Earnings call transcript Q3 2022

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Operator

Thank you for standing by. Good afternoon, and welcome to the Ultragenyx Third Quarter Financial Results Conference Call. At this time, all participants are in a listen-only mode. At the end of the prepared remarks, you will have the opportunity to ask questions during the Question-and-Answer portion of the call.

It is now my pleasure to turn the call over to Joshua Higa, Executive Director and Head of Investor Relations.

Joshua Higa

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

Mardi Harris, Joining Emil me Medical Commercial Officer. Erik Bedrosian, Kakkis, are Financial Officer; Chief Chief Executive and on Officer and Chief this Camille Dier, Chief call Officer; President;

the will actual Please subject I'd risks to remind we and during factors results statements. materially. forward-looking risk uncertainties today's that to making our to These filings. our may in certain refer differ like call, and latest everyone be discussed statements are SEC

the I'll over now turn call to Emil.

Emil Kakkis

afternoon, Thanks, Josh, and and a XXXX good both our for in everyone. execution focused our commercially team year on been pipeline. has

the We progress broaden to and our continue issues from third achieve have strong priorities year-over-year In revenue commercial commercial key to quarter, across continue substantive programs and growth we despite made in base. our our challenges. ongoing macroeconomic

regulatory countries Evkeeza In In commercial lipid are seeing we potential positive experts and and on underway is our multiple momentum significant Europe, are the we Latin enthusiasm across Evkeeza seeing from revenue. increasing launch America, for with of country-specific for to discussions. enthusiasm in submitted treatment paradigm. beginning will believe discussions. or reimbursement We supporting in the expert product the reimbursement multiple will and of effective are already lipid dossiers the countries change submit We've pricing help

that GTX-XXX With we data full in promising shared on program the and regards science pipeline, interim of our I/II we to control July. the Phase acquired

patients profile the XX now with being in continue mid-doses encouraged We progress and program to to be safety the the maintenance. in by XX of used

well Phase a has III anticipate of we months, which enrollment option. with study very new treatment the GSDIa, for for enthusiasm few the in patients enrolled next the Over completing

two mid-XXXX, Phase should of to plan of bone We levels. in us study the also enrollment for biomarker give data osteogenesis response imperfecta dose the II portion on which the of complete

and this for chain project. of therapy the facts we our team operations Boston focused the of remained solid big gene continue should gene pandemic to and month, on building and be has been the manufacturing Last track quality on next are of I for years supply maintain Board with the have exceed the I facility by and therapy issues, Ultragenyx work boost impressed manufacturing facility to global a begin our outside year. budget also plant. Directors within the the with franchise. visited greenfield our It Despite laying financial foundation or a timelines ahead. We've be costs our hosted met to the

of operating objectives. headcount manage with will the quarter, specifics In $XXX July, our and as to worth we wanted our provide our cost will have partnership raised the priorities controls Kirin. while our additional royalty million have but expenses achieving performance limiting sale actively in the we Crysvita in We on XXXX. more headcount, believe achieve we and into to Erik third these a and touch span on right team well portion place briefly commercial of Kyowa established I team's

throughout We ensuring partners established. the field U.S. on in make very an disease Crysvita our will rare successful solid our period recently for commercialization with closely process have extended be the launch. building Crysvita to foundations worked agreement have We of companies both collaboration time, continue franchise to allow amended a U.S. we

transition increase April, additional after the operations U.S. XXXX field ahead began recently field the the further Under In terms the after joint to our that and Ultragenyx details here an one-year and full-year the today, maintain for revised will commercial commercial responsibilities transition through a of increases we in April, amendments release presence shared XXXX. consistent KKNA we transition until effort of earlier for press the coverage. issued

and patients additional opportunity them highlights with of surge franchise. an XLH each Crysvita partner's providing identifying more continuity to to help the commitments The efforts maintaining treated should and be with

to KKNA. salesforce with will time, Ultragenyx expenses this continue be During shared

disease In and our TIO programs. addition, treat and clinical agreement. our co-lead partnership Medical with to Genetics diseases based an Genetics many in Ultragenyx programs continue original and will XLH transition indefinitely after call monitoring will healthcare of important collaboration terms of the And targeted on providers with we by America segment on are the the who KKNA. other in Medical North patients XLH the of stage

provide call specifics quarter. the the the to on With performance to over that, for commercial more I turn Erik

Erik Harris

team's the and supporting you, good the quarter, launch efforts I'll Dojolvi Emil, Thank Evkeeza everyone. third afternoon, commercial of Europe. and on upcoming Crysvita, in the section my focus across

a within key therapy of territory, reimbursed start continue number forms, metrics, profit including meaningful Crysvita, and to in on the prescribers. we unique see share patients For increases

have the unique In in increase is steady time the These a reimbursed XXX of patients an any forms start were quarter, saw last base therapy type on by impressive of in completed. particularly increase led quarter the number this to forms expanding approximately prescribers. third This driven we was XXXX. in given start of early ever given

new the prescription providers approximately XX a third healthcare wrote In quarter, for Crysvita.

