RARE - Earnings call transcript Q3 2017 | Ultragenyx Pharmaceutical Inc.

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Operator

Good day, ladies and gentlemen, and welcome to the Third Quarter 2017 Financial Results and Corporate Update Conference Call.[Operator Instructions] As a reminder, this conference is being recorded. I would now like to introduce your host for today's conference, Danielle Keatley.

You may begin.

Danielle Keatley

Good afternoon, and welcome to the Ultragenyx Pharmaceutical Financial Results and Corporate Update Conference Call for the Third Quarter of 2017.

We have issued a press release detailing our financial results, which you can find on our website at ultragenyx.com. Director Relations. Keatley, Danielle am of Investor I With and Financial President; are Emil Officer. Executive Officer Kakkis, Chief and me today Shalini Sharp, Chief include but of as quarterly the I this Securities statements Litigation meaning remind identified to of Private to, the forward-looking XXXX report the Act will of including, of provisions would our the limited statements on forward-looking Harbor investors Safe in Reform within on July be all XX, with not for XXXX; like call our Form Form that XX-Q in report XX, will XX-Q which be quarter quarterly that subsequent filed website our filed the September ended will on on the SEC, filed the section. our Investors and available periodic XXXX, types reports soon; represent of are These call uncertainties, our of this risks as involve that including many views date only and control. the substantial and our forward-looking beyond statements could any from our Please materially those differ that in projected actual note statement. results forward-looking

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

Emil Kakkis

results. alfa two joining quarter XLH for or continued Good Shalini the progress quarter clinical by and of to vestronidase afternoon, will overview during us. milestones. make our MPS progress with then you everyone, We've rhGUS and X. discussing programs: thank third and for our And start regulatory I'll our recent of give for burosumab an

PDUFA or License status review XX, to XXXX. BLA, the Biologics burosumab For priority April granted and a set of FDA Application date U.S. our

internal FDA from that we the filing is we review review meeting. advisory timelines the in the that FDA, stating by to their proceeding process our for the indicating part the received the expectations. according currently the XX is and recently feedback an not holding with on received to is of We're and date agency committee As encouraged process, Day planning from review review communication the line

FDA and multiple a based children The paediatric for studies the available from and our accepted in adult reminder, and As priority adults. accepted both indications BLA review. application data is ongoing on granted our currently the

a includes the substantial indication, data young some lives. study rickets the readily olds showed from in XX-week These that was improvement to data showed paediatric studied we For at typically the XX-week showed X-year week September, the Phase in on ASBMR X previously patients on find from At could patients Phase XX-week early These the to visible than throughout X-year we children concept II the significant healing patients which also patients in have at their data and older study which also only to II patient more and would XLH of rickets a effect presented affect olds, study the bowing. severe XX. had younger of bowing, from both II and that XX-year in olds. treatment X x-rays at in improvement every XX weeks. that profound exciting in bone rickets Phase deformity The data the the significant supported baseline more X the

a levels, which an patients the the indication, study, also Moving to and be with in and of III among placebo-controlled considered improvements osteomalacia our in Phase in the significant showed clinical patients important indicated could healing adults burosumab. with pseudofractures adult fractures with XLH. BLA improvement increase XX-week In data for outcome the FDA, of phosphorus from adult includes serum discussion improvement treated

improvement The bone XX-week included in review. demonstrates the baseline patients X.X% and bone osteoid will they paired label six These osteomalacia quality compared provides underlying a now adult biopsies to fractures substantial to patients. open of submitted to the of and study had total have volume BLA from the We which biopsy with XLH, the be bone disease bone data information bone available bone/bone of important treatment substantial to severe treatment, six disease In X.X%. volume bowl representing study, osteomalacia. had XX% normal a severe decreases X% of X to in in XX of osteomalacia reduction. range patients, of XX% can The data a after the this osteoid on adult patients from mean lead support these mean the osteomalacia of severity present in in the the XX.X% underlying volume/bone [Ph] XLH observed mild

