Ultragenyx Pharmaceutical (RARE) 7 May 192019 Q1 Earnings call transcript

Company Profile

Participants

Danielle Keatley	Senior Director, IR and Corporate Communications
Emil Kakkis	CEO and President
Vlad Hogenhuis	COO
Shalini Sharp	CFO
Camille Bedrosian	CMO
Gena Wang	Barclays
Yaron Werber	Cowen
Fang-Ke Huang	SunTrust Robinson Humphrey
Joseph Schwartz	SVB Leerink
Edwin Zhang	Stifel, Nicolaus & Company
Andrea Tan	Goldman Sachs
Yigal Nochomovitz	Citigroup
Vincent Chen	Bernstein
Arlinda Lee	Canaccord

Call transcript

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Operator

Good afternoon. My name is Jerome, and I will be your conference operator today. At this time, I would like to welcome everyone to the Ultragenyx First Quarter 2019 Financial Results and Corporate Update. All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question-and-answer session. you. Thank Instructions] [Operator Ms. may conference. you begin Keatley, your Danielle

Danielle Keatley

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

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

Emil Kakkis

us. the will and Good providing turning the to you afternoon, will Vlad, call before some for XXXX. start the by over introductory first brief I commercial performance remarks who everyone today of discuss quarter in thank joining

Next, at Shalini will these latest looking on look the XXXX, disorders. quarter and a also prepared to clinical by our of organization third commercial month. will progress product, In programs, depth of discuss to remarks treatment the We're on Analyst we've our territories oxidation of fatty first milestones last a with update many throughout greater acid Crysvita later major across the our for you summer new were first end the UXXXX, which some results. forward significant long-chain year. financial world. Mepsevii this application drug launches our momentum three in the quarter built submitting Investor this the established of highlighted Day come and back then close in during I'll to also Camille sharing forward for

the DTXXXX, our we platform, disease treatment program, concept for For approved gene therapy Xa. in clinical saw the glycogen storage of second

Phase for be the studies therapy as data This year programs. programs and that we will X late-stage allow manufacturing facility our capital clinical in commercialization will Phase therapy advance will will gene plan build clinical enable and We to GMP-compliant our and pivotal our two gene will us a and scale other successfully gene therapy development fully programs that initiate therapy gene gene therapy X/X also us a XXXX. also our that from raised of anticipate support to

glycogen reach storage Wilson programs expected as clinic we are the for gene for to Disease, in program our X INDs XXXX. preclinical program dual-triggered Type therapy We've three preparing discussed we is disease also Analyst for submit lead the and deficiency program As at year. identified creatine next Day, the program to transporter mRNA

through Now quarter first the I’ll through turn of year. the call over performance the to walk to the Vlad you commercial

Vlad Hogenhuis

and afternoon, everyone. you, Thank good Emil,

quarter build on the Crysvita the to the XXXX, in we've For successful of continued of first States. launch United

to continue from XLH and prescribers patients a has We with feedback meaningful patient's lives. that impact on receive Crysvita

patients Over quarter approximately By transitioned commercial the to the have start we've therapy. of launching clinical treating the received XXXX, of completed from physicians. last nearly XX fourth end product, months since the X,XXX all forms filed successfully

XX% a forward, patients Approximately physicians we naïve Crysvita. reimbursed split new to Crysvita not continues pediatric therapy. have of expect and adult patients will be on unique prescribed Going XX% be nearly prior commercial There to all treatment. XXX

more As this proportionately writing than base grown prescription one than for X/X patients. has physicians with of the prescription has grown, more multiple number of writing prescribers

specific as will year, the this Earlier prescribers As more experience total buy prescribers. and patients. we has treatment the simplified payers. and believe of Medicaid of to XX% XX continue number this government payer first-time well growth as for as reflects and was of number change process, payers prescriptions process gain other and received private Medicare with a bill XX% see March the the providers J-Code, reimbursement mix writing multiple which the The we in and

approximately payers U.S. for As lives. we policies stated million published covering last XXX have quarter, Crysvita,

of across across pleased commercial to a formal within case-by-case reimbursed U.S. full of coverage of launching basis, April without generate the on We're XXXX. continues types approving XXX payer early metrics. lives therapy launch U.S. approximately are coverage broad since The on has in patients payers ensuring all quarter-on-quarter the to policy these Additional momentum Crysvita all nearly growth led meaningful

to the reduce reimbursed takes genetic more well initiatives these on For time the believe get patients confirmed potentially and members in with as access amount We family genetic will confirmatory continue recently have of who We out XLH. to the diagnosis it Crysvita U.S. identify help to therapy identify of counseling XXXX, testing, reach implemented as a we'll the remaining analysis pedigree independent easy commercial programs. months build and nine of

pleased American XLH that Now, of Brazil for March. within the approval in treatment the adults received we're Crysvita in in children and Latin market,

eager authorities year this and responding and also requests. in from patient markets will multiple to next. named decisions treatments Colombia, Crysvita physician We Argentina, Chilean, health to these look We're reimburse to forward in disease and Brazil the regulatory rare with key continue and launch Mexican Colombian

to with Over we'll Portuguese meantime, provide in Shalini this and in the difference Chile Mepsevii, few call working government We're pleased United to requests. months, regulatory named this patients forward Briefly a but we'll geographic make Colombia Brazil. discussions year the in continue and around take update. coming who continue for the with a the the and and broad EU these markets major for in approved of Spanish authorities in French, We're that, we additional ensure patients. Crysvita authorities and to next. MPS decisions is product States, German, look turning including with have to to Mexico, respond patient regions world, currently decisions can Mepsevii health years, to access to financial Reimbursement regions VII all to with the will world. continues encouraged turn the I'll With these three