are from over from We existing with Almost the product physicians. other writers, XX% patients new into rare finding the years of launch and this is I new of to more continued those applaud while coming adults physicians now as team's prescription disease community-based our four are find increasingly prescribers. XX% efforts community are we the well coming

offices of and and physicians to open we We COVID field come face-to-face activities personnel. our educating are seeing on many as focused new as increasing are now out

maximally us transition. The sales specialists signed several years, to our patients long-term working April, potential. enhances recently Kirin ensure amendment we Crysvita's KKNA has month during of this our the last field been While support team XXXX combination of the and team Kyowa side-by-side closely our can field additional with allows U.S. beginning through extension in to work for the

Brazil, countries six profit including America, for This Argentina, months drive Crysvita quarter-to-quarter children five known Brazil now Mexico. at to and XLH variability, territory, and uneven steady and demand the market in therapy the largest of continues share older. underlying in of Latin grow and in well adults be recently Argentina is America the was treatment Latin some a Chile, Outside ordering continues pace. approved approved to in while the the Colombia, patterns in

XXXX revenue is forecast million first in period regions, of Across of compared We nine revenue Ultragenyx all Ultragenyx are supports know same and in XXXX and XXXX. territories months reaffirming Crysvita the demand of of the our full-year to million. guidance grew our the XX% underlying to $XXX we $XXX our Crysvita revenue strong

start including and exceed in therapy, Turning efforts and with now beginning the number Many on U.S. to reimbursed forms expectations. patients the of the the our of continued Dojolvi leading to indicators,

third providers launch, with As of have XXX quarter, XXX on the patients therapy. metrics, is for optimal approximately Since completed we of supported prescriptions educating these are to written to receiving assure start Dojolvi, XXX the providers more clinical healthcare on one. than dose opportunity strong nearly healthcare optimal dose. titration and reimbursed approximately an with half patients an have there for continue forms, received Despite benefits writing by our over studies

recognition, we to Dojolvi of programs continuing and France by continues Italy. be Latin in where is led America, get few get enable approval approved orphan Europe, benefits currently U.S., to with is access which work through faster. Brazil the in even to to In Mexico, this to Outside demand are us and a in to process therapy similar Dojolvi particularly approval. we early will named In that patients the reimbursement full receiving for recently patients product important received

set in maximal to range of While better effect need we for dose $XX to are for year time, we $XX guidance titration million. work we beginning the the reaffirming the continue at address at the this of to U.S, million the

The America, then homozygous by for hypercholesterolemia. study with Japan Evkeeza, Closing Europe reduction we all standard and LDL-lowering clinically familial with in launch of which in with improvement data Canada, preparing over The briefly for on Regeneron's Evkeeza drug demonstrated a top existing LDL-C to diagnosed significant very care are treatments. were a XX% from of patients pivotal followed impressive. Latin

the that also patients. of novel activity. studies LDL-C which to Evkeeza key Feedback positive, alternative remnant is XX% mechanism can blocks less that LDL This who Evkeeza reduced convert cholesterol be leaders lipid from the for to of LDL-C understand that bypasses in the ANGPTLX, normal pathway value enables VLDL easily than process study have They opinion metabolic of levels receptor liver. especially patients. the a of by proven severe clinical leads the patients in showed into The high seen action X% with their participated this by cleared in dangerously these and those most very

access, Across In Dojolvi, which later and to the parallel, reimbursement pricing could requests access from filing for orders our this first on delivering of Evkeeza are could the disease is team named a and are evaluating Mepsevii we commercial Europe, global in generate in ensuring to the process benefit look therapies. patient Crysvita, leader by discussions. country-specific rare summary, all forward to In we year. patients beginning the who dossiers mission being now these to

financial With that, quarter. more to for Mardi the turn results I'll to on the details the share call

Mardi Dier

totaled territory release the for other including included regions. ended Company XX, financial Crysvita Erik. million. $X.X was North Thanks, revenue sales three Ultragenyx period sales million Dojolvi was related issued results summarize. the for was Crysvita a share million the third will $X which $XX.X months million. that net European the in profit and same revenue territories territory XXXX, quarter, for the product $XX.X America quarter the of million, Mepsevii the million. we Great. $XX.X revenue Earlier September today, press to $XX.X for royalty XXXX was in from revenue $XX.X million. of revenue in I Total briefly

quarter of expense for compensation of of operating approximately quarter our SG&A million million, expenses expenses for the cost $XXX.X million. million, of of expenses related Total $XX Operating and $XX.X sales million non-cash includes $XX.X GeneTx. of to which of were expenses XXXX million a included R&D one-time $X.X acquisition stock-based and third $XXX.X R&D the

million, our loss $XX to non-cash million This approximately and For the are that $XX $XX net during non-cash million the $X.XX to interest or expense stock-based Solid in and the highlight Bio. and Arcturus ended share. approximately the items XX, million of non-cash to net in These first of September related $XXX decline impacted of XXXX, handful the OMERS royalty. of important compensation, loss nine value revenue Pharma investments It's by per equity transactions, Royalty $XXX.X of fair have million a was quarter EU of offset to year. included related related the months

cash, quarter with in the cash approximately ended equivalents billion We marketable securities. and $X

are While manage we continuing cash well are also to to decisions our burn make strategic operating capitalized, we rate.

