Ultragenyx Pharmaceutical (RARE) 29 Jul 222022 Q2 Earnings call transcript

Company Profile

Participants

Joshua Higa	Executive Director & Head, Investor Relations
Emil Kakkis	Chief Executive Officer & President
Erik Harris	Chief Commercial Officer
Mardi Dier	Chief Financial Officer
Camille Bedrosian	Chief Medical Officer
Gena Wang	Barclays
Joel Beatty	Baird
Dae Gon Ha	Stifel
Tazeen Ahmad	Bank of America
Cory Kasimov	JPMorgan
Yaron Werber	Cowen
Joon Lee	Truist
Liisa Bayko	Evercore ISI
Ashiq Mubarack	Citi

Call transcript

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Operator

Good afternoon and welcome to the Ultragenyx Second Quarter Financial Results Conference Call. At this time, all participants are in a listen-only mode. At the end of the prepared remarks, you will have the opportunity to ask the question during the Q&A portion of the call. It is now my pleasure to turn today's call over to Joshua Higa, Executive Director and Head of Investor Relations.

Joshua Higa

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

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

Emil Kakkis

the good Starting Angelman in we year. meaningful UXXXX Thanks provide touch all continue another GTX-XXX of the program. These company, as Josh members turning more acquired on in reached commercialize along the over early and against afternoon, position progress solid a We're growth they of our of to the with control the we detail bolstered over MPS a for, pipeline with a extensive across I and acquisition late-stage good us commercial products this control July, the on coming In across their to from to quarter Genetics MPS our want And and value also program. the program updates programs years progress IIIA we experience. a that team creation. over everyone. moment team which to Angelman us make now goals. of call cash and full strong we important acquire late-stage full position with area into quarter. our financing for syndrome, the the clinical and similar leadership across The put the Syndrome MPS gain substantial our revenue with delivered activities for We of months disease product before GTX-XXX now enabled globe. couple six to Sanfilippo have or or royalty genetics exceptional other III

cognitive investigator, Bayley, by is remind delay a that of level the of used and in in As age allows GTX-XXX is the clinic changes maternal seen clinical These with the in our responses mutation in or often of recently. the natural of measure that this principal Development for exceptional patient Dr. or latest and and history doses that Bayley clinical language providing Last when I'd measures this and ability know Bayley week, highlight independently We type measure further that the excellent and mutations. me to seen more these with therapists this to Bayley. do the version and in Angelman Receptive we score syndrome showed last different across to patient The significant are call region group with I by meaningfully of Angelman effects extensive been said a something exceed with development patients due region. is history sub-score predicts of This we week. our on domains with with is as administered severely One call important regarding threshold expressed confirmed therapists is childhood In results We Angelman doses. see an these to clinician’s, data for the I/II families. improvement they're improvement for Across the loading Bayley data Cohort impacted up particularly in observed time children increasing of minimum signs to some at not we're of study. trials meaningful and We clinical a enhance multiple individual to the children. the by have syndrome Bayley would to the statistical observed for allows by syndrome improvements like development. to of Scales statistic daily measure of specific the those now the very slow is of used rate standardized of with on Infant XXX This date like diagnose X safely on always neurotypical using the evaluation not been as said children. investors treated function data with development Bayley variation. motor seven XX-milligram children children skills. deletion lab significant study Berry-Kravis for target limit we've of in study. with on XX improvement haven't historical severe we which we early the through all study and evaluating disease Phase a in ability our the years demonstrate a drug in It's a of rare gene delays who receptive… showing set an for from from UBEXA multiple change Bayley have communication shared particularly either learning in measure clinical patients to on last deletion one and expect I nine part patients significant communications to seen patients are children days, X of with dose a week in trained and error in

Joshua Higa

might audio you have Operator it confirm Can we like if connection. there? out lost we're still seems Emil's

Operator

connected. One moment. I do show still saw him -- him

Emil Kakkis

I'm here Josh.

Joshua Higa

Go ahead, Continue. Emil.

Emil Kakkis

who of patients the important X or favorably original syndrome communication US XXX. have and lack recent been Angelman the change improvement an most three two compares is were the minimum of who This communication seven communication significant who particularly days, the treated expressive have at statistically in as children five store a score -- Bayley with Bayley treated of in for XXX day X Across receptive nine in a in either skills. evaluation. expressed statistic Cohort to part patients for significant their of of and the Receptive showed patients same

us depend Bayley of we patients are in will findings Bayley syndrome higher studies from full well for included time were manner. objective The also point, of The time at doses. months the results potential of improvements reported dose past natural the release. Our measure this history combined original a the improving confidence of four patients were XXX last longer give skills measures that following starting time that Angelman likely patient results patients on communication data the that and past many since with in third-party period a their day results communication five the with from meaningful

two US patients out dosed no dosing and to important Canada the at originally week day and results have ex-US patient all evaluation the first treat UK safety and already dosing day before the in other begun patients fact, doses both their the to Canada first cohort Expressive phase the We've selection in from with X. One mg the X.X dose patients we've higher also the last beyond in patients that was XXX reported drug-related step improved current additional of events. used dose. X In starting study reach loading read cohorts communication and with in in treated XXX three in their forward, of maintenance for receiving amended and the the the Cohort Receptive protocol X

