Ultragenyx Pharmaceutical (RARE) 15 Feb 242023 Q4 Earnings call transcript

Company Profile

Participants

Joshua Higa	executive
Emil Kakkis	executive
Erik Harris	executive
Howard Horn	executive
Eric Crombez	executive
Huidong Wang	analyst
Maurice Raycroft	analyst
Anupam Rama	analyst
Tazeen Ahmad	analyst
Dae Gon Ha	analyst
Mehdi Goudarzi	analyst
Brendan Smith	analyst
Kristen Kluska	analyst
Lee Hung	analyst
Jack Allen	analyst

Call transcript

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Operator

Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2023 Financial Results Conference Call. [Operator Instructions]. It is now my pleasure to turn to Josh -- turn the call to Joshua Higa, Vice President of Investor Relations.

Joshua Higa

Thank you.

We have issued a press release detailing our financial results, which you can find on our website at ultragenyx.com.

Chief call Harris, Kakkis, Horn, Chief Officer; Eric this and Executive Chief on and Erik President, Commercial Crombez, me are Chief Emil Officer Financial Joining Officer. Medical Officer; Howard making remind to today's will that I'd forward-looking like everyone statements. call, during we be results These actual to and subject materially. are risks and differ statements and uncertainties certain our may refer to SEC latest filings. the Please risk our factors discussed in call the turn over now to I'll Emil.

Emil Kakkis

programs our that everyone. In set XXXX, generated commercial Thanks, and Catalyst and across clinical for good afternoon, up Josh, have significant us we XXXX. momentum bridge

put we On the add our maintain international commercial positive expansion our negotiations reimbursement robust us across of approvals trajectory markets growing new marketed front, reimbursement growth. to in progressed continuing position with regulatory This geographic in along and to products, decisions. year-over-year and our demand to existing

in radiographically XXXX. showing showed fracture and just patients the osteogenesis XXX at UX Phase II analyzed with of On key side, our treatment. and of real XX interim months accelerate in our we this our exciting new programs X XX advanced shared additional fractures Analyst the no a data October, data priority data company for programs reduction in value programs the on Z enrollment we to X in substantial three years. At catalysts successfully XX% will can the bone coming have On from all that for our in and after clinical of that increases imperfecta, density Day confirmed score released months. creation in data led XXXX This year

tapering dose syndrome, their UXXXX dose year including fleet. therapy. for clinically X standard improvements Phase completely much or than key communication, are zinc data motors, cognition, Angelman across show repeatedly begun disease, two data including the multiple cohort patients presented longer GTX-XXX and the lowest from On cohorts in demonstrated domains, long-term profile. care, acceptable meaningful data safety can behavior the Wilson I/II receptive growth who X showed have we with off neurodevelopmental for These study GTX-XXX and/or On extension we X letters of for

While have UXXXX the are data observed. for still encouraged program, we the by early we

continuing granted interactions need into week, will The development clinical U.S. unmet long-term data medicine designation to development by or to program. the momentum for clinical positive early European we've the this updates. the The based positive GTX-XXX is Last this data shared and with address European in give cohorts clinical GTX-XXX regulatory XXXX on with multiple extension potential of with priority Our the confidence for and treatment Medical authority was navigate the us that from disease. Street, path along the we able our be PRIME

improved data of Phase we in Diego. UX-XXX III A San released newly For Symposium Filippo The syndrome, patients week shared in with levels CSF correlated we and extension at treated Research the treatment San resulted cognitive in demonstrated results Meeting WORLD exposure from sustained last reductions Annual with in and fluid sulfate in cerebrospinal the development. sulfate time long-term over decreased of the heparan UX-XXX heparan rapid sustained was

Our accelerated strong FDA made a case and to an there's with ongoing, we this to in continue pathway discussions the on are believe program. be approval

will $XXX offering drive and sheet, to spend our continuing that for XXXX $XXX entering balance are programs we from that raised the [indiscernible] many company, ahead, proceeds on QX robust on months. million. the including in Looking in key also in the value We're our with position equity the a million clinical financial resources our coming focus

medicines. to to future lead his section. efforts rare of we of more to There's a Eric the Chief look forward Officer, Medical our these Our Crombez as disease in lot view continue will updates

Harris, Erik the update provide last revenue turn I'll XXXX. an Officer, year in that to Chief Commercial XX% to on to growth led Now call a commercial over efforts our our

Erik Harris

start in good performance that we everyone. the Kyowa promotion on of to Emil, and for will successfully afternoon, you, that growing Crysvita I'll XX, North transitioned with Crysvita to to America. Thank the April in XXXX, Crysvita's generate revenue remind Kirin our U.S. company. I everyone and continue want

the During to while Ultragenyx transition the find field field key team community teams. to continued from introducing accounts Kirin period, physicians, patients Kyowa

continuity Ultragenyx services support In the and reimbursement. from transition U.S. demand the to the for and hub supported Crysvita to the patient helped Our Kyowa field additional hub patients transition XXXX, Canada. and the growing smooth in ensure of Kirin of the resourcing support team treatment

continually a approximately we to year in reimbursed success saw team to forms were there transitioned fully Crysvita continue prescribers in support us Crysvita U.S. unique of fact, the XXXX led strong North and XXX America. services growing support generated demand that field from that for focused and another experienced team, be start Kyowa robust has amend and to therapy. our XXXX will Kirin a with field The this a of support In base on Ultragenyx XXXX allow patient we in agreement from further confident year our the in through Ultragenyx' will XXXX, feel hub from patients with

approximately with XXXX. included new QX therapy, commercial began Shifting reimbursed over new Crysvita in America. which of who finished to patients Latin XXX We XX patients in year therapy the on

nations Over our added approximately the base therapy. to year, on the XXX course we of commercial of growing patients

