Earnings call transcript Q1 2023

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Good afternoon, and welcome to the Ultragenyx First Quarter 2023 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 an opportunity to ask questions during the Q&A portion of the call. It is now my pleasure to turn the call over to Joshua Higa, Vice President and Head of Investor Relations.

Joshua Higa

Thank you.

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

this Erik Officer; Finance; and on Officer Vice President Officer; me call Huizenga, Strategy Kakkis, Senior Emil Joining and Corporate Chief Aaron and Olsen, President; Crombez, Chief Harris, Eric Commercial Ted of Chief Chief Medical are Executive Accounting Officer.

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

turn the now Emil. I'll over call to

Emil Kakkis

clinical everyone. in mid-XXXX. UXXXX enrolled year, Josh, meaningful analyzing data and osteogenesis progress study good afternoon, part key X the begun data first generating for the fully release we The of portion of In programs. Thanks, pivotal imperfecta Phase the made the the of is in towards has data and our advance team the from

that closer in the therapy begin Around X swiftly us patients the Phase the portion X KOL we begin placebo-controlled patients study. bone much potential believe and and expect of to this roll feedback, same that disease. Phase -- on randomized to dosing the we a should we Based for move time, with

we've expanding in in study. to participate and Outside conduct GTX-XXX and This with with sites multiple Phase experience Similarly, activating and Europe. the In cohorts. had seen a number for dosing can U.S., of the possibly ex-U.S. and the discussions our are expansion Angelman, Australia Phase the Phase we've protocol have X/X forward feedback to dosing FDA future the with U.S. support we X have look sites productive in the begun of who GTX-XXX broadens the U.S., across X/X positive in on

compared first business, on therapies quarter expanding Five the world. to and access we the On the of significant grow grew In around launch in revenue growth. prior Latin XXX% $X Crysvita the the continue meaningful are opportunities in revenue our $XX million to commercial year. there America the still to million of in side XXXX, make progress years for to

next Kirin primary North Crysvita We commercialization program in responsibilities the chapter assuming last for U.S. in and with Kyowa the for America began recently week. Canada the

And We joint promoting will blockbuster in continue medical our we'll efforts to U.S. beyond continue XXXX. April the through therapy that, commercial geneticists. this

has I of our their patients patient top a smooth recent and with and In the disorders priority. and rare genetic Kirin want span world. Kyowa thousands therefore, the transition, experience years, in their most Ultragenyx in reached development as acknowledge around to approvals for Crysvita the planning and the to of community moved of two in late-stage integrated XX work bone through and patient efforts the achieving team with commitment dedication

I a comment also Orphan made marks speed want the Act. of type the disease. Peter to recent XXth anniversary rare the warp Drug on an year This by proposing alter approach to operation remarks briefly Marks,

has left diseases diseases While it ultrarare rare progress in to has behind. instrumental treating been

and We now and hope and to diseases path across make an for to have children the life-threatening progress forward there treatment offer currently opportunity children. to the many right no of are afflicting which parents these infants families

approval limited companies providing have will Too ultra-rare agency the I'm with I'm hopeful of genetic makes many pathway in clear to And framework the programs. agency with the diseases. fully supportive therapies to very -- halt resources promising accelerated small had new that urgency accessible supportive work the required. that

agency I'm is treatments community. that and new the we majority We encouraged lose greatly for fear the listening the diseases. that receptivity may these showing to of ultra-rare

programs, our seamless position together rare has transition going new role her closely for Medical programs to Crombez forward. experience full-time on Eric of Ultragenyx Erik want our turn a our therapy force and like over over as in driving gene call Eric also and it to He and in our grateful a I adviser. Harris, I'd welcome his Camille She's Executive to passion to Vice Camille new Officer pipeline, has on community. extensive been and advancing she and been clinical Chief I'm five development into thank ongoing the the worked her as clinical rare years for been has partnership I with and President. strategic to a steps instrumental for and both as disorders. the Before call dedication metabolic disease

Now provide to quarter. call our I'll the on update efforts Harris the turn an over commercial to for Erik

Erik Harris

top In Thank the shared you, with cause which line U.S. children and targeting in approximately in our generated we In the offering hope XXXX, a cumulative afternoon, $X.X years we is breakthrough April adults sales, with five everyone. the therapy underlying and of disease. billion partner, product Crysvita good launched Emil, KKC. their since the of

find In we up it would therapy. approximately in therapy. utilization Crysvita, all well represent patients of efforts to PDs. by start XXXX, the first the than on received approximately be led nearly they similar not forms to who in When XX% more launched adult of X,XXX reimbursed And reimbursed was make patients. quarter now fact, patient start XX% half, and Our have adults patients forms have whether X,XXX understood

