BMRN - Earnings call transcript Q1 2019 | BioMarin Pharmaceutical Inc.

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Operator

Welcome to the BioMarin First Quarter and Full Year 2018 Financial Results Conference Call. Hosting the conference call today from BioMarin is Traci McCarty, Vice President of Investor Relations. Please go ahead, Traci.

Traci McCarty

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

quarterly today team Research the are Bienaimé, from the note, Spiegelman, of repetitive length. call can Vice not management On Chief so questions Officer; Hank the Financial call Worldwide BioMarin's out Chief President and President, feedback Executive request time, to Chief hour Commercial President try of of Officer; be try Technical Executive and call your and us get J.J. Operations. on everyone. Executive our to Jeff and keep portion in respect call from Chairman Daniel Ajer, we an Q&A Robert and Please Officer; Bear and for Vice based Vice Baffi, Fuchs, with to as that we to President Development; we during the last Executive

please If get a understanding. e-mail call you do right Thank send and back get you your or not your question, for I'll an me give me to you.

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

J.J. Bienaimé

joining thank for And Good us on you, Thank afternoon. you the today Traci. call.

We launch action the quarter. with U.S. Palynziq important PKU. early has opinion pace of to Palynziq we solid XX% end topline with million very positive last Beginning We on horizon. corrective first continues commercial in very $XXX XXXX, end basically. result brought from with the to of contributing begin commercial during over product, XXX many that and that's year-over-year, that received an the XXX adults Recall this of of quarter the year, will results are a quarter were our likely QX Palynziq grown at in in the us this CHMP XXXX pleased catalysts of our the BioMarin for first the European quarter. patients seventh a product QX market Commission of XX% increase first of year. strong, the in having the Palynziq The fourth a quarter base to to at second quarter patients be the

So the we are pleased U.S. expansion of very with the market.

order through [indiscernible] actually even patients is our phase titration progress in Palynziq. continues maintenance patients better. from dosing Actually, their been and efficacy optimal for the life and importantly, changing more to results-seeking to motivated for them Palynziq have and the we're is to that achieve work Importantly, highly therapy hearing what life-changing with

team preparing launch and this On Jeff the are later a heels European year. of potential his this success, for

Moving now Brineura.

said of accelerated Phase from we X have quarter, study support the As last patients would we treated approval. the that subset

extremely been gene the A. year. has in We on based for study patients, enrollments quarter transformational are this complete approval people the therapy X track characteristics full hemophilia for on severe potential from and positive physicians of entire the treatment living of Feedback also Phase with to third payers

at are operations, excitement whether the we a data groundbreaking in gene accomplished that teams BioMarin. the work program and in therapy. here hemophilia to and the we is field our literally hemophilia by to to technical as R&D, truly who accelerated across want As or clinical of history making acknowledge organization and our potential continuing readouts across get is approval in pathway, I being community valrox to a the and closer advance decision pursue key not build

anticipation. to the proud developing emerging we with great an of really therapeutic look valrox forefront modality, the and to be are forward We milestones again at next

moment. to stages to with in be the end vosoritide. achondroplasia to the say, completed are Needless the final the vosoritide year. commercial if on potentially very current results the development of at briefly are needs just an Phase year, global the will update XX-week last around this nearly the excited XX,XXX we in of enrollment We corner and arm vosoritide, could our approved a treatment X the in children of of an trial with of program address which Hank Turning territories. provide

are potential to -- potential represent over contemplate and for These As one are is over trajectory near front development we completion products three patients years, of growth our both programs. or we coming both XX,XXX BioMarin one across commercial center. global transform these footprint. opportunities and anticipate to The as to combined the valrox vosoritide of our exciting

the Turning today in track products, this current valrox potential we to year in $X.X on billion on contribution outlook approximately deliver are approved $X to XXXX vosoritide. a from financial billion revenues based before or and

an provide programs. Now, our to would development the I over update to call like Hank? Hank on to turn

Hank Fuchs

J.J. Thank you,

Europe. CHMP on Palynziq X that adopted We for for authorization a announced company's Palynziq. the the for opinion First positive application March marketing

release long-term shortly approval. noted forward to improved. Importantly, treatments, learning making Palynziq European outcome the Commission to their psychiatric opinions after and continued look in press that PKU We the of later the and available with quarter symptoms patients' this patients CHMP European cognitive

across all our are progressing smoothly. global vosoritide, studies the underway to development of Moving currently program

opened families children, dosed to has enrolling in which interest X, is study XX XX by enrollment will year, age, also followed Cohort age X treatment. X the will to Cohort a X, of months months compete children year. age, complete enrolled patients with participants of to level and newborn shown in the of recently have Our youngest X of this up age. this includes children years of rapidly, X, Cohort which most subjects to enrolling very and X sentinel months high XX enrolling the includes of as

apprised We life the keeping to there most you may we committee thank in achondroplasia. to our for of participate especially progress for the advisory data this our early who at towards look dramatic many look support progress, FDA the families our stated forward to afford last want treatment as on spring, benefit X we meeting strong Phase also in of As year. have study. achondroplasia children chosen with reader later is our And starting

valrox. Turning to

patients X As subset of results studies valrox. J.J. from could whose accelerated been the with Phase all treated said, support have an approval

addition, campaigns we reiterate of intend of package. our In coming the for filing the in our now to expedited process in Ahead the valrox inclusion new communicate the and update are X CMC want how analyzed manufacturing now for required to completed being over I months, have information.

