Earnings call transcript Q4 2022

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Operator

Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2022 Financial Results Conference Call. [Operator Instructions] It is now my pleasure to turn the call over to Joshua Higa, Executive Director and Head 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.

Joining me on this call are Emil Kakkis, Chief Executive Officer and President; Erik Harris, Chief Commercial Officer; Camille Bedrosian, Chief Medical Officer; Aaron Olson, Senior Vice President of Corporate Strategy and Finance; and Ted Hazinga, Chief Accounting Officer.

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

now to turn the I'll over Emil. call

Emil Kakkis

afternoon, good and Josh, everyone. Thanks,

Healthcare highest the you we'll for on efficiency our JPMorgan areas around and completed Over year, where pivotal of for for the listened year, studies. may several on about we aligned have XXXX. while my Angelman, Some I programs from gene resources company. This in our our value-generating strategic at continue the data talked presentation imperfecta in programs to operational recent Conference and key focus therapy osteogenesis focus teams investments, clinical generating our last

than XXXX, $XXX across million company. commercial be from four $XXX year, generated In revenue year our fifth in as in of more to to $XXX revenue a indications. we our range the we expect This million million five global products

We as still the expect of phases existing to commercialization. continue growth relatively early are in products steady revenue a

opportunity the like and time, imperfecta, At business with we over positioned large steps. us for investments the GTX-XXX to have same drive made recently UXXXX from value for years bolstered our few Angelman meaningful last the osteogenesis more have and programs additional strategic

will the which commercialize clinical capabilities. homozygous for US, agreement our familial infrastructure Evkeeza secured countries we to and in outside license Specifically, of collaboration a global hypercholesterolemia and leverage commercial

of marker and in A therapy from based of study. syndrome generated rise global Abeona pivotal the for AAV to data strength the the acquired UXXXX treatment data Transfer gene We in clinical on Sanfilippo the

begin us of which treatment GeneTx plant our Phase full in Massachusetts, option the control data is program. for Bedford, we to our that gives the this completed to therapy build-out of spring. the We production GTX-XXX interim And gene GeneTx the following expected acquisition of manufacturing syndrome also over exercised acquire from Angelman I/II

scale production us rise control our our therapy the a therapy marker therapies, of acquired generated Abeona and gene ability This facility for gene Sanfilippo cost to grant as strength manufacturing gene better capabilities of study. commercial-ready on and We from of company. of data Transfer syndrome us based global the and the timing, pivotal A in the clinical in UXXXX the the the to AAV treatment data establishing

which exercised program. the acquisition the the acquire also for syndrome manufacturing GeneTx from build-out this interim completed in us gene Phase Angelman treatment cost and Bedford, full to facility our as option therapies, gene us This to better our grant And the following gives scale we data production production gene of is GeneTx the to of I/II capabilities of of that over and control Massachusetts, the ability expected commercial-ready establishing therapy therapy our us a the manufacturing We company. our timing, control GTX-XXX begin of plant spring.

in generated therapy of data Abeona We rise Transfer of marker AAV the acquired strength in from study. treatment to syndrome Sanfilippo A pivotal and the data gene for based the global the UXXXX on clinical

Massachusetts, therapy interim acquisition GeneTx acquire And which syndrome Angelman over completed that gives We to production us gene exercised treatment spring. control the data expected plant GeneTx begin program. our build-out manufacturing option for in Phase of is following the to GTX-XXX this of the our the of also full I/II from we Bedford,

therapies, of our our ability of gene the and facility manufacturing establishing better and the the a to us This commercial-ready cost gene as company. production us capabilities therapy timing, control scale grant

manufacturing the the Angelman over build-out program. for Bedford, I/II full expected we Phase therapy GeneTx to And Massachusetts, the control this plant production interim We that GeneTx also exercised spring. of GTX-XXX treatment gene us acquisition which to our in following option the completed from acquire syndrome gives of is begin of our data