America. growth is patterns XX% expect While Crysvita patients seeing in is adult patients, by America, across underlying an organization global by the Latin we most our uptake our reflecting confident America in remain that increasing for revenue products. generated the in Latin we XXXX. These a ordering driven demand of million, $XXX in to XXXX in similar pediatric uneven pattern efforts the quarter-to-quarter are region, across increase which combined we but demand compared variability of driven current North In revenue a saw

approximately on remains start strong. XXXX, grow, In reimbursed patients in added resulting XX start The prescribers reimbursed For adding for the U.S. therapy, of XXX in number XXX approximately new in forms over patients Dojolvi the and prescribers XXXX. and U.S. demand continue since launch. XX Canada, in we the to forms

France, In is as we progress named Across in demand screening deepen National XXXX. Most are Dojolvi lack despite throughout expanding of Dojolvi reimbursed step requests the Mexico, for approved Compendium, important awareness Latin the Europe patients the are is HCA increased patient are EMEA Medicine seeing an more teams driven majority the steady sales recently key medical America, access in in in Though XXXX, from the by LC-FAOD by Dojolvi while current inclusion East. Dojolvi patients. and In which to the growth XXXX. $XX teams. with was region, finding continue region the the to an toward the revenue, we increasing our newborn XX% making is in of stakeholders of in for through to Middle which generated request compared the we coming million patients

on progressing reimbursement Canada Lastly, steadily positive EMEA, EMEA, and infusion clinical markets In HTAs processes. we FSA, provided working recommendations continue have with launch and Japan. across European through the our are in we

Japan, on accelerate the reimbursement. are population start for public In validates indication [indiscernible] for in we HTA Health to EMEA In forms clinical authorities. Evkeeza. patient the On the The received children from is with to preparing we expanded payers January HOFH. European with approval uptake approval. the have reimbursement months. older authorities provided expands approval the to recommendations the give with further The initial -- clinical the Canadian and expedite private for process is coming drug's Commission regulatory team been pricing years in meantime, and the and underway in bodies, since In December the region. on prepared discussions addressable board we value Japanese positive approval X XX, are XX has Canada, Evkeeza This received steady working on Canada's aged an both

by of the team launch to more the Evkeeza Crysvita busy and the XXXX, I'll revenue results Building followed in and call drive With appropriate performance Evkeeza. approval, portfolio educating financial strong reimbursement continue on Evkeeza-label Following to from the we will guidance. is benefit turn XXXX. share The across to expect Howard the we in our on our Dojolvi could Japan to success details treatment. majority quarter. patients XXXX identifying the next that, on and who continued physicians the in expect

Howard Horn

good and performance. product I'll individual everyone. focus with revenue. our Starting year covered just Erik, we on afternoon, Thanks, since full corporate total financials

expenses For of cost included stock million $XXX for Operating billion, XXXX, SG&A $XXX $XXX and expenses Total of expenses representing of compensation we reported XX% over noncash operating growth million $X the $XX were noncash million, included -- $XXX stock-based million. expenses million. of sales R&D XXXX. year which

net loss share. was per $XXX year, million the or For $X.XX

used and year-end. higher due was in of receipts million in which December of when In had As million, $XXX payments XXXX, timing cash cash, marketable than equivalents securities. XX, around landed net $XXX and was cash to forecasted certain we operations

used expected net which cash Our in is million last less than is with month. provided guidance the XXXX we consistent $XXX to operations be

We last are ranges XXXX reaffirming also guidance the we month. revenue stated

which XX% all expected $XXX revenue all $XXX million, be million, is XXXX. Total forms includes between and versus expected million million to Ultragenyx. $XXX regions $XXX XX% represents is and Crysvita to growth revenue which revenue of and to between to Crysvita be

Turkey from and Crysvita from and Europe. and North includes royalties revenue cash product America it the noncash America Specifically, Latin and

represents between expected XXXX. Our XX% to X% XX% Crysvita be versus -- me, growth to guidance which versus range represents $XX X% and is XX% XX% million million, to XXXX. Dojolvi growth $XX revenue excuse to

we represent key Our will our requests. Crombez, our where update in data CMO, we growth faster in name expected call I'll blend on respond where a the commercialize to growth to year. turn of countries readouts patient countries lower provide With an this that, Dojolvi projections and clinical Eric who

Eric Crombez

everyone. and good Howard, you, Thank afternoon,

that clinical from UXXXX DTXXXX expansion presented data XXXX. UX-XXX, data at second the be have dose-finding week, These to finally, the quarter, III include the ongoing readouts study. the data, to Phase cohort II/III of longer-term data of a addition data mid-first data and the GTX-XXX UX-XXX last In II number we important then Phase in follow-up presented part Symposium III planned we half, Phase Phase WORLD for

the pivotal Phase there from data read of the around end for the out potential the UX-XXX, For III to the first study analysis is interim also year.

