BMRN - Earnings call transcript Q3 2018 | BioMarin Pharmaceutical Inc.

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Operator

Welcome to the BioMarin Third Quarter 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. To remind you, today's call is non-confidential and contains forward-looking statements about the business prospects of BioMarin Pharmaceutical, including expectations regarding BioMarin's financial performance, commercial products and potential future products in different areas of therapeutic research and development. developments depending regulatory progress future filings authorities, the market, materially programs, BioMarin's actions with differ those reports. pharmaceutical of and as actions availability and and the may SEC on in XX-Q, of the product in factors such Results detailed competitors, XX-K BioMarin's X-K by of capital,

Spiegelman, Officer; President, J.J. Chief Executive and Ajer, the today Worldwide and Commercial Robert On Research Chairman Officer; call Financial from Technical Baffi, Bienaimé, BioMarin's EVP, Development; Chief EVP, EVP Dan Hank Jeff Operations. management Fuchs, Chief are and Officer; of

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

Jean-Jacques Bienaimé

Good on and you, you for afternoon today's Traci. call. joining us Thank thank

us for for data top for three DQ regulatory XXXX. IND results, potential increased first of been quarters extremely positioned The line results, and have in XXXX a filings new productive have and

has So the of Palynziq. with our revenue commercial quarter team in this addition delivered record

to and continue trend. expect record in in billion remain are million results. quarters the top revenues $XXX three on billion line both approximately year end And first U.S. this year the third well in in billion and $X.XX We Starting launch of had in XXX during XXXX. we $X.X for of we approval third patients quarter this track this and year full July the in quarter. quarter Palynziq after May, with at launch $X initial extremely the the Palynziq the went commercial reimbursed

patients reimbursed, by XXX and referrals We we between case. starts adult risk expect end. as PKU at And and beyond PKU clinics to new and year to drive commercial patient QX add patients on in expect Palynziq be XXX a

approval and level pleased moment. Palynziq in will us launch a details in very with walk of interest are additional through We May in Jeff the since

in EC second of action is U.S., if the the followed we CHMP expect potential launch of first Application Marketing our XXXX. Authorization in of opinion Commission the in positive, outside And half that the a expect opinion in on of the Palynziq we Europe half Palynziq For XXXX. by EU

in next As studies. vosoritide both approval vosoritide XX to for XXXX half with the follow. XXXX potential second half forward, are growth from in and of potentially XeXX our by results valrox of X week Phase of in the of for the excited the are in we the of data tracking look full we're is for targeted valrox filings important late-stage second for to their hemophilia dose next Results valrox products, plan and achondroplasia prospects drivers accelerated approval of Moreover, filing mid regulatory with year.

develop we the products gene and clinical beyond you our at data platform Day We plan our we how excellent our this gene X, and look with capabilities, gene Looking seeing next the November valrox are to in therapy leverage deep products, projects, PKU our there. experience R&D York on on thinking landscape New to about our forward we development dive our upcoming late-stage one being to other therapy's to a extensive clinical into phenylketonuria. plan provide therapy

our U.S. excited So Jeff to pleased it prospects will conclusion, products. XXXX. in very on XXXX details in who for update our commercial are share beyond. other turn over will We're well about we of to-date now accomplishments launch I with and the as as Jeff? our Palynziq

Jeffrey Robert Ajer

inquiries morning, on I'd of you today received to some a plans. drug and to the proposed acknowledge this Due J.J. President pricing from like you, Thank speech few Medicare

of is less we of than a business, Part and scope of global Given maintain commercial Medicare revenue pricing book from across innovative generally corridor our tight To business. remind the our X% products. our global you, B

more until closely impact but early available. BioMarin, might changes it to that details monitor is to too potential continue policy say will are We in additional

PKU launch of Palynziq over U.S. are in quarter XX,XXX the U.S., a of PKU adult actively results one-third business. base then patients to With the of them and a to for all the products. approved clinic. of that now PKU walk-through U.S. being largest XX% our adult opportunity a third patients only Moving in tremendous treated in patient treated Kuvan, Palynziq ahead. our we all U.S. Recall population are represents with of the being have so

the are period, of indicators following measured of a were key focused end all third The launch on this of During are revenue the lead growth. we number future metrics of as quarter. that

to XXX XX from were there with treatment studies receiving were reimbursed the commercial were Palynziq naïve patients these, Palynziq. First, Of treatment. XX formerly clinical and

were there be system quarter enrolled waiting on our prescribed expect end, patients and commercial patients now end, enroll are Palynziq, for and reimbursed shipped. their then adult forms, in PKU naïve to signed injection In REMS all addition year an we By to authorization additional RareConnections be first XX XXX XXX between at have who Palynziq. then

active Palynziq patients. at third one had of Second, clinics the end more quarter, the or XX

phase to Kuvan unachievable maintenance had were constituted important induction/titration has education enthusiasm a greater patient our meaningful still be of will outcomes Long-term the it's is weeks, XX least To one when revenue. to consideration daily achieved, An for simplify, with contribution Palynziq. encouraging naïve of patient level. and PKU certified. some of have time two in-service dose, alone. active of Palynziq. meaningful that milligrams on on for that dosing seek in that meaningful dynamic. the the the recognized. driving understanding intent patients. revenue. A use this expected An context XX% of half the of takes is are the expected This in of indicator in on in for In revenue treatment, a approximately XX the adult efforts XX% a to early past working very $XXX,XXX per project when half still Palynziq the is shown Once of at patients daily, injection and treatment years, first XX% we control the patient market loss milligrams cases enrollments positive is Palynziq, have more the patients significant and XX% from to revenue their maintenance of Kuvan and of approximately year with Phe revenue our for REMS complementary contributions optimize patient of in it the was adult been date, patients Kuvan Kuvan To patient are fractional, at exclusivity per through their almost total year assuming number is been with $XXX,XXX on net provider addition, clinics to a are clinics prescribe XX Kuvan franchise their commercial is level observation of Once there the and key in quarter healthcare per per per months, to provide

