Ultragenyx Pharmaceutical (RARE) 2 Aug 192019 Q2 Earnings call transcript

Company Profile

Participants

Danielle Keatley	Senior Director of Investor Relations & Corporate Communication
Emil Kakkis	Chief Executive Officer & President
Shalini Sharp	Chief Financial Officer
Vlad Hogenhuis	Chief Operating Officer
Camille Bedrosian	Chief Medical Officer
Tazeen Ahmad	Bank of America
Maury Raycroft	Jefferies
Gena Wang	Barclays
Fang-Ke Huang	SunTrust Robinson
Arlinda Lee	Canaccord
Vincent Chen	Bernstein
Laura Chico	Wedbush Sec
Yigal Nochomovitz	Citigroup
Jeff Hung	Morgan Stanley
Nicole Gabreski	Piper Jaffray
Yaron Werber	Cowen
Adam Walsh	Stifel

Call transcript

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Operator

Good afternoon, ladies and gentlemen and welcome to the Ultragenyx Second Quarter 2019 Financial Results and Corporate Update. At this time, all participants are in listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference call is being recorded.

I would now like to turn the conference over to your host, Ms. now conference. may you Keatley, begin your Danielle

Danielle Keatley

our Good results for and welcome our to website release the find you Pharmaceutical financial the Ultragenyx detailing press corporate a quarter at We've afternoon results, on XXXX. which can and second issued financial update ultragenyx.com. conference call

and Sharp, Director Chief Investor Emil Officer Executive Kakkis, and Danielle Bedrosian, Chief Officer; Communication. Hogenhuis, Corporate Camille Shalini Vlad Financial am Officer; Officer. I of With Medical Keatley, Operating Chief and Senior Relations me are Chief President; today

was filed as May soon meaning XX-Q I'd which the Act call XXXX, that quarterly like X, the quarterly to will Form reports Form filed but will will of available the report include remind forward-looking Reform our the identified to, be investors report XXXX of periodic with that forward-looking on in filed this subsequent statements on Safe limited website our within on in of XX-Q on Litigation Investors types SEC, not of section. provisions Harbor including, be our Private Securities that the and the statements our

These forward-looking the involve of many from materially of any risks statement. only statements in views forward-looking and note including our call that results represent and uncertainties, beyond control. could differ as substantial are this Please date actual our that those projected

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

over to call turn now the I'll Emil.

Emil Kakkis

then introductory to before by will for in update on results. will joining the Vlad, second turning second commercial XXXX. providing and our you the thank some I'll us. afternoon, And financial over of you start quarter everyone the quarter discuss Shalini performance who call today remarks brief Good

rest of then and the the and programs the will outlook I'll year. across progress discuss discuss Camille come back for Finally, the our clinical

Our in Crysvita this strong the to our triheptanoin Yesterday, territories grow long-chain Application or in year, quarter Mepsevii submitted oxidation UXXXX for both continues three and world. with major disorders. the acid of second fatty progress treatment we the Drug New commercial momentum in of launch for throughout

focused through quickly the clinic get relentlessly this advancing in regulatory to possible. as on order have review We into UXXXX therapy and patients as to

and move For forward clinical to disease this therapy treatment we One glycogen Ia. storage the continue our for deficiency our programs gene the platform, type quarter. of ornithine two other transcarbamylase

our licensing collaboration results siRNA recently mRNA, we from provides therapeutics. our now The Importantly, solidified more treat agreeing XX this We've seen far. disease DNA rare and and mRNA opportunities includes by platform up a with with Arcturus. to so diseases and collaboration expanding to partnership greater promising targets

Type to the mRNA GST expected III is to Disease Glycogen program III lead IND UXXXX Storage advance of Our in XXXX. treatment for or

addition therapy In other Transporter have and filings. Wilson's we for III, to Creatine gene for These dual-trigger GST pro-drug program our our Deficiency. two IND disease our ahead moving pre-clinical include with towards mRNA program programs

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

Vlad Hogenhuis

afternoon, you, everyone. Thank and good Emil,

The from XXXX, second start of strong we've quarter. patients April prior received to the X,XXX pediatric XX% quarter steady with patients and forms this quarter completed number launch our hold of is XX% The adult quarter growth adult of in XXXX line pediatric split completed start United States. continued continue among in for in patients. forms the Crysvita physicians. with Since was and approximately in the The a treating roughly

Approximately XXX unique Crysvita. physicians have now prescribed

prescriptions seeing for the and these wide prescribers with time experience first reimbursement prescriber gain more we're As patients. therapy multiple the process,

than one encouraged than of prescriptions one-third that more prescribers for numbers today with writing grow are these more We to continue patient.

Crysvita. for This this for Earlier J-Code year, and the patients. helps a buy-and-bill process, Medicare especially simplify received Medicaid we specific

increasing We to-date. approximately private As mix of of Medicaid policy, J-Code payers the to continues and state have payer XX% and XX% currently payer’s number government a the shift. result and other

full point. of at lives nearly U.S. within this We the coverage have

without a case-by-case are on basis. policies approving payers Additional Crysvita formal

With payer we this now initiatives diagnosis easy process, patients finding then XXX patients broad Earlier to genetic therapy. with confirmatory but counseling, reimburse to therapy. all and and a confirmed a pedigree independent converting access types, reimburse coverage testing, on we this improved These across programs. have approximately commercial year, patients implemented have more genetic analysis

Consequently, of quarter. substantial we who second therapy the increase in number a had in patients began reimburse

among we're year seeing Crysvita and uptake in private which two Canada earlier can launched take insurance. pursuing public to and strong with reimbursement interest Canada patients years. We this up also We're

where America, in to the Brazil Moving year continuing where earlier and Crysvita reimbursement. for this see you Latin to pricing pursue pool we're

and respond We to also decisions Reimbursement a Chilean, next. patients' in request. markets we meantime, year and all Colombian, decision these and authorities regulatory this Mexican years to expect can take from continue the the the health of named few in

turning well. to the to and Europe, in more order Briefly launch patients Mexico, decisions world, continuing various we're expand Mepsevii, reimbursement next. is government approved expect we additional this regulatory go In and continues this of and the availability to States, the and Chile around year Colombia, health Brazil authorities United discussions therapy to with and Mepsevii

financial update. turn call a provide I'll will to who the that, Shalini, With

Shalini Sharp

press you, earlier June for We ending issued Thank was revenue the three that Total which $XX.X briefly release afternoon, will financial good summarize. a XXth, XXXX and everyone. months included update Vlad, million. I today a