advance we Once discuss showed will on At weeks the completes, and indication. burosumab, XX increases patients ASBMR our study presented in phosphorus Phase completed II histomorphometric significant serum [Indiscernible] the in TIO. TIO data biopsy, significant September, osteomalacia, X mean we in of II our tumor-induced bone that statistically we of treatment markers, program forward Phase of the with in treated that patients Lastly, and of determine levels, improvements also continue who to X path in with turnover outcomes. the increases and in patient results indices improvements in burosumab, osteomalacia or bone showing

to away. away Turning regulatory FDA under rhGUS just patient are identification a XX, action ongoing Launch active and November with disease for efforts. weeks with date preparations the X well X, alfa and our continued MPS filings EMA to of education or vestronidase the PDUFA review

is approval. Our and to receive if in place we commercial team ready go

in half to vestronidase priority alfa, Europe review the anticipate if moving voucher We as is In for XXXX. review process is it first CHMP We of the a approved. expected. along expect receive a opinion

launch XXXX term In of active number the U.S. continue 'XX the with addition remainder and the catalysts. vestronidase of weeks, to alfa next to nearer be few potential in the of

additional and a from XX reviewed biopsy in PDUFA patients for expecting have For CHMP year. burosumab, our we in in the In for Europe, have being is XX-week quality XX, bone are also early opinion both of from the the end XXXX. data paediatric U.S. our study study BLA and bone we date adult around We'll April III from paediatric all adult results XXXX. burosumab patients the patients in Phase the and of

$XXX the the in deficiency into GlutX plan share by well. enrolment III the focused the shares a is Phase years end outstanding of stock also agreement Dimension When started which approximately or to all million And few place discussions cash, a as of Ultragenyx seven continue as year. agreement. Therapeutics. common young for $X we date per of small I'll ago, should acquire in Dimension UXXXX, merger advancing of proposed in company, take to movement the we to in proteins, definitive on of to early-stage while initially technology focused study a the and on to October, study, going with Dimension traditional III in spend Phase approval the entered minimize a capital and in syndrome promptly for regulators FAOD, patients manner. progress with we our we merger efficient of small acquisition Moving regarding we investment on minutes molecules platforms In a

treatment and advance With gene of gene in the into expand would financial also We first, the I'll after we've you we've this from us and satisfaction play a it will and closes right an our research programs commercialization therapy. successfully conditions now forward as this strategy officially expertise existing expertise important Therapies And now to treating of therapy to and, modalities on to Tuesday, which and more step expertise, company Arcturus to over early merger. results. base these new capabilities number moved X, with and of and and with The on Shalini involvement and set have next us Dimension could if would that mRNA time programs [Indiscernible] time, turn the call love look sharing be the our a is rapidly select particular who technology to broadened optimal details November for programs will the them disease the the for to infrastructure best rare Now -- the we depending the We disease as early-stage provide built the provide believe for overview clinical specific potentially deal advance take over that, for of right options commercialize commercial metabolic Dimension's of any combined this approved. technology. the and diseases. genetic near-term to

Shalini Sharp

and period third share, which $XXX I compared good of update, We and a basic issued briefly the first same was Total per Thank $XX.X the cash with for reflected of in for diluted, operations or of use XXXX. XXXX, $XX.X will per for months time diluted, afternoon, nine you, and everyone. press the Emil, This quarter a loss financial compared XXXX. included with million million that $XXX.X for million release $X.XX $X.XX million net share, today of and summarize. XXXX earlier basic or quarter third

first noncash increase gain cash included continue use million. the charges, stock-based amortization months the noncash Net and for foreign re-measurement remainder by for in million, to We of expect purchased the million other intercompany amortization $XX.X of year. compensation approximately of nine including $XX.X non-cash charges, to on premiums of investments, XXXX of depreciation operations in loss exchange offset $X.X a

time. recognized We third expect quarter vestronidase patient sales revenue to to increase over stock the in a In for we named continue expenses of compensation alfa. $XXX,XXX

programs this R&D XXXX. expenses translational accounting Phase were pre-clinical were advance period the costs. Costs the for programs multiple operating towards development research total of proportion during and of third quarter million million our Research the as for costs Our increasing III are greatest $XX.X for $XX.X also with clinic. programs our