we trial of summary, story. patients for between naïve the successful and pleased clinical patients are patients, long-term and is we patients received we progress conversion contributor our Palynziq Palynziq very launch reimbursed contribution to anticipated growth community million. to the well months modest beginning the $XX XXXX and revenue the transition to and year will revenue U.S. reimbursed full the commercial predicted during be the believe trial million Given made Palynziq the treatment clinical in by of PKU this of with In Palynziq another product. initial only believe has $XX to commercial been

a year last products, which last of the year-to-date year-to-date net to compared quarter of periods Health Brazilian from the drove same growth in year. Starting quarter, strong and revenue growth other almost resulted brand. our commercial Now which product in compared revenue Vimizim, the for with benefited turning to record Ministry year-to-date large order to XX% the XX% in XX%

uneven experience has patterns. from ordering to patterns of uneven markets, which quarterly the impact continue We certain revenue

As Vimizim dynamics Naglazyme, respectively, same Naglazyme we were to increasing annual and in in place Similar third and revenue market year. with the reaffirm. always, guidance net we refer for periods to last compared XX% the to encourage both XX%, the quarter which you product year-to-date, and revenue for

Moving Brineura the to since quarter first being the of in add $X.X CLNX, for revenue of treatment full net of quarter EU million. mid-XXXX. new commercial XXXX, fourth in resulting approved and now third patients, is U.S. continued sales we which of its commercial to just the in In

disease twin and from programs goals We progress of our screening diagnostics identifying approvals. and against are reimbursement our awareness making national new patients

of strong we net delivered of optimistic laying expect growth are material the that our and confident steady will Brineura significant in The of And are seventh more XX% and of revenue commercial Palynziq, forward revenues into expectations the but and terms develop going third in are commercial in patient our team we a for year-to-date. closing, product it quarter quarters. slow revenue interest. contributor clinician coming a exceeding groundwork the XX% We launch the contributor. In is of product,

details more quarter. to on the financial turn Dan? call the I'd Dan like to results over provide to in third the Now,

Daniel K. Spiegelman

pleased report strong increase our by $X.XX XXXX of record year-over-year, contributions billion, refer we release an today, for we Jeff. our as XX% the to for a third summarizing mentioned, financial and all with press were to you, three results Thank I Starting Jeff that the quarters total revenues J.J. full that you results, details. are for top line first marketed driven Earlier of of and release a from issued quarter products.

until be we billion be goal revenues expect contribution for of Looking XXXX beyond couple have XXXX that we over around vosoritide next what and expect the that paces from that decade. the and our with line XX%, to top should current the said above from previously, at to And level of keeping we $X percentage growth products. valrox revenue the years growth start next total approximately hopefully track

R&D million Moving referred expenses. head enrollment October, year-to-date expenses $XXX.X $XXX.X as to periods XXX, programs. I valrox which to and to ongoing what trial of in Phase back, quarter full production is was $XXX.X primarily in The expenses, the X of XXXX increase Phase it trial XXXX. compared is expenses R&D is due in third X production Vosoritide BMN and towards and million $XXX.X those the clinical as support still of respective year-to-date operating increased be hoping to formerly in the called the clinical million expenses, increased to XXXX million in

XXXX. of XXXX. to QX, quarter in clinical expenses marketing continue our $XXX.X year. $XXX.X $XXX.X to XXXX while the the Palynziq compared primarily commercial associated to expand expenses commercialization slightly these programs, credential balance EU prior of The million remain and primarily increased relative expenses for next and campaigns guidance valrox expected increase a respective preliminary U.S. due launch, for full Palynziq year third progress quarters, full of period and to Brineura million early and the into the year consistent efforts. to SG&A QX, with guidance sales though prior launch increased $XXX.X year was for over to increase year-to-date forward and remaining Kuvan, launch, and year-to-date for increases to major million Palynziq are the and of will Looking were the million by SG&A Brineura driven expenses efforts. expenses R&D the should within with The by within product driven

Turning in at $XX.X net to loss to flat year to the in $XX.X third GAAP the quarter XXXX in GAAP XXXX. million bottom results, loss net $XX.X in compared third same compared quarter XXXX to marginally the Year-to-date, $XX.X was XXXX. last million in essentially period compared million line million XXXX to of increased

excludes basis, XXXX increased know, and the contingent other to on quarter, you and certain which XXXX quarter performance QX XXXX. our and to consideration measure for taxes, consecutive also amortization, $X.X compared also fourth specified compensation, the million EBITDA in depreciation on interest and and in which As of excludes is third income, items. non-GAAP recorded a based we $XX.X we million In non-GAAP stock

Brazil Naglazyme due than both to large though still order revenues, XXXX, guidance. Looking of expect ahead receipt a revenues quarter and for to within MOH that be lower QX QX fourth full-year in Vimizim, the will we QX of

