Ultragenyx Pharmaceutical (RARE) 19 Feb 192018 Q4 Earnings call transcript

Company Profile

Participants

Danielle Keatley	Senior Director-Investor Relations and Corporate Communications
Emil Kakkis	Chief Executive Officer and President
Vlad Hogenhuis	Chief Operating Officer
Shalini Sharp	Chief Financial Officer
Carmen Augustine	JPMorgan
Gena Wang	Barclays
Joseph Schwartz	SVB Leerink
Chris Raymond	Piper Jaffray
Maurice Raycroft	Jefferies
Vincent Chen	Bernstein
Arlinda Lee	Canaccord
Jeff Hung	Morgan Stanley

Call transcript

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Operator

Good day, ladies and gentlemen, and welcome to the Ultragenyx Fourth Quarter and Full Year 2018 Financial Results Conference Call. At this time, all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] I would now like to turn the call over to Danielle Keatley.

You may begin.

Danielle Keatley

and update the fourth conference full XXXX. afternoon results Pharmaceutical quarter good year corporate and call you, welcome Ultragenyx to financial and Thank for the you release ultragenyx.com. financial We've website detailing a on our press results, issued which can find at our Keatley, of Investor Danielle Corporate Relations Director Senior am and Communications. I Kakkis, Executive Officer; Chief Hogenhuis, and and today and President; me With Chief Shalini are Vlad Operating Emil Officer Sharp, Financial Officer. Chief for the safe not ended XXXX, that investors was filed and available the like will call provisions include forward-looking our which XX, filed Private soon; remind on within the Litigation statements year XXXX, be the the November quarterly I'd website statements that be Act all meaning section. that of Form SEC, subsequent on in with our of the Securities on will this our harbor as filed XX-K the in periodic limited annual of will of reports to our XXXX; forward-looking types December XX-Q report Form identified X, report on Reform Investors including, to, but represent statements of many substantial are of including the control. and views call and forward-looking These involve only this risks our beyond that uncertainties, our as date in results materially could Please note that actual statements. from any those differ projected forward-looking

see For actual as to Emil. statements well uncertainties and results periodic description those call now over a cause filed in further to turn reports SEC. the forward-looking I'll as business, of that to our from could our the with risks the relating expressed differ the materially risks

Emil Kakkis

provide by managing operation introductory our update last before on XX Vlad providing launches. thank Chief to joined as will you for Ultragenyx an the brief and and turning Good Operating Vlad us. remarks over joining start and Officer he development tactical afternoon today Hogenhuis. I'll commercial business commercial everyone the September call some

The on quarter close our our for update call providing will Camille on back update and been financial an look you have as truly things forward XX well the will as I year in come months and to transformative fourth last clinical XXXX. full programs Next results. Ultragenyx. to

Crysvita Over we geographic the course Mepsevii simultaneously regions XXXX, in major the of world. and have three of launched

successfully drug in submission for the to our enable Storage have third application we therapy Type FDA also concept year. Glycogen both with Cohort Ia. ornithine for gene our parent deficiency new [indiscernible] in patients launching application a a have product achieving two shown proof strong of Disease with results clinical worked UXXXX transcarbamylase programs, towards We In a for of our this X that

approvals early us substantial growth. fuel continue therapies patients Vlad. will currently development are approved future enable commercial to will to engine With Our limited who rare diseases, no or and with successes develop have the availability I'll This options. of a for our expand now over medicines that, and to it our new creating that turn

Vlad Hogenhuis

you, Emil, everyone. I'll launch with April. our Thank months available XXXX, commercially late in following was eight start for Crysvita, which and in afternoon good

and in XLH policies published Nearly be formal the start policies XX payers. continues States without the types writing split the and nearly multiple has payer on the who includes commercial XX% The a in and This plans XX% brings bill under from and and coverage mix We these of the patients have doctors in which that comprised to approximately remaining payers XXX on of a to with covers with end forms are quarter. million treating the basis. private XXX prescriptions. believe coverage prescribed XXXX many extensive payer a number have quarter additional We’re pediatric across commercial government States. we Medicare remains fourth By by patients of strong This pleased of had approving therapy. XXX Over to fourth other forms XX% therapy adult for patients on previously Crysvita with are process. of full have covering and quarter Crysvita as XXX between we December patients not United the enthusiasm XXst buy unique now total as all in adult on Crysvita United Part physicians, B, XX:XX United more approximately nearly for start Crysvita of be adding the the treated commercial launch with continues alone. increase with pediatric of to reimbursed This Crysvita Approximately great therapy payers patients approximately compared completed lives. XXX received States. to XX% reimbursed than that case led the at patients XXXX, the case prior end

usual by sales teams. the Now of Ultragenyx, and liaison improve to the process science physician enhancing a from the patient to separate medical and addition beginning we and saw dedicated development team in with commercial advocation

are community support the demand doctors with the alike. experience order serve with gaining guides early seeing to a size we've doctors we and into patients the our demand XLH. UltraCare Crysvita identifying these for the and in placing access for distinct service a three on to launch three-pronged have additional patient guides prepared submitted, as the continue in are therapy. many and patients. and the healthcare we needed reimbursed to evaluate team is on UltraCare on there this responsibilities Once we’re we months reached to success, meet to that patients additional best the and the therapy. provides Eight teams with experience The approach possible. and meet broadly Based positive have our the well of doctors, who may seamless liaisons forms team providers patients to team liaisons liaisons information launch, added growing This UltraCare diagnostic created working information launch patients for to quickly has assist the shares focused ensure we've as UltraCare and solely ensure in to As our patients established start and as are them