us facility commercial-ready our and gene scale timing, control therapy better our company. to of cost a capabilities the establishing the grant the gene ability manufacturing as therapies, This us and production of

to for GeneTx exercised also gives Phase over the us option data following of interim acquisition our treatment syndrome of I/II the full GeneTx program. from that acquire We the control Angelman GTX-XXX

control we capabilities Massachusetts, spring. cost this our the the of expected of scale grant of the and build-out to And as therapy production Bedford, us commercial-ready and company. our is begin gene gene therapy manufacturing in the completed facility a establishing manufacturing production our plant gene This ability which to better therapies, us timing,

we year initiated We completed pipeline, II/III to progressing have and made and program enrollment of of OI Phase past our Within II Phase over strong UXXXX in study. a the pivotal program. addition portion the the progress our Angelman for

DTXXXX period. last for in advanced two Phase III into the baseline and screening We GSDIa enrolled the pivotal study patient have

first in for a patients additional And We study this initiated and screening in patient disease. also seamless the DTXXXX OTC, with Wilson Phase where of the dose randomized study III dosed in we UXXXX we of period. initiated in have pivotal patients study the baseline

company evaluated clinical ways in we our improve a growing pipeline, revenue commercial to recently operating efficiencies. As with a late-stage diverse

We established the actively and programs. and clinical on important launches will our focus headcount expenses leveraging from to our commercial global drive most infrastructure managing on execute value

commercial over call year. Erik that, last on specifics to their more the With successes and turn programs to I'll provide the

Erik Harris

all and the the challenges growth presented of Emil, they delivered afternoon, for and year-over-year some the everyone. Across teams. you, XX% by commercial XXXX was only variant. an early overcoming good the year field products, ran Thank important

grew first our disease grew $X meaningful revenue are can proving generate Crysvita Crysvita year XXXX was XX%; rare also from Mepsevii, ultrarare grew after growth efforts billion. launch. X-plus years XX%; Revenue the sales products global in our team's of Dojolvi product XX%. The where territories exceeded and

caring the total Europe Latin approximately Crossing our to North in sales in key the the threshold Asia. million made also efforts of Ultragenyx team's America across that America through efforts possible $XXX we product and led and and

In of April continue will on this we reimbursed expect in approximately strong as the Since in to approximately fourth we established added year. unique new start XXXX, that with we XX Kirin business Crysvita responsibilities U.S., therapy, along to quarter patients grow commercialization the in primary XX the launching forms prescribers. April we transition and base a have XXX of Kyowa

start and where to nearing metrics support XX% revenue annual the pre-pandemic are their are team XX% community harder-to-reach this clinics adults more particularly These of as levels the impressive efforts into generated. is growth This has in shifted than our forms region. finding

teams started patient Latin find to the and America, which and setting. Crysvita XLH, and approvals reimbursement In raising Kirin in have Kyowa combined and in awareness community helped additional us in pediatric to Ultragenyx efforts. continues supported our by the from of steady QX, demand With and in build prescribers adult many more grow, we also reach regulatory has

to XXXX more XXX to approximately prescribers and continue ended more patients will Crysvita. grow reimbursed benefits We experience on therapy patients in with this from as expect their region, this we the and

As broad total but which guidance. continues is grow reach variability of Crysvita In underlying in and of steady to at patterns we job demand quarter-to-quarter revenue, This a the pace. common in this drive region, will XXXX, Ultragenyx. the a the the ordering for regions representing better revenue to issue does Crysvita updated some we

all to and reaffirmed types Europe. share, issued inclusive $XXX America same XX% or of types of for XXXX At and today, profit the guidance revenue, to this year noncash. Latin royalty, and XX% between but is million regions the we of growth. range of This our Compared million for North Crysvita and $XXX beginning the revenue, cash America, of in represents