QX, will in In noted total, also guidance. within full-year but In still SG&A R&D guidance unchanged. our GAAP addition, remains expenses above, as and non-GAAP also and increase

and expenses stock from and contributions results strong utilized cash net delivered notes approximately In continued cash from at revenues, counterparties. In full billion also compared the robust shares received with non-GAAP quarter increasing Kuvan this profitability BioMarin our of XXXX the convertible have equivalents repay XX, of billion as investments, also XXXX. that XX,XXX XXXX. retired. as conversion, Naglazyme, shares of had steady we been for we matured. year progress net $XXX common and closing, terms end XXX,XXX principal on year, issued million call of and These this the $X.X to the XXXX back XXXX, operating In year-to-date October $X.X to our controlled product compared Vimizim cash, a to shares With we of in towards subsequently and September

like over Now, call the to I Hank. would to turn

Henry J. Fuchs, M.D.

regulatory year because to just provided new so we provide two J.J. Dan. we'll or of Day an have this updates, X, overview in has many far few a already accomplishments a where R&D Since development November Thanks, touch and items. on I'll minute take and

review For decision CHMP Palynziq is application Europe, of XXXX. and first in half expected the tracking the the under with market a authorization unexpected

that we're for in raises valrox clinical design Given translate of the demand the enthusiasm are draft Palynziq demonstrated products recent accelerated that our increases for activity in believe and the and into in range valrox, we're in therapy optimistic lowering recommendation factor Phe our discussions of approval gene to confidence to studies bar competitive Briefly potentially plans on regions. activity the development advantage. for – we guidance results below an approval pathway. an FDA file normal the our health ex-U.S. seeing The gives Recent study well for the in our the us U.S., dramatic with will registration. the authorities supportive range read important expedite with for the FDA factor

In very exciting from by in New are to and we November encouraged Day to you to aligning finally And European upcoming to research health our York, R&D updates development what health authorities. we group. remind with conversation with ongoing planning forward of of at the look in X authorities, expect a number addition U.S. well our

achondroplasia updates, as all In pre-clinical with extensive including addition provide PKU gene of with for for data review products, vosoritide our as to children development to we well therapy product. months XX the key plan

growth Chief about of leverages rare into BioMarin's And should and the the he in relevance and attend It interesting regulatory insights stage get treat of and share of our Policy recent an be approved. approaches will one so how his future the we're perspective tune to and products scientific have how or achieve Cardon, on please diseases Lon next key pipeline to webcast. policy Given talented via in of our person to the innovation thinks Officer, leveraging in from colleagues Engagement advances, genomics excited event, and Regulatory to finally, our share BioMarin informative Scientific beyond. plan Patient

continued your now the for you your thank always, support to I'll and As call questions open

Operator

our And question Joseph with from Schwartz is first Leerink Partners.

Joseph P. Schwartz

Thanks very Great. Hi. much.

your with pricing But could think that this you new hemophilia, valrox could current for you pricing given in potential much impact your lower Medicare don't factor wondering proposal offset to be have replacement reference system know strategy exposure by there So associated I with extension I therapy. health save gene on factor was replacement therapy therapy cost your how impact HHS' if products. the could international since and spending

Jean-Jacques Bienaimé

mean, yes, I'll comments. start and give I I just think this –

haven't haven't priced because not approved As valrox, U.S., you the VIII, Europe. have obviously, in not know, we we so it yet, we Factor is priced it priced in

because we on is but indeed come regulation to So of with if pricing details and best to time here this obviously, strategies becomes a of consideration in happen, here, both have indeed important this a sides think up discounts I this will an lot if are also does there be flexibility Atlantic getting need U.S. of Medicaid the lot covered be that already a and

European average So you and huge know is not for us our give going details, is to products. the more to but price can different compare difference price if for this government Jeff our current U.S. I a make that don't that

Jeffrey Robert Ajer

much Yeah. Thanks, J.J. to add Not to that.

be relates models CMS And reimbursement the refer other facilitate you got and and the developments recent there we've remarks advanced may novel that gene time for products, Verma's details. Seema would be within to subject. pricing on of plenty reimbursement working to as it think to I out therapy

Joseph P. Schwartz

That's data it And great. your where patients but Brineura then, Okay, helpful. like all like recent looked think. at on did patients some in BMN update I SSIEM, stabilized, it wasn't XXX

existing market data? and So likely do think from there clarity that BMN on regulatory when most chance you you that the path the for think XXX can to BMN regulators? get expect you any to do is What is XXX do you

Henry J. Fuchs, M.D.

Jeff. Hi,

dramatic the at improvement Quotient, diseases, comes size hepatomegaly patients. think DQ I it's which think draft all seven in a documenting something stabilization to heparan partly of checking in who kinds on in the demonstrated presented progressive fluids, of recognition and of like of tralesinidase. close five out endpoint. cognition patients diseases, tralesinidase pretty of a slowly levels issued challenge therapies of in sulfate these in and for reductions development have guidance SSIEM boxes for document the we FDA brain Development the tough liver And the is data in normalization So was in

fair be So whether a guidance Suffice I Food which would our and the that Administration documents to conversation conversation. to to apply we a right us Drug next now, with amount particular about on you those to step opportunity regulatory we an around presents path. want don't to say, how have so that plate a to for on have expedite comment and report back timeframe

Joseph P. Schwartz

super my Okay, questions. great. That's for helpful. Thanks taking

Operator

next your from And question with Goldman Salveen Sachs. Richter is

Salveen Richter

Thanks for my question. taking

regard that with you're this pursuing there the approach. seem to like Phase other play it approval valrox, data be do from accelerated will to decision a from pathway. the And X your on So in role the looks factors apart study,

between are DNA? is for based Just you circulating help with remaining a intact us just profile And in then wondering, work non-circulating a be there to wondering continuous the to or appears still that can do there if secondly, around that and X order that thesis still was when mind. comes the so episomal and to Phase from why assay, discrepancy I the your discrepancy expecting have set the versus regard the you one-stage intact, pursue ongoing to the understand on it And and there assay in and filing, chromogenic the the stabilization data filing. being to that study you

Henry J. Fuchs, M.D.

