BioMarin Pharmaceutical (BMRN) 3 Aug 182018 Q2 Earnings call transcript

Company Profile

Participants

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.
Robert A. Baffi	BioMarin Pharmaceutical, Inc.
Cory W. Kasimov	JPMorgan Securities LLC
Salveen Richter	Goldman Sachs & Co. LLC
Joseph P. Schwartz	Leerink Partners LLC
Robyn Karnauskas	Citigroup Global Markets, Inc.
Christopher J. Raymond	Piper Jaffray & Co.
Matthew K. Harrison	Morgan Stanley & Co. LLC
Phil Nadeau	Cowen and Company, LLC
Ying Huang	Bank of America Merrill Lynch
Ashiq Mubarack	William Blair & Co. LLC
Martin Auster	Credit Suisse Securities (NYSE:USA) LLC
Timur Ivannikov	Raymond James & Associates, Inc.
Kennen MacKay	RBC Capital Markets LLC
Vincent Chen	Sanford C. Bernstein & Co. LLC

Call transcript

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Operator

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

Traci McCarty

Thank you, operator. Thank you, everyone, for joining us today. management J.J. BioMarin's from me team are With and Bienaimé, Global EVP Research Chief Executive Commercial and Operations. of Fuchs, President, Officer; Hank Officer; Chief & EVP, Financial Jeff Chairman Baffi, Officer; EVP Technical and Chief Ajer, Robert Development; Dan Spiegelman, To products contains statements and of products different business non-confidential potential about regarding BioMarin's therapeutic presentation including areas development. of future BioMarin, expectations this research financial remind the you, forward-looking prospects performance, and commercial in progress of actions Results product materially market X-K may filings on the Securities Exchange availability those future differ of with actions capital, and and and and authorities, reports. BioMarin's Commission in BioMarin's regulatory detailed competitors, depending programs, pharmaceutical in such factors the XX-K developments as of by the XX-Q,

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

Jean-Jacques Bienaimé

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

track in year. So, and quarter we on on of the build another half successes very delivered first to our solid the

billion and products the update Our two-year Palynziq FDA us revenues approved commercialized With significant drug regulatory $X our quarters. accomplishment and accomplishments in in May six program. the development's guidelines These next over in of the financial for currently in approval on the of XXXX committee valrox for our meeting alone. advisory achievements set a an second of in now expansion can achondroplasia XXXX, approximately we generate of productive U.S. half track with on the up approval EU we believe very Palynziq, and included

QX last in $XXX XX% revenues and year. of addition a delivered than of we over quarter, second regulatory strong to In increase the million clinical progress, more

Jeff products very strong that. going pace for is on our with and and to launch commercial continue to the patient of you give details demand Palynziq, We some are see of the pleased legacy

$XXX million milestones achieved GAAP total across roughly a with to who the XXXX year profitability for expect the Business of and more incur will half a billion. to And deliver strong the XXXX, revenues net key non-GAAP review call Having on in while detail. Jeff remain for track full to excess results, we loss of While the we financial in year. of accomplishments I Commercial are the pleased pipeline in over first will delivering turn Jeff? late-stage we the our $X.X

Jeffrey Robert Ajer

the same you, last with the commercial the revenues year. team's BioMarin generating in to quarter XX% Thank of second I'm extremely increase period in J.J. execution a XXXX, total pleased compared

in commentary approval product Before Palynziq reviewing launch some U.S. like the provide specific the in following revenues, to on I May. would

early were clinics the XXX-subject been since referred we high-level treated received said, III from have than Phase J.J. interest clinical physicians, patients of to As accounts also been launch being trial All patients. approval. for patients XX-milligram to dose a up More the XX dynamics study. which with naïve-to-treatment reflect a have RareConnections, and from referred,

third mind that our very So, line with the of enrollment far. by that patients to the commercial and timing Keep the we vast to patients converted of each visit scheduling majority pace of end-of-study quarter the to therapy pleased patients clinical REMS year-end. variable in trial smoothly the commercial trial These transition visit the their through which with progress performing. majority completed subject is We sites. in and end-of-study process the is is and subject trial are how patients Palynziq rollout to launch are working so have to an patient, expectations by therapy clinical with are for to anticipate end of the be of

of XXX certification and physicians REMS prescribers. unique ready prescribe, number includes is Already pace launch REMS at by the Another which certified to naïve-to-treatment clinics the important measure of XX the clinics. from initial of are positions

to numbers. the would trends which was want revenue look across no quarter metrics. on from revenue call, product markets. conference new all share approval to I from week the they induction subjects two commercial one on forward dose, active include products, favorable for turn benefited We of Naglazyme and the number contributor Naglazyme Recall, results those saw to July. only also the behind key update first the as on available more other like a providing quarter. phase generated during on second now quarter-to-quarter. of therapy defined as in as product, region approved third as unpredictable the both Vimizim, Vimizim of to patients revenues patterns the commercial our total a America, in ordering Latin therapy quarter as orders supply second and results certain MPS those portfolio our sites with large and We to key in Palynziq. as to was Despite in in and financial launch growth treating the contributed order call guided that results and EU/EMEA due timing number well from or to strong a well became of we our As sharing maintenance titration couple having quarter

last delivered As revenue XX% for the a second $XX quarter, X% million to the year; quarter Vimizim increase million, compared last result, to of and quarter was a of year. $XXX second in second compared an the increase Naglazyme revenue

guidance Brazil remainder patterns of MPS brands. the due on previously revenue we annual uneven reaffirm remain that XXXX, cautious anticipated for quarter-to-quarter, to both provided buying For

early identifying are approved Brineura to the EU/EMEA was course in globally with three the of dozens markets many with well. the We're Moving progressing the treatment in pleased in which EU the the of treating both children their efforts children XXXX. quarter We're XX patients and new U.S. diseases CLNX, in for now across from of region. our to

working making a challenges in major steady markets. and EU with in and Even approvals are patient patients diagnosing markets coordinating on treatment, seeing we're name in presented We gaining progress the increase processes price also reimbursement and through smaller unique by therapy.

that million optimistic an contributor $XX are diversified from an our become increasing The business. important our Brineura it revenues of quarter second to part base total will as contribution and we was

Kuvan. on Finally, to

the year-over-year. in by quarter in America resulted strong were Kuvan million $XXX of revenue in continuing growth North markets. growth X% ex-U.S. and results driven of development Solid

Importantly, North saw we in patients XX% growth commercial America, year-over-year. in

revenues next the specific So, generated the and of launch to second quarter on metrics closing, we company commercial in strong starting U.S. you XXXX in look with forward Palynziq quarter. updating