XXXX completed GeneTx of acquisition gene have of and for late-stage are programs clinical we the As the burn year Evkeeza, our peak us multiple is manufacturing have completing initiated as build-out we said, facility. a therapy in-licensed

the began we maintaining of implementing we As initiatives head Emil XXXX, drivers. will process into quarter, mentioned, key in operating value and headcount our on as that expenses the third limit while focus

With to that, over Camille. turn I now will it

Camille Bedrosian

too everyone late-stage continue Thank one most we rare you, and the diverse third in pipelines quarter, good I afternoon. of In Mardi, and to the disease. wish advance clinical

clinical made our enrollment cuts enable data well We to of number pivotal the our studies changes a that of also programs. will interim have strategic as faster from as

in gene clinic, programs for osteogenesis therapy currently and the few I/II Phase of the II/III Of imperfecta for Phase will Angelman the GTX-XXX, a I UXXXX, on the syndrome, briefly programs. touch

serious least cohorts enroll we in patients to XX of of a for original a reports dose at and five have escalation the Starting and to received cumulative patients patients Crossing minimum is no no GTX-XXX with XX Phase Angelman XXX GTX-XXX at an XX safety safely drug-related Thus I/II XXX extremity have events, study. experienced dose dose exposure days. milligrams exposure far, the to weakness. XX as important continue over lower dose of lower days syndrome, had weakness, reported patients of thresholds milligrams of at and extremity the cumulative including with these up

five continues confidence safely give dose in us administration strategy that allow the the titration us to recreate This patients. and to original may the slower amended seen efficacy

a lower one These are including from have no originally event. reported drug-related and doing at the patients patients cohort previously there redosed first also lower had U.S. doses with weakness weakness. no including extremity significant each two serious in the well, least have that extremity been treated Canada, safety two We events,

clarity and We are U.S. to the the protocol of the in to process ex-U.S. work few through next have aligning months. and intend continuing

once cohort. expansion in the provide half first data expect enrollment and data in begin update of we the We gathered XXXX, the substantial will to have cohorts a we from

substantially. to And setrusumab the collagen osteogenesis in now for mechanism and bones potential mutations for patients reverse mechanism strong with The imperfecta. value response UXXXX belief Turning or to strengthen we this osteogenesis setrusumab imperfecta. anti-sclerostin the has of maladaptive the particularly bones majority have to of a of the

II/III of months. remove study. sites mid-XXXX, to are we will including study placebo to program. the our of us with Phase data recently to will expected this complete the believe Data real-time along randomized setrusumab patients next Phase changes larger few stage readout, available study the dose-ranging from establish with Phase the to the protocol Phase the III algorithm. enrollment this amended enable study pivotal of Concurrent the the plan in the the the of in the response in bone transition stage biomarker For this we that few portion the be do allow ongoing III change, analysis portion study, last dosing two-month from to activating us XX of We of in II to the to

and gene was and a Phase lead designation DTXXXX designation Ia, are approval currently program for the interactions starting provides prime This therapy opportunities with EMA. III agency we to Glycogen with the study. could Storage Moving additional Medicine placebo-controlled XX-week from accelerated Type randomized, to evaluating in or granted for programs an Medical of This Authority, pathway. Disease of which our double-blind, recently treatment European the pivotal

Recall of efforts year completion data the these enrollment. on earlier helped focused rate and have this the the thereafter. anticipate of end Phase increase one-year we enrolling around currently of this with enrollment We year, III approximately study, resources

continues study. Wilson to stage the placebo study. patients UXXXX, the to from the recently for UXXXX Similar in dose-finding we pivotal to now the the which of of stage removed Turning disease, enroll

plan While dictates XXXX complete late and early on patient, to share between each period the to progressively of in safety of this protocol a activity and data signs or observation initial in shorter we expect XXXX. enrollment mid-XXXX stage clinical

in my hemophilia I'll remarks The last DTXXXX mention is A. for program prepared

this has to development Bayer As discontinue part review, of and Ultragenyx. our decided the to regular return of program rights

better to A right version the this that is development a gene is believe continue in We of hands. therapy hem

we interest We DTXXXX in hem A in but would that be development a of rare and hematological will AAV initiate not disorders in further partner, high-quality a a developing clinical interested quality partner expertise without has therapy. a an

A hemophilia and our will We landscape partners. respect therapy potential continue with to the monitor gene options to explore

Thank you. I updates, to now call turn these With will back the Emil.

Emil Kakkis

Camille. you, Thank

quick upcoming the of I'll Before provide the to the we portion shift Q&A call, a of reminder milestones.

to based be the on enrolled We year end later. it is expect now one-year well the approximately around GSDIa Phase the III data how rolling fully of with to be shared

enrollment in UXXXX expect and For III complete the few share dose-finding transition next we in to osteogenesis and II data stage to the in to Phase Phase imperfecta, months mid-XXXX. of expect

XXXX update the to in are to expect by of highly data and substantial expansion GTX-XXX we a Angelman first half begin encouraged progress of XXXX. we've enrollment In gathered syndrome, and cohorts in the and large data plan provide once

Lastly, syndrome. UXXXX for Sanfilippo

approach These filing and execution for to commercial our position control mentioned, based Mardi are current this and generated an date. deliver value finalizing on We clinical and data patients us FDA continued team's Abeona catalysts, in the field approval are other to in shareholders. cost continuing the meaningful evaluate to biomarker to measures by the excellent put regarding

that, move on let's Operator, the your With provide to instructions. Q&A please questions.