Most We’re and approval also of XXXX, Canada making at progress sharing in American commercial Crysvita and end patients, adults of global the launched our the same as one received notably territories. or North the in and the Hakko the have and agreement of KHK. profit by Canada and of with year partner covered for in expanding reach older. we age treatment Crysvita Kirin, pediatric is XLH Kyowa U.S. our

physician in as Turning Brazil reimburse approved treatment named Colombia we as Argentina multiple well and anticipate in continued to launches XXXX, to and in request. response to patient additional

to patients life and to continue regulatory major European encouraged the with new important Mepsevii Columbia of to financial we geographic Mepsevii, in currently Brazil. MPS three we're will turning Reimbursement call identify to an continues around additional with make including Overall, approved the With that Shalini, ongoing and update. EU Now MPS VII, United Chile. in look the briefly which regions the are and payers is to patients will discussions available. in turn XXXX, a world. that, provide we decisions difference the are VII In forward updates provide they with who as I'll States,

Shalini Sharp

month quarter briefly was XXXX of product ending following afternoon included million. a summarize. of a good and that XX million period XXXX Vlad, press was and breakdown will The by update, December $XX.X for today earlier figures. the you, these I which is Ultragenyx’s financial product issued XX, the release total fourth a $XX.X revenue We everyone. Thank for

revenue Crysvita million royalty For share year revenue in total December Crysvita ended for XXst, our April territory million. U.S. Crysvita revenue million sales This totaled in profit or America. of in Earlier XXXX. approximately Europe the product Crysvita $XX XXXX. regions collaboration. for $X.X product KHK collaboration their America, European royalty billion These Crysvita regions includes North we $X.X U.S. fourth were lunching $X.X This $X.X includes include revenues territory all during million. top-line from and months license and agreement revenue sales with in XXXX $X.X million totaling XX, and yen in revenue million. European regions, sales reported sales of Crysvita collaboration from which other and quarter this of of Net international month, the the $XX.X Keep the are mind sales profit our collaboration and XXXX in represents after recognized $XX.X X.X in revenue the in South $XX.X million the territory KHK. million in territory in share million. Net from eight these in on in was other

year KHK Mepsevii product million. and quarter, reported same approximately the for Crysvita fourth revenue was the came year for the $XX.X the quarter was revenue $X.X was XX, yen, research XXXX Bayer, fourth ended we of million. for ended agreement the revenue million ended For the million XXXX, XXXX $XX In million regions. $X.X billion UXXXX December our in and fourth the XXXX of patient XXXX. in million X.X quarter for quarter $X.X December which of million of or year named was $X.X recognized $X.X international December from in top-line with fourth the XX, XX, sales

minimal forward. revenues going We these to be expect

including to and guidance and will this patients the growth quarters unique strength launch. therapy, We've providing reminder, metrics with Mepsevii a financial provided continuing not we on launch are and As experience commercial gain be other at Crysvita time. launch. plan of in provide We help of characterize granularity our early start forms prescribers only in reimbursed of to the level this to

for for year. the the of quarter the XXXX year, fourth $XXX including development expenses of for research million $XXX.X operating million Our and costs full for total $XX.X and million were and quarter the fourth million $XXX.X full

into increasing product we We continue R&D development expect cost our preclinical our advance studies. over as pivotal to candidates early from time

over our increase multiple support to as SG&A programs expect geographies. We in time we commercial

SG&A $XX.X million expect per fairly or loss and quarter basic with $X.XX versus remain the and for XXXX diluted. R&D Net the of million, of $X.XX diluted, split per basic was a fourth the net also for loss share $XX.X of fourth of to XXXX We quarter share consistent. or expense

Cash exchange amortization adjustments of cash losses remeasurement rates currency related will the continue includes in stock-based same share December net ended year equivalents and was $XX.X depreciation net be call of diluted. We company's Voucher of year sale Review basic of was sales ended investments million, net loss should per turn ended to with in This to cash research was non-cash in the $XXX.X from sheet. the the XXXX XXXX. the expense structure of years per Priority and portion PRV compared clinical the proceeds $XXX.X XXXX, with million, million, transactions. and to with non-cash $X.XX, approval. intercompany ended in in or Crysvita XXXX same million $XXX.X connection for in Mepsevii million us net back strategic Crysvita $X.X that on I the loss for compared $X.XX compensation a to the operations the our the and investments XX, portfolio. gain million $XXX.X for change year and to for foreign the with We $XX.X $XXX.X of share full to diluted translational loss in Emil. For in of for support and balance by Ultragenyx’s in for period believe of including and Mepsevii $XX.X $XXX significant charges reduced year making million XXXX the XX, tax the million The developing continue fluctuations and the period initial December million the basic launch or allow January and our and received used the resources sufficient of now XXXX cash, million