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

Daniel K. Spiegelman

Starting Thank XXXX $XXX were for of million, revenues contributions an BioMarin all full from record to half you, line top our press summarizing second the by Jeff. of first a release year-over-year increase of driven Total marketed results products. refer on the today's Please quarter. results. with XX% financial our details strong for

commercial growth on current believe XX% year vosoritide. we after grow As and portfolio based J.J. and alone, a we then generate approximately that $X accelerate mentioned XXXX revenues in our can valrox revenues revenue billion with in and/or

one addition reduces no strong there of business, more third territory. the any relatively product roughly regional In a reliance therefore we single To is diverse of secure diversified very Moreover, mix. base responsible is a and to for mix a that product start, net growing product revenue on also have revenues. and than our a

to of the sell, example, net year revenues XX%. For products U.S. we the XX% ago, to four for this it's of years product and drop the accounted expected

hedge in years contribute a accounted In products, approximately is product where revenues XX% broaden regional to building year In more markets and the this expected risk in ago, expect to the any essentially four more of those of volatility X%. we add future, revenue continue and or for country. new addition, mix times to Brazil we as and one uncertainty reducing

mid-single now the is European XX particular The on for Pfizer's Agency in sale NDA FDA's of $XX a the and Marketing under royalty Inc. $XXX the talazoparib. quarter; accounted for a expenses. in a Turning Medicines Authorization as if digit note note, acceptance we milestone XXXX, of milestones, by be million items milestones million regulatory from operating as of sales would intangible Application Of an entitled June payments approved, to to triggered up gain as Pfizer in well being additional sales. submission million payment of talazoparib asset is to we an a in and an in received on

second R&D spending increased the million Moving year-over-year increased the of spending of to programs second XXXX. in well due quarter expenses MPS III on operating on in XXXX as quarter IIIB $XXX and This valrox to to compared as the million Phase $XXX to increase expenses. program. with was primarily vosoritide the

XXX be global gold increase year Phase this the the guidance total expenses patients in from similar order an study III has to in announced the This planned patients of year, we resulted rate For standard standard bleeding patients quarter, versus planned we program R&D of $XXX the GENErX-X accelerating levels XX to R&D demonstrate additional study. first between to half enrollment on on endpoint Last while an and in in care. in of increase half potentially second annualized to our million. enrollment million full $XXX expanded the superiority in to originally expect

this guidance, line including non-GAAP we while other importantly, bottom absorb GAAP expect to all So, spending R&D increased and results leaving unchanged.

increases million second quarter net loss net Turning payment revenues increased received offset partially to the were in a to loss Vimizim driven net the as by by year-over-year the GAAP bottom Pfizer in R&D quarter compared was as product XXXX. These decrease results. $XX loss to net as GAAP $XX due profit expenses. line of gross in increased was million Brineura talazoparib. primarily milestone for GAAP from The higher and well

of of the $XX in $XX income was to income non-GAAP XXXX quarter non-GAAP quarter the million XXXX. second million in Our second compared

track income remain $XXX million non-GAAP on full As between we and noted, million. $XXX for year of

at of to we equivalents year-end of terms as and cash $X.X billion XXXX, cash, as had XX, $X.X billion June XXXX. investments, compared In

and of in convertible a senior forward will subordinated $XXX approximately which as Looking we million notes, October mature have XXXX. reminder,

Under equal conversion observation maturity, excess or any average shares notes. offset these promised, surrendered these into XXXX. the dilution previously BioMarin settle of on XX% arrangements entered based issued counterparty shares call and be be previously cat price conversion BioMarin in the principal were XX-day conversion receive a if conversion from stock value, agreements excess BioMarin's to conversion who of net their representing settlement the the call this will banks we amount utilizing the at portion BioMarin bonds net their of those will have cash of we from of common that issuing. time receive BioMarin notes for value. that and for at By of with will dilution call notes cash small. the maturity of very excess the share would arrangement, and next the our to of shares settlement holders VWAP As is have period, no volume-weighted stock price, cash exceeds $XX.XX, a the To means receive elected conversion What notes also

is issued For VWAP if the will total approximately XXX,XXX be would be XXX,XXX the shares example, net observation $XXX period shares to equal shares. approximately at $XXX, and

the more Kuvan another to expenses indenture Vimizim, For controlled XX, closing, supplemental settlements, Naglazyme in information XXXX this BioMarin increasing indenture year. and and quarter and refer excellent half first our please continued strong the the about non-GAAP profitability XXXX. first convertible precise delivered progress In dated note with October operating solid sales, towards

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

Henry J. Fuchs, M.D.

Thanks, positively July the We Evaluation Jeff. as walked important pertain valrox program. impacting our for hemophilia in but our were quarter, development. draft Biological on severe the of Drug Centers global and issued and this On a scope guidance Research development has program a highlights specific through the Since J.J. the spend the to with guidelines valrox treatment of they product gene for more very I'd to for pleased Administration key already and A. recent moment Food our like XX, regulatory equally therapy

guidelines the treatments. focus innovative approval industry, For development therapy the the the eliminated expediting gene and on agency's of

for are X with Phase BioMarin, aligned the program. draft path and our For development specifically that we guidelines the pleased design ongoing well

to or to a are reminder, that by should fully of considerations dose Back and XeXX already one be of after a GENErX-X the using name our is quarter fully the dose the to quarters two the most of germane enrolled just GENErX-X be with development guidelines, the program. few enrolled expected XXXX of draft that. first XeXX end part As the

GENErX-X may data patients subject rates our missing for observing collect six information; for medical bleeding to records through annualized and a and recall bleeding for months lead-in importantly, data and rates; this annualized collection is in bias designs. First, period on part the retrospective based be to GENErX-X of the study study

need view levels on should assays evaluate as between development, surrogate the of BioMarin's that the a that chromogenic risk sponsors and activity we made valrox's provide delineating discrepancies confident information prior considering we're pursue accelerated approval they'll Food progress this endpoint. at early substrate tremendous whether Administration assays of evaluate we Second, the these one-stage understanding clinical and the can Drug factor time to in to clotting a of and and filing. have and basis point using course findings is benefit to

confidential. such as one-stage pivotal range before. a one-stage out, further the remind course, the groups approximately indicate one-stage discrepancies published study in Of reports the reading activity end assay was one-stage details proprietary still be you one, and remain that within of investigations this reminder, And XXX% conferences previous XX% chromogenic well our result. and and chromogenics We've approximately Further, specific at result normal. have between of in pertaining reported chromogenic mean reported and than the would and at other discrepancy assay that to have was result medical previous will our XX% the higher when our year the assay

aware means in of to importantly, copy these For a et in note take is the the are that that types. of the Also, traditional can make rate But the endpoint hemophilia factor endpoint for be have we're clinical for in to program. endpoint the A these to may for be al. stage observed And pertains one advantage Journal results, regard approvals. primary of of. prove is higher no this December and to efficacy generalized as our point roughly Annualized accelerated clinical Tracy assay considered transgene client activity this benefit, XX% that assay strategy third won't activity approval plans you'd our our chromogenic findings by assay factor of Medicine if approval can and an in and efficacy of comments before, surrogate with by provide reasons. England opportunities bleeding reference, publication one-stage between this and factor specific present expression and that a as note pathway, discrepant for regulatory levels chromogenic and like the under so of talked well these a are way regard to And approval in accelerate the than primary endpoints the unprecedented. pathways. there IX VIII is consideration action would one different recombinant our we considered FDA discrepancies authors about we've been results. we're assessment up we understanding last details about be provide can for and in phenomenon on is endpoint this have a agency of endpoints could demonstrate products to way competitive while accelerated George New example, to have the to early we that In in year, activity this our humans of yourself. may