ended horizon, quarter for discuss in balance. to with we due expectations equivalents both and the vestronidase increase activities potential cash XXXX, to and around product I expect U.S. launch $XXX to operating two on to the third We burosumab. on investments vestronidase During wanted the million a burosumab and minutes alfa take potentially revenue launches With and expenses few cash, alfa. the

date imminent. for know, is alfa vestronidase PDUFA you As the

However, the not from the it's product expect we obviously revenues late XXXX. in do significant year and in

on we reimbursed With we X burosumab be upon launch. as ultimately alfa, MPS put rare As not a patients disease, time patients an will gradual build are and found, reimbursed. is immediately and all that expect vestronidase extraordinarily both know therapy over

to payers conduct We new will in-house they these nine expect do months reviews for can other as clinical complete. of products, which three take to that therapies,

do penetration process expect on these in we programs. expect the While we build is gradual typical the of impact value essentially disease In launch products the significant demonstrated most revenue new, are has the which the over in period, of terms also all a as when will in that the see of treatments. our time early clinical rare burosumab, reflect ultimately case of patients revenue to true reimbursement for

may the We uptake expect than be that patients. in adults paediatric slower also in

adults study complications independence of negatively progressively that have burden illness shows quality debilitating functional that and of life. Our impact

with children treated adoption issues and being a with adult adults portion not will take However, associated therapy oral result, a with significant of today, I as reasons including a will who among KHK. for And compared for the of to D. the longer safety typically conventional potential of actively are being treated. review terms are patients variety it collaboration phosphate also vitamin

book or to manufacturing percentage After net In commercialization of in product and the take over KHK a higher the we that and goods with XX% development Ultragenyx costs. outlines share half associated as the the pay compensates Throughout America, the in Canada. we essentially agreement will U.S. profit a tiered period. of will Canada, will U.S. states required mechanism, Ultragenyx a supply Canada, Europe. be payments, for profit range. biologic, will to shown that cost net the the economics upfront post-launch, cost during and years the and majority a with KHK also of first the price years, supply America, our reflect pay royalty were agreement share in not same U.S. and for fixed territories: margin development Canada, and share supply periods, for XX-XX five for the reflects that these KHK typical price Our launch will a the KHK This paid any of burosumab fact intended during the three This sales royalty of nor Ultragenyx receive burosumab the is partner have price. instead the double-digit or our of the five and the In North revenues and milestone any sales. than mid-to-high period commercial is U.S. will efforts of costs first KHK. Latin for and burosumab

significant a will commercialize the America, pay state will while will of KHK, In Ultragenyx In revenue, burosumab on Turning burosumab to receive the commercialize Canada, a low net sales. Latin double-digit the of will Ultragenyx paying book and royalty while recognize price agreements XX% Ultragenyx Latin a American in KHK as of and same supply fixed single-digit Europe, U.S. KHK net percentage up that to net sales, royalty sales. and sales.

As XX the hold I'll a receive And the roughly by approved reminder, to agreement. that we value today. basis. in country over Europe, reimbursement it on to burosumab are products a for This one territories remarks of estimate can for to to back Overall, in of call the with my third subject that, XX-months country concludes the take Emil. that turn we

Emil Kakkis

Shalini. you, Thank

approval As on potential with the active alfa European we've for progress and and first front you've later and quarter our vestronidase filings heard, made tremendous U.S. this burosumab month. both regulatory this launch

Therapeutics clinical commercial We will and an and launch key are underway hold our acquisition additional Analyst on planning New December there. additional programs on pre-critical that Day the after the including experts closes. are team programs where We X, many and around information in forward York, management of to readiness, our Dimension you provide well. as We'll provide lead preparations our seeing detail look to

So the for call? portion instructions to Q&A the please your provide questions. Operator, move now can the let's you of