invited the X. which top of the X data X management BioMarin and in usage any the that valrox we World remain Factor are to the bleed Once annualized update we line on place put We at previously, for VIII the valrox data. Phase we data, Phase all Federation comprehensive May. Factor rate since a completely see year precludes public assay, levels -- provides Hemophilia As X Phase not communication X VIII the everyone the stage year X and last seen access determined will have by plan at data said on chromogenic a Phase our data update together a team we previewing both We've to X data. have X-year [indiscernible] including valrox

any we As in for is late interest potential possible breaker abstract line front granted should level without at and late have the line running breaker a have before included that communicated. we you there understand should very communication X-year assuming top-line detailed time. much a at update the we information We presentation additional X, X, one. the wasn't high expect in is Phase another late we indicated, as ISTH, late-breaking ISTH, a that The and reflects will that, presentation breaker of June communication the that submitting as be After that will is granularity provide a provide top top deadline update. that so we already public

per So decision very will meaningful an of pathway communication breaker. to a chromogenic no as States. data VLA valrox of describes number that the demonstrating discrepancy [indiscernible] and statistical and and following part subjects filing United component; X continued provided for between stage be Phase study patients the package. at the will X assigned best subset IU the least and provide assays the X that end potential weeks; the four, the demonstrating similarity not deciliter be the or XX from the decision late data XX approval on the The be the in the lots at campaigns the will from to across comparability durability, before but accelerated pre-specified we'll including this will "checking the regarding That all off" need threshold to our Phase without been or package; be safety do components next you one, integrity the pursue in three, supplied manufacturing of two, our jeopardizing whether number to the achieve of based on filing year CMC of has including:

the you summarize, a should data the expect finally, get whether components year decision an update on top day should accelerated of Should outlined. late the granular Phase not end that X, line X. update the breaker late you expect it on reflects expect path deadline, the or ISTH forward you to submission a presented the by to of in And X-year time Australia. a we So, June breaker, more by is our learn ISTH approval of presentation pursue based should just

excited to We quarters. becomes organization very next over the weeks, developments you, is turning come months when to other valrox this few look in what year. seeing and data And all And the later like finally, it R&D are about available. expected forward to the we

of into phenotype. within drug putting weeks, the leveraging initiate the shared preparing based for XXX. are to to normalized development XXX much that and untreated We've expected Recall from treatment the January candidates experience beyond of programs development BMN our studies is XX we Research In clinical years to clinic we Worldwide lifespan the forward conducted rapid to in organization. preclinical extremely mouse. were human well look the with weeks, therapy of on are We with anticipate stages Palynziq, summary, close the commercial with productivity of and the BioMarin out our PKU levels and manufacturing demonstrated experiments data continuing enable ENUX ENUX so with over We to out Phe effective final phenylketonuria mouse coming material. valrox past of our our the this which the scale pleased model, we products, BMN the which development of use to best new focus product filing and gene different on research how flow IND to learned organization. X in We

our continue for vosoritide I to and rare benefit valrox highly prepare with good we are for team the the anticipated work more patients my and disorders. As than inspired of readouts, data ever

Now will Jeff, more detail. the Jeff? call business in to commercial I'll turn over who review the

Jeff Ajer

Thank you, Hank.

of U.S., injection, that performance pleased in working very the enrollments, As and revenues. we with date very marketed to revenues and team's the past Notably, followed this the I Starting from with million In performance The contributions patients XXXX, the totaled are population. product phase patients Aldurazyme through first XX% we in net on Palynziq, year-over-year. of regions. with the begin we XXX we BioMarin's launch. move through. the revenue finished focused across as are XX% all brands adult over majority $XXX principally and launch titration, other for the all first contributions am representing in-clinic from QX grew team Total quarter, still and brands pleased continuum quarter included of with the initial managing by the is the trajectory enrollment

receiving in So very less approval with since commercial U.S. Starting reimbursed strong results a the patients. year than

quarter, naive end formerly of of XXX end, pipeline As patients. reimbursed total enrollment patients nice to At an and from we of XXX very and XXX the transitioned Of naïve first with clinical Palynziq. our quarter Palynziq. those, injection; patients of a of their there the were studies shipments first on complete new had XXX additional are pending