This are the and U.S. bringing patient growing The is against the opportunities one most continue franchise. teams launch, of success important support to of goal still and well have plenty field be of this there and of yet driving adult executed and this the that work Kyowa this a our base their proud will growing XLH build and Kirin to from with forward. am support transition able extended with going team therapy pediatric strong establishing to TIO. factors of on patients I

disease thank the phase, be team successful to our efforts next relentless to to on move launch. American would I we from very led North As like this that a the rare

made America to of also has Latin in success meaningful overall the team contributions The Crysvita.

we by and all commercialization impacted transition, region leading the reminder, is will a As efforts. continue this the KKC not

team continues regulatory to country-by-country and obtain Our additional work approvals. reimbursement to

reimbursement where has reimbursement Crysvita. Technology creating received population, with patient growing the Crysvita a Most opportunity recommended be added Mexican for pediatric the of has Mexico list for recently, positive opinion. Agency the was to these treated to The countries Health patients the

quarter In approximately there XXXX, on the XXX patients first were reimbursed therapy.

the in the last approval with acceleration quarters to Over following in patients, two couple this pediatric has approximately grown happening of XXX years, the reimbursement greatest Brazil. last

patterns it to underlying grow. in quarter-to-quarter ordering drive in variability is the revenue, this continuing is demand As some but common region,

the profit the it and million Today, Crysvita Crysvita we revenue are the reaffirming regions all and of beginning year, noncash range of $XXX forms of More all the we issued in to includes at $XXX from revenue. the guidance Turkey, includes and and Latin Crysvita revenue North specifically, America transition. from the to product collaboration the Europe royalties prior share cash America million

there to the completed demand the launch. to indicators added leading of since first approximately had quarter, the a In show is Dojolvi. In is this I'll the for which start the product forms from to have we XX the strongest we continue quarters of top U.S., shift now real patient community. the one funnel,

Dojolvi. care the of on XXX quarter, therapy were for providers approximately reimbursed end one As prescription patients of the writing approximately health XXX least at there with

to have able of care to providers We achieve supported are educate begun look early pay forward these titration. titration optimal efforts ensure dose continuing off. benefits on clinical to to by patients dose the Our efforts health studies to the our

and Dojolvi. America, Mexico, receive injunction the through Latin plans. patients to continuing In leverage the commercialize process our insurance are in infrastructure existing we Brazilian therapy, commercial year, to first to begun this Earlier therapy have receiving patient navigated private

authorities work to important the enable Mexico to continuing We in the in and region this other countries are the greater Brazil, to access therapy.

Dojolvi. drive continue early access Europe, for Across patient named demand the and programs to

regions, said to Italy and France between and $XX we and announced coming beginning East the way, with countries. are million, at Middle we As the from before, Across Germany, have and expect Dojolvi revenue we of reaffirming range million the the certain year. more be $XX requests Austria leading more all

guidance also latest product, of product, to $XXX includes This total Evkeeza. of a Crysvita our XXXX million. million as revenue our the are as $XXX with range revenues -- in ultra-rare our the Dojolvi, reaffirming We well year beginning and commercial revenue Mepsevii for issued range

expected I it strong to to Japanese second touch launch meaningful the will begin from European, KOLs Throughout are Evkeeza continue the year, of outside teams wanted applications commercial received U.S. Aaron, country have and will over efforts they feedback base in I Canadian, the Latin in made on various to filing the this those that Evkeeza, have the launches American the year. of half support Before regions. that and the progress to hand in their

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

Aaron Olsen

$XX quarter revenue first was was quarter the European revenue revenue North period Latin of and the million. financial million included XXXX product in in that the briefly and Thanks, today, quarter, ended Erik. XXXX, $XX million press I time Company $XXX for the includes from release for noncash America totaled royalty was we the which which March summarize. America, million Dojolvi Crysvita million. $X will a issued revenue. from million, XX, $X revenue $XX $XX Mepsevii other for Earlier results same quarter for million.

noncash cost Operating $XXX for for $XXX Our were expenses expenses $XX of compensation of of of R&D include March million, the quarter expenses includes total the SG&A ended stock-based million. which million. of and expenses million XXXX operating quarter XX, million, $XX $XX sales

of multiple later-stage is to Similarly, for single focus planned release our to time. our we dose part safety. have that ascending dose portion greater were issued deferred initially the not press early-stage of decided to study ascending UXXXX we conserve today, Phase that programs company's cohorts and filings we and the this have is the IND have that we patients on at completed related in and product not of this the In enroll This focus X/X note for certain enrollment UXXXX year resources. to resources decision programs

million marketable do uses cash quarter are our cash, repeat quarterly For on securities. In $X.XX the bonuses. the the that $XXX quarter attributable or in share. annual XXXX, there payment a and the million $XXX equivalents primarily of ended approximately to loss first not We per This was basis. cash of with first net is certain employee of quarter,

support period. transition. will our commercial with the sharing commercial Kirin cost Additionally, North our full last funding Crysvita QX reduced of was period share Kyowa be XXXX post America substantially of profit And during

We continue be net $XXX in expect operations to less million. cash than XXXX to used

to who'll clinical update an Now provide programs. key on I'll turn our call the Crombez, Eric