Ultragenyx working with study is lead FDA for US now harmonization development file team to is that support coming would clinical to protocols. for the of and this to a all right these discussions point also an the the components ex-US this report program. Our allow of interim With together,

agreement the we take the for we with for pursuit gene program right Scott syndrome. regulatory past, to closely the partner. of signed who we trial firmly approval. We we the alternative relentless for Sanfilippo meaningful pathway. right have story for the and In have science the Dindot's of The a diseases and of that lab expertise their and make the use one also community. ensure on parents of history believe strategy Angelman now treatments strong search worked an in In treatment GTX-XXX second endpoint defies and we in odds story the our therapy or an and are data announced a and And the this of size opportunity for the our MPS a the and believe for the group are territory FDA It's difference the gene of is we bring the was GTX-XXX. clinical trial right approval quarter, Sanfilippo of community. a use the UXXXX capability can the to UXXXX believe to achieve to establish I first-ever therapies the right support to discovered of IIIA of robust team. us MPS familiar AAV development to inspiring and

providing for on year. on hard Our our the later agency we discussion with forward turn strategy updates their team work the is I'll provide to in team With and updates functions. to it that, over at to the filing the look

Erik Harris

our I'll and commercializing Crysvita section continued my discussing Thank to team's you, Emil, shifting afternoon, good Dojolvi. success everyone. before start

within territory, product. than find to launching share profit adult new Crysvita the the more since years, For continue four and we patients pediatric

X,XXX consolidated is to centers of end quarter, similar the and US, XX% scattered are half being many finding market. excellence. we of second the of by than prescribed into penetrated to other finding market physicians almost community-based This have rare Recall, the In diseases treated the of the pediatric over and As adults. who is XX% patients country. adult more of have the been treatment pediatric contrasts where the were mostly the approximately Crysvita patients adult patients across

presence. digital Our providers enhancing as and meetings with team programs interactive healthcare and is education our traditional to along mix as virtual online well of patients leveraging a innovative in-person educate

also even existing that patients plus grow for initiatives with opportunity plan meaningful launch. often strong family We treatment with physicians identified work and We a believe four assistance adherence and there specifically education to the into as to within to franchise entire well Crysvita steadily the years is recently continued target caregivers new patients patients. develop as launched comprehensive nurses

already Across in the XXX first XXXX. responsibilities second year, efforts. to as surpassed launch reimbursed have territory, Latin In revenue quarters. the grow, the $XXX XX% geneticists momentum named to results support our to In of Ultragenyx gaining launches Outside our patient in on the Brazil XXXX in Ultragenyx the continue and America, year. versus grew the gain primarily and to continue America, first our to as in therapy. the quarter, we will early their half outside towards transitioning profit all from over Colombia of and XXXX to performance we expanding last first Latin requests regulatory as the seeing the we the medical revenue reaffirming of regions, This Kirin We guidance we XXXX. XXXX. Kyowa April in of are our million forward there continue quarter Argentina, XX% recent Mexico. in efforts the Crysvita are approval territories we we this our total revenue over share of now grew compared half generated first will of revenue are And patients look date, region continue million. coming in commercial In in we on $XXX to of Based look half the of

Dojolvi now the in our to and efforts US. beginning Turning with

We indicators as broad across clinics the of metabolic across of leading in continue use growth to result key steady the US. see the genetic all a

through to XX beyond care are for reimbursement team Europe. for early we health programs France that Dojolvi a to requests high for continues of commercialization. adult approved is in more year. US, other XX% also across and In Dojolvi countries major machine of generate faster In cases demand Crysvita This to other for starting for patient late are in in stage is metabolism to statistics add learning errors through professionals. pediatric health artificial LC-FAOD the is treatment the similar than the at centers days. and and access expanding One the and meaningful program approximately respond to intelligence with an coverage there we They the our time to is physicians new promising leveraging of approved leads saw are the patient patients of of potential what doing Italy, be named than even and similar call are inborn moving last Outside less new turnaround The all patient patients of Brazil, our authorities for Europe. use named we Crysvita. of programs named continues and both to and

that to can we this the approval, We million get are of to and of through look the the to continuing other on to complete. results process the million details little are $XX quarter. year, call the providing on time out next commercial forward we work to update bit over but to At in this to take in another $XX turn full more guidance reaffirming programs the quarter. share a that, financial I put I'll January. reimbursement With this Mardi point for

Mardi Dier

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

sales Our $X.X quarter XXXX $XXX.X of cost total million the and development $XXX.X expenses for of million. and of $XX.X operating were SG&A million, includes expenses second million, expenses of which research

For loss per $X.XX XXXX, XX or the was million second share. $XXX.X ended June net quarter

to of number During our approximately offset value includes net approximately stock-based these year, transaction, Markets of quarter Crysvita of in dilutive raised decline the impacted with $XX sale half million equivalents $XXX the non-cash This revenue quarter, cash, fair dilutive non-equity the the approximately of $XX cash of $XX EU the compensation, million transactions in expense $XXX are Subsequent with there capital by America million items million $XX July capital to of in related securities. million to Capital We ended have and Pharma of a investments North to Royalty the a that royalty. interest of our first non-cash marketable the been have in equity related million loss. the in of royalty. related also non-equity OMERS and portion we non-cash the for end

acquire paid GTX-XXX. the option to exercised which over to us $XX allows development also genetics our in of We and July, million take

$X are development decisions our capitalized to slowing in our operating and the spend are billion We well in with to and manage burn. bank making stage headcount programs we order over growth on

