RARE - Earnings call transcript Q4 2020 | Ultragenyx Pharmaceutical Inc.

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Operator

Good afternoon, ladies and gentlemen, and welcome to the Fourth Quarter and Full Year 2020 Financial Results and Corporate Update Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. [Operator Instructions] I would now like to turn the conference over to your host, Mr. Joshua Higa. begin. may you Sir,

Joshua Higa

welcome Call Ultragenyx and for XXXX. and Corporate full Results Update Conference the to afternoon, Financial the and year Good fourth quarter

projected involve including any Annual the was which that our XX-Q control. reports statements of website be only risks of October views These XX, in types on Report call, Dier, have our call with that, subsequent me the I Erik Financial Commercial Executive Form our actual date Camille statements note am Harris, Private and substantial financial Higa, Please Mardi Reform Director on materially our of many remind Safe Kakkis, detailing uncertainties, this and Chief would find We those Securities on identified Chief Relations. I that within the in forward-looking of Officer differ available Act forward-looking Quarterly our to, call to Joshua the represent XXXX, and that from press all statements filed Bedrosian, SEC, soon And as will today Investor Officer; Chief like provisions the and you Emil of joining on Officer; in are this filed be filed forward-looking on ultragenyx.com. Officer. President; which forward-looking this of including, Report Form Chief can our that Harbor our will periodic the Litigation limited the section. at as but Investors investors meaning website include of XXXX, a issued our beyond could results not on and will results, XX-K Medical release are statement.

our and the now a in to those For to call as description please to Emil. materially reports filed SEC. forward-looking differ of actual I'll see turn from the results with risks periodic the statements, expressed related risks business, well our uncertainties that as cause the could over further

Emil Kakkis

portfolio, afternoon, Ultragenyx XXXX from joining everyone, thank We of us year transformative over for we and approval clinical as on which refilled the programs, priorities. the years substantial you, And many portfolio now opportunities of today. stage Good pivotal have our at fourth completed strategic six studies. are for executed with four we our last in now entering was had IPO. couple a our of

patients for perspective, and have with are treat advancing allow despite also our mRNA programs the We our conversion the rare year. a this efficient creation a value end and to approvals to will treatment. our other treatments. our programs, next disease two pandemic first no and launch more pipeline pre-clinical Crysvita therapy that treatments specific US first-ever the suggest to received programs commercials Angelman guidance upper in clinical From approved accelerate gene approved potential. and more of no global From activity two realize we OTC updates released rare do to from this therapies, in GSDIa for large oligonucleotide the as well Angelman with we syndrome. antisense certainly GTX-XXX our to relatively results disease, deficiency, initial the with of more results but much glimpse program The first opportunities our revenue achieved as diseases important Those XX clinic of promising the us data in

manufacturing their technology. Bedford, Massachusetts runs we variants the PCL our differentiated with began acquisition per on we licensing manufacturing the provide XX Dimension Later construction and recently, to our deals the side, one gene gene has Sankyo scale. ever construct from facility providing to ground will producer of XXXX. system. initiated which of of therapy in selling gene in our therapy franchises, biosciences at platform, manufacturing microdystrophin one Most in broadest rises Ultragenyx our originated utilize year broke non-exclusive our therapy On the X,XXX-liter to year, including Dystrophy the HeLa in manufacturing productive initially AAVX by HeLa with Muscular gene now we solid that in partnered largest Daiichi therapy Duchenne Therapeutics our pair

Our now in-house one clinical preclinical pivotal stage partnered program portfolio program, three with in-house programs. two publicly additional Bayer and includes clinical disclosed

vectors therapeutic programs. is will us Our to manufacturing portfolio. have gene other robust is enable choose the process of business, efficient the that modality the of PCL disease, large in clinical variety side scale, HeLa next the platforms at robust But therapy enable to a we our our strategy just development that make highly each the and modes generation AAV larger of right for

this most For there UXXXX perfect will monoclonal In effective the of a their But our very and study by and Mereo bone in for OI, of physicians complement a adult the for note of are detail antibodies built development that way I and setrusumab franchise new The significant from biology. with with which one development UXXXX, monoclonal program more bone, XLH between antibody of Xb will learnings be in program the program multiple BioPharma, or UXXXX in our change genetic have Crysvita we than program expertise Crysvita. be is OI a significantly December, late-stage on osteogenesis Camille can collaboration the added diseases more overlap XLH. effecting and the genetic treat Phase rare helpful common provide later. OI, largest completed TIO. we has bone is patients imperfecta, and or the with types who will disease future

of us the a royalty. provides we where receive rights of with Europe, the collaboration throughout commercial this exception Importantly, the world with product

challenges, and executing more great performance was substantial able year. commercial I'll our of year. to year strategic preclinical across with While past over Ultragenyx to XXXX a plan detail this Erik our the on success make hand programs progress to was it provide unforeseen full for our commercial

