Ultragenyx Pharmaceutical (RARE) 11 Feb 222021 Q4 Earnings call transcript

Company Profile

Participants

Joshua Higa	Senior Director of Investor Relations
Emil Kakkis	President, Chief Executive Officer & Director
Erik Harris	Executive Vice President & Chief Commercial Officer
Mardi Dier	Executive Vice President & Chief Financial Officer
Camille Bedrosian	Chief Medical Officer & Executive Vice President
Yigal Nochomovitz	Citigroup
Gena Wang	Barclays
Tazeen Ahmad	Bank of America
Cory Kasimov	JPMorgan
Chris Raymond	Piper Sandler
Brendan Smith	Cowen
Maury Raycroft	Jefferies
Dae Gon Ha	Stifel
Elizabeth Webster	Goldman Sachs
Joseph Schwartz	SVB Securities
Joon Lee	Truist Securities
Jeff Hung	Morgan Stanley
Liisa Bayko	Evercore ISI
Joel Beatty	Baird
Laura Chico	Wedbush

Call transcript

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Operator

Good day, ladies and gentlemen. Thank you for standing by and welcome to the Fourth Quarter 2021 Financial Results and Corporate Update Conference Call. At this time all participants are in a listen-only-mode. After the speaker’s presentation there will be a question-and-answer session [Operator Instructions] Please be advised that today's conference is being recorded. [Operator Instructions]. to Higa. conference like host to now Joshua today would hand over the your I go please Sir, ahead.

Joshua Higa

the full and Pharmaceutical Corporate for Financial afternoon, Results quarter Good welcome and Update to Conference XXXX. year the Ultragenyx fourth and Call

our Relations. We you results, at I can release Senior Higa, website which am our financial Joshua have on Director press issued ultragenyx.com. a detailing find of Investor

quarterly and Camille Harris, are on Kakkis, Chief remind Officer; President; this Chief and Erik this are SEC, from uncertainties in involve with annual, the of Officer; as call to reports Emil differ Dier, Officer. Bedrosian, identified forward-looking me available Financial website. materially Medical forward-looking in Joining Officer substantial Chief like Chief only those in and which time control. I Mardi section all Commercial Executive would and represent actual as the investors could meaning to of limited on our statements that of and this Investors that statements. call XXXX our These note the will provisions Act include not our filed beyond forward-looking any but risks the are call statements results types of within views periodic our the safe Private the including Reform that projected of the many Securities statements including Please Litigation of forward-looking harbor

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

Emil Kakkis

potential start Regeneron the a concluded strategic good second disease global rights to afternoon, Evinacumab. rare both the everyone. disease therapy rare for of exciting to year. the We with and a for and outside Thanks, license commercial Evkeeza deal US We've had Josh an

and This the manufacturing to Mardi Regeneron accomplishments commercialization the discuss let commercial deal finally, technology antibody absent of finished Genetics to and -- across Evkeeza that's Regeneron receptors low all tolerated marking XX treatment investigational well biologic US. HoFH does or with one LDL-C been the strategic across study Evkeeza copies LDL-C. showing mechanism lower by in a and XXXX significant dangerously have ectopic commercial in high-quality severe for all resulting the XX% to these also has the for cardiac from partner muscles to years, reduce study despite LDL LDL-C positive a studies premature LDL freezing treatment than weakness study program to commercial activity X% of reduces three or events we've patients FOP, by same for the global receptors. update of the were functions. of approved receptors. ancient when Evkeeza occurs VLDL the clinical this pandemic, and risk safety the in genetic a ongoing that We traditional with participants. the We is on of for medical reduction observed. showed four – in this by deal treatment been cholesterol the ANGPTLX less in affairs patients of and over not severe outside ANGPTLX LDL Evkeeza enables XXXX completed in abnormal in extremity us FOP the the The in financial disease patients commercial Daiichi patients a exceeding currently keynote populations. regulatory is targets scale my Los EMA atherosclerotic are showing that met disease will receptors HoFH US. minutes of Patients that our and difficulties uptake for two Sankyo. to Camille FDA first antibody XXs. challenges an also strengthens data clinical the of is recently remnants And their the strong development. leverage Progressiva partner call. alternative by by rapidly Evkeeza that do Along of are clinical in ex-U.S. advantage appropriate with HoFH events. with liver IFOPA done is an levels in lowering discovery blocker our both an work the with good our progress position most XX-week natural Regeneron's a this protect the lead its disease standard set lead and drug in spend Taking product of also XX in at underlying devastating this are male has we activity the instead also consistent meeting. and approved also and Despite establishes reduced and Asia. approximately expectations our by agreement Ossificans very presented with Fibrodysplasia XX% portfolio leader for high profile true license released the way right with of includes products. and top pathway regulation late-stage our in presented to over novel also negotiate great move in on atherosclerosis. has include broad to direct collaboration significantly another existing rights with for or drug reduced our in and HoFH. more eating, formation deal that pivotal older. atherosclerosis. with movement working homozygous in disease capabilities launch FOP. with level improvement may care each and broad patients enrollment the of value Evkeeza, to whose a antibody include binds into America novel other patients hypercholesterolemia and Phase our in LDL-cholesterol with familial many including premature atherosclerotic X/X from liver a a and study patients be are with have It and LDL by which details receptor walking on single to will XX% expand inherited regions of with and pivotal clinical [ph] VLDL This and of mechanism program US disease demonstrated where the patient and homozygous affects hypercholesterolemia LDL cleared triglycerides programs In all We that breathing In we transfer are receptor all good genes tolerate occurs the familial is to the COVID protein Evkeeza the blood treating exclusive Latin on liver present of alternative plays despite of I'll each Regeneron separate that the also partnership is in lack few Europe, with Erik, this I on I want the FOP the patients commoditization called supported That week an parent deficient to treatment treatment mice In targets another also from training cumbersome. lead potent our and through in will also made a The on the pipeline, patients successfully converted leading treatments. LDL year. provide while death don't tissue cause a all that commercial excluding countries and FOP. later ANGPTLX, severe genetic territory. a is patients initiated development [indiscernible] in The performance enables single and which like of by at and blocking their role we mutations very a Angeles every the of bone ultra-rare and very still Evkeeza the soft in approved difficult to stage a bone

familiar a talk completely to teaming to for hand and seeing Evkeeza. Erik, I'm forget. what now to I'll disease, waiting to terrible or someone last positions team commercial is not readily them is about over freeze in the who will launch this you So, call patients with site year the is doing frozen save performance