Dojolvi. strong begin The patient with with the where we Dojolvi, to in XXX one unique U.S. continues XXX start the grow, I'll with forms prescription XXX We now therapy. demand. with approximately prescribers the of ended to XXXX, In Turning by team's reimbursed number least at encouraged providers year on health writing truly approximately care now approximately work patients completed were for

educate XXXX, on the we to care continuing providers will Europe, titration clinical in dose on our be France In be are optimal studies demand patients patient to ensure name of achieve titration. access able the dose early benefits continues particularly Italy. supported by In Dojolvi focused to to and for led by and programs, the health

certain see America, in our Latin Austria leveraging the across infrastructure to have region. through work expand requests also process, In and existing countries. East to continuing begun to are the We come Middle we Germany, from

full Brazil, get from Dojolvi process In currently Ministry is Finance. of the reimbursement approved, and to approval are working we through the

$XX anticipate million, we also revenue reaffirming country additional the and range Across later between XXXX expect this year. We regulatory region we the filings month. all million $XX to last announced be in Dojolvi regions,

versus XXXX. mentioned This million and the million the beginning includes the and includes for At Dojolvi which for range $XXX estimates we also we XX% also I Evkeeza. over revenue, Mepsevii $XXX XX% percent just XXXX. issued represents represents and XX-plus year, growth between that to guidance are reaffirming today, growth total of Crysvita It of and

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

Aaron Olson

Thanks, XX $XXX the territories including December Crysvita other North territory the Erik. Ultragenyx that months of today, million, XXXX, ended net we million will revenue $XXX for results and regions. the which $XX a American profit million, in in Earlier million for release quarter, included financial share summarize. press briefly issued was sales Company XX, totaled I product revenue in $XXX

million. for XXXX revenue Mepsevii the in revenue was time the million, the for was related Crysvita $XX royalty to territory million, $XX of European sales Total Dojolvi period same revenue $XX

expenses of of million, Our ended expenses total $XX $X expenses the which SG&A billion, million $XXX for year and were sales $XXX of operating R&D XXXX, XX, million. December of included cost

stock-based compensation $XX approximately R&D of to Operating year onetime expenses of of expense and acquisition include the $XXX million a million for noncash related expense GeneTx. our

was cash, ended and $XX.XX net We equivalents with loss ended $XXX or share. year approximately XX, million securities. year December the the in million XXXX, For marketable per cash $XXX

uses leverage the and infrastructure clinical line. financial grow This portfolio. XXXX, are by to net discipline, growth of be top revenue as expected focus on significantly late-stage our to and our decrease our In enabled operational we continue will cash established

Crysvita these will will now be royalty to be of in While offset financing America improvements portion cash the our North by partially net partner. our that use

of us fundamentals allow will towards confident that the We in drive the coming strength years. are to continuing profitability business our

Now read the recap I'll the the turn catalyst. to to Emil back call clinical XXXX

Emil Kakkis

dual Thank clinical If action open resorption. body's for provides the collagen. UXXXX their cells to a the up you, we broad diverse those limiting mechanism response portfolio bone our bad Aaron. increase anti-sclerostin unique and setrusumab's Starting and more bone Q&A. the into address for to the imperfecta, osteogenesis osteoblast also production maximum stimulates before bone I'll mature summarize while cells from maladaptive making with catalysts makes unique to

total transition The are we fractures Separately, in that portion playing Phase to data assess and compares which been much fully study have expect to higher in patient enrolled, a to in younger the fracture initiate these share II a pediatric mid-XXXX. population, frequency. a III has bisphosphonates the we of study setrusumab Phase to young

Next, GTX-XXX in Angelman syndrome.