Eric Crombez

everyone. you, Thank afternoon, and good Aaron,

would the before close of call out. I syndrome clinical potential back of the the treatment to Angelman two treatment brief updates of for Now imperfecta osteogenesis and like to programs, turning potential Emil GTX-XXX on provide our for to UXXXX key

domains. discussed, of Angelman disease neurogenetic previously is that severe multiple syndrome As spectrum a broad a manifestations developmental with affect disorder

least several for encouraging patients XX escalation dose cohorts. first in been more exposure at nine there In and GTX-XXX part including date, been have with GTX-XXX, patients of the patients treating enrollment we years To have the been for clinical have on six dose year. to of activity. therapy We months of patients and study, time-dependent seen continuous have one completed who than with

Based old; to in verify A, the we seen age. X patients now on to years we've B, the eight will eight plan we in XX between of patients dosing approximately dose in Cohort that which and years escalation expansion and Cohort GTX-XXX the Phase between study. U.S., four and dose have patients regimen XX cohort, to designed the enrolling treatment Outside in cohorts, be what of used and advanced patients enroll and are

be cohorts to Phase optimize the dose ability these be in the and as individually have expect clinical to titrate with the dosed benefit. Patients what the we will X will way to

enrolled the We expect to have patients months. of in coming the majority

have and U.S., discussions originally The the ex-U.S. with the goal In continue do U.S. strategy with in of the harmonizing patients redosing are with treated who well. to the FDA the the three dose ongoing protocol. were begun

encouraged are clinical activity safety that lower favorable are with We at doses we profile. seeing a these

making be nearly The and is disease a the of provides setrusumab We UXXXX bone makes defect. of bone action bone to resorption. anti-sclerostin while bone of into body's turning the and correcting that also of to a of stimulates metabolism. mutated increased osteogenesis to cells to or the defective production normalized setrusumab collagen college underlying mechanism strength without for limiting Now the collagen. directly cells maladaptive imperfecta. bone OI unique dual address believe amount abnormal those Setrusumab response treatment osteoblast the can potential mature

serum expected Phase show X change a data. generated, look the to substantial are currently patients. the are conducting for Mereo PXNP the percent and all to and We include analysis this a include on six data mineral is suffering and bringing Phase to that making has with the We CTx bone forward inform we stronger builds we The much-needed plan patients and and and treatment data to study continuing three Phase OI. changes Phase are available density. also lumber in data X month data sharing X help It expected safety towards bone path the and X that in spine to use dosing on forward for

call provide milestones key and back the highlight remarks. the upcoming turn now I'll Emil to closing to

Emil Kakkis

dose-finding half share XXXX. X XX-week we UXXXX expect Thank the therapy year. the summarize patient before of is programs. in patients the stage. this disease you, Q&A. dosed and it last and this start We're with our I'll GSDIA and clinical Eric. to earlier study up we now Wilson pivotal window, the key in for catalysts Phase for DTXXXX gene first data enrolling open for I'll

this expect XXXX. half in the safety, of signs expected of second initial in on XXXX the to of half connectivity complete data We first with

We've expect and syndrome when are the study with now an progressing, the Sanfilippo with review it available. a for dosed this seeking are program. they DTXXXX multiple Phase baseline period. to we're forward to A up Discussions more you enrollment We more accelerated FDA patients on patients sharing to pick path details for through UXXXX as X look screening in and make

osteogenesis fracture UXXXX expect in fracture in assessing the for the data, share would rates to imperfecta. and we're portion to to young to X a expected higher This have population, Moving transition patient begin quarter. pediatric of enrolled, been bisphosphonates, UXXXX compares initiate which a be much younger study fully these study study that Separately, this frequency. Phase to X The and Phase later mid-XXXX. has is a to planning we

Lastly, Angelman we continue clinical exposure long-term for belief GTX-XXX syndrome. could in to support change mentioned future activity my a Angelman Eric therapy that syndrome. and the have The

to you FDA We're We're next clinical of key our year. efficient data discussion also has investing cut our Our we later look year. in enable by and in and with today the and broader with geographies, X GTXXX investigators will productive network lot studies. in us making early a UXXXX the drive in which more our with been Phase a X sharing faster study to more forward this programs Phase programs and sites establishing and progress

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

Operator

question with Our Thank comes first you. Wang [Operator Instructions] from Gena Barclays.

proceed. may You

Gena Wang

taking Thank you I my questions. questions. have two for

for is update. imperfecta one X the First osteogenesis Phase

design So design, with on study to inform show benefit? low be year? what you sufficient be to that and too would X per looking for two to related Phase you patient baseline optimal Phase clinical will fractures X study Would the

OMS? XX% very $XXX guidance My to for million guidance. on Was more XXXX. second royalty U.S. Crysvita question before The $XXX is to the or after quickly that million