the various goes determine and results on methods on from populations. are working range high assay we of use resolve high FDA said, activity the say a goes a clearly, a activity have Drug support activity we you factor level Administration level we activity very representing factor bar. as and I that of of as recommend the surrogate on factor achieve of and we within that discrepancies then And demonstrated to normal factor a activity this and to the However, to endpoint; this factor endpoint, Food say targeting

products in on BioMarin factor are together and gives prospects manufacturing in the our along us outside The results The Valrox guidelines Taken scale commercial important for reach advantage. Valrox. gene believes capabilities bar very design with an range an for draft our gives our recommendation the raises FDA approval that the with greater and activities draft study development for us that of the of approval normal confidence pathways. guidelines therapy supportive competitive accelerated

followed the We are that. quarter QX we've to decision XeXX a by ever after XXXX and in look the right as and more develop that path XeXX GENErX-X the the encouraged enrollment GENErX-X chosen completing than dose in to or as well two we forward

ongoing health we're parties. very we're aligning development and encouraged with EU with regulatory addition, planning well the U.S. conversations by In authorities and our

pleased IIIB. of forward, of our And for to refer we'll another to Name, programs, XXX or development name, introduce the briefly going for alfa that International a we the Turning program. we BMN treatment Meeting INN we're product at that tralesinidase you on Type that September. to to Nonproprietary few look updates you update when MPS the note, SSIEM as providing ongoing study On year's forward in early from this

So stay tuned.

calendars development in-depth for R&D the November we're research the you you'll BioMarin's be will about as near Date how sharing to to will to team IR Day to now capabilities the The up and on pipeline it I Finally, your mark questions. X. remind at open thinking support, an future look Save attend; future. notices and setting Thank very as you I We of look your our beyond. much We hope up the for forward well want in

Operator

Thank you.

Your question JPMorgan. from comes Cory from the Kasimov of line first

is open. line Your

Cory W. Kasimov

Good guys. think couple Palynziq few how Hey. product all, discounting the here. over of taking my then wondering for second for guess, A I'm the for we the modeling launch. quarters probably of question about them Jeff Thanks questions. anecdotally naïve-to-treatment drug have on afternoon, I the those XX it's first have Or Thanks. but on drug primarily to free referred at first patients patients should RareConnections And switching a point? been this I in whether the is, these roughly you for that that if have Kuvan? for clinic, your at sense looking Palynziq, patients from this are yet not are do are

Jeffrey Robert Ajer

do question about starter Hi, don't Starting to have great try I'll starter that we don't intend free unlike a have a of This Jeff. drug program them. Cory. to reinforce the questions. for cover to a couple Palynziq. I and we modeling, is want program Kuvan, of with Thanks both

we a their So, six starting starting third when by followed induction quarters at and up patients quarter. those on numbers patient maintenance reminder dosing of we'll titrating have for dose with the end reporting be to the

So, coming for therapy. in to that when all reimbursed you see place patient. play a those The those clinical only material transition patients commercial is be will the trial free drug is in patients, way

patients introduced parallel, RareConnections end multiple we commercial available program. Through things it's study are concept or at patients they're trial that, those through their through managing already on referred sites. do those clinical parallel. gate, of that clinical clinical all visits have I we In learned our drug, of experiences So, drug. the their to best commercially-labeled Once to they trials get from patients with that transitioning in for into two

First is, label can to fine. that's we on transition getting work drug reimbursement. the free drug, work switch on on In we and approvals commercial that's getting so parallel, on patients the that if then reimbursement

free saying expectations with there. reporting then, our can't mainly Palynziq a be pleased all are in XX-plus will only therapy. numbers, first naïve handful launch. role again, to patients, the this in So, at the rapid we're meaningful those There possibly switch really U.S. resist of types exceeding of a patients. I months patient enrollments those for are in reimbursed about is am They're relative question naïve there. quarter of that now, Kuvan how your from of drug to a the a inside several mix patient And when play uptake I And patients stage. There patient naïve

Cory W. Kasimov

you. Okay. Thank That's helpful.

Operator

from from line Richter the question comes Salveen Sachs. next from Goldman Your

line Your is open. now

Salveen Richter

payer could an decisions step trending how then how incorporated? give program, just Palynziq, to is Heme for on coverage my secondly, on there? A enrollment update With edits just questions. is Kuvan on taking us you being with the Thanks And regard

Jeffrey Robert Ajer

Okay.

the first So, jump and will the one. is in hi, question This on Salveen. second Hank cover I'll Jeff.

place. our Palynziq presentations. effort those payer So, we're of in getting put policies of accounts payers reflection national to And see kind major are of in front the starting a coverage with

requiring Existing level that And edit course, and than for a existing so indication introducing therapy specifically. our different policies on failure or management. Kuvan reminder is Kuvan. coverage far of these look diet than with management, consistent payer solely include That's both a would step patients XXX-micromolar label. our A greater fee label

And forward, with yet, minority on be therapy patient through So far, that importantly, we if sign we're in payer the as crossed up question? it authorization policies coverage perhaps hasn't does or haven't wouldn't Kuvan of Hank, surprised in the run no explicitly into but so step of cases individual second going with more that far the going payers yet.

Henry J. Fuchs, M.D.

amount enrollment, don't There's by as And so far. we're you during as Salveen, updates we imagine enthusiasm of busy for of patients. specific trial. we're Yeah. know, really lives give could On might so change pleased trials. the the seeing on enormous getting the that modeling clinical profoundly enrollment to suffice course But and we're those treatments enrolled an our patients you enthusiasm of say, what

Salveen Richter

Thank you.

Operator

comes from of Partners. Leerink Your from question Schwartz next the line Joseph

Your open. is line

Joseph P. Schwartz

fragment Great. wondering, risk event Thanks production batches view current the very an processes given was for just how your your valrox? space, a of much. you the entering DNA procedures in on I and anomalous based

Jeffrey Robert Ajer

question? you Robert, won't answering mind So, that

Robert A. Baffi

We starting Yes. been that They're that raw followed the I you have can and very our materials and And we materials. understanding is good GMP and materials. of specification, those of by all released certificate starting a quality They characterized. recent very analysis are have of event tell materials what closely.

working batches would that appropriate is mention meet vigilant starting place, master they to talked a we very sure our we material as that about and I that sort it, production bank system but of produce we have type those So, make have bank, makes starting materials we not methodology our those of of specifically mode. are while in specifically, levels also use conservative

so, material And batch batch our routinely with are from to for not one starting we each production. going

Phase So, I, that quality testing generated starting They've used to and feel We that very we've with we in have of II overall along. all confident the been material experienced the materials material. thus III far. been the generate

Joseph P. Schwartz

with you you to patients a in for And I the measure hypothesis? WFH Thanks. to DNA consistent able Is but the you it your your this to wanted who've just And longer haven't period leads human And, that something provided data, or been I can then, way you're patient hypothesis. treated expand time Okay, about circular this level also seen circularization know great. any in theoretical? commentary have is more Hank. stable this is in stabilization hepatocytes ask for on expression. that of observe probably or

Henry J. Fuchs, M.D.

Hi, Joe.