North from our revenue territory. Net million ended million Ultragenyx XXXX, in in the KKC in sales $XX.X in includes European partner, for the Kyowa regions totaled revenue in million. royalty the For June quarter and total Crysvita Crysvita of share recognized revenue $X.X revenue or XXth, Kirin territory $X product million. other America profit collaboration This $XX.X

KKC. across of Topline Europe, America, shared totaled and sales revenue approximately America, of total represents This million. portion the are worldwide a Crysvita which Latin $XX.X North with

quarter was of patient was XXXX second $X.X million for $X.X named UXXXX revenue Mepsevii the and product million. revenue

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

As we've going revenues previously, continue to we expect agreement forward. to from be this stated minimal

this not providing be financial Mepsevii are of guidance experience We Crysvita will gain continuing at time. to both commercial and and

on We have strength and the including launch unique momentum start characterize reimbursed launch. provided prescribers therapy, forums of growth metrics to and our in help patients

We experience plan of in the and early we revenue level only appropriate granularity guidance this of are quarters provide to multiple shift gain the in launch specific to to launches as time we territories. with the evaluating

$XXX.X expenses operating total second of are Our quarter XXXX. the for million

$XX a were costs development Arcturus and expense and research amendment. research from collaboration including $XX.X million development million the Our

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

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

We the fairly SG&A consistent. versus of split expect R&D expense remain to

the basic of Net loss gain on the second quarter and of second or for for of quarter XXXX includes from of $X.XX diluted, related the was compared loss million million $X.XX value the the million net the with per second share, diluted, XXXX. or per fair $XX.X share, $XX.X and the expense The million the basic quarter investment to for in XXXX adjustment loss amendment Arcturus. $XX.X Arcturus $X.X R&D unrealized a and

change voucher the of of received priority approval. future a Ultragenyx’s loss value FDA to this quarter reflected in the the sale the the pursuant Arcturus second expense, periods. will R&D in fair XXXX for investment the million portion amendment $XX.X review includes with be the equity, of of that gain Crysvita Unlike in Recall the the from

stock-based non-cash which for expense in non-cash $XX.X and including significant exchange compared amortization of $XXX.X same foreign is adjustments as, used $XXX.X $XX.X rates. the non-cash transaction half adjustments such R&D stock-based includes first significant charges, -- the of of This subsidiaries million, the for restructuring rate in million and the some Arcturus resulting $XX.X expense included $XXX.X and depreciation includes million from million, million, fluctuations. and amortization fluctuations in For in and foreign currency cash the currency $XX.X of in and million non-cash for XXXX, compensation $X.X losses million of million to remeasurement, $X.X compensation operations million remeasurement our in due which to million foreign exchange charges from depreciation was $X.X period also expense in XXXX,

We million our Arcturus collaboration second in should believe and translational $XXX.X equivalents clinical the in of for which sale cash million investments, Mepsevii XXXX ended paid of quarter us available factors We years initial for agreement. pipeline. allow Crysvita to the advance $XX and the launch the with cash, program and sufficient be and and support our resources build research continue to to cash continue to in

clinical to update programs. I who would turn now our on the call like provide to will over Camille an

Camille Bedrosian

consequences Shalini. triglyceride lead medium-chain And or disorders oxidation these that for and a to are wish good despite is LC-FAOD. for a serious have There we metabolic patients unpredictable screening crises management that Therefore long-chain fatty with for and afternoon. high needed MCT disorders you, everyone frequent patients I UXXXX also and new Starting acid newborn current LC-FAOD, can with precipitous rate mortality believe oil. treatments urgently with hospitalizations. or Thank

Yesterday, The over whom FDA. majority a with a been XXX treated package of to bringing submission supported step the data triheptanoin broad our of the We of range goal team therapy is toward the a UXXXX time. to long patients. completed or a drug by our have new comprehensive patients, of application significant over submitted from period

access review a data the which -- safety includes company-sponsored retrospective patients function. of previously studies, impact sponsored includes cardiac results study, the study and from XX submission and the efficacy use review UXXXX, randomized patients, investigator extension on showing to compassionate triheptanoin X of The record the expanded Phase long-term naive controlled medical

disease for enables the priority granted FDA this Earlier year rare for designation eligibility Fast-Track UXXXX and which review. designation pediatric

to Now priority acceptance that we within the expect FDA review designation the submitted from submission days. XX we hear back have application, on and

our full disorder deficiency ornithine gene to ammonia coma is OTC metabolize for Patients and into with X-linked up toxicities OTC crisis, deteriorate other deficiency, their transcarbamylase excessive leading an deficiency, hospitalization, urea the of suffering to limits build even death. neurological ammonia. can and or deficiency metabolic deficits Moving DTXXXX, amounts and cycle to program ability OTC that in body's therapy quickly blood from

infections from two dosing cohorts, maintained genesis levels We normalized and XX study and their the liberalize weeks since the these urea respectively, responders -- continue ammonia who protein had their levels. medications, cohorts two of first for in their have tolerate OTC rises through more previously include ammonia XX data manage of reported we experiencing without scavenger discontinuation and to to diet the

or this related data These events adverse related gene infusion so cure metabolic treatment any OTC seen efficiency serious therapy. not a possible suggest for events. adverse far is have that We with

GC/kg We update are X.X dose at and in program of currently XX^XX provide an quarter. third a will the we DTXXXX the × evaluating on

lead potentially program glucose-X-phosphatase allow defective during therapy in the resulting gene constant severe to This glucose storage release disease for during GSDXa to well. liver alpha inability continues as glycogen increased or by enzyme as our caused DTXXXX into metabolic is stress to circulation. Type progress could in GSDXa gene or to hypoglycemia a fasting, Xa infection. such the the

dose point previously data GC/kg. or the XX^XX × XX X.X setting. to this a increased provided patients and show for week three in durable fasting all the in clinical maintained time hypoglycemia cohort We of responses their At lowest controlled time patients

their baseline liver. three further Importantly, XX% improvement reductions and of at demonstrates which patients reductions compared in the all cornstarch weeks in XX%, have with sustained to XX in XX% metabolism glucose

We plan cohort of to of the third to well. dose are and quarter in XX^XX We provide second now GC/kg. this the DTXXXX back program will call to I have update an a X.X Emil. × on turn evaluating this as at moved the year dose