Emil Kakkis

Thank you, call progress open to Shalini. on up will questions. your and few the development our spend will I we then a minutes recent

Long-Chain For have the with UXXXX study, previously in received study. as Fatty long-term had down extension study going patient In study Phase prior UXXXX and released diseases, both been UXXXX duration showed annualized zero major the to patients zero on with clinical well rates. from as for median we've data treatment. who further efficacy The the previously annualized events to extension Acid our treated includes treated Oxidation clinical patients patients sustained rate who X XX not

in in patients study demonstrated In duration are of the addition, that extension in XX% at reduction of patients we in that to median Phase X encouraging saw XX% the median reconfirm extension population. and to patient we The fact the and the that and saw a set can UXXXX for similar reduction broader who annualized patients the they are previous beginning we've all replicating event rate UXXXX more was safety These XX in profile studies. seen studies naïve rate in across other the previously drugs study the annualized what separate study. diverse are

a three positive program top the All and no early study Ia, therapy gene approved demonstrated first in and hypoglycemia. treatment-related hypoglycemia, therapy, or reported. strength were look providing possible in control, sleep the through adverse seizures we Disease therapy the events for improvement which dose DTXXXX, forward from results severe hypoglycemia gene of is patients severe raw Typically required the tube response and the reported night clinically X/X or events of lowest adverse the reduced lowest to and need line There to increased program. encouraged by glucose for dose updates cornstarch without during clinical meaningful We're January. of the response to making and prevent patients Glycogen death. no time cohort night our it to on cause fasting. Type by Moving time serious even feeding to patients infusion-related Storage consistency can GSDIa demonstrated hypoglycemia reflected Two to Phase during cornstarch clinical the and

recently a conventional Turning Vitamin a we in phosphate that in head pediatric demonstrated to randomized active efficacy data and reached to oral XX-week comparison head believe D patients. superior Crysvita, with treatment

improvements oral care vitamin phosphate XX for these of been weeks D For the we has regimen what procedure records at similar greater in other observed studies. weeks was in showed Crysvita standard patients years. pediatric and healing compared last the growth [indiscernible] XX XX the XLH in profile of safety and The with that to significantly

our innovative our ensure will pediatric gained to label patients help have We support and XLH of receive with this therapy. all

that months to continue our drive milestones XXXX, to we progress. in upcoming Turning a of the will number important forward have

the quarterly front, to progress and reaching updating and on look you XLH MPSX on On we forward patients calls. commercial our more earnings

for Mepsevii throughout active year. Crysvita We ex-U.S. expected a both have also number filings of regulatory additional the and decisions reimbursement

expanded with For sponsored mid studies, track a of package XX company and to in compassionate XXXX. long-term showing review UXXXX for use more than extension UXXXX, access patients, and medical the comprehensive the submit efficacy Submission investigator-sponsored Phase study record function. we randomized controlled patients, study, safety of are effect NDA includes on cardiac a retrospective an a data FDA to including on our X data

GSDIa, cohort the long-term patients of dose in from further from for DTXXXX to treatment the XXXX data the first expect first we three patients. data For of mid cohort second our and

from mid-XXXX. and our we the year launched closing, XXXX for expect AAV third our DTXXXX, of for therapy was For gene out dose treatment cohort in two OTC breakthrough as built we deficiency, organizations products. In data a commercial us

will specifically data potential the Crysvita, have XLH. for eight months the For with to first launch therapy of success support translate can shown strength the tremendous of and clinical and commercial support patients our

with translational forward more Day in research forward on details our you program and coming City We and global translational deeper reach sharing research our an most in advanced stage. are XX, we've Analyst nearing and clinical York product our to weeks. New Analyst on look the commercial Day development expand – commercial continue more and April our advanced to line planned programs On also Looking clinical to detail to into reach provide we hold dive that program. the

to can you the Q&A now for questions. portion call. of the provide move Let's the you please Operator instructions

are ready the We begin questions. to

Operator

that. Sorry And our Instructions] of Cory the question about line comes Kasimov first from [Operator JPMorgan.

begin. may You

Carmen Augustine

Thanks Hi, this for Corey. for is on question. taking Carmen the

could kind of are press events regards a likely? them medical therapy meeting datasets year. expect whether as you to more the Could and give a release of with sense us So of cadence expected or later gene this at the a we

Emil Kakkis

programs. The data data towards midyear mid-year, expect is sooner we on will middle also XX-week GSDXa, have from in OTC things one hear than data cohort we and which the that. two which data second case the is expected also some second both might to so forward, is year come somewhere those GSDXa, of Well, cohort have coming would you of

Our meetingand expectation appropriate month for announce the until rather release a top line when have in A then a and greater at tendency we scientific been it has provide it would meetings. data detail press holding than we to