in in Palynziq time from Our adult Kuvan benefit efforts lowering were will our last revenues community job confirm increasing patient on enrollments patient unique Based Fully dosing, are to additional might team convert PKU QX we now of successful. to an patients of quarter the initial be execution to launch who The been past tracking up summary, are Palynziq naive enrollment. in tremendous be were Palynziq net United Palynziq phase, relationships from XX% quite number the promote results of there titrate doing the XXX of U.S. Palynziq according going XXXX. driving least product that leveraging have is a a revenues for plan of patients XXXX. the patients clinics pick new of and now required those the U.S. plan. uptake and treatment had to million is to the in at the X availability that Kuvan daily number commercial can patients with year, quarters Phe we turn $XX.X to deep the year. conversions. States We of experience In the for of next XX the in the clinics to our focus PKU began in to are maintenance completed expect Considering the dosing, remainder first treatment we that and achieving typical the

increases representing Vimizim. growth, XX% year-over-year. patient sales $XXX.X quarter X% Vimizim now all other regions. revenues was in to first in Turning year-over-year with quarter, starting from the million across Net benefiting growth contributed product the

For the represented This a XX% with for coupled patient well-established growth growth. totaled revenues $XX.X brand, X% year-over-year million. Naglazyme,

As and patients growth difference largely are the to timing. with Vimizim, revenue between order due

Next in well seasonal a experience $XX.X over QX. $XXX.X in as net to increased guidance track decline timer revenues. Palynziq uneven late due the in QX relatively order revenues, brand for XXXX. We anticipated, year net insurance achieve the flat increase due Brineura to million And million, order contributed changes finally, X% United keeping fiscal in net -- which patterns as product on Kuvan. conversions. to in claim was States, In revenue Year-over-year we as to product product an QX saw

Importantly, QX in efforts the XX. raise progress bringing patient to in new the good saw country of addition X awareness markets, count suggesting that was year-over-year Net in diversification with are patients of making our CLNX. to growth growth XX%, we

revenue were I'm We XXXX. throughout results and will very on expect and quarter encouraging, growth All-in-all, with all of the that this performance fronts. rest first continue steady strong pleased

Dan? I'd Now, results financial in the turn detail. like to review over more call to to Dan

Daniel Spiegelman

Thank you, for Jeff. results for Please full the financial see today's summarizing our press first quarter. details release

already achieving cover top will billion Jeff As for XXXX the in solely I our full on other results, individual communicated QX Consistent anticipate currently approved we based reviewed goals, revenue $X.XX would quarter. $X.XX midpoint XX% to first be line on year items has with growth billion. product This the products. of

above and with total and revenues J.J. grow that should additional take be billion, significantly contributions the approximately years expectation will that as addition, in $X XXXX an vosoritide XX% valrox growth In reiterated, the beyond. rate from us

results. turning Now, to quarterly

were first First to a revenue of $XX.X item that Jeff did not entirely cover. product QX Aldurazyme reduction XXXX revenues quarter million of for to timing the This XXXX shipments below due to the Genzyme. the XX% level. of was

we Genzyme of that went time we recall in compared revenue increased the are first accept year, the rules and reported by $XX.X from delivery GAAP quarter required net or new now the full when the XX% Aldurazyme us, sales same recognition XXXX. from may of $XX.X to user. recognize that product last the the to million You up in place revenue the at our estimate it quarter under sell end Significantly, not to product will Genzyme receive they into to they million

with compared These which its these our by later their Brineura Phase year, reduction study remain late-stage is year. of were in in preparation trends largely is of R&D in driving programs. accelerated Palynziq Moving quarter the R&D to with the level, vosoritide Palynziq XXXX, million Phase in the full looking for year, less and BMN expenses expenses, to valrox XXXX QX should U.S. XXX of essentially our R&D These a as expenses XX% unchanged ‘XX year forward and first the of expenses. and of to stabilized, than first the reductions near a over of and million. in offset marketing million. X spending tralesinidase $XXX expenses increases halfway have launch launch manufacturing and XXXX even and potential quarter expenses R&D to following ‘XX enrolled, product be fully valrox, last still to increase And or QX which On decision full look track progress largely the XXXX. a and X at Sales to full levels this continued guidance programs with we compared approval the on alfa we year a $XXX launch $XXX throughout due continue enrollment

expenses slightly for of prepare marketing prior to intact. of levels SG&A as in continue full will the guidance flat $XXX in However, remains million QX launch to Europe $XXX million year we essentially $XXX expense and the and were valrox XXXX. SG&A Moreover, year expand compared throughout while and to sales million vosoritide, Palynziq quarter,

probability tax line net increase a progress XXXX, outlined, bottom to million first same the benefit to compared just a Serono. due a in milestone SG&A consideration and continued million. in loss to of primarily the net expense of GAAP a Merck income $XX.X in Palynziq quarter quarter of improvement of GAAP operating and the income the quarter in the results. slight certain due We associated Turning of expenses million an increased the loss first contingent in to payments GAAP was XXXX million over reported $XX.X $XX.X non-GAAP Non-GAAP $XX.X to increased reduction as