Shalini Sharp

Kyowa for included revenue or $XX.X I issued everyone. will and Europe are portion earlier and briefly Total shared the $XX.X ending Kirin, partner Thank was revenue of Crysvita a three that XXXX the Latin today totaled America, afternoon, worldwide release good million. which XX, financial which months Topline March North a approximately represents America, our press We update, you, summarize. with Hakko total million. across a This KHK. of sales Vlad,

territory. revenue in For Ultragenyx collaboration royalty includes $X.X the named in million. $X.X revenue for ended recognized million of patient quarter the share $XX.X This revenue was first Net profit Mepsevii million. million $XX.X revenue in Crysvita in European the UXXXX product March of million. territory other regions for and in totaled XX, quarter XXXX, sales revenue total product and the was $X.X Crysvita XXXX U.S. $X.X million

with million We our revenue also $X.X agreement recognized Bayer. from in research

is period in $X.X million the $XXX $XX.X of who manufacturing amortization to first rates the use million, net for was XXXX in of equity share the gene the $XX.X $XXX expect XXXX. million As including future. with or sales facility agreement diluted vouchers. and with and stock-based income We of exchange offering turn the proceeds in related ended quarter the quarter non-cash the and This depreciation the used for expectation compensation fluctuations reducing Camille, $X.XX on fluctuations stated intend income million per compensation to $XX.X exchange which now We to for to cost gain from which significant $X.X this intercompany which change and amortization update to ended tax therapy XXXX continue depreciation cash $XX.X $XX.X to per This forward. review losses, first Net XXXX. million closed basic in underwritten first XXXX clinical priority in the the for like -- compared cash, on expense This will build the charges in $X.XX million share includes Recall million call our with in own rates. $X.X used charges, programs or the of March and cash transactions. we operations and sheet. structure approximately in the of to of program. the therapy our per operations public previously, investments we on included million same in our company's first from share in have in includes adjustments net remeasurement foreign equivalents gene significant with first stock-based the $XX.X over non-cash non-cash XXXX. the quarter of for Cash to connection quarter speed provide was proceeds balance XXXX an execute of ended to the in diluted minimal a going of includes currency million quarter $XXX.X million would can from $X.XX of compared the increasing raised GMP portion net expense I XX, adjustments an million we the the $X.X million, loss be of of and March of for of net and due quarter to including with million, revenues and

Camille Bedrosian

LC-FAOD. Thank into of for oxidation disorder, genetic in and afternoon. everyone group good the Starting energy. convert a too I unable acids long-chain which wish a UXXXX fatty is is disorders you, LC-FAOD fatty long-chain body Shalini or to with acid

high with current new we With the that disease. for the disorders patients and treatments and MCT or severity treatment these mortality newborn needed are triglyceride rate devastating with urgently despite medium-chain this of believe oil, screening

in on new track FDA drug mid-XXXX. are UXXXX for We the to to application submit a

criteria relevant regulatory well communication frequent and eligibility disease with met. the this as UXXXX. quarter, earlier enables FDA front, are as for FDA. for granted the for fast-track Review, designation designations rare also early allows pediatric Fast-track a Priority the On if U.S. designation It

from submitting We expect days our back designation NDA. after XX Priority to hear Review FDA on

program that our urea to Moving metabolize an or DTXXXX, gene ammonia. deficiency body's ability cycle OTC therapy limits the transcarbamylase deficiency, disorder to X-linked deficiency. for is OTC ornithine

provided As you deteriorate quickly of into is metabolic very are other ammonia High Society Meeting to neurotoxin. coma & and blood infection. Therapy the their deficits by neurological of Day. deficiency the aware, with Analyst Patients build can first OTC induced recent ammonia and ammonia Phase and American up at of an in We often in from excessive XXXX and amounts additional full data patients crisis, potent results the a Annual toxicities, leading cohorts two X/X hospitalizations, our study in Gene even Cell death.

two experience the first responders, to these for two metabolic could XX to one enzyme patients from term decreased of of time typically been patients patients program As points, recent in and into patients cohort. Weinstein, a has a improvement the center include which to lowest each for a circulation. Analyst responders the this of defined reported that at first additional tolerate from stay in experiencing can and longer Day, without glucose during cornstarch an to had and our least therapy our out ureagenesis medications these At a protect on This of observations university, data the dose responses, the resulting further such as lab-documented patient to time these that scavenger type in of severe increase their GSDIa he ammonia ammonia is the metabolic further to liver to recent decreased fasting, cohort would a in with weight mix now possible observed at able Xa crisis All consumption in as diet is even his and experiencing of study. so patients case consumption DTXXXX OTC have rates for the compared a extension. in of their lead data release more inability alpha X/X have have David the We hypoglycemia cohorts, This time Professor XX% OTC And the are gene shared provided have glucose-X-phosphatase to and to been without recently protein. one Pediatrics treated discontinuation or We maintained a in patients of followed Disease University showing gene as scavenger ureagenesis, had cure levels weeks. of and all ammonia that initiate XX and XX also durable reminder, are hospital crisis. also medications. At weeks to GSDIa rises Furthermore, the now needing dose therapy. defective Because or and hypoglycemia additional We was without their their stress in by allow Dr. so continue deficiency during infection. an at the the of have substantially, two moving is their the hyper first potentially have liberalized they the and as caused during far baseline. significant the increase setting. normalized we further The XX%, of infection. three view cornstarch influenza, data a followed investigator, by Connecticut Glycogen program M&A by His shrinking from the DTXXXX, suggest and Storage GSDIa. this validation clinical and for with are livers rate improved metabolism, loss. glucose three the the controlled Director fasting patient deficiency gene Phase metabolic

metabolic of with not GSDIa. normal The Physically, was one region be able observed glucose to back transgene the had the infection. the support from gastrointestinal fact the control levels to despite that major a this was hypoglycemia will stresses turn with for maintain suggests glucose call patient's addition, and is would hypoglycemic normal expression challenge its gastroenteritis that a recently Emil. I In patients the able now despite to infection. patients the needs

Emil Kakkis

Thank our you, can coming I'll a to question-and-answer of progress. And the discussing spend then we that Camille. minutes important to a continue milestones move number drive months the period. few in will

prescribe adults continue in calls. to the to both will and we'll expect and children breadth we Crysvita, For you XLH and depth our update increase, to treating on earnings progress our subsequent