Erik Harris

of in-person before challenges. many of guidance months XX the proud I'm we afternoon, pre-COVID, uncertainty for of in our Thank ago, commercial revenue orders, $XXX million approximately Crysvita truly to closing the temporarily team issued visits of in face pandemic, offices XXXX to everyone. were right in the how you, seeing Emil, never guidance non-essential $XXX territories executed and the the range. we able top $XXX doctors' at of end year all million. our million, stay-at-home the a global at and finish to good Despite

be increasing launched we indication launch XX% on middle on Crysvita year, enabled in in out medical issued ended This as our territories. efforts patients Crysvita XLH For guidance Crysvita's adult on by who other of have XXXX, these efforts, ensuring that more educate shift field is our the Crysvita XLH while the commercial To growth this existing finding XX% TIO Crysvita. to Ultragenyx see will we towards and will strategies in is the focused steady expand both to these well. reach we our fourth stay pandemic. going on year-over-year for of on launch. patients we The year last $XXX to support $XXX patients by community digital many teams these In mix adult represents will the physicians million adult to of XLH of million very This and patients believe during growth a between finding of revenue This patients patients. by for endocrinologists, a harder-to-find be the will driven patients. we and specialties We setting. getting greater portion Crysvita, expanding and nephrologists continue

for at centers. We to the Reimbursement patients TIO stage the major of of well patients is are converted bone from and same majority trial the Start metabolic have reimbursement clinical launch. and consistent progressing TIO XLH receiving drug reimburse Forms with for is

specific We products as region. we being Outside indication to to Latin required strong in making will health that revenue reimbursement to of our more efficient. injunctions continue the US, across inconsistent, are disease we rare patient TIO, numbers Demand in are steady not name in progress to Crysvita the revenue be overall that consistent be leading and with is from discussions patients remained patient for to region health as in providing our sales America, ordering this in with gain guidance. and access other tend lumpiness, sales. more The authorities patterns some see which granted included

full updated look receiving you other keep in Ultragenyx as we territories. will forward to We reimbursement

community. of prescribers Dojolvi, of unique December, interest from end includes seeing long-chain of last which which oxidation approximately launched clinical studies. year all also breadth of XXX approximately acid which At to was our received in participated we middle commercial patients States. for fatty the to completed treatment the and led in for Dojolvi, This product approximately United therapy, we speaks now Start XX the are disorders patients who reimbursed from this physician on Moving the also XX XXX to in launch the Forms the

payers. from support strong seeing also are We

be Dojolvi of I'll to that, call January XX these over the in Dojolvi US of As the coverage a first now Forms the turn guidance to over launch, and In the the the the of million launch. metrics With payers. sense believe of policies, includes revenue results. share will provide and better end Start some we prescriber of which the strength XXX providing the from over Mardi quarters largest of financial XXXX, lives national have

Mardi Dier

territories $XXX everyone, will ending was million, from included XXXX, revenue press a territory the related the $XX.X revenue afternoon, $XX.X $XXX.X earlier year call. in briefly Total million for and share financial other I in revenue release and XXXX Company of sale thank royalty including North Crysvita joining Good Crysvita update, European summarize. $XXX.X million. was territory regions. million Eric. product XX, that Mepsevii Ultragenyx profit for issued was the in which We net you of Thanks, totaled revenue sales today a million. American the for million. $XX.X today's to December

time. revenue end Dojolvi as revenue of July named includes of XXXX also regions. to revenues provided technology. and related $XX.X months to HeLa – manufacturing Dojolvi approximately our toward patient the strategic non-cash for tech may revenue partnership of increase of five This of around included the after services Sankyo year the in includes We modestly transfer expect PCL million. these months the sales of XX the $XX Daiichi over US other total was HEKXXX revenue part and million launch

pivotal clinical and expenses gene advance X/X IND-enabling of research number Phase support increase million. in other of $XXX.X programs In ready X/X we into studies, three total to year our $XXX.X Phase activities study and to III clinical clinical OI. the our R&D clinic. includes were first for $XXX.X get we as Our GSD operating SG&A as million, development UXXXX next therapy and study million expenses we the which expect in the cetuximab cost of a XXXX, mRNA expenses the

as modestly and Ajovy SG&A Crysvita, increase expect of launches and to we to Mepsevii. expansion in XXXX, the continue We support

net the of a loss December We to support For the and in ended the used the and for to $XXX.X This execution million the ended loss increase or in $XXX.X was of $X.XX excellent year and late-stage or million XXXX year share. securities. ended cash in achieve in with December same XXst, XXst, $XXX.X net period same per XXXX. in operations for million milestones loss key position the marketable investments pipeline cash, $XXX.X clinical to XXXX, to million, per fair Net equivalents for in year an puts $XXX.X billion expansion. was value commercial able this period cash securities. us our compared $X.X share, of be in equity includes XXXX, the in $X.XX XXXX Net million for compares

programs. I'll Now, Camille clinical on let our touch of some

Camille Bedrosian

EMA. our has with first storage meeting barring we for good initiate or deficiency, Phase these transcarbamylase DTXXXX Medicines and stage we advice this the have for status discussions, design end the to quarter. where regards current on issued the Phase Out this second feel will with for Phase of have release, of Shifting as completed unforeseen And disease, with with and currently the gene as Type progress and from X, by study, well on design initiate DTXXXX or the programs. delays, end initial the X comprehensive we'll an afternoon, We half Agency, study. about and we up the are our initial confident held briefly pivotal of FDA. EMA, X study, this track XXXX. January Therefore, summarize plans Phase in advice currently discussions detailed scientific end Mardi, half process our are Ornithine meetings, to glycogen therapy that we now scientific our X an of end the data, in have We for received three today, gene next Xa, will Based this European study the the on of aligned any X endpoints. the everyone. and I on track Thanks, regulatory a latest to On for endpoints pivotal XXXX. to of meeting the program. therapy with feedback steps FDA, we Phase OTC X press