Carmen Augustine

in then additional Thanks. on you of potentially to the to Okay, toward expect to you And position program gene additional program? what soon. When a your were to see mentioned front clinic? prior needs done to work clinic follow-up could for we disease that preclinical characterization one moving be thanks. And therapy move more Wilson the

Emil Kakkis

order We Analyst through of parts on the to program expect scale in our treatment patients additional to you up data at Day where on of data patients regulators, the that need working we our the glimpse that present April to with disease manufacturing the some one the to clinical April XX and required of We're of – do and nonclinical on first be data. see with first and commercial some the do probably Wilson places XX. various give will you’ll work commercial

Carmen Augustine

you. Thank great. Okay,

Operator

mine question the begin. comes from may And Gena Barclays, next line our Wang of from of you

Gena Wang

Thank you for about questions related Crysvita taking my questions. launch. I have two

So revenue but seems million. Should continued, modest and in million based more calculation, my U.S. $XX.X the Europe EU U.S.? revenue be roughly was will the EU and was out, of on revenue last expect U.S. the doubling than flattening major from growth roughly a we revenue quarter. the driver $XX.X

Emil Kakkis

with. number lot the that but under think that European Well question. I at to the launch the launches and obviously quite And important In well. dragged same it's separate and of with, and of the have have time. it look answer launch, we deal authorities reimbursement as deal gone over is that's the a U.S. involves country you not of plans think, have a we KHK it's very care launches. I the and how But let to reimbursement health take remember Obviously time me think I particular of territories it’s period in European which a

cadence grow. we're about we and how U.S. those in And of reimbursement pretty with Europe pleased and and there may authorizations We're the the going. be progress So we're how both sequence some differences seeing. the bullish are

Gena Wang

And I Thank patients? identification patients percentage paediatric of follow-up patients have with through on based adult for quick a the tree Okay. you. family What Crysvita. on

Emil Kakkis

this certainly There a I clinic matter; pedigrees get quantified you think we number have the total, will family on identification. specific number cases pedigree add big families it's want are a don't have for a at a in don't where the to that but of don't the tree point. do a that treated. believe you factor strategy… did to I together of on word came number to I

Vlad Hogenhuis

and gives pedigree in see connect them the them patients and connect analysis physician to by future opportunity families an as forward think the to I be desire. their program help they particular if opportunity disease and for launching who for affected discuss have the with with patients represents might to and Crysvita that with a that appropriate, an which therapy those well, we pedigree is look see their to near whether

Emil Kakkis

so specific this time. think part the important pedigree family was way Yes, in how numbers launch. real But this you the in I of much point a be can't connection I at found overall moment give part will that at

Gena Wang

minority is the small fair through to of it percentages So say family pedigree?

Emil Kakkis

Well, distributed XXX lot so say doctors. of we doctors, firms, announced it’s I greater a clearly than right, we with would start you that to have XXX

don't it opposed point matters. this So as to it’s families of individuals. at think But less groups I

launch. a think are part launch. escalation in excellent succeeding really an beginning I the And the we're of think important family I of in connections

Gena Wang

Thank very you much.

Operator

from question from of next Leerink. SVB Joseph line our comes And the Schwartz

You may begin.

Unidentified Analyst

Dig This questions. our taking for thanks on and dialling for afternoon Joe. good in is Hey,

XXX Emil on question So a GSDIa. UXXXX and the one on other for

you you coverage then can before ongoing a us the on discussions. terms NDA the mid-XXXX, side? end discussions profile you the how filing question do sides want on guess any the the you can envision Phase there? of second you by drug are approval need What what X for under DTXXXX, of Phase how would of X the ultimate talk little before to patent file thoughts And XXX, question I the is what unmet accelerated UXXXX, about remind you And do on your both you filing but And the on is significant or bit briefly given alternatively, can FDA as mentioned they see design? in on EMA see can maybe First the Atlantic? approach for trial that

Vlad Hogenhuis

you. good, Very thank

working UXXXX So and we for the advice but are through continues. process regional and those complete scientific ongoing authorities we discussing authorities, the at European discussion have well have conclusion and this begun still with with no that point, we're

On the patents, patents do to regard issued cover the they U.S. we have

go think can it's on patents. through I on we various probably more probably to too and that follow-up the process complicated here

we in it’s But in that are I So product. patents the do that the seven there's some composition that where also drug patents Europe like do have XX-year U.S. years. remind orphan protect exclusivity, you

important So exclusivity, think, I is with that the XX-year protection comes piece Europe.

aspects the key we’re happening FDA their first Phase hypoglycemia supportive promptly we're But at clinically our going I and of with that on that other certainly of and can to as day. what's think get be in that And that during clinical in time not risk, hypoglycemia end threatens sufficient. And that FDA will is for do for of And XXX, pursue night. the to during patients that the think to We enough not a recognize that risk our our one it's that get us fair and X/X. expectation should would patients every expectation think, that to and these change. that look trial hypoglycemic a expectation Phase meaningful is safely meaningful to every be the Phase talk is metabolism. to we on fasting risk file as X So fundamental I go that. But X. really livers our clinically look. night and the a are is going clinical piece them for problem points reduction that planning We'll we at patients the about a confident we But can feel extend as

our that dose. three, get at treat would is would six to for is want patients still that just cohort the the right X/X, another plan dose, order that expectation make really but And file some would than Phase the Our do we in to more second we for only was approval.