Erik Harris

Thank you, commercialization Emil, Omicron and the section performance good the afternoon, variant. the impact everyone. I'll despite discussing start my XXXX in of team's

patients of from continue pediatric we therapy we remains see the to demand markets. underlying adult and to the XX while steady total fourth the the within approximately split US patients quarter, In North alone. pediatric XX-XX in continues both unique The prescribers the added and increase. American number over Crysvita, of adult For territory, on

portion shifting as with have teams of The towards expect on adult adults who in will high. split doctors this greater TIO remain We increasingly compliance XLH continue Crysvita setting. the or community the rates finding a patients are

years better of Crysvita grew momentum. once Outside million. much for from receiving North product gain they four XXXX feel begin In fact we how XXX% to demand launch into to In about continue $XX.X revenue hear patients this continues therapy. stories they America, to

providers teams of them work demand direct there and patients find is While doing and are patterns in work have it's regulatory for the variability to quarter-to-quarter This clear may in Crysvita. fluctuations result that caused underlying reimbursement educate a be ordering been the strong revenue we with the a authorities. all

an a expect be For year-over-year range $XXX growth disease midpoint into $XXX between of million rare impressive represents territories launch. XXXX, and to revenue Ultragenyx four metric years this we The Crysvita in XX% million.

start to with XX since bringing fourth Turning In launch XXXX quarter approximately start starting XXX forms. launch now we metrics. to total of approximately the Dojolvi forms and US the the added

patient of patient a in Dojolvi of many to Outside in XXX and early patients the Italy. providers Dojolvi care approximately Dojolvi continues growth writing the named them prescriptions for for increases of therapy. programs. XXX of patients. quarter, led reimbursed As significant by our US driven this have has end prescription France Europe the In on access unique written Approximately the of was and health named requests use multiple through fourth with

this LC-FAOD. to process, are to Health all National Late with with begun believe the have final that the Brazil's Surveillance and I regional to approval therapy. few adult $XX year working authorities will launch of specific our representation months. We to we We expand is $XX them for a get Brazilian note last continuing on quarter XXXX Agency getting pediatric of a Dojolvi. access could we for and Dojolvi the of from for last ability look the we range work guidance Ministry finding patients metrics the better to is be million last and The have full for provide million confidence should who through the both approved we step from continue reimbursed patients of Finance benefit as reimbursement treatment Dojolvi. patients

a approved efforts XXXX relatively Now between to is be with also Ultragenyx patients will patients. shifting have XXXX of patients and Evkeeza organization. of opportunity, error where the with HoFH inborn gears approximately like be This on lipid commercial to HoFH. we estimate rate Across we and identified most by global the fairly market a an is Europe Initially to estimated territories, establish truly will commercialization These diagnosis of us treatment patients. protocols with seen HoFH portfolio. specialists. and help established for the high of as well-developed further XXXX focus knowledgeable our typically program which a estimate there cardiovascular metabolism are we physicians

is respond As name that supporting be discussions. work is reimbursement team the for will to KOL Mepsevii interest and in which dossiers and requests current seen will America given their established and portfolios. ready add and the from Dojolvi, could also our Latin patient modestly This to build already in commercial begin also we begin affairs Evkeeza access, XXXX to medical infrastructure and we infrastructure to Canada, place community. to the on In strong well we submit have

and of addition portfolio efforts, the support stage teams to services a today This into as prescriber little successfully to required. also have for launch expand very We field needed a Evkeeza hired new recently the have patient and long-term and we Evkeeza Ultragenyx place build-out region. where with entity commercial management. sets the is geography our The for our general Japanese in we established APAC a

these regions of While global help should that disease and mission rare reimbursement have company, processes. along being its to advance note a of unique Evkeeza pricing and will we truly each Ultragenyx are complex

Evkeeza feedback our for it offering colleagues on over In forward revenue proud I'll We speak the opinion continue from as all the like as to through call begun efforts. new for on have financial significant time closing, look just these see option and am a to HoFH. to patients very some team's But with I look themselves you patients. With these leap supportive processes can reiterate forward a new the Key in to know clinical program therapy. results. leader and I and build work take XXXX share to treatment forward data for that strong how and is of momentum. XXXX would Mardi turn the to compelling efforts as and will we I the that, I to My

Mardi Dier

profit grew $XXX.X million Great. summarize. XXXX release I'll Company of America XX December to the $XXX.X for net Total included Crysvita to revenue a the press including territories revenue earlier European financial XX, the totaled We in $XX.X year a regions. Crysvita which in share briefly and Thanks million. was issued royalty $XXX.X for ended that million, other $XX.X Erik. million. The today Ultragenyx the Dojolvi North revenue the months of from in territory product sales million. was territory $XX.X revenue sales related million update

for of ongoing range. XXXX Erik launch was $XX reflected our at growth the represents guidance approximately strength midpoint of XX% million. is which the guidance XXXX Dojolvi, As the the mentioned, for Mepsevii in

over have tech $XX stated, modestly transfer time. with technologies. substantially these In strategic and technology XXXX, license may increase Daiichi to we manufacturing our is fourth part revenues XXXX total partnership agreement of As included previously also recognized through and we million HEKXXX completed quarter as revenues Sankyo expect related the the were revenue around our $XXX.X activities this PCL transfer December XX, under million. therapy gene the XXXX

a million, were expenses research and payment total this includes million. of setrusumab of $XXX.X and collaboration of cost note should regarding million related also Our SG&A $XX license $XX Mereo million, operating for $XXX.X development for I expense of the agreement to the year one-time sales includes for $XXX expenses OI. which upfront and million expenses the of

as X/X program, of most next clinic. We mRNA to UXXXX ready pivotal and clinical number XXXX for we studies support expect the study X/X we GSDIII our activities spend study ICIEM NOI, R&D continue our the study preclinical advance to Phase programs in UXXXX increase the get to advanced Phase three therapy clinical X/X somewhat and other and as the the a into gene Phase

in XXXX we Evkeeza. launches expect also support of and expansion We continue and SG&A our to to modestly products increase launch commercial as existing the the of

or billion of Sankyo For $XXX agreement. million the of the the operations collaboration XXXX the year per a includes operating equivalents from received net used cash period in same ended decrease achieve Daiichi and December XX, value cash the closing ended the in million a XXXX investments. included the with net related compared included cash license strong ended cash was agreement. loss the XXXX. the million share. ended was few critical presence and XXXX our Net approximately the to in commercial December of that million $XXX.X used to and fair in $XX.X Net for of This We net position marketable expand $XXX $X million, $X.XX million loss months The cash December XX, to XX, the the in puts Mereo for compared for XX This XXXX securities. used payment same us year XX-month approximately period period the in over upfront $XX milestones $XXX.X cash, next equity to years. in

our turn clinical to the touch over call some Camille of I'll Now on programs.