Wilson Moving program also designation, third which half in FDA. feedback clinic now will we the the pivotal The fast enter enable to protocol dialogue track our on cleared additional seamless, this IND to, the UXXXX review. FDA gene in track year, to currently announced X/X/X that disease for the recently from has This received first XXXX. program study is therapy initiate of XXXX. and single application Earlier Phase

of devastating SAE the Late Syndrome. a announced much partner, paused Observed and preliminary lower spectrum five associated submitted GTX-XXX. dysfunction and from we Improvement also disorder, is clinic. onset including increases of lower including follow-up first by local of Ability patient’s grade with the fully in and beyond measured least also of the domains Reported oligonucleotide patients last one for cognitive Phase or clinical by sleep has of doses the two our in very primates. measures, which partner, supported Now SAE the amendment manifestations, These the as three scores or Dosing touch of FDA improved study inflammation GeneTx in Angelman and or speech reach X/X Angelman after serious had treatment a as antisense extremity Bayley-X scale, on, GTX-XXX, with disease were Clinical in improved impairment, seizures. I'll ataxia positive Angelman All much year, five for improvements with the antisense first well who improvements EEG GTX-XXX safety in communication GeneTx, developed been broad of two disease disease being the patients readings. at interim demonstrated Global Communication ORCA at have a was information nonclinical balance the for has is treated, The GTX-XXX. our scale All of ongoing is administration resolved syndrome. and were the as of Impression the by in domains data dosed program Angelman including was data five oligonucleotide, neurogenetic syndrome to issues, adverse weakness the on currently substantial in five information resolution and back or events all study the other and region sustained observed higher improvements the and clinical hold. patients for SAE. to nonhuman a is

We administration the following the we the Furthermore, resolution changes questions of first to we the observed but to XXXX. the the some Once These received FDA dosing in the of of reduce started, time were new concentration range an from amended in requests associated FDA and interim dosing FDA. is that contact and from proceed. local resume expected have well-below within expected study plan proposed dosing to clinical SAE. ASO. GeneTx the and additional the activity of the plan doses are are expect half of We and second and enrollment to the half currently approval responses expect study data provided. XXXX, with

FDA protocol Canada, similar and also of A process study filed. trial in application are information to be to expanding other including was previously will proposed the clinical to our countries, where submitted. that We the amendment

study. OI, adult has randomized the with and or improved dose the and and leading XX defect with which others is fracture three anatomical program deformities. partnership clinic in is on XX of OI The our abnormal and excessive can dose-dependent results to production Oftentimes, in ability you of to the released data also the OI that by amount formation types research, a types study This among different across Mereo. X setrusumab, sponsor of UXXXX osteogenesis to an to safety strength, OI The major X, result XXXX through we bone pediatric triggered the be for pivotal you believe in a and enroll in create in indicated abnormal believed three collagen bone our collagen this work The bone fourth are even increase show stiffness, to in in the that a very results showed improvements strength. is is and with improve future or substantial could an achieve weakness with to sites. Study now excess monoclonal cetuximab in disease, the mineral patients a pain, fragility if bone the profile antibody X/X were OI. a X, different data included inadequate with bone we Our OI. But OI absorption mass. a observed patients months. prevention. bone in be the response X bone insight for the significant will Mereo enter agencies, that was monthly could understand We with for cause patients. simply the treatment and bone bone density collagen bone density factor regulatory patients important significant for was and bone in OI patients bone, preclinical of new bone weak to discussions Phase change is absorption, the improving increase These fractures based believe of Pending reduce planning levels. leading we extremely in favorable collagen. for serious where low Phase due Perfecta study dosed

period, their and mineral role density reduction estimated first months. bone based at marker, into in an dose a on identify XX optimal to the over increases serum bone rate control XX PXNP, fracture randomized will looking formation The and the

Through for being pivotal OI, to pediatric avenue for we with also approval study expect half of pediatric The planned adults production the of is metabolism of the which population briefly A the enzyme glycogen debrancher for caused summarize impairs is us While is breakdown developed second Phase an the a the that and GSDIII the have our UXXXX glycogen we fastest toxic the responsible residual glycogen a error We metabolism will in Arcturus, for by enzyme. the the thereby debrancher XXXX. initiate toxic the leads give I gene, deficiency inborn product. and carbohydrate. to is believe the license liver or GSDIII. AGL the the currently the type normalizing in for of nanoparticle will from with X restore glycogen enzyme carbohydrate storage in mutations function, clearing X/X parenteral disease accumulated last program intended in an product to lipid separate is mRNA a liver. residual of and

the currently are year. the I'll in Emil wrap up. to a second half track for IND X/X half to turn back call We the of on in to XXXX now first of and an study initiate Phase the