We of of been We are program who encouraging across in activity and dosed a expanded signs protocols. update screening began cohorts later provide the see number continuing data the larger patients this on year. and both under have in to next expect access patients recently expansion amended clinical based the to the patients

Turning expecting to to syndrome, to a approval to UXXXX plan the meeting have XXXX a for based file therapy of first we're Sanfilippo on gene endpoint. accelerated the biomarker programs. in With for discuss the FDA half with

patient to XX-week pivotal III this Phase this dosed, for XXXX. the begin, enrolled expect first last period DTXXXX Once share half the For in for in the been clock we we data year. screening the will of baseline has the this patient earlier study GSDIa,

DTXXXX patient for baseline momentum in the as stage. more we screening this first the deficiency in Phase in III it dosed patients UXXXX the year, Wilson will study earlier study make through the enrollment disease patients enrolling this increase period. OTC for anticipate dose-finding

mid-XXXX and data signs activity expect XXXX. expected clinical be We safety complete in with initial to of early on the

steps completed mRNA Based other single our and has half in cohorts in on review I/II we'll plans the the UXXXX, then to for program. Finally, data analyses work, of in have ascending these dose Phase the GSDIa of enrollment these XXXX. program we next the expect study, and our for first the

in XXXX. products have $XXX commercial Ultragenyx founded and approaching are later, of XX million. four I years we revenue

also next priorities. We revenues, clinical robust one in the late-stage of with have of growing rare new most the diseases. our pipelines company development of poised fueling for We're phase pipeline the now

a years, financial base, to will future discipline also while larger the opportunities rare allow us growing the of profitability In leading medicine. reach disease indication and coming revenue

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

Operator

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

Gena Wang

each I and will Angelman also ask one question osteogenesis for program imperfecta.

imperfecta, A use, in how portion? obvious enable and for response you Phase the the first, you II expansion used density for cohort program, in would For Angelman selected you And those mineral Phase the us establish osteogenesis B? are what the the biomarker midyear algorithm would for And the to how use can III remind then you doses the doses portion? X update,

Emil Kakkis

you, Great. Gena. Thank

been, in evaluating period. Angelman, So titration dose higher course, the doses of a we have through and

the put established out and maintenance put we we've the believe their of the been that risk haven't but some what we'll patients levels. more in We we're are doses be have out an benefit doses have using both loading information we we'll out We've using. exact information trying, in doses range that doses it that we some using, but when on of appropriate we've expansion put

comparing XX in ages With XX. regard both to imperfecta, to we're XX, adults we're children doses from and to five to osteogenesis comparing

of more achieve optimal drug using order results. to concentration is Our enough need in younger analysis pharmacokinetic that may to get expectation antibodies children the the

the to we'll bone So have and at bone PXNP, the and higher effect of concentration the marl young or as at also determine patients drug we'll data old, in not? younger in and and do is to compare the concentrations we'll production drug biomarkers look the well, of also and use achieve we to density the those optimal look for need some a the goal

will as at a do the tuning that little finding. you'll what We disease on be least patients this at in past. bone been than dose. based responsive patients in get to this our and think time, I before, younger at on patients read in The does treated -- that the the young know is dose question have look little than more we higher So which a dose need go patients older treated drug expectation the we've rather

Operator

comes the Sandler. Chris [Operator line Piper next Ramsey from our Instructions] And from of question

Chris Raymond

collaboration a I've on don't you guys question heard about. one I Just think that talk

maybe guys would just think, wheelhouse disease. they a asset that to collaboration would that XLH this be Emil, mentioned started that with phenotypically today seems I find seems something Enozyme ENPPX developmentally. overlap in So It misdiagnosed you would, to your fit patients. to within seem with

of thoughts any had of going on had in curious guys kind thinking collaboration Just maybe today you that line and light the itself, least the especially and at this on drug thoughts forward? if about data how any you're top relationship these this

Emil Kakkis

with help just all planning the we're diagnosis issue with and share lot of our find information point, work we not a any and each companies patients products this We Yes. on companies, of other. not other trying portfolio. more are At to that on bringing into

the and watch but -- have to including point, need what partners in do this our we continue plate all opportunities finding is and on the regard our we to Indesign field for rare others we working at diseases. But And something with with field, others patients doing new we and manage and routinely. is to