XXXX us facility. gene or US are we don't late-stage this to acquisition of In responsibilities therapy one-time As programs as we XXXX, have anticipate additional Crysvita have said, will initiated year KKC. we commercialization our completed multiple of of SG&A XXXX and compared in-licensed events we burn anticipate is nature and transition we to manufacturing build-out large Evkeeza, capital Canadian decrease a for the the clinical as and completing peak for expenditures Genetics

net overall invest our across continue be as in to and effect then company the net clinical will preclinical the decrease and will a in We programs burn. discussed, cash

to Now, I'll turn call Camille. over the

Camille Bedrosian

everyone wish too I development afternoon. clinical and an for Mardi, Ultragenyx. you, time at good exciting is This Thank truly

including X, clinic discussed call, late-stage antibody that for mRNA Emil Phase four ASO Type glycogen and will and antibody In earlier this imperfecta. therapy storage section the programs osteogenesis for a on seven trials monoclonal or on X/X I in have focus disease UXXXX our setrusumab. for syndrome gene We my today's Angelman

stiffness are developing imperfecta. imperfecta bone fragility, We to and OI. bone that significant for is there are fractures, results weakness deformities, therapies osteogenesis Currently a OI defect leading for in approved by caused in no and collagen or Osteogenesis pain.

for XLH, spent the As preclinical setrusumab. time a and did bone of company, a before biology we we for candidate OI, have studying lot TIO our with deal

bone across by issue abnormal is for bone formation this imperfecta. is One new What collagen. to back Xb you the want to do adult the study This an that months, very time We the substantial believe, resorption, anatomical details to go bone a weak collagen strength we bone blinded XX the osteogenesis leads different won't few formation, improving has patients statistically patients After the found excessive mineral future have production indicated take we this the excessive the data mineral occurred of density. points. bone improve Mereo improve with mass. is a bone density presented. you and levels. with bone's the that abnormal large change OI all a trial was reverse results to And of the fracture gives being improvements of already bone and dose-dependent bone today of observed important low triggered of prevention. is opportunity with strength. collagen not and of is that Phase resorption ASTEROID on and remind you insights bone the the and most Furthermore, increase significant effect inadequate sites. factor I formation even that to multiple in randomized insight XX It of OI studied three believe our can across important bone key can dose simply if substantial I us

developed of favorable All Similar accompanied safety to profile. when how development were burosumab we XLH, over by Kirin. a Kyowa took we from for findings these

dose turn on strategy, to PXNP the back Once We now taking marker in we patients using of directly to and enrolling Thank the I five you. benefit goal and we into of pediatric OI with XX to update, looking call are improve Emil. between upon have adults results III, the of them further the the with with the serum this With optimize patients. years, these and formation are bone ages dose. will the determined fractures. setrusumab pediatric setrusumab evaluating Currently, dosing we impressive OI Phase transition for will

Erik Harris

out. a happy Emil, finish hard of bit we're a little I'm this to having time.

Emil Kakkis

you. phone. my with But problem had just now. I I'm fine. Thank I'm No, fine

thanks, So Camille.

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

continuing we enrolling our evaluate approval portion and the study an continue III study of continuing filing with expect biomarker therapy DTXXXX In for the the Phase enrolling III pivotal to under imperfecta, the for for of half Separately, five IPO, we the continue in convincing the around study. data. II gene for in strategy who old osteogenesis in stage been are to the feasibility dose expect pipeline on of a in the have and children to We'll year. Phase Phase sample we'll to the the initiate in first of the patients years patients portion UXXXX of based on UXXXX update dosed and UXXXX second For year. end provide follow the study

expect also activities begin for and dosing this start-up We to DTXXXX finalize year. patients later

action we will is build-out On material our on of track in the map the of in side, producing to continue Massachusetts, Bedford, half begin the first facility which XXXX.

and half the UXXXX DTX we I/II the dosing the data repeat second Glycogen year, study Storage Phase the part to of stage. first share For from the to X of single-dose in expect initiate and

to off in to GTX-XXX X syndrome, or we're For continuing milligrams the outside Cohort XX enroll and Angelman at US. X X.X

to expectation expansion gathered data one Our is substantial and determined update, an the provide from cohort. have optimal seems the next dose

a please we that reasons instructions. rare patients All to of With Session Operator, believe we are difference move robust, pipelines in most provide diverse, to diseases. the let's the create Q&A questions. of meaningful opportunity Question-and-Answer for late-stage these your that programs distinct make on have the one

Operator

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

Your open. line is

Gena Wang

I quick questions. have two

First one Crysvita. is on

share you active Angelman how wondering question XX% you you the make and that's penetrated are second the And adult leverage regarding how Just case that wanted I tree. the If correctly to it is family program. market. the would sure adult I'm heard market to expand

to two – would know still a you will dose to benefit to be update, open clinical think I XX And domains? that score I that wanted two this are in early the higher be week cohort willing would than expansion milligrams? definition the since

Emil Kakkis

Okay.

the can answer penetration first. Let speed this you one And me touch then second adult on Erik, Crysvita question.

be the close a going expect expansion, to need we and we very using on are we're to where we set still titration, to two-plus So that for be. to the domain criteria

don't at results In efficacy fact, be often beyond XX about results expect go we the all what but that's We expect. we're a to their at to have we're us where going frankly. little tell looking to including more longer-term

I think close. very we're

it I now it's the think cohort right dose right. we don't on level. this speculate we we'll do loading make But So necessary that. next once to want get wouldn't I to sure start going be

a what longer also the So in we'll getting seeing benefit sure of quantitative clinical we're also study will dose to to CGI both time III. we over that Phase but in scores a we're level to meaningful us substantial, continue evaluate the and periods could that in make score provide

finding I prescribed. get for part we've challenges talking but Crysvita, of about for find they been the them penetration, the Now adult the of them do good think we

on it's So XX% what Erik, and expectations to Ps, much maybe and are? about with challenges touch regard higher adult in the issues can asking the she's are market the you what where our penetration versus

Erik Harris

is XX% Thanks, market Emil. heard versus the you Yes, penetration the adult for of pediatric market. XX% in overall prevalence. the correctly, represents adult two-thirds about market And the

I family pedigree tree Emil to significant for to pull thing in is the XLH them system there do a offer necessarily work. because, date, So as loss through But we convert these treated done many rate XLH. stated we're members able think signs and are longer to genetic four but work two XLH, adults. each opportunity does takes for we've And not they is stated, community of physician related find as XLH and high at about counseling program. the excluding a therapy. just we to being growth very stay the symptoms a patients, which one with do them, We when on It family of to there

patients take So for and each identified finding analysis. that of family there to two advantage on tree patient, four counseling been that's genetic when we're around average

something we do is leverage. that that So

Gena Wang

Thank you.

