Ultragenyx Pharmaceutical (RARE) 1 Aug 242024 Q2 Earnings call transcript

Company Profile

Participants

Joshua Higa	executive
Emil Kakkis	executive
Erik Harris	executive
Howard Horn	executive
Eric Crombez	executive
Huidong Wang	analyst
Salveen Richter	analyst
Dae Gon Ha	analyst
Tazeen Ahmad	analyst
Kristen Kluska	analyst
Priyanka Grover	analyst
Joon Lee	analyst
Maurice Raycroft	analyst
Will Soghikian	analyst
Yaron Werber	analyst
Michael Riad	analyst
Jack Allen	analyst

Call transcript

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

Emil Kakkis

afternoon, good and Josh, everyone. Thanks, incredible We've and of year half forward had to an look today. sharing the you first more with

the positive can this our we strong the On revenue guidance we're our puts happy our drugs programs total access data clinical front, on with to We're the pipeline, us what raising have revenue our revenue trajectory for projections, critically interactions. prior advanced on performance and outperform range. and means important so to through globally, and readouts multiple share Eric and successful details. Within more and late-stage meaningfully Howard our regulatory our commercial

Angelman On results with the on syndrome, positive announced the positive this from treatment to Sanfilippo antibody the year additional and addition the both positive earlier front, Type data results UX-XXX gene for we in DTXXXX syndrome the we Phase therapy in for patients recently treatment Imperfecta. III UX shared with glycosidase in results Xa, of GTX-XXX and patients Phase Osteogenesis from XXX II long-term the of

of for completion announced study we on and evaluated. III Angelman we to of with endpoints end FDA, be the key syndrome, align Phase II successful where design GTX-XXX the Phase For meeting an

agreed FDA our reasonable path also study agreement have by of of study cerebral a to BLA we our surrogate submission accelerated support has fluid data by to syndrome with to teams this Angelman a sites the [indiscernible] heparan year. On clinical seek The approval. is date. working Our endpoint reached with forward global been A, spinal sulfate a to supported our type end initiate on to Phase III UX-XXX for

developing a the and next. Selectively, have; this Harris, ahead have details drug to excellent marketing with all submission things multiple to to pre-BLA submit later intend regulatory from very across development. is are our the us puts a with a disease key total my this extraordinary. you these next the next of achievements to exciting early never finalize time agency forward. These our depth with clinical rare result is of now for move disease, and the and our us ability to in I an direct revenue It's Chief us we to our execution call our position which or BLA this Ultragenyx. I'll approach Our can on breadth months, a momentum submissions commercial update and committed tell to nor to for fees meeting, X to career year Eric our raise we opportunities Commercial guidance I company step rare in therapies the the and led the in over spent I've XX a of data of over our rare company has seen best-in-class that turn our across year. the Officer, provide portfolio readouts employees

Erik Harris

adult and everyone. XX% performance with encouraging, continue forms to QX I'll ample in afternoon, Crysvita physicians, in new Crysvita's the in U.S. community over is adult the growing. by in for Emil, you, of United States. XXXX. Thank good from came demand in The strong Approximately stock the the remained start patients and XX quarter. has room penetration XXs, were low This given Crysvita implies prescribed prescribers and the is with

commercialization, another on XXX delivered Crysvita, in strength we year patients the lead of our are the team LatAm successful Latin We quarter adding by over XX new revenue given where demand. our see we demand. Shifting totaling region. confident by Mexico, Argentina patient contributions beginning and largest Brazil revenue are reimbursed therapy full like America, in since projections patients is supported launch. Crysvita to underlying more approximately of U.S. to in the America the That the and Latin market continues drive from majority the underlying meaningful to said, countries to in

As I revenue demand for remain calls, to in uneven quarter-to-quarter patterns, but our we growth the expect LatAm confident previous on in mentioned due ordering underlying products. earnings variability

forms and patients be Moving XXXX, therapy, them in added XX% The in patients on XX% patients XX approximately to start we peds Dojolvi. of between number on prescription. XX growth split of new adult and writing to half new approximately new reimbursed and X resulting starts the approximately In to grow, XXX prescribers of than adults. remained prescribers reimbursed continue more since continues QX XX The approximately adding strong. with launch. pediatric U.S.,

under approximately Dojolvi, region, majority is across an currently we the across MENA driven named number countries France, increasing request. the patients The EMEA from XX Middle of Europe treated requests MPS countries revenue is in within from demand including but For are East. region, XXX are and by region. other sales There the receiving the patient of

processed being the meaningful where treated option. [indiscernible] approval from April. processes reimbursement have As before, all XXXX has very approval. done the in reimbursement through gained In starting, regular second launch an XXXX. In and of important and year following in January and XX of stock a region, already our Japan, physician continue to team HOFH there excellent EMEA is majority new and I the patient are a The we added job as who in regulatory feedback have treatment patients MPS country we Crysvita. patients positive quarter, and the said the they has is contribution identify in quarter we communities, forms receive an in for the second and XX pricing to excited launch to have approval map And territories, has through but approximately and are

We this HOFH. as with expect important we growing to patients for bring to continue therapy [indiscernible] demand

was QX a strong for Overall, XXXX quarter Ultragenyx.