Operator

of Joon the And our from next comes line from Lee Truist. question

Joon Lee

to where safety to maybe issues, you formation exceed. for bone in growing patients, of want wouldn't bones young With biomarker too is there lead loss, for could much formation issues, particularly Are there the potential level safety a data setrusumab, example. hearing bone potential

Emil Kakkis

kind question growth whether much the nerves patients So otherwise, is the in impinge are you too production. is think in bone very bone of hearing deficient stimulate their can or truth like I and these

state. down get while genetic deficiency think, for from a different level to deficiency. the we're from these the to required, defect of very genetic And sclerostin, us might cost is place a starting So if ability the you degree you a had of of problems I knock

are patients of Secondly, deficient position very I would the at beginning quantity. a say, bone

risk get maintenance resorption, strength we -- dosing. it a let's improved we the mitigate, where patients them having kind. of at can long-term which bone was point think their and frequent we study treat their looks a based loaded establishing establish, maintenance strength we'll and And there treatment less bone like asteroid example, that look say, grow, stopped on of for we we've -- As improved, if fully dosing at once was at after we the I would a and looked

day. are But the right it going concerned of beginning order for this of and But in are bones patients of drugs we'll not out now, that's face always effect disintegrating managing every forward. it, but business, suffering at point, about this watch Their these program. let's we're first the our

Operator

the of our question line from Yigal comes And next from Nochomovitz Citi.

Yigal Nochomovitz

which obviously, So the a are anti-sclerostin. quite obviously, know, romo is on OI lot comes getting we lot that questions you of conversation, program lately in and up a as

with the be potentially us help could and you quick romo, setrusumab elevator if option your pitch on why differentiated NOI? the preferred So is from will antibody why

Answer

Emil Kakkis

Sure.

of designed XX optimizing osteoporosis going not be the of What not and that labeled was for approved doing we have that developed indication, in romo to treatment, imperfecta all, and for area. dose will a treatment osteogenesis limit. of we're of months is limit osteoporosis They First and indication. the have

We will will disease, lifelong. which in which dosing, is chronic be able to important be this have very

by secondly, a dosing treat also way able as specifically. company rare an we'll patients patients optimal be optimizing be And disease support to in able regimen, the will to

combination the regimen will romo support setrusumab differentiate what out and the there from right of So dose, now. the programs at

factors, be So will think, some the of those I important. are that

Yigal Nochomovitz

the dose, versus year-old the of you're on is dose out the head-to-head And five you're question just in will two study the when read you used the follow-up going selection the the that patients in to that middle a Once the year. OI that for be younger select in post? that same earlier Gena's comparing regarding dose to

Emil Kakkis

We well. needs dose isn't dose, dose, then over that study Well, would expect to to that adapt it the be we'll for higher, XX if the higher, be if other as example, to. turns to out

Operator

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

Tazeen Ahmad

UXXXX. follow what Can opportunity to like facility do you for wanted support the would on in going us Sanfilippo the today? of think competitive landscape And there that And the follow-up then on just forward? patients quick new just remind a are in therapy maybe you up how manufacturing many I looks all syndrome gene the abroad. your one, Emil, programs and U.S. on your

Emil Kakkis

Thanks, Tazeen.

do the wasn't one we decisions of very picking IIIa, around well. was Sanfilippo And we was with make there good So view as a whether to there had could program option that was really. in the our up just

standpoint I The they was effect. they're that actually product, are product business. better out lifetime, a -- other of program biomarker than in from reductions going think, This in of

think early on. lentivirus own effective There there really, think, has fairly stage But which another, therapy challenges, a gene is at approach, there says. it's AAV the its that I isn't I that's out

our seen So I've this and feeling biomarker best was the be that from the a had would program at least that efficacy lead standpoint.

was that our decision. So

kids appears regard the a this the a bit have of With thousand population, you more to like two to be little regulatory perhaps, of on years think process. to we the an probably few a treat Because that in the it best older right? was bit happens Zolgensma, there's best get patients, what young are so under would benefit world, need effect depends to little -- in the the as population old, patients, but of they to it incident be