breakeven GAAP we result For remain $XXX original of to income non-GAAP results, and look for our GAAP guidances unchanged basis $XX on a and million million of to $XX million. non-GAAP a both and $XXX million nearly loss

to working our GAAP at end with usage capital consistent we with the similar be results, impacts investments and end XXXX. expect balances have planned And limited levels, cash timings current small year in while cash our cash at ended $X.X non-GAAP quarter can of Finally, and cash level, QX billion. actual the on to the

and remember Please for p.m. X:XX feedback, call lines. by operator of start this at will to we on Eastern still tomorrow to reach the to If to the your like please wrap mentioned open question. and open afternoon open that or request call, we the to would follow-up order call X-part I the ask limit Now, as Traci this questions based the keep end call have line. up free plan questions, you you Operator, single to timeline, the out to Traci please yourself In to the morning. we that feel time. this

Operator

question Our is first from Bank Instructions] Ying America. the [Operator of line Huang from of

Ying Huang

question rule. X abide the I'll by

the the level So maybe, about update. the A focused importance have Factor bleeding on from X-year because won't squarely ask investors Hank, I rate, of

So maybe agency how bleeding discussion with in rate your is and the payers, consideration? also even in important their

Hank Fuchs

pretty important to the rate's Bleeding FDA. darn

reduce scenario with For the it. want ongoing non-hemophilic no do the of of notion no the basis statistical that but support is likely tests the reasonably bleeding become rates from about tests to important there statistical approval accelerated perspective range where, approval, the to a associated clinical approval endpoint bleeding, confirmatory to on Where Factor primary They Factor is levels the it's evidence U.S. in full have are bleeding. for there's regulatory in really and are the trial. achieving

part for are in U.S. the they for test the statistical for likely test a expectation, no of full approval, reasonably decision-making, So accelerated regulatory statistical approval, full

Operator

JPMorgan. of question next Kasimov Our the is Cory from line from

Cory Kasimov

we about already Factor what or we in? the in confidence But as to I'm not this update valrox good. would to VIII levels a which give ABR? What to be you have of this not in report, a that bit on of and ask you you well. are what surprised terms closer you program, hoping would confidence more quite a when get are speculating success wanted early lot investors X-year I'm so much you consider see might good what to obviously curious, you summer, And as

Hank Fuchs

morbidity, bleeding bifurcate, were going would system care I about of Cory, the far going X-year experience, And most That's about. rate data. care to a important the won't patients if but out be the reasons about thing the for that by to payers what costs say that care a what they to clinical are: lot sure of update for X the bleeding other lot one, data. what is so money, not I it's say to the that's things, healthcare that's they're

innocent or particular that. And problematic way great. if is a if Factor speculate a and joints whom in third at little day breakthrough at of type, not But all and to And so there about premature about patients that about have be patients to understand year the we'll breakthrough activity, that or all. of to going of no it's bleeding, is, I of little think what who to low one interpret is bleeding, relatively And who, to in a in frequency is push way in physically to bit how think have kind what the bleeding extremely on themselves be is in may that a that target type.

to So are of pertain in bleeding. to good outcomes reduction sustained going evidence

I we as or eye far goes, great think patient expression. consider of in there's As Factor a inter- for Factor don't is mind's quite would specific number first, have and have to twofold: bit -- variability be good. activity I And about that a intra- reason we what the don't in our

time that at any subsequent could look such different, point, or a up down. point time So given

makes clinical is exist and relevance Factor the to data. the level. VIII you it reliable, the challenge difficult transgene as control bleeding VIII Factor established. hasn't relationship second what of assigning know, variability And about trajectory a the might And of a a in be reasonably certain the to been accurate, number So

period of we're database. going time, to company building long be first for a follow to the that patients As

So for specific number. give X reasons, you I a can't

of thing. zoom in in earlier, with are levels, kind Factor to So important have back amalgam an most them prognosticating. at look interested out because Ying's bleeding, people question and

it and off, course, time did so. was go if data what happened, to look, that's I how bleeding Going to why you the difficult to back think have very prognosticate be to its

J.J. Bienaimé

we questions way, VIII the And enter all. said which will So approval, payers, by because answer into to forgot FDA's approval, second, need previous get regarding full consideration full need, approval, we not or at full accelerated for for approval, does person. the from the to Factor what Hank the earlier, reiterate not we whether we

our usage, payers, to want going And rates, Factor how are is much On levels to linked is this impact save the pretty main money payers reimbursement thing primary with the [indiscernible]. bleeding product. directly Factor in zero to know Factor VIII, of that impacts And generally annualized the What especially are VIII they the the recombinant full VIII which usage Factor recombinant the VIII usage. endpoint is on study.