Canada growing are in are also our we and decision and pursuing Latin year. We expect recent commercial in and other this countries Brazil, America with filings to regulatory reach approvals the

the of controlled study compassionate of function. showing impact the on XX data long-term the studies, efficacy drug mid-XXXX. the the sponsor we're than medical a and Submission submit expanded For include access new our data will and for more in UXXXX, randomized comprehensive record track to application on safety review FDA in of package UXXXX retrospective a cardiac X use investigator Phase patients, extension study, the company-sponsored patients, including the

Moving DTXXXX the cohort of expect OTC to for deficiency, for mid-XXXX. the third data we therapy from programs the treatment dose our in gene

For GSDIa, in DTXXXX also the from three data for anticipate second dose we in the treatment patients mid-XXXX. of cohort

dose, optimum of have data each these both data a before the Phase the path dose enrolling additional study. suggest, we and then at be to If we reached we'll the for patients For program. X three evaluate forward determine moving programs, would

to and facility therapy support gene GMP bidding We of platform. across manufacturing efforts also fully that will construction look site the the forward finalizing development our selection scale our

the strength of our we're and of and model actually to chelator from pipeline Day Analyst preclinical for the effective option for copper more Finally, or therapy that preclinical in shared much the by the Disease, we cytoplasm a preclinical pump data AAV of research illustrate at treatment be could pipeline, therapies. UXXXX, current encouraged the Wilson the appears

down into Type Disease be liver's and study of treatment it that suggests dose for a to UXXXX, toxicology completed mRNA Glycogen program release successfully to finding glucose have the For range into the break way we glycogen meaningful or circulations. ability Storage and reduce non-clinical could III,

creatine early continue UXXXX, concept approved top to effectively drug's data treatment relentlessly will the of cross and and for the accumulation dual-trigger the of remainder the fifth of for to in and year, can to the what the this For creatine celebrated our the shown choice Earlier the this founding molecule candidates. brain. of our has ninth anniversary blood-brain the we done We ability and to still deliver Stepping us to deficiency, of enable Through rapid to program transporter submit clinic our I'm the studies have the brain find in nonclinical to from studies patches IPO. barrier XXXX creatine we company's company need this finalize to necessary suggesting exact INDs the conduct proud XXXX, three XXXX. back, very that of patients. anniversary solutions preclinical assemble bring

on also or This rare products nine successful treatments has platform no eight in world have with the limited received and clinic. and established options. two XX who genetic more for We simultaneously two we've and still gene last a bring track diseases file. geographic we treatment clinical them new the Over other company rare therapies conducted the helped to robust will worked disease therapy XX manufacturing in that with record us keep and different two patients a launched We've become products identifying in have years, approvals studies in and diseases. three different regions major programs clinical diseases

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

Operator

Ms. from first line comes Your Wang Gena Barclays. of from question Instructions] [Operator the

now open. Your line is

Gena Wang

questions. my have I just questions. two very you for Thank taking much

revenue. The first is one regarding the

into wondering profit roughly follow-up the year? revenue on exactly over quarter And million, about I when $XX.X take into it's; quarter? a consideration our $XX calculation rest growth Just seasonality million question. U.S. we representing the that should million, for any of So, in the have XX% growth this sharing of US$XX trajectory base A, for last think we the

Emil Kakkis

hard. Gena, partnership can because I the parts. appreciate Thanks, numbers think moving of complicated a We revenue the are be and that all the little bit

I that with things, early can numbers think anything. this in too point, comfortable a too down kinds regions, be bit to seasonality, that from progressing. don't we're can know the I just we look we at things those so regional are for there's -- I end seasonality. would there I think early really We're reason the don't the the gear, in of as of up to I launch or that. are of little any more and if be seeing give natural some know answer whether progression to comfortable anything At feel specific And I how launch. see think than

Gena Wang

regarding is And OTC great. quick follow-up Okay, the GSDIa and a program.

confirm, -- Just want HEKXXX manufacturing to commercial production. commercial already cell and suspension in using cells lines

future Just wondering any need for studies?

Emil Kakkis

an are and scale a HEKXXX version that Both be continue study important in is in make production GSDI do It's is There commercial GSDIa The and in be We currently. to quality. programs of XXX-liter scale will think XXXxX, not that GSDI. modest improvements OTC will program question. we required. for may

a to will move GSDIa. We platform eventually for HeLa

far, this some done than in and we but the a proceed the well, given future -- it at program change as promptly to at head the now HeLa the think we important do platform success and we rather introducing via point have do an ability moment. will We a to commercial so to it haven't of

a giving cells HEKXXX now suspension is original to we're We'll the a liter. approval. originally which XXX run GSDI, is moving are they are processes. OTC the but X/X size very with to also what cells similar type with be OTC, system doing For multiple process staying be a XXX which Those again, around containers XXX-liter adherent, the very for that's heading Phase system, are similar to will to running but

Gena Wang

very helpful. Thank you. That’s

Operator

the question in next with Kasimov comes of Your line Cory JPMorgan.

now is open. line Your

Unidentified Analyst

the How for online? on we one about questions. ramp in should [ph] your my be XX Matthew months Cory thinking cash burn. taking the thanks next My on is and This and with guys. R&D is for gene Hi, over it coming manufacturing first

Emil Kakkis

There color Sharp are let a lot of provide parts, Yes. that some things, Shalini on I'll Shalini obviously, Emil. Thank you, one. but moving

different expect time. GMP to side. expenses revenue do we time in we on to have begin the of program. dynamics more two manufacturing the Those for looking same increase modest also more at So, therapy the for XXXX We also in R&D but lines facility do offsetting to the gene are and our though and moving are those expect in expenses expect and increases to SG&A build-out directions, expense different over are that the the

Unidentified Analyst

to position for to you'll a no-go it. in to the update, that Got expect make reasonable then just data have DTXXXX is decision? And midyear that go a or enough be