Yeah. Hi, Salveen.

discrepancy replacement the was other assay no factory of observed in These kinds more that. of in And products. guidance document a is is overdosing that the wants to for of products general basis been incorrectly a what or make that extent FDA of a issue for on to So underdosing interpreted there recombinant have bit the in potential raised matter phenomenon discrepancies for the the that assay some result. is of sure products

gene sponsors inform to has And so they're therapy familiarity. with that a And from of of into be they asked may that gene which FDA extent than the decisions. the is where their products coming that different that might look factor the therapy biologically products more products the recombinant to lot the in transgene context

the to Factor you they VIII accelerated context for kinds So establishing either represent pieces the you're would which the went approval the the consider of of bleed is reasonably levels this a be to or indicate two are under for data range. levels would VIII – likely which has And likely. believe Agency Factor of Factor value that present point to benefit level, reduction, further clinical why that Factor reasonably VIII asked FDA on of that of the these VIII in observing resolve only that say normal a information, that achieve

look after important received the next call you And animals look the and Proof let's who regulatory second in is obviously course, clinically outer BMN reproducible forms reliable, And real, XXX. observe for to have to at likely of it reasonably expression. things apply of persistent persistent operationalized will the transgene, providing we and think is phenomena gene more So, that transfer in the in, on out for we it the year. terms how the are relevant the is DNA of we that in years update we an vivo of – the forward they an part pudding more where convinced of of the stand sort in and on of to and in question, middle settling guidance question literature why documents. in their and I that's this context your

Jean-Jacques Bienaimé

conversion, our based think on And are significant discrepancy R&D so to believe be cover going Day. to had to in also, assay we authorities, we I don't with The we've cover circular is detail And some issue going this. at for to that a us. VDR regulatory we're going this interaction

Salveen Richter

Thank you.

Operator

next Piper your And you. Chris question with Jaffray. from Raymond comes Thank

Christopher J. Raymond

Hey, taking thanks for question. the

you gotten but to it's the I of So know Palynziq, maybe early still through, are months patients on days, folks kind launching. these just you've decent guys number XX sort five so of gotten X-week treatment-naïve now timeframe. of with arguably critical a have or that into

period those like? that along? this during some describe who that maybe describe chance for think, you've run you folks And might are are discontinuations biggest part Thank running anything look Kuvan I of the to into described anaphylaxis? any then for you. can those sort that Hank, any part a when two into maybe enough Can seen timeframe some of maybe question, unexpected as far have a patients you you of And and what of XX? you

Jeffrey Robert Ajer

So this is questions, me. Jeff. Great for Chris,

see if them. I me cover Let can

were numbers those events Palynziq. So end of got launch. note which that have reactions, completely the majority reported, is, on practical so to we the that were we've injection going and – another are with quoting for vast been expected of experience of site month The September were reactions as adverse first of thing

process, start patients seen other don't of the have site is dosing which expect to to the and at really is the serious adverse beyond We very time the on just that more get many The XX anaphylaxis point gotten we reactions. we seeing that has milligrams which or up with types have that case injection daily through events beyond, of to management, reported, reimbursement haven't taken date. caveat and that adverse it patients start new events to the because arthralgia would

might three I question now. good for a to be So ask from think months

of naïve been XX% Kuvan Kuvan have of patient patients. patients, terms enrollments active our In approaching

thing going loss for dosing ahead Palynziq maintenance patients of patients there active amount switching exclusivity. a of and the on, to fair onto – So onto PKU a those overall really is well of which we adult a positive get think franchise is

Jean-Jacques Bienaimé

was discontinuation. on patient a There question

Jeffrey Robert Ajer

Discontinuation.

Jean-Jacques Bienaimé

Yeah.

Jeffrey Robert Ajer

any getting patients and discontinuations induction naïve haven't far So with seen from that patients. But again, we're started in we just titration phase.

Christopher J. Raymond

very you much. Thank Okay.

Operator

Cory Kasimov JPMorgan. And next with questions comes your from

Carmen Augustine

This on taking Thanks for Hi. is Carmen question. Cory. our for

your for which patient for another correctly, XXXX, I implies, the on Palynziq you're mentioned guidance. commercial if XXX or to XX at guidance patients am new you the starts low So so this XXX end expecting year drug, full counting of

So that's you're And kind what of you an pace think uptick quarter the in saw of we with starts in XXXX you I'm on Thanks. about how line of as of patients get into launch. wondering new patient thinking first the kind this? catalyze what compared new starts? will to

Jeffrey Robert Ajer

Hi.

see. let's So

Let's those back that... to get reported numbers. We

Daniel K. Spiegelman

XXX.

Carmen Augustine

XXX.

Daniel K. Spiegelman

therefore, She XX our is and lowest of first the balance patients assuming you that end the of to ones the about And guidance. the clinical kind get in that's XX. XXX would of are another at

that's So she I what think said.