Operator

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

Unidentified Analyst

This was significant [ph] eight for on data is make valrox. any is Marianna some compared we and or seen differences Nathwani I out be hemophilia comparison Salveen. it I actually allow had have from made the not? a A, And study that would years, given follow-up wondering, to to to reasonable

Hank Fuchs

think Factor IX levels. XXXX, think the of durability his years field more fact, through was of and the 'XX spite presented according dose in X% I of by a And and on high me it's were all Nathwani's study was importantly, in you, started X X the great sustained Factor X-year update just mid-dose way, reduced dose after about X-year in that low and and by the the flat and X to he original his got let study. gene substantially in at knowledge in to cohort therapy expression think is levels X a remind it in only bleeding high doses. question, highly an on X-year I informed NASH an published update Factor he data. hemophilia I And update and December, cohort

X community VIII substantial the Factor expectation. program programs. that now, for their and encouraged of humans in couple good think given X evidence is of two There's essential the transgene correction passed inform in expectation. life. with out looks scientific between expression in gene preclinical about is Uniqure and durable And evidence the of and message a although differences durability years. establishing with years his is a So in numbers passing hemophilia canine other Factor of I of by durability of years, those experience that Spark IX two I are really there Factor an given which is there's for data now our therapy think IX program, or are precedent canine They hemophilia, pieces entire canines

So high-quality the gene that in gene therapy will established be product, after expectation your vector expression is is the a field that provided durable transfer.

Operator

is from the question Chris Raymond Jaffray. next of line Piper Our from

Chris Raymond

here and Palynziq, break XXX ask paying -- a guess Hank think give XXX question. now a In Jeff, patients, non-valrox I on maybe you you on so I'll said. and Maybe have I naive, therapy

think the to -- us quarter, get trying Just guys has X-or-so naive XXX rate the can where see patients, Jeff, of a of you if I give you the sense experience, gone? a can And discontinuation had sense last was if XX%. think, I I in had you clinical it around mentioned discontinued.

appreciated. is So rate? Any stable now? can you color it -- give be relatively would that number And of is sort a it

Jeff Ajer

Yes, course we're could to on by saying to that for of patients And watching caveat most Chris, Palynziq their possible commercial context, it's that that started in I'll my early treatment, milligrams XX therapy still getting would fastest relatively things the naïve closely. great patients the and titration be question, answer weeks have so in reaching change. example, being to just point now for per mid-QX, they with XX put where once of schedule, day. are that in

induction of bit the just kind schedule. So of a path of about long context titration that little

I later no are last with far, So, quarter that quarter one Consistent number and the the so clinical quoted they materially the those in experience surprises we discontinuations, that what with discontinuation than inside has of experienced real trial. was less been figures.

Palynziq about of Palynziq. I really other will to program, the patients lot reasons. -- point And out Some patients the discontinuing learned we've learnings other trial a we've and due discontinuing those our shaped that clinical use thinking the about how tolerability through to for issues, commercial use got support of

way with of adopt their number best try to up working they're the clinical as following the clinics getting support to their a in and possible patients that U.S. their guidance clinics field and providing experience to got of So out maintenance dose. they the coordinators titrate a to to ensure

Chris Raymond

So to XX% it you number, in just so say so single is the than clear, lower when to the materially you're digits? safe still see be low

Jeff Ajer

numbers Yes. the Consistent quoted with last I quarter.

Operator

question Our line Phil from of Company. Nadeau the and from Cowen is next

Phil Nadeau

levels Unfortunately, I'm said going to the that the data bleeds line trends to to clinical top the susceptible important, fall rates released, -- data to everything certain become of that at people currently. go when if Hank, about when determine Factor X-year on they the XX%, bleeding back going even you to aren't going I'm to going in Factor particular, longevity Hank look effect. people are patients' appreciating assume to and the that And most below being they're are valrox.

what analysis? should it? we Would try from of So effect? doing to the to longevity interpret dissuade us you mind of we out extrapolate data keep figure do and point think as the in you this the What

Hank Fuchs

Oh, be boy, reaction. don't, would first instance

Just so simply it's you could -- you Factor multiplicity. you almost could construct give yourself transgene Meaning you many to anything subject a bleeding. to about tell to because there's and and just want to answer curve regression you too any wanted

measure let at interpret And something surrogate patient experience we're never significance these durability relationships it. the I to think about X long-term our of of its industrial year a in the gene that's And that in we that world. speculate inform than so remember, been of I'm segment. keen transfer data of done and rather to people And in that's the have X a understanding

on and into relevance the factors. VIII to perspective clinical give advice levels that's my prognosticating about future of those the best So Factor

J.J. Bienaimé

being contradicts And data also Factor control XXX% with [indiscernible] add and X% actually assay. comment IX I below Hank, bleeding this again. mean I for X% be the levels years not IX years December, there Dr. I'm threshold this at X around making ASH But know no idea, it's patients where agree Factor because years that chromogenic magic with with X the and to would the have at that going we Hank from have control, is X the Factor are data I that Nathwani presentation per X just to still the as IX the threshold. XX%, or again, actually I XX% bleeding