Emil Kakkis

Aaron Very I good. I'll answer go is revenue. we're then part the doing can answer the second ahead question particular really question. and OI the on how And the royalty think

we dosing. for? Phase know kilo might in We're older We young to to simply the the to know We whether are just trying might need the help out to portion a the exposure exposure higher do better normally same expect dose. and to dose. X its drug us X, Phase -- kids. figure work that We're we a with the looking works, achieve per XX-milligram OI higher what get fine-tune dose on can mainly the patients So looking

of is dose design number. purpose it trial. all set primary in the is that's The So finding the the just

time will the Well, number enough that's the and -- enter all, per have average And trial. design have also patients patient powering we've of of minimum look we you the thing long the should and the The the long at year operating going on fracture and it plenty XXX for a fractures XX first is could and decide could a detect asked months. power last at the the be total The of are done are with placebo patient should exposure the the the we -- second is recognize two studied. of we're trial months could last a groups what some how to be ones interim the threshold. look designed should patient comparing to study longer, an the change. be XX how be followed, be the question of and way is first or will question And fracture to how the or work amount key year look to study we've

adjusted power. based the trial And will do interim reassurance the determine the to have to be so help the again, of provide on we look length

rapidly disease, that respond is kids more than though expectation when they you treat adults. bone with much Our

differential So XLH quickly our as did, in treatment. come the expectation they relatively for that with example, bones will is

the royalty. So forecast is question what the for regard interpretation is second the OMERS part the to Crysvita with the of and

Aaron Olsen

$XXX OMERS The of million Crysvita $XXX guidance is the million before to royalty share.

recognize share. be will to OMERS continue all We certain portion revenue, although noncash a with royalty

Emil Kakkis

This back in to royalty have the and are way they accounting at royalty and to a we sold is cash looked cap house what income. be a the streams what reporting to come apples-to-apples year-to-year. you region, us that can still revenue income I recording compare have done that the the then needs so royalty also you is we picture addition is better royalty noncash gives I therefore, us mean the from it overall of the -- think

Okay. Thank you.

Operator

Thank you.

Maury comes Our with Jefferies. from next Raycroft question

You may proceed.

Maury Raycroft

questions. my taking for thanks Hi,

path get ahead you you received? key update? For data the feedback And on more considerations Or on just registrational is harmonization? if then design wondering dependent dose the you the can alignment of elaborate Angelman program, on the can

Emil Kakkis

I to... Maury U.S. about talking you're the assume just Yes. feedback

Maury Raycroft

Yes, that's correct.

Emil Kakkis

get we're really being what's it's detail. on issues. loading detailed that's make past, in end at What practical regimens. and we think to different our But a the feedback I how the that here the looking the we might the in is regimen to a doses it get really going, we don't different provide won't to we said productive, through goal we said slightly until decision. close regimens, meaning and in equalized be conversation and But Well, said is good Good. we've having try go is

from and would would Phase derive of. this But X we in we're run path is about trial try a I single get it, Phase program that to be. is -- plan Phase loading a But talking X section remember, could a and X we to would look that our what the maintenance

should able get get right out to -- a figured I I'm we encouraged be So this dose. feel and to

registrational yes. So on dose, the

So the the for expectation in dose I general, responses But doses of the will rare thing a disease have wells is about always. doses is dose. globally really one is and we're going in to in Angelman. There have the There dosing range no will say load. one

with on with may need or higher that of And Angelman along need individual expectation it. we part regular there will some efficacy may dose. a get that prove can less, become Our will individuals that is some and be the titration. for There be by

optimal important treatment, in that individuals their out, effect dose Phase here for you would way. point with have example, of that we The on we load study majority long X I reach situation. large Kuvan had of kind titrate in to goal in may just one did of Phase we the patients for in individual the a X guide an particular dose the and expectation where to to for own that get think the their the I'd titrate their clinical benefits need. those PKU patients to work and run show effect then that and can

Operator

you. Thank

Ha next Dae Stifel. comes Gon Our with question from

proceed. may You

Dae Gon Ha

therapy on question. if one-year question again. comment the treatment Emil, tachyphylaxis does if data wondering for And have good recommend even any previously, Great, implications setrusumab can on that's Thanks ASTEROID, setrusumab? presented afternoon. Thanks back our been bone appropriate. ask that's you biomarker chronic looking a on taking so much. Romos duration at I'll a for

Emil Kakkis

see is you're of goes you're PXNP look right you steady of look But each at kind I as so in it comes, settles dose that talking year, in. a if wave setrusumab the about continuous data you're it losing what looks peak PXNP you down, PXNP, Yes. think at the the will you actually -- if like at and the of area is the to it's of right? is And the you that you're not beginning the areas of end a then get curve, up. What's the as you're synchronization for think have really. now happening a harmonic going And the than pan, is So pebble the important peak the but a response, less essentially. losing and start. pattern, curve of a response the of sharp like you it's it effect, on

even At marrow If density entire XX, marrow a more fading you look XX at end at continuous, see and piece, was going eight, year up at was linearly at good not year. for of continuously is the will of you the bone the density it ongoing it the a at differently, the look at bone it little but data the clip end at eight. the