Traci McCarty

Carmen, your how what worked? is – math your that

Carmen Augustine

Yes, it. how calculated that's I

Daniel K. Spiegelman

year. question And of into looking rate so next is, new the is going what for patients we in are forward that uptick

Jeffrey Robert Ajer

that quarter we additional enrollment rate Recall are been clinics adult has been patient clinics XX expect that it – out our of continue coming of reported that active. more them we trial Recall XX for account Tier-X about patients Tier-X for are adult XX% that clinics, launch and and the patients. new active. clinical of that as that are we XX% that of our account said third the target clinics now at become of of the picking So would throughout has up XX clinics to

of Also, we've become active, trial of in experienced is efforts up. pick a not drug, clinics new the start should the as third clinical more transitioning now patients. those trial drug, enrolling lot what focused patients. enrollment rate patients commercially-labeled should sites should the have with So if their that facilitate initial on in they quarter clinical fully-reimbursed their of to as clinics those most And

Carmen Augustine

Okay.

Operator

from Karnauskas next with And question your Citi. comes Robyn

Srikripa Devarakonda

is Hey for Thank guys. taking question. Kripa Robyn. so you for my This on much

convert? Kuvan Palynziq Kuvan interest switching terms us? – the of Is that in provide you get of there can to expect patients Thank convert any you. a Given you long-term? or to them percentage in you think patients should how of of or the to of indicated Is you that on patients Palynziq, that for to color you think all will majority there do conversion in

Jeffrey Robert Ajer

that's Well, Kuvan objective to And to we've patients converted it's the we the time Kuvan in QX have of ours XXXX. at many explicit by end of expected as treatment the that to be stated adult United Palynziq for as lose in possible exclusivity States.

numbers the new referrals of patient are Now, far so small.

on may small that a are year positive are some possible. effect and small So XX% is Overall, late patients. from The next by next Kuvan project and experience active the to to year, patients. Kuvan percent Kuvan early expect too too have what material numbers on revenue dynamic,

Jean-Jacques Bienaimé

Kuvan get entirely not as need your a we'll even are we've modeling a lost which account generics lot market, are have chance you that I a But Palynziq is a something that actually and is sign if not switched forward, not generic. U.S. news we currently, Palynziq, get the of good could naturally, interested, take this means think in patients because that even are still promoting pretty if there Kuvan which the into they I got patient The is of hit patient really we're number good they Palynziq patients. the to encouraging a And go on good mean, to later into substantial that to is to generic, them on convert. Kuvan We're time by we switch. trying – a that to promote the

patients. longer, pretty to that need of we current Kuvan convert the don't do So, after a it's number little significant can actually We market. the actually the patients Palynziq the all to long, window a is hit generics these to capture

Srikripa Devarakonda

you gene R&D And you. Thank we Okay, on at great. of one detail quick PKU what can program more therapy expect under see provide to Can any question. sort Day? the

Jeffrey Robert Ajer

considerations, considerations, considerations, man considerations, manufacturing that durability efficacy there biological is don't I about considerations, design, in know, dosing considerations, Information else safety vector missed? marker anything I

Unknown Speaker

I to (XX:XX:XX) we're going think don't figure

Srikripa Devarakonda

Okay, you so Thank great. much.

Operator

taking top question enroll. kind patients - LLC Martin patients have that about six-month you you question. we data? the Thanks Suisse. maybe looking Securities Suisse in with on us update (USA) all for trial's phase? press one expect Phase release to if the is Martin for that Credit when of Auster with could into measured you're continuing might running before found Hey, to notice to of X. want still I the are next Thanks. looking the XXX dosing, you enter Credit see to clarify, vosoritide Auster had And the your And I guys. type or velocity for line I

Henry J. Fuchs, M.D.

end line the because the enrollment Yeah. data occur And for expected that's the in at We're still close track year XXXX. of imminently. to is top on of

we project fact, the And remaining said those are put making that. patients KOLs, slowest our – to activators able to we're have previous last is an all site that on calls, have sure we that really as opportunity were doing in. In we're their well

we the of fully So that And Suisse you'll then just we year QX that then, X, the expect - the have for Auster enrolled in of Martin Credit running this the by next enrollment And by close dosing valrox complete (USA) trial Okay. on Securities end to announcement expect the right year. can and to make is clarifying that. LLC Thanks imminently. for occur end continuing conclusion XXXX? of to Phase that patients you're

Henry J. Fuchs, M.D.

- Auster target. Yes, (USA) Thanks. LLC that's Martin the Securities Suisse Credit Excellent.

Operator

question is next Bank Huang of of America. your And with Ying

Aspen Mori

what give the Thanks. ones. ex-EU? us any question seeing – on couple beyond Aspen growth we're anything update a revenue It's currency A quick you velocity? can of data then, And XX-month on questions. And breakdown Can for Thanks we and from there? for expect of impact kind if kind the ex-U.S. of Ying. vosoritide, taking quick Hi.

Daniel K. Spiegelman

don't near a impact U.S.-ex have year XX-Q the give FX breakdown. be relative currency zero, that I'd will million I unchanged. that nearly numbers had expected ex-U.S. of into relative what And last bunch to the dollars. have a on it's And taking I front me. net of couple we rate similarly, right net can you, U.S., it's to tell for the tomorrow. out you of budgeted year's on changes, in and breakout to rates, account basis in is

Henry J. Fuchs, M.D.