Emil Kakkis

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

expect we patient with the America. expand Crysvita, regulatory our recent to prescribers to in and launch decisions For and Latin strong our in reimburse and Canadian formed launch growth start named U.S. see and reach continued we'll continue patients sales

gain As we for company of to the U.S. broadening expect needs to contribute the and efforts a precedes our product to base launch of the coming pass territories over the early filing revenue alone, Crysvita approval years.

and therapy review defects. acceptance XX FDA for For could ultimately continue days. in long-chain believe option process the meaningful throughout UXXXX, acid a to on patients back we work next treatment We'll hearing the this the and designation NDA with fatty submission anticipate offer with the oxidation

in and Moving third we two gene continue to both clinical therapy transcarbamylase programs progress to Glycogen and programs, ornithine programs update Xa deficiency on the our Disease provide type an will the quarter. in Storage

submit three three for programs several and are XXXX. Finally advance and data to of continuing preclinical we our pipeline, to in the packages preclinical on-track we're INDs

first approved our grow summarize To continues to far therapies behind. is briefly launch UXXXX with two not our

IND. promising gene and We preclinical stage three all two have pipeline fulfilled have programs program -- and refilled we nearing our clinical with stage therapy

been clinical option have five continue therapies have and companies, few few filed well rapidly poised do since for BLAs timeframe. are multiple not deliver. successful are to the four there and very in successful or a are programs We this treatment patients developing There to also who have who IPO, rare NDAs good completed years companies three last in disease

develop, The to progress in approval gene in new product catalyst us a obtain will on ability company. and globally commercialization and Our as therapy, commercialize filings, skills and terms unique productive a for puts disease of rare had regulatory a efficiencies we make year. category

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

Operator

first [Operator of line Your question comes Bank America. the Tazeen Instructions] from of from Ahmad

is line open. Your now

Tazeen Ahmad

cohorts. about taking readouts for Emil for Thanks the XXX XXX afternoon. good Hi, questions. the ask to wanted dose you and in my higher just

steps you are for would take? positive, data would be next that the -- the if So what both

Emil Kakkis

that Well, would another with both then we're at to continue, dose to the And we patients that ready and if define cohort protocols be we'd add that then plan reached that XXX we information for X. going Phase dose. three XXX

as talk also move are program. proceed enhancing we confirm, studies in first cohort ahead. about treatment find to would we But that and other more will Phase few both another both want about looking X we'd we FDA program a -- dose whatever We in at patients, and to before for the with to might -- do we want talk we things

Tazeen Ahmad

how other? Or much move And are cost, in intention look which choose the constraints the over of to data one that both your would is terms it forward? have for these positive both any if you to there

Emil Kakkis

products the as clinical At we perspective, are planning putting both perspective. this our we're a well and development include X Phase point, planning, that's plan in CMC the as from

Tazeen Ahmad

Okay. Thanks.

Operator

Your Jefferies. second from line comes Maury Raycroft question the from of

is Your now open. line

Maury Raycroft

on my and if named-patient my thanks this from questions. in -- on you requests question taking can the few America any far, provide work received comment quarters? was Hi, pricing over on everyone. just the that the you've contribution and Congrats region next Latin for how named-patient the progress and details My was so will revenue the

Emil Kakkis

I'll named-patient it’s little think been already responding we've a some America. receiving important, revenue going I provide what's I to. let have detail that been on more putting request we and think Vlad in from South on perhaps

Vlad Hogenhuis

Thank you, Emil.

Argentina, number and we're getting So named-patients countries, request Brazil. Colombia a in of

were submitting and Authorities. Care We to the recognize are processing invoiced, the with Health actually we appropriate these revenue. documentation requests them Developmental And once

an process give a The with bit to in And color. for to little the patients of you named-patient going and the legal patients that looking have to Latin Brazil, all through In of its go system injunction is helping we're course, to product. request. America get forward

Maury Raycroft

it. Got

the are guidance on the Okay. at just then, noticed therapy so question two titles for gene a -- XQ with the And that there program, SSEIN we've conference.

you if that whether line would wondering, more any on around Just can top be specifics conference? provide the data

Vlad Hogenhuis

we will is be in haven't but We conferences. But possible. be haven't the prescribed it conference yet shown planned, the yet, what at would precisely certainly what

data. I the abstract include think existing

Maury Raycroft

it. Got

very Okay. much. Thank you

Operator

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

Your is open. line now

Gena Wang

my you will that taking America. Maury’s for regarding question, -- reimbursement get commercial contribution, the questions. meaningful you How Latin revenue soon follow just Thank think patients the do the we process? Maybe

Emil Kakkis

each reimbursement. that in Well, the have different. named-patient a was past, for as unlike country, Argentina, law allows Vlad new noted, they In

it and from some Colombia. In talked first. what have time go patients there right start ago that to process, legal through request have that gotten we once treated. number seeking approval than. get changed, moment the patients just patients process And through We've And Argentina about may are So some that, process, Vlad in process faster a their gotten takes actually a was, get of things and we to getting then filed have there. Brazil, that constitutional

a But is and and lot investigators meetings substantial down seeking can take there conferences I patients that are treatment of a that with Brazil time, four there this can Brazil. anticipation us, in that. of some in you step this maybe We're in myself there and move has And for see the number and but their put there -- timeline Brazil tell process of Vlad, also excited Brazil able in treatment them patients the about and on tell being to reimbursement ahead. for interest itself. to was

Vlad Hogenhuis

got in Sure, in we approval know, so you March as regulatory don't Brazil.

to and now getting We are proceeding pricing approval. reimbursement

dose, XQ said by this patients taking them And to that that for reimbursement the XLH. submitting will in we're soon. of two we expect can request of meantime getting for we're but of the we supporting five take to process these Health, by in appropriately of year. which our slide hope end years of evaluate who We that's we're Once Brazil. complete as Ministry want of getting start reimbursed preparing and Emil and to lots that accelerate between And Crysvita to all interest up from see and

Gena Wang

And the patient, -- patients your just rough also you. family and XX% wanted calculation revenue And existing tree pediatric resources these the U.S. percentage that roughly the versus numbers how and if for Crysvita? with for adult based in-line the just wondering Thank see how of Okay. to they're through the the of $XX of new about patients? is regarding much many like million just the is the revenue U.S. on my patient