X point, this is. the I would to what data we the our have at with forward get So FDA will to talking about Phase path soon expect

Unidentified Analyst

taking for thanks questions. our Great,

Operator

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

You begin. maybe

Chris Raymond

two questions. just Yes, thanks. Great,

guess, you think, Crysvita. piece – the was last could I you quarter, to mentioned I on First mix XX:XX adults.

little that example? this year? for if assessing that hypoglycemia. think was there Could expression will I color get study? then go there, I on time slight data one were January that but a you just to cornstarch an might think more one Thanks. GSDIa down you time, Can on said intake Was the there increase driven was that, update patient? from one the that that update think down there you they give respect saw, on program, was that mix on a trend that still also are I no an slide dramatic, question seeing with a same are to some dramatic, baseline on you there you at gene an that DTXXXX there you baseline that discontinuations. And go patient the from at you patient at saw, patient maybe And maybe but back then think, just all and I I And in give had update in have third the at the and could more discontinuations? I have all characteristic that, third then think

Emil Kakkis

XX:XX piece still really The continues the let's same It the hasn't as XX:XX with so start adults. before. changed. Okay,

so with comfortable And we’re that.

very about really haven't In few. discontinuation. the We clinical program, it's talked been

And so in we're far, shape. good

let just So that. me stop with

time kind patient GSDXa third to patient three the I X.X third the think, hours. and have one hours you data, X.X baseline a of higher column, has questions. of

So they they’re though if that they were needs already but even not patient, they affected. time the hypoglycemia approximately that was less about through was the night, The important thing have changed, their getting already almost cornstarch severely XX%. of decreasing were less

cornstarch allow fatty intake. to them And were so, – improving were there reduce sufficiently glucose their

how believe very GSDI, all now, three variation right do are responded change comfortable to with So some I and But were there severe responders. big all degree patients our we're is. of and therefore the they how three of

we're the go enzymes, release gene have not their going liver, do of I glucose-X-phosphatase of is these they glucose the their it to – think are expression, the extended a in GSD essentially to genotypes sense expression expressed of Their null. we Therefore, into be have gene regard with enzymes in body's ability are to don't – measuring no that really assay that want Now are directly to all. and at a expression patients tissue source. null for enzyme. for period whole for times because

And think assay, so enzyme than we and measuring specific their sense planned a more that measuring just that. in glucose haven't simply we anything

Chris Raymond

much. you very thank Great,

Operator

question from Raycroft line next our And comes the Maurice of from Jefferies.

You may begin.

Maurice Raycroft

thanks Hi, and for the taking my questions.

The is Crysvita. on first question

Walk in Six-Minute data, data. was showed benefit the shown the Test, which statistic XX-week you not on new the that we For noticed XX-week

wondering comments points so can of the label. any if And that its make you meaningfulness into on way if have data the just I'm the

Emil Kakkis

of the was that walking. an Phase have quite effect on X patients shown those we Well, impairment had even on substantial, in particularly study walking that

the because for is is walking an of this improved test. time it has hard effort again Therefore, trial So a an as getting the the is is although And into challenge with placebo-controlled trial, as total randomized controlled. the a verifying be it The once a can't have test component reason. might not effort that base walking it's on to simply genotype. label

patients phosphate better to efficacy another at no of so you and walking I much for the Crysvita standard I is ask. walking what think, can that being I look single question measure, data, every care. just There's the to reason you whether than certainly any line think we if However, would no and it's say And is any up on parameter other the down longer, one. about there it endpoint, given oral be

think And so, to is talk advice patients be label what to other hope would with I it's But think label because the doctors and XLH. think, get be doing agency not best give we I it their right is but our actually blinded, or with to harder be things. on important, label with the on the and the certainly to walking may open get

Maurice Raycroft

in a helpful. it. Crysvita And on approval countries as of first out? then for how That's still to expect XXXX? half track can the follow-up in you Got on play you then for in American to countries, starting And the provide launch any are granularity Latin you specific

Emil Kakkis

time press through the because the regulatory we're several a expect as exactly just the we're coming where approval. some haven't can get not that’s it partly out be any in we’ll right South of which beyond we us are to and approval play Argentina. well. patient time as quite approval, countries [indiscernible]and what we reimbursement confident now begin granularity, couple But in right that predicting we process well approvals those filingsin ahead through process to we launch will process able have but Well, work any for America we take in and at Crysvita provided XXXX reimbursement take out, work more the But least and figure have now, on going getting get when would to