Operator

question and Cowen Eric [Operator Company. from Instructions] Schmidt Our is first from

line now is open. Your

Eric Schmidt

on Good have Emil, getting no Are process, the CHMP just whether be burosumab thanks taking that it And quite to afternoon sense the ahead is proceeding the AdCom discussions is they're Phase on filing been will highly I know the that the FDA date? congrats as would quite could for question, engaged well, early informed and and Thanks. Maybe based complete approval second question, my the FDA the progress. required. and that seems on you the be data? this there review PDUFA on then for II

Emil Kakkis

takes I the don't planning Eric. for that I by they're question. AdCom usually think to the and XX, it's execute determine unusual, whether that AdCom Sure, them it plan around normal think you inform Thank you so months an it's to Well for they Day because time. it,

but that. I working point point, application, we finish couldn't engaged and so necessarily I think are they wouldn't readily the early on at this that would this quickly that say expect

opinion On through review, a end. you it's are essentially and going year well where this worked we're last obviously year, are point, end reach toward time, well. the at the is but of some steps still you so will there's our -- that -- various the the European Day the it's process they passed the to still along And We've expectation tracking moving point point the then in the steps but by in we've XXX Europeans as of finish with well. the expecting the put far of

Eric Schmidt

you. Thank

Operator

comes our of And Bank from America. question Thank you. next Tazeen from Ahmad

line Your now open. is

Tazeen Ahmad

how to one thoughts Hi, wanted good relative lot where a time, that's presumably you from afternoon, a sense you development, taking thanks have indications are appropriate for take just other question. and for you but in your Obviously, Emil, maybe get patients. long terms actually the to commercialization do of to it pre-identifying of on going to be my to start get your will wanted rhGUS. things in smaller of get approved,

Emil Kakkis

person Well, we in indication. time projected of U.S., -- we the years. spent found much for a if in part, latter obviously time you been around have parts the America. very the every some identifying the single Europe, is that doing still have We've rare not a in on been XXX, South couple world. patients, as We've more It in really

been piece not we the hydrops that handling the diagnosed, hydrops MPS babies first to step of try obviously story life is market unknowns though use, have say compassionate X on diagnosed. we few an that's patients find year hydrops is to in run. might step do properly working in probably we're treating becomes year XX additional there's hydrops, people patients by of XX I getting have of who so born year process. that or the a under to are a the important that and We be in make and patients X with that perinatologists a with think age. there sure a continue are to long had We babies number of one it with the And predicted born would

course, on I Our possible. delay as as product. plan promptly get of would the to, expect launching out is a long launching the product not in

from have launch to labels and But enzyme before do and We distribute feel just and to rapid it. product with agreement the things get therapy. right is planning ready can that we go, it for do we're a to put final obviously have you what the FDA we packaging the out

Tazeen Ahmad

for you pricing an is appropriate should we think do then And range here? Shalini, that considering maybe what of be

Shalini Sharp

specific clarity later we disorders. at this is a for out the and haven't this what we on will product other point more provide Well, expect dollar be MPS said given but a at approved ERTs to priced date, that similarly amount we we have to that

rare Now the much we're similar this product other actually than a the to you in note range products. still that the other more ERTs should is but MPS pricing to committed

Tazeen Ahmad

Thank you.

Operator

Thank question JPMorgan. you. And Cory from our next Kasimov from comes

Your now open. line is

Unidentified Analyst

or bit about or just perhaps from this seen what on Shawn you comment other guys, a you for Spark about Bayer, your how could program, if different maybe little have like say Cory. with in this Maybe you BioMarin? we've VIII Factor some a might that partnership could example, [ph] product from little Hey bit be companies, for just talk similar

Emil Kakkis

Factor another really have I it going haven't it's way really Obviously, it's it's VIII in be and evaluating better, when ours. Yes. and obviously very right? Well, for completed been of tremendous all, complete They and knowledge experience ours, first VIII has But acquisition. Factor VIII to long a obviously, so the hemophilia. we No. in Factor is say, we space Bayer yet, acquisition program we will can't the time. after be

are and partnership closely that VIII program. in going that not and We work forward we'll in as knowledgeable experienced advancing Factor area on