if opportunity a first data met. and means the early that is alpha have strict This compelling the the the end are value interim while the providing this of for for primary spend analysis. X effectively We study is endpoint unblinding set p to X.XXX

stage DTXXXX. and the a few [indiscernible] the II built at occur in I'll also expansion the annualized clinically of Shifting of to study. expect III with readouts significant the the study The now domains shared we improvements a clinical that ASBMR data and us across last to event meaningful in the Day disclosed safety efficacy. the and understand to for included will October, share clinically longer-term see initial start reduction and that near-term fracture Phase and cohorts. in demonstrating statistically we dose Angelman final extension would treatment fracture study meaningful saw Phase a potential the syndrome in the GTX-XXX year of Last data have for confidence at gives reduction New the data, in the these we in next York, II in our extension the and that Phase Regardless Analyst timing, in we GTX-XXX cohort multiple We interim escalation patients for analysis second We for in that XXXX. study months. rate rate two

XX-plus enrollment cohort year Analyst at with enrolled on at was upcoming efficacy XX began been changes. higher We shown Earlier have trial. at in end further we study achieved focus dose data cohorts developmental year to the and patients was XXX highlight milestones data least in have that half completed a many to to in last of expansion safety days. therapy intend The these will first of these for evaluate III These loading doses a include of we meaningfulness patients Phase what also on similar We intend clinical format to the the last who to larger the expansion number present that a year, cohorts patients mid-first and Day. regimen patients expansion the ever of globally. of this dosing in showed

expect domain With the changes Phase These loading higher will presented doses we main meaningful study. showing for Day. verify period Specifically patients, dose and at natural inform by selection with comparisons the and data efficacy III no evaluation a that to Analyst greater history. of to number was

anticipate we later with data FDA planning Phase will the start discussions on that and Phase to end III up an Phase XXXX. II in continue Our meeting of well, mid-XXXX, will expansion the which in III go support study enable

the for is of type treatment DTXXXX readout gene Ia. investigational data our near-term next The and for glycogen therapy storage disease

data largest our first pipeline, III We this readout. the have be therapy will Phase one of late-stage gene and

to I/II earliest All therapy. see meaningful of fifth follow-up. expect Phase has statistically year their study, III demonstrated patients the DTXXXX reductions with response patients the a significant patients of and the the that placebo-controlled clinically meaningful randomized, entering we proven in with treated durable Phase In cornstarch to in clinically

expect to provide call of and study this We turn DTXXXX the remarks. back top data to closing some second line III Emil now in quarter the the unblind share from to year. I'll Phase

Emil Kakkis

Thank you, Eric. a expect. a catalysts, by of the year. across We've I'll this better incredible number clinical what us sense catalysts close sets for summarizing which of progress made data everyone pipeline, those up get to

top This the is we'll line cohort III share to As the from second expansion quarter. DTXXXX GTX-XXX followed study. in Phase data the Phase be Eric next mentioned, few by II the expected data in the months,

sharing Phase data X are III share first Phase complete recently track in Next, and the patients Orbit is This the enrollment be Stage we with studies quarter. the disease half dose that Closing well, end we approximately in the plan Wilson will of on X patient. last for the we imperfecta, from year. escalation UX-XXX dosed XXX the to with on data of cohorts the the expect in II around Enrollment longer-term to for mid-XXXX. study in the osteogenesis second data UXXXX going

therapies path to XXXX expanding our our more is XX is important The access more the expected year or on compact commercial for time and $XXX is after. enrolling to well patients is important and just more toward also to than or We'll to continue bringing these million an track in products, more enroll patients same and study Ultragenyx. global generate profitability. approximately support revenue around

from of questions. in stage four share updates one move programs, our making to your company's the later clinical let's data-rich provide on please also instructions. most We Q&A the expect history. that, meaningful to this years our of With Operator,

Operator

Instructions]. [Operator

question first line Gena Our will Wang from come of the with Barclays.

Huidong Wang

the in one question GTX-XXX for Angelman have one regarding and I setrusumab.

prime obvious approval Phase second inform Phase and So the feedback have 'XX. regarding And with year-end you for trial half first setrusumab, 'XX also the design? how will what II design? Phase interim III in the the And long-term the received is for path GTX-XXX, trial before the you met III early designation, will the Phase FDA data regarding for III

Emil Kakkis

good. Gena, for Thanks, [ Very question. the

of was designation So just of the we seen presentation And data saw. basically that we've the far. a prime so recognize data the close

plan to We on III will no have designation with a not the that's FDA. the FDA on European regard the have settled With authorities become. the prime Phase something influence to

a think terms our plan III. discussion Phase we and We've Phase data progress, of way our have we're discussions. of the We end for good forward putting FDA. had the have we encouraged when we conversations there's finalizing making comfortable feel II, them by initiated with with good together a plan We're when and hand but Phase about in II meet in

for design. we're III knows double-blind be planning a the Phase that And to don't there just with design. a that European traditional I be FDA randomized either to reference in XXX basic problem authorities that's around XXX said think have that. patients I design, trial the think We with should any and