$XXX across off results through more million bounced turn gives of first and quarter quarter seasonality months that, the that Howard revenue. guidance call broad us quarter in portfolio to confidence X put second share year. With leading beginning range the the on will we first expected, the the a for the financial to commercial As year. us at strength year the I'll we the for that to total guidance exceed our on the the The established trajectory of the details

Howard Horn

I'll start and good that by in earlier our Erik, reported afternoon, press Thanks, our release summarizing were financials today. briefly everyone.

million, As Erik million Crysvita reported for including second in $XXX of and America, and $X total quarter revenue $XX from was Turkey million second in million $XX revenue Europe. Dojolvi million. was million, quarter the $XXX the XXXX. North from second from America $X million the was $XX noted, quarter contributed the $X revenue quarter and revenue in second we in million Mepsevii [indiscernible] Latin

$XXX of million, or the of million. Operating $XXX sales share. were net in million quarter the expenses which expenses SG&A included cost of million $XX $XX included and noncash million. In $XX operating expenses total per stock-based loss million, R&D expenses of second quarter, $X.XX of Our $XXX second was compensation

in As million we proceeds XXXX, offering from for net million June of quarter, our in included $XX of was million half the cash of second operations $XXX in had first and the cash, XX, which the total was million in $XXX securities, used equivalents cash marketable June. Net $XXX and for year.

on seasonality like includes As our items call, it of the quarter bonuses. payment first because in discussed we the there annual is May

$XXX less operations guidance all million range than year. for to all end guidance, we increasing and $XXX remains performance now expected unchanged Crysvita in the our be forms includes cash our and net million and for for Our strong of to we This of of revenue expected million the and Accordingly, to Ultragenyx. in revenue, be to globally existing regions trajectory used between the across [indiscernible] of revenue and launch targeting are Crysvita which our reflects our products, upper revenue range including of $XXX Crysvita $XXX territories. $XXX which to total the towards year. Shifting to million, XXXX are million be for is is all

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

be With I'll between Dojolvi We Eric turn million to Crombez. the continue $XX expect to $XX and the CMO, million. that, revenue our call to

Eric Crombez

Thank you, Howard, good and everyone. afternoon,

the long-term approval. a surge cognitive was UX-XXX heparan accelerated IIIA. be of with correlated sulfate used June clinical catalysts of demonstrating can of This The reached function. the sulfate a we that improved announcement data clinically that announced number heparin significant with agreement treatment important up half first has for endpoint year for as FDA our in in with reductions this followed for seen that We [indiscernible] MPS

year We this goal expect around the meeting to details filing finalize BLA the pre-BLA with of our later the package in filing XXXX. of a of end

of of DTXXXX resulted study with For XXXX. a GSDIa, Phase that treatment maintenance announced a meeting with from DTXXXX from for at daily the half control. Results reduction statistically pre-BLA III XX in of with second cornstarch data treatment line will in regulatory strong in that discussed significant top glucose this study the be the week in authorities we showed positive

and the the For of XX% Imperfecta, a and UX-XXX, fracture fracture data annual we X.XX. mineral Phase Osteogenesis continued mean in reduction of persistent portion rate XX in sustained in mean setrusumab also XX% with were median bone improvement for X. score substantial of a with Orbit showed density annualized month that improvements treatment of II from Z There increase of baseline in resulted rate announced treatment a in from

the These shared at that the or cohorts clinically those treatment meaningful data escalation of rapid improvements of for I/II across consistent cohorts showed multiple additional data syndrome, [ were in we April, ]. domains. XXX Angelman expansion from For improvements the GTX-XXX day and exceeding Phase dose

the in data long-term escalation in by lives showed benefit feel sustained of through of dose a XXX flow and recently, news increasing additional clinical good improve lot we about ]. that cohorts shared GTX-XXX also space disorder. patients the been day affected There's to We and this [ very of ability the

portion activities. Last and We strong we randomized developing on in patients across believe we the of no a advance with as in study serious multiple meeting for position continue III we skills global blinded FDA, to a new XX-week start-up Phase adverse Phase with domains completed III where a design Phase study. successful II the month, the sham-controlled we Phase are events, II end the the new see we of aligned

communication, deletion. between we secondary behavior, motor of will endpoints secondary by enroll the in also and study domains a cognition some respond us to communication, full ongoing also design, to be evaluating expect for and we II will study X motor types on activities year-end. the behavior, for by sleep. a assessed and XXX and Phase [indiscernible] begin who already function for X in alignment, to are Phase index, endpoint improvement start-up our ongoing And and approximately experience Phase and FDA The primary of the XX FDA sleep. Individual years score. track X receptive by study endpoint countries, been which responder activities III allowed We of The gross with on now gain the to age startup study and aligned a on cognitive initiating demand have study to of global of study with in across [indiscernible] the have to were year. patients cognition, with alignment III discussed GTX-XXX. With the raw participate time. multi-domain UBEXA these key are enrollment end XX The significant with include this includes focused progress FDA

