.

Jean-Jacques Bienaimé

Thank you.

Operator

the ahead Kennen line of Mackay please. RBC. comes Go question next from from Your

open. is line Your

Kennen Mackay

mentioned? Hi. wanted had Dr. that you perhaps to is surrounding from again, One Thanks for this study. that to and used IX year, to on taking growth to again be whether U.S. the in this we recent term, sort pegvaliase the I the here is a in unique perspective get KUVAN be And of this had saw study year-over-year going pigs concerns long should we the Jeff, monkeys, that X% you specifically unique Westin's in transferring safety for the of AAV pegvaliase the one the KUVAN the or safety. to thinking with market your Thank we high-dose growing the sort beyond preclinical should question. is the anticipating from premium had on for that Hank over lab AAV really on that the (XX:XX:XX) vectors, used the you there of growth something article for you. or and about last come then patient pricing

Henry J. Fuchs, M.D.

Yeah.

it's, So compare trials to as to it hard clinical is across compare across impossible as say, and agents, I vectors, species. capsids of humans would similar in different

area, For are may we I have (XX:XX:XX) know, there consideration think on area the the relevance we're and not and that for doses our – human muscular an human safety atrophy, of I data clinical the the that product. don't in of be we're some findings, that using, safety important our data

Kennen Mackay

you. Thank

Jeffrey Robert Ajer

to your And question relative about...

Operator

question next line... the comes Your from

Jean-Jacques Bienaimé

finished. not We're Jeff?

Jeffrey Robert Ajer

today, approximately that focus At over Relative are there's on be treated one the presently to patients launch, adult X,XXX potential presently question of States about PKU adult are on KUVAN. that your United third two-thirds patients which being in PKU of are clinics pegvaliase that will our time, KUVAN. in not

access and to So on the we revenue incremental delivering a market, portion than PKU very be incremental would PKU that we will in more think focusing total. hard that of getting bigger be

would well years. adequately adult KUVAN like trial will face that controlled we the on on motivation to as limitations that we patients KUVAN be to our not controlled logic pegvaliase launch, KUVAN Beyond are or couple help to exclusivity as they likely have a in on not a of

Vincent Chen

you. Thank

Operator

Go from next comes the please. Wang ahead, question Gena from of Your Barclays. line

Your line is open.

Gena Wang

the questions. taking would quick for you questions. two I my Thank just ask

So pegvaliase revenue XXXX include one, billion any confirm assumptions? of to $X.XX not $X.XX just billion the did with wanted guidance you to that

Daniel K. Spiegelman

No. No.

what within peg so is we obviously I we is of range think our but the the we – there. but can said be assumptions lower, to without try in express, within about range does still pegvaliase, it pegvaliase think we include

Gena Wang

for Okay, another rox regarding then sounds good. question is And hemophilia A. the

Phase the trial, for the product? only GMP X will So receive patient new

Henry J. Fuchs, M.D.

out you, at a include with commercialized The started the to simply from was of to trial contractor So process the first commercialized of same the our purposes. to If portion made scale study. of be that smaller part pivotal us of to the by process. facility are the manufacture. of for size patients the Xb contractor using using bulk can material data expand trial the not believe the the in Xb to patients will the from comparability the manufactured sufficient the enable commercialized study the treated second Novato remind BioMarin larger who will at part be pool we but to We the turns facility of the of larger trial that scale case, the that

Gena Wang

the... then And Okay.

Operator

of the ahead, Vincent next from please. Go Chen. Your question comes line

open. line Your is

Vincent Chen

One quick question on pegvaliase.

costly? the potentially likely are might is some payers (XX:XX:XX) that pegvaliase you if From this speak what potentially and or patients by guidelines a towards there you pricing? a require hearing to for heard, payers, what you've KUVAN vary As coverage are look to more about pegvaliase and could fail launch how diet indeed

Jeffrey Robert Ajer

Yes.

comfortable surprisingly would the So are run KUVAN. with Generally with And the PKU, we've up an payers very notion from are obviously say our adequate payers. we very not have in of for that's PDUFA conducted the lowering and levels of as reimbursement of good gotten their comfortable date, so payer phenylalanine speaking, measure pegvaliase. to payers research of managing a feedback I mix

So baseline next also potential generation going into that approval sets of a good therapy. a the

policies Beyond hard that, kind would or of coverage variety and policies, gotten some that of however. board that step on see could failures but policies, possible, of thing. suggestive be It's strict across or creep therapy a the we but of of feedbacks we've in, of in that in sort KUVAN terms of initial coverage nothing

Vincent Chen

very you Thank much. Great.

Operator

There are no further questions at this time.

Traci McCarty

final the goodbye for say then you. turn J.J. Thank his the to and we'll over like make call We'd to comments, to day. Okay.

Jean-Jacques Bienaimé

Thank you.

you, Thank So thank you, operator. Traci.

record progress only we revenues position valrox to $X.XX in will expect hope for a pipeline among our rare billion. growth program, as Not to and billion. between In leadership XX%, companies. pegvaliase PDUFA, we XXXX late-stage XXXX. summary, was established billion line in of development, commercial every defining firmly our top XXXX, data In the year did for business we BioMarin In paving enrollment development over full revenue our way year across $X.X anticipated in vosoritide disease results to So revenue the guidance late approval. XX% we May a $X.XX of to scheduled continued our XXXX, a see receive at U.S. to

all providers, of with are or with pegvaliase successful who well care trials. experience either years KUVAN have clinical launch working the direct participated XX a to equipped patients those executing We PKU the directly given and our in committee

followed in BioMarin. support, the We're late-stage quarters. like ahead we to and for at very is Thank term, approvals On coming the be up this of of the energized the growth next our business, forth keeping research any you organization forward development, working growth candidates, set across The team and research later bring side year. to of R&D announced of the that execution which could are BioMarin you near vosoritide. diligently commercial, manufacturing look opportunities our our steady for confluence of by accelerated tremendously and lay over valrox your by programs upon apprised the continued

Operator

conference call. today's This concludes

You disconnect. may now

BioMarin Pharmaceutical (BMRN) 23 Feb 182017 Q4 Earnings call transcript