Emil Kakkis

for finance numbers together. on there. give we I I where we've Several are, to on the couldn't just We could is perhaps getting on what that link this detail questions provide think the point, put mom some to and forth on treatment the put I definitely families could at information don't revenue. are thought that be out going family are keen answer you But kids closest is able treatment Shalini we second wouldn't I the there sure to know Right. patients. know trees would part, between what we and

Shalini Sharp

Gena. the noticed not break does as question, territory. the revenue down may you And for Thanks Kyowa have by Sure. Kirin

by for an at not But up of profit $XX.X share Europe, our them that's to quarter royalty million. revenue is So in royalty The this share specifically. the disclosed we territory is collaboration is $X.X which North disclose and America rate million. the in what this revenue XX% is

are disclosed at the those numbers that this can all we So time publicly discuss

Gena Wang

you. Okay. Thank

Operator

the Your from Robinson. Lee Joon SunTrust comes line next from question of

Your open. line now is

Fang-Ke Huang

that, are one question. mentioned for elaborate may for Maybe question, remember on Quick thank response. you XXX Is seen? And And you what differences variability comes that, the Hey, it clinical therapy? from of taking the conference a from Joon. follow-up. Would have the evidence this pre-clinical to studies, a this evidence at some any of as or gender differences Emil, have you is do evidence is on mainly which supporting of the their gender can you it constitute animals response terms and Fang on gene other you there in see from just well? I

Emil Kakkis

you. Thank Yes.

explanation possible could particularly one differences being animal is examples cohorts not for observed in efficiency more literature resistant. with our models proven and females different the response There be in a in different that. patients -- is we So in of gender early gender, the have to we

patients manage patients looking We're possibility. the we in And on immunity able We there dose, at is looking don't for some how our hormones with immunity. that and was as evidences evidence all the on that to that certainly happening relates though in that. to possible be response, believe a their we're able too. female effect that enhancing provide was to immunity are truthful one increasing and in We to may overcome that be angles have female

have be that's and that therapy. something male, been seeing many but will considered something to known programs of clinical not is and released difference, other before The been you're a have gene therefore female I in programs, think have that excellent those it

Fang-Ke Huang

patient expression very some on And more there is produce suppress question also helpful. I as mentioned starch to transient Great. And degree. is going in-turn and going That's insulin, the one then XXX. reduction think in and you to that second a insulin if patient, consumption

there the to -- help And you just transgene the where promoter it be be suppress that. transgene, loop could is insulin Thanks. used your -- for that's more why understand for going about helpful. mentioned understand probably that, a expression? will to what think you contribute maybe help that how Maybe feedback us normal can you I us

Emil Kakkis

Sure.

in glucose physiologic So that control control respond your needs to or normal signal, X-phosphate body. the we believe like glucagon, the things to of insulin that glucose

responses so respond which promoter as of region, is that as to concluded as insulin, both kilometers design And includes three well steroids our to the that glucagon stress the well. elements or

to optimal to the insulin condition increase expression will phenotype of is the their the on in-vitro. an would let able come those that We three rather as shown And levels expression the need they that let of in the all their that promoter, will to starch come we received to process think transgene reduced been We wane fourth need patient And normally expression do to clearly the potential the maintain give provide down glucose-X-phosphatase. their and than to All that glucose. the first that and a normal. under suppress we've patients. would patient, gene in their that insulin enzyme. starch starch, But know they've back increase of has

is second lower of in patient started starch, a with. had to three you relatively all amount into number small at reduction, because a begin one as heard actually And reduction. XX% the are at a the XX% reduction, down one now, is lower Really

So actually low are all that starch amount of now. relatively

and their what manage long provided. the think their that as as transgene I shows, glucose ramping they internal So new keep starch, come back, with we

enzyme their So program waning we're how switch provided that's on away get maybe the system to starch, we of that dependent insulin part from gene therapy. to we the forward look starch metabolism one is new through confident as we the this based them on

Fang-Ke Huang

helpful. you much. Okay. very so Thank That's

Operator

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

now is line open. Your

Arlinda Lee

seem with different the what dosing you I'm as you're I Emil, all my cohort taking have wondering I'm XXX. set dosing would kind those mean do the the curious, think I'm of the thanks looking guys, looking cohort -- at expanding to an data Thank XXX, as XXX with well And in Hi, and these kind patients had also first for question. schemes. what to upcoming of you. for particular schemes? effect. what about curious that you potentially talked do Curious for be? to then threshold about

Emil Kakkis

Sure.

lever to EXX So, we'll we've now level. patients, the put data in gotten one get we make is And out conclusion. data XXX, and talked a the about when our -- we on to to dose enough

Our we've the improve see adding expectation is that level that enough. if if lever talked as we about another be steroids necessary. possibly prophylactic should perhaps What enhance in order efficacy, to and

not So that's have terms results look And an is only what make option what like we in decision. dose the might of and that the we see lever we'll a do.

to way study approaches. want do comparative get need right patients to actually we in them answer. Could to our get study affecting life, to we I in order treatment the right program need these experimental with more make and decisions is are What in what to whole do the their and

pretty in question, metabolic -- close we findings reiterated Day gotten For were at Analyst the complete that discussed to XXX control. we've haven't other patients now. the detail, which three the have included that And

to I But see good is if that but try next we it that as that to the to achievable is that level there can second what continue it first it to and a as level were level, and ensure pretty question efficacy that dose actually level moves some of we understand achievable, enough. We're also level is I higher at cohort or the it that dose the looking enough. -- be think was explore different could know. think up better or of And if it

they result, time to the see potential to of proceed need the to and we'll it effect, and that high will third maximum wean that but and think data the that, put able be dose see a there or out start is the how from do quarter. I this we'll achieve to be quarter, XeXX takes the to because respond sufficient enough

Arlinda Lee

you. Thank

Operator

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

open. now is line Your

Unidentified Analyst

for for Judi Joe. a DTXXXX. I'm if dialing Thanks I taking in I question. Hi. our question could wondering [ph] on was ask

third of humans such compared levels tested Specifically in percent to expression of normal dose what And gene have based different mice, models And in achieved different --what need up. of deficient doses percent how the in I does correction tested mice. OTC being a as pre-clinical the and on patients mutation. follow to was