XX beyond two WFH I So, there, we years. (XX:XX) difference provided between and we're plateau VIII through what did and week more biggest at And expression than XX. already the that week XX, little looked was, the XXX week to to similar can't updates us think, Week any pattern a gives factor the XX provide it occurred, that bit in of nearing confidence phase.

half piece settles the The gives is confidence that that phase. plateau out studies settling other us that a the the key peak about in about phase show preclinical of

levels. tuned the of activity that variations because factor tissues, So, And explain of will biopsy undergo and and or extent individual the for form. form data. case for the is the saying patients regards imagine phenomenon. now activity with that patients in as and in that say, a going the in assessing to of yet of more next variation in to typically that's as individual liver information middle for require with world in you And whether there's bleeding disorder. it'll humans, reporting is the the begun, stable question. which can far while That this human that That in stable does there we're I'd whether to basis we able the circular sites next we sub-study magnitude accessing investigators DNA we're presence or what time initiate stay be take look ordinarily has is be of of form circularization their for studies the the changed but hemophilia particular active as to now fact as work is promised expression in patients. are means which not then as the liver to And confident tuned And don't stay as year three-year longer to at settling discussions how that update ask the

many transgene of As systems, an non-integrating abundant form different mediated delivery there look case circular. a we is summarized forms. forms about particularly persistent at of in only AAV And the no WFH, really That expression if past circular can recoverable is by out the variety that. literature you and be than there's years other evidence that DNA

make animals period taken combined we've together immunosuppressed of taken message. So, that into is can and demonstrating express product fact sustainably can done now for and transgene studies circles of that can durable factor that cells producing humans. VIII believe express in in capable is and in time the stably valrox good with liver preclinical We

Joseph P. Schwartz

you. Thank helpful. Very

Henry J. Fuchs, M.D.

Thank you.

Operator

from comes Robyn question from next of Your the line Karnauskas Citi.

Your line is open.

Robyn Karnauskas

sense Thank guys. Hi, the you any like for you to patients PKU (XX:XX). but do for are now on couple dosed harp like, Not this, very much dosed have taking questions. a who the just questions getting are being

given there had And from hoping this thought easier on meeting? your this vosoritide, amendments your FDA that or post. the to than and you concern? then with in the the you release that, was prophylactic I you know give think expectations are trial wrote to thoughts what difficult call, press anaphylactic of what give your it more is that So, segue this is or big in this clinical to will line what be you any and line Thanks. Do on

Jean-Jacques Bienaimé

Sorry, Robyn, to of a bit. It's you're your question. difficult kind little breaking up understand

which (XX:XX) one, early titrating patients terms Jeff, the naïve, and starting what's terms our think is in figured is I them. talking of we naïve which of about guess you're probably in I out first experience

as much... we've done think I

Jeffrey Robert Ajer

of indeed question, your the that Robyn? Is Yes. nature

Robyn Karnauskas

you more originally risk and trend not we handful whether doing anticipated. patients long. having anaphylaxis seeing of (XX:XX) being Yeah. what to – than or of and like smoothly there I'm hearing are stay the about whole patients just about curious that's the going

Jeffrey Robert Ajer

not clinic product, hands. enough so clinic first say, the conclusions based our in that team patient has injection, gotten the count of draw back referral, scheduling patients been two positive gotten have end signal positive can on visit reimbursement on or shipment any of approval, visits or first the around of of I that far to have anecdotal been but first So, in firm I've can way that number which all through very probably The I far. reports those from so an those

Jean-Jacques Bienaimé

second think... question I your So,

Robyn Karnauskas

second it's expected. Got that press payroll? question in the have with release was and on dialogue – the the FDA else yeah, it. since that wrote what the Have you had in regarding with a And you the the panel trial you vosoritide you designed. you've Anything that line the

Jeffrey Robert Ajer

Yeah.

from line on panel discussion The little So, that. give to take vosoritide up the surprise kinds incredibly detail panel. No our the you just on with a of the well we more program. that heard bit development things

want call conducted just you, want see now They want randomized And They a have placebo to placebo trials. more They trials than single a to we trial. controlled controlled groups. remind trials. randomized see to to few in So, ongoing to see two clinical being things out different specifically. age

encourage patients sponsors achondroplasia achondroplasia. primary we've have contemporaneous annualized as randomized endpoints average olds data panel primary They X the of wanted growth of The a velocity So, current randomized are on collect under annualized more XX year other endpoint, history see growth the in to be on data individuals endpoint. as trial to to velocity one The in and who panel trial years we another five age. that evaluated. additional untreated recommended natural in

example, apnea functional or sleep For measures.

amount (XX:XX) several that incredibly said, an opinion elements. we're have because the of was pleased very are incredibly as to done we published members say And that studying. who also the I work so, investigators and data agency on And was on encouraged or by surprise, that much program track. referenced principal of our leaders Committee And Scientific development occasions well I that critical meeting no enormous with have on left academics that we that

So to right about people the that I the things. means that we're right think talking

we through measuring by And height attrition through ongoing. preclinical item outstanding our well adult-type is were a that durability marker any be by of our data as effect, to what We amounts adulthood treated our biological be gains. one expected there the in to obviously as effect regards of in assessed final data cohort biological the as the be also rests gains question much which ascertained clinical data believe evaluating very as in is final meaningful that on whether vosoritide treatment well and a patients be approach thesis that patients will treatment duration there was long-term and controlled all ethical, caveat the benefit we long-term. about study support duration, of so feasible, believe but As no one-year trials, a is should establishing the responsible around of of panel noted of the that effectiveness durability the the discussion to placebo can the of that expectation at there a attrition some over

active agency wherever our the same And in sure the that that we be with you committee everybody at saw make establish see dialogue look continue on to are of work advisory you to page. lot the and think and We and as page we've program, a possible the to from on you done I same get page.

Robyn Karnauskas

thing follow a that up. affects Durability approvability. same And

fine can with about So, think get volumes actually the thought like there's though does you up, is that approved would you you tell one and be you follow Anything What year that? that it's on reimbursability? think but approving feasibility your even what have under not reimbursement. and don't with you issues

Jeffrey Robert Ajer

been for remind Well, have now been vosoritide going next just is months data R&D to XX-something that We're with precise treated the Day, of Obviously, durability together treated of first their since effect last have XX haven't on our by as R&D months with timelines be, we'll to is to be the line durable try if XX and month that data in XX, now. that. through you to vosoritide. that when already of have given of the from the we months We're the just Day cohort you, to tuned cetera. et for years a of patients. anticipate And be And a up submission we the months III many Phase data, of to timings sort encouraged imagine going establish of readout when sufficient couple trial going the well, of XX. history stay natural XX, all for patients

Jean-Jacques Bienaimé

Or the biomarkers...

Jeffrey Robert Ajer

granted biomarker, this, much is this established reminding that so I has J. because as we've long is the a of seen bone time there me is excretion the the which And cGMP take didn't ex-bone for biomarker. treatment it been with to of When evidence for J. biomarkers, you and related being more cGMP the, that at look no precisely that's urinary stick of biomarkers call, effect. such attenuation

a together should durable have a substantiate we That that data together, treatment providing submission the and taken that biological indicates of the vosoritide. effect with of in lot time of maintained. data human So that be treatment be would we NDA should effect the the

Robyn Karnauskas

Thank Great. you.