Hank Fuchs

that Factor know they're a patients XX.X%, if bleeding. back to felt because don't $XXX,XXX I And therapy replacement not year Factor be their going to all going payers to excited that I are that might level at about add letting especially

suspend they the a So that picture the being, to interest I context to role but developed, try for that's overly time keen Factor we going now. to in in have are think be understand the play our right overall levels, where we of understand

J.J. Bienaimé

are want out around be, I [indiscernible] point X believe heard X only we is but at this data pointing available. where we going to [indiscernible] the like X% this to we and that are is years, also because this not to comment, because this X% because I've is And

Operator

Our next Joseph question is from the line from [Operator Instructions] Leerink. Schwartz of SVB

Traci McCarty

Okay.

go the operator. Let's to next,

Joseph Schwartz

you hear Hello, me? can guys

J.J. Bienaimé

Yes, Joseph.

Joseph Schwartz

happened there. what sure Not

attribute predictability less are gene considerable any that patients doing variability, an determine valrox administration? you've that with their important correspond you So, is more recognizing had work or to such and for to who one-time therapy factors respond the

Hank Fuchs

patients. Joe, We've done a that the bit little in of work X on area,

in expression of variability who got into variability of patient levels it there's But X the only XX to -- a non-hemophilic make deciliter group also the there's range. first the above IU little bit that transgene got know, in who to year. you didn't per As the

identify So the we've looked predictors initial a the attributes level all, responsiveness. to if of X -- can us other identify take And predictor, to response. patient we better versus been at at the determine patients, different that also X that X factors for there's the time patient of a I patient and can we see or product if understand, lot understand patients, at X anything it's if between and to can't we other manufacturing significantly of all to magnitude patients. think We've that that's chemistry going X looked control haven't the X product-related X able more

Now awesome spite which to I target of the gene sustained end in response had that transfer what benefit. be the we've XXX a of in this study, already what joints, an talking majority consisting about of turning elimination hope vast by is repeat bleeding, seen the of of year of variability, of off the patients treatment we'll is

out robust all keep is, will got to on really eye response have in forward. of a our we we learn this we to try at an more go But variability go So predictors from and the patients. about point,

J.J. Bienaimé

Factor have lower Right? at -- was year X Factor bleeding variability recombinant has in in and get-go, and been And under bleeding been our VIII I has patients although had VIII to control, the who Factor would -- there does control the even from there found it levels had say in his was variability Yes. not response X levels, injections. -- in no patients

Hank Fuchs

Yes.

J.J. Bienaimé

variable. not the outcome clinical is So,

Operator

next the from is Josh Our of from line Schimmer Evercore. question

Josh Schimmer

all consider your the out the questions, all going to are despite ask. still If turn it prior to that durability years, levels X continue and decline potentially about commentary does a interpreting despite to of why yourself to either information? challenges previous questions And your quickly VIII the at Factor Hank, to answers vectors? importance or to strategy there about I'm information, then then some the the or alternative strategic redosing that considering to consider solutions, move have alternatives blind you that, that do need

Hank Fuchs

gene fairly area, focused obviously. limited said the the persist. also expectation, that before Josh, our of What part Nobody the because for redosing And is that what said, ourselves potential in activities the that is our as that the reason to to program. benefit clinical for But in is we're doing in Street consideration going thanks in largely competitive, we're blinding of for blinded is part it's transfer will is, I done and of the the reason of research about for redosing Wall question. we've anything redosing.

imagine talking a but suppose we're the But it X doing be about proprietary. but expression. relevant we're it stuff redosing, It and to investment it. ask point, haven't in of And doing question. we materials it's thinking mean that that gene update, could to about and only one will it trial. lose at a I year actually a forward did a clinical somebody Now that I can then, the result look subsequent about nothing means doesn't redosing it, major triggered not we're that about we're it and mean doesn't

Josh Schimmer

yourself I informative strategies of may guess the to strategies. urgency data those for Because the blind potential why be data? and

Hank Fuchs

Josh, to you're question two. on

I a So very strategy into data trial, at all arm's but discussion undertook pointing and interpretation of friends that at a here we was would of baked to between the decision it Factor conduct is the clinical manage is me, of my length to trial big to dispositive that all we say about activity the just understand what to unbiased of good authorities product. think out know of to from the very health of really of be the all availability data made terms decision about that decision And our the the could relationship we with trying of want keep integrity therapy most things in came earlier. of bleeding that the And we why for -- going a and availability and element data the accelerated gene us. might

that. activity I redosing, put committee's said into decided the -- concerns move we'd to into if before, to have the data monitoring around our that we or heard once So as they phase, the hands. reactivate a needed pivotal we we suggested all And increase of significant registration data had

are. manage And with the where trial made about decision. the so we how sticking the we We're here's decision to and