Operator

Our from first line Instructions] Wang the of [Operator Gena question Barclays. from Certainly. comes

question Your please.

Gena Wang

I and what the for escalation? timing to sufficient score that next be Since release, cohort think that said expansion Does do will data question. the update? taking don't half Angelman. further were first Thank dose you dosing improvement the an X And you cohort mean the you have X CGI the regarding also, you my I showed XXXX. one for press in need the

Emil Kakkis

Yes.

it's more making going time, we're of the escalation feel We're commenting now whole So the be encouraged. cohorts. and an the expansion cohorts the cohorts. general a dose when giving progress, on cohorts dose data we to not from we're the at provide we just we're looking appropriate on And you expansion to but timing when get data program good and we'll

Gena Wang

the what Okay. patients doses dosing? two they received And each quickly for two frequent in how doses was Also receive? who did Canada,

Emil Kakkis

in the protocol depending on initiated five, we their the that protocol for they age. four Cohorts Well, on same had place and

X.X the X that And a same so one. dose the either was start dose, or to

they think, other and four I receive two received so one the received And would already. – doses, two

adverse The and important all, thing is we're doing. they've had at encouraged effect no how by they're

more there's So We're hoping other get get patients who treated. want to the treated. to ones

Gena Wang

very you Thank Great. much.

Operator

from Thank from our the Schwartz Joseph you. line SVB Instructions] next [Operator question Securities. comes And of

please. question Your

Joori Park

dialing Thank Joe. Joori for taking I'm you our Hi. for in questions.

six first cohort XXXX late XXXX, GTX-XXX. XXXX XXXX, previously I it early looks early late – in you is believe The but one expansion to or or that seven on and update guided sorry, you enrollment first order there to XXXX. some in done was of if need like begin enrollment the just first in to half in of will I be that the wondering begin things XXXX. are half f

Emil Kakkis

cohorts. been Well, enrolled. enrolling already been we've They've already

is what by the those we're So completing we cohorts and the initiating in enrollment early said year. in expansion

we've underway. haven't that bits with of data progress. we more decided cohort. announce had substantial So We that's than And – to what provide not of we each all a amount out when just before we data would data is dribbling rather said point-by-point

have a So the but we're we're progress. going of wait encouraged substantial until data, by body to we

Joori Park

on to was with studies are it. release, a the to then I happen. the is much. very the for you order in you follow-up, FDA in guess make as and FDA looking Okay. that ongoing. what U.S. color provide And could Got if you the press harmonize, discussions wondering Thank mentioned this I ex-U.S. some

Emil Kakkis

of terms don't go prepared. process. noted previously and the hope we we'll we're which to and through information U.S. CSR, the to report before the a run U.S. that the other provided current through wanted requested, sites We they at in FDA, data Well, back set working we study. some sufficient and we And they of we'll – reopen usually details that the information complete had forth, and of then be provide the study but we the

that's been them. So complete we've But ongoing. for just preparing they that a and set, more wanted

Operator

question the Thank you. line from comes Raymond Piper [Operator next from Instructions] our And Christopher Sandler. of

Your question please.

Christopher Raymond

Thanks. Hey.

questions. two Just

First on DTXXXX. – XXX

here XX. it about approval? basis kind at you of data, just saw Just review. – have for that totally looking that? guys I reduction prior a look that review know thinking as chance is clarify, you kind the and I'm this prime the Thanks. but process in then any an interim the corn starch setrusumab, you know to off reason protocol Can Is meaningfully serve finishing announced amendment. back you sort use even for for at guys just the of the on clarify week of the just European there of the designation. with curious enrollment pretty III, just is And sort of accelerated talked Or the about just of I table? Phase guys delay before,

Emil Kakkis

blinded. serve have U.S. DTXXXX, Okay. With acquired we and Europe both and to the trials

XX-week So even both. six-month blinded we were if data committed for data of to had better,

no will accelerate. we to complete there's get us help way data, to process get though, to designation So review the blinded to prime a our use file. Once discussion that accelerate the

study. of that the it. the the be to honor But use body to shorten the we so couldn't of We'd would And that main blinded XX-week study. benefit have main

under I the year, say, in because general, of are setrusumab, beginning Omicron look, For duress. all would of sites the in year

had ahead. lot managed getting of have a and challenges pushing going, that. think We're studies I people we've

dosing. placebo on, doing data while wide able we'll study. what's the to of removal just feed going We're the ages. along dosing us doses. on to It think help of a we're a allow enrolling don't dose. And our be us just an through all gives in one to like opportunity could range each studies though, patient. The placebo of are on ages picking different algorithm the different going We think see I get kind time real more

through able faster. and just being to So it data us that through fly by will help get see the

final when to for we ages. need whether bit the patients in, decision get higher a we of what younger data we for the can So go on make and

to benefit it's us. a main So

its very Street look what the all will data, well as our and profound OI. patients, though, drug dose in the the with anti-sclerostin globally. data, the we animal and as drug to a III. be help of why it And like for in the think able also for a awareness having when of it's way we'll raising be at strategy probably us important people to hard data you us That's and looks inform both With driving and human, the course [indiscernible] in effect also a will helpful Phase the and raising very younger awareness