well So The view marrow information that if as the in continuously there's from reversal strong withdrawal indicates bone effect the based as for setrusumab. on of the that don't treat. of this that is you decline disease a information density our

as one after after a is bone we into, And that, years or to so say, normal could we a that maintenance let's density to is of regimen is our continuously regimen. to other marrow sufficiently expectation we're possible achieving you every let's go. look beyond And couple need say, will strong at level month an of that that and year. that It continue or back going treat some

right is now, OI think different osteoporosis. we from But

would from us As stronger get ASTEROID absorption and that And we've will bone continuous there's we production. stimulus you exposure. counteract continuous therapy, continuous there treatment now that a chronic for tell you with seen need everything to do need

that we different distinctly look reason. being so And disease at for these

Operator

you. Thank

with question comes Rama Anupam next JPMorgan. Our from

may You proceed.

Unidentified Analyst

on the is Kuno [ph] actually for thanks taking Hi, Malcolm Anupam. question. This for

read for therapy AdCom looking broader So that looking program? would that through could Sarepta's gene ahead, next is there in week your be you to panel anything

Emil Kakkis

think We they're of looking to going panel approval their principal in approval. whether The genetic is be in the trial. at think in Well, accelerated test specifics the I accelerated the particular to give own accelerated may And addition them really at important is there. diseases. -- rare really approval about our

think in that approval. will So -- treatments accelerated of developed. for think We Duchenne don't We program in not other fact, approval will we're Muscular that help improvements an better to support or approval be treatments that cause Dystrophy developed. be further

think I the that. needs understand to panel

From affecting and outcomes, other and be -- of the agreed programs own three to programs, other are our our -- programs is our standard approvals. clinical really at upon endpoints they're combination endpoints excel would drugs all of program all point, approval and endpoints, this with and dependent FDA, so have it's endpoints of approvals removal based on their expected of their whether what standard are for not set or on be three really the none the those biochemical

accelerated are of for true And in be you to a With I long-term that heparins But think situation, on treatments that follow-up highly companies. and regard sulfate reduction situation approach right sulfate, an We that's sulfate. if predictive achieving reduce I was heparin use sufficient have program, of that about think of of we to the thing. number that there think We companies think filing. Sanfilippo understanding MPS achieve IIIA will clinical-based good a approval heparan evaluating the is a outcomes a the number and biomarker.

get be be the to whether that these other we consider we're rare Duchenne it's FDA about or that on I not diseases, say as opportunity to meaning approved. say that, of the treatments that center going we to medicine and than MPS treat IIIA available think some more maybe to treat than But able approval is types we Sarepta to of precision that think using in supportive diseases would company, we've should a problem. pathway a more rare novel but biomarker there So us. been That should to -- an now have will

you. Thank Great. Q:

Operator

Thank you.

with comes Securities. Our next Lee Joon question Truist from

proceed. may You

Joon Lee

our question. taking for Thanks

CAS the ongoing seeing Angelman of you're latest On your the Thank that trial the what efficacy with thinking And a in still so FDA? Is or different? far, in signal of you. your approval part is it something CGI is also endpoint? terms discussions

Emil Kakkis

Yes.

mean, and we're important, So improvements I receptive there's in I what we've are and And communication we're continues. several seeing about domains. said seeing expressive improvements sleep. have we both there. I And before mean actually, talked before think

a of into communication to potential But and see powerful picking lot communication we a in pushed being of important pick we any important, domain that's were these. since patients. is unique if and So as one, would one we the

how and In those to our They could open But on them. point, what Phase X open expect on, to understanding X getting the with domains of important, on we're We've my optimize able certainly that be focus our to globally the discussions of the decide you any portion of plan have touched view, this into into our the to number the get endpoints. gone later are with year them. and for the program. working this them. Phase at now, study FDA we not But right understand Angelman on the

Operator

you. Thank

Yigal comes next Nochomovitz Our Citi. with question from

proceed. may You

Unidentified Analyst

Yigal. for Wilson taking from for that positive a We for put you year. data I Hi, can questions. part Thanks to outcome to on context into ask about of you. study? wanted expected and team. would Carly initial guess, the next early you constitute what is what help Thank show our believe you This plan disease the

Emil Kakkis

and dose, So study most just at will at look study, I we're effective evaluating. which look right? The in Phase levels, looking our basically Eric part the FDA, had mean we've what primary we'll at discussion the discussion is lead for the The and with purposes had on a dose we is to pick touched Phase That a and approach X X remind design. I'll study three and endpoint? will is of number ways. doses, X, Yes. touch the Phase what three But let the you, have is to on a it's Eric seamless right a

Eric Crombez

Great. Yes.