Emil Kakkis

little mice are good. translate mouse Very dosing because is AAV easily much with treated vectors. The a more to difficult

of dose dosing in we're the doses mice clear, kilo the to translate hard, to is translate the per that in so exact a the humans way So should XXXth range just XXXth using mouse but the be at eXX be is therapeutic. what the to in that reach but But I is XXX% even cured, a to say fully mouse, should be would significantly you humans less primates, easily the liver higher doses. in it's number achievable to non-human

is the really think The have. of normal XX% which ureagenesis doubling of cheaper amount maybe we have we is to a what get in this program ureagenesis, to is to they about of get

XX% X% like type they X% and more them into So of get to say the patients need we to of have to range. let's to these normal may be X%

them equivalent So normal patients get sufficiently is is the to XXX%, mean is doesn't give convert they around normal probably convert it two But ureagenesis normal them first normal. that XX% XXX% or in in ureagenesis, XXX% of urea the of ability level sufficient we enzyme ureagenesis. to a enzyme achieve reach of means normal don't In their that did will to their ammonia and hold have to are to to the range ureagenesis. XX% to that the give means their them enough normal that able cells what now or gene XXX% level have

But animal to overcome different And ability levels, resistant But that primates, dose it's working is as we compared everyone relatively going were a to clear, -- in human’s to looking mice what in the we're vector the therapy are to to this it's easy these in AAV. pretty to a the space in small to AAV. humans resist what regard gene more and easy So to is with be increase. mouse at XXX%. at get know

Unidentified Analyst

question strategies, the has on next Thank I is was to approved. And provide great. some your color you could my commercialization Okay, do if drug for wondering you assuming the UXXXX. with clarification.

field your with patients that base, working with Given the physicians Mepsevii. force Crysvita and with different treatment is and currently

Emil Kakkis

Yeah.

So institution. disorders that handle doctors some do treat that some are endocrinologists are the type actually on too, of depends different, the Crysvita -- are metabolic that it there

five familiar products relatively patients very metabolic, launch that. We're XXX and a the in launches. or [indiscernible] generic have around team is where that centers with multiple into manage managed are six concentrated the XXX, actually these and are There involved can of smaller

looking existing will at forth. launch. But for we that launch. MSL time and commercial in team. wouldn't the have us the And there team so We leverage it's But figure team want best to I think to the way options, already metabolic impair some how to Crysvita the is be a whether out

launch. with because of we near have product same size smaller to the team number as number number this and given sure think to want make a time, targets of in with don't we the the Crysvita, we'll the we need see. doctors Crysvita, anywhere large are At we compared the as of of dealing to we targets re-sourcing But it's we're relatively a

Unidentified Analyst

very you much. Thank great. Okay,

Operator

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

Your line is open. now

Vincent Chen

much very question. taking you for Thank the

programs, the are therapy assess leading and alluded heterogeneity back result thinking in gene I on the been some hypothesis? what of driving revisiting that wondering results. what and you've some going to that was DTXXXX, trying previously is the you to latest and one, the the Another especially what's of

questions be some to third this? a the updated help but commentary, prophylactic system. steps the as potential lot other dose their of I the quarter of B something related you've might the the continues the steroids, ability? that reach alluded in immune What of show range then for are data earlier to of understand increasing addressing to to what if levers would you elements suppress of heterogeneity, and range, the me analysis range cells drivers consider or you know in And corollary appending the a in could the your of of

Emil Kakkis

first I the Very good, going any hypotheses so what's don't on. with new regard have we to on question, think

all in that gene-therapy think is, could is which programs. I gender the of the source immunity, innate one variation of be factors clearly dependent

EXX and later of time that been overcome we'll the remains able And to data it be quarter. I And Most does people to that provide that. hope still dose, dose with be the possibilities. this of one the that would that

about the One think when immunity And things is suppress prophylactic been how considering the to steroids from of have to shown AAV. improve the beginning, you we've it. by been any transduction

steroids. it is other impact companies' seen the from products expression, with significant primate We've that, prophylactic We've steroids and in also non-human hired done, using that both think on ways, I it clearly there have is prophylactic productivity of without a studies.

thing the if, just it's gave think thing And weeks I levels it a normal even expression it’s adding a we are add-on. another can fold, to very add-on, boost, enhance to if And steroids twofold simple few if a achieving a So, of steroids. natural of needed. you couple

worth therapy, hitting doing cells It's important. whether even gene think expressing, their prevalence XXX% these you're normal, enhancing of benefit because the if long-term of is are I

the us of not in here we whether as, might something targeted that So types that be do looking we benefit, might program, We're tell added are drugs' to relatively at other looking X simply. currently an Phase at

a is difficult lot area. there don't much out think there. a It think necessary. takes I into you it's And I more

on adaptive little immunity. a bit focus less Our

a more seeing we're Gene being And expression B-cells. the question the responses. corpus mentioned You antibodies immuno of on of or core the immunity, kind as We're getting seeing innate lot Trans initially. the not because directed

think you're of then Gene, part If about the want antibodies seeing affect to you that that things Trans might to immunity.

we innate dose. enhancing suppressing easy immunity, focus steroids potentially on compression. And might boost right to an as prophylactic So through at option now look

Vincent Chen

Thank Great. much. very you

Operator

Thanks.

line Wedbush Sec. from Next comes Laura of from Chico question the

Your now line open. is

Laura Chico

wondering genetic XLH a Hello. Good around was could for earlier your back moment for go we questions. comments to thanks if taking and the testing. I And afternoon. the

I'm just have of the whether curious, of might if to shift with you. figure you've had population, you then, quick any how perhaps sense as genetic testing? put the I that And implementation for terms a follow-up in out there XX,XXX that

Emil Kakkis

I so program far, put clinically seeing results high among genetic but we're haven't we frequency think Well, our of diagnosed testing exhalation. confirmed out for very explanatories

clinical are the accurate genetic at the diagnostic feel comfortable those and wouldn't we alter prevalence this point So, we testing that numbers time. numbers think would

Laura Chico

comment And titrated now more kind adult all across a versus few up. you've And have perhaps you at of of it. persistency wondering rates patients as as the and differences patients? there related can belt more then Thanks. I observing got are to had here, your that Okay, on XLH, kind pediatric quarters any under you're compliance guess

Emil Kakkis

haven't the on We yet in essentially compliance and anyone trial aware had don't -- but I'm is all we haven't were put so actually out rates what who can of. say that pediatric and drugs them, stayed we formal persist -- I patients have lost I don't

the were certainly there been some excellent; the from don't we source may population this -- our who the it the continued, so it's in trials it's And a compliance have confidence been at adults not has very problems and point. among to as relatively just not program So, good in good. was but few far a think an persists of of

Laura Chico

Thanks very much.