after being we we that, XX% of I statistically further from us have up level us as It fracture -- all over see It So see the settle patients data treatment, have on fractures reduction And a any now true out efficacy time. a move already profound positive very endpoint a lot is setrusumab longer will period to the to at endpoint estimate might that puts about of good III that higher over longer effect final and study help a rate the a fracture I of sooner that patients. we'll we that position we would little potentially Phase X, us the have will in reduction potentially and end to rate But get time. significant before a a understand II the to in mean, XXXX. that we'll on highly put able Phase periods of sense we data. of depend felt there reduction. more of have the With efficacy standard, the results. still give we We Phase we'll in tell result appeared Since getting through us X, that the us level the few not very good position could variation reduction if and another were and more month X get maximum the interim good hit interim. fracture will in

the great very forward. setrusumab the So bit see And help little understand data about a encouraged we're how the about II us is and shall forward. it path in Phase more path

Operator

question. our for Raycroft with of line Jefferies. come next Maury from moment that One the And will

Maurice Raycroft

Based longitudinal far, on totality from study. your your data including natural history data the so Angelman of

As a you to Phase the about do and what you what a optimal think age is have maximum efficacy frame benefits? assess time of design, III could group sense show

Emil Kakkis

Yes.

So looks gain be anywhere Day, data at right? last study is And continued little XXX. XXX day to look XXX So day it put day year longer, you like And the data at looks we a around like is early. ground it much at our view from if Analyst XXX, over day stronger. the you going if to we've time, out look could

will for results. that data study, the Obviously, not right the [indiscernible] II what's on likely I'd a longer gives want us the time separation help to be time means It [indiscernible] figure Phase number. more out found that it do Somewhere needs the time in frame range meaningful is the that is, And placebo. more do the frame. to for in the would to time we try study.

In using good II, been age kids, we've the people is age, we're our the Phase too. right continue thought patients same terms of X- would older now, in seeing but early to XX-year-old respond, I the plan on, think in range only with responses young to

So to I don't order shouldn't need think efficacy. that that limit limit best in get the should we to

group we design. XX Phase that in to our whole think I X conclude III

the to though types study, potentially younger data open-label do kids, the study set. expectation other and fill older at look an generic separate is Our to out a help kids to

III main XX-year-old study, With Phase days XXX X- the expect between to XXX long. days be to somewhere be and we to

Operator

moment one question. And the Rama next will with our And of Anupam for that from come JPMorgan. line

Anupam Rama

Maybe just the What WORLD? feedback XXX the push-pull in on there process what the in accelerated program an And with program meeting. of update a was here quick one you for where me. at are discussions MPS levers the FDA approval on like are terms III

Emil Kakkis

meeting show being the consistent the with had the there, the people I in extended developmental really positive treated to data progression good sulfate That's is heparan predictive outcomes. Well, clinical patients. good we from of feedback continuing think because among longer and

so and and so value. of been number has FDA for using similar there's going showing HF a predictive on encouraged been people the we're And marker a while.

it affect area, benefit which if believe, predict at in a Peter organized as we other MPS [indiscernible], the solid marker multiple with We data the disorders, XX. February think will FDA I help than doing brain likely in be should to approvals which happens, move disease I there look heparan genetic that sulfate workshops encouraged And programs. brain. by particularly planned clinical that moving idea to start with heparan has should accepting of the been stand the the take encourage us and very Marks sulfate feel across will the reasonably biomarker rare forward the

disease the tell progress MPS-IIIA particularly hasn't the think others treat a data I single the a that's to good disease there we on that about workshop, released, handle have the data released So approved how us today. have forward on for trend we a MPS we're encouraged MPS and thing brain because been and

Operator

question. with Ahmad of moment next for Tazeen that the of our from And Thank America. will you. One Bank come line

Tazeen Ahmad

the XXX have noise. I for Wilson. just for couple on Sorry background a

line. time readout the changed You've

talked is Is from have that same how to composite a a FDA just ] [ discussions just data it the that including there with I had nomenclature time have that traditional a the that that first a and -- originally yours. might of the Angelman going the back we've a think reason endpoint. to that viewpoint affect their shift? guess composite been saying And a was endpoint about relation Angelman, around own question FDA's I measures, of as you're time includes but you lot question getting on main half, more slight is with endpoint? now it's recently to I and that around CGI, that for using And Baily mid-XXXX. was then think coming detailed the competitor And of potential using is we've

Emil Kakkis

Okay.

layers, just line enrolled and has at months layers. X key whole We and them enough to process year. titrate role. the to the opportunity their get last time time kind done on this the patient the last So We key early cohort least titrate give of treatment Wilson through have patient to an want

So but where different we meaningfully. not it's from off slightly from have

time if So sure that's to key to be want get We give enough patients they off their to the can. to layers able timing. make

be endpoints endpoint whether MDRI whether case traditional single will something with now or stick we press a just other of do FDA. that had they still Whether on think FDA. the using a in should ways two discussions to of GTX-XXX, and for picking there. showed we've primary. endpoints open want story to on they're ways to various think question we the that's I working looking looking, But many or of through ways. and both the we're we MDI our basic And flexibility With of novel a So I the at lot

the not They point. think efficacy behind is about don't of are us But at going really out this the not [indiscernible], I the we be the to outcome end our think And will change I driver I they do. it's efficacy I'm been points. been data has worried a that impressive. -- competitor put on what think good. there

in future our So some at I all it this team and I for And point, developing think around best honestly, endpoints, in support And think nothing our the in hands. of is it. have methodology able we work suggests and listening a anyone to else, in us on our FDA they're be this. that seem conversations with ready They to when to final on and the the willing endpoint. particularly solution