with important Certainly we copper clinical I that's disorder been a disease that's the is that established, Wilson measure mean, understand. metabolism. So of us to ways. And many for can precedents fundamentally, regulatory in

on addition -- looking of actual to are we the That's is in ways that what I'll to and guess, copper. call of for loading also measuring way trafficking assay, the copper place So because protein which be to an to copper, loading to establishing the ceruloplasmin. producing the activity-based I is at typical normal that kind transgene only for take measure of for the important,

be not is levels ceruloplasmin down. Ceruloplasmin themselves, if will copper. to We broken measuring quickly also bound

to this and increases that another show can we're of the in of we normal transgene if establishing So is way trafficking show stabilization levels. producing ceruloplasmin That's copper. protein,

looking yes, correct the again, that a the in key do, loading copper these tissues toxicity the and of a that which cofactor So for later as onto deficiency functional you're free copper deficiency meaning copper, need having enzymes. cells also also ceruloplasmin, correct copper of but we're functional to without

Operator

Thank you.

Yaron TD Our on next with Werber from Cowen. question comes

may proceed. You

Yaron Werber

Great. had us have there mentioned of two dose interrelated press at XX patients right nine a that are and months in give a mg on XX Yes. you Is dose? doses. a six that the little you're with different expanding year. least than Is an have more release I exposure at just now? also. the Angelman the Can Or only you question bit sense

the And release Thank efficacy well? only? second Or you're about that going in you update safety at secondly, to talking of point this year, that some data really next you. to when do expect as be is half

Emil Kakkis

Yes.

to patients, XX. loaded they're and between at X.X extension on load enter doses really the X.X X.X then there's phase, in remember, to and XX they and maintenance between were -- -- they a So

than actually XX maintenance QX is the dosing is patients all the that. all long-term to therapy That the But getting So those therapy during dosing is XX starting right? maintenance period. on dosing, lower are loading at

said haven't the current dosing back. Phase is We've expansion. our in hold we decided So that to what X information

plan as rise it we've dosing it a can -- and it from first have so of you to study We based other part maintain on what may seen that study. the we'll -- we lower our in and doses a dose we've the know learned certain far, where the in load higher

just The be in data the not year later safety. will

out information put to tended efficacy. the Our on

from both when data and provide the we'll and what long-term expansion through working we're the so cohort And separately.

Operator

you. Thank

Kluska question Fitzgerald. Kristen comes next Cantor Our with from

may proceed. You

Kristen Kluska

good afternoon. Hi,

number and next global years. potential wanted thinking around you're to ex-U.S. Just some these some how you maintain rights? considering candidates where late-stage ask of late-stage to I of about on approval and the markets footprint of in address your cadence leveraging your nature the a and building few

Emil Kakkis

thank Well, you.

So our commercially, Turkey have leverage multiple several infrastructure. company because one and Dojolvi of brought effectively we footprint adding certain we we in especially and global gap us the long-term America, Evkeeza could with we particularly it And felt territories, between leverage benefits that we opportunity Mepsevii. then an And precedent having in to approvals. had this in currently. by commercial does while in that, approvals Latin Globally, give approvals next more primarily, we the had

launch product added put sort Evkeeza to the so And another mode in of U.S. a ex

-- capital key the being important do done. the launch have, people teams of able And things One to a products of think leverage in high-quality advantage we combination smaller that. are that's we with lot of relatively disease, we with efficient can this. efficient we And that the rare of can how you that get

then about into think whether be set and four setup having several South new products disease. launch disease America, products, expanding up now three you those able well to the to the If metabolic neurologic -- we're or products in globally

what's the you principle to commercialize for real sets commercialize So important I was a beginning from do us up for to Ultragenyx gain and need and globally. optimally to think value, really that it

You can't just do the U.S.

all revenue you've globally pulling from to an get done the really product. approvable develop You once to need work

from that's to off as gain I pay U.S. and so outside we And our it's And revenue start grow starting to beginning. think the been the philosophy

Kristen Kluska

you presence Wondering looks significant in there's focusing anything have ASGCT this if we again. Thanks pretty And like it be you. at year. a you particular Thank think Great. on? should

Emil Kakkis

Well, X programs Is we've anyone Phase aware else. there's therapy gene think got with I that a of that no programs, than one probably more more lot in of. I'm true?

information and intrathecal some platform has some exciting our animal on strong well as a in So out gene there PCL and the very new have a -- which we and pigs. UXXXX, even primates also Pinnacle models which therapy is program, about CDKLX as data there's very in platform,

good and for AAVX So delivery that to to to innovations brain gene the and our enhance how an be about IND. we therapy, coming will one feel

that on data ASGCT. there's well -- that as So at

that busy So we and we're on. are technology there working with these all products

Operator

you. Thank

with from Jeffrey Hung Stanley. Morgan comes question next Our

may You proceed.