Operator

Beatty question from Joel next Our comes of Baird.

open. is line Your

Joel Beatty

taking Thanks for All questions. right. the

what from on cash plans hand new this your for cash? the the are deal, recent With

Emil Kakkis

you think want I do answer? Mardi maybe -- Well, to

Mardi Dier

Sure. Yes. Thanks, Joel.

to in pathway really our million. used the going a this hand now the we more this and feel with acquire So profitability. plans to that little for bit billion we bit $XX fund well on course and But really have that the put cash is to -- the genetics are bank, $X of really cash refined. great on program we development us good little over A already

cash also maintain have allows very -- of next to clearly, the in where said We few milestones I it. but those will I And think, And our balance us over that that flexibility I us script well, get some years. lot through how we terms It meaningful we milestones. in employing operate. the lot should have this of a in of is discipline in prudence and a and as spend how we

headcount our of year managing peak slowing I is we're reiterate to again, use should cash growth and say, rate So the a this for once growth there. And a just net number use expenses And and the will the we et continue time. that and in-licensing cetera. forward number acquisitions to decrease of look a start of programs over cash net of onetime

puts So, in it Joel, us forward. good moving shape,

Joel Beatty

Great. you. Thank

Operator

you. Thank

Gon next comes Stifel. of Ha Dae from Our question

open. is line Your

Dae Gon Ha

Mardi. I guidance One you for dosing it's Thanks would us. our then Two the be on substantial individualized Emil, I for when data. question commentary an is to duration with that also us upon And next seems data guess what better on contingent improvements? more of afternoon. questions. I because follow-up substantial guess, given functional longer-term or XXX. Great. Good a for of the maybe per from patient taking second guess correlate walk definition basis, can that through, update

what five I maybe some a guess, $X down. through you that, the factors billion kind seemingly about runway years plus walk us net think can of versus guess of I burn that of total cash cash say into Thanks. a coming profitability? As sensitivity and we like

Emil Kakkis

start and with I'll you deal question. can cash Mardi, the Sure.

escalation patients think next expand we've people update confident we to said we without the a of put patients. than And dose that think be of events we'll in to the can operating be forward. treat be rather it was substantial at small and we efficacy treat four enough we're result three about forth we what what we to safety a next of going dose are we're that So, We clear clarity us give plan. drug-related then can that on We'll next is this that that to drug output, worth I release going cohort bits our talking not cohorts the out or and just having and want larger because speak number to data patient’s can when data in come data seeing cohorts. very We'll do as it important, efficacy. you and major the can the

as talked that game in the we're means want talk that of III. have opportunity we to quantitatively. make the to all provide clearly before Phase a will sure data in of about, to an want saw to now So I that the we're we we I a and toward the are confidence time amount toward substantial right that direction meaningful demonstrate -- we have kind results and seeing we've today But next heading

Mardi Dier

little use say, you. that and of development, towards the do this net many moving also [Technical flexibility net I our to increased I've But times. of a pull cash moving and did burn to those And us a manage we need said on lot give a has if But we we Difficulty] don't lot number when we’ll development levers Yes. give has just No, programs forward reiterate be more our goal and flexibility put forward our towards cash us positive be in to cash down of be manage flow we levers to profitability. for will on growth what comment flow cash cash we about positive. runway to specifics our our forward given see coming revenues profitability. our the get pathway as bit if ultimate moving pull would and net towards to and need burn to

prudence know, position, more importantly, We're like specifics. going help Gon don't a not forward, cash year any cash manage we're a So pull to want and in going good we specifically. to flow going I give to feel the if levers Dae But revenues. you forward increasing

So a financially like forward. to in we're we position move good feel

Dae Gon Ha

Okay, much. you Thank great. very

Operator

you. Thank

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

open. is line Your

Tazeen Ahmad

questions. you taking on Thank my for Minor Angelman's.

discussing relates dosing, this actually to that anticipate titration. align to got you just the it think with a And you you So the about to do at originally, FDA when had that ex-US try where US? protocol dose as been do with you may want to slower you've trying to Emil, ultimately dosing

you to could say that's again? take want how do mg that long let's if something And to could table? the So on you go that's think Thanks. XX

Emil Kakkis

Yes.

the a on US priority. top Our plan alignment serve

requires data that that to provide we because as this we -- have we going We in doing, We'd get certainly would it format, which were US just hope We're right to now. which patients us CSR enough submitted get doing expand. a year, we're now we effort it think asked help effort. bit and the the more

So be going this various ways talk to continuing of US. to that in and numerous goal the done. I've to conversations and are them be that division plan we in and believe expansion, our had we part that's would get can that our with year get the

regard the need to I dosing, with to we Now, don't XX. think to go

based need number get to in you single what -- dose was a single the don't it. can we think I remember knock that down, to on originally but data was monkey already happened you dose,

giving with can times. X You maximal of equivalent to knockdown XX near milligrams the get

data I you'd where say, data don't to talking with were. very think we similar it's I a if think quite don't place about quantitative look quantitative our Bayley, at were at there. deck, So I loaded actually get yet. would necessary it the think, actually a in see just also enough But honestly I we've we're from you the to we

off, need So we getting XX I of I give Once to range doses don't we're to it. where going far get we think us is into go. multiple think so the

necessary. don't now, would that's I think not I -- right So

I there just think to need we time up I but times we multiple and give necessary. give can is need we think don't it And to are. therefore, we to XX enough act, to get where going