Young. benefit it's there think clearly benefit be you when treat the optimal but in may happens I patients, older

won't get treated doing what none is doing want because this this If you other see every gene options sample we What other to point treatments. it So of some way. the believe therapy I the we I other and unlike time. would that's are add diseases essentially we just look a gene there was now, patient are saying right where therapy about at at in that we to think

of has think that high And I can and would promptly. devastating rate get so have approved very impact the the it if adoption we kind SMA, you it be with

two that other capacity. plant runs has can One was the suites Now operating manufacturing the The and run XX right is now. a year. question plant suite

that will talking you're handle gets don't need -- that. runs needs operate what the on we'll a we we of because I year We of programs suite. XXX% achieve can done. which second it about depends lot know XX to a

rate, some have greenfield always plant plan our right a long has think the we that XX we some own. can take runs green a And way long our will us many manufacturing necessary, a But take But could we of And moment. further. need Our double that plant some capacity to treating if that and at we're a if now, then we space space I hybrids, do have the high-level plant success what therapies, for will way expanding us contract it that been to at gene invest further. in next the future. in

Operator

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

Jeff Hung

XX? talked Evkeeza the that incremental per of talk see with setrusumab, the you've about or doses the would to for Europe? guess hearing about two what XX-milligram benefit the the over I launch you then you dose can kilogram expect kind in of feedback been want And you on

Emil Kakkis

gradations think with we we idea, setrusumab, set were rough -- really I concentration production, give production. more to look want the you more haven't say don't as it. worth to bone I We bone and the So you dose of a if gave drug you XX% if criteria. gave it would go it. there precise But But if XX% along. worth at that's drug effect it's then doubling

we more I more that and gives would would be a be that that if is think kind giving something what meaningful for rough of So drug. warrant spending looking you idea much to

program access had and there, and with And because part think uptick get the think reason first on to regard will so can drug and we've With interest depend be partner the that place. in had strong to in use it all that we see reimbursed and out of decided from And I doctors about given few [indiscernible] both it. a is there monthly we're we a receptor just great as we IV -- the I get Regeneron really if really and in doesn't it, patients in a first LDL feedback can at tremendously on the treated. we've positive on requests potential excited Europe Evkeeza, the compassionate good

Operator

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

Unidentified Analyst

This on Salveen. for is Tommy

you for any terms here of you number remind this can data where a of in what year, coming benchmarks duration, the some positive us or signal? So the see are bars of UXXXX would details patients and

Emil Kakkis

the program looking that an and release that's in for branders We'll is enzyme this or around glycogen changes will of response, help biomarkers accumulating UXXXX etcetera. metabolism. biomarkers fragments producing as are well Certainly, glucose glucose restore immune as that LNP be mRNA and safety liver

So it kind will primarily a both be of biochemical control as at as look release fragments. of glycogen well glucose

get those patients two idea cohort. dose of help things were kind So that should an us about dose. escalated These single-dose are all single-dose ascending at

we will it a of primarily So what achieve correction first be terms of on metabolic indication can based misers. in biochemical --

Operator

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

Maury Raycroft

setrusumab update, XX. two are Phase I was age if have you'll out the the But each you are older what for doses all the is breaking and younger? range of wondering clarify patients versus can to at five you II proportion

Emil Kakkis

Yes.

two all their least what will have So patients patients those is through have including the data for at -- XX for months, in. we'll last

at were the of started of done will patients months six time that last For year points. which will some BMDs were that data, have interim include maybe

kind of So that broad be will of set data. a

determine at allow look both much have to -- distributed We'll try doses. exposure and we modeling biomarker groups, the need lower we we We age are need ages will how decided that at to the whether to look us have to and disclose. will to PK increase around haven't use ages age and at range drug different that the we different the

Operator

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

Dae Gon Ha

GTX-XXX. update maybe FDA submission fully any on Maybe on protocol the time a that to Has the scoliosis line dose? of the Can to the for of provide question harmonization? updates package the resolved continue patient And you receiving any patient?