Michael Riad

question. Michael Jeff for is taking this Thank Hi, our for on Hung. Riad you

suggest First benefit one tolerance a for building the phase, for I dose during follow-up. out go, especially been and have Thanks. who GTX-XXX, maintenance the been on what are escalation? The any there are further learning you from nine has can patients year evidence patients for one have And to plus. you

Emil Kakkis

Okay.

happens the to these long-term any I a were certainly that dosing amount in things then a piece chronically is, if to think one very mentioned key you The might have drug certain how me safety important that of drug so event give problem needs be levels some of much a at from safety. kids many learn story, because matter the fact just a point we're first, you dose we at time. our -- of have accumulated, is

turn on start ability or important And takes way they developing, development you a the and once this mean can time. can is whether and The their language doesn't interesting and fact have instantly, given is develop develop, to which more think more long-term the means to important. can given below threshold we it disease. even be it drug thing give of -- it's this that's to chronically, some understand the The is do that you things, drug it they other we that

that that the we and in patients. dependent that we've gives we're improvements, working dose said seeing what gaining so that can and as is meaning feel we can we're like improving, a watch And us confidence change some these of people future and be time dose ground range effective and are

we're ability can both -- to still maintenance can fact So the safety gains. that's what we and from progressive long-term the dose see we dosing, learning and drug chronically that get

Michael Riad

a you. very then just Thank That's helpful. quick follow-up. And maybe

COSMIC, seems deformation and It much. deformation what patients. be pain? but bone about bone obviously, these measure Is these So patients? bone for to clinically ability going any so there Thanks fracture meaningful in the rate, is for measure to clearest that these be would

Emil Kakkis

kind with bone something where have of XLH the and all kind The while Well, to that readers problem developed work is We to it going that. harder you tricky. in deformation power. sort may that's patients regulators. it's help of know, you is did scales, to is us not help And really of thing

regard we but quality very just chronic being don't people good also life, have do functional in the pain, feel activity, they to are their bones, the trial. fragile those Now measured measuring in and We're because with also certainly pain all time. of

be them Even things and if go a they feel et that not sedentary, bad that fracture, have cause don't on active, microfracture, they have stay to make cetera. them

of functioning So on quality help practical doing the we'll the will of that what fractures. interpretation kinds and assessments be is support life

thing at But we see something you more two-year-old to look so at their is you deformed. of way deformation but in at particularly at the assess We'll -- enough take to then fractures, formally versus to one but an and x-rays learn case the think we how a haven't treat are I not or most and to would look a them, really maintained that. patient about do doing XOI important And our look and went one look want do them. thing. just from and appreciate that post-marketing I probably are bones to to need that's look to it's total as that we the

that dramatically didn't think I fractures old, have strengthen they five someone see who bone different who strength instead. if years years in the what they're several bones, for be right or we from when four you'd could their had

the And bodies into long-term. that these it's in you pain, that. because a a the program wheelchair, we and don't to place we necessarily able will to be of before future that's a want main That but kid But think So Phase it a the future got we'll change are change live starting in is patients. one up think will walk their life. this end X. be great their look young be for to about of destroyed I real but chronic doesn't something can actually And things we've

Operator

Our next Beatty Joel question comes Baird. from with

You may proceed.

Joel Beatty

question. Thanks Great. the taking for

the that are agreement have be therapy, monitor GSDXa you do are being And or gene the with primary will FDA filing on commercialization? harder endpoint? clinical for regulatory that For endpoints important there

Emil Kakkis

Yes, will has will in endpoint the as But that reduction looked we been and We're have based the starch, corn on be maintaining of just will glucose clinical on the primary endpoint agreement control agreed to. in not search while alone. at do primary corn There secondary meaningfulness assessment. advocacy be agreed. the using reduction that the that. part was be endpoints But other clinical

Operator

Thank you.

question Richter from Salveen Sachs. next comes Our with Goldman

proceed. may You

Unidentified Analyst

is for Tommie Salveen. This on question. taking thanks Hi, our for

efficacy? or of how you follow-up any details months there And of the here? program? could can we enrollment on from the pace to headwinds see on you on helpful terms about this interpretability So the Angelman update range on data OTC, question, and are cohorts thinking and of of be any in doses when Thank and Maybe would exposure, this just you. tailwinds comment of

Emil Kakkis

efficacy that in proper So put that is when putting and it we out context. what do need make we've sure we on to heard data Angelman. Yes, we're

only history not in this a around what natural propensity patients. does it's help the do doing just compare comparable compare and greater to create to confidence patient see a patients these normally dose maybe how natural But to to treated type the and exposure. match you where from we're history difference analysis So and you your score time patients patients between

So intend that. do we to

for for do. would also we the second other have what calibrate the clinically clinically? change, which But mean that to of The to it would in certainly -- thing patients those Is sense. big What is efficacy context So the that change? meaningful? the been effectively you compare sure providing score these will terms did proper be results can original help of is expect of sensitive how you probably their that we the give some on We'll data we patients. how element issue, we're of magnitude and five they to make is what you compared that re-dosed before, the to are does

Eric Crombez

timing and OTC expectations. enrollment was on Next data of the question

Emil Kakkis

right? about talking you're OTC, on therapy. were you Is gene -- the that Is that

Eric Crombez

Yes. It was.