Tazeen Ahmad

like matter? -- Okay. you confidence go collectively, be clarify will you with final should about that to Thanks, you're you the have feel with regimen meet or what on FDA, your Emil. need it collecting FDA, And just you'll discussions before does now more not information do to dosing need

Emil Kakkis

No.

the drug-related and that we We clinic monitor the now. enough data we Our not can dosing include doing levels enter seeing That we're right in doing we're US and have carefully. any at dose feeling the program events. patients safety it we're was can to at that

phase So determination that afterwards. is the involved US, the point in dose to get not

US, think I to occur. things think number the don't about certain of And say, certain been shown and able which we've questions I there not dose that we've concerns a do assays, now -- were are So, let's of fact, or biologies, the safely should strategy, dose going to think new be elements study, what have including door the the sufficient titration of to administration as the part forward. I well of number the as open in we with the

those So in we're to done going work US the as year. in we that promptly get get can, patients program and as to the this

Tazeen Ahmad

Thank you. Great. Okay.

Operator

you. Thank

of JPMorgan. comes question from Kasimov Cory next Our

Your is line open.

Cory Kasimov

next be readout? I original for was included in you around just provide could patients dosing the And the of Angelman will color, Thanks the incremental any redosing question. Thanks. data on five wondering their if the paradigm? their

Emil Kakkis

Yes.

those thing. wanted I been because get We've it's So very think pursuing redosed. important this a to patients

in and be is someone going That's what ladder point they're and do same who's this So that to for given with. the young US, we're older, patient, we they're And us a would dosing to to dosing that what X. at be it's they're at X dose. time. the we wanted regimen, used eight in five that for which Cohort to dose in good avenue, right, right? Cohort Canada So them get same Cohort X the of or started that get give to it doing We're unable is, X for to one all X.X an in able site going dose

drug-related signed safety patient know try patients We'll is that Well done much as hasn't no actually has But dosed. as can been get issue. first patient get US as a we safely, way, second not one, have the up. first hopefully the and the to US did the in open did the dosed we back So until we get problem, well

what There based exposure. which is was we the of expected. reaction So to on a no history drug,

the all true. be which So said -- biology, what would was

started. the to happy the want taken they that back all to -- that benefit back just though got they get was saw stuff get -- So just long on we're they're these on, that because sad back because to it. get I'm they kids it's before so aching to

as it can. we to So as soon them, to we got get

Cory Kasimov

you. Great. Thank

Operator

you. Thank

Werber next comes Our from question Cowen. Yaron of

is open. Your line

Yaron Werber

I've on Angelman, and actually one quick got two ones on XXX.

you're CSIAS look of the from actually Phase secondly, to study So wanted an to expand history considering some then opposed XXX. I to natural kind once And say, have Berry-Kravis to if a you there if the right? dose, of I to They're just that just not MDRI, do FDA assume just the placebo on site choose reports, doing question a then the Angelman, And at III? have obviously let's controls. an endpoint as is separately a the to on as clinical that Liz then waiting maybe necessarily you and four have going experience before plan also have for

Emil Kakkis

And at have have not just part really safety are data, gone Okay. that The assessing the part control day time of controls XXX. story those before. the they efficacy. didn't for We release of of their the through

can't that was for. I study information, the the information compliant complete we're we format, that's clinical GCP in think administration what reg report So detail format, compliant, asked safety important more but and CSR dosing include

a adding regard lot guess and be a have the little we have control good an the And ex-US theme. So domains, us and But With that we that there's will should dose why method mandate is important in us it. state. I change the we'll to we hit conducted to provide the dose update. regard our US, the the the group, the to how is, be a trial. – the and and for multiple planning not by the the expansion, versus into do seems be be responder get how how interest to a as and MDRI so insight, information control really we're doing data But types is color, I it and we'll we patient multi-domain the of it youngest sure, data With on think more look there's what's analyze ex-US more in there. get once help the more index we'll will MDRI at we'll data the there. a in much of interest banding I'm the in all now for oldest on dose maybe provide patients optimized really

kind group, think complicated. does could control we I that but of make things it add

do. you would say where that track its we're credible we're or the whether will be we're have, that think effect beyond believing And we group effect data it's and looking placebo I say more magnitude. is you're think size to is as it it or that placebo. effect on large that and that effect, effect incontrovertible the dose, to is. I to I comfortable whether think And we clear. your clearly you our optimal is of the real would question But I what and on, which look a you the allow and magnitude now an can would on amount hopefully, the to data believe magnet seeing of a we If about right just be to about is for help, control but think we're hope get is

Yaron Werber

mean, then Also obviously random it's bend X.X, obviously Right. more Yes. power look making up, you I us, of obviously. how I a guess XX for for importantly. numbers, you little endpoint I'm it's regulators I bit of in new a for one just And for it's or in difference a thing numbers weeks Pivotal? terms And two-point it's a mean, X.X if different do not just discussion. if

Joshua Higa

again. Well, phone It your looks have like in. clicked I'm off jumping might

Emil Kakkis

goes screens it at Yes. turns Unfortunately, sleeps it it on, when remote, my phone basically whatever, off appears.

for So sorry that.