Operator

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

now is line Your open.

Yigal Nochomovitz

just taking of to the XQ to understand trends properly quarter-over-quarter Hi, from XQ. want I Emil question. and Shalini. I for Thanks some make the sure

was -- reimbursed So, America. higher But in U.S. saw North and there in you a XX% much increase in patients a share increase profit the in XX% XX% approximately collaboration

there And pediatrics mentioned you growth patient earlier adults. price was no far was as just mix the you on and this as for XX/XX consistent was there that a there split understand

some or that Thanks. growth in patients up? of how into I'm of an to in for not we could just patients insight a is. skewing trying payer the cadence it quarter in shift or get So, Is a provide the accounting just if potentially the understanding to lines the the mix related in what maybe discrepancy the Canadian the

Emil Kakkis

is more to we on closed reimbursed that the change reimbursed how the can the one comparison think I that patients patients. getting gap many generation, we Sure, the definitely compared form we the that. e did -- -- do of start start things were to of do rat

quarter. can the or on input maybe was provide definitely enough you have the question numbers feel that some a difference I the for So don't Shalini in of these differences.

Shalini Sharp

question higher Yes increase and is the story the I proportion start that's think than in that -- quarters. no, patients it's probably the the reimbursed versus prior half in form reimbursed a great is

well. you're the profit that U.S.-only obviously with Canada whereas we you provide about start as secondly revenue data that the North actually share absolutely And right American includes form Canada

the are key explanations. those So two

Emil Kakkis

therapy on in Canada we insurance. continuing have grow. who that's patients And to do have private And reimburse

Yigal Nochomovitz

Okay.

So the both profit the profit that's getting you're that, share a constant. though a but calculation, I know not you -- details better haven't at higher gotten higher… into the margin It's as on

Emil Kakkis

No.

Yigal Nochomovitz

Right. revenue. …on

Okay.

Shalini Sharp

That’s right.

Emil Kakkis

Yeah.

Yigal Nochomovitz

Right.

noticed wondering, trials, other steroids prophylactic particular and the same hadn't the clinic the experimental at mentioned just then that we for yet. I because least regimen exists disclosed prednisone. for XXX, had, you cohort detail we -- you And XXX that prophylactic whether Thanks. least for question posted of we on at Okay. XXX were have publicly plan And with seen

Emil Kakkis

considering are we weather steroids clinic. level, at seen enhance of expression animals, based as the in any dose the well peoples other Well, use as on in data could the we've

we're So XXX. considering the

because For has use steroids how metabolism. of affects XXX, the some their of glucose complexities, it

that we're that decision. final made could potentially we we haven't through working discussion, So add it,

to overall the with was verify, worth and felt steroids if enhance help it it just worth we see. doing simple, to got be a it prevalence the expression bump XXX, would we something it For capturing so for we

Yigal Nochomovitz

right. All Thank it. much. you very Got

Operator

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

is line open. Your now

Jeff Hung

the If the on Thanks outstanding pricing talk that of remain questions. a in about can Brazil, the dig formal steps bit I little more taking dossier? you the reimbursement approval for can ahead

Emil Kakkis

start Vlad I'll with and finish. Sure.

-- The or filed wants setting. pre-approval in things be we than do order why named they clear, changed to patient immediately to legal process approved. rather want the doing reimbursements; have process that drug to time. in Brazilian partly government So in see over is This Brazil, the

the to requests three adjudicated get you cases That process to the other this legal there's get the two where get taken response, which approved, access. to filed cases more we patient starts; the and get And first So named-patient therapy through. perhaps, allows you, do or few. process promptly then enables will than

in a that up few Brazilian process and the that who patients middle were and now we're process. in queued right So of quite have

other to of process, in course, I'll are look There a Vlad And process. of bit going time the addition let about exact And, steps the talk unknown. are is normal several the and amount we little to if can tell to go commercial we the just faster to reminded that -- process again what process. what negotiate usual maybe the me But an reimbursement process. you briefly formal the was, Vlad access can than program

Vlad Hogenhuis

back the Ministry pricing the that completed, we price. we report is are approval Once Brazilian December. we so to submitting from Health submit around approval, to price the a we process, after that of hear So to close be expect and U.S. expect November,

XQ be two between can anywhere submitting XXXX in and dossier years, get five that time to We responded typical for will reimbursement five. vary to closer the a to

There actually are opportunities that a do -- partnership. public to private

receive We for experienced will be is I'd approval. who patient exploring XQ to we though, programs named team a companies. pricing those in other done after But many Brazil very has have that the we add like in

through the ensuring that where now to well versed legal to the named kind getting we required the So patients these they're for of requests Health Ministry patients sales and from first get we're up they're compiling patient the waiting see of Ministry the legal systems, materializing. so the all Health, of experienced in paths system the will and these physicians, follow well by through information the appropriate

Jeff Hung

support you remind to guidance? in given then, quarter? the about Thanks. it appropriate Great. part a of the a to mentioned number you're Thanks. you are change of guidance. And you've time for amount you how initial been waiting in a Can timing sales launches, and us after an of a thing medical quarters? certain Is Shalini, certain Or providing thinking

Shalini Sharp

question. good a That's Yes.

a other recent studied We product And in depending we're on wide around we products sales, other when not also North there only stages disease the in launch, practice and but territories guidance. range is which have rare in to which provide have analogous because in years of America, of launches and in territory. various

we a like we to Kyowa top Kirin, provide Crysvita when also collaboration they so would with align with on have that, have And to them to guidance. with

So of those are be all things we'll considering. kinds the

guidance to one once we'll to So than agreement and able partner, then in have further provide level you. with of be products a for we our maturity these market more

Jeff Hung

Great. Thank you.