Operator

Jaffray. from Raymond Chris the from comes Piper of question line next Your

line open. Your is

Christopher J. Raymond

you. Hey, thank

Just two questions.

be bit the the Hank first, least seem a guess, to I the on possibilities. using and issue factor, maybe, normal even the a calling with would guidance. range you're FDA out range. decades factor close Especially, activity should little be experience to target that where a in of Hank, normal draft So, is of of this odds at couple of not at for

have factor stand. guidance, we've guidance speculated folks that not talked this Some to targeting targeting might

is factor, you days I of early still Palynziq, hypersensitivity phase. And the in then kind occur that, thoughts but do on it's happen, into on eight it's so if to on here, reactions know Just any think, clinical titration experiences think I or insights expect given this recombinant weeks they when not have wondering you'd therapy. especially your I like Any that gene obviously that?

of Just that guys remind XX% saw in the think us, trials. clinical sort bend world dropout expect a are I would down like XX% and you to doing Thanks. what or you you lower? real the rate What number in something

Henry J. Fuchs, M.D.

factor-based regulatory wonder Chris. Hi, to We'll not assessments agency's guidance the activity if some clinicians Okay. let stand. for

Full on is normal The me accelerated based you about from need approval. factor perspective to provides benefit. that's agency measure reasonably believe a approval evidence reliable, an want an something, predict want to to the and likely they that they endpoint in context to activity of levels rates. that robust, treatment in still clinical Let behind of a to motivation context lead their – be likely language, remind which is that accelerate enabling the talk approvals, is they targeting in effect reasonably the substantial that

said, looking using recombinant could have lower activity factor one former imagine are same not you're when as those situations and there are are the Now understanding in transgene activity the factor levels that factor at of X% expression. replacement telling meaning you, factor assays a the biology therapy as what that doesn't I where range

that's wonder the that going as normal about to to bar we that the the is expressions between be transgene take whether it factor the who range more some So, bar and those will are to clinicians lower, sustaining or under if relationship time learn going bleeding. to be elucidate

while efficacy We prove as in a for it'll will be are level have that it factor before anybody a efficacy while range. it'll to clinical long activity lowered bar while could a same produces be be sample based factor substantial think before factor I they before, rationalize are can range we VIII that lower So companies require levels investments who levels where a fairly normal and lower the size a like normal. product and then which a on more of a targeting really

Jean-Jacques Bienaimé

whatever a regulatory approval. obviously do Chris, they mean, for regulatory said, not it's accelerated is hurdle. want. it's can this one, hurdle Hank a clinicians as a mean, clinical specifically But I So, I

Christopher J. Raymond

Got Palynziq? on it. And then

Jeffrey Robert Ajer

Yeah.

is the on telling you What seeing Chris, The and launch do of and you the both would are question with kind dropout you the we trial. regarding trial. about how learned was reactions far? And real the the point through of lowering of in what and those clinical the you quoted call So, to can we manage so rates gist induction think clinical and through rates what first patients thing titration were do I world? hypersensitivity the you expect a I patients what out lot your real in

made that what the clinical a and pretty we in and call. the And adjustments on study. with of by coming wound printed good you protocol been had that's the we up induction time there for what was was and out U.S. to portion we we So, that thought the titration launch had the of labeling trial, the protocol reviewed

to a at and and dose. to week based XX-milligram more they the dose carefully to XX weeks all adjustments and that's for expectation is in the reminder a dose milligrams based a that through feedback tolerability dosing, started that clinical way the to transition weeks. four to on our get do once They twice X.X fastest once-a-day that once they XX Just investigators are on day. real a managed pace world The that they is trial the a process, but the patient. they from milligrams then accelerate week, very XX-milligram They moved a up then to frequently, patients get – accelerate

carefully the would in phase reduce rate incidence I the trial. reduce and So, slowly to that and for reaction the deliberately hypersensitivity induction appropriately will go expect dropout of titration to and patients ultimately clinical

language we're trial who of really prescribers the world? with clinical PK the webinars have a clinical number do trials. clinical I already the we're that how expect mentioned, the first doing that we thing that the is that Now, in leveraging real we're experience don't The trial first label are run by had beyond the other Palynziq experience doing We've investigators through investigators. is, that thing of

titration, dosing monitor the So, Palynziq, and manage to tolerability through induction how through use they're for changes. on to how guiding particularly to

had to investigators individual Palynziq. looking a fair use trial speaking clinics learn events that at number how In doing to addition, are we've clinical of

training follow That on we Palynziq. with Now done that use and sales deeper MSLs questions. be by of up the of on their rigorous team. personnel BioMarin That's would mix a

I So, for pop-up. so to seen other all very early report patients, this far far that haven't any we in, than earlier the underway. early is small problems the so Too naïve days, tell anecdotal mentioned but

Henry J. Fuchs, M.D.

would I thing add. One

and way a amplify about by behaviors data place two kind an don't a first rogue so we've program back or to One REMS physicians. in our mess performance up I REMS to sites have And barriers of to are titration. program, outlying you absolutely They the just lot go call. this also. bit REMS any be hired on are to of jump a to types on eyes is This rate we have exactly have induction get prescribe. your we of that of discontinuation participate put program And that bunch little acceptable not physicians puts context. that, And them that this our think on that would wanting for so systems BioMarin's REMS people. into in specialty the active

to particular of side if also our this quickly very in business. the is So, very interest react the see the going equation patients, interest the and In problems, of but anybody's having of commercial quickly.

track So, to be then but I also key learned. that relearn watching of already people the experienced as going the to record have good they and new new also the think people follow people, things that a been Jeff people the to having the the have – that's teach said, bending down don't good

Christopher J. Raymond

you. Thank helpful. That's Great.

Operator

from line Your next Harrison the of from Matthew Stanley. Morgan question comes

Your open. line is

Matthew K. Harrison

taking Great. Thanks you. two question. the probably for Hank, for

the me to It going Thanks. you're if clear employ accelerated how details you – know your wasn't accelerate hemophilia want another Could you to I to not you're that you I much potential about deploy comment. actually guess, said and going didn't to about talk could earlier if I and to you an you or talk ask you're to about from question So, willing are, file? plans think way maybe, strategy filing. and see if your could if comments strategy. accelerate whether

Jeffrey Robert Ajer

an is, can I market can value everybody And whether quickly we in humanly yourself fairly possible. can I the virtue information recognize and assess pull document approval we've relevant of getting FDA's what and as And it's our for been for accelerated transparent together findings. about as think support that the read you Well, think valrox say likely the the can to guidance BioMarin yourself, strategy.

For advance to of documents. short market. of us, we This you were an chromogenic clotting is a a highly number in could proprietary of the timing to kind and talking not stop of than said drivers year ago, and more this issuing these talking scope think, competitive related we and than It's just because example, as confidential to issues year being key are these say discrepancies on assay one-stage considerations. want a occasions, methodology, is about valuable information. and not We enormously news more I about the FDA I of substrate

our shareholders we our patients capital maximum making we of duty-bound are for have get to lot that benefit in a enterprise that and in sure invested Our protect the them interest. for

more. And just stay so, tuned for

Jean-Jacques Bienaimé

here obviously, time, do are a gave that same the guidelines new these seriously at offer considering opportunity as I (XX:XX). we But

Matthew K. Harrison

And can helpful. just a I very Thank That's ask up. you. then, follow

next-generation in we those, on think as from pegval for specifically time, initial mean, about therapy be of other from of to programs, need those do you program? some guess, initial maybe how datasets your, call, programs you of patients many signal I'll I gene I may terms some see Just think your maybe a

Jeffrey Robert Ajer

That's good.