Operator

you. for And question. come of our Ha the with One Dae Thank moment that next from will Stifel. line Gon

Dae Gon Ha

on ask first just guess is, Emil, can reduction. one I cornstarch the shed the I'll you two GSDXa. some more on insights

from So the the is we've heard if some important, sleeping important, docs more itself. as night also through that not reduction than

provide you about Can address talk when the ensure training caution? And corn done what out investigators think to appropriately monitoring So the then sleeping versus about GSDXa. sticking reduction of of kind here, And cornstarch meaningful with you slower you of level is starch data than and these you just expected clinically the physicians? aspect the a for the reduction? outcome what will do consider to

Emil Kakkis

question the the and then with with the training question, maybe second deal I'll first could you forth. sites Eric, about so the deal of

a burden every horrible and day each we its hours. to more the the forget dependent haven't on live lives I what are cornstarch, with with Because with on because something die, actual for problem The is starch as problem patients reduction to than you X patient each a minimum a if head I cornstarch. agree change to thinking, take that cornstarch taking that, to So of is could is gun whether corn cornstarch we threshold is have the critically with important not make set survive. probably

study were So were no at what did night drop our X. have and we maintained to many know Phase blue not They patients is maintained. and cornstarch that to patients their I/II from were pulses -- able supported. They

night, So of we're administration Well, be be cornstarch have we'll allows tedious what something taking have? the at also also look take, looking us we'll whether We'll we -- statistical will they at. they're during number cornstarch at data at night, significance monitoring, the will control monitor which reduction. to glucose primary looking which glucose and clinical

it's dependency, feel amount of the of reduction So in the the who they where going they the part to addition you story. happen that's exercise can the they are safe like of now why characterization hypoglycemic will which hear to and think to night. And out to many transformed cornstarch, cornstarch house go we've feeling for about tell get the don't important feel not an drop can part had sleep is many important patients and at going I starch them. without patients And of seeing we're other take through the they're that And that somewhere. us an night

those to quantitative of are meaningful the So clinical I aspects data the think come all will reduction. to start what around argument out addition with provide help III Phase in corn the

maybe how can on a Eric, with sites. Now you provide more little the training and we're doing this

Eric Crombez

want participating a III. teams, of build in lot also independent did physicians who the who on and very as here. really GSDXa work study experts principal of group on Phase to gained for nature the sure to the clinical Phase experts this program rely we aren't experience sites of with Because biggest this the trial during important Phase truly point, is sites of of our safely. the We III your sites part To monitoring and guide continue training to in we're consistently Yes. really them and to III investigators the doing double-blind study, the an to wanted trial make

Operator

Lee one Joon Securities. and with of our And question, will line Truist the that from for next come moment

Mehdi Goudarzi

exclude potential you treatment? with provide I experience that Could would on questions. on on setrusumab, Mahdi patients what mix the ASOs. on for like have question III, patient Phase gives And label is patients clarification UPD prior please This the you you with or be two Joon. dosing other for is for some that chronic would you the are envisioning confidence a this

Emil Kakkis

Yes.

Correct. of speaking you're GTX-XXX. So

look open because those support intent stick So we'll UPD types help we're company, the for them cohort patients, haven't to is tried already deletion of Phase we wouldn't and patients their the label which of we if the in expand to where XX% III treated our a at control type of efficacy misses on and going but gene Phase the the be -- at don't gene been randomized have to part patients studying, look and within won't Phase of label, be they conclusion III. we times in to study the haven't the a genetic basket for patients II. studied the we a we habit is types, But which provide method is as type,

deletion So be it type. will -- the Phase all III will

patients, I most improving smaller studies able to types. efficacy are be think, on So with the with puts patients, most support to deal benefit in us to other severe those the severe path the

who had example, of safety haven't prior crossing to right to But is acquirer. point we challenge this very and going going efficacy a time. yet. our look We at we experience, patients We've had would Phase patients something III the number Roche do. now, events drop into Roche had have particularly complicating a The that patient just those program. for our ASO on It's Now not take study. work for still to We're interested for we certainly program. and been safety complexities. it to in on there's patients those those access out We over do done at at in ways what in look patients forward have net can another

And you was had which so program? question the remind on last me

Mehdi Goudarzi

OI. product dosing for believe I its dosing chronic

Emil Kakkis

chronic dosing for OI.

that know chronic we it. Well, we Why we you believe probably do need Okay. get dosing? can

were losing you gain just were and we real that had, turning density, patients we of better the around bones when the took diseases. was of we diseases, of imperfecta is are put age the their What we different kid their gained. bisphosphonates, the biology osteoporosis them patients, direction bone setrusumab very as treating chronically state study know of First different that particularly bone ground It's they of showed off now because [indiscernible] they very including to months and What the that better. treatment from on from and chronic you, their the they disease. the They back do clear [indiscernible] they and that them one bone conducted and dosing. all, osteogenesis know need are continue

we move bones a all OI we at they from their going able a the may genetic you at that stimulating, And need don't So into need effect to different. point particularly at frequent dosing we less say an or do makes believe their their have and to to make said By are maintenance And their them to maintain dosing bone [indiscernible] regimen. we'll point, younger look there everything bones and disease maintenance where we're density indication, what that which dense. they're for induction in be more further. different,