XX bigger about And that durability. remind discussion of remind months the to which durability the may a the of is be biology a about you really at your suggests a had part FDA you, then evidence me question And just question committee context the problem. is question, about the in consideration Durability of let second advisory about issue in durability.

of the speaks to out proportionality couple preclinical a at patient biomarker the and things, for data market R&D us durability. safety. so causes be biology. We'll joints, present on, effect the plates product. velocity vosoritide believe that the growth present on for what's datas you achondroplasia you novel to XX preclinical potential present And the XD we'll So, treated does growth annualized it for Day, the the for some with some example, re-review of We'll months going that durable, of what's to is we'll ancillary that biology to

months been I the attention think the the patients main thing be velocity annualized will data therapy. to be treated XX pay that Day and who through at will biomarker have to new R&D in actual growth

Jeffrey Robert Ajer

dollars, been point And maybe other a have U.S. I'll is our In our euro more you about balance other represents within the in add obviously just of be about might the continue data third. sales U.S., in the one currencies, to XX% and asking. sort currencies,

Henry J. Fuchs, M.D.

appendix, the (XX:XX:XX) in not was That

Jeffrey Robert Ajer

of prior completion the was That questions. the

Aspen Mori

Thanks.

Operator

Fitzgerald. And Ellie Merle your from question next with is Cantor

Eliana Merle

Hi, question. guys. for so much taking Thanks the

of and Just back durability. to sort therapy gene

DNA of You've and talked durability. about about the circular thinking long-term the importance

you circular are of do think think you And to Thank a less there you. what make therapy likely the of like, DNA? circular are key that determinants them more the sort designed not? whether lead terms aspects are curious or products like can in how DNA or long-term, I vector turned gene of Just into guess,

Henry J. Fuchs, M.D.

concluded been it subject question XX years of a is intense has about but ago. a research, that That

you – affect vectors to truncations some you there how and remind can were with summarize aren't things at that we which of R&D for going we're So, like assure how the Day could we and the circularization. that ITRs genome, petrological to about packaged

So good I'd say, will come attention to those to. be R&D pay things Day and to

Eliana Merle

Thanks. it. Got

Operator

from Paul with Stifel. is question Matteis next your And

Paul Matteis

Thanks for much taking so Great. question. the

the physician, Just your wondering approval? do you've how over U.S., is suggest quarters. that at experienced bandwidth wondering the titration once and seeing terms patients a you in coming on if of I'm so less much. could that the the physician FDA's versus in doctor and individual get how period launch for an what Palynziq many European an the through you're experience the process, any so think out and would dis-coordinated risk far seen And there then Palynziq the interactions just plays result on review the of anything from through Thanks

Jeffrey Robert Ajer

question patient United Palynziq clinic States, the I'll first maybe capacity and experience level. take by in the the the bandwidth So about

launch peers. or – non-experienced handling that that and will call, trial past in that think initially to we the clinicians clinical than clinic thought more be phase on and in handle guided an severalish the clinics able we expectation probably the groups be speculated would patients in small their we the induction/titration to patients experienced So,

we've naïve empirically have number. a clinics have So of seen one is to to patients naïve number small forth pretty referrals, the have put small so aggressive few new Most far. more of patients, referred clinics what that referred than that treatment to-date patients patients patients, several

largely clinical a We may from X the separation of quarter the of impacting trial trial is that effort focus support patients in also experience dynamics than manage haven't the numbers that products X and will of But the the one clinics those commercial-labeled which to clinical be gaining to fourth seen on transition done third reimbursement, trial Tier the the clinics. to of likely in Tier clinical quarter. be in their the

in have is, that time bit little have phase goes. in I they another the reported referred a have the groups. start number And most that to at are small need how and think manage time. groups dynamic patients we induction/titration want them more that of interesting still kind of There in referred see to patients to of So that one through a clinics clinics maintenance a

that be color more that we'll Maybe another quarter. on one that's draw have So full next conclusions. watching. early too Probably we'll to

Henry J. Fuchs, M.D.

the regulatory regulators I ongoing arrangement during simple of time having because a have licensing the European in that And is would we the development think didn't more fact that protein were dialogue difference and similar than it that I at Kuvan. The seen possible offset the a like is be different. biggest PKUs. FDA the is worldwide experience of there by the benefit to case the of we the simply mainstay development. patients are the a reduction fact additionally the the who phenylketonuria that phase is of not and this that that management of diagnosis then we regulatory, basis had with intake two in It's on respond different EU But substantially EU of Kuvan and a in PKU just that use of management between think The is global the the diagnosis. lot of

the a there think and our very relatively for Phe but submission the that think intake doesn't that's call intake. not in in the EU And face recognized is for tied for didn't in little is hypersensitivity bit – that restricting that freedom that there's Europe particularly, United it difference Palynziq thing they U.S. that compared States needed both protein IgE Europe clear is intake reducing of positive in perception to in substantial which diet. in we Europe, the complete type on offer unmet going pretty non-responders and points Europe, the that modest Kuvan of What and reactions being intake. favor normal U.S. what was about restriction is and more have – the being data anaphylaxis patients demonstrates by regulators protein delevering the relatively have to who I problem how powerful to the protein between documented of it they're restrictions in IgE. evidence from I an also protein need their Kuvan a require Kuvan-treated still a even Final our it

until experience good opinion in EU beginning referred which expecting commission this that process, lead about in we're you And to the And the authorization, prospects. a dialogue, of about we doesn't being that of to So feel actually able European next remind just approval for adjusting to full to in year. CHNP of quarter then happen in of lack would the HX spite is opinion second pretty commercialize legal year.

Paul Matteis

Thanks, Hank. Got it. Great.

Henry J. Fuchs, M.D.

Yeah.