While with other Phase patients patients XXXX. of study genotype we for patients age this who in planning with will majority in also with on groups focus of full other GTX-XXX initial initiate treatment study to are III Angelman an deletions, evaluate open-label syndrome, to and represent the the

back application. I'll to now include both data closing provide of in some Emil remarks. to plan turn expect to studies enroll BLA call a parallel in both sets the and future We to

Emil Kakkis

summarizing Thank catalysts the we I'll the pipeline, year, on of first clinical our and made the close performed key part X significant year. progress clinical for Eric. commercialization our In products. advancing globally the by of rest well you, of quickly the we

For how study on GTX-XXX share are year, working for to II they the Phase end Angelman the to of and initiate maintenance over patients syndrome, Phase doses. by the doing time, as we're plan the III receiving continue updates

excellent date, superior of this to the data Our position program. ASOs data puts future us other long-term any in for is and to that for far presented Angelman on

study, Orbit XXXX. early planned there first Phase year-end the or X was interim III analyses anticipated by portion are of UX-XXX For the the

a analysis than stringent a equal or at months to met, a will first by interim The not months. later, value XX will threshold of analysis X.XXX. [indiscernible] occur have If a p followed the less is second threshold few

collect study and would would interim the for these Committee event The data study the a run it's to a met results there's and analysis by unless share very then data threshold, for company announced of very immediately the as prepare top final line be integrity patients to the formal an this to the first threshold, over of reported not Data stringent have and analysis Monitoring we their require time would outcome, visits will rigorously. In carefully analysis not interim but to that the clears the and be months couple complete analysis. important

to For as more, readout, second we we'll disease, Wilson dose [indiscernible] time the with in months last or of In all analyze once the stage share UXXXX for and Stage data additional therapy year. data. finding expect half of collect X prompted the to followed be I X Cohort the data by some for of the patient

Xa, expected for UX-XXX of All end is X executing at catalysts the on is working is programs, and the around [indiscernible] which XXXX. Ultragenyx an will clinical of disease the inflection for these and point. type other for on exciting are Sanfilippo BLA submissions, incredible we in be year; very DTXXXX while teams are these X which also expected the syndrome, for

months, next year. between on XX be GTX-XXX have should study, generating way and X provide to million XX revenue now all we the million while, $XXX is the $XXX III filed Phase with and expect growth data our this meaningful we to expected be to are Over Phase the [indiscernible] BLAs well III

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

Operator

first The Gena Instructions] comes we Wang [Operator Barclays. question have that from of

Huidong Wang

data update. syndrome Angelman

such Ionis loading also competitive quite especially at your communication saw landscape from regarding expressive and design, endpoints your and other update the we're both to trial the the a read-through Maybe is for if as also frequency, what We and latest the Bailey? data looking dose few Roche. thoughts

Joshua Higa

muted? help that difficulty question? your you. All to of having like maybe technical hear a can't Emil's looks little out lost we're Was line It line. Eric, We we've bit you right. and with able are

Eric Crombez

been Yes.And released of data we've important time around following the certainly, along ASF. updates the that were

very successful data end from more feel II end Phase -- we importantly of good from at Roche patient Phase to we've Ionis. meeting II very locked had the first both certainly really we're the looking We our mentioned, we for in stand, year. as as forward where of FDA, we and So about with and closely a have plans by looking

which been to under domains. have III doses, a dose X a to important dose individually the to really X readout across done and of in secondary for thought loading some we're also from data and ability get talking to us the year all doses our in of collectively movement shorter loading reducing about We and time. Again, increase gives and seeing Phase endpoints, higher on drive the as MDRI looking that X all across domains we've great those period of

We are looking but secondary endpoint, expressive cognition communication important our an certainly primary with endpoint for raw moving as scores forward at

Emil Kakkis

back. I'm Hello

Joshua Higa

the took of there Eric care any had Emil? follow-up are for call. there that you Gena,

Huidong Wang

Yes. Sure.

wish of data maybe in patients this year, biomarker So on maybe [indiscernible]? regarding Wilson you more second the you what how and kind half many you do data with

Emil Kakkis

Well, each there patients, in XX is a of X cohort. total

data have of will lot copper primarily will so not enough aspects be levels, you a each patients [indiscernible]. levels that biochemical. right, There's and other clinical patients in of be dose XX that will -- information, it focused could worth be there on it at distribution It of So level.