Emil Kakkis

for and great Canada outside option. with to find Dojolvi and Camille, is treatment to to launch will also and the important America, has urgent with multiple in portfolio, across I In Mardi the in efforts of forward in are Crysvita one portfolios Erik. Latin refilled a continue and team greater market to the US, expand late-stage analysis. progress BD lots a the commercial start, Thanks, to outlying continuing patients continue pipeline a full commercial and Crysvita expanding The patients best Turkey. LC-FAOD, XXXX our strong use looking clinical the of the through of disease year know in to off we're and pedigree who need work active be new going and through for programs. adult is with execute clinics the rare catalysts with business. We're

clinical three business, adapted therapy Bedford, Affairs Our COVID very well products three a Within and we've in the our environment. launching broken teams four commercial ground the trials. Med in to going programs diseases in facility to have manufacturing late-stage Massachusetts, gene

system work of therapy reduced continuing dose will that with disease new also Dystrophy. We a have the higher manufacturing X.X these making COGs, neuromuscular higher Duchenne advance bio-modality improve larger and We're a our on the Hela early-stage in on gene continue and CDKLX even to indications productivity to Muscular global deficiency for technology basis.

acid progress The also the in Phase the for Glycogen nucleic X/X initiate resuming and SOP will We program enrollment We're that efforts making mRNA GSDIII therapeutics Angelman. metabolism have to in complement broader acid been leverage diseases. a means. will to advance. de-risk errors pipeline clinical treated UXXXX Storage plan not and Our study by creating mRNA for Disease our that for type our at portfolio GSDIa pathways therapies with our therapy X in The With on of our mRNA or inborn up -- genetic traditional rare other lives in provides is builds to addition of of first a program loan more out aimed rights of come great clinical targets. license Debrancher nucleic deficiency. also also gives cures setrusumab us expertise and late-stage to our bone our XX

all As moment you the modalities, strong enabled areas evolution in our investing, financial across in continued multiple a our can late-stage programs, from and assets great by company of approved strategic diligence. and performance we're with therapeutic see,

Operator, portion on let's the Now, to the call. the of Q&A your please instructions for provide move questions.

Operator

[Operator Maury Jefferies. from sir. you question comes of from first Instructions] line Thank the Your Raycroft

Your is line open.

Emil Kakkis

question. Maury? there that’s very good you a Are Well, Maury. Hello

Maury his and great onto doing fixed. communications you asking. we go mute. the next for caller on don’t may Maury, until you are We thank why Operator, gets

Operator

Yes sir.

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

Your open. line is

Yaron Werber

Hey Thanks question. afternoon. good for taking my

And -- then, sense. have the year? any I give know question, So, looks now essentially going the I model Emil, you're with it's Do you Thank two you good, another really could II/III. the good Are Mardi, potentially I you that looking? fairly revenues? maybe us a Phase I got pivotal, full. into you've Do pipeline visibility still into mean have do you is How room you. if of for Daiichi-related you we drugs share Or can mean sort don't next us? one BD? for more first right? the

Emil Kakkis

at to look with having if portfolio, tend six stage we right And would you target have -- clinical to programs. think what our doing. seven five now, we're I Yaron, Great. we

in in stage development programs. So, we're relatively state full terms of a

earlier We still commercial If But opportunity. we organization are to are with ability continue we and later development better stage stage potentially, I opportunities, we've lot put look. to would past walk excited to believe what together look work at a it. continuing years. we're want to think that been because from opportunity. to I over imagine were an than we loss have amazing, and -- a of and very we've something about our We don't to But portfolio. you can got full there always the last do, of for I'm do able wouldn't a been place being the couple leverage be we've in

So, you answer question. other the can Mardi, maybe

Mardi Dier

Yes. of back that deal. deal for transfer in we believe Real have with tech with is million completed of this we upfront. we From was the an Yaron associated the Daiichi, accounting revenue course, purpose, March the recognition $XXX simply, that non-cash

million be about recognition in on plus that and So, should $XX XXXX. revenue there's completed that left of

that be it model should way to So in XXXX. a pretty specific

Yaron Werber

Thank you. Great.

Operator

from next Your of comes Barclays. Wang line from question Gena the

is Your line open.

Gena Wang

change the versus there my dosing is little And regarding is have regarding taking a already the original bit Angelman for any Is I change. regarding and amendment? the color the to wondering give It the you protocol. protocol my additional you Thank feedback, your second submission amendment more seems Emil, question if they give syndrome. that, questions. can you questions. two if One just regarding

the and dose changed XeXX. GSDI for ddPCR from used pivotal XeXX study You it the to

the be do you will Just OTC for going similar you that programs wondering, all forward? to do more the accurate program? need future the across use ddPCR change And dosing will so

Emil Kakkis

Sure. Great.

plan provided IV required had We and all them and together very order put questions, amendment cetera, proposed. specific in. the things additional not those gave interested change the to They were with provided we They et an they the protocol in inquiry some response substantial them questions the this information, that what on proceed Angelman, large So, now listened amendment. a did ahead. to -- to they we and

They in wait we'll questions line. our to response that. really didn't their But So basically plan. and see change the were

with I could think, settle think pretty I back do to feedback just their and you see patients -- to or to feel patients plan do is have assure it. get is it, happy want that manage to there the to they don't like thought into wait change and not maybe back treatment. we're well inquiry, that And we work I going to But out. we'll our for we been treating they to these but want comfortable, and much get get them we're safety to think ways done. there's really

to to get we're So -- we want back it.