Next generation phenylalanine hydroxylase.

Just thing to that's a human this about the naturally remind an end-to-end, substitution cool And you is, work. the deliver required have sacrouterine enzyme. enormous to that in the pegvaliase would missing but amount to occurring human is it we done really enzyme, would for of love it

about is on pegvaliase. central talk numbers, It's in whose pull in the Kuvan the the BioMarin's advantage a here tell too PAH patient occasion primary therapy benefit And gene to gene little FDA to specifically intervention the is approach. ability can of to early what has liver than one lower but I and blood you speak the approved phenylalanine So, levels. BioMarin's more is the of I the really

effects is example observed drugs with levels pegvaliase phenylalanine that for blood thing that of Second the like substantial. we've quite are

talk run so that we great reduction amount levels. data thing a in would is it that require in sample PKU the efficacy our think we've what Kuvan the path development I therapy disease. definitively data Phe of prove a enormous to and pegvaliase R&D preclinical So bit gene far. you doesn't an little blood a great, give be. know Models I size of generated models the of that And the that'll third more we've to mention quite And got could that going about lot Day, we're well. and to insight into with Come

you and and introducing FDA think what gene therapies, assure will therapy proactive way contributor from that guidance how I I is put in with PAH about (XX:XX). similar thinks you position active document the BioMarin a a ourselves to the innovative we an hemophilia can on can energetic tell gene therapy

Jean-Jacques Bienaimé

to on should our things maybe you the a add gene Yeah. Novato here also manufacturing a this therapy Robert, the we're planning with basically program time and in about few that fact front start facility PKU California. And also

plan be the should scale, that first, – product. – is the the be scale want this as the commercial one So, to the I'll will with Robert? first-in-human potentially in that, start few Do to commercial it words see potentially a we so guaranty to you

Robert A. Baffi

Thanks Yes. J.J.

is we has a what or Hank talked about leverage alluded Phase that valrox. of pivotal and material That for demonstrated and simplifies knowledge early having clinical that this that encumbered we gives very our to own development seeing the in can in the manufacturing valrox study technology hand the benefit supporting support year to, leverage is, earlier base having us platform like the advantages as production III somewhere to tremendous capability built the a BioMarin the of the It has have with production program programs that of of it potential One up Hank quite great think, be production manufacturing this bit at currently plan other scale in very not many with to with by gene produce do aligns transition PKU to, manufacturing profound for us point, will that initial else. material would going development is and commercial because at on anticipate specifically clinical is be potential launch. manufacturing clinical experience that to facility. well a what in to as of products a production other the of the What to upside to we product. manufacturing rapid using I this our this material studies case commercial a that well allows has scale is The hand the all produce of the therapy for commercial

Matthew K. Harrison

Great. much. very Thank you

Operator

line of next from Nadeau Cowen. Phil comes question Your from the

open. is line Your

Phil Nadeau

for afternoon. then my Thanks One, one questions. taking Hank. commercial and Good follow-up for

of better a naïve trial the little naïve Hank, hemophilia, I'm your predominantly second are they can been time. to thoughts commercial how Where patients, get you patients both prescribing? have from? patient the or bit centers, you, coming that Then trial commitment the about at Palynziq? are on your same who treatment to the XeXX a physicians, with talk curious for treatment therapy the XeXX for Jeff, lack coming the filing they're term, side, new On recommended non-clinical and from on doses there clinical Are for

like and to quite given guidance arm doesn't at on getting to factor single levels XeXX could factor seemed that study well of behind as Just actually the the least the seem fit the not the normal six document. be up design with it with months levels as new

later? So, Thanks. is it to with and that possible what out XeXX do you file first figure XeXX

Jeffrey Robert Ajer

Yeah.

is this is I'll Jeff. Palynziq where coming first Phil, question, the for referrals naïve they're the which patient in from. So, cover

includes, Just able prescribe say, we REMS have process XXX certification greater sites. a let's That prescribers XX to of reinforce PKU at now that background, clinics XX are the clinical majority and trial than the little to through Palynziq.

the other so few of clinics. Most kind of you're of larger broader in XX, clinics. total group are Some and get have clinical trial time a the the PKU of smaller some other not clinical smaller trial to larger group in treatment are clinics from least past the clinics other That's the far. from you participated and some handful PKU naïve that first clinics experience, sites, that the PKU patients from by a of the get coming XX from naïve targeted large patients to at XXX experience first the into PKU starting that clinics. that are that But

Henry J. Fuchs, M.D.

enrollment registration question, that file will much we're XX approximately extremely the it's reasonable in three-fold be I is for dose we what gave, study the data comments it's expect that to group level a Xe dose still dose group crosses fully Based a even the XX I second the that enabling The On smaller, to enroll quickly the the getting faster. study Phil, after is, that's though to line. on high. interest your make assumption think going group finish we first believe

in anticipate to filed XX. will waiting of wouldn't therefore, availability major valrox for some dynamic would advance XX us unless so, general, second hold well shift, that be the study. ready I And in were of be to And

Phil Nadeau

guys. Thanks, Thank you.

Operator

the In of time... interest

Jean-Jacques Bienaimé

me let add. And,

would to quite for made the that good might a new it's with XX little not apply high You the potentially below also that competitors. filing, comment XX%. a And our because accelerated guidelines

Operator

the interest you that time, to of limit In ask yourself question. we one

of line Merrill Ying Lynch. comes question Your from next Huang the from

is open. line Your

Ying Huang

Thanks taking for Hi. my question.

you Phase study program. update patients for Hank, at on heard sometime would one the the be in II I next think, the saying that Just hemophilia next I, I the maybe year. from valrox

ASH? Day? mean you're at at not that So, we R&D talking Or does won't And is your November clinical that? this get about why any X update

Henry J. Fuchs, M.D.

So, really is chronic yes, means. it issue And because what expression. why by the clinical moving we're decided that's really more outcome. a to now be and phase gene And into durability that's of going

years between get That's patient. in activity As relationship now, of possible. using Take every may events same levels about I But not crystal equation. is, may start X bleeding the and for and breakthrough learn We what X mentioned, to it out it's when lower high from factor are. I it the level in what not the and activity as we bleeding what to factor the know be don't translates to why of clear that the question reduction as transgene. want

As mean for factor own because lower activity of approvals, levels uncertainty for J.J. just that our lower be to pointed factor not XeXX, bleeding. the activity out, are accelerated what part studies, eligible including level of the about reason going

want the That's to not way the the experiment we that frequency reporting it. reason the way activity that running we and factor running, want other principal and the we're do levels, what around. So, data relate experiment to reporting the bleeding to back observe is for we're that

Ying Huang

Hank. Thanks, Okay.

Henry J. Fuchs, M.D.

Yeah.