Our the team, to on product encouraged Bayer around area position. think Dimension think of but closes in really them. opportunity responsibilities, strong producing The the work the if Bayer's acquiring acquisition put for And I good with and part producing we're are presence about the technology hemophilia us the product we're excellent. primarily in is we that why them

Unidentifed Analyst

that of vein of had hem walk just is Thanks. through And that happened kind in the potential read-through you story behind the maybe, and any keeping kind B maybe so program there Great. just of if hemophilia, briefly us can what kind of you and if may? that Dimension whatsoever there

Emil Kakkis

being that program, not so that obviously B the would and vectors. Factor not there B know inflammation the different. that them. happened in experts used was the vector VIII that we I used is not in what's have recombinant say the Well, program and programs being to that vector in novel we're we other read-through for -- would that, that our is hem in potentially -- what programs, what that the a to think program in other acquire, public we the be other factor the a but do vector that for reported And it's is is nor on vector may used no and related to more not particular is they hem

Unidentifed Analyst

Thank Operator Okay, again. you. great. Thanks

from next Our Leerink Schwartz Partners. Joseph from is question

now is line Your open.

Joseph Schwartz

questions. taking my Great. Thanks for

about give and burosumab. center structure? I of already date, are and in you you gradual for mapping these identified, treatment you Can any into activities many field accomplish what build have how pre-clinical you'd able and time like how us to insight patient been commercial expect your for to So, and said, size been over patients you thinking have know, a

Emil Kakkis

XXX the would think along the disease and for patients doctors places field programs, give XX identification. we various targeting on have and the disease that some frankly, we they to primarily, the seeing doing all that group on pediatrics the our so patient – territories, forth. sort our marketplace now, XX and important and proposed. something in just can that these a on about. really general have ICD-XX a centers it are UltraCare we numbers our in we I those right, to the or But patients patients are focus that of finding we work on developed. a United that, currently. patients we've are we right across in of sort adults focused divide places. across studied and our special yet team key out of to are Liaisons do, about But of rapidly think as as burden. And using state Well, those and carry in It's its that put we and and are really States what per of space that XLH team awareness That's team we diagnosis secondary we number of and of clinics. and expect in field has targets is identifying liaison both learning currently, based we the ICD-X released and the Ultragenyx’s haven't leader the the around get treating and information and past specialties team XX-person field only out The and tools targets which sizing A work number codes, basically looking of liaison country opinion around patients country have is patient main That's you the XX finding that's in get that we that eventually to

Joseph Schwartz

or or different I sophisticated what different bone the the of fractures, And differ then, the think does to and in -- centers serum peripheral that it, follow-on you see more for to be are most or do those market emphasized option that this right of [Indiscernible]. order out What segments segments helpful. stiffness convinced market? is how That's guess there, the a need between you phosphorus the and message pain be is

Emil Kakkis

them word, are things far people acronym, not use the a lot of centers XLH, that -- the one even a outside may around knowledge term, modern we're We're gaps I the the is science the vitamin more Well, disease some D through and first finding main other discovering resistant is of that rickets which use think, the or of basic terms. naming. the certain

of illness The doctors issues. around peds. give and understanding from of the a type burden FGFXX burden -- of patients, understanding phosphate the learn disease biggest part of to the and It's are to impact greater certainly adults knowledge so, them the there The of they terms life that. treatment. kind that the phosphate and out of knowledge also the of across goal are is the full of XLH their just have them in informing and understand about number is see and it breadth is They of vitamin problems And understand are a effect are an in that in trying the them, illness low across what of patient. explanations the that not the agent using may necessarily of about causes D the so we are

Unidentified Analyst

helpful. Very you. Thank

Operator

you. Thank

Our comes from next question Laura from Raymond James. Chico

is line open. Your now

Laura Chico

Hi. questions Thank been have All my you much. so answered.