I think shape we're good say. and in for that, what we'll they see

On we're dose. the really measuring What we're changing changing is not the GSDI how story, dose. doing the we're

of which that, out what amount GSDXa were what's reproducible, happened higher the PCR it's just what more important a closer has -- is therefore, a method, And what in So, digital is And on just drop the turned and is we digital effect accurate eXX It's techniques before. we're the calling considered vector original some and we're working They're and giving, them. we've before certain acute to total really And not it same the the relates the more of when real you biases dose use we changed. been limitations drop methods good, we're variability it to giving actually the a was have method. as it as techniques. validatable representation. and for more was it more which XeXX accurate

accurate quality of particles therapy. is dose and methods, is can And use gene how a that are also continue labeling and us we're this give program, improve think the as these has the the drop. in is we the as But difference, a one the OTC the we that, method. there is accurate what same it it the the representations and FDA more would of of the vector we of was. want product be digital For the been things It's all for is using everyone dose on clear alive to I based many just on to to to are well

it standpoint of we're it speaking, anything. FDA process, change practically confidence the and every So of lot. give quality administering from helps the a on what the But doesn't ourselves

Gena Wang

you. Thank Great.

Operator

of Your from from line comes next Cory JPMorgan. the Kasimov question

Your line is open.

Cory Kasimov

Hey, the good afternoon guys. Thanks for questions. taking

have the First And up working do, the that dosing the first in you and you one know you'll on one running as Angelman, I fact, think, you're do originally? get plan the year in is and you SAEs assuming of Wilsons. expecting. administration saw whether How then half avoiding and follow-up quickly study amended on

Emil Kakkis

proposed redose the we'll way will they'll months. and a existing patients, staged and another in into patients, the the at every the XX also is we then basically maintenance doses X X five that manner a go protocol and Well, look patients enroll

So we sense X get safety? we patient by over multiple both doses the after thought patients of with and efficacy So about months to year we'd And through each seeing are get a dosing. it lower additional that the dosing for get to at that level? the in later because enrolled expect story the these X XX, give time will to effect X us accumulating months of we in a the through the we getting are takes

then The had maintenance dosing, and be that and safety was doses feel Once monthly a improve the the to saw we phase. the X-month we on going safety So more better. they not problem we it. show through the that X they're will only little before helps we same get efficacy go substantial think can comfortable every where during I have

think the can go it to through monthly for, this are and should and monthly we'll later efficacy hoping the then phase, year. dosing on phase maintenance get we the if we're and get good happen through know I So

Cory Kasimov

for move into goes seamless very stage next And that a particular it -- to I/II/III your making truncated in think what's Phase in design? is we phases? typical Is else as then the terms of Wilsons, of all go/no-go there to helpful. of just here decisions Or type the it? something across for process this three That's study it should Okay.

Emil Kakkis

patients through we're three to to XeXX, X, Well, eXX, there's sequence first a drug three X a So cohort, it's going dose dose stages. in In and cohorts. of placebo be all XeXX, Stage cohorts, going right? a

period, show is and dose what's know the interim those seeing dose efficacy be this like dose on -- and what At on During fashion interim blinded assessment that forward. will a to whether make that's an looking happening, in a there we'll the appropriate, and assessment, are decision we that in cohorts. safety pick after we'll move a would in to three the we point, group blinded

about have we'd sure but where to the out harm make out to stage but we've that from data into we some the study, to put of dosing not So stand that some somewhere would and we the the helpful? we -- in expect stage. not want we'll Is crossed that information put conduct there would that pivotal

Cory Kasimov

Yes, Thanks Emil. definitely. it. Appreciate

Operator

of Joon Securities. from question from Truist Your Lee the next line comes

your You question. may ask

Joon Lee

Hi, thanks for taking my question.

when trendelenburg would can in FDA generally Or could Emil, entirely issue an if was from to placebo Angelman? that agreement then And you. if lease to from curious avoid be Is that the is different? learned So possibly OVXXX and Thank study? you effect failure your that that IS? secondly, apply to and using study want the something lower Just affirm SAEs? dosing CTI the be did anything there's sufficient any they of what there

Emil Kakkis

The provided question because I years, they've in justification it's FDA way been been the years, flush chemotherapy for reduce Well, this trendelenburg It's the of not done actually we that local been procedures in and widely. did made established we to toxicity chemotherapy like by think Sure. well up. used And because the drugs. not

been that's -- before. we're something not like So it's done never

wasn't really is a more thing. It was event going that. what's there what of kind So on about and question about the

just we information can a what event. required, more and that interpretation let's local it understand And things inflammatory those our confidence can of right just happening of understand. what's so the want interpretation around of detailed they about they we and being make kind MRIs -- Just the other

capping no there strategy the the other lower this and at about So changes on was point. question dosing,

think pretty think there's basis beneficial. So right be and for I good where we're we're good making our we feel I about a at. to changes choices, do the

label. much on If not how course, patients approved was, during read and average its remember complexity effect it open Now not more I it the regard we're value, the it our points. five The magnitude to it. were on patient patients probably Phase don't managing With create X.X for seeing regard through it for to fact appreciate has of or with points, OVXXX, the think X, is was I the we're of CGI five that too would an our X.X it. product that