Operator

Thank you.

line next Our comes Ahmad from of Tazeen of question from America. the Bank

Your please. question

Tazeen Ahmad

my Hi, good afternoon. question. Thanks for taking

data? how expectations for just data point Thanks. to your you for when to for of OI secondly, And would maybe [indiscernible] be you to be And indication, remind think a clarify, clarification can large addressable DTXXXX? Just UXXXX could population clinically for what you this expect Phase patients? on consider us meaningful about see III then we should

Emil Kakkis

Sure.

let's So size. start the with

X For the and be developed X X, to setrusumab both patients powerful the all type That in types. type to to us XX,XXX should UXXXX address amazing type very is allow do on order would that group, world. which treat and of in three OI able or

what than larger XLH essentially So is.

population. very a So it's meaningful

the the establish study, right the dose. Within help study, of part the II, will Phase first the

So decision. we'll have make biomarker-driven data that basically to

Phase young the III drug running where can a to a way. between be age another is Phase main younger used clinical which the very We're study be really we'll experience looking at young very our rate also in with bisphosphonate, own young which diseases, bone – the study, from a profound look III, way patients severe XXX group. fractures treated to fraction, setrusumab in rare at us FDA the from patients, high going effect, the to particularly in gives those the or another the we'll new clinical fractures, demonstrate we'll of which powerful respond we which in For XXX discussion, placebo randomize with and patients

product. another for the us way to it's So demonstrate think we

be randomized. That study open but label, will

that will one total And look – at fractures. so both

one maybe meaningful Xx So fascinates but the this clinical fractures, looking them study, effect And fractures. I at Xx Well, that's reduction are meaningful. both reduction is that think than how powered better XX% total fractures, a We drug we've of fracture, fractures. would in think certainly proximate this other will the clinically in We out be reduction. have about think XX% that the of a failed a and studies.

there's help But I that more of we'll at than respond fractures, drug enhance things, how a will be and because be those clinical the endpoints, meaningfulness, impacts now, right in are just disease the they which think think have probably will those we daily lives. number and looking like in their how how feel, how is on used it live will their well important patients

Operator

Thank you.

next comes from line the from Ha Our of Stifel. Gon Dae question

question Your please.

Dae Gon Ha

my One question on KKC Great. clarification taking Thanks amendment. Good for questions. afternoon. the

expect to April So that much. XXXX? or wanted in perhaps clarify, should April or guess, dose April starting this transition royalty to we any profit another to also for the Thank at base, XXXX the patients for then Mardi, just as share? way, apply very extension XX-milligram have And does you share XXXX follow-up to just And cost to GTX-XXX, occur a point? reached I

Mardi Dier

go straightforward. Very in. just I'll jump first,

at royalty we'll have structure April so time that transition, initial amendment. the the place So XXXX, takes of the

be – the the under have we to and grow to additional will folks sharing We field help make transition a that arrangement. Crysvita have cost to smooth we be will continue and sure in able to

Emil Kakkis

reached have a titration, to patient XX patients large and there there's in the is received GTX-XXX, the but one number that the regard cohorts. and from XX XX earlier With many Angelman of

achieved and what So by seeing. a we've of moved we've significant without we're having encouraged level side drug effect, we're – a

Operator

you. Thank

of line comes Raycroft from question next Jefferies. Our Maury the from

Your question please.

Maurice Raycroft

thanks questions. and my update the taking on Congrats Hi. for

to I July dosed wanted can patients see For – for cutoff XX? you if been have many new July how the Angelman, data clarify since in

Emil Kakkis

give don't that can you precise think I Yes. we detail.

all expansion them escalation you're We're is to be in of fully and XX we're early cohorts, doing we them expect think around. we end the enrolled the are going four but triangulate the And said first one – through each expansion. year. working which would larger which the trying be I to before group be two half, dose to the – cohorts, patients, can through

kind at that's any that detail we So moment. provide I the of where on won't more are.

Maurice Raycroft

also standardize sites on for in States assessment Angelman, discussion measure? potential United score and the in reduced for it. CGI to Canada. a strategy some you been across versus EU CGI Got have the And buys there's Do place and discordance

Emil Kakkis

that's to Yes. raised. a question that's fair Certainly, be

I [indiscernible] where the think that the parent. calibrated we're PI, the score of we've some But the score assessment that scoring is that. measuring of not Bayley and shown say score. it's would not on one example, additional way the kind the the the specific working to or with we're psychology third-party objectivity. gain ensure for And data, though, training also because in Bayley administered, that's particularly psychologist the original the the to on done I reliance you're like performance normalizing scoring system

important training we're of I also scores effect. what confidence that a So these us an of seeing independent help not that reliance placebo dependence a give and effect combination and some and have on is