Operator

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

line Your open. is now

Nicole Gabreski

Hi. for for taking Chris. This Nicole is Gabreski on Thanks question. the

being HEKXXX for two used within these are your of manufacturing HeLa was therapy gene facilities? and the Kyowa is to wondering just you just up. on same precautions within I safety platform? just from platform So of that set bit centers as to manufacturing take contamination the could you of risk And the if so, adenovirus limit us give the a color excellence,

Emil Kakkis

manufacturing, cell based contract Thank where That you in in for the you the from so still same we're process XXX manufacturing, HeLa locations. cell we're have said the question. certainly. they're different doing the doing control to Currently, really places is not the

including all system XXXX of HeLa run reactions disposal system. liter the bioreactor is and disposal these cell what think I

controls And of that feel have problem and so with a the comfortable we question. about not seen the

Nicole Gabreski

your this? HeLa and reasons And Okay against plasma of been if so, reason a over cost that to production the has the then But use to were is costs. GMP high mentioned just you've system to to plasma bringing production HEK's. there one any as follow-up, reduce for in-house what thought

Emil Kakkis

done become for We that have we something supply could XXX, that certainly just, build done currently plant a the It manufacturer. and of plasma have include certainly and made to we another and own plasma production. could our complexity, people could some adds chain and decision it level be

a another not really I plasma, the smaller think and cost and and much the with up using people XXXX-liter probably issue one growth HeLa, a process. transaction reproducibility run is to cooling they're can reactors. But commercial factor with is factor, include running possible, we scale most manufacturing going is robust to just end liters are the it really cells but sizes reactor reproducible of create more XXXX

is manufacturing. for undergoes XX,XXX-liter, no plasma it dedicated if of was doing could make but XX,XXX-liter. vaccine XXXX-liter, one it you akin and said used barrier transaction we The And

truly the commercial HeLa cost reproducibility AAV the sort manufacturing, commercial kind with think you're -- we it's we system of of create what think for program. structure is generation, next where going the truly AAV required to a So and of

We it's better certainly be rare believe as to approach lines a producer indications long-run, we are and still HeLa be can but manufacturer. in the approach faster done can in cell using XXX. upfront, AAV It strongly these to commercial long-term

Nicole Gabreski

Thanks. Great.

Operator

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

is line open. Your now

Yaron Werber

question. my Great. Thanks for taking

gene two Shalini. maybe So, Maybe just for a on about follow-up therapy.

So to. a quick question relating

a steroids So of give and the is help what to what out then three initial few us sort also into you patients, have trying of – patients. plan well. next follow-up do whether you I terms figure at get the this want step you as And expand try a the I'm dose? point will more a and higher to make decision a sense to to XXX, after a the thought is next in consider

Emil Kakkis

we're that another to as protocol arm second we're with comparator group Well to in then add do continue, achieve let's get the see the say if of add but still and out The thing, steroids. the the three, two responses without of see will two doing what do, an we same is good really response even steroids. To arms. arm current to is The could response, idea do what better continuing substantial we we a get of steroids arm. the planning If it, study that we matter protocol whole now no with three that the that do three. at dose

the that's So argument.

the it's to go if option where certainly off range. be could to get we in however, higher We we not an need the table,

a potentially simply possible. than improvement as thing, get If a couple add the it really you answer and think steroid We simple increasing dose is can want. fold to better usage to something a simple enhance that's what that's we as temporary further of

of make gene no we this and the and experiments doing therapy is where X. a add situation, Phase do and do should and making right we a look inline, on they patients won't tweaks of how this thing without now, experiment one steroids group lot comparative decision linear comparative a and doing then is programs, a one a good with that steroids look you good So if think

enhance to done, arm steroid three tell we'll us another without three another further add gives a two be their of a will proceeding, that we've going that's add we're simple regardless that use can and assuming you we steroids. idea. of look We're at we hopefully the that addition results So getting then to three-responders. steroids an will arm will effect how it by whatever And or

Yaron Werber

look cohort probably Got prior Phase obviously do like to trial starting to then what depending figure may a next potential with X Phase you see Okay. will terms like terms now, X steroid in endpoints? the year, the was cohort know sounds dose based path to of dose do you but going next It what what in shifting and And out good. in on a on But you what design pretty of it. we'll element. the you XXX we're think registrational expand

Emil Kakkis

control general studies. clinical metabolic endpoint. disorders, metabolic tractable, would they're now believe for our for And these right that will Well, since metabolic, and preference FDA's endpoints it's more is be a pure view we to make have example, doing them the a

or which XXX for but So stability, whether and a whether it's time around have glucose control at tractable looking removal and is be to during glucose been glucose we've going of but think maintenance control, we for issue some cornstarch of aspect it's we're the FDA. relevant working or hypoglycemia time glycemia of also the hypoglycemia to

FDA. discussed We the with it haven't yet

of program a placebo will a XX for that of think -- likely example, XX maybe kind patients study. We be, XX treated,

thinking And the needs are in that were about discussion thing we complete As before of both plan. an idea FDA planning. current that's planning we for we But however this kind is our we share we in that just the made Phase want best be choices. plan, we've year, in but II, Phase next X program we do to make what sure to that our

Yaron Werber

And timing segment Europe by potential expansion KHK great. adult XLH? the any filing update to for That's age in Okay. label on for a into

Emil Kakkis

they to update publicly. they've disclosed because have that planning together any know do the I know forth that put they've if filing. I are publicly, don't you that Well put Vlad,

Vlad Hogenhuis

if publicly. know don't I they've disclosed that

Emil Kakkis

historically they been we the putting required. have all information together, it they've know Well,

they doing the think And if their to Europe. in we it's have in I haven't But so them filing. court disclosed what yet, make to disclose it let they're

Yaron Werber

Shalini. for Okay. And last then maybe is question

around reported $XX.X reporting $XX million So roughly. when morning they the and you're that was KHK sales we this calculate

there Latin or reporting and we're So now is is not seeing? else something the which is potentially America not Canada that KHK delta

Shalini Sharp

you differences that no, good as very of Yeah, the America report or read any way named-patient hard book two. the the in as sales couple footnotes revenue But it's we way also a total to in question, are in figure. we the disclosure are noted. in their different of they Latin the not if is, you their reconcile report, they access sales include And early do

Yaron Werber

Okay. Terrific. Thank you.

Operator

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

Your line is now open.