So from see was effect. the if degree that you might placebo of efficacy changes far larger than placebo,

changes in other addition including mobility. and things, other changes in supported the observed, other multiple sleep including communication evaluations So trial the that, there and that to were

actually just change. wasn't the secondary, supporting was valuations host of also it So CGI. a the It whole

the some course, we So is with the for it's months that have four, And ground these and real, and were it's five know, is in have of of this could profound. maybe time time patients know the started that's get effect We patients. be course, it's quite to symptoms. again. lasted are passed, of as why we many something anxious important -- the by way, patients think the lost passing, know But we we

in case, So confident is XXX% our this effect. not placebo I'm

too course, just support placebo have beyond And, effects of it. assume a data much you effect. of to would magnitude We the be part what to

Joon Lee

following just up Angelman's. And quickly the on

the exact -- [ph] approach, product have better? you There's do protect companies created kind same yourself. What your franchise know, to you your target not stop. a that have node for around start You could of well pursuing keep

Emil Kakkis

to make, run you've got target you've think fast. I Well, a first thing the got

that's, hard but of, they're -- truthful So too. hit. to the But sort

the rare comfortable. truth clinical about is on you execute The going It larger some disease We're more there, to the to OTC, will ones, if thing other is like GSDIII, we GeneTx play. want works they in and be only done ability improvement. we vitro but Angelman other much be areas. really that it's do, what were will when all produced work we looking there than potent, program. But said, region transcripts scientific And antisense this felt doing doing. the the done had are that have program, people far that more it's at, to the our picked to property, data pretty is going alone -- to to distinct like insight knockdown we work was here's a reason able is they're targeting GSDI, we they're we a day. the the think be not important. company, intellectual We The I some the whole as what and day real all others, all

hard because methods will expression gene the replicate deliver actually have neuron cannot trying therapy will think and and every too that you variations do, The to do to of we're doing I method this be thing. very what ASO other hard. better be evenly, high. can't of you is They're

the ASO, than deal be ASO going allows a to on of trickier maxes with basis than it's to a expressed. gene enabled with we be force to don't EB-XA but It be expressed, an amount lot So basis out protein. therapy we even this endogenous because this it an regulatory to gene to

and we you get confidence So allow saying maybe ASO neurons scientific going targeted allow what believe detail. more is little properly. ASO those. the the methods, best we're that's At this bit think run for I method same best to is time, And among and was the regulated And of a very strong we're our region, why basis doing. that's to fast. the why them be active have I'm to in to there we to What a among going you,

Joon Lee

you. Thank

Operator

the from Your Citigroup. line of question Nochomovitz comes from Yigal next

Your line is open.

Yigal Nochomovitz

you Hi, the taking Erik. for Hi, question. Thank Emil.

was from to Sclerostin antibodies a profile you a of imperfecta. you to you given, osteogenesis secondarily, know, RANKL opposed on TGF-beta romo in or provide on osteogenesis as comment the both in trials? could Phase And target on targeting clinical Amgen light could fact And with perspective Sclerostin. that as recently of that posted strategy imperfecta differentiate then pursuing cetuximab So burosumab I the Ultragenyx for if also is advantages of bit

Given is is you Phase with III OI that Sanofi pursuing know pursuing OI as I. and OI in Thanks. and Amgen with Phase Prolia LENUMAB

Emil Kakkis

you. potential question. knowledge. lot for for And familiar But -- with been variation XX us, of has on This another good for look, fact, so sounds and Its commercial we're works with a years target a Thank Amgen in romo. worked It's very around osteoporosis. romo on that of main anti-flu Michael antibody. Good. like Minsky has Amgen back our

they of development part pediatric As which plan study. for program, Europe, are to run to the them a requires do required investigational their

in apply pediatrics. doing year, chronic look to higher, they test for therapy long not least they're in population, and the their but have the run dosing we And is try going one And running to whereas to the on mines study. may uniquely program. dose they we the dosing which a But it's do in to not have and regimen be products at think What at So they idea is will think of an that in OI. more unclear. population. are that they're do or need just that to the is will their to with phase, I that induction focused to We're the need part PIP

types us we commercially? and with to will. That We'll indication. still people deal romo a OI I allow play we very folks to there's is different will adapt on a we said, OI pricing OI will our the product of patients it. benefited. to and with that off-label in we think lot a there have But the the could lot that And say, to and And be with issues So dosing could somewhere dosing other the important that deal for and optimize regardless of regimen achieve what use can that out. issues have yes,

you that good. particularly molecules, think features point second competing are I unique Sclerostin other is the The some has asked about

bone also re-absorption, inadequate but have you at anabolism. the If look they OI, have much patients too

very anabolic strong definitely in you that anabolic be a And agent. good fracture. agent. And is potent Sclerostin bones formation very a inducing at appears to strong animal strong So OI resist we results of in that want think model the bone

to a doing. Sanofi they're on now. we what So re-absorption, effective. approach, denosumab I on in on on It's to to bisphosphonate. but OI with strategy. I But think OI also some that could product or I RANK given for hormone the keep combination another maybe size I talking best. bone don't is eye have you're valuable, think confidence is expertise that different. And think bone make difference the seeing effect is is our will assume second-generation re-absorption. molecule the optimize. It's Sclerostin context have be a Certainly, a big have bases Ligand -- say development, related to of add about. another probably lot little particular ultimately We'll based what it's real a may forward right what working first running and we is patients. version a just will a of on the be say to our in need It involved It's more regulation. I step another I another is as push we good to like would Sclerostin and focus think we're especially anabolism they of can TGF-betas, in the

Yigal Nochomovitz

Thank you. Super Emil. helpful. Thank you,

Operator

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

Your line is open.