Operator

you. Thank

from Citi. question line Nochomovitz Yigal next from the of Our comes

please. question Your

Yigal Nochomovitz

and Mardi. for on Emil questions. the very Hi, much for think, maybe I commercial the Thank I you had one taking team. business,

to fourth regard to the for trajectory Just looks range quarter, Dojolvi, you reacceleration guidance the guidance. of lower significant the meet in the for it end need like a

a few the just just spend that assumptions if guidance you. getting range? you minutes Thank into could clarifying So around

Mardi Dier

what's on – with the Dojolvi I Dojolvi. do been not take indeed commentary Erik give can for start, just have always. quarter both of hasn't Sure. a launched We that Crysvita going as a then here on long. It percentage he that some and is well, and But seen I'll we've – you but as heavier always actually revenue. with what fourth total mean

it's number in we're and seen the treated, the quarter and and we on that of health fourth start I we expect you the commentary the we you. of doing. – give to and expect we've patients given Erik, this So know going think any Crysvita, again if that the year growth mind don't that the how business Dojolvi more for into just Dojolvi want But forms well

Erik Harris

stated, as a and strong. expectations identifying therapy to the demand in our optimizing on commercial I continues Dojolvi, to patients manner. have dosing. to it regards those patients is is converting exceed timely And that challenge we're working with The

campaign working we providers increase patients have of closely at dosing awareness and treatment. both this titration optimal underway around educational and so and to the time And with timely an

Yigal Nochomovitz

for Got it. disease I placebo wondering pipeline. removed part the the one independent and and or the if setrusumab that were disease, as quick decisions as if therapy connection totally Wilson's you. for was just interesting on you thought both just there's just well finding. they antibody the And coincident. follow-up Thank it gene for the any those I'm unrelated the between in dose

Erik Harris

have and just four now. independent right been coincidence, at same time running trials They're the we

at more that they're things. placebo on the We do can and stage. your So that. the And design, But all and seek to going learn always to sometimes actually. we perfect – the is be which think practical it's what's know considered same have reality I ability then

part. of what maintain the for the study, like And body be it to going the the learn us was better was happening placebo on for we felt simplify so to the and but upfront main fly

haven't talk be also it seen to didn't it, rolling It doctors with and able helps about to know about the just patients they makes drug if program discussion. in because easier that anything the to the

we're stage just to and everything. we enrolled fast the best adapting drug can. happens as at totally for we're separate. It get what's to the forward be as advanced this way And it's So same

Operator

Thank you.

the from of Our Joon Truist. line Lee next question from comes

question Your please.

Joon Lee

our Thanks questions. for Hi. taking

a responder And there? which multi-domain runway? for to alignment you. mentioned mitigate possible comment XXXX. on? areas way expertise cash outline You cost Can your given XXX, focused is on noise Thank for any index you And you're

Emil Kakkis

Yes. a I'll detail? provide with and can you start more expense, little

Mardi Dier

Yes, sure.

Emil Kakkis

provide The that a not We've drill big value. down growth. here to of main money and place had a that. is Mardi it. job spend in lot in harder that to on Maybe need on manage That's more growth, don't can create some to our color thing really

Mardi Dier

Sure. Yes.

Dimension really a the past of So growth we example, of had for have we mentioned for years, starting headcount, couple lot with the acquisition.

keep as flat be looking to do as as forward spend really into Emil where drive keep we limit that would is value can to operating exactly well, our keep saying, flat to well. headcount OpEx – and – And and is is not goal prioritize that so And XXXX going and as money. our well going goal expenses what and our headcount

results. perhaps We're we're think on housekeeping, that see that things near-term. I of the programs these would already seen value are growth. that's not drive good we've I And right? this period we'll want think where had that implementing, So in we results And and we've the

a So good standpoint, that's we runway cash are in great. shape. From

XXX and some and have about asked $X years the that milestones always billion you've questions of about through on the next we haven't say us takes sheet, We definitely specific balance – and couple given XXX. on but dates, we critical

cash So we shape. cash standpoint, of forward. from a care want that that feel we're take moving good and in lucky to We

Emil Kakkis

Mardi. Thanks,

potentially that, a or evaluating population. you a could So is different state, on big there the the heterogeneous We that way multiple those in importance familiar think domains given are five domains six changes fact a domains, Angelman is of capturing MDRI, that you and the or of responder for prime wins disease different big evaluate. many very multi-domain index with not of clinical

evaluate add given difference the with wins minimally big up endpoints filter. you through important You and

that between a population, select for which thing think capture to them efficacy vary patients population particular [indiscernible] the to looking we're would that We in allow have carefully the not you might to method at. allows

you're would that to expressive do you Bayley I score, example, third-party who say a can can way for and other having that you So a primary endpoint, professionals if measures using receptive have imagine assessments, as you forward, test communication control as get of types or certainly the well, it's and you Bayley’s measure it's one potential. a

lots we're they're it. features it. The good MDRI It So look to it level could discussions all FDA. there's in it. lot We of a widely lot Mepsevii, of but senior something there's great that to did at be a where through those of We've going options there, for of disease is not a would use accepted like Angelman and by at be heterogeneity. work aware a had

problem we optimize to of on there's figure so how pallet out, to out a to in of have endpoint. yet figure trying a matter but a efficacy It's So not that much we the options capture now, it's but Angelman heterogeneous it's the time in going across the population. really right

Operator

Thank you.