Operator

Thank you.

from from question ISI. Steven next comes Our Evercore Breazzano

line is now open. Your

Steven Breazzano

clinically taking Deficiency program? you for about And in be meaningful? to threshold where the for could that for the Thanks bar something you're Hi. for you you question. my talk just expect see is looking expression maybe the OTC Maybe

Emil Kakkis

of ureagenesis at fraction and to what obviously looking is a we that's proposed measure of see the our in for ureagenesis look level program and normal we're normal you haven't being acquisition to there is program. the Well, achieve. The OTC finished

dose would I seeing just meaningful not optimize significant would cohorts to to is, would acquisition, of or quantity that that impact for determined, on will be think, needing be not of verify therapy goal we we our complete and the use the some been that try and closer events. them has the self-sufficient diversion fraction be bring ureagenesis now think ureagenesis well having and to different if right to we're normal to being ureagenesis clinically can

is, can't urea what will known give number idea, what and to an you you perfectly well now. I the I'm right it's whether not right percent an to ureagenesis sure but ability the restoring make degree. we're the So, give exact

as we're treatment normal be can a achieve So a acquisition Those level is but some time to able cohorts dose But some yet going take that to what that a to done know doing, we'll we're to to have through would ureagenesis. dose percent of significant at expect a of move reduction I is this get very not to and just its get cohorts. at program the little point, the beginning. through

Steven Breazzano

Thank you.

Operator

you. from our Gena Wang question next And Barclays. Thank comes from

Your is now line open.

Unidentified Analyst

[Indiscernible] for Thank for taking question. This is so you much Gena.

few question the XLH. in So, around adult maybe just burosumab to

about want follow-up adult what as to Just see do data. to you expectation XX-week the get biopsy there your the the III for Phase trial as expect and well bone

for this so what XLH? you. data the submit Thank all FDA well submission So you your as do to also EU and also what plan adult the for

Emil Kakkis

We placebo the to we believe they'll will review, from it data. is have that additional which in change an data. the adult the the healing we expecting expect body begin data would moving we're a the crossover necessarily to think in and not think, patient XX-week it to all would use same mild. expect file of update, reduction don't but in safety direction the to biopsies healing also to the we'd is in bone submitted do to will fractures. point, drug view. continued healing data, We We bone be the the be showed XX-week that Yes, at the which that this we osteomalacia And just described so X severe

expectation. expectation the XX-week in able review. data data to is don't quantity XX-week would data think peds point piece is that good the get so which they additional data would [ph] this will submitted were the and data So think, what that comfortable supportive the the But data that's data, our the now are eventually, is including get provide, additional that come. a of only we at they filing, what additional we deficiency biopsy be is We our is safety feel available. once think And probably data any of to the about from submitted the but the necessary we will talk it's have, and I in is we and with we there review FDA, after

Unidentified Analyst

it. Got

just maybe a So quick follow-up.

So do on just what's on the the use timeline? of expectation you like -- your expect,

Emil Kakkis

We there be questions, restarts. are of them, that the Well, give the and things, then stops. on assuming starts give it we we will there it The by on get answer They year we the no think you stops. process still to end is you opinion. further stops, track it has to track, back

now hit expectation our opinion the of we point that the right of is So should year. end

Unidentified Analyst

Okay. Thank much. so you

Operator

Thank you.

question from Raycroft from is next Jefferies. Our Maury

open. Your now line is

Maury Raycroft

Thank you question. for taking Hi. my

For Disorder if the in you any that III mutations study, GlutX where you types recruiting SLCXAX that the I'm you can are and any on into baseline other on are of enrollment based characteristics comment patients trihep Phase perspective the and protein comment can on? you Movement maybe and wondering levels,

Emil Kakkis

where with we're enrolling year, of out would and there put the that fairly more not enrolling. on we the next end Well, it's a before haven't exactly It's and that. in track expectation year. finish putting enrolling comfortable so early we're our But we by is well I’m

substantial is of that patients, fact, number events. not finding of trouble patients events. effort, There are both both new to qualify of We with enough patient significant an that a number few that yielding PDLs, movement we're MSLs patient a with our having effort that Europe, amount have patients U.S. movement is doing and and events, quite important disorder diagnosis, and in people is and have

So that part gone has well.