– don't when able not we would seeing substantial rarely don't seeing think already be should we case, – that you any data I be I I that think you placebo. – should seeing, are us And know are is think I that blinded in all before, trial. I to actually beyond confident the seen So never lost see make just it's me are things – variation seen think right? but kinds you I've test open-label things that I've of and the just significant of seeing something move beyond test not placebo the And of the movements see Bayley normally. already. We're variability run I the

confident, And they're we're the little I'm more a about an a are they're how quantitative data feeling think meaningful change So than past actually well rather I because absolute that. changing.

that will III, I Yaron. is is we're we're confident understand real about call. having the the sustain in placebo-controlled that placebo So I withstand need to we'll what a trial will help we question might actually that we're sense control Phase what excited think for us is make be but seeing making don't seeing, a call effect your how a some which

that So I appreciate point.

Yaron Werber

if I really look Yes. point than a you a less data, know Because well, mean, at year. you it's placebo, at IBIT did which a

maybe – it's far? mRNA a in subcu? tolerability anyway in about for given And to for – so weeks I secondly, And the GSDIII, is IV so XXX you. QX given question. chance Thank dosing early all these the apologize just I a So think it's just quick questions there going you're very then therapeutics. on know

Emil Kakkis

don't That is more weeks. not go to dosing yet. program. a month early want of the very edge in It's in details It's into two likely expectation I But very this to go thing. early. once the limit every the our

plan enough, Once month. clear and be once doesn't be storage think once that case Our in it is dextrin we because dextrin to a fast. abnormal material. clear to this a limit We need it, month will you accumulate the

have and make If the You can doesn't will the clear be clear okay Does has two, a clear it, you can continuously to be again. away still get three weeks sense? of benefit for that drug. weeks, map then completely it even don't faded it to couple drug the after of you

before. So subcu hasn't we're subcu wouldn't for certainly but looking it done monthly and go we to necessarily

Yaron Werber

you Great. Thank so much.

Emil Kakkis

All right.

Operator

you. Thank

Sachs. of Our Goldman next question from Richter Salveen comes

line is open. Your

Unidentified Analyst

Tommy Hi. Salveen. Thanks on question. [ph] is This our for for taking

Angelman. We on two have

they the age the in much versus study, have might see given the younger more Phase any dosing for under like that are seems quick Thanks. we're naturally that update a geography? needed you align patients a would I/II, on that same do of Phase drug secondly when So wondering where and different be you it protocol, geographies And the develop or different patients earlier more interim is first the from the due the the benefited this to based do when at Would on to might how begin? you the design older? think is to younger III about guidance as this protocol similar could wait be

Emil Kakkis

from benefit definitely from first first currently more get now – the the younger five rapidly quickly. that is question, more patients five Well the better, Sure. think part certainly, original it's pretty they even clear I patients clearly that

that of getting a it it's other. think the I for is than they're dosing as a dose higher was of them for potential factor also

seven the the in getting best, a old current much cohort dose bigger. the than looked really four-year-old but kid relatively the who who's So is years that right, the four-year-old higher is

some nice an think improvement. age improvements I So even just could in It being XX-year-old and dosing. of seen indicator it's be we've the do earlier showed both the but,

us good stop completely So going think don't effects. I age getting is to from

to do effects of we're them. all be good see It to in may think see domains, think it. I going different but I going we're in

just sure is and not make dosing optimized, the flat. we just think I need to

it's be going for adjusted have think I age. to to

X. our a U.S. global on We want would don't separate Phase with think the we in We U.S. to think regard smart to that's program. Phase X move. need in board. plan not the study, get would to get involved the a be rhGUS, have the I synchronized Phase Phase X X U.S. Now certainly would can a

I being and ultimately Phase think get at I on they patients X but information board conservative get and and in think them provide Phase require moment, can they're X. the the going the we

Operator

Thank you.

Joshua Higa

Thank question? next you, Operator,

Operator

Raycroft of Jefferies. Our next from comes Maurice question

open. is line Your

Unidentified Analyst

patients to to Jean or expansion just time of been first gap group enroll the dosed patients on have cohorts is needed how next the clarify, much seven? two in after for six Maurice,

Emil Kakkis

Okay.

each doses assess begin, will first are next is patients their and seven while titrated should two second right six begin get cohort right away and first next They doses are the in So going. the they two the two will the the then -- cohort the titrated. work way get getting away. If will cohorts their where they're be we cohort

help? that little a turnaround in terms there's So dosing up. faster of now Does

Unidentified Analyst

Okay. Makes sense.

Emil Kakkis

Okay.

Unidentified Analyst

quick to early Wilson year-end pace how the more do question of year? by like And one selection pivotal Stage next a you can the the data Stage the or or up then and get you think And enrollment? mid-year is of on to

Emil Kakkis

Yeah.

As was the in enrollment several Wilson we've weeks before before the separated XXX patient. -- a in said required program, or which staged the by each FDA between

Enrollment basically of the So that there timeframe time in has enrollment all, would begun. the part -- of wouldn't enrollment the to the get stretches Phase the program, and get patients there. X significantly. be out possible And but it's are through we enrolling it for

So by we can. are it We'll when forward. year-end. But won't have on an data there Wilson moving provide update we out we're

Phase from before it bit data take XXX. a more little we see time So will X/X

Unidentified Analyst

Thank you.

Operator

Thank you.