Emil Kakkis

Salveen? Is Hello. that

Operator

sir. returned, been has question The

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

is line Your open.

Liisa Bayko

question. Hi, taking there. the for Thanks

those? going Thanks. all And expect are efficacy similar we For types? DTXXXX enrolling of should for the mutation Phase GSDIa, to in be you X from kind different patients across

Emil Kakkis

mutation. remember in GSDIa, the not particular about restricting a of patients are to XX% are null. But We

it's get. of phenotype expression as can you null as probably homogeneous So

less effect patients we And or as all let's so deficiency -- comfortable effect sorry, because the most better if we would feel expect say, that the we that there patients to have with even treating nulls. of less good we've pretty if is mutation, some, -- be lesser been been

we the already of them based treated. treat we treating the feel not treat. all nulls easier, nulls pretty if So confident right? can So we many should treat harder, then one effectively, on how we've can be We can missense

Liisa Bayko

Okay. Thanks.

Operator

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

ask your may question. You

Jeff Hung

for Thanks questions. taking the

for the the plans For went through single-protocol X/X/X. Phase UXXXX, you

for there program see that as was single-protocol going for design? standard a specific this for So allows do forward, this Or you conducting be study other therapies? studies something you'll the gene way

Emil Kakkis

urinary there's execution single-protocol Wilson There case, know copper we endpoint. Jeff, endpoint. In can factors urinary like ones, that products, the the copper And primary FDA all design one is these make are agreed excretion endpoints, can the XX-hour because possible. the and approved has be key already that primary some of --

design then program straight entire can defined, With you through. the primary endpoint an

for a have do criteria endpoint. in has primary try don't sense? a Does next confidence without analyzed having inclusion that the to configure your make to it's little defined, stage. Phase X/X/X you really in the define hard a right If So, what to the it with of endpoint and level

have there endpoints, Now be X/X/X a knowledge possible for the Duchenne, Phase where may example, been of where is then standpoints for because those that's one developed.

For meeting be in CDKLX year bit is could through that little a it in new that's is query the getting or have X any the may it where the neurologic there, and one harder case, That's different. one like separate of advantage program. straight-through together. something preparing little In time go middle, creating disorder you up it don't is submitting, the burn The you a regulatory the months, sometimes That's endpoints. a right? a that the any to We're way you savings. design do of data, key a thing you almost step to making continuous

we try that take then path. can design, wherever upfront So confidence to on rapid can from the endpoints agency we and get

Jeff Hung

think then talked treated adult And current you about on in the focus the Great. are past, the I XX%. about Crysvita, for of pediatric? proportion finding it's what's patients patients. that mentioned But you

Thanks. time? over Just curious if that's much changed

Emil Kakkis

know, in basically, adult you of don't the Erik, of more been that? want From it's Yes. terms the new anything to to fraction toward add But current patients, prescriptions. shifting I do

Erik Harris

about time shift last adults. to more versus patients toward peds pediatric patients. mix heavily couple last -- this, XX% of we reported overall was the quarters, in XX% finding the continued to XX%. toward XX% adult we've We've adult the see than out more The patients. been And on

heavily. continue that expect to to we So more shift

Jeff Hung

you. Thank Great.

Operator

question comes from Wedbush of next Securities. Your from Laura Chico the line

You ask your may question.

Laura Chico

afternoon. you Thank good very and much,

the co-promote. on up following just So Crysvita

between from you. how wondering following XXX mentioned to about second in guess, key trajectory? as are of it's know I And bit a I towards just the change shifting biomarkers one therapy study, Thank just entering a kind little and just regards the space? part drivers quick the patients. as initiatives be And guess set we're the bit competitor DMD about Or the but maximize co-promotion focus one could is I'm maybe DMD. to you the the the on looking ahead, to you're I if strategic in thinking be follow-up of the there probably kind update just I'm remaining curious adults, gene of this should other, of to theme the agreement. mix then pediatric thinking a adult about? with you thinking alternative opine a in XXXX? So Is on we

Emil Kakkis

you thank Good, Laura.

to we adult We're with and So that the market. not the both still regard to have room Crysvita, finished process. in peds penetrate

about to peds. So XX% -- but got the of we have

So the we little as market, the growing the of lost, they're the to that adult peds larger work to a well as on market It's more adult. unmet probably harder a little continue right? is patients need just there find. And a now, number right

just are deal So don't great growth transition. mainly ahead has that a it. of I we know of product making It's it effort. the to big with about the the optimizing a do

anything about So if, you most else to see that? don't it more really the we opportunity. had where Erik, is to add I know

Erik Harris

as to both the that our to so stated, of And most to just from patients target adult the The finding I that priorities patients. to digital only more able adult But we expand opportunity. is we've of to I certainly and resources teams are, take have as thing and shifted one setting is be add the do community to advantage the commercial that would in medical in where it well plans physicians both of field virtual. top that