Maurice Raycroft

Thank much. you it. Got very

Operator

line the Edward Robinson. And our of comes from Nash question SunTrust from next

begin. may Your

Fang-Ke Huang

for manufacturing are I our the for taking the another talk side call on manufacturing? transition briefly questions. capacity If with for have using correctly, cells. [indiscernible] to question you. can for Hi manufacturing have platform the to this I in or any that manufacturing is first HeLa Huang have the Edward. you maybe Do Fang-Ke therapy, DTXXXX just the both Then of currently is therapy on you plans DTXXXX Thank what a follow-up. gene cells? My you it Thank gene you

Emil Kakkis

Very good.

suspension we then but we XXX to our gene do HEKXXX manufacturing fully transfer developed. cell have team do currently, is for process, program. manufacturing used X. which We're as no to manufacturing a and that manufacturer would being we operations developed set format, cells of for for OTC contract Currently, us using have inherent have Phase our own. efforts an are in So capacity a the What contract or format, that we run we to up the full-scale and develop the allow

[indiscernible] cell run suspension transfection the is HEKXXX they a in not adhering strategy, reactors, suspension also the are product are litre but that in XXX to we program DTXXXX already the than four suspension For we process. so

which the that to then program. cells, plan we is to have something for the GSDXa to ability HeLa convert do We

go the where plan for cell for starting partner HeLa the is A our system. is program Hem the we For that’s to line And to with of Wilson suspension cell system it with. HeLa straight we’re Bayer

Fang-Ke Huang

Great, helpful. that may be

program. Second more question related to DXTXXX the is

the right just You Thank like when second can you can And could you. stuff saying provide think cohort derive mentioned some the dose? those. be magic you dose quantitative do it

Emil Kakkis

it's getting think right. important I question dose the an

risk hypoglycemia. through Our be expectation the we any want eight-hour them getting but an last is in without expect night full what of night, is to to beyond other words period,

taking cure. good, there criteria. their dose will that will So the to also barely just those to the specified that's would patients. comfortably a be want kind We want needs at be but a complete specific to I know metabolism and the thing. of the night effect, the very through to safely their essentially is we hypoglycemia are that basic be we with the wouldn't focus we not improvements that’s stop And a elements liver, that get important in patient at but XXs totality, But all in because through in consistent for to look not yet achieving on any public time haven't of we additional

Unidentified Analyst

you thank very it, Got much.

Operator

line our And question Chen next Bernstein. the of comes from Vincent from

begin. may You

Vincent Chen

couple questions. very thank much the quick ones. of A Great, you for taking

and of of LC-FAOD. a between sort advantage to choosing bit ones, [indiscernible] manufacturing, the later I'll on time in scalability of the curious manufacturing you little trade platforms, first opportunity that. more more different in Really offs transitions. be HeLa you how making the on those of cells. follow-up about different you to previous love in I'd bit how follow-up but there’s And gene then suspension talk the then would think a a about a hopefully and think sort little the about hear The I bit the therapy a involved hear UXXXX one, question on second to

Emil Kakkis

is transaction view to is that development. essentially a is clinic multiple and to rapid the our get initiate to HEKXXX Well, transaction, way suspension plasma

provides can see in think commercial – cell further. run you a have it call it a XXXX and you vaccine use and can AAV. to to HeLa a that’s consistent The litersproduction which viable strategy liters up very lot producing where lines a have we the create recover your I by is cells scale, way, X,XXX like approach scaling to grow then I and virus helper more to be line with producercell think larger long-term commercially

advantages cells structure HeLa is XXX. that So bit system widely takes cost get provides and longer to get XXX can and but approved. get a better the you there about used very with long maybe scalability and a I well it’s you systems, cell in faster setup, there think little to little run a The

We clinic our the we to ahead and for and these a both and long-term scale as armamentarium as in prompt using to also matching business. as to to we can manufacturing and create the are mixing necessary move manage costs of required therapy get gene

Vincent Chen

UXXXX then expect I how the LC-FAOD the think the identified as as U.S. these well would break of so, we what into or prevalence or about see. patients segment segments more LC-FAOD how and to X,XXX different each of LC-FAODs, sub-segments do and And down you less to X,XXX on see up-taken? sort opportunity of

Emil Kakkis

I you sub-segments, genotypes. There mean are… say you when So different assume the

Vincent Chen

Yes, different guess yes, different the I the – yes. LC-FAODs,

Emil Kakkis

studies a our the of CPT but that gotten the and LC-FAOD is in expect and LCHAD very been see CPT The the those have degree. our Yes. we'd number, are compassion [indiscernible]. small The to smaller five CPT any II clinical have within of patients less very lethal significant total. of and is also small have failure types a very VLCAD the for There been different are patients. are common some use common fraction six and heart fraction, and potent, genotypes of form most internal them The the type type considered that in type most pool, into

– sense. numerous the using sense other it think of or screening CPT the can the states a will is fraction at give information I either literature II on you ratios. the is definitely for total. that least from program VLCAD you gives is So This largest find TFP, this data a and you California public smaller in group LCHAD, a the