Emil Kakkis

said patients, a resolved as had. the The before, we resolved they essentially problems Yes.

evaluating we're to that yet. think I not continuing redosed him. He's

how which will second We encouraged at his until levels have was intervals measure, protein we do to resolved, wait that's gone have and yes. question we the but -- will his on and wait until we're

how through ahead U.S. good expecting expansion meeting. them discussions we'll to with presenting there's we're So gotten discussion we're we've moving opportunity cohorts like ahead. I and and shortly, the with move be We planning an in approach think a to feel lately that responses And good

need amount formal will position U.S., to being of about protocol confident the enough still that we and think data get to a be to we important But up in which a designing the in open be study in rapidly in Phase expand able have discussion put to the III. order to large us

Operator

question the our of from from comes next Liisa And line Bayko Evercore.

Liisa Bayko

Could you -- with royalty the kind royalty. profit run changing that the just split, the how from just of And in net you to Can helpful. us that you'd that? getting to is through somewhat would the seeing from profit of the you're remind change equivalent what be be

Emil Kakkis

Yes.

you're share So talking our talking a profit about to from what That's the to partner, period. be clear, you're switch with Kirin the royalty Is about? correct? that

Liisa Bayko

yes. Exactly, Yes.

Emil Kakkis

There won't of it The actually be. really. general, made be be should pretty modeled well way change, we share will what Well, structured a predicting essentially significant our originally even. crossover that terms was in in

expect of top commercial the the So the and tier XX%, costs. to cover royalty cover some will

We them. sharing costs with some are

total of designed picture how and similar. pretty how was if -- at the is But you this share the it's profit at this flat, look point, pretty

off very that we there's more, good know feel that. you significant you in don't don't if And to see wouldn't so or stream. say revenue a to want anything step the Aaron, I change

Emil Kakkis

that well Thanks, summarized. Emil. No, was

Operator

Instructions] Our from Anupam [Operator Rama JPMorgan. from comes of next line the question

Anupam Rama

me. Two quick ones from

your accelerated us maybe with the programs. that scenarios FDA to that here. broader with an Following path for calendared? on program, up III And you And has forward could read-through Tazeen's MPS is the if and walk to meeting been any UXXXX on out been through go play there gene question therapy are able

the question in that on a in helpful. statement on be provide the with press And of what That seeing then expand time activity any dose time-dependent continues you could two, period? type and you clinical GTX-XXX. And color There population. maybe if to be you're type maybe the could release would over benefit what of

Emil Kakkis

events and is a precisely degree already the a authority Yes. is endpoint evaluate. them and discussions, that we with But of meet meeting FDA the Normally, longer don't on point planning with the shortly. agreed set, exact clinical meeting put with the we're and does had meetings out of to time regulatory of timing to the period that

they However, started as that sulfate predictive. acceptance late, of some have of showing heparan is

in with data. to than as And the as allow Peter sulfate Marks waiting marker for believe which the file earlier settings possible our clinical and discussion would may us in primary, heparan public be the we other

heparan of sulfate focus will on of particular for to being discussion our filing clinical used clinical purposes we with data, use to the verify a the follow-up can complete So course, benefit. of that's assay commitment the high-quality

come faster, will that mean up lot we program trying it we answer a just question. If soon can our what just to means is relatively have faster. can as the we'll have still on set run allows we're us And do as the that that because a plate. so CMC we pieces doesn't to let go It know to the possible -- a to as file program. soon. And it manufacturing of little you We the go And know earlier. we efficient is way in capital

some was there disclosed and time-dependent So GTX-XXX, differences. we dose that

mg the were put out about groups mentioned dose Bailey We and this lowest before. the at the quantitative endpoints the two around have had talked we we the with in U.S. group dose that information fact actually When with the earlier, better higher

improve to said piece. that's patients the time-dependent So seem over dose-dependent piece. time, that and was the we've that And

ones we're with So things, cohorts. many and talking disclosed, multiple in evaluating the and drivers into we're which of things main moving we're looking why already both the dose are language we've we're it's the we about and seeing about we've -- been scores at time-dependent seeing and response are in but feel And Bailey sleep. that's pattern the it's good and information expansion