Camille Bedrosian

are With this as repeat forward data dosing Phase Preliminary update you. the call our to disease on and I of updates X/X good first currently turn to programs; in baseline back single Starting treatment look X the X the of storage therapy placebo-controlled dosed X now back providing following of study. reported imperfecta. this expanded, will screening young arm dosing be extremity GTX-XXX, with to no in the anticipate or which phase enter the genetics UXXXX disease the will shown adult The or program initiation this of ascending this the Emil. the development dose Wilson Angelman randomized syndrome of will startup. and DTXXXX dose disease Phase antibody to after stage adding enrolling treatment X/X Phase UXXXX the enrollment the osteogenesis the for will the eight with enroll initial the treatment in XXXX, the Canada UK provide of DSMB study. ASO our with under I Xa of We and Phase treatment that placebo-controlled therapy, In four in protocol. patients seamless gene the the X study. as in anticipated study. patients collaborator and in storage call US Phase the our mRNA of second arm DTXXXX, X continues signs GSDXa first well weakness. glycogen Mardi, glycogen the an briefly on early mid-XXXX. clinical randomized before afternoon. the to to have patients in protocol wish stage for turning UK, update, the everyone eight-week for for clinical they six gene Thank study I a the in UXXXX, in type of of with assessments a pediatric half placebo-controlled patients or is programs patients half half and ornithine have currently the first deficiency in will begin seamless amended study is X/X Outside patients lower from the Canada the in Emil. of is OTC currently three with in Cohorts antiscarostin We status Thank dosing in cetruzumab Phase and is the final an too X/X/X section, of you, type and activity. first randomized patients support of the study additional transcarbamylase for XXXX period patients for year. my

Emil Kakkis

you, Thank Camille.

of Before quick reminder we key close I'll company. a milestones the the provide out upcoming for

provide For safety data U.K. Canada as X as mid-XXXX in GTX-XXX treated patients efficacy and arm the the in the of Cohorts X update and Angelman available from well an and study in on in U.S. the will

programs. durability conferences. continue major at our longer-term And gene clinical provide studies from For pipeline, late-stage and three for X/X the enrolling OTC GSDIa we'll we'll therapy the Phase data

osteogenesis begin pivotal half of an in in for For the pediatric old. the half years and we'll year. study under to Imperfecta expect the UXXXX the strategy X/X the initiate we have selected dosing study X dose on a children update year first supportive second Phase Phase In in five of the the we in provide portion

the advancing XXXX, the our on that, UXXXX of half Operator, For X/X and portfolio clinical Phase bolstering we manufacturing please tech completing dosing look data expect we With transfer technology initiate and this a our sharing first of provide position dose Q&A let's part stage. In ahead the XXXX, steps directions. In year, the our from building share move study in financial second of therapy the strong in to updates schedule. a questions. single your with made programs building we commercial you. gene the repeat on and to forward the to meaningful to

Operator

question Nochomovitz first with of from Yigal the Our Instructions] Citigroup. line [Operator you. Thank comes

Please open. go is line ahead. Your

Yigal Nochomovitz

with had randomized Great. trial. taking for respect question you're to for Presumably planning placebo-controlled to your going pivotal my a very trial I a you the just Thank do questions. Angelman. much for

have Angelman observed of there's a the that the main observed, clinicians the far in XXXX given developmental How function overtime? where X/X/? are Phase instance a makes assuming And separation For the point change from X.X my history slow you. thinking see increase pretty you publication this that Thank Journal quite question of placebo in natural actually about disorders is nicely. so the so with the you've line in in you

Emil Kakkis

Yes. Thank Yigal. you,

trial. though, are gains change time in about as thing to kind the the may gains modest Angelman small observed the there one compared be think I pretty some over of

subjective before is it approach in to be we'll no that CGI able concern measuring our if from can Phase have the a tool the really I placebo that at as opinion disease, efficacy question of depending one a the is to but we no X, approach achieve we've more on So the and all. seen distinguish The investigator.

other I use currently us function underlying allow effect specific what to our that on before say, study are But question time that dependent, a we're going effect I such our have that working Right magnitude with and out understand be particular need and we CGI which genetics seen up understand we've certainly we the no we're profound study will in view was instruments still with will walk endpoints, others effects disease. given figure And assuring, of we to look development and capture doubt think endpoints that's a now that all. and get or change and study. figure it's the will which that or on very raised, in these figure as measures used out are separation. also -- powering optimize have Phase carefully we and some our certainly it's before I We meaningful. come we to would our the a to have in at the a can design studying we appropriate fact dosing Whether the of and design this of will whether concern X magnitude on observing for which out this. your based We'll

Yigal Nochomovitz

follow-up And topic Thank to super this helpful. interesting. very another deal. Okay. was regarding That's Obviously, Regeneron the on you. then just

the usually smaller license obviously you see but reverse to asset, an happened company the larger to the As here. commercialize

what or following one-off that of this presented itself more a with situation transactions was Regeneron? with more see we happened could is, question the So Evkeeza, Thanks.

Emil Kakkis

big and and we I act Yeah. how are a just behave. we company, think Well,

said, felt of but in they on Regeneron what great company, for is best the and what's the that's capability, going care confidence So it want had, who rare especially want a and built global they to take and to haven't get appreciative these here. we commercial do their and were because thing handle to they But we this patients they they them space, care, have business a guys of really well. good product you product about care

for pretty busy it's of So we're that pipeline anything, deal. now. and Look, reputation our two-product correctly full care if taking in together is there's draw right it people a

important So right now, we think And want deals view, take get put be in to an us in not to that deals. And do to is we're work our pick of be potential on want think precious on expecting – to time we proud commercial FOP. our want in we a are and standard be We effort. to life-changing has them many to order that But care. we're a Evkeeza, burosumab on to think to we really selective. products for change change and other we of, people's will that care

if could won't keeping So going what we'll comes. – and we're we we'll think now, and And just see else excited right about of stick about both those it be wait those.

Yigal Nochomovitz

Thank you. it. Appreciate

Operator

you. And line the Thank Wang with from comes our Barclays. Gena of next question

Your line go Please ahead. is open.

Gena Wang

I taking my one have Thank Angelman. regarding you for also question questions.

January So CGI score. of in Emil terms said the update, in you of improvement sign the early

second loading part is and example answer score that we patient what are the cohort, mentioned for past year, like data if you improvement? you the which out see you will to of out regarding trend, a the that a you patients first for dose mid you what kind forward XX mid could the year pivotal you And additional this of then remaining expecting in that initial study? we can specifically that definitive what In will patients criteria? the two also did like for domains. Did more continue And a eight be can from two data move So the five by have two little quantified see? we dose to lay after this loading see to bit dose, from could improvement see each know meet score

Emil Kakkis

good. Very

you, thank So Gena.

patients improving we'll they said, reiterate is we in and that just in cohort was plus we Angelman achieved clinic lowest domains the patients For the next In is those also escalate met and the two than not scores the but the X plus. they that Cohort activity that what in said improvements more level. seeing improvements their cohort had but is patients the dose for two were therefore in two were patients also to the that X, escalated had that we older has cohort. allowed CGI four that expansion domains, the dose of additional two hadn't And

beginning any we're We're activity. So extremity to right the get at clinic not in titration. We started. the seeing get We're the wanted lower seeing of issues now.