Operator

And from question. line Yaron come next And one for moment the with Cowen. of that will Werber our

Brendan Smith

This is on for Brendan Yaron.

expect any know to you one FDA filing how First, I might timing be you, on to Filippo. But after you you could on soon primary really after I want ongoing. that to regulatory Has year. quickly see QX San with just on submitted. you to get talks this able on readout durability wanted to just patients quick based the when to these you that and those think another file. endpoint, what still follow or and on that long whether understand understand talks be you And have make before they just able really confirmation are expect given you just decision then wondering if are to happen GSDXa. a how to just going how

Emil Kakkis

would that coming MPS to off whether we data have clinical will to important be follow think, the point. biomarker-based up alone. I workshop up, for half you data that read could on IIIA, file I clinical an almost this will data. we've So able expect of assume I'd tell enough collected after that, be the And first touch a will I

we treating the what built find field it's is way are with approval in workshop diseases. we open to for to The think the the the able let meaningful us half. et we more a may now know will forward this first and being better if should biomarker, the door but approval MPS we we moment, for path our that to then field, follow-up accelerated us and IIIA, think more division, either it's important occur meeting review get can way both However, the an ultra-rare but just know

time for haven't readout, put GSDXa a line filing. For we

that the bring in-house, of plan do. which an part be noted are FDA will CMC the time, our and expected will manufacturing but precisely require and in packages, meet But we have with some -- we'll is to go we We the have of all from more meeting important to package understand pre-BLA to have different it. what the a to the what rest

we'll will toward to as be if as diligent help we we I've which that data determine can positive, the so And the -- But move working parameters a expect said. happens. are we're filing given what

Operator

that from One next of Kluska come question, Fitzgerald. moment and for Cantor Kristen will line with the our

Kristen Kluska

that worth II mean second Phase at with least think of So of X.X guided puts you the years of the for follow-up. half setrusumab year, I data at the

wanted dosing further the anything about then impact comments in prevention, looking further to to to to What general, essential relates dosing. deformities if there's about going OI as I cetera? structure, goal. bone the your of if think that of et as But beyond which, course, some also features be ask or be is of even you're in chronic So chronic it fracture earlier improvement OI do for and such you there the could preventing of other

Emil Kakkis

Yes.

right. you're So

that if patient range we're think XX using say of playing. described And he's could X.X that in and you had I He the running around treatment. we probably and like anymore it wasn't months wheelchair years.

reduction is more better at to and clinical that's look feel hoping not only secondary of for but think signs we're that fractures, or fractures. not kind people of having a I what those active still

of as the those clinical story for that going well side we're look in patients. So meaningful to

the expect not [indiscernible]. stop you But it particularly and The in that months, deformity so to they to having also give dosing that improve and of the degeneration and is time X to where spinal sufficiently things every happen. in maintenance in fractures, the the fortrebral the devastated, particularly point absolutely this and the density hope maintain devastating have deformity two fractures, result. fracture, are try and the that we expect would interested lose X the thing OI, have the to that most most which we're X kind we the wheelchairs they the in capture would place be in the go be of can Our who future. of function when column. bone if spinal wheelchair of maintenance allows to us which the at -- type is prevent that in to that preventing decline bone relates tetrizumab one having and their to spinal things thinking, the why should or able to that Those and very we fractures lives change bound. the part becoming able are spines, X, wheel to progressive dosing, the -- see X- stop density old we'd partly few to are be column running is kind study we the you we're in will It's having COSMIC We stop X-year

with treating you we So to setrusumab to help be can need filing a going approval, at the head scanner age forward. when patients new for that prevent OI deformities young establish we a to care for a

you've asked case will that deformities. that the hopefully, question changing about So add that to

that be wheelchair-bound OI think what do can terrific and result that so you seen ending both far. would type X a that's that If what X you could we've what we and be, stop and could

Operator

line with will our Goldman the moment One come of from Salveen Richter for next question. Sachs. And that

Unknown Analyst

Salveen. for on Lidia is This

you much have how frame we we just expect year, UXXXX, of could specifically So the expected help one could proof this around just clinical here? concept update us on

Emil Kakkis

Yes.

detoxification. as the see data. -- patients information going chelators maintain can What a can is Wilson we'd gene least the small the at obtain what can replace replace gene looking X we remove place concept a with where that chelators Because we into is in be the to you as we still right? get well the as can we just doses, still get need just chelators, more treatment. some profound treatment are chelators. set have a we restore that at relatively to can their there copper and the We distribution, because thing So distribution think rid copper to restores you business it's therapy you're that that copper to eliminate didn't in So of therapy letters got get X exceed urinary beneficial a is the is we patients can second for to chelators

and the that And If that might would a with will could of though, about beneficial. the potentially on could up that be the tolerant study are what's known more a able beneficial likely stronger fraction larger is we a dose patients. the done to and very Wilson more And at it's the be happening. that larger it than III will not of are our the to that are become show optimal benefit it to to want disease. rolling and given that The of chelators we'd patients well would be the of us can sets immediately us be both show So dose adopted open things of at both expectation, patients it Wilson safety, Phase those their If moving chelators imagine, distribution great having those regimen then will drug you for allow by fraction that then that we're the copper [indiscernible] at toxicity. the patients XX% respond. that outcomes XX%, happening, door

of the per So of Wilson patients it only Disease. will framing group. give least those remember, aspects the there's two us beginning at But five

grouping might to having effects, clinical us meanfulness outcome distribution. there So that have say expect secondary But about that Phase restoration with the to answer. more of the something in we in otherwise, you that, to cannot neurologic enough the by the clinical study, copper or start obtain definitive have to we'd following results tell III patients