Joon comes next Truist. Lee question from of Our

is open. line Your

Joon Lee

Yeah. Thanks.

you. get any you appreciate models to you that it's but publicly I I'm in actually Thank a if wrong, expression? leading aware preclinical animal any UBEXA in data UBEXA antisense. evidence GTX-XXX, that's do have models that complete XX-milligram I'm been knockdown disclosed. your But of near Regarding Correct meaningful me expression comment of the not

Emil Kakkis

induce or direct can It situations. a sequence if monkey, talking UBEXA does induced be expression result us, other for we wouldn't for monkey it about it our Joon, get Yeah. didn't. to we so particularly homologous because unlike

individual will is -- a chromosome. in nucleotide from -- have also which from turned paternal can some the between help So in and result some polymorphism single the get the to because we you monkeys a the distinguish direct versus out us monkeys UBEXA UBEXA maternal coming there

dose paternal animals we -- the we're getting level. allows right? for at SNP the that fact us expression to And verified the monkey. paternal expression we of can the expression UBEXA those look in determine gene, in in So And in have that that in

Joon Lee

Can quantifiable you quantify a more -- in can that context? you put

Emil Kakkis

they're end all auto level you So like up getting Well, expression, what XX-XX. you similar maternal, between regulation, you'll happens very end so when right? get getting that up

Joon Lee

Okay, Thank you. great.

Operator

you. Thank

Bayko Liisa ISI. from comes Evercore of question next Our

is line Your open.

Liisa Bayko

you for question. Hi, taking thank the

of wasn't versus much speak maybe tracking that you and so Emil, why Angelman. noise. totally and out or on that's it like to just understand I Can more completely CGI didn't the kind Just convincing with the Bayley's again? deciphering

Emil Kakkis

dependent improvement is relative to ask plus And scale one where to plus the minimal because or usually plus straight Well, require CGI do move. [ph] to very or moderate The in conduct test of be is the score, before investigator quantitatives minimum absolute process a rigorous whereas, order that what trying the the scale they're two, be right, a is up. move harder specific in what it's stronger and compare they more after has measure -- things view because to to can and tends their to are to sensitive three, therapist and change, the on of other kid do what… somewhat a order to to

Liisa Bayko

Understood.

Okay.

Emil Kakkis

Yeah.

why showing So look looking doing different quantitative, it's investigators the people of at trying when in the way therapy different how because much therefore, this the a think by same show Bayley study, same more test is the and sites. to objective to quantitative the a I that's an with why we're kind comparing you I of comparison trying done is you're better. was I test, across gives investigators, And different, of way comparable think think right? little That's but still a we're the is it's

Liisa Bayko

they're skills on Bayley? into -- rate. at your just kids to said kind the I'm naturally just sure does a that factor I'm trying mean, on this, as of the I as quantitative the as of are Okay. Bayley's, with -- more it's And change acquiring if rate like that they how the do then thinking you that just lower think background Is

Joshua Higa

Emil you're mute again. on Sorry,

Emil Kakkis

went Sorry. off. The… phone My

Liisa Bayko

No worries.

Emil Kakkis

Yes.

I natural Bayley's about So it severity. It the changing. the development if of to Camille None changes have you in history change. anything been natural studied the in say the was number not doesn't of the but Bayley, and history. in I thought programs know the really don't

Camille Bedrosian

illustrate relative Thank the question. and a and quite Thank communication deletion you at individuals one studying the of this deck, measures are references Liisa our cited to in actually The these also and those that. Bayley the improvement corporate in correct. of we're with flat number we're those we particular time. over follow-up you the in also that's of that references Yeah, particular is in for happy flatness time Emil. are in with and patients you have mutations on There

Liisa Bayko

just into this And to Phase you. Thank take Okay. then III. now

thinking So I endpoint. kind think about you a were multi-domain, of,

be would other combination and So some there that? should thinking about how and of be we CGI or things, Bayley

Emil Kakkis

Yeah. quantitative respondents multi-gene use Well, only would like the Bayley. measures

could we could communication score So and sleep, could a the for expressive well. measure receptive a cetera. like Bayley domain sleep severity Angel gross motor change, look do and an be at Bayley those two change for as different use for et We measure we the communication

the So for minimum value do order analysis of analysis is in these android that, set considered we'll each type and responder type in we powerful there'll domains. It's can of The course, clinically be a multiple to quantitative is other meaningful a multi-domain amount more benefit test a to across really of domain there order tenfold is to have approach. lot be multi-immune of that type some heterogeneity types powerful. disorders. This for is of complex than many capture more change

Liisa Bayko

explain questions. into agreement maybe about setrusumab it used? on there's treatments thinking quick. target paradigm in and is Is in, And you're how that be beginning product that two the They're treatment have context more Okay. the just One today? of where some other it like questions relatively can I and you fits profile, Can fit okay? where given would the

Emil Kakkis

have a don't of Look do are Therefore, on compounds that Yeah, bone I'll lot just that. anti-resorptives resorption, they production. effect they that there's I improve related think to reduction bisphosphonate of really an aren't absorption. amount and the the only two studies of trying them access the randomized effect and the magnitude were of strength bone positive fractures. and improving in XX% of five reduce In but

problem solve patients feel really. So, it doesn't better makes it but fracture their

and think think lot improvement room inducing of that there's cells regard phenotype what two other production the are or to osteocyte The to With bone. bone but to anti-sclerostin combination bone-producing last of there. become I is of in really phenomenal, it maybe of stimulate to those best far we also to anti-resorptive. is setrusumab the [ph] so is looked by that of biologics the cells And terms at, a

And denosumab program. other of we the really true powerful don't one it's think the sides molecules not them anywhere out and on So, story stopped other the that does. the this all for pulled at I a of effect. potency that’s biological has looked biologics. some think I the both including and working It's been near of And have

confident at this above So, it point. I'm pretty that the others stands

Liisa Bayko

this year. And just for shape then final Okay. question is of R&D

know I the -- increase it part year, of lot shape of on I about like, as be a here how think were just we year, So, next go into the understand should continuing will and lower R&D -- of you wanted should or from commenting we I know we you this spend? -- studies. you've to said then starting to or