patient I in about genotypes the of the encouraged detail to this in we tell the enough know So about now, drive and continue study process more the more forward diagnosis protein to run. you going is how going don't long there. we're

on they're topic, of say, course, to phenotype from tell perhaps, be But GlutX to can't be small There's we have information it's -- make can, more relationship diagnostic going information at that any, variation to a might of different on literature really are, really don't individually amounts severe. lot need getting what but genotype genotypes, really the others. but that even clear there that provide on pretty enough or those less and it's let's the We genotype, patients like. patients you there's like look each patient you how of or

Maury Raycroft

as Got study? you you getting more -- in more goes, children whether on age the what about it. adults And as can are far the comment or

Emil Kakkis

is of don't of of actually both and There types enough detail. I children adults. a the No, patients, variety know

we're So other. seeing or I don't think any the one particular

Maury Raycroft

Got it.

Okay. Thank you very much.

Operator

Thank you.

next Our Genuity. from Lee Canaccord question from Arlinda comes

open. now is line Your

Arlinda Lee

for potentially while Hi, that the Thanks. And one my think patients patients as that questions. the maybe least possibility I theoretical Thanks treating at there like hydrops -- about on was in update that taking these were wondering well? treated? treated utero, I'm a ago. of a provide an then was Can you was one of there guys. and patients

Emil Kakkis

kids couple a from like There general, in age. that a on so have ones treated, the young been very have six are continuing age, We under U.S. of months in treatment

treatment. outcomes, are So probably growth that it's little And I their more profound we really involved take is young, how think update, And to should it. think been just skeletal on we other some a kid you don't I say take but of though, any from in so really physical -- the we to well a with, all change What can really better know continuing age, I've more get health-wise. resulted are their changed on going in get kids if treating is, those few months We and guys asking MPS in to specific a has the programs they we've I are under other I a and can way? question Arlinda, time much that can't comfortable probably feel progress. have -- doing, future information we how

these and an years lives important their change from that old of really physically harmed. some way a are very age early treat So it's we are kids think who when are taking in bodies can't XX patients and to you you

to there is program topic. currently and report. they Now the doing at a to have anything clinical scientists will leave a is report treatment that collaborating a the that are in update at utero trial. that But results. we're on research the planning their looking program in That who we looking utero, with We're don't an not

Arlinda Lee

well. what could your for line that? And program intention be voucher to this Okay. And then a would happen? time sell that Is eligible as

Emil Kakkis

let I that. Shalini answer

Shalini Sharp

Yes.

because, review a voucher pediatric vestronidase Arlinda, priority a we for to X rare for do product disorder. receive expect obviously, for So MPS this alfa

products anyway receive most because review intention to expected be voucher. our of divest all all our to are would Our We priority approval. upon or of -- products review a priority the receive should it

the we following be process we could simultaneously that PRV receive and sale time. the anytime do if with So approved, should then get that

Arlinda Lee

those burosumab? force maybe And launch think working that liaisons any we that about? years, then to And are after one-time medical should what on there great. burosumab, on costs sales the five we’re or happens then Okay,

Emil Kakkis

a force -- can burden work. that On the continue level. now at run think patient [ph] big may do that have diagnosis costs, I not the particular. and continue not anything we in issue patients helping that's indefinitely that map diagnosis contract, of out That long make it's the might We don't disease do one-time awareness us all patient

the Sorry, second question was?

Arlinda Lee

the of were years The for were that guys you sales first forces the that collaboration. -- five using

Emil Kakkis

would with What we do them?

Arlinda Lee

Yes.

Emil Kakkis

We the market other we'll market. the use have to continue to that on many products them

and get Our have But think intelligent as years help able the products a handle getting in We'll time able partner, the in Kirin, that two to and there patients. the will that be will that. going, point start to be that over, transition to is be market smooth. afterward. the a for and that's way expectation we products on in other people our with managing market will up. that our which are the They make make we'll it. way And to putting we'll expectation work to kind it set we transition transition with of field help at well have flow on might effective in is other partner the our then manage But before

Arlinda Lee

Thanks. great. Okay,

Operator

you. Thank

is Our JMP with Securities. Liisa Bayko next from question

is line open. now Your

Unidentified Analyst

Hi, is there. Liisa. for Amy This

sales expectation us driving maybe quarter? can And it? what's What's on you really -- Thank your going sales the forward? named named for the the patient remind So, you. just for this stream revenue

Emil Kakkis

handle let I Shalini that.