Operator

that our Citi. One next moment the line from with will for Yigal come And question. of Nochomovitz

Unknown Analyst

thinking a various earlier to you before III? expansion you're take end just traditional novel you're Yigal. generate you this regulators, on the is point maybe you're between novel thinking towards novel I'm Phase sharing the cohort might the had guess capture think I'm a scenarios Phase you potential endpoint. end is you Kubar III I point. that you for how more expansion to as I start curious And beyond you follow-up that need is a of submit on planning spectrum and if that. with there on the potential to the for a through for data versus how curious data, end discussion. Ashu more about the lean additional This Angelman more more

Emil Kakkis

a that Yes. so we mean, tested we've Well, focus think what be it and new primarily ones we're main choice thing I pattern study the of which of right, of get have endpoints our brand see. dependent large the which that same not out. we'll number give of result. shown adding The see thinking, patients wishful before be it but or will focus loaded to. necessarily though not that will happen We're I on large is more come expecting our to will of already -- patients endpoint to we change -- be we've going showing in terms But the the precise more estimate reliable, similar number we way, a a is us in very had, see a and haven't better to a

communication. three five five be those and other and to drivers. that express main behavior for points, the the talked sleep. and But talked which will be of about with. two There domains working five goal and the be for the to we've going will going core are be was fine about we're are endpoints daily domains domains, the end the assessments other motor Those from for we've Our expectations

start don't and think have among we we I endpoints a So with that know conclusion agreement. really over. we the We the not how structure We want regulatory best us there's a going on point with think gets to to something ones endpoint enough do. start new up make be with gives the come need wouldn't we to we'll discussion. to we're data a enough and have certainly that going fresh drug That's the where the way and to agency. But works fresheners a to conclusion insight do off believe good in restart

Operator

the And for will of that Stanley. with our question. line next Morgan Hung moment come from One Jeff

Lee Hung

clinical with we to you about HS enough UX-XXX, you've file mentioned do data how need almost long-term you potential alone. course think that the to off normalization time for sustained for follow-up the accurately measure clinical development collected What development? should improved For data length of

Emil Kakkis

our is the requirement discussion had a with X one and had discussions agency. age patients at to they this least Abeona in told areas for the see years. of Well, Originally, reach

in period. been in to is able at patients XX that change estimated period, development decline readily normally look between during and in and there yearly you we've history. the between if distinguish you that development, However, show most then months the the rapid XX that months, natural trajectory can And of

between, has XX over change of who update. we sulfate of think a our months the was what's are of what that's we're trajectory But patient already is on now someone understand have to age X and months to analysis, of say, And new happening also benefit, in the better of means demonstrate let's XX have elements exposure number working interpret number of We yearly patients into can between that of the that helps X estimated years. better insight part trajectory the a or area XX curve clinically we relationship others are may how to age provide months XX understand in of past which And those adequate And good that heparan and the untreated. already this to there showed to we a enough to time. gone what's think trajectory combination on showing we of know sample with be reduction. development. patients this But

what just XX, to have XX already we'll even from XX months, effective. clinical drug patients be of for. minimum will is we the months, be those saying XX so But a pressing And data

all for data the simply XX to all evidenced the get it treatment is another far the standard. go predict XX approval accept to So way to to forward, the toward accelerated but reasonably and more and likely include is us an valuable data minimum patients

Operator

Schwartz with from next the Joseph that will come One Leerink. question. moment for of our line And

Unknown Analyst

today. on This Will is for Joe

just both quickly So Angelman. us on from to

often as just the see expansion prior want to the expect that cut, the skills lead expectation, was response. higher there for to to the also it community, more make are patients that the in that and you a Angelman, still I in hear doses could some we previously, noted So of gaining constantly is this these and placebo data hope compared given to which sure an cohort. be then increased effect may

potential help So just wondering what mitigate strategies in can pivotal a response a trial?