I think We certainly an of that encouraging. early us the working it give is gene very will indication was understanding an well. and therapy had

point. primarily the at we're part of story the focus this So on biochemical

Operator

of Richter Goldman Salveen Sachs. Question comes we have

Salveen Richter

Can you hear me now?

Joshua Higa

ahead. clear, Loud Salveen. and Go

Salveen Richter

patients that your the Phase understand population, kind with III around assumptions for fractures Phase So impacts regard how in that sorry how and here regard of to Phase the here? And about of look III help particularly the improvements, at AI see rate trial? data how OI translating you as the Could you -- II the you to us that. look with on

Emil Kakkis

got shown I Well, fact, at data was, continues increase p-value dramatically. the much And what the think in smaller. to XX-month marrow density we've that bone the

So are of all the that remember, you not all the but at tells it patients a looking in the the median, that's p-value reduction substantially just toward fractures. tells declining -- moving patients,

feel very the is effect large. So that we

see In we placebos improvement know to this density do marrow few from of time. bone had not that data period III, we a they terms translating of the during Phase in

to effect on both doing. So that. With factors be regard like is there environmental are and no fractures also fractures, will placebo from severity what the dependent disease patient

when not is better, more have we that patients seen feel have treatment for is gotten patients, Our falls on start work, expectation time and that stronger doing what period and have they could longer a but have have will fractures. of been

data bones more confident to we for feel do will strength activity way patients patients most think patients pretty value the I means which will even product the were because think compensate support as that of doing, any clinical such But their thing on Did supportive this. Salveen? the for are I bode might that feel you change are clinical the in, for of interested well active. we its occur and the hit how the So and

Salveen Richter

helpful. That's really

Operator

we Dan next comes The Gon question have Ho from Stifel. from

Dae Gon Ha

I'm Hope off. coming

Can you me? hear guys Okay.

Emil Kakkis

clear. and Loud

Dae Gon Ha

Awesome.

Emil, with perhaps rewording just have maybe management then reservoir its thought differentiation earlier And is if the wondering mean, you I impact coming maybe tangential that thinking product affiliated bit reservoir greater different you drug coming a feel get its ASOs. can trial intrathecal effects? I about side and just through a [indiscernible] as fairly just strategy life using more and cycle certainly have an here, GTX-XXX, [indiscernible] details where the other bit III of domains you ever be potency? more out is like guys strategy but quickly? administration about GTX-XXX's design maybe little was and I safely for wouldn't there a maximizing the revisiting in So question, you Phase we or the now about by terms guess of Gena's ASF, set, ASO around certain that some

Emil Kakkis

Dae Gon. Thanks,

condition is double-digit at the think thing the first, you of So the for data that long majority long-term the we showing is the look part about range I to our on enough. X and are data actually looking Bailey showing if patients into

we of So years anyone. that haven't were I any data. of X from seen plus showing

showing So program that's from kind right we're only data. that the now, of

Our drug have XX-milligram and effective. science tells is is we that to been right. That than you about we've The talking the range, more We're potent far what the in that. potent drugs. more operating region below is the of targeted added other more X-

waiting differentiate your what I'm will will on point, people to point. to have. With regard we actually other from efficacy. think And believe we that a So I superior data match see it's good

[indiscernible] think in our things I we make we them make one philosophies and of is work, that first easy. then

Our which invasive a and approach. you that inside, downsides. goal It as have soon has or is at you would thing. an It procedure idea. similar certainly its to [indiscernible] one lumber is this for through get look patients port, Angelman we'll puncture approved drug are easier There make possible. catheters which lumbar potent. devices, this as Omya is a much drug for But also But a a catheter-type which can syndrome access, do simpler a how is then upsides -- make more than

drive we're be So possible certainly, and those are we, we approved, will company, to the never and still. If best cycle improve ways continue a do things we life into management. get forward things looking with experience, consider, still improve we going a sit the efficacy to patient to are constantly can outcome. the as as Those

seen we're that anything like me in feel way. ASF, I tells great we any position. after a different haven't So And

Operator

next Tazeen of Ahmad we America. of The have question comes Bank from

Tazeen Ahmad

on, would half I half of first about And couple was to your a readout then a interim you year. get the maybe moved initially, a to thought sense second the it's that and of talking now times X wanted you're have that Wilson, Stage readout. On doing in

once for curious Just delay. we that those do of program? then should reason in what see data, the the we that steps development expect And the next

Emil Kakkis

Yes.