So They'll XX we're those encouraged. patients. and with move we continue forward to as titrate dose

So that's kind of where we're at.

level or we titrate out saw specific if domains they haven't to they plus We put would efficacy we two had said the scores. before. two of But wouldn't further. That be getting considered

continuing we're to and with but we're titration. forward dosing move So encouraged,

what four be kind data from just that Cohort would whole data includes about which first of their -- as that we doses well endpoints Baileys would behavior the and it midyear. five of – We're talking but language XXX and and CGIs be the and see? That not include expectation we expressive Now see and would series would -- scores will Cohort gotten a day have data evaluation. evaluations domains, a the evaluated as what now was sleep

number of be a have that from would psychologist. supporting. both investigator would end come a endpoints So, points would families, patients or or the we Those

evaluators. there's different of So types three

they're is with We doing. and the can we different will evaluators the synchrony looking how think between of we hope at these at So different this be endpoints. look how drug a assessment robust

see titrate at be we'll are see and point we point would get actually dosing that that of that and the clinical cohort we forward. a where moved we expand beginning this to And add from patients program we to regard our separate new of with achieve substantial would established level a more data placebo patients. at we kind that and efficacy and at level. close dose to So sufficient are until if to we If trial the that the benefit in is level, we dose, we look had that debt we expect

deal higher we identify to Now we got midyear efficacy sense the later try That I'm get could you cohort a how help or see The that we expansion be the tune results idea idea regarding are up older But dose be and us or we we And range. we're dose have guessing that we're the and that by we're is asked, a year not of manage will something it to dose. with young safe a how going really learn to the and to allow with consider what the dose seeing anything dose what idea we'll the verify that going to know for the range all to within when to dose. a do. it it about but encouraged lower optimal could the be is else could risk in But so event. would dosing extremity far the of an level. at dose, is.

into range move as seeing we're think encouraged we and in work need activity we the -- we deeper just we're so, And to forward. therapeutic the

So we're with excited Angelman. potential about

Gena Wang

much. very you, Thank Great.

Operator

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

open. is line Your

Tazeen Ahmad

you taking Thank for questions. guys. my Hey

what to us about five Just yet, for drug? would remind What you Angelman's. able be If would more then patients decided agency? part are where get of just need been market market the discussion currently for that them synergies between not that you original it's out you had -- products detail redosing? to you And yet and on know Evkeeza. you can if comfortable of or to Thanks. talk to Can not one be going HOFH? little secondly, you to a that you the you is those point don't build Emil, clarification bit with redose at the need will whether about the with

Emil Kakkis

Very good. Tazeen. Thanks,

So in interested redosing for Angelman, those we're obviously original very X.

that seen those away has are them. because, course so pulled much tough families So they and for had of been have

they're anxiously so waiting. And

method look the in at to we belief program dose from and back ongoing want Our agency. safely. two order the to is it dose the right things verify We collect to we help data go striation that now just to can need ex-US to show that’s to and help

and year response -- world -- are that year those back and is doing hemological programs I on in which agency redose issues redosing. The US hand, that expect this original essentially all eventually to the would second of which dosing go things at and middle is get also get a of elements them no concern And think the patients those some part show to two we'd will in on hope that on evaluations ex-US was the other we thing hemological With to of in we'd the program treatment. plan administration. seek aligned to the terms to there

order next of those in the So, to of five the we as to these on patients want evaluation well we have from data some set the of think step as redosing. to get additional next

country centers with excellence is Evkeeza, management centers on very run So, model elements. common some very is have have are number a rare centers. of common. is knowledge government. people the go or But small always number this detailed limited and defined thing. piece by a that in out like centers And therefore kind are really to the the a orphan disease and rare of field manage Europe, There a particularly key it easy certainly of launches reimbursement disease leader In very there products is base which medical of are --

that have So we're us as go number centers. of we'll very limited basic that a us supply are bit which inter-workings those that to and growth to go we of a well revenue to we would people will can that in gain medical comfortable fairs countries things the currently reimbursement very as thing will will footprint will enable which more countries than are. also expand for in is more The allow to Europe our Evkeeza happen use

the think up gain will where so will that's have we rest will set the And we from which work for do. Evkeeza the benefit of the programs us other I actually we with

is move it. is be will we genetic is leverage of capable we and while doing team disorder So, Evkeeza a different who working but we'll get the be from we're we Evkeeza. growth Remember have of metabolism benefit think well on still involved, lot may a and some there metabolism fundamentally doctors that it HoFH can our lipid can And disorder. a to comfortable team sometimes

not disease as about this more a area The about areas other well-known it's be like of thing Everyone common disease it's because I'll diagnosis knows point knows hard Everyone a really might out rare as is right? some challenging. not such It's LDL. disease. disease mysterious which

find if a is centers at on and Evkeeza. commercialize They we fishing set of patients just is known. be how major the drug to already and in are get it lot people them them they're Here and won't take The go places. we'll up challenge the

Tazeen Ahmad

Thank you.

Operator

the with Thank you. question Cory next comes our from JPMorgan. And of line Kasimov

Your go line is open, ahead. please

Cory Kasimov

to can learn change chose various is it update might second Phase or just from then I thanks do get talk Angelman, of plan and full-on we UXXXX or dose and cadence you. it a follow-up coming for for more dosing think contribute the X X? questions. and me the in potentially like topic. for XXXX? like we here? these safety efficacy for well. Two afternoon around cetrizumab good Do year. from the first about Evkeeza of taking doses, guys. the talk of just a a Hey And what anticipated guess we LatAm in you the the just this little or Can you half XXXX, should as more my bit update Like Thank the more a on online Canada you countries about as about Phase

Emil Kakkis

good. Very Okay.

is the in go we this And to process obviously But consistent launch region. through usually countries and Italy achieve to these filings try there a moment. And probably work manage like get things the to Germany that going people other you the I by to across pricing and have at quickly, it timing work pricing France, through. through through of but that sequence we're and more through have a process to can wouldn't Evkeeza work So country country. to

point and regard still generator now to right that going filing requires ultimately course But the And in still would this approval and process sooner. the and XXXX the also some countries. through in country now. would LATAM of won't launching you spent getting beginning this Germany of LATAM, obviously growth. we Canada be revenue And be will in reimbursement With go at of I one Canada so not it can something So right its primarily is Crysvita do to And big these launch plan. LATAM filing in it's also reimbursement is and be in year. is beginning our a Dojolvi. And

plenty rest but for fall after to get do again Europe. the will So more year about they in there Evkeeza and be And World of we'll will this for But have that. driving filings Evkeeza. the

UXXXX. was Now for next it just Angelman both and one both it UXXXX was the or

So point.