Vincent Chen

much. Great, thank you very

Operator

from next question our Arlinda Canaccord. from Lee line And you. comes of Thank

begin. may You

Arlinda Lee

wanted Hi, guys. up questions. my question. on also Thanks for follow to taking I the gene therapy

you're that profile bit what cohort the in like in to of you go So trial a would XXX. Thanks. Could of little go talked cohort And third to second a might looking you kind and might to registrational also on talk talk see about you XXX you'd the about next what forward like dose? decide what for to can cohort? be or what about a you lead a then steps gating the be

Emil Kakkis

Sure.

ornithine I So the three about a all hope we what is clinical we're out off see will drugs dose. XXX We acids, for you're certainly allows feel what response. come What restriction. of normalized of out two or three an looking asking achieving of off can is out adequate their to We three program. having three. and to as achieve dietary cure two by that potentially consider the We cure patients transcarbamylase that fatty mean three, which for

higher lower make have normality. may move to and Some OTC our to allow general next us consistency view dose. will next full this more to stage At on patients, ones we it which is harder lead level, to to shown with the effect III. that we us cure have reach two a the have to of are towards of But that get patients of a start effect Phase give proceed who we a treated point, hoping dose patients across the

Arlinda Lee

with you cohort can are additional dose information a and expanding discussions start ahead then what talk of you in dose Thanks. or about the you looking could gather selected? kinds regulatory go maybe And maybe things that for of you agencies or

Emil Kakkis

Yes.

some manage discussions our expectation promptly happen, would and time to to we not strategy if crisply the meeting been the from initiate timeline. while is and information cohort, talking By move – ahead. we wanted the of sit probably three see, next we would have take but to same other what the do is the in to with the to cohort shows So, the the of to always has patients patients. Arlinda, regulatory move And those treat have as for the data if dose. we the timeline to briskly our next authorities manage can. that as hit parallel another teeded But up process wait with those We've we what regulatory authorities we to we would our then we third that

Arlinda Lee

very much. you Great, thank

Operator

Suisse. of And Credit our from Martin line next comes from Auster question

begin. may You

Unidentified Analyst

Hey, Marty. This guys. Tiago is for

give enrolling or of Crysvita if broad now. curious right pursuing So patient is launch? just and you population patients color you. on profile us on [indiscernible] that gene spectrum, could is you're opportunity could Thank and segmentation the for how identification established actual that given the the and how outline could do the various how for you're if that and registries, therapy America, well those the how what considering GSDXa, think well you just the is the you impact in Latin dispersed addressable target standard product patients care, any patient there established And

Emil Kakkis

same is our it to elsewhere best in Latin America, what there. challenges in Crysvita believe because We is I'll are differences extremely we doing more the America of very a we're doing usually but and for about the of bit manage large up America. patients. patients will it to with more for Vlad areas in Kennedy outlet set frequency have is get on foreign how of what go is all America and Excellence, Centers you diagnosed. as reach course But to about is Latin general little of get which excelling product Latin place give at Care, course, that little become identifying primary in to do I'll coverage been other is Centers reach to to through doing of patients dominant. operate. may have start there's general, America we're which launches Latin a talk there specifically the Latin launch and Obviously, their local, In to team of Well, the the set I touch on XLH Latin Vlad Excellence then involved American XLH. let with,

Vlad Hogenhuis

Thanks, Emil.

and Edward who teams Colombia with strong liaisons, in three a America. has of centers, manager, and MSLs named with have of metabolic patient we has Thompson, a different Brazil in the experience So and a mixture supporting. Latin a to are of program are the request general UltraCare patients liaisons working we responding number bone Argentina, lot strong of He mapping in who now process that built

for and strong approvals We create also a got reimbursement share a have function up we and to task a different we’ve not and reimbursement soon we only updates built also you. that team submissions with regulatory Mepsevii for as them established have Crysvita, Brazil. well the we with will that's experienced in helps But on reimbursement strong approved as but are where

Operator

our from from and comes question Sachs. right, the next of Richter Goldman line All Salveen

may begin. You

Unidentified Analyst

the the for Thanks received one one on post doctors taking Andrea can My year is, Crysvita you've gating as you feedback Salveen. factors have thus in far this prescribing is U.S. speak This that on to and questions. first drug? up on Thanks. you any our you launch for seen the come

Emil Kakkis

patients not or – think doctors it is a positive? are had treatment. the because – feeling better a that more still they thought feedback we've doctors, could, number And they how bad, patients I have thought are got who the sick doctors I were. lot better that didn't were always better than on us doctors than they very think from they the patients appreciate discovering tell the actually treating of experienced patients

that treated what forward and needs stepping we're adults of as sure have a to really view So out they in of and happen. making habit the interested start aren’t the get really changing of not to doctors while for treating

else have what would any a we're you Vlad, you thoughts I if so, on do doing bit thought with – what results. might little with the And

Vlad Hogenhuis

observations supporting the reimbursed also to is endocrinologist – their available and I the clinical other the I patients endocrinologist pediatrics, assisting a forgetting long, point. making as they're the piece patients and by don't the that so addition Yes, feedback would sure of process process offices compiled, information appreciation apply think, that physician second complete, have them, all of wait the we're their of therapies. by hospitalization the getting how would in with to is too add

Unidentified Analyst

Great, and then second my question.