Emil Kakkis

be to able make to if expect X to a decision ready Phase we're We move not. to or

only cohort’s what so three another We and information the that effective suggest patients. remember proceed three I’d think enough us to would which is that ahead little patients, We that patients, to us X another three response an I point therapy. give do certainly you a showed would was Cohort deal more treat with. we to felt would out that

three some we if clear kind still for, would two Phase you of it's responders out we're So, would less of believe from when or we curers, in that life-changing had position what way What have of results, in with three something we a you altering, terms away because their and And that working or the of be forward we're not is that X. hard life we out to of walk that response. proceed would have find results three suggest a looking say ahead get to responders is we I proceed outcome.

to based consistency dose in a will So, the lot seen we've therapy I that of on, actually need but we things what be confident increasingly other towards there do feel many work on may programs. increasing

Unidentified Analyst

questions. Got taking it. for my Thanks

Operator

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

open. Your line now is

Unidentified Analyst

taking the question. Eli Thanks for is This for Chris. [Indiscernible] Hi. on

launch. So Crysvita one the first on

this XXX mentioned physicians point. at prescribing You having

then about that and on have year a better comes of you're is trial XXXX, in prescribing? versus depth you're could quick do than importance design? number now you remind think about a you generating one about that this XXXX as help just I include data one thoughts us, indicated growth Phase us personal you talk and could the setting could the relative of And just that And DTXXXX, second just initial you think your previously play X breadth do into about a us expectations increasing you on guess young X, or launch? in into XX? how as patients targeting about thinking of you that just pediatric on your remind how into Since prescribers So I drive number as So think that. prescriber Phase of out Thanks.

Emil Kakkis

Good.

So, that I've for extraordinary soon number I launch involved prescribers into disease with XXX been of think is rare any launch. the this prescribers

many the U.S. work in had we think that never products XXX and I

out the a That prescribers doctor talk about number. and still prescribing. where patient of the there's each on of question there, bit more with impressed the that versus are maybe prescribers is I'll said, little depth we So are let breadth Vlad -- each we

Vlad Hogenhuis

Emil. you, Thank

the to metabolic top So at and What's their kind and also to adults of convert is enough to a of number But Crysvita not still XLH, the in expand prescribe for to I the knowledge both who us of continue be we'll breadth to have to patients centers that haven't patients, to is some yet to patients may and and in continue pleased yet. launch the moving smaller important the pretty the who have academic XLH all priority. soon and the who prescribed with first exposure with of may their that's pursue community specialists pediatric managing we're prescribers moving prescribers. with bone are patients of think product kind

Crysvita. and moving compared to with on them to their patients diagnosed be educating we'll So them therapy helping

Emil Kakkis

on and the Then treating Based I'll DTXXXX gain adults safety there. from on strategy, question ask focus first, second then the the people to answer move information FDA generally to setting. on on down

forth. Our that of from view terms the not of and different and XX-year in XX-year is are so XX-year old GSDIa old, metabolism a their standpoint old that

down result So, dosing much more model our move something expectation age consider in like here that effect factor and therefore there to for lifelong early dilution saying Phase the grows and the size of that the treatment. be to liver dog liver to patient is is could and near is XX. important in some the now X how probably in is a The it would of effective would before the treatment potential full-sized grown, was from for fully result evidence

what question still prudent a the of are we're of population and younger the And we down be I to dilution. and patients. on early So in make lower? younger we in children. this on move approved will XX early is, and we get but what to the patients to with get go to going some goal question it's deal goal we the our XX. then have process, well, our And something XX with explore do age treatment the think will on should while do think we with as will to up down there in I like younger the Obviously, isn't judgment is

Unidentified Analyst

you. Thank

Operator

comes question of the next Your from Ahmad. line [Indiscernible]

Your line is now open.

Unidentified Analyst

for price questions. my in which LatAm a the difference taking percentage that price we least point And differences and price. for highest Thanks of the first us relative give Europe noticing the price kind any of you're out at U.S., the is is to basis, Can sense Crysvita? presume a on you on

Emil Kakkis

there's rebates Well, pricing complicated a required because other Crysvita? on. lot is and factors of For including going other things

and consistent for upon try range. agreed our to to companies a range that between a standard a strategies we was have have We globally set pricing within and to the pricing achieve

at Of different negotiation. a I point. full this different situations give comparison all you of the we can have prices course, think regarding don't countries

to we a I think planning to so what more say little now we response went that, think seeing moderated we’re we hold would can't pricing a too which detail price to to achieve haven't allow range to different territories -- within and that a that, us the consistent we us in more the better give dramatic and early it's you the differential has U.S. payers not pricing try for globally, pricing, is think U.S. but are I more is to responsible approach what we in yet. say from I fair so given are that purposely price with the actual a

Unidentified Analyst

need Okay. for And NDA, should for how if kind at the you get next? sales about are what then you of all expansion force FAOD upcoming thinking that approved

Emil Kakkis

can to that with talk start it. I Vlad let and I'll

of the all, manage and work with in-born the generally that error U.S, FAOD across certain of is FAOD. we XXX around by centers First U.S. the a lot that a managed centers the the usually centers world in number

So, there's concentrated Crysvita, hasn't unlike that. of targets many in targets set which relatively more a

-- happened be the there's to those So, MPS as X. same one centers

field talk So, about Vlad I'll let our moment. strategy force at the

Vlad Hogenhuis

Yes.

be that our presence, looking overlap areas address we a said, Emil our physicians our But these working that think, of metabolism able interactions FAOD MPSX inborn and of best on currently there's with physician as that So patients centers patient I population well. the with with focus see number narrow serve kind how to and of synergies to significant at to within fundamentally, current these represent that servicing them. big we're a option

Emil Kakkis

some -- think would to think many. I with manage So, we synergies very we not able be

we occur. a list, narrow already doctors. We'll large to of the look build-out have I'm And because this a at current -- would I huge have target going to large some not say change. -- managing as field for There's So, be. expecting this some not members

Unidentified Analyst

Thanks. Okay.

Operator

Cowen. from Your next question line the of Yaron of comes Werber

is now line Your open.