So Tazeen, thank you for the question.

up we X the first qualifies up to and longer, finish ended last that having that out X the dragging patient the think patient. of end we and I thing is took something had out happened, cohort

drug. in a saw more out, took time. months of that of going It's -- One is that effects problems. it thing Cohort some part took not behave did we like put was for so X And the actually It the transporter more to the you of enzymes. day of X one our It's the than we to wasn't see copper.

a bit learning we're So little it. about

to need last in, the is. that's least months what our and data from So X timing go take was at we patient the of

international I you to to [indiscernible] think data, Phase have an that out. it time clean for to study the and put add on it II/III is

forth we horizon. So treatment a we're heading and encouraged to this data is. by put right important But the into decisions for gene it come the get with that's we'll our is where think it's saw. on and continue Phase for plan make III. the so up we early therapy Wilson and I there And felt that to And data

Joshua Higa

question, next Operator, please.

Operator

we The have next comes Kristen CounseliGerald. from question of Kluska

Kristen Kluska

a show on wanted this setrusumab, both and on relates potentially think there I quarter. those On could patients has to pain? the great a fractures think an bone that Congrats Is to these experience. better down to the drug that potential you benefits any have pain are reason to impact putting as there if reducing ask it

Emil Kakkis

Yes.

we fractures means having impression all look bones. the patients the have Our lots increased with pain. about activity, micro -- while better. They're weak And particularly of feeling Phase And that time, what talk patients, their fractures. less is they're from II OI

activity, micro So fractures. if induced heavily have of terrible probably feel bunch next they do the they'll a some they strong because day

not as wheelchairs, a beyond, patients or not needing see X-year not having it's patients fracture. from point at What being are we afraid physical more the much can treated activity. So of single the point activity,

pain. that So fractures we'll we have reduce will confidence and micro improve bones stronger

quality power both those should it's the that enough study measures study. in large and endpoints. evaluating us other are a And we pain, of so life And help

one setrusumab OI. really think think, for we'll ways important make, it's we therapy that I of So the a

Kristen Kluska

doing that that on down density bone end feeling potential you're And spectrum, for more type open ask, exercising if maybe then other and fractures? just on the But sometimes the further your any point, people loss? loss of that any better could doing or if are help does door and even that slow more activities, people activity, I know of

Emil Kakkis

Yes, it's point. good a very

I I'm great think have who and if feel one not patient, did you now again certainly don't We have doing the a now, increase it fracture, a to started a risk. did could fracture patient sports that, with just anything weaker. right? you're do do is I It's that. in to our bones don't and I rational our not if What you bed sedentary, get patients, these or we enough, sit say think but will tell

exercise their enhance they bone actually bone do lay the is bone down will So will It their stimulate where bones strength the to weakest. their actually further.

So effect with I we'll and have sports be think a active to them more or beneficial else. -- anything for

the about these worried not fractures. it's bone part of stronger We of OI patients. risk more we're towards getting better and activity, a think pattern healthy for more lives So moral

Operator

next Anupam The comes Rama we have from question of JPMorgan.

Priyanka Grover

Priyanka This for is on Anupam.

this on Just gating available any into For or the for a early filing will year? BLA from filing there UX-XXX, be later are based even data, quick us factors the though year BLA question next

Emil Kakkis

with They work. This know an good thing contract they're they derived Well, make [indiscernible]. children's together their need one the are do our people. put sure manufacturer, nationwide to the stuff, CMC from that the doing is They is excellent we job.

about right exactly has what in That now, expected much that need what BLA. line. factors. what to we of in would can be any feel year. but to -- be needs be we're in flexibility agency get with a where needs a agency see so And BLA shown to we putting get be has be there I on to thing gating go. And on I we're to and don't It We're one having our pretty a work track [indiscernible] this together isn't lot to later. now, proper the think, And

Joshua Higa

Operator, next question, please.

Operator

The Trace Lee we next of from Securities. have comes question Joon

Joon Lee

Knowing been on about this IONIS why ASO for go data no [indiscernible]? go, strong GTX-XXX what not program words, consider a Biogen preset the on Angelman would know the they GTX-XXX call opt other what ways as the program? have you not Piaget do from what's threshold. and be the point you publicly or in In during when competing disclosed. quarter may morning, may did to Ionis' selling Biogen's conference cross what Congrats option think for patients better be patients enrolling In for your study, asked

Emil Kakkis

multiple I sure data in growth substantially at higher over I set. and amount Biogen daily considering that condition seen is a in steady X levels domains achievement publicly think of am time Since longer think data and is I from competitor are our periods fact, Ionis Well, limited of we What months. show available. that's of strong through data the we've looking a all

was that it point, So I'm has they compare, And sure me we to unlikely Biogen their I think the and term point. data #X look And that product don't for seems equal how might at feel wouldn't they to that But this their a It ours. were their think stronger. and capability the our the think have to substantially we that opted like to understand and equal. product, is data made meet what they choice. in if it longer had at I not ASO potency we And product. have didn't fact our met ours doing is it's this now, and if about Angelman good it's a criteria right

Operator

question Maury from The have Jefferies. Raycroft of next we comes

Maurice Raycroft

X And Wilson's disease. I'll about ask

by further of scenarios change to advance Phase require your optimization? or to what Or What you're global be see to For what you need assessing considered biomarkers. III? metabolism a could magnitude do would copper update, upcoming win