Vlad Hogenhuis

ahead. Go

Unidentified Analyst

launch then regards be any force have second and FAOD may program, in launch that preparations with would necessary? early already assuming was speak And Can and to sales on approval been you if XXXX. my additional question started

Emil Kakkis

the is Well, we're expectation UXXXX the point to I think that question. would our in is launching a with this is U.S., the out team that launch good and you What I of same the with Latin products Europe America.

and it the VII that fact, as In MPS are is same well. Mepsevii treating doctors

that we'd and think be to globally. I So efficiency, this able do with launching great synergy

substantial not important think expect our event. acid synergy and I the do oxidation other But will of we the programs team the change there I a well. fatty would goes metabolic but So need, in course between what launch need that assure is we the we to that

Unidentified Analyst

so Great, you thank much.

Operator

from you. next Thank And Citi. of Nochomovitz line our Yigal from comes question

may You begin.

Unidentified Analyst

sort Samantha the Thanks using work the of out three for our there X three of your or questions. sufficient and DTXXXX, could taking I is for you’re in relative dose this confidence X you wonder demonstrate that two preclinical gives anything Hi, earlier in to three of the on dose Cohorts out that Cohort Yigal. response call? for be X to you mentioned the in is

Emil Kakkis

no doses expression dose. better this get in Well, I is effects think non-clinical that's plateau the maximum think, there do I that model or we seen in have consistent, so very shown we higher at

So is that higher effect. doses showing will data get better there a

expect So from higher from we standpoint expression a would doses. to better get that we're comfortable nonclinical

Unidentified Analyst

then the assume for thoughts the one Is up are XLH, you to Crysvita. into oral new you’ve in some and into to market U.S.? XX,XXX gears into on of updated you've approaching vitamin families or the D who and estimate With you year thus the the just XLH additional is have maintaining be down, the far, it done gathered your would the And the patients either all with phosphate all insight switching identification Great. that launch? launch reasonable XLH

Emil Kakkis

And say that to prescriptions the various number probably you I found eight XXX getting in pretty what months also think from real on consistent is we based with maintaining that think number. We're still XX,XXX would number, seems I have happen. that channels I getting tells would everything think we

have of found triangulate seems change to at And So to kind that we point, toward don't reason a this number. we've everything it.

sticking that be Now will of and of definitely I are. you efforts XX,XXX mean they doctors – thoughts or who patients diagnosed the now, There would our those number. lot all other But finding right they're lots found be some tell to on doesn't readily whoever available. and we're adults, will are focused patients a

Unidentified Analyst

so Great, thank you much.

Operator

next Jeff comes from the question Stanley. from of our Morgan line Hung And

begin. may You

Jeff Hung

taking Thanks for questions. the

on For are remind you with think Crysvita, can what you biggest provide and an adults hurdles update the us that process? identifying

Emil Kakkis

managed being are pediatrics to doctor Well, frequently. different from going treatment the are with where the already patients

that As these seeing their seeing all to prescriber different adults doctors. have doctor, going prescribe, pain the they they're so basis five, on endocrinologists be may not person. nothing orthopedic their a and rheumatologist their ago. they not regular seeing because were management They're to seeing years They're six

really product. they're to so And or not this somewhere at able prescribe who's ready

tradition But doctors you patients. to of prescriber not ICD-XX been need be a get deep from and to we find to people those little [indiscernible], including for just we'll So find time been codes that adult it's in many That's by got really find stop into the we tools when think then secondary the new as get back. to efforts had our just bit that PDL population, to to have the it and continue take going get seen to to our you treating number those more with them them the a but years been specialties through matters. able the – and them

see Vlad in of the and patients, is addition, before, sure make some using In mentioned pedigree aunts what uncles the doctors had We're cases, for to our techniques. course, the on finding codes the other pediatric reasons the outreaching we're other and might which grandmothers. also, patients doing work of

the wherever We important right other triangulate part it’s feel we well. launch, going of patients find now, pedigree more like grandmothers – will be pediatric the but have who through system. an and the found we are that It's effort the one ways they of –

Jeff Hung

And Great. then remains what in TIO remind for you you can your and Crysvita on us collect? the and what to the need outstanding path data regulatory additional with discussions FDA might

Emil Kakkis

with like of biopsy be question, whether some Phase on a question study TIO the which not. right move. had them have and we're data is course, to X and TIOs We discussion more or data behaving data have continuing on rambling that think we that off have to the we with and required and that would XLH we initial We than is TIO think be show XLH the adequate file existing the discussion to able data,

continues discussion in that have the So we year. update should later an and

Jeff Hung

you. Thank

Operator

And Danielle you. the turn this no back time. call showing remarks. questions closing further I’d Thank I’m at to like for to

Danielle Keatley

This call soon. and concludes Thank a be joining our you us. will for today available replay

contact phone If additional for at Thank you any have us free you feel ir@ultragenyx.com. us. questions, by please joining to or

Operator

the program This conference. all today’s participating may Ladies in does and you conclude great a gentlemen, have for disconnect. and day. thank you Everyone,

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