Yaron Werber

Great. Thank you.

cohorts, and to a of fourth decide based point second let's in at the cohort questions you got low couple what the Emil, say, I've if and to the obviously? on do of on increase XXX also XX first really to third So, terms the go maybe

Emil Kakkis

EXX, to Well, what by dose. the don't We protocol, dose. you for a would or for to high that better not have yet, think step aggressively. it that go to is we I could on we long and benign lot a and use them, protocol a periods range change think pass that seeing successful dose, the at currently be because substantial reactions in start higher people genesis that top steroid people or not EXX of would the the are think EXX threshold too shown have the understand a We up go also would we answer think we've and to normalization whether to of so successful, before EXX example. a a your an I dose. we do cure is you Because is dose, lot do is effects. want really that that smart more that very of lower seen all we that to to doing we've inflammatory can't lead of

have people something we other So, to also good is there a we will immune other be something our settings. be immune need enough many if enhance little bit, that's probably that response that as for like an at factor given innate efficiency and at look dose, believe we a at so looked EXX factors in example, modulation, response a could

strategies we're We'll the eventually So, should an to dose look any we it's EXX those safety that -- effective dose to other we out not have feel get question that although we be we'll issues. the could dose at not things. to we raised. I think within whether that both at able EXX, I've don't because of about consider impossible go I gate that but some need pretty right have comfortable the increasing further, looking think and it's of to

Yaron Werber

that the I presentation meeting, will don't at the subjects [Indiscernible] know Then enrolled I cohort, useful. two noted additional one and enrolled. very dose captivated and been be it's the has third Okay, that patient later in if sometime data data, the get give XX-week on with QX you us that presumably imagine, update would we'll probably that. on what is it's an thinking? can And you're

Emil Kakkis

said. well When feel say studies track the mid, in one-by-one we Yes, put mid to a decided do. in we frame. just We on we still haven't weren't the is data formal QX time to we enrollment basis. It's XX-week going something frame QX, for we time out all we're get

for So, that build well program. do we GSDIa to track are on program still the as that to

Yaron Werber

what and Okay. you as cohort data optimism sufficient. you maybe on in At there's well Dr. question dose at pretty data second sufficient And as with looked from cohort then the of fast good. pivotal? point prior move just based with it with how first the point is to Obviously, if what on can Weinstein a XXX the the terms --

Emil Kakkis

Yes.

the is do to metabolism to need, hope elimination well if that very base, So are the changing we we all to has XX% XX% of cohort, starch for glucose be there's of effects to glucose. significant expect a higher the as the their which jump up sure see than who's dose getting their someone that support in could metabolism dependency the Xx We second get so changed. on reduction enough we'd Xx want no that and we completely make it's secondary that given think we're maintenance if to cornstarch

very to we planning about But all possible think already and positive. three design and Phase treat and patients we another thinking into are So, that's if dose X. X certainly we that are plan Phase execution the at it's remember,

go having So, you in I up possible enough it's and right agree to that dose it's we If as be will our in patients setting three XXXX. would the but would probably quite to know I which confident. dose, biologic be not only think three optimal Phase as start say at the Phase X, they’re and dose,

the both to on be second can of So, the buy got made plan probably any we'll study. the to we that improvements for we've light as do manufacturing it FDA to manufacturing going in finish and three we on have the the assure design pivotal

Yaron Werber

on X,XXX based patients Okay, now Any your you as like how question. great. latest many Thank you. sense Crysvita. It in started about are patients noted been on work? looks the actually there's diagnosed U.S. Final

Emil Kakkis

Yaron. Yes,

of believe our still XX,XXX that projections We've not and the or we've very put XX,XXX very we would said U.S., a we a I on haven't and patients say of have pretty We is number there's here. identified number feel been identified individually And our people substantial that you fraction based out what happened disease doesn’t lot of that you've comfortable therapy got a that's number a patients unless first in population that to we launches. to of get means you, thousands just get, whole had year, you've because -- patients fast that the significant diagnosed. be on specific around rare but To expect. we of XX,XXX identified accurate number. And the to already there's X,XXX a seen

So, don't and we a think number. numbers we we and found to comfortable But right, be the there's right, pretty lot next All that diagnosed. evolving, have value. XX,XXX feel haven't put out of it's right lot about more is, question? like I reason and the the just going but feel And constantly a patients, of are is we

Operator

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

Your line is open. now

Unidentified Analyst

Hannah is Hi. for Jeff. This on

said that point has South Europe patients For new Mepsevii, in you've been finding its and America. focus

are So or surprises learnings how are those efforts any there Thank those from new you. going? geographies? And

Emil Kakkis

I'll on you. three about talk Thank direct rare regionally. and the of been let is With -- that start, others. launches Vlad which actually what's through, and I've any I'll rare on several, going of finish disease right MPs

we finding. in and steady launch both of phase and So, the step-by-step the development process looking slow all slow what call over products growth time, in process a is

Sometimes not the it's -- done. getting getting it's not

the patients, to additional one-by-one We're sound patient pool and the doing all we pulling finding patients lost a follow Sometimes up. steadily. to additional stuff bit are

that it's Naglazyme, been around when product. were the launched same and and been found, the not I don't almost time, but have Some somehow MPS whole is, every know why we

Vlad just about finding little bit our for level. So, what's regions a let's high a from Mepsevii on going talk general in

Vlad Hogenhuis

now hydrops in a have are example, MPS those I the MPS in get we're a to have patients, works an Latin In able there, patients. patients. And which and Mepsevii of couple been the effect MPS in level, those evaluate did looking evaluating X, because deficiency we're is And of kind U.S. fetalis, non-immunologic either diagnosis, into available. but we U.S., as who how of in patients X that to past, and with those so to continue in patients therapy, who patients Yes, identify wasn't are we find looked so high for actually attest the diagnose to testing we've evaluate our not it continuing Europe, the final can think in America, we of from to the a presence the situations, think patients, in wasn't are add I suspected reimbursed patients and number that. diagnosis the And well. who on to so we're continue of efforts

Emil Kakkis

And think progressing. we're It will the with definitely relative patients finding take it's time, I but frequency.

Next question then.

Operator

Nash line Robinson Edward from the Humphrey. of question comes of next Your SunTrust

Your is now line open.