Erik Harris

Just the one for

Emil Kakkis

fine-tuning It's is is Angelman know. test a like need that's story. kept -- and The already of the not We look all I you UXXXX, per -- to We're dose they really hearing the to well question to And quite it's works proved higher than of is don't kids going and maybe probably Okay. so pretty so compare. all. that kind purpose time going now We we that dose kind we're it. XX. kilo that XX the know a dose do right? go higher simple meg we're know young the

do so five-year-old to figure XX in XX between. to out old and And ones maybe is somewhere maybe we're is what the needs in try and going are

distribution create for to different how do going just pharmacodynamic we dosing effect look algorithm. age than we're doses So kind assess at of drug and

less hands correlation. would in optimize to in of age. the study it And algorithm think our to how as dosing hands the which showed or really a is based a least to on at I showed PXNP good

to doing dosing We say what a data because blinded high at level probably talk about So we're it's not our of we're We out that's kind significant in middle the provide put We'll expecting. great study. here's algorithm. of dosing. detailed would what amount a of data

how be And so figure we're to provide. update enough working. we program. much detail be heading into But important can I we'll would then have the dose X. an some it drugs restrictions And out to and on think say Phase going We that how should for we're

Cory Kasimov

Okay. helpful. Thank very That’s you.

Operator

with Raymond of from Thank line comes the you. next Chris Sandler. And our Piper question

open. is line Please Your go ahead.

Chris Raymond

that's Thanks. I've non-Angelman one. okay. Maybe got the Yes. a also just first questions couple of if

the you've second Regeneron before potential of -- that drug drug the second Emil potentially setrusumab. Speaking I in deal, highlighted a is think,

went with I Just end just was that expand the at guess And SAEs if safety it a -- signal. maybe when on seems guess deaths some just maybe developing but were the in guess, it's of not clear Regeneron, with into And could a were but treatment-related this of they I just path ran kind silent on digging these question I issues fatal see on on there XXXX. of it some Cory's then to into forward? Emil curious still be you setrusumab. Maybe follow-up molecule what terms you that they

just maybe that or I've next guess? think be as the that treatment. might big a up thinking just you of compare little catalyst that bit high expand setting as of in you in year that And I that kind when level XLH greater terms the this a I maybe of end see? the heard market the about you a to opportunity year demand you past opportunity this you an in Can say as even on framed for

Emil Kakkis

at the that going comfortable people is cause with what we're to not they obviously And is program study all look Setrusumab their those drug the to why conclusion is, Sure. drug have I The very and very I details in they very their advanced that advanced our sick on. What of are the can the need have and of Regeneron in some who may is advanced. that defer is patients were events. had say just disease we scores both. in we evaluated

confident confident profound feel through so not that disease We talk but I what get them are horrible is say disease all, drug is it's effect the you on we going right development strategy. should the that to can to so work approved. things. refer good would there and But developing look on to I Regeneron their doing them development a that at from product our in there's now, strategy is we're and And

Xs. Type Xs patient and as X OI as be Type well Type the can both setrusumab, that For treated population

and have be if a Type you degrade. the types; at look Now can there Xs fractures variable

Some some than occasion, have more people have occasional fractures more significant.

with wheelchairs kids, X and not devastated They are now horrible. X no patients And there's Those talking right? and compared is advanced end patients devastated and So XLH them those leg. are physically to a more far is Type even of Type are more right? with let's fraction kids These some being disintegrating. X with to whatever are X about say done close in bodies, child really right? So Type the treatment. issues. We're are significantly patients than bend doubt having are just crooked up adequate are bodies nowhere impaired their more

And XLH been looking OI XX% the So a need among population. the sorry the very last that the there's and potent better. anabolic has think in in -- basically in very just [indiscernible] spine the strength other biology with collapse agents of year density in is impressed more in patients agent one what drug high And models. is we've normalize is years And I'm density few we we've had sufficient they're bones X% This been than is any the improvement bone for lumbar to the their line. in study places spines essentially disintegrate. of the drug improvement and one double in which anabolic bone showed which X dramatic in and X disintegrate. Their lumbar Type to the

profound for So to growth of strength lumber young achieved one then to future stabilize could patients example to declining change would that constantly for and function. our improve bones spine post-improving want function we from think the be and and broken

generally big KOLs XXX% to more driver. talk that -- in to need they'll larger that patients than you given they the XLH severity believe top OI XX% So patients clinic. a their of of KOLs when on be we're we But especially unmet

to OI by we a you good and one. add a we're a a why could more significant OI it's population this in XX,XXX amount. prevalence could there's about XLH XX,XXX new the needing so high population. But get think if be same unmet there need needing on that. that position vary. higher that's that There's drug our than with help than We with And think a And excalation population it

Chris Raymond

Thank very much. Great. you

Operator

our next line from Cowen. question And the you. of Werber comes Yaron Thank with

Brendan Smith

Thanks for Yaron. This is question the guys. taking Brendan on for

I America. understand choppy. of pretty a be was impressive one bit quick quarter-over-quarter it the a can North in one obviously But Just Crysvita. jump rest world on

you we GSDIII drove what half. how see in forward want think in then maybe What so the about expecting said second patients are much QX readout by maybe this and one and expecting kind it on moving just of the will the data that if Just just in repeat then? year? And quick could context I should you oxidation. we we or maybe how Thanks wasn't from, to if be sure a many dosing get data it

Emil Kakkis

and Thank the you, I'll Crysvita Erik then Brendan. handle question quarter-on-quarter let handle Camille number can patient GSDIII you. Thank briefly?

Erik Harris

driven of surge was -- the lower going activity where strong lot a fourth some Delta volumes midyear into with was I the -- offices. had a think, barriers there we we quarter lower -- patient So which the following in by

we part latter coming that of year we the rebounded surge of variant. at Delta out a as So saw the

Mardi Dier

of on -- half have we XXXX. portion doses Great. name open-label the cohort. as the the of Thanks question XX ascending program. in escalated second eight study data to dose data will for GSDIII be by Approximately our of single Yes about the will UXXXX patients after our the suggests

from blinded portion clinical will initial foremost as well The we'll have randomized that and so dosing information of additional study. first the -- and some And and on study safety blinded. of the be portion pharmacodynamic repeat the as data activity

the you won't an where have half on portion in as second update been the give has certainly completed but until stands that we So we'll well. of data year portion that

Brendan Smith

Thank Great. Okay. you.