J.J. Bienaimé

much, as is integrity want own critical will X product, working Hank be patients be data the pivotal much meant for the fail are, and believe but approval, clean, there the who redosing shoot too is And of the opportunities just to the for trial to that's of potential don't as we Phase we what trying accelerated said, a to integrity to but actually not trial lot important. on we therapies. squeaky our Actually are strategy, we are we that has that's especially about the Hank here, of clarify that to advertise competitors' our redose what

Operator

Karnauskas of Citi. line the from is question Robyn from next Our

Robyn Karnauskas

Palynziq. to back going Just

do almost forward? are Palynziq changes patients mentioned mean market also So that about you the that European in new you Europe? for you What drugs, any you for And as opportunities on you then the have given label. see in broad a trajectory does that go XXX for

Hank Fuchs

first. I trajectory I we which The think I but Great questions, can question both. can think heard cover we them -- trajectory as cover. X, The

the at treatment we Palynziq provided to you not QX, at pretty naïve you a what on just numbers QX line -- you data, reported previously were that If pointed therapy ago, of the see end mid-quarter you commercial take basically we end is look in the that straight a update kind the that end imply for we that patient while add the referrals And that points of at in reported a earlyish the patients. the stability direction, same lot which is is we of in QX of would And that pretty the were largely trajectory up QX, the in patients in staying total. those uptake of stable. to got that because that

on can the patients that watch number other hey, you is looks that therapy. that pretty The not are stable say, number enrolled, of and but yet

we and of in that's continuing that our treatment. patients getting queue, to So should that's trajectory number, a It our we're about of enroll good that getting as if stays kind therapy good-looking a The same patients expectation. indication if too. steady, right, doing of that's number job the the indicates them our that on continue pipeline, of patients kind remain steadily onto

good. that's So

European in which will give draft that And won't deal language us positive. large is patients EC seen a looks we've label, holds an until that very potential entirely give got pool that terms know think label the to a Europe, the approval. of up, market nice we that that'll In of we've what is very if I with. really us, opportunity But

excited about So really that prospect.

Operator

from is Matteis question next Our Paul of from line the Stifel.

Unidentified Analyst

Nate anyway? for protocol has titration it first been How patients to [ph] on enrollment is And Palynziq by gated the quickly their This Paul. progressing for rapid are from treatment?

Hank Fuchs

sure correctly. to make just Sorry, heard I your want question

from of REMS patients enrollment a -- -- barrier our Your through to Perfect. REMS question was quickly is therapies, titration enrollment barrier initiation the titration. progressing induction production and is to how to to a

clinics some an step been to pretty average time variability Vimizim. with Kuvan, couple about, months not added we so has inconsistent enrollment it's the well on of that enrollment is steps running of basically barrier which currently But schedule I according than REMS and clinics know new that know by trial. X has the not in experience schedule patients, clinical organized reports being all on defines other we consistent, or the Palynziq that really And way. I an United surprises quarters between there. patients. from reinforce, their patient of onerous anecdotally, or -- therapy as were about and And some the pretty is not the through to The fastest the we've to patient. our than far, Palynziq been and other the drugs no titration then want step. and with Naglazyme expectations, we expected, problems more I've that a using last just anything path. been slowdown heard far, X It's have additional to starting States induction along what with what steady and than And so is and It's with including label conservative So, anecdotal starts. that to require the to lot what are a are around the don't experienced

Operator

the of Merle Fitzgerald. Ellie line from from Our next is question Cantor

Ellie Merle

the say gene into, And you clear are, your just the like and what having, might your this prioritize like it I gating clinical sort from is to more I gene I into mean PKU? of disease of of preclinical outside just optimization hemophilia taking think initially new strategic guess, therapy versus that's and programs? valrox, do unleash X you factors of So manufacturing in that say, you or a choice it the time? capability given therapy expanding vector terms strategy XXXX currently that guide mean in programs X Or work expertise, when And for? is rare

J.J. Bienaimé

of candidates physical every that's an the of it. pipeline. have So of the therapy we more clinical business -- other product to compared in gene We assessment it's possible that opportunity

component said therapy time, of on very we obviously this although turning into therapy the planning are gene not gene a at as because future, we before, pure BioMarin is So company. have a in

therapy therapy next next factor here and -- what's we candidates. more a to financial likely are anything gene there gene therapy a but therapy, [indiscernible] year -- the after going So so candidates gene there bit but after -- non-gene next there -- therapy, like than And have are IND to hopefully gene components sometime decide are be year. candidates it's our so other that is actually PKU else. And relativity are

those need just all we to probably depending competitive success the of and levels, a what we of them the considerations. technical believe on market So is the prioritize size

into kind few enters generation about the a of what that's Do you the want So, words say of manufacturing this, component here. to Robert?