Fang-Ke Huang

you Huang our This Hey. for for Edward. Thank is Fang-Ke questions. taking on

If know mentioned and the HEKXXX. stick to going transition the is I to recall why that you then follow-up HeLa let If you. going manufacturing. and you correctly, the to Just can gene to transitioning therapy the it XXX, to you platform, to for one stick program that's XXX just the are you're program Thank to correct, choose one you us other HeLa HEKXXX?

Emil Kakkis

the promptly program better the But, the -- we in ahead producing HeLa then, that process move system system we the sooner. put with to and a smarter The it's to post approach transform to get for a just we'll HeLa HeLa do Sure. transfer scale place appropriate will And in be approval GSDXa. long-term could approved. more could situation. GSDXa But the and think approval current after manufacturing manage think

to HeLa stay OTC, product. we We was not been factors that answers provide For have to why one at but, it it's mean many with as additional strategy. cell programs, But XXX doesn't for done. since some to don't just successful that at look this decided for the We need done for one we can't less it it specific viable. produced to We've productive producer and make we it when haven't other enough particular be point. applying would to for that it line but

Fang-Ke Huang

approved then Brazil, just you And you. think about Thank we Brazil is how helpful. specifically very in recently about should That’s one, since can second reimbursement? talk the Crysvita in

Emil Kakkis

For Crysvita?

Fang-Ke Huang

That's Crysvita. right,

Emil Kakkis

Yes.

worldwide both a be a that achieve for establish the on process can well team. reimbursing we but are little we any and regions a within in Vlad, will thoughtful very want responses including as touch access bit in-patient ongoing to Brazil. that a going that of ban good what Brazilian pricing to, Brazil, getting and ongoing the we're are have our We majority about as Maybe own you goal for

Vlad Hogenhuis

Sure.

for Emil. you the thank So Thank you, question.

us of number are system see meantime, formal patients requests will put approval and the get take pursuing for patients to be step at who by a should reimbursement announcement number up six reimbursement recent can a up Typically, new Brazil, next are that we discussions government on to we done formal of the years, get pricing in reimbursement or time, the framework patients' time be pricing formal for a significant have two which has approval. the and in that months, continue to the pursuing the For currently Spinraza getting we that and Brazil, through get Brazilian a court we to after Crysvita, been in preparing will approval. and we but In named honor applies one with in how and for patient additional and studying dossier. programs, access pursue who us amount the we'll government, of we three Brazilian where the to prior new Brazil

Fang-Ke Huang

Thank Great, details. the appreciate so much. you

Operator

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

Your line open. now is

Joseph Schwartz

for to DTXXXX course, and see was Hi. the it clinical Thanks in this able sense to time. if to want could -- pace. your give effect target you much. us durable low, of is fairly you GX very benefit are terms but over I or is clinically see be a the you what given profile hoping wondering What a of wanes threshold expression seems like to of have

bit to want little You a perhaps. higher aim

have see hoping Phase X? then follow-up. trigger on advancing what a pull the before to study I into you you And ongoing wondering are this So, in

Emil Kakkis

Great. Thanks question. for the

seen way of can better protein. number capture, other is of patient's do information measuring the have talking we're it hoping is better concerns for We size we clearly and heard look we're about liver improving. assess it. there the a The time of will the metabolic kind one is we certainly the profile markers is challenge try have to doing a of certainly patient. that, cure think program. for from membrane-bound liver-based at expression what at how that that to exist are standpoints I necessarily is to what to the on look metabolic I also There other and that patients and feel or health level targeted work are the more capture a are The

achieved, to the really there. darn were out to we And level just biopsies. figure we get necessary biopsies need to the expression So, didn't decide things

results So, improved of the clinical glucose I have secondary to improved metabolism patients. look the at health metabolism, secondary and of

this not the tolerate expect indicate that maintain would Our would clear able metabolic that to markers to is the without need full if the cornstarch around that the And to be lipids, having to and risk glucose patient in liver enough should glycogen. be at uric would acid would shrunken their target and all other liver order is treated have have to other abnormal the normalized. enlarged of things that hypoglycemia, night that

as beginning we're are put foreseeing within I dose see feel what of to Xx we this So, at required. should low-dose, we good think the at that range what's well that a

Joseph Schwartz

Great.

an give could study an Okay. you progressing? Thanks. I Phase wondering on that us DTXXXX, is X/X How then update when program. if And And expect there? the was update we

Emil Kakkis

A, Hem the A this GMP year. update on successfully remind include you is, deal before which had in the provide the running it. program, Street Hem that authorities being within now which is an patients by by and that on those We a to you. They remember happening what's a that the in product it with to driven HeLa provide and are it mentioned for expect But program. has regulatory buyer. the in their control And consider We the on XXX does been updates for clinic. XXXX-liter made it's with don’t our clinic update system, did what at I'll treating acquired won't accepted them partner clinic partner the we develop is

utility process important an of AV. terms of it's reproducible think scalable, in manufacturing program do to that we create So, of demonstrating

Joseph Schwartz

Thank you.

Operator

Your Stifel. Walsh next question comes from the line of of Adam

now open. is line Your

Edwin Zhang

Two Adam. question. Hey, is Zhang taking for questions the Thanks for Crysvita. This on Edwin on

here? patient XXXX XQ math, about added Do think a growth of quarters? pace have could recall a fourth patients in Going on if by quarter you how, seasonality? XXXX. Then the reimbursed therapy. new reimbursed few And commercial added XX reasons last you of Thanks. patients slower not quarter next the the be what XXX in of see have we do I forward First, follow-up. I my you

Emil Kakkis

of in trajectory on with Essentially this addition start patients crossing question, not then were traversed Vlad it. na��ve, very left. onto Thank to patients QX, clinical the included a remember, have drug bit launch, now. a were those naive to QX that perhaps let therapy in add drug people trial large number The I'll and a that more you. coming of on all but majority number small patients

And now in patients. so you're QX, naïve only seeing

not much clinical a seeing as You're patient.