Operator

question from with And Jefferies. Maury next our Raycroft you. Thank line comes the of

open. Your line ahead. Please is go

Maury Raycroft

enrollment US And dosing do your US update? the plan you United during on what on the the the Wondering the or be patients my could time the and a have US ability going I adapt expand Congrats I Hi, How progress. the in of four-month data Angelman. for to midyear guess dosing States. there will one in two frame? enrolling ask in at on study mgs the update before like could taking to you was thanks questions. cadence many look

Emil Kakkis

four I The we're -- enrolled that planned for essentially enrolled patients they're patients the mg. are two essentially were think so --

already then an with there. we're started get that's and we're So And to to with and get -- started. FDA go them letting agreement planning

the yet. get have protocol our to expect middle was we're of current from year. to by towards do know the US of set that that to the much update enough would I I data to the would as we think can plan try our the don't changed data planning the back ex come US, through update somewhere, from program. to the look what the US But I take and for agency patients. we to

their we go agreement. the crisp agency a package back to So very that get with and single

So other go information that back once in US. we'd ex the align and hope the the with US have we safety protocol to

have they probably the -- to with that to in participants the By the cohort We on as of doses deal we're mg. in expanded we two patients doing. probably expect the who point but are enroll them in protocol next have would their completed that

you but to midyear. tell exactly that So can't certainly to to we're get try we going

Maury Raycroft

Evkeeza, And guess, wondering option. negotiations the I'm I a launches question may helpful. exclusive had are quick factor for it. with into the related Got with near-term FOP That's on FOP I Regeneron. just there obligations option, to if that then

Emil Kakkis

in our and we’ll they on agreed on aspects on share are is need really to option with depend that opt for they that's have just start we'll other. at depend to. Evkeeza, setrusumab. then tied will requirement certainly, Well, what the the is. have in each agreement will is and to not option are execute on of the provide There plan doesn't information that on development point two ability to all and have things those, our -- costs But performance The the their we in -- time, of some what to

this the are each things other point. to -- related not So two -- but and at

Maury Raycroft

Got it.

questions. my taking for Thanks Okay.

Operator

our Ha question of Thank Dae from next you. Gon Stifel. And comes line the from

line Your Please open. now is go ahead.

Dae Gon Ha

that be Angelman driven, get for going but more it setrizumab, I to was Thanks consider ASTEROID you Evkeeza, study. the remind your wise And can on you approach ANGPTLX take how just bone you. fracture on in One, measured not could gene secondly, I'll powering or you're guess, targeting that commercially questions. from also was I you us went tonight. if before seems after versus then and you're taking whether afternoon. of on Emil, wondering in our stay density that thinking I terms the with Good Great. Evkeeza. away editing Thank

Emil Kakkis

Okay.

gene submitted other commercial is side, but ways knockout exciting, editing on talk And very to years through. having the the which in there's gene get trying but, obviously the So, to have not to at of certainly strategies the safe, going But is right field. watching about is this ANGPTLX, to just antibodies we're in first very very patients, to goal take works this do few editing treating gene editing. The is people me, now hand stage well.

them take company a to advantage of have that ourselves. likes not but We’re develop to platforms,

experiment early-stage program a with, to another not was that needed if road, your we honestly, spend we few XX it the ANGPTLX, is, really be the very is while. years, few few a number. point the what money worked. And Evkeeza long. that that something going how could this Could maybe. editing on to product people sell but treat or view, our gene And in is years, in it So well, replace But could down to next are for all

can trying do now feel right figured comfortable, works. yet that have convenient. pretty doesn't the It So issues But genetic a to program are bigger if happen. unknown that that will It's it point be out. at some sell maybe well. we now to is they're any we market, Works

of is down. gene at watching this it in a just ups place and we're like then and so, editing, And time. But in there comfortable we're point our all good have portfolio you for they'll

not sufficiently For their FDA mind. considered by correlate where been UXXXX, didn't clinical bone the have There moral well. generally density situations it is in

with think I better correlates anabolic Although, with agents outcomes. it

of be bone and in view will be big the from that requirement the So work. moral fractures But have higher we on trend to seeing know the ASTEROID the the -- was enough in very were it density improvement that dose clinically have clinically our was comfortable feel observed is fractures endpoint. primary readily we study, in was would their it of important that patients improvements data we're fracture be and trends didn't there the would What time. and not that to

respond -onset ASTEROID powerful And XLH that's efficacy Adult bones like that out got remember adult would we can Same bones remodel much thing, more and is I'm of respond build Second kid Kids remember, don't faster I story. UXXXX. and group bone for peds true study was very be will drive faster. that in adult. than going with the we faster peds in greater, fractures thing that adults. and bones. much to you why, even confident improve And would readily that

way we're pretty kids That So was are powerful. and comfortable. that's did we work why faster more by in everything the true

into to actually see kids months years But it some. the kids like I In was in take fact, pediatric in nine of two as should That bone got XLH, went right? pediatrician, because originally That's unexpected we only, they as change a get why remember medicine to people benefit opposed said better. but to in XX% I

Dae Gon Ha

Thanks very much.

Emil Kakkis

Thanks.

Operator

Sachs. Thank of you. from comes with Salveen line And the Goldman Richter our next question

Your line go ahead. is Please open.

Elizabeth Webster

Thanks Hey. on This question. Salveen. our for is Elizabeth for taking

Crysvita just from the America other XXXX versus could your come on comment if world North you you of breakdown rest the wondering expect guidance, may geographies? For

Emil Kakkis

we breakdown past. I allowing to don't in what want the Well, the don't don't there. of from I make comment. the purpose if the we've sales But to know you has room he to to generally, do do Erik, sole Erik made breakdown that think the get

Mardi Dier

from in. I'll numbers jump and then comment just you bit the and a just can

Erik Harris

right, All Mardi.

Mardi Dier

Yeah.

year Latin So we and coming it's about other were from Crysvita million I America North although product press mean sales, territories that release down, in break put $XXX the we was last million. $XX mostly the that America didn't but

-- the point within So of that the that difference gives magnitude guidance. you

come more? numbers see if Latin growth don't strong from at the continue want biggest to to anything Crysvita know clearly So growth generally, America. North largest you But the in in we I very America. Erik, add

Emil Kakkis

what Exactly say. was I going to

Mardi Dier

Sorry just.