Robert Baffi

to therapy with capacity. Yes, put candidates, And evaluate certainly, can additional produce and programs, therapy those the valrox we programs to we certainly as team our capacity and propose both XXX. gene current accommodate substantial clinic, for gene have Hank additional as into we his the

Operator

Our the William is Blair. next question of from line Timothy Lugo from

Myles Minter

is on Tim. for This Myles

therapy. the have would PKU to with would at if therapy predisposed one the for Palynziq a the decision to public gene you'd endpoint strength there? see Considering be to program gene from And be on trials cognitive relates Just [indiscernible]. quest that looking the happen Palynziq, potential neurocognitive benchmark that in I'm the that actually positive whether started benefit. wondering

Hank Fuchs

of there like think don't control either to authorities clinical uncontrolled expect been Palynziq therapy and think ever I is trials I setting, therapy have that a be observed to to compare otherwise trials. larger than will to requirement controlled a is room. in But or better might than could that, clinical or health the gene gene untreated what natural you an if effect Clearly, large Kuvan

said program in clinical year. file our yet, my IND comments, to this launched later we haven't going we're Now I as

Phe have believe get from I their Europe, a own remind let's with so United in looking the you United would believe So I compare say, far, health of in But controlled in for know could Palynziq on the study patients supported in that do authority just Palynziq effect specific things, States, large clinical And blood let's while certainly landscape will I'll Europe. registration. reason more support to do Phe what support Kuvan control States, and to that descriptions. Kuvan a the very blood registration stay trial, that to of explicit at you regulatory tuned there's ample least

anything Phe So real for endpoint evidence beyond approval. an regulatory that for there's no would be required

Operator

Capital RBC from question line Kennen is Markets. from next Our the MacKay of

Kennen MacKay

and just and ask competition Congrats the achondroplasia, break both from you're a on a thinking the maybe quarter. from valrox of team how standpoint commercial theoretically sort about trials a I mechanistic to well physicians about the clinical couple will in how mean commercial as we as potential competitor versus choosing on patients a on And put agent? years the in I valrox side, standpoint? commercial registrational thinking be should of give potential

Hank Fuchs

answer. of middle of the in valrox end the said You --

this think is all vosoritide. I about

Kennen MacKay

achondroplasia, Sorry, vosoritide. yes,

Hank Fuchs

the of of restoring number single so target competitors first getting competitive and a we into the of thinking point to and no view, the without growth of point approaches approach a of we're who's of Well, achondroplasia. are of competitor they product, data and very all, have one, similar There of a but preparation the view us, biomarker a mechanism to taking. And different a that taking approaching long-acting single data, like Yes. healthy is specificity there every details the this from dose from approach volunteers.

are they So behind us. years

manufacturers. wishes. of And according or into or go interests vosoritide so their question, they with on other not other from how -- may will and And on amenable the part now less experience go vosoritide, depending your more be alternative to own individual to options payments will their

time that we'll products investigating in interval, next-generation Now be ourselves.

is high and thing off vosoritide will and a instance, the much moving product, of working that. hopefully be so only the And the of that is that in would our presentation relatively -- vosoritide expectation satisfaction people be on first better we're with

J.J. Bienaimé

the any I closed, if clinical is treatment as is the the also, base window opportunity because this no is -- may, here. work you limited, know, there -- for And for benefit unlikely product patient’s once window there's

would So miracle waiting X to growth. XX centimeters XX they of or next X years product, the for about lose

Operator

from Our is Nash of next Edward question Humphrey. SunTrust the line Robinson from

Fang-Ke Huang

for Nash. Fang-Ke on is This Edward

hemophilia. back topic to variability the going on Just

So is to we A, your more you the based hemophilia in and B hemophilia compared based for think that on hemophilia there think all that kind A observation hemophilia therapy variable on is that Do trial, gene observation comparing always of experience correct B. field...

Hank Fuchs

compare to you did have -- difficult sorry, more so It's questions?

Fang-Ke Huang

follow to want just Yes, up.

reason is behind it? correct, secondly, any Just that potential is correction if there then

Hank Fuchs

Yes.

to it's across difficult I across interventions. diseases, clinical compare across very trials, think

VIII mean products. ready know of still gene I a So categorical therapy, and Factor very early B, I'm comparison to Factor hemophilia A our I and make and IX it's products don't days. that hemophilia other

Operator

now I back call the call. and This the Q&A J.J. end Go over CEO, will be Chairman ahead. And Bienaimé. of hand to of the portion the the will

J.J. Bienaimé

Thank you, operator.

and So, the X the Phase by accelerated the future, vosoritide the therapy energized continued with year the global end prospect year with a as PKU potential commercial of the growth our our of end and to some brands. XXX data look current -- valrox, of of gene of we we of approval our Phase forward the global trial are at the readouts at products BMN also,

the So look a manufacturing quarters. and in in position continued and organizations, Goodbye. BioMarin great never ahead for has to across the things thank stronger been we your commercial, forward you R&D coming And support.

Operator

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

now may disconnect. You

BioMarin Pharmaceutical (BMRN) 27 Apr 192019 Q1 Earnings call transcript