Operator

And from question our comes from W. of next R the Beatty line Baird. Joel

Joel Beatty

For revenue, QX any year of are last of of? from aware to effects year QX be this there and seasonal

Emil Kakkis

particularly have expect think there did can burst America off but And terms seasonal always more with Well, not actually, Latin Aaron we in But of hitting you lumpiness always would I I'm are think well the I on. and effects, think in because year I sure marks. and to say. come anything some our orders last

just bit little lumpiness? business. nature predict. Erik, But well. add else the of a part it's rare I so think did to have to are things of you the harder And on It's disease anything

Erik Harris

I Latin as think important gold last it recognize five of we've we as treatment expect and indicators, and continue key concerned, it's we've the as in consistent finding, is increases as start Crysvita. for prescribers. growth patients performance covered that had unique mean, America seen It such for establishes You to reimbursed steady to I forms, far XLH over grow the patient years it to standard

back do So in look the experience strong. has some you insurance lighter seasonality which reauthorization year-over-year, consistent as That previous changeable been patterns expected demand as QX remains we little a QX over said, through and work ceasing if in patients process. QX, and being ordering years. the

reaffirmed So it confident get on But remain just why little to takes our which the we demand and guidance. longer we on. a is in underlying

Operator

from comes from question Cowen. line the next of Yaron And our Smith

Unidentified Analyst

[ph] on Brendan is for This Yaron.

answer was to did just Angelman. forward? I on this underlying was a hoping And wanted just call you up if relative hold that any ask noticed really to some jump Crysvita, then my second. as what us I QX for a if expecting QX. just in trends that bit. you can you're But Sorry, QX forward give maybe just for sales to hold is to And phone of kind really moving I color

But just you bar maybe patients a include readout to of really just how out to and in kind sense here expect us know in of of sense whether in things get many how to for thinking you're data. day when I next of of to expansion long of lot dosing can terms the a we there's where all cohorts trying on this. really the give include a what could and questions like over they'll put from internal is want terms you them the the you

Emil Kakkis

on touch Sure. agent And you can strong the first. question Crysvita maybe, QX the Well and I'll the then Erik, question. answer

study. relatively So clear been small these can enough XX costs, of were at and need kind releasing. on just doing like a each projection Angelman, statistics But data in which something of on power how least we group that it's groups. at us do been for to patient kind understand to give the dose we've do to we to We've least titration have patients

would what allow idea. what is I giving would maintenance which XX to have have Phase rough have in for of confident look announced titrated will maybe now allow will the Phase kind is volume see will for That's who program. them for data patients need about the the I III in looking that to I'm project patients enroll. be like, And of a a that the data ask have or what to investors to So of we in to expect us would mode, half been III you accurately addition, already we program that the think and we've would us treated future

think want that. a to to that's Perhaps pattern. seen Erik. So pattern before, I Crysvita add And the it's you we've for QX,

Erik Harris

we as more meetings strong educational main the was expected in hampered more live And increase had We well We were throughout the driving they saw demand as of in the offices patient growing continued as second volumes Yes. XXXX I Omicron a year factors QX. up the year with strong programs. of as one opened the early half in variant. consistently in and participation think and of the

Emil Kakkis

of first in but a bit has strong it's generally So out it the rebound of QX. hampered a the of year, part been

Operator

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

Joshua Higa

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

Operator

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

disconnect. now may Good day. You

Ultragenyx Pharmaceutical (RARE) 16 Feb 232022 Q4 Earnings call transcript