Emil Kakkis

I doing patient using average, Yes. important. countries pedigree them four think the three is enough relative has find is of on one are across pediatric likely to thing to related And adults or that drug on because help each we're the

be There a going if transition. treated, that that is, recognition need we how at to on more still many lot are there's to adults But The population that's you doing, we're treatment happening of total there's adult feedback more treat, that an area other lot where think number us the of and thing look in could help. I I adults lot and to the a for getting patients is a adults with patients more and think, that's adults. they're

all So revenue. planning but And same the of transition. remember, we matter. partner product about our growth with we're peds it's optimizing and adults both transition, with essential course, KHK after of the But and working the the continue

revenue much, If you the we at the doing the be genetics post. It's look just not we're commercial of medical will doing but promotion after as transition.

for the promotion of segment in product at role a have will we doctors. least So one in

the story. biomarker Let's – the about talk other

seen another to question, in And you bring what how engaging from efficacy. losing. the outcome accurately think which – like it just at whether compound, and a not having the that once an it do you've really going treating into integrating that, with will biomarkers sounds And do help. muscles to on function. So is effect muscle, function not that were about think is would then after maintaining have that – the asking and the And look markers is early And and data we have we we if challenge about. how think and proving about methods in that's it others always familiar they that and we for before lot time. suggest with it the I think are treatments a takes we're do mindset where translate generally addition, there's here any an think that think stroke be think deliver question. And impression of clinical function, on the have probably it the is, into you should doing maybe how lost knowing And look It's measuring But the almost I to back at I disease truth I to lost think, and carefully muscle partly is of I factor, function they've at tricky. because do there benefit, good I is even, the going clinical achieving. important. you're do is means I the will on impact what though, methods, want which is – to

Difficulty] things So quality the that ensure guarantee all doing we're going But biomarkers are to of validatable. that and I we think coming in we're [Technical is look at the are

Camille Bedrosian

bit little Emil, coming your phone. of we’re from audio a problem having

Emil Kakkis

can You all hear now, right? me

Camille Bedrosian

Yes.

Emil Kakkis

Okay.

but maybe may there a too, hot getting phone I The think sun. questions. is hot problem from good be my the anyways, company

Laura Chico

Emil. you, Thank

Operator

Instructions] of Jefferies. [Operator from the from comes Your line Raycroft last Maury question

Your line is open.

Maury Raycroft

taking my for questions. everyone. Hi, Thanks

I'm like check guessing, newborn I in So on to good going a just with off seems Dojolvi, And say these if screening it's wanted much to wondering in not provide specifics around too naive it in to more I'm on patients patients? use. really you you're progress and But uptake traction can getting and any start.

Emil Kakkis

that that in whole process there's prescribed, still we There have to any have don't quickly happening had also newborns. to had being some newborns story. more – reimbursement without – are Well, emergency a to Yeah. separate we we've we and haven't report cases some have respond

the time in – really have work policies especially it worked. reimbursement But any think whenever we're comes they process if make that that whether doing to will symptoms, is care already, of reimbursement uptake have I allow babies, we'll not, about what for treat don't them, don't been be So and through once and process. they sure I continues. don't talking patient say situation, for you we remember, that treated. need newborn would got patients this we to for to some treat you taken the the continue can to more patient time of have be and be treated, I or to have specific babies the you we newborns to that we've and about But in

drug free issue of anything comfortable immediately could to don't know, acids patients And if newborns. then so time. and you reimbursement I over handling else we're Erik, very on provide process providing the there's the with

Erik Harris

is these Ultragenyx I newborns, been would XX certainly with under to those as product for in six we've very newborns our responsive able some that cases. early been say has as requests hours to is hub get emergency where hours,

such disease, life-threatening newborns. set these up address disease for we're a So need for to fatal this

Mary Raycroft

is Yes, And on question that's helpful. UXXXX. last just

patients you XXXX? provide we wondering, perspective plan study of Just would now more end types potentially what see look and enrolling? by And could like, any you on the if the data on from of can the

Emil Kakkis

the it'll more in clinicaltrials.gov nugget we a expect would that up Maybe, I'm have at – early study? you with data brief just come We detail. may sure end Yes. the some can provide on the in Camille, year. of

Camille Bedrosian

Yes. the a dose drug. Emil. looking Thank It at followed we'll question. will. the ranging Thank and the foremost, It by and finding and study, will. mutation, you safety the dose, randomized of the There'll for patients, in cohorts the and we'll Yes, also placebo patients doses is in with out, first identifying you, by with multiple dose have study pediatric second of for followed the be round patients. be patients. cohorts of addition, start And and GSDIII, there'll initially regardless adults, first

Mary Raycroft

my good. for Very it. taking Thank Got you questions.

Camille Bedrosian

you. Thank Sure.

Operator

the at Joshua Mr. further like conference to question no showing to this would back now am I time. I turn And Higa.

Joshua Higa

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

Operator

participation, Ladies and you Thank gentlemen, day. your wonderful for today's conference have a this and call. concludes

You may disconnect. all

Ultragenyx Pharmaceutical (RARE) 12 Feb 212020 Q4 Earnings call transcript