Erik Harris

Perfect.

answer. your there's Elizabeth, So

Elizabeth Webster

Great. you. Thank

Operator

next line you. SVB Schwartz our question Joseph comes Thank Securities. with of from the And

Your open. go line ahead. is Please

Joseph Schwartz

is on conference, Dr. much. by Angelman past saying [ph] question the very I first last value At the this preclinical my not. is emphasized second Thanks I'll But year conference, at preface guess and the models of late my for FAST Angelman. Dindo

and in in so, model. response studied pig to if to I if line working observing so patients I model what that did what you with any see the the be with the dose value you're out And particularly wondering And therapeutic was GTX-XXX in index of and was GTX-XXX. wondering And if you've of pig respect then now. and is

Emil Kakkis

Yeah. -- pig Well, and was more But A&M Dr. talk recently the Dindo Texas school the drop. animal of would about ag models it course that being is.

the So They're -- we we we're pig. the pig. now, don't in the They done developing have actually but the in got have [Indiscernible] now it clinic.

front. So we haven't gone back GT with of pig the

really So those I answer couldn't details.

stranger strange come where pigs humans the a it -- are Angelman of because acts doesn't I it will to up has anxiety. like to interesting strangers. It up think patients those have to is pig come reluctant most

additional of out fundamentally improvements whatever these what's interesting, to helpful could the it that human but or But is be happening. deal So other approaches. behavioral terms work, which the or molecule monkey work the pig developing in types done to retain, are to figure haven’t which subtle other do got yet. there's with patients But or truth patterns we I you are of think in

biomarker maybe developed the out pig, figure And the have so we'll the but the a completed clinical we also in validated already be time by program. story is biomarker could honestly

the in for do on that now but you're find we the expand is future will clinical right models program. think help truth us future, have is history the our clinic. I And dependent the the the going what we options path in don't in So the forward to

Joseph Schwartz

disease hear Okay. taken can your sense portion points. study given I of us the the into have we seamless a might of about will dose then you pivotal study? when we'll something that or effect Wilson the decision the and data be was wondering first And if a give

Emil Kakkis

Sure.

the -- of We study the be didn't the to expectation But the won't assessment all will start program, have. interim program. it's But we we our of an have because of disclose blinded now. that able data just starting

this an get and is able be not that are the So to time because time. markers of is now where there be are looks expectation what to on But the and we impact our that drug working line year is the we in like that to dosing interim importance proceeding. response that choosing going dose is of to know a the

about we have So because it, the should dose that's of granularity drug the level to damages here the Phase of be proceeding we're and of you X. have to to and be But the is objectivity study. provide just don't X the tell it careful we'll to probably want kind Phase enough that anything

Joseph Schwartz

you. good. Thank Okay,

Operator

Thank comes you. our from of next question line Truist And Securities. the Joon with Lee

Please line is open. go Your ahead.

Joon Lee

taking for thanks question. Hey, our

your dose. or midyear? milligrams, between you your And by Canada ability the get time the before a on and the XX-milligram the XX you sufficient have definition all UK studying midyear, dose is is all to top doses of data? to patients patients to maximum those and have you what Do data dose XX-milligram Angelman the have line to goal For few do skip

Emil Kakkis

really QX We'll annoying midyear of QX interesting. broad is be to it's with you. Very enough start to all or the the last

we a it of can escalate months So is three truth through executed. have number the they then after the maintenance every phase study that pick But further. steps escalate that's why they get way. the a protocol The we and and get to doses ha two study during

of to there. However, we were if getting XX will to a months number get take

protocol to is does, just doses. in question we So jump normal your can clinical jump not

June expansion higher But level started cleared four higher let's we are the jumping at say dosing dose at midyear. doses to we as by dose of a level. an level get data have this initiate load start and look where instead which to data and level we that going And so is that to allow with So expectation us cohort, our far. to we're at to dose a now, see at would now at

what get if we're to higher -- doing our ahead. and dose jump June, allow continue will at that will further the us efficacy we're to be bit allow you're at a by we want to by us getting time jump a that little to it midyear not the where and So at

the therapeutic through and work window safety to have window. the establish We

us to patients. I the a there so gives think figure what We to efficacy just dose for of we've think window. we get meaningful will out the is seen take would therapeutic that far need what be kind

will dose, titrate information going there will we're up and We'll rather the that this jump we'd that jumping year X. by have those expansion which run Phase clarity us to between then level cohort, somewhere later an all than add and to expect dose another will hopefully that So of what give in continue. in from

Joon Lee

quickly Got more there any meaning then prespecified or is are no And the it. Data planned there for Monitoring Board Safety

Emil Kakkis

or of regular happened, the continuously related safety That's have to the but enrollment ongoing. on those Well, because a yes, the they meet need to evaluate that's two rhythm were further cadence, enrollment. prespecified how expansion they will

they Of course, is an are if ad special goes yet no And meeting notified regular it's doing. a going But there's that a the that there a except and otherwise, of be they meeting, lower cadence triggered hoc extremity be a would cadence as regular then event. but forward. to happen immediately have

Joon Lee

you. Thank

Operator

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

go is open. Please Your ahead. line

Jeff Hung

Thanks for taking the question.

For partnerships, Evkeeza, further the considerations geographic beyond were what to for the how important as expansion then And Europe. is indication into the expansion? potential APAC you and there mentioned for the such

Emil Kakkis

the because and well, in trial there other territories the that regard in as to Regeneron I countries all the we think, that are support considerations, So places. down patients in commercialize we that patients importance was are all Africa with South in for the some

we'll a the that Japanese actually this approved be to So wanted to commercialize country. it positive programs it we help on on. has broad requirement in Japan array we course, a tool in there's very, data the go the countries Asia Japanese a to the want of here that into quickly. patient is to get any And support, of involves and and in any study very patient are us Because we working certainly the going KOL go Evkeeza and

should have Japan potentially into in filed package we Japan already So that a that's going get us earlier.

our And filing. so that will of be immediate part the expansion of

it's to something with set which have So the drug worth I we'll making into talking it's that time option, is With I the them patients. have would by where regard that that's that get important effect detail. a at us say at development is think most and I understanding they and point, profound set -- and so point, beyond the a think, discussion really doing program and I looking going for about data going more that is would sit patients going want current the don't it's forward. very would And treated it. And these profound about at with that to think this down would

we're we at So move point time. encouraged would this expect forward and in highly to

Jeff Hung

what the the follow-up to Latin for America, on further in growth key year? America are a Latin this as factors then question And

Emil Kakkis

America story way. always a have been is the great we Latin Well always team -- by the has

best gotten I think In disease that The of the done have through has been reimbursement. now politics team this COVID final always regard Brazil, that and have at Latin some programs. reverse the in that the America a the process step. challenge rare for of people of We we've well lot and setting pieces to important the look one

there'll reimbursement. set begin In will adding in we'll certainly patient So Brazil other degree sales price the expand be once further. working and we're are filings and on growth on -- want we're point? and there to be on traction that can anything know I a ability working happens America. in else at then countries, the working the Erik where add we seeing But to there's to Latin now story might you don't growth America through we if this Latin

Erik Harris

community and No as I to expand patients America particularly we setting. as into in we continue did as with reached find similar more US in the to North further out the just think growth we

full can and full Something you. reimbursement go similar in Thank activities. especially, we will into occur we as commercialization attain promotional Brazil,