Emil Kakkis

OTC.

our sites is of and effort the OTC And now. gene enrolling in GSDX and therapy study will growing the interest. we how depend and study driver there's just and as primary the on -- international we our effort. enrolling be to terms It are back drive. the Okay. is long, Well, is put open, plan push study It now,

enrolling not it's this be should XX. the because it So longer, XX little the year. study a that is timeline weeks, of study is But

So data gets XX weeks timeline last patient after would the enrolled. the of be

we if not the even be will means that obviously, I'll longer this year, the because up enroll time after before end year year, of next being -- not frame. that's data So end of the

Operator

you. Thank

Our Chattopadhyay with Guggenheim. next Debjit comes question from

You may proceed.

Unidentified Analyst

the Debjit. We this of to [indiscernible] translatability for PXNP BMD setting individuals your OI. translated on pediatric in thoughts want with on is to postmenopausal get to Hi, osteoporosis correlations the of

Emil Kakkis

responses. different is Okay. dose worry responses There Well, about levels enough PXNP PXNP and there really of we three ASTEROID don't to BMD. that from different osteoporosis, and doses because data different patients were have BMD XX at had

patients, show was later. day you dose clearly worth PXNP, on a was that actually one we and translate is at there year in highly patients what What predictive one correlation. So and that at have of dependent the XX see to that PXNP these is month found

you're The generate one pretty what's much beginning process much a and density. bone laying which way bone respond will motion that you marrow much you're sets PXNP But in a how how at so the is making is measure then interesting -- the down. really it of

our pretty how we're said, So study. impressed well BMD we're correlated. That in measuring they're dosing also

through to too. us about telling we which Phase will data us confirm is So BMD some some allow have patients our will the actual actually PXNP months for in dose with data, X six what directly study,

more dose terms a the have our PXNP optimize for to X. our Phase of understanding just X in little for than how Phase we'll So bit

Unidentified Analyst

of [Indiscernible] then sort that. the translate what expect adults in it you for in Is pediatric one-to-one? Thanks population? to

Emil Kakkis

the reasons the is to study relates response. the the do one OI to of actually with response determine Well, peds how adult

Our faster. higher metabolism, real patients expectation, PXNP that far active. more the are The their more they'll love respond have question could the they because to and begin surge other larger will fact younger faster have level they much bones that young with, patient a antibody strongly clear because metabolically is,

want exposure too. at to And we'd so look

us, at And exposure, tell what exposure -- tweaking the do the with more adjustment that the how different at exposure sensitive relates are young they if them of patient. needed the will they then XX-year-old same combination no. equally how so look really then patients, be to so age that correlates at at are, give do exposure at and needed drug? we'll is doses that and assure that can we the of PXNP more antibody to or that look drug. would different And of adults yes the if four-year-old than benefit ages help we'll and The the kind patients of young need would optimal And that us make

looking Hopefully, helpful. So that's of that's way we're it. the kind at

Operator

you. Thank

Our Evercore. next with Bayko from Liisa comes question

proceed. may You

Unidentified Analyst

question. is for Julian our question taking setrusumab. My for thanks on [ph] is Hi, This on Liisa.

provide you'll some data. BMD met I So

patients Thank data so, And and should is between correlation If expect? any factor? we you. many the worth how how strong density bone of correlation? there marrow is So

Emil Kakkis

Yes.

have them. patients We'll probably ago how variable they the group. some data have we So XX data data months more expect six enrolled. have three enrolled in this of BMD to of study. And amounts we'd were long month have PXNP and data in on all in We'll depending BMD

BMD does was, question the follow-up then So you asked predict fracture?

to weren't ASTEROID able dose, power there OI will trend was consistent, tell. the to enough the from in which at I study, adults think fractures they but enough have high in showed look the to a be

is So are strength moving increases laying with mechanism weak we're of or bones can the the the unstable. very fact that in restored anti-sclerostin down should bone that needed, in and quickly bone the is on this be be is basing how it's on where where where BMD what

And so just well. BMD the means should the very alone mechanism predict

the agents not phosphates, but anti-catabolic right in bone BMD with like predicted is, it may When where just keeping it place. you're this be hasn't wherever is usually

but right discrepancy this lay bone And it block been why right strength, the in in and make predict past. in will in somewhere, end the up improvement bone you it that's be when down the you marrow So bone you density place. the bone some not just mechanism places absorption, whereas may there's if

we But mass in bone feel strength bone more anabolic animal this well agent models increased bone with wasn't mass bone improvement, whereas did. very It that like bone strength well than strength that the correlate. will true, the correlate biphosphate, did the

in is So strength. a marrow mechanism density data this bone improving there's bone us give that effective to I will us think enough that tell

Operator

the Joshua This for like to Thank to concludes call turn remarks. session. over Higa the you. closing I'd any now back Q&A

Joshua Higa

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

Operator

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

You may now disconnect.

Ultragenyx Pharmaceutical (RARE) 4 May 232023 Q1 Earnings call transcript