Operator

next your Markets. Kennen from And comes with question RBC MacKay Capital

Kennen Mackay

research, if other relating to of go I PKU comps the bit these still or question. lastly, just then the this patients? we how stage entry where should wanted your that there more I taking on your And Palynziq thinking overlap could negotiated for back gene wondering to any are your of us in generization prior one pending the patients about should congrats rates wondering on was of to Kuvan and if generic the Maybe a thinking step be you thanks from if more of there? would sort understand or little help Kuvan about saw the I responses. Hi, Thank was much we you you be therapies space and the the market fitting but market, from very guess, quarter. success with sort

Jean-Jacques Bienaimé

ahead. Go

Jeffrey Robert Ajer

Yeah.

understand see me I question. So, Kennen, let your if

would of you generic to branded analogs guide loss asking were I States, United that conversion Regarding for in the of any of kind Kuvan was of there if exclusivity think business. the pace expectations the of

I that guess So right? if about

Kennen Mackay

some staging or any are had staged there come I was of to. around settlements or some that the comps wondering that you unfortunate. more generization the of was I it negotiations you from just if was a it thought that if had

Jeffrey Robert Ajer

That of business, actually – – which standard months. because entry one is generic generic part later, industry out business staging other a large in then have six first the staging generic comes from the being what of the is only the and the one a profits of I for the is of of believe, we the entry in and actually, six months sort

is is personalized is lack. our where So that is for benchmark standard. there of comparisons thing That's pretty specialty sort medicine. high-priced highly lack a there which of The

We erosion. expect generic

a that degree We analogs high not have confidence. that, to of there look but lots you are great efforts in with to place of modulate attempt to can

Kennen Mackay

Got the there you. volume were color. for if Thank limitations. you sure wasn't I

Jean-Jacques Bienaimé

second your was What Sorry. question?

Kennen Mackay

sort wasn't Oh, of I I if sure settlements, appreciate there anything from the were limitations if that. those like no. – color. volume any or

So I there. color appreciate the

Jeffrey Robert Ajer

No, there aren't.

Jean-Jacques Bienaimé

with in protection erosion, I as think, in, although Europe – XXXX. this (XX:XX:XX) I XXXX there be issue that is don't only, the we But until U.S. reinforces think will some

that specialty pharmacies buy your we they same the product you model again, a can around picking that's as be pharmacy that we're and corner. receive a providing So, support This the see the is be to in erosion retail for when will you patients distributed Kuvan. believe of generic don't much the through entirely lot a someone

we somewhat future. the So traditional than an be will no the should your study it for believe lower real available, be is case there erosion interesting model erosion. But

Kennen Mackay

you. Got the for Thanks color.

Operator

next your with question And Gena Wang Barclays. is from

Gena Wang

Thank you question. my taking for

valrox Factor regarding VIII accelerated will one have XX%. I be you I approval. over for So just the level looking would assume

approval the how So, longer half X, first will VIII? that case which accelerated FDA X say impact Factor year. is wondering if year be Would just there specifically in somewhat the for of declining likely follow-up decision, data, would next X, Phase – further

Henry J. Fuchs, M.D.

support using interactions this document Phase document, that with Well, we're earlier clear FDA, levels approval to-be-commercialized based doesn't on X predicated that is And for will durable X, principal the using tell is from I from registration. had benefits the the going guidance to Factor initial trial the risks primary decision population material. the the the the from expression we be what the we've on get and to guidance the But FDA's in VIII And view mentioned registration ongoing going X and that considerations. net mainly from that can pivotal subsequent our very be Phase is VIII Factor of durability come on on for trial.

data coloring, to term the will So essential that longer be but not initial decision.

Gena Wang

helpful. very you. Thank that's Okay,

Operator

next from Vincent Bernstein. question And your with comes Chen

Vincent Chen

one you therapy. gene the with a ask broader taking to I respect very Thank back, on I'd taking like Great. your guess to strategy much question. for step

therapy a pretty gene almost in proven the to And gene seen companies out, of the argument less is that is the science seems we've deals other therapy more space. or strike really So environment of flocking to favorable. regulatory number the

rare natural therapy to portfolio. robust capability is quickly in-license a expertise disease out a of the bit gene and So gene broad able manufacturing market a build readily that's bringing a company to therapies got owner for assets, a

was BioMarin me quite for strategy, given least impressive, a would So manufacturing platform but been be and situated bit deals. expertise that this at established of it ideally terms disclosed to your quieter really seems your in you certainly

guess you the developments? therapy thinking versus portfolio for two, gene use gene that I how the therapy, about your and internal right this the the in example, areas of way? broader And focus, LNA am in plays about business I of thinking role First, a strategy are

Jean-Jacques Bienaimé

I think you're right.

Henry J. Fuchs, M.D.

perfectly We're poised.

Jean-Jacques Bienaimé

some And we We're with are we that and up reason candidates. we I looking perfectly done think poised come with are deals That's many reason. to haven't is just one at capabilities why the – because did we ensuring we economically PKU that viable. whereby gene are orphan ultra we don't and also that a economic believe maybe the and different looking than approach therapy, viability, players of after we opportunities at have have gene diseases is going therapy

So that is our position thought.

great great it's releases, line. the So we not doing be bottom are gene going a lot is of therapy sure press deals for to little ultra-orphan for

Vincent Chen

And how - think about you do

Jean-Jacques Bienaimé

indications. larger (XX:XX:XX)

Sorry.