Emil Kakkis

R&D. think And trying We manage it. can what have to lot going a Mardi of sense on. you We're I a give

Mardi Dier

cash clinical Liisa, commercial -- believe funding general OpEx next some efforts we as gave our And R&D, about Yes. of I examples right? SG&A KKC. Hi in mean next we're I -- will seven year, talk and down particular, programs, it's our to in don't But have We trials. we Crysvita particularly So we that net transition Mardi. SG&A, we development go our use.

going lot levers our we're be that programs. that, various among conscious said continue of what Having of major forward. to have So and spending very of we're will the a spend part a we

and measured need paced still milestones. be. We're where major are we we So, moving to through

Liisa Bayko

Okay. you. Thank

Operator

Thank you.

question comes of Securities. SVB from next Schwartz Joseph Our

Your open. line is

Unidentified Analyst

they going on to the frequent reported the it Thank dosing for keep in a patients wanted regimen on provide you just you're schedule you. to going more the dose more we could would in phase, adjust see but to maintenance the these GTX-XXX. Joe. in next getting you the benefit Thank that. for be to I'm your update? taking be And I Are increasing you assuming, question. our possible In data patients to more And confirm question a in maintenance space? have I when if

Emil Kakkis

Yes.

looking near the on we at we to make broad protocols are built through hit we we dose think the we will data. extra when hit the in the have anyone an current when where program on in period. we're patients as we the monthly we a three-month ability the until to scale right for They dose, dose hit provide on a and And titrating next longer-term dosing. where need think So, provide more we update, our we data

need two top get extra let's load the optimized, right or the So once will ability to we doses can we them that dose dose be. then order that to to them right do just one We give and we give two think or we -- if level, should would than we have what an that rather extra in up, figure we to them simply say, out get so more. think

we're So them data ways sure optimally. dosing and to the and on we have forward set get make contributing more current that they're fully

Unidentified Analyst

very Okay. Thank you much.

Operator

Thank you.

Citi. comes Nochomovitz question next of from Our Yigal

Your line is open.

Ashiq Mubarack

It couple a quarter quarter. nice second like the taking a questions. your of I for showed my looks Thanks Yigal. for over guess last sales just on questions. commercial Mubarack Ashiq Crysvita bump

So the growth midpoint during looking to this if second correctly, expecting decelerating the hit maybe your I'm at it guidance like you're -- Crysvita modestly half. of but seems quarterly

your we're commercial curious any seems in there. it just to half. a like guidance, And second see that some need growth your on how dynamics guidance you and be question midpoint So, some or of accelerating as why to at think growth the Dojolvi, might be and conservative the then might you'll similar

acceleration you that key is will what drivers be? So, our question do of the Thanks. think

Emil Kakkis

you because a about but estimate we we know. Great. let's lumpiness I guidance guidance, best equal on want that is a certainly But of to are going of forward bit way. see. put on Yes. any our put something for Erik, say then think thoughts based Crysvita forward We and Perhaps bump, Dojolvi? is going which kind our parameters. we to little other guidance and going caveat see it's Erik, this the Well, it big don't we did

Erik Harris

Yeah.

much half case the at the second which you for year, look same. sales previous has of the been strong For if We're it's the both products, the pretty in expecting Crysvita. years

get So converted ability grow to we're growth, to and accelerate as those treatment. continue patients in to find our new confident to patients -- continue we

Emil Kakkis

Yeah.

here second stronger a had half generally than Erik. we So first you're half what saying is

our So for expectation that's both products.

Ashiq Mubarack

gene maybe of a And Got conceptual therapy question Okay. pipeline. then on it. more your

gene I think you're up therapy to six distinct programs.

you're managing development And about so from comments I'm just earlier spend standpoint, about curious your about given thinking a these burn how programs all prioritizing from but -- standpoint both also time? between these over cash a

Emil Kakkis

and is or been when GSDIa lead very It staged and OTC, we demand Yeah. little order the It We've more take believe to getting be of further with we because program desire the we've the forward was going in added now XX-week between. of we It's a push high we patients back all off. more time question program, treated UXXXX quickly. could enrolled. doing bit off -- study. file. and top priority a a actually has required in that strong of staging data. take bit Wilson has little the would very thought in top the to put file. already we XXX just burn some It's to It's kind is somewhere one for position had

spend-wise piece So X the need like and how we we'll Phase commercial part of in treat bit little is eventually manage to additional is a on. some to manufacturing But program -- required that's more program might manufacturing we that and the that patients. products in absolutely focus a post really should we're only the managing the program have we it

the goods also how improve manage Stage year ones the but kind allow so a more that's lot. cost have reduce up manufacturing back, have. gene the for and earlier therapy bring especially earlier need our of the is we're it And And Xs it. picking down we our hope consistency. our well as and us stage trials to The we ultimately of I cost of and Stage their load, will are a start costs We in will X, manufacturing agree reduce working clinical to which for plant our

and this is your our So own going traction that's to in necessary with how invest gain we we're is products in forward. move as programs manufacturing of to gene to like plan in therapy control continue sophisticated being

Ashiq Mubarack

Q&A Okay. End Great. for of answering Thanks my questions.

Operator

time. questions Higa Thank I'm you. Joshua to you. I'd the for closing turn showing Thank any back call like further remarks. over at this to no

Joshua Higa

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

Operator

you. for you conference. conclude and all gentlemen, Thank Ladies this Thank today's does participating.

Have a great now may You disconnect. day.

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