Vlad, expectations to of that's know, patient you So at are if bit where of reimbursed what we're right add for I now. Crysvita. want on don't a growth little our more

Vlad Hogenhuis

we'll prescribers also the larger Crysvita quicker center, base. well greater see to within as to the continue reimbursement. go help year, expect of remind and patients a analysis, and be to new of will us our diagnosis them. to genetic counseling patients help essentially are on as those XXX to we grow programs continue also the of you, and pursuing number just These launched family of a we're confirmation continued March in represents quarter to patients grow so tree shepherd were to existing the new represented And growth XLH this patients. at continue forms 'XX penetration therapy who with And to patients and physicians number start allow April naïve in testing, positive in population. well, of aggressively and the see will last Yes. The patient experience their reimbursed more all kind opportunity an patients as But in Crysvita the which increase new some identify patients treating not get as XQ,

Edwin Zhang

Great.

your FDA? are Phase Second, is X you. data? to the Crysvita agency in And terms Have the Thank Where you allow filing the studied plan? PAO. likely how of with the conversation aiming we with now registration

Emil Kakkis

we about some answer our with for have plan had to FDA some the we additional and until discussion. provide some are the discussions so continuing we Yes, an set the after year in know information. additional filing the won't filing have Crysvita, later data that We

that have And we is adequate and saw have on information Our the bone we structure with biopsy same symptoms. therefore, benefits same in XLH associated on we the effect that data sense osteomalacia filing confident. position in that and the support function are bone showing we

I to should on you think -- else I'll let TIO. Camille, if talk there's like, add if about we anything

Camille Bedrosian

Thank Emil. increased trial of also with an our in of important for in marker TIO the Yes, the you, you the or normalization phosphorus patients effectiveness which Sure. with Crysvita seeing phosphate, Thank setting. question. is we're

said, As have our with will and the be updates you more we'll for we progressing continuing year. and Emil discussions this later agency,

Emil Kakkis

not we data and it us to the later bone the have structure, suggests delays drugs for But else subject to able to we off year. we and the So, everything sense will in you just file the further phosphate think update work makes population. same be

Operator

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

Your open. line is now

Andrea Tan

a if Andrea what Great. Salveen. index to comment of This real Maybe taking Crysvita you one of potential impacts quick if question. international Thanks could for be Tan Mepsevii? our and on could just the is the pricing any for

Emil Kakkis

think, from pricing right there'll question that large the us an targeting be. we pricing to similar maximizing are set earlier. come we strategies and pricing don't to because been interesting if the where about patients. to little on programs an these find be That's be us also -- manage that might Therefore up huge able But It's and be other across there's but thinking well a perhaps trying that sort between pricing of forward we're access to a that We revenue, our balance think question, we strategy issue. different similar territories. differentials and maximizing to will that we've achieving think talking to impact on for

were certainly and So, us we close will it important have keeping but it. a think impact eye on, an don't

Andrea Tan

so Got it. much. Thanks

Operator

the Citigroup. of Your Yigal next line question from of Nochomovitz comes

is now open. line Your

Yigal Nochomovitz

Emil question. Shalini. and for the Hi taking Thanks

was in they third Dr. a regarding XXX addition the I weeks. think of and fine-tune I made rapidly at comment anything only hypoglycemia, though with tweak the increase that coming you the apparently Are or in study to cohort Day, to an off cornstarch. to it And in the the cornstarch? subject for he that Weinstein interesting that time impressed three second result obviously titration think the perhaps XX% in Thanks. even off very tripling was the had doing the dose Analyst

Emil Kakkis

Yigal. Thanks,

he's getting at I think mode got What this age the of and are produced. sad is issue in constant cornstarch we've is at dose an getting their is constantly chronically. important patients a being is one. large They insulin

can adapt now is being so And a trying we're them their new where into still but release glucose, to insulin overproduced. they actually world

get is may them So, of may to back down. back slow ramp insulin to you it what it be step time off, modulation be of -- need you're insulin a a but saying to glucose may time that a down for the

were well. view to are that doing finding his lot patients thought was insulin. is responding, patient losing He's a he think comfortable just the But I clinical patients compelling him. very of how that

taking patients larger starch. time doesn't so him closely going little he overtake were insulin levels do. to to have We're on going his treatment who don't look slowing not question -- of more that will he's his down, on other So, in amounts at a what too fast

consideration. for a get and lot is you're So, their time it's hypoglycemia. up, little remember harder they we making they off, around hypoglycemia to their them test, important cornstarch of not But as right? the insulin actually them us reduce a kept tolerate and an Because take if it starch

going we at that don't to it works. carefully how that are sure a look to So, make But I think problem. is we're managing

have manage The patient change we to of timing think it's the goes. well. and and still that in I that issue fundamentals an don't doing over just dropped time the it's something think we cornstarch the what I do. has will process his how

So, whole we're to to have going we're really that encouraged attention pay story. to that by

Yigal Nochomovitz

sales that I of any KHK couple that. believe data I can for on study the the Europe strategy update to you get continuing partnership a approval seeking in more there's And and momentum. got order Phase to ride developed I still they're is just understand expansion obviously, And then good generally that waiting your just or in year file a long-term safety And lot on running obviously Xb United in in so, don't with market. adults know that States, for questions better in you've a the safety a the force even last providing clinical Europe. educated with the if of on you've adults, for is then experience product the KHK.

to in So, for potential the keep to Thanks. years with renegotiations KHK that profit respect turn the own the to is of over sorry, and let the to salesforce five respect royalty to so you them the your it switch to years, sort split in switch as could from start -- five with supposed any scratch? process

Emil Kakkis

already data adult they waiting with really it's start that They're to adults warrant. in I'll the the They have on controlled but file. to adult, all randomized right. file not Europe. planning need All

process, their they're it's steps But working have on of rather related the them other the to more I'd just more their not think updates others trial timing I them. related through provide is than data, execution what to aspects it for let of plan. some do in kind any and of it's

On to partnership U.S. looking good that also the have become commercial needs and in launching they company for be global deal we royalty, the with us optimize indication I an question our patients, important a the open decision it execution. that manage and for part will and the joint of thing and to product antibody between an ways product a Kirin the was We of switch think certainly right to I and goals the a for them Kirin that them cancer -- certainly do how we're the But at with want always that think to I transition. they're

gene products that expand so far on we we So other for done that also out point it's example, it. feel metabolic well, to work that and we'll you are that might, so bone to metabolic well do, I'd at do I looking probably so potential approval, for proceed bone comfortable or force in products programs -- otherwise. we do therapy have other to plus field many will as other as might things can our we including products

products an So, us for as I to as in work opportunity summary. will well. look on at that other Anyways,

Kirin to as in we've think we'll I right decision, a and and predict I getting revision and partnership but to Ultragenyx in want great but decision. other work deal have and the the done always the at wouldn't we'll point right this time, very there well make get them we at with the work

Yigal Nochomovitz

Thank Great. you.