Operator

line the comes from question Tim Your Blair. next Lugo of of William

is line open. Your

Ashiq Mubarack

is for Mubarack Thanks my guys. This Tim. Ashiq Hi, on taking question. for

follow-up question. to on So, last the

VIII As levels? And strategy? Thanks. you durability, what of capacity? limited you be you you consider by scenario, activity continue valrox multiple in continues your long-term at factor would would to kind forward, at manufacturing And point look a would your that reconsider if contract dosing dose how to moving

Henry J. Fuchs, M.D.

once dogs potential contraction the collected driven in re-dosing important there. of data transgene not in so stays in factor much subject you humans, Well, in get been that does there, indicate is it in because hemophilia by IX that's hemophilia

is an delusional where you we important that's And that have it important. as talked re-dosing and could but really being effects, phenomena. adult liver don't will If become pediatric really see about phenomena

from for we're along concrete what our to getting to around yet us and us keen And on question most this hope see when concepts it's because data factor VIII Phase go in phase and either results. study re-dosing, in said but reason, of want and pertains protecting to you'll it previous integrity some all I GENErX-X bear us study We're the we data thoughts the I/II points as the that's from you'll not of not you the we when that really with program you, either. really understand integral of see to GENErX-X. about running And so It's Phase the should this are pass a going I should so interpretation program. really as see it in gave us that just through lockdown that and And have point to just the important for you're don't or develop I Ying bit to mode, is road. III focused none our I time see registration. the and remind and down the

know of a its to a we must quality. is but going high practice. quality going be landmark be data too need keen an are change to It's These We This to medical study run way. to kind. experiment in high

Ashiq Mubarack

might anything design on see sense. you of draft potential and how anything to kind you the PKU or made And anything you that studies, are makes studies hear just Did your interest reconsider forward? related That of there Thanks. moving Okay. FDA those quickly guidance.

Henry J. Fuchs, M.D.

a said, Like and mean, disease hemophilia gene is therapy the rare guidance it's particularly. I I guidance for whatnot. Not

company a for this patients like diseases. drugs terrible been years, the BioMarin, has XX-something rare with For our living of availability expediting for

honestly, feel like language I documents guidance So, answer. these we in our is the real see

working as to as more said, proclamations and closely fertile strategy look programs. it me be lot coming whatnot like suspect we development identify and we'll closer I with see So, if more won't the to path. most surprise the we out the a I agency that And get BioMarin's

Operator

the Your Credit from Auster comes from next question Suisse. line of Marty

(USA) of meaningfully for obsolete Securities proposition curious curious a you kind recognized of if And gene update approval Auster is develop Suisse its or values, line a curious guys. regulators. create my therapy, in years to LLC Obviously, is Palynziq's to Thanks as proportion taking been Credit of value value - be has Hey, going Martin Kuvan globally. larger comments efficacy Your then European you kind for on a open. if majority kind enough in with I'm but Thanks. question. different Palynziq, Probably very post U.S. few of but got any of Palynziq conversations will have Jeff, ex-U.S. the

Henry J. Fuchs, M.D.

publicly say And communicated I done seen what side. would that on I there's the we process we review critical to-date. the pouring regulatory filed. submission. after And we're you familiar you're can milestone with European a we're one the know not we've regulatory that the over say if about comments On when a process, is, four months

that as get the On the other too through FDA, the optimistic we very are through got EMA. we shall we hand, so

optimistic cycle first to and in of cycle we the through first As we we will approval. EMA the a approval, got get are that got FDA

of if over use the we're interpret process in in the that, cautiously the because term the future, middle optimistic, I Now, don't please here.

we're bring second that approved think through to half an be is XXXX. of MAA, belief and expectation patients European seeing Palynziq Our we'll I able

to than that Palynziq we're think nothing talk report with you're substantially U.S. not that the environment on currently Palynziq, could the subject in what launching respect be So, And us that's I for proposition ex-U.S. pointing long we're where worries value is big going time. it But different a ex-U.S. out for timeline. that to is a about to

and payers, a therapeutic also the and decade professional on spent both receptive a including and developing For we of stake with BioMarin's too market portfolio, market is it's important most and where treated the PKU terms associations taking intervention. U.S. market best is prescribers a the the in using in payer PKU hand, one how environment over that pretty

the is U.S. best. So,

markets, to last the it been other X.X for it's years. relates Europe great As and

and to used rights and develop X.X-year BioMarin the worth need tend to PKU developed that price approvals. European how recovered and and Palynziq. treatment patients about It's also specific can that go we've guidelines that reimbursement the to that to to prescribers, are noting you both both were I more after recovered Kuvan other we've published you PKU period notable programs And have for franchise. thinking through that the rights So, systems payers. tell markets EU

prepare So, we – to different hard differently. working are behave systems

degree for cost on has clinical based program. a example. of UK an incremental response France innovation assesses and effectiveness value So,

for systems. and adult the So, for strategies has really develop proposition. But It's is to each the second of the piece of powerful phenylalanine. patients. our we there're EU fundamentally, working different Palynziq remaining systems unmet PKU the illness are profound burden need and value a rooted reduction in And major of

that's really - the So, of for detailed response Credit we're Auster control on Suisse Palynziq in providing (USA) moving adults moving from Securities based a a power towards Appreciate world to phenylalanine it. seek to which the impact and world we we're with levels but for Palynziq. in Thanks Martin response. such Kuvan phenylalanine LLC

Operator

comes line from of Chico question Raymond Laura the James. from next Your

open. is line Your

Timur Ivannikov

Timur minimum about we think We market have had you what level we actually if on of Ivannikov is discussions. Thank question the should valrox? payer minimum the don't today. about valrox This for durability Hi. Yes. valrox. be do duration your thinking And for another based How you. you've And assumptions, Laura treatments? of know is

Jean-Jacques Bienaimé

to saying you're what payers, is So, payers? related specifically to

Timur Ivannikov

about duration specifically necessarily general, treatment? payers be maybe of this would assumptions but your market feasible what to a minimum in Not

Jeffrey Robert Ajer

bag. around eventual next require. payers. starts we'll It of I regulators from then, kind It are saying, into built to of expectation of Well, moves expectations And the payers out the clinical durability of seeing will end durability development an pay on durability at be what program. I are you price an based what look, are durability, will The are line. the kind finally, payers what your which with think mean, of

gene XX a happened, payers to say, down think, after price go fine, therapy an I'm said went expectation exactly So, XXX% it'll suggesting if last and able to on But Hank... that's response. months system be months we'll of to but payer will XX a X%, what based of at you that and and negotiate for will our that you I not

Jean-Jacques Bienaimé

that, time we I that. years, same by related that, years a I well, came something than research. of it. has more the to mean, x data payer the past that months in not historical another are product four going time, communicated way Hank to, as your because said we're several to the have And if no doesn't to get we At interested expect payer doing probably approval, we're it x with patients. to in lot actually five Nobody we think up actually, explain said, or covering – of ever have in to respect in your on work

to and the you no have so, by time mainly factor assuming And bleedings we'll bleeding but five by have document at on that be whether be four we after the launch. will able here years, – visibility to the we'll demonstrated factors not way, still rates VIII, by of

anyway on five payer We also on the current years more of base based value the reimbursement negotiations potentially than research, difficult of have seen, that treatment. to might early be it

although kind to really to way of there not your that answer a minimum. express is another question, that's So it's

expense so And a far. so, we have minimum

Henry J. Fuchs, M.D.