Emil Kakkis

Maury. Thanks,

the through maintain therapy, first be right, copper they now copper. to copper necessary top and to effective think urinary weighted patients free levels I to right? get think gene an standard to a are and copper chelators have of indicates as off, able it's accretion Wilson, see with to replace we that care, I be So off able to that have detox proper pathway,

places. some copper copper contributor [indiscernible] in the of to in And the and Wilson phenotype. is in very are the significant these that improvement other patients a is which distribution brain patients a low in see are Secondly, levels, the deficiency to generally we'd think copper and majority we patients, to of source like copper many that [indiscernible]

So it's things control. their and in a patients distribution copper were looking the low, and those in over toxicity for, are X maintain the care those background we're baseline, remove substantial of standard to particularly improvement ability

Operator

question from Schwartz The next comes Joseph Partners. have of Leerink we

Will Soghikian

Will Congrats design us endpoints. GTX-XXX, on on the of on quarter. for progress for Joe. outside One study is this the This and

of Just be has clinically wondering color benefit our the meaningful on can And endpoint? the you provided us Bailey of a on X the would for the approval? if bar along remind lines, same FDA any success understanding what

Emil Kakkis

they off, to a variable right? there thought the agreed right way. established first analysis required. They set X, Baily the continues variable was or threshold Well, no was approach So for

analysis. part So they the a didn't as maintenance. We require which MDRI, responder will of the [indiscernible] responder support do

I Baily a X, primary. be that's X, clear significant But what patients to in can already They quite haven't daily we about that's of a in us. they've which the what X to is while think Baily So the improvement what majority dip we if Phase into seen In numbers. II, our important. considered set mind, want any that's essentially twice required range statistic I it, of as show achieving a we've double-digit is

MRI, story The of the the and for this that patients. we of is change the other a factors through combination the value is the of see as end it life it, part eyes of of points are [indiscernible]

think the score improvement language is these drug picture, why are is I some patients have this life falling calmer, sleeps whole you would to going for multiple at Knowing story. that not much, behavior So tell doesn't Bayley change well, down for all states as be understanding of important instructions tell the the at the home. And spoken big these of self are is X better, a patients. we domains language are things patient look of

Phase So can if that good showing we replicate in we're in III, in we're II, I shape. you think Phase what already

Operator

we Wara TD The comes next have question Cohen. Aaron of from

Yaron Werber

in there give is just see point? And how of about kind the going powering, in terms kind little have of -- here? speak of sense, in as talked let's here? in million are feasible age, a price we're I to how or a be see endpoint, I what's of planning X but don't of the follow-up. kind maybe you commercial to Emil, can III? what think longer and to of GSDIa, a so. about just get the secondary the think Phase maybe think terms sort a $X we beginning I hoping you've just want market delta of look to XX-week just say, a of mid- an a the being, of end And you, X opportunity us then bit can for and on kind questions to you big to

Emil Kakkis

Great.

showing we group So you there, assume well actually if actually in AAN in you we the placebo we -- our more the Xx history history, above or data, the slide than on had natural powering XX-week deck, we're times XX% the look at still percentile. put -- X if had a April [indiscernible] power. natural Even the XX well X

we fact, history natural control powered. typical be above would the XX% in with well group fact, In

effect very weeks. talk -- we're well see into is now, seeing range in when were about the double-digit you XX right it which powered So the to size

reasons the and that went is, a more better longer accumulate of we overall. we you One felt story made it improvements

them us high to what Phase inability extension. didn't But urgency this because a as their we're better were, sugars is a amount doses, patients the is, to regard their if thing to are and seen cornstarch there. We number opportunity, of nature in to and no disease. powering GSTXa longer seeing strong change And dying their know and of think than of dependent in happening as for their the recognize will much blinding reduction what because their the on patients on cornstarch that's physiology outlook a of data feeling less issue is brittle call the seeing doctors they having we the the with disease. think get III in very think Angelman. With better patients, We their an the as effect are of a get better and our to the of they these the that there's change we miss fear what's we've So survive. changes. and I highly of But a the commercial life exact is

for corn to is have nothing And [indiscernible] people would starch representation that every And regard with to like urgency think die. occurs day. of the some to just it's the head, I them the gone other waiting exists. that risk of a diseases that the

been success It's will we this urgency, pricing of we And dosed. to the be been not at too it to think what's a we and pricing, have regard commercially happening. With put get been So what's bit highly that's the think that's a desired key has certainly patient out population, to defining level in to point. early. patient gene expect patients we therapy want

range said past in possible. is million have that million $X We in the to pricing $X the

some and have haven't I points and it. opportunity. even price plan our I think gotten we for is. this going what is look come very to higher carefully We're But GSDIa reasonable down But important a at set programs. think at

new be I think I will therapy. levels well. an more And do to where of high programs like think it perform other urgency. of the will I important it some there's it expect