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

Your question please.

Yaron Werber

that they Maybe, accepted a and taking give to ORCA fan the communication. And you academic primary a communication, that placebo and there endpoint that you just us priority, for do a give Emil, much question. believe you been endpoint that's as know take Where toward I my of FDA, II requires for might then site. the on too to grant a Thanks pivotal? that? Great. a you Phase validation I a just might an validate secondly, that sort Thank as select a long. and move have on is even you just Can chance going maybe point, well, believe very you before very of – would And validation? run once that explore is any to dose, to you. Or is

Emil Kakkis

So and ORCA, seen the we're ORCA. we're running the in we've improvements familiar,

I will validation think be ORCA – the done. the

I it's now either be to done going done. think or

a I is development there's putting can that is would think and will at it that to primary The that and often as thing caregiver you look happen. all thing when be assessment clinical you a is noise. tricky I because critiqued So say complicated

to sole regard. I challenge. caregivers think, – be putting would In a assessments I it generally the fact, with would endpoint, this primary in

it is But see might you that. rather professional much professional would behave. like something an missed not when independent comfortable, the you I alter patients how have for valuation valuator setting a

of all aware that. we're So

for time have us it to We figure out.

validation language of overall – be ORCA do. the comfortable being important primary, how could think But of the the patients I a of I will components with assessment whether be would variably would it primary. an of part less it be one will happen,

Yaron Werber

introduced My was II, we second Phase the placebo. around question when

Emil Kakkis

kind running for item we and really placebo. that common you're to not think the Yaron, to but us then large question compelling an our it a seen is would more thing have get trial. data. going think asking, placebo Yes, efficacy less The gives not confidence frankly, fair kind we're treatment comfortable plan have a have but think treatment we're I we be And want a Phase Well, we question, dose will It's we know Phase looking change lot we the that of people, worth is we III feel include that's II additional is be right If now does effect right. persistent. can in. that sufficiently is we definitive how right effect investing don't the or of the seeing because we're discussion were not, for unless certain, raise to of enter I we data will been

need would preference. But in your well to that amount could is think we taken, right we'd then now, we looking move that our is if was kind consider we're If the get III. efficacy mind, but point efficacy can I of of incontrovertible to Phase That head for there for before we right solid But our III. it. that, really be large Phase

Operator

Thank you.

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

please. question Your

Elizabeth Webster

for taking Hey. Salveen. is This our Good questions. afternoon, Elizabeth and on thanks for

on could progress? end early you you the for? on to next discuss you. on then signs looking provide Thank the or program, update of expect you data how year OTC could XXXX, And you're what color enrollment Wilsons' the For if

Emil Kakkis

Certainly.

of to to open patients planning cohorts, label. in enroll So three five we're Wilson what for is have update, the we now each all will expect

of include telling So the to dose what be like that would looks in urinary different core would copper you population. dose we that that going we the would cohorts. measures copper, tell Those dose expect us, is what and Wilson. our data hitting safety worth We'd patients are XX FDA the to relate in on physiology metabolism data. be the ceruloplasmin right about I and have other and the forward and parameters

we expect at to what time. out do that's put that So

OTC, as start-up after we many trials consciously For GSDI as we OTC are. in put enrollment running

enroll. enroll So We'll get get be is take time It trial, and randomized time lot enroll. focus enrolled. to adding more on a our enrolling, the we be to so of And will it's more longer. our probably GSD a pushed we'll outside to and than in have expectation. it OTC to It somewhat that more XX-week prime the now will more going GSDI effort it a bit XX-patient U.S., to a study, which it still probably take U.S. it's to has. out it the little and But is

Operator

Thank you.

line Joel RW the from Beatty from Our next question Baird. of comes

please. question Your

Joel Beatty

Thanks.

across from new the coming program For and mentioned new new then are scripts on could And Crysvita, question I think second that attractive a half to How from existing B returned new you make prescribers. remaining does addressable Bayer, hemophilia the is split what the partner? prescribers market about that prescribers? the

Emil Kakkis

then, Well, let first answer one the first, Erik, me answer the can you one. and second

it events. hemophilia So they data, on had what and they decided a wanted who criteria A patients reduction They they came our had meeting acquired priorities. back wasn’t had had with in significant program. other a And great was number of

to plate an us. What company. bring put they as where so into looking don't we our to all, to have want money We experts. lot a back we're decided it area And on first a we're for – more of not

think room. has we still hemophilia So

better getting a development, I that We still therapy. we a I they're But think room for and the benefit. to large-scale partner of be type our we'd gene happy think think think, looked do pick And who the manufacturing. manufacturing [Bayer] is AAV to manufacturing, program be, for up there. process and we've do our wants would

for lot product. room the we think We the still Pinnacle why and have a which be as our platform left give better. something PCL edge looking manufacture that new to and question plant But prescribers? don't there's well, Erik, an answer we'll of a that use to you us on would

Erik Harris

Yes.