Emil Kakkis

you. Thank

to seen. at be what improvement we've accelerated So should doses expectation the loading that our higher that relative

we is all remember magnitude the XXX on you We days our load thing haven't see you in treatment, of The way went XXX is, So the we changed that dose to out from I data effect would to extension, view II showed see that. days Phase the right? not going real at you're -- effect. be to and going that patients allow us XXX should days. same a number at expect large of should do verify and we the to that the make that we're that the us higher But seeing say

optimistic. effect. time What we for the third-party are lot like of worry psychologist with value a to you go and placebo the is key use doing Now response, recognize because at placebo well, with thinking, there's is doing you to effect see though, the see things. evaluators goes clearly In looking what the III, if while for I beyond you're response gain people are show able a to be regard placebo gain to third-party kid then have it's they that is on It linear data -- something already on forever. a continued placebo people eventually think of happening. doesn't ground continue ground. what Bailey, the what you endpoints, would not the Phase over But longer-term the are the not The

report for are they While looking kid may to have impact, do the some things. patient the

things So and we're effect large said, large effect. able wishful objectivity That of actually patients and that a kid some seeing to expectation is the enough thinking. past it's that not enough just a helps power have population our the be provide do to placebo to

past longer frame of would what is placebo addition, the In or maybe time XXX be. days days about, window normal the should be talking we're well which XXX

how X in evident improving in who's most are become the patients will patients, domains responding. domains it at look you of to when think X I responding many improvement who's and people to not

length time we've risk And study. response so clipping will about the results a the of the But help impairing factors, think of of one and effect, that the thinking issue lot been it. raised is which are of do reduce a the negative I placebo importance certainly or

Operator

that One moment Jack from next our line And will of come for the Allen with Baird. question.

Jack Allen

quick I ones. two have

numbers, products, all, the as know namely out end analyze first and guidance. when seems there's of at seasonality the commercial it The Dojolvi fourth the quarter Crysvita. the I of quarter some like fourth lower already numbers. you're you in But

gene really was thoughts provide adopt over we any to here? more should could are XXXX? it of I a to of Is then you the a So here. some color the patients think asked products more severe And of how course those market could your GSDXa, about on growth hoping on research relates subset be early as two therapy that that there

Emil Kakkis

That's GSDXa? for

Jack Allen

Yes.

Emil Kakkis

Okay.

is Eric we So that that XX% with may growth see to patient because still is art to Crysvita, commercializing, I achieving. in we're some confident our this. best Dojolvi an We're Dojolvi just rate, but slower We're and give we're and of excellent there that feel I any a sales, think form, see with think always issue we're rate to think XX% at guidance the territories view don't looking we're rate. is but the to the growth. Street navigate guidance continuing trying named is close responding we numbers little to something in -- think, territories our have I continue And growth the growth I XX% I be made clear. think,

is. have lumpiness, with is been in theme. these patterns recurring a QXs Latin the It what it So America

I think, ultimately, just -- it's it down. up and swings

and be months think -- have new to some I year. always it's something But overall. winter just higher. Is the manage before that buying tends and going quarter to to before we're Fourth there the

sense, to we but that growth to XX% should it and good -- we rate the smooth have be guidance have excellent. about makes I out feel you guidance have with So

patients no regard have to genotype, of genotype the like With or right, XX%. XX% GSDXa, severe

patients There some the are So but majority severe. are severe. of milder are majority vast

our And being homogeneous than regard of so we'd study. in that look more we gene that therapy disease many at the population

danger having in and So and at on crashing dying phenotype greatest most at from suddenly. in and probably need that disease the I patients point, severe very of dependent this look the of starch, highly would being most the terms

Operator

with moment from H.C. our And of that line will for one And Ed Arce come next Wainwright. the question.

Unknown Analyst

Good asking afternoon. This Ed. of is for a Thomas questions couple here

Can you So also, of what tell additional just days. cohort. GTX-XXX in and Angelman, amount Phase us in longest these And compared the are some for focus that III the patients patients are current on study? the duration investors expansion previous some that what the can endpoints have to

Emil Kakkis

I heard the cohort. extension

all to of a -- asking year of of X of patients. the years for how have will they long years exposure be treatment presented probably, we'll but by who patients get X go somewhere between we time from out. have You're the treatment II, ones Phase The had we to end those it

the patients time. age And long of a who a beyond good are Day meeting. quite XXX we patients day already So have Analyst -- at chunk

problem actually a chronically drug shows going think you that accumulating safety disease. that's good not exposed -- the in cause to this are I So doses

your that. So question because I these that didn't the catch treatments an I think of quite I issue. and profile we Reestate part have doing second an important can safety that's

Joshua Higa

considering. question I was think should the about endpoints additional be second they that

discussed perhaps we've Beyond Day. Analyst at what already

Emil Kakkis

them severity different Well, the Bailey an -- either before. Angelman used of scale the or a before. or assessment be we behavior The ones or sleep core, seen two alternative see. are lot the the we scale [indiscernible] looking ones We've for the behavior talked sleep we've I we're could have those about, or used at.

include didn't data. have recent in we -- them extension haven't We didn't most the

So endpoint. just for questions I endpoints pulling relying it. collaborative those but with key not those to say of with information those and few and were choosing like the were to questions great a I us out in the rigidity flexible to endpoints. would our on discussion we'll flexibility FDA, more ways and alternative They showed get on they up calling doing you a little think highly regard

those And set, particular example, so existing out data for and can on for we behavior questions in those for within just even our use hone sleep. questions, and pull

go we but to So be but before, new at sensitive are coming what not we data to won't out haven't data up look our goal be would with there sources don't things you've things the for patients are that that to never and seen have and with pull any seen those or clinically agent, brand on. new change in the best meaningful we have

Operator

time. to call in remarks. I'm questions for back queue any this would over closing like showing to turn Higa further the no the I now at Joshua

Joshua Higa

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

Operator

program. Thank you all for This participating. concludes today's

disconnect. now may You

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