Operator

we question next Hung comes Stanley. of The Jeffrey from have Morgan

Michael Riad

is for on Jeff Riad Michael Hung. This

regardless more Going pediatric is you options if patients it it to about role a think better adults, most and you'd thinking loss factors setrusumab type? mean bone What for do course? or on the Whereas leading I activity. bigger then a do profile, type? of expect to like play you the cycle deformation treatment like and type-dependent OI the for think that view which now you age as -- fractures OI broadly penetration? of Is of -- back about

Emil Kakkis

be It patient have different. is each reason to a to may think going treated. I Well, be

severe bone or Type see when years data hope, really will If and a X, treated you're X or that X a age is of stopping vertical not compression wheelchair. ending in show X destroying terms up stopping your your be and skeleton fracture, in of years and your disease, old, you're we X and a or basically Phase III could we patient X our before type with the with transformed

be could that you what that So are you're treating do when young. kids would

pain they're able highly and can't the a by would fracturing, Being in still all when you're stopping being all or However, adult even Type you the in you're track change fracturing, to time. an bones, pain form stop if X. to have in still still if because valuable information, wheel even you X it's you're like

Type been activities might have milder, those be X, it's comfortable, not I in Type think for able an not Type get addressable half as any fractures young in sports as need participating don't much will at the some to patients. old, be and don't may doing where Xs as information, There age, might will have going be before, much Xs being probably For sphere to treated. of that you we'll or population are be -- as have addressable but beneficial. They have who or many fractures, enough there

Xs. expect So Type well the Type Type I we treatment we've Xs data on Xs all you can shown as though, from the would Xs. and the What do really the say do

reason all in diseases. think a So to that of X all a in have these as treat types there's because good life we'd we we any of point well at any ages penetration expect as

Michael Riad

circle I then something. back to to And color. for appreciate want that just Angelman, added I

that up longer-term Phase saw giving Phase So like to months, XX weeks, more good a insight the a unique but to X out the responses showed is and obviously, benefits. data of bit I/II III that competitor makes we those into

that can have been aspect like developmental gains like be achieved? just that you clinical how think was used and I data like durable that wondering it for more So the evaluating

Emil Kakkis

Are data? didn't data else's quite... or you or asking I someone about

Michael Riad

I'm larger-term about I/II Phase like data the how III, elite asking Phase like data can... that

Emil Kakkis

Our data?

Michael Riad

yes.

Emil Kakkis

important tells data these to XX longer is that weeks still better what's patients point only where continue journey is ground. is gain Well, I see they don't to which the they kids a can to go you long-term are how think to could X gain get. on continue where going, about unclear They much like ground. -- they

have time point to I learn does would had time a functioning make you and as better, things, more while time like can't is is that learn other kids growing this component, to something in The to up, understand. brain So a -- take developmental repressive right? start took particularly we them, get communication, essentially didn't think it take that right? that they kids learn to might Just and know receptive they you for communication

III. is enough there's and approved, but long-term continued to XX drug take the complex we demonstrate the will functions shift drug why time, patients if in why good weeks product point more shift improvement [indiscernible] capture be a might improvement time control but population on will the feel -- So that the penetrate that Phase to to group, the the successful gets stay from

Operator

Baird. comes The next Jack have Allen of from question we

Jack Allen

quarter. few I on the the to ask a on I little Congratulations progress question. parts wanted over of setrusumab, guess, this

it second was are BMD to presentation wondering color Do over we setrusumab? Z we of provide relates should continuing as for the improve just you're like The seeing the to half the correlates and ask scores about expect first Phase and of study additional I enrollment of you of which, and I as to we're and year comment the of look the expect the time. to up as data what then in It that towards and portion biomarkers fracture looks additional any types also patients to time versus looking with over there? And how with the that at the the wanted setrusumab, ORBIT trial in that related II rates study? follow in it differences

Emil Kakkis

Sure.

set more So goal right the able as file necessary in to set prepared honestly, Phase III if second the is the continued haven't now, our hit patient the it's we regard to well prevent I'm that of have XX right BLA now plan now, the to to -- have transformative. interim. end to crank we're and be the very patients will not But been encouraging extremely clearly we we and not. with who not a it's particular months half, is out if I -- of certainly months And a do might XX on to sure have data or to a mean, continue and the over They continued been but put data, improvement are story. XX time.

in people point time, turned at true. some that's put eyes out may We in think I XXXX. toward it but already analyses think are definitely interim we So most

ratio. number regard have to X We But in So enroll did those. Type now it's And we and Xs. of II more patients, to what other and study, severe study, have of get more the medical is the and the Xs. which we're had we lot we Xs wanted Xs fractures, haven't first We Xs Xs Type patients Orbit more Type up significantly yet. With that committed put and the we to Phase that enroll more to need. data type Orbit Type a And in more able Xs larger wanted. XX

started -- them saw Phase I stimulated before were get the because the II think in apprehensive, data, once putting then patients doctors most they they involved in data to actually their most need.