treatment. believe is the XX%, up And So patients that of treated. half adult and if of pediatric patients about market get think about on we patients is you about XX% adult about the the to the for current will majority eventually end patients pediatric penetration will continue XX%,

center. doctors community bone with associated we've these been these necessarily metabolic the of these most that many are are As stating, if patients with we're patients setting days, of traditional in finding that not not the

new find patients to – them prescribers. initiate treatment to and going time that's either or that them someone to those we takes it refer get So for treat

Operator

you. Thank

next ISI. from question Bayko of Evercore Liisa the Our from line comes

Liisa Bayko

Hi there, doing? how are you

Emil Kakkis

there. Hi,

Liisa Bayko

disease. couple I a just questions of wanted to on ask Wilson's

be efficacy? this initial in signals trial evaluate you'd any and doses. any And couple potential Like the If we there a to efficacy? that what should be for data be data you'll if talk preliminary you design, be for about can in in to you read-through able evaluate that just amended looking any looking will set? there's of Emil,

Emil Kakkis

on Well, all, of first all while. for chelator a therapy the in the stable been trial patients have would

in urinary copper. looking the be looking after through So the at that drugs a up drained chelator some their much their which But urine activity. remove at also copper then copper plasma, copper or part, whether copper through they loaded on in already is is tell with first ends sort very how looking through getting We'll at have That low at loaded be we're of amount their plasma urinary the we to plasma us not. and copper point. We're going versus will of we're reducing Wilson.

normal gene can't the therapy copper a that of of unique distribution, do restore with is you which we features one It's chelator. can

So the animal that really important we we get that's increases in in And ceruloplasmin a feature. showed significant delivery. could and we to model think loading

would dosing. will urine with basic bloodstream. probably So that's a that – the on be few measures, an copper the make easy be way and we'll biochemical decision other copper, Combine measure it from serum the that

So I as core be or various it the ceruloplasmin, of types copper would well is urine think, copper, serum it. as at

most transaminase copper biochemistry. off to overall. and is But driven think how it safety I patients Of there's course, be are safety, going and doing of

Liisa Bayko

it's well? understand us – disease. maybe give if Okay. And could I some the like endpoint background. you just Or of the is critical to regulatory kind that as would be that more

how curious on bigger relevant Just for it the is picture.

Emil Kakkis

Yes.

or people agency the regulatory using discussion been So on – copper. free copper, have some plasma preferred the urinary copper actually focused urinary

copper chelation take while And also endpoint going control drug, getting and accepted any on. but – so copper they're the keep urinary we'll drug we're low show on at that them look to urinary going off that while is less to well, won't loading their do the to it we they primary copper can control. have they're And –

makes that if sense. So

show we'll still to treatment So treatment low copper. urinary of and old the able rid maintain use to and their new be get

the approvals. endpoints that So One meaningful was has that because in there is that FDA use of program is biomarker-type that accepted the their the agreed-upon prior clear Wilson advantage already of evaluation. are chelators the of

Operator

question follow-up you. today our from a Thank line And is final for from the Yigal Nochomovitz Citi. of

question please. Your

Yigal Nochomovitz

follow to taking III. Phase much for very I Phase on wanted for an up the thanks or II Hi, placebo just Yaron's around follow-up. Angelman question the

Phase the trial, of II or in only substantially mutation it the placebo a do Angelman, literature X.X. suggest Phase some III you was placebo prior assuming analysis in below a effect know. As know, a our that deletion that the That you'll mixture of of effect the you and would open if was And patients. as based I'm around enroll deletion place patients, X.X. you

what your that be confirm in it could thoughts XXX% pivotal if you trial wondering that Thank And do would Angelman deletion that? you on you. a are patients? Just

Emil Kakkis

Yes.

only because not all around the to around other is plan do of pivotal will but think, lot variance, CGI score, a variation type, create Our the all scores. a deletion of including we also

the heterogeneity, there's heterogeneity. already more So last of – thing you a want you lot want

companion UPD, other drug types help strictly be patients. we'll use studies So will disomy population, and type, missense the some in the the the and the uniparental would or which the deletion other III type Phase understand of is like the of of XX% expand conduct that

for the if will placebo severe severe a reach study, label, are whole population. we trial honestly, randomized And we the want focus thing in population, be on prove the can but we stories other to treating the the consensus. easy cover So will to that

want so type your it, by it would controlling these effect. it in think it could extremely noise resist and if is more might I a because I even harm important of and even designs regulators – though create outcomes types, mixing

the not severe you ground. are on at ground history they not do patients, gain communication, flat, If in change, natural they they do significant look very expressive receptive

on avoid ground think some noise. that will mixing some with who expressive, gain by So patients you do we not receptive

of part tight it be to just FX and is about more it this strategy seeing. So we're confident keep our therefore,

Operator

question-and-answer you. any today's program session remarks. does program. the to the Thank Joshua for to This Higa conclude of like I'd back further hand

Joshua Higa

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

Operator

gentlemen, you, conclude the for program. does participation Thank in ladies today's This your and conference.

Good You may now day. disconnect.

Ultragenyx Pharmaceutical (RARE) 2 Nov 222022 Q3 Earnings call transcript