Adam Walsh

of used guys, brief The I gene thanks by you my your on on trial? in XXX Can most comment Now with first. current filing made, there accounting Hey, sodium historical measure therapy And on variability helpful. have OTC trial. color been then FDA second interactions, you're acceptance speak UXXXX level you for in assay potential you based to for how in the variability filing Thank the a the for ones approval your that assay be as you. package I and label increase assay the in can drug? taking that of the ureagenesis That's the ultimate assay. observed acetate two here. believe, measured your and the questions. has to any the any for confidence would one NDA recent

Emil Kakkis

good. Very

So, NIH method the assay we a the assay repeatedly the work it's within they've best XX% our you when study, developed doing consortium, where funded for patients look range. ureagenesis disorder, number some And an patients, stable pretty within a of for that been are if years, was for a have around range. urea by that they that cycle it's Usually ureagenesis. feel consortium assay question, to were of and in measure, even are in on

doing to but are on it repeated occasion more consistent. people define not who had period we time, collecting now data -- over We've help been, of appears but have variation, that reasonably

is that patient long prescribed in has the example. as sources fasted feel for happy, we So, and and is the doing sneak of they're test, in that cannot is while the for food done as way one variation, it

they in less it Therefore, as the of just change the we're long way, seeing be past through we're well feel the that the be amount variation do and you in observe should what we would that answer can't variation, consistent as So, calling XX%. patients it than responders to as part. prescribed

that ongoing and we've high the information, question with have XXX Well, you had reviewed. detailed discussion FDA a was great both accepted previously the second we an asked we're confidence will and on The an that assuring dialog be level's providing confidence. the pre-NDA filing so

they review, we added time accepted, feel XX We review; between of that the let's the patients get filing additional worth data. for to

of patients in the number had an naive reduction of that the to XX data quarter X prior study, had with study. Phase Another the third as days the in two, our well hospital retrospective in treated median we consistent as XX% in days that from

had we we more now data getting when decision. than are made feel they So, actually they actually the

added we've that we to access think strongly which been collecting, information is support addition of. In we've expanded

them and talking exposure, is of XX seriousness it is we're magnitude confidence of that of high continuous than our the size the of for with and of about. sufficient some combined type exposed, the So, years XXX packages patients the more data

the a clinically hospitalization changes of oral of These types toxicities aren't substantive meaningful that downside. some GI Combined simply drug GI these rates; are but observed issues, no changes. very large a there's is drug, changes with and that small at in upset very safety profile

we and pretty feel think approved. we we benefit confident, this can I risk -- is the through and strong of get review feel get

had work in questions FDA we're they're we want have sure right process and As make we'll the the good and getting they raised, to them answers. said to through questions those the and with questions

Adam Walsh

Thanks Emil. Great.

Operator

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

open. now line Your is

Unidentified Analyst

This for the taking question. Hi. for Cory. Nina is on Thanks [ph]

to Just quick want questions. two ask

the the one and kind level have about thoughts little quick going a Medicare to in And just follow-up. one how about just and and about, that – exposure impact you pricing I business the on question, bit quick recent U.S., proposals might higher talked hear So Medicaid your wanted of forward? then the a a

Emil Kakkis

as they hearing and with we Medicare support our at look good. of are the treat now risk. benefit patients creating that don't -- approving, for policies policies Medicare are We responding Well, exposure Very look are at patients. and the to stories is

there the its we've is just think, that to we seeing the terms is far that that of impact to because and fact for in in support interest bigger a this that drug, So disease. you going much profound other rickets show percentage aspects of of been

a on think of table, the we reimbursement So are we -- going question Government lot the it Group that, questions that on a similarly we're pricing closely. in have confident and on and follow proposals Affairs I

I think the issue pricing problem. that Europe cause is we’ve to asked, whether reference ask us about would people

issue work And not us. is say we We should have pricing together other I to U.S. not think in strategy maybe unlike we feel and thoughtful about and particular situation. on would actually very differentiated for on with pricing how our that from a we have been significant Europe that the an some way Kirin impact

So, we feel like we are in good shape from that standpoint.

effect hand that is going very to think rare situations space people rare of and the these money and and drug. think lot disease market done special kind huge there and conditions, I kids disease amounts important, we is be think it's is thing of parents at are their of issues of there diseases I whatever want always pricing get I larger think that where but get -- is really to rare of just people will happens, fundamentally believe I that treated. and all a there very from profound want kind

So to happen. that's going

Unidentified Analyst

quick filing then for Crysvita? for on follow-up, there And are great. TIO Okay, updates the any just one plants

Emil Kakkis

are that. the guidance, TIO FDA provided yet haven't We we on with working

we'll that will details continue our update could but get that be the then details all we expect this But are want able confirmed, is and to those expectation we point. Street able we confirm details file we at but come. and TIO, discussions hope to we file, when but on to the we yet Our be to the

Unidentified Analyst

great. Okay, Thank you.

Operator

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

Your line open. is now

Unidentified Analyst

Wilson's the in thanks file IND I This for you're the Ross planning you taking Hi, to XXXX. on question. know for is – said

look here think just trial would what Just Wilson's and like of and about help as be clinical what we us interest? program clinical understand endpoints of you a a what kind be Could can like. disease would

Emil Kakkis

Yeah. Thank you.

So more other copper knowledge the important, then the clinical fortunately a regulatory other copper is on had of and copper approved, is people. except, about Wilson us therapeutic down that a disease Alexion currently lot loading metabolic in gives used for prism been a disease throughput us indications. and or has that's the the in move gene the for Wilson was copper marker, basis use the a that new the is which primary has path. biochemical particularly – copper animals. our is freeze in as but a We copper, and are abnormal using to endpoint feel, trial, [ph] this We also chelator is think And in think therapy is free metabolic use levels clearly you we which has going is and go thing you in there strong when program, which

the situations gene think have are study for what who CNS, long-run. we're liver liver I as help in at product weren't else to as And therapy look to to both doing with we well that benefit the have also the the patients the disease, at look of injury, support in said,

our what biomarkers. expect neurologic very part and liver important for supporting in their We while the benefit. primarily the And are in copper would study, study which phenotype and focus it might we on to so look prism also of

discussion to that plan be that history are hope a with design in becoming programs. be that to past. is move Wilson there agreement been for not the that to that, because is terms to is do what program But that choice endpoints through more forward-thinking think is, development good group have and adaptive more contiguous design established yet. control FDA something to FDA said we and haven't have they is clinical we an signals that our looking our we including plan require this We pivotal, the the initially. And from from would they very adaptively of a our provide goes at ways the smoother, think a and able will into dose randomized And hope working cohort and faster

Unidentified Analyst

Great. Thank you.

Operator

at to no to Danielle the this further questions turn back would now showing Mr. am like conference I I time, Keatley.

You may proceed.

Danielle Keatley

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

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

Operator

Ladies for concludes today gentlemen, have joining you day. wonderful conference. this and Thank and a

disconnect. You all now may

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