Operator

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

open. now is line Your

Vincent Chen

at end Great. on patients? little XXXX drug. of XXX to to the it's XXX I that start I at of as start end longer much XXX the XQ suggest respect why would you the XQ But quarter, provide the in. be on Could which could to that reimbursed the a to briefly you the with slowdown aggregate Thank the trends get start forms is commercial translate of complete the of for end taking timing notice factor there and over was sort third to the on forms, that's patients their seems some in conversion you some to translated aggregate disposition. XXX U.S. seeing quarter, color at had fitting patients taken into other forms forms of XXXX. commercial could for driving forms drug to of start patients the on longer quarter later me it you're commercial for you start reimburse a Or end conversion which very comment Crysvita? if translates first of of wondering between

Emil Kakkis

of some sort Again, then the let that I'll start to of out and detail. finish Vlad question

genetic it, testing, plans things want the is few of to important requiring initially doing one that very started one went plans think recognize that's I as while were majority there genetic on now these the year testing. after of

drivers. I was think one the biggest of probably that

testing test weeks. requiring their hung And then plan, and and then patients goes some have test, then will then process to takes of it that to sent And to their they form, rejection test several the resubmitted they the had the are request start they the get in fill up the get they reviewed. then because

some not we're working a that problem. So, there will there factors start And to It's happen. are through. that big

forms to to need think talk start We conversions. But I and is work to improve that. to the help think of we're Vlad just can excellent. what trajectory those address through that doing I

you about maybe help accelerate that conversion. can programs the little bit doing, a So to talk we're

Vlad Hogenhuis

which the we Yes, no all forms start helps quality kind in having the errors improvement doing addition initiatives the that And with tends confirmatory since before things. are make to to the some sure submit delay We're testing to test compete. managed of care have that results.

the the completed In fact, start forms our we've seen trend an payers. especially accelerating with in commercial hearing

I getting XQ think also what you is new J the used to code. see patients, new the in Medicare/Medicaid

Some Medicaid policies in their states publishing take for longer little bit a Crysvita.

in fiscal then, to a a Just Wisconsin mention their the April. Illinois, year delay. New had And bit little starts in of York,

in that, reimbursed actually forms through accelerating are patients. and seeing but fully trend working So, generally carrying an into to start

Vincent Chen

much Thank Great. very you for details.

Operator

of Your the from line question Arlinda from Canaccord. Lee next comes

Your now open. line is

Arlinda Lee

guys. Hi Thanks for question. taking my

Emil Kakkis

Hi Arlinda.

Arlinda Lee

I on had couple more follow-ups XXX. a

in, You here. nights mentioned manufactured have we that currently it's two

will the how And incorporate are And Do maybe able will bridging now maybe convert which kind to that? regulatory you of that inter-patient next to technical can you this to requirements get you on dosing little the dose on? do secondly, -- dose Thanks. for a that? help important above or convert need study level is more little suspension? you what maybe do that be patients need the later Or regulators? inform to to variability. help then, think Or think you So, it

Emil Kakkis

have the had the in They place. comparability the evaluated. a on plan with have we with adherence we're already we comfortable a protocol, so, first about which just the already of very really regulators Okay, required and have question suspension. discussion a And

cell a And So, et cetera. it's step. is this same plasma, substrate, relatively the and modest it's same

pretty people many which suspension, adherence that. have and seem from made comfortable shift a really with it's So they

don't a being I bridging as see a I that study a required. So, see don't barrier.

probably going far that consistency, be think that, we're data think we get to able close and EXX should The to get place second question, through. to a the threshold where I that that we seen is we've so suggests to

environment is AVX more case, some so was kind need AVX. up seen in to, let's potent to view. programs several folds up have to XX:XX say get our We does of either the that other in but than And AVX

that think using we only another treatment a as enabling of said would all better also population I prophylactic the at limitations should place. any of we the we And a types if response patients. their consistent so strategy, prophylactic affecting see steroids, at example, for of not way EXX patients, from potency. be see across look dose, lot start of on before, Again, is at improve but that we triangulation, to with good And to medications potentially help responders

that's encouraged many are confident some responded us us And far, that feel unknowns, not options, real So, a using for close we're what But another that or good them we look patients. makes still modulation as think effect about has we for immunity but at improve therapeutic will have that's particularly has and our those And have seeing get further. way if EXX, a to dose. we their more. year been we're be what there only for would meaningful most, should and I a because so around now sustained of the hope strategy who that in possibly

Arlinda Lee

help to commercial the build start just do facility. therapy lastly, Maybe get then batches product? When or And think to we you clinical gene first Thanks. might of out

Emil Kakkis

would reluctant I go-live do -- you sure use time. take also time we if Well, plants validate, plant. to get them our design for they and the reluctant staff have manufacturing approval a to build me to our live are they but call, But, the predict would I'm be on too. that you to product. and have them Because know and prepare, be

expect In run. use And manage several even But and have think we plant the to in greater still for partners put more is And always should good quickly. CMO. long us we and manage CMO year work clinic the the after you the meantime, at a we of would our expect over doing us. and cost more now plant CMO give the So, between to products then, continue we as least just place. couple The process. in flexibility it

Operator

no Ms. may at further time. continue. are Keatley, this questions There you

Danielle Keatley

and Thank replay today you. shortly. on our our concludes available call will This the be website

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

Operator

today's conference call. This concludes

You Thank you. may disconnect. now

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