Vincent Chen

opportunity think that attractive finding ways therapy gene therapy indications where to about there. do look in in you opportunities, How that truly the about the agree got science something I do terms how where attractive readily confluence find is of bit addressed gene great, but to something be in finding really that's many a think things tend And by the gene little challenging can do of you've therapy you and more those? because those

Jean-Jacques Bienaimé

believe we believe exploring we and in we of using there actually, you challenge sense but now to need that that opportunities, enough economic But gene long-term Well, incidence that some. to what is as that allows we're the market some – market, significant which is finding your patients, your opportunity a explain second are existing in the it's somewhat question the right have – product therapy be a viability. prevalent has not an large are only

Daniel K. Spiegelman

pipeline sort of of on the we we're couple strategy part And coming in thoughts the to is to and good our going talk have our how a what news there. about up Day of R&D is and weeks long-term build

at that provide hopefully, So, can to answers you we time. more

Jean-Jacques Bienaimé

and because gene are companies. manufacturing regular our a And needless approached are that on gene we emerging in therapy, say of we to therapy capability expertise basis by

likely deals could some the that So in future. it's there

Operator

Lugo William with Tim comes next Your question from Blair.

Myles Minter

Hi. fit trial field with potential just taking that when putting looking I would can safety you about in something the pertains update sera sort top Thanks category? Lugo. line like positive ongoing question. for patients Myles is data. just which expect the that Mine for very and to of is valrox how Thanks. Tim on in And whether aren't enrollment, sort can AX. part supplementary we the sera that on the positive label on AX the in am concerns the valrox you second an be, approved, label I'd much imagine hopefully could seeing provide extensions, of of wondering any question the Minter to case

Henry J. Fuchs, M.D.

no Yes, things usually We give enrollment safety updates. so detail, don't to report. blow-by-blow

bit is safety a the of to antibody it's time slow the a study. it's study It's put you, there remind anybody a tried has because to first a purposely positive gene that in, Just knowing titer.

are might we lifecycle that be So and have we think early and about we're – some a about label ideas initial mapping action post positives, haven't we little it's concrete completion the that data sera given data where taken haven't some of be gotten to first as the that's an our that And as supplements to the specific going looking beyond secure And be and at we've to approval but still going guidance for more about indication. reimbursement. area valrox to is be, by required a to informed really play broaden to how to it's payers at what it, extent order we and but clinicians carefully and a date. priority looking it, the far very relative out

Myles Minter

Okay.

Operator

question your with Joseph Company. comes Thome And from & next Cowen

Joseph Thome

questions. Thank you taking my there. for Hi

Brineura's on launch. one Just

the broad indicated look at other? the and up of gating How Are aims, kind prepared getting of or you you. the in to two reimbursement inflection launch sales. of more we in of an You identification more a these that either patients. guide one Thank assume steady that? could we about you're than going remarks grind after more when you're of thinking And

Jeffrey Robert Ajer

combination Probably of both issues. a it's

of is a A Such we of for could go Morquio prevalent kind big that after. for that. wasn't launch disorders, we time we've the think launched as from patient Vimizim warehouse population guided there other when the that of some like case I of including

not of we going to guided in pretty of it effort CLNX incidence XXX,XXX. is incidence, X The So be rare. that prevalence. We commercial was estimate a

time pieces pick in working consuming we're we named as efforts to time, programs screening successful, the sales be patients we same At need are these the through targeted and the That's So in that. well. reimbursement able putting to up on time processes. consuming. disease That's awareness it's out including We're appropriate. and be approvals progress markets patients to making where been utilizing field, but

going in forward. the and the CLNX make will contributor noted slow Brineura slow really but combination part said revenue of prepared expect, So is that, progress we'll on Having picture be of factors ramp. we those and more the was – as revenue of rarity that are the steady remarks, that meaningful revenue a of

Operator

will I back to the Q&A call the turn concludes J.J. session. This Bienaimé. now over Mr.

Jean-Jacques Bienaimé

Thank our and you you. call today. on operator, thank for all participating

the XXXX revenues another growing compared to revenues in $X this alone. we believe and year on strong $X.X we the quarter, with commercialized U.S. can continuing products approximately in So With year. we half of opinion and Palynziq full the XX% year-to-date deliver XXXX, year last for revenues a delivered in currently path in billion billion generate EU on track to first

help with So expect decade we plus it contributions coming we the quarters. launch days, experience are the that U.S. is significant early over of pleased even very Palynziq. us though Palynziq our drive Kuvan And will of from

pipeline look to at of November forward we the thorough relevant So and providing other overview X. on a R&D Day topics

there good-bye. your and you to you hope continued we So see support thank and for

Operator

today's conference call. concludes This

Traci McCarty	BioMarin Pharmaceutical, Inc.
Jean-Jacques Bienaimé	BioMarin Pharmaceutical, Inc.
Jeffrey Robert Ajer	BioMarin Pharmaceutical, Inc.
Daniel K. Spiegelman	BioMarin Pharmaceutical, Inc.
Henry J. Fuchs, M.D.	BioMarin Pharmaceutical, Inc.
Joseph P. Schwartz	Leerink Partners LLC
Salveen Richter	Goldman Sachs & Co. LLC
Christopher J. Raymond	Piper Jaffray & Co.
Carmen Augustine	JPMorgan Securities LLC
Srikripa Devarakonda	Citigroup Global Markets, Inc.
Martin Auster	Credit Suisse Securities (NYSE:USA) LLC
Aspen Mori	Merrill Lynch, Pierce, Fenner & Smith, Inc.
Eliana Merle	Cantor Fitzgerald
Paul Matteis	Stifel Financial Corp.
Kennen Mackay	RBC Capital Markets LLC
Gena Wang	Barclays Capital, Inc.
Vincent Chen	Sanford C. Bernstein & Co. LLC
Myles Minter	William Blair
Joseph Thome	Cowen & Co. LLC

BioMarin Pharmaceutical (BMRN) 26 Oct 182018 Q3 Earnings call transcript