say regulatory the about the two-year thing was there to that our question, a to or a is, if And presented authority. health clinical major we data I I'd components think

one health hmmm, like, was patient. all was One authorities no of something, mark, from at something of some of the give the me hemophilia bleeding, joints target us to two-year that striking made the the comments that breakthrough resolved,

Timur Ivannikov

helpful. Very much. very you Thank

Operator

of from from Capital Your next question Markets. comes RBC line Kennen the MacKay

line is open. Your

Kennen MacKay

much could of that when obviously, chance moving guidance of I wondering much. and are there with approvals caveating, of so we XXXX recent of initial parts could at in and you point for sort to parts was Thanks Kuvan here. term any moving the Thank beyond Brineura very sort with there if that help you And vosoritide are squeezing of, sort valrox. stage some that? and/or me patient, Brineura the the see pegvaliase, was with preventing longer some maybe not, what sort

Jean-Jacques Bienaimé

in we to But, all, and start I'll report the QX in. year one guidance previous. generally probably give first Yeah. we of can step Dan when

bit first long-term of we to do actual during are year, You saw more that. in revenues. will the Actually continue guidance terms we quarter today, a of the giving

anticipate of generate commercialized As currency around by products we that we XXXX. will said, billion our $X revenue

that beginning introduction is some the of only just QX potential of XXXX. talked the until in So generic reminder that's Kuvan Dan for XXXX x-U.S. about projecting we market, long-term so a – Dan? U.S. guidance. is in are

Daniel K. Spiegelman

it think think your lot guidance others we and will anticipate add guidance moment. about Yeah. But moment. is to to most the I road. I feel doing don't down the don't the have When at that comfortable the we at appropriate, a

Kennen MacKay

you. Thank

Operator

Your next from Gena of Barclays. the question Wang comes from line

Your open. line is

Unknown Speaker

is This Gena. for Hey. on (XX:XX:XX)

asked. data, the Just could on additional color the a kind going of you couple you where questions at your if antibodies, to is? number SSIEM? trial any additional on is, patients MPS the for since very one quick questions, many on are of see asset give AAVX most any color the we Number are patients two of with could already what give that for how And

Jeffrey Robert Ajer

Yeah.

lot to level who yet. have patients and that titers when measure AAVX chance then patients by a titer to so to several first expression transgene is treating patients in way XXX way, for a study. activity the as immunity expose the And is weakest this don't study, that who also for treating but of because event under have the work. are For the study patients. titers known it slow stronger And preexisting it in in complete we also the weeks designed is factor positive patients slow It's stronger no before subsequent is we affect a we does there valrox, followed want

full is, a So, just we study. completion history And XXX MPS IIIB. a is enrollment slow update for I natural say give we cohort or remind enrolled have not started to anticipation. a And timeline can you an SSIEM, really all

enrolled, the of that Just that to remind after in you part that's the of essentially and fully study year has year a a we are period run observation.

at of at complete will roll for So will A that to observation a April drug completed in treated all patients which active XXXX, XXXX. of the time year. year and over be

of we're they'll year run-in reported who untreated in a of that the been on a XXXX, side of having more reported in we year, heparan be normalization heading. the normalization patients We'll all of have their data. Congress of levels, with in a World XX treated study. three brain for own some roughly patients, three, at relatively few April patients, of that a on direction signs natural position the function. this tralesinidase Therefore update but really have SSIEM, completed I of sulfate at just organ still patients treated is to size, that complete January year, cognitive we we're it's about stabilization of year think, a So, in the of not When enrollment of in definite early

can see and you the about to talk data. hope So, conference at we the

Unknown Speaker

Thanks much. it. Got so

Operator

from question line from Chen Vincent next Bernstein. the of comes Your

is Your open. line

Vincent Chen

questions. Thank on ones valrox, quick A the manufacturing path on you one development the very much for forward. one taking of on couple

runs different but manufacturing treated were one on vector? did in multiple the one or potentially I run? of the with prior don't valrox the in together group all So, products mixed get valrox from patients manufacturing run, XeXX to and made same mean just vat patients different dosing, initial manufacturing

Jeffrey Robert Ajer

you to Robert, that? answer want

Robert A. Baffi

Yeah.

presented I/II used different runs different And May. trial. have of batches a be will been from clinical treated our we produced produced in that production So, materials number which have we've III we've initial throughout both results the with clinical genesis Phase patients and Phase process, the of was the several batches of

and that the end strong batches So different number processes of have, a with scales we the will the comparability our by different facilities, us of gives and day, characterization production a of and at sense that experience clinical analytical made of material.

be treated the we production that material. so, batches on few a outcome clinical to significant patients not with of And be will basing entire batches number and our of produced

Vincent Chen

supporting additional then treating plan data could I your on see. And into trial hemophilia A with color you pre-existing a antibodies. to move provide patients the

are the what would that gene ability what have And that? to the So, would therapies? re-dose on data I this data implications guess, support

Jeffrey Robert Ajer

think, antibodies was AAVX, to transgene and of transgene a demonstrable pre-existing have of ASH last of is in I humoral elimination showed And complete to we expression non-human We treatment primates at AAVX. presented activity. year but on immunity patients poster, total not that attenuation of it titers with who an the there

past the immune that's get in gene the the idea liver. capsid presenting the to the amount is massive the that of we're vector break basis. is a and of that underneath to scientific concept so system swappable a protein system And and immune And defense the to

basis. the remind scientific And second And so me... the the question, of part that's

Vincent Chen

What will be with data like implications of this opportunities re-dose? the

Jeffrey Robert Ajer

implication valrox to diagnostic level, that level, one could of valrox be have for development, that that there one event work, AAVX in well have benefit not as the another not for doing may that to In and that way could that diagnostics not be in by FDA patients the is is for kind registration required backup contemplated a our naïve case we're diagnostic in everybody. at We've possibility having already accountable of patients So, a on requirement And, towards is companion risk well At versus we for example, companion regard. it needed. of we patients. same. companion your which would AAV a guidance two the if we're the identify study underway positive

Vincent Chen

congrats taking much All and very for right. you again. Thank the questions,

Jeffrey Robert Ajer

Yeah.

Operator

back Bienaimé. call further There Chairman, no time. this J.J. CEO I'll questions are Mr. over the at and turn to

Jean-Jacques Bienaimé

our Thank Thank you you, in for operator. today. participating all call

track. year $X.X conclude, billion on revenue very reach on quarter, to we believe track are we in in We another delivered XXXX. approximately full solid to we So, and remain

launch We an U.S. weeks on commercial metrics second forward in to million. the long-term in million we $XXX R&D the For and and also the $X on U.S. income a approved we guidance. approximately for that's of on a only. at billion an full look generate Day year, Palynziq And commercialized And of believe update specific after in between quarter now approval providing that. and revenues $XXX can EU XXXX. currently you track little few half with bit year, of non-GAAP third products in the in Palynziq And next we

So, thank support and goodbye. you continued for your

Operator

today's This concludes conference call.

disconnect. now may You

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