Shalini Sharp

patient who paid from approved, for country to not have So, product be is which are vary for, country. we programs, can it the but Europe, that circumstances, under in under patients these patients, treatment which do means on certain yet named

this. patient patient But on We small the hard we remain alfa have to recognize do anticipate. vestronidase a the negligible, it's a historically and revenue don't is we Europe sales very We to to significant revenue collections really number had for because because revenue expect timing expect revenues named basis. of named some of in previous patients, and

Unidentified Analyst

Great. Thank you.

Operator

you. Thank

is Morgan Harrison with Stanley. question from Our next Matthew

is line Your open. now

Matthew Harrison

Thanks the have Good taking Great. two. afternoon. for questions. I

to related America struggling So or heard can your on some I guess the peers then from value related Latin that they've of question pipeline. how you first been is, guess of a Can you to we've one XLH? just little bit. you not in the changing comment, second the orphan see I I is comment changing And the territory that have just [ph]

different thinking new You've should obviously pipeline a assets. of allocation new handful assets now? your among prioritization about about are -- add capital the we added you assets a to and think or of about of handful How how Thanks. right

Emil Kakkis

Okay.

in always local lumpy there's distributions sort challenges been -- and payments, America the a Latin of economies. in history there's been So, of and

going be number forward. that will products increase that's issue become the more always be disease the so may rare in with It of challenging. And an

Eduardo way. We in manage substantially would there kind fixing primarily smartly, focus head, dysmorphology features strong handle that well-versed result -- thing disability work. need child's that very to results I countries has and that on products an We think to health. are are process extremely the in a the from and We improved being to is it have lot think and Thompson, we its that intelligently. a work our think the the elegant the important reimburse Latin down going and bone-related and and very of sales the able in is We're done American of it

we it's that America kind target well think of the would the probably So populations. in Latin treatment do from

we now XLH. they countries about on affected right while a are appreciates for So, with pressures though, in how of be access we to and smart increasing helping have, the the commercialization, way American Latin that appreciate, patients that health have we and their challenges handle really there press

products had have Factor -- which we and assets new if complete are tractability execute. the acquisition Phase to really with acquire really in inborn try the -- are and for that after Dimension, one do, We of are were product overall Two in programs and that is and we we going OTC, our at other we to stage. pipeline. them look be at to products the in stopped. a have are GSD-I kind Now, inborn X as our would gap with to error on area. -- products They error early ours manage we're their of the ability do for II IND we'll products prioritization, manage the our partnered Phase carefully actually of Because because three And phase I, program Bayer. products flow

three are that into nicely So of fit -- kind programs gap. there

in out. the to programs really year early other year two Our are pipeline another

prioritization R&D of the portfolio, is like will the be the think of pre-clinical but I the So XX% investment. part that easy look actually to manage. We'll at more

to think together out. So another fits or well XX% I very without push in the having clinic thing one

about be we're what how Day little and haven't pipeline ahead. at where it, an we a and we give area in maybe Dimension we're in anymore. is give clinical us So could think talk else R&D errors strong in acquisition the on said good But is there would you the early-stage in, inborn a how We'll that better that area. progressed the managing some picture work

So I do think well. it fits

Matthew Harrison

you. helpful. Its Thank Okay.

Operator

remarks. to am further to closing like would Thank you. I Danielle no back Keatley I questions. time, this over for now turn call the showing At

Danielle Keatley

a replay Thank you. our available be and This shortly. will concludes call

additional at contact ir@ultragenyx.com. or If joining for please you us Thanks questions, have XXX-XXX-XXXX us.

Operator

program. Ladies concludes and thank in conference. your This the participation gentlemen, today's you for

all now You disconnect. may

Ultragenyx Pharmaceutical (RARE) 5 Nov 172017 Q3 Earnings call transcript