but So don't shift compared think different see be II there it Phase will is I to there, we in little bit of what data. a our substantially

Jack Allen

in thoughts But the were just of of impact Maybe severe you it. power? may questions Got know when the get more how what enroll guess, a brief portion about that than your enrollment lot powering III how X context, you potentially the expected? follow-up. I I of expectations on Orbit to enroll think set Orbit to for and you on Any study. that did patients the out Phase

Emil Kakkis

subsets, at at you adults, XX% overall to is benefit in to X look if frankly. not Well, pretty Xs. because to of the months, have XXX, down each Type data peds Xs keep we look -- right? Xs, but look enough I at right? understand you at age Xs, the XXX, also So we fracture We what's look started having powering, that originally reduction And XXX-patient our we of had a at power, So there's clear also with or it difference Type old able could number way and the which is to the past and or you the look saw it right? study. enough to and look And get large data then young huge that you it want Xs needed. to as in their of be at to brought the when Type But X group wanted first was that subset, or

of maintaining X the So the that in number was of drivers XXX.

assuming I fraction try you types was types the have and data the could the when we with made and think was design and there the reduction power, study, age in smaller. But across I think a of of fracture plenty all severities a capture I the the good study the groups, rate think, will of rate Hawaii. that we to what cover put, amount higher

Operator

Walter of RBC. question next have The from we Lisa comes

Unknown Analyst

get is if This More It Luca. Lisa is on for on disease of picture we question. thoughts your Congress. voucher rare expire to it Wondering September the This can pediatric big set on like XX, program. by me. question for this the a is sounds is future unless of reauthorized

drug forward? appreciated. reauthorized affect rare there going Any might be this would if much it's not disease So color how development

Emil Kakkis

Yes.

able to setting to year being the -- them through happening next being this and for year. able get new So later ones apply of sun is

to. as us we PRVs UXXXX be available if or DTXXXX, X and us should to there a should be for we company, to get file able For

vouchers ability rare anything. those drugs. think us our to PRV it supportive helm we some the $XXX happens had really has doesn't support We're of million, We for for $XXX in on submitted We've short disease highly run, bipartisan So planning. on million that on major a something PRV. almost vouchers, of sold for the the us, ultra-rare additional cash information. other that long X like is of rarely the for the Ultragenyx equation us, impact our develop In financial changed in the we've in And term -- sold own the affect diseases. what

will rare yet regard doesn't the year a now, it feel election done, it's are you to hasn't party that a right and to So with rare we get something do things, crazy you but I is if disease. happened but that think what time matter have

the hope to will support. we PRB get So

certainly providing it. are We

Joshua Higa

operator, can next the question? to go

Operator

Lisa question Evercore ISI. we comes Bayko next of The have from

Unknown Analyst

for on Ming Jim is This Lisa.

and an and open-label we they study Phase that the is in that indicated Phase approval opportunity to if may is launch romosozumab So running an they Amgen pursue III have OI, OI. positive, noticed for have III in study

think setrusumab if just you approval to in OI? what it wondering have would implications decides So I'm do for pursue Amgen

Emil Kakkis

the to Well, that's given already us. us intellectual access. property They've news

So the substantially the clinicaltrials.gov, I to [indiscernible] them, of less They comments biologic they're osteoporosis their It's regard don't their in understand in think for interest really seen -- now, levels density dosing they've European anabolic they're With indication. marrow toward bone There's as version their a a dose I it. Right we've had a growing. that's at the data. or huge shift getting much agents published OI, in using. II We that from focus. is big it. think the Phase

improvement, in a reduction. we're our treatment So then fracture and modes of be we will superior superior in terms bone

done So in Phase I or presentation think is as an you III for OI. the What optimized drug they've should the not this their unclear look at story.

And so this them really now at dosing point. dramatically have for we data is which right Phase III, don't would know to from to likely to because concerns I our superior far our your happen get is not data, they have to change

will will this be at think point, inferred a factor. think I So that there be and I us,

but drug more differentiation Now they then to or to Xx if higher goes match use Xx potentially, of to with you our give away a [indiscernible], dosing pricing regard the have level. dose because

us heard this and has all. for of their believe by part So as and disease at in osteoporosis listed we're I we before. that position, anything main feel the space from haven't like in I their good franchise a Amgen rare been

changing they're be I'd So that. if surprised

Operator

the closing for like we call Joshua I gentlemen, reached and sir. session. to question-and-answer of ahead, remarks. Higa turn have And Please back would the to end Ladies our go

Joshua Higa

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

Operator

that conference. us. Ladies and gentlemen, Thank you. Thank joining concludes then you for today's

now lines. your You may disconnect

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