Jeff Hung

Yes, I think that’s so. be to important going

Emil Kakkis

Erik. Thanks,

Operator

our And ISI. next of line question from you. Evercore Liisa comes Thank with Bayko the

ahead. Please is open. go line Your

Liisa Bayko

Hi. Yes.

but one on Just one one And just question. quick maybe answered, Angelman. my been most quick of financial questions have

you in So of you through the a good rate just us -- Is that was can especially for this walk us year? to about, bit. up shape give year spend parameters maybe a guide your just us or thinking think the R&D some run Thanks. about just number for I R&D. can of

Emil Kakkis

there Sure. has is spend as There's There more. in been some items the some onetime in too obviously it increase mix. have little -- a Mardi described bit

So why give sense? don't you Mardi some

Mardi Dier

cetera we the $XX as sure. continued the out much now R&D But there. in in gene next we in than I collaboration our upfront focus stay expect for R&D see think programs mostly have the our growth will our to in year million going Europe late-stage for with reimbursement therapy multiple same. development the would highlighted work the build into would in line and say We we'll the other the pipeline Yes. do on we countries growth that's right pretty XXXX Evkeeza some on et But for in in

Liisa Bayko

looking things with you're then And of of looking Okay. help and any the respect just Thanks. like to would telling kind other or metrics that like you at? of guide on you're Angelman's. there onto sort to that at biomarkers Are . that be indicative

Emil Kakkis

-- because it UBEXA blood the biomarkers contamination all, soluble of a presented UBEXA first UBEX were fluid what Well, fact probably people as in we Sure. like spinal -- in cells. the there's was thought blood

of in power an patients patients to now look at was the neurophysiology a turned not detect and so to more may is a at amount UBEX looking And neurophysiology kind and leak of the The biomarker. the neurologic is what tap means bit to there updated not blood the those due they're in you MLB read of spinal probably I'd and Delta going Delta the and what thinking on or in is fluid showed of at fast that dependent we epileptiform that should it it little change looking be directly EEG trial find the Power showing EEG. were at which But out data they're doing that look meaningful. that trace whatever function assessments the in patients guys on EEGs detailed that's

would neurophysiology abnormal very is shows patient would it think this the to information before. The subjective fundamental look evidence. you I are probably So tell you to just differently the best And a biomarker type these neurophysiological that as happening stimuli I than or something at being not they you did brains respond to is better. and starting that

Liisa Bayko

Thank Okay. you.

Operator

of question comes next you. line Beatty from Joel our And Thank the Baird. with

open. is go Your line Please ahead.

Joel Beatty

taking questions. Hi. for Thanks the

potential Wilson's about Stage question X disease the typical of learn in for could the First one on then to to outside how US. in part any second efficacy is fast how might is drug. sales the SKUs on After ramp-up much time of endpoint? otherwise rate And approval at the compared for the is trial, there be fee that what diseased a neuro

Emil Kakkis

Okay.

first or question US about was something Evkeeza the outside on the else? So

Joel Beatty

Yes Evkeeza.

Emil Kakkis

Europe little hard Europe, behind growth much a so going. other getting countries this it's the going I set going for guide it's wouldn't about – launch And the in what and regard to of are filings that I all to will is able I tell to outside and So them, for work be think and to be. to be that U.S. approvals how is couple mean about going. But of year it's going But year be the in sales that's to to going are it you team yet. reimbursement would a somewhat us about this Wilson get reimbursement be the on. take focusing there years scales Wilson expect ex are neurocognitive an program. dramatically With is some I to and

that a lot the have not abnormality remember However, the patients at beginning. at of may –

So were abnormalities. hard difference you would talking about the that to be people Trying to do score dosing. do on have their determine

the ceruloplasmin the would highest it's to all that control and going would if that should dosing on most forms same the give is going good – to focus is the you right assuming highest if say data levels, we're does really and be biomarkers say there's up dosing. And to and I dose. on the the to end safety So I to highest would eXX control things which gives answer it us the as likely of equal, the that, us think copper basically dose likely safe copper various

little we because have a treatment much me assuming why rid be to gene copper better stages that pretty more you're range of But first that's so this is is coming the variable eXX mystery gene are work low expect It focus that I of copper reason just and distribution, fact clear achieve. necessarily end that's once man, you – enough there would that And that to know three and that real we'll why detoxifying, there's should harder distribution. everything copper And one copper of get see. doesn't that get in And shouldn't that restoring in detoxification real homing up the from you would which the occurring in excellent else. eXX not restoring dose the we we're getting be copper, be you the therapy we that toxic about we to two than want it's personally animals, because eXX that that the could And which range safety level the going setrusumab actually higher, process, right? through dose. actually the you're the on But new a the part therefore, would over therapy be of some story to being do doses. benefit would

Joel Beatty

Great. Thank you.

Operator

with our comes question next And Thank line Chico the from Laura you. Wedbush. of

ahead. go Your is open. Please line

Laura Chico

for Good on squeezing have I just Angelman. afternoon thanks and in. one me

case of about you're sites Assuming wondering could a towards and talk in of be on execution if what would I'm Thanks. best recruitment studies, kind the advancing U.S. the scenario feasible number pivotal you the side,

Emil Kakkis

a lot loading would U.S. to are than That's our be in are but of think that loading and with I number frankly. would six-month XXX, -- be a Because lot somewhere as effect over not sites, at question population just patients data great can XXX of And manage where to look but X anger going that intrathecal I focus. less -- smaller lot a then But the our but patients a only Laura. and be hope That's of going there. the to an trial services good that a talked of study. in study a as And administration haven't a number the lot from set therefore, patients really about fairly are -- we question for say than just hope run just huge that is able patients the there. with usual would a a a required tendencies support study need clinical promising the drug study we're be there a to difference very And sites There more if assuming going interesting clinical period. run we've we based be, studies be it's I'm the international we centres have will ability are of sites be size who Phase our it number, to we probably will study enrol they a of of on be to large a show would treat the most

patients. now in of But lot of is successful. in X So in there's be patients issue be question the I the So could showing ongoing. sites? sites. are on patients, important like to in a or Angelman of also in do there any Phase will we I'd think venture is point I programs competition other be Angelman I But time. number and don't competition can't exact then what the even everything there'll think at more I asking drug number this with you about tell our if that come moment But studies that our for there programs think are Well, guess patients enough out, safe can a and if enrolled else to going you're context there's get other there for right?

Laura Chico

thanks. Emil helpful. Very

Operator

further remarks. like the conference And question-and-answer to turn Joshua does over back for session. And Higa would you. this to any conclude Thank today's I

Joshua Higa

by If ir@ultragenyx.com. us any questions, please contact there us joining This concludes you. additional for are today's Thanks or phone at call. Thank today.

Operator

Thank call. conference This concludes you today's for participating.

now may You disconnect.

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