Ultragenyx Pharmaceutical (RARE) 5 Nov 242024 Q3 Earnings call transcript

Company Profile

Participants

Joshua Higa	executive
Emil Kakkis	executive
Erik Harris	executive
Howard Horn	executive
Eric Crombez	executive
Christopher Raymond	analyst
Tazeen Ahmad	analyst
Malcolm Kuno	analyst
Huidong Wang	analyst
Maurice Raycroft	analyst
Mehdi Goudarzi	analyst
Yaron Werber	analyst
Yigal Nochomovitz	analyst
Wing Yip	analyst
Jack Allen	analyst
Joseph Schwartz	analyst
Michael Riad	analyst
Kristen Kluska	analyst
Dae Gon Ha	analyst

Call transcript

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Operator

Good afternoon, and welcome to the Ultragenyx Third Quarter 2024 Financial Results Conference Call. [Operator Instructions] It is now my pleasure to turn the call to Joshua Higa, Vice President of Investor Relations.

Joshua Higa

Thank you.

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

Joining me on this call are Emil Kakkis, Chief Executive Officer and President; Erik Harris, Chief Commercial Officer; Howard Horn, Chief Financial Officer; and Eric Crombez, Chief Medical Officer.

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

Emil Kakkis

late-stage for programs everyone. from Ultragenyx good to now achieve Josh, afternoon, It work growing on is momentum across great we development and of further build the our we as regulatory reaching commercial our time and successfully are expect If submission existing completed Thanks, a portfolio. revenue top authorities, stage. we approval our

with we a our development further the more the opportunity approved treatment and importance syndrome same X for for could X improve expand late-stage and geographic for to patient year dramatically We efforts for within their the of clinical This so understand X patients see the medicines. community. in and or near-term imperfecta. approvals GSDIa Sanfilippo these These and continue accelerate is potential to access patients BLA reach as substantial last up with first-ever treatments our families. could earned the in with programs both we've growth Doing be our we lives investment year-over-year submissions further growth special and to years, osteogenesis A submissions of to type potentially these revenue

heparan reached for as back discuss UXXXX prepared Medical to points and clinical On I'll And for of to efficacy with support cerebral for his progress could of Eric our point the end FDA rest portfolio. already of on accelerated that in to agreement remarks. well. Sanfilippo have touch fluid data product a make continued have the Chief support we is in treatment Sanfilippo couple be spinal syndrome. announced syndrome, Officer, Crombez, used our we we the June, across quarter, reasonable as sulfate This to a leave approval surrogate that we the clinical clinical

from in pre-BLA where we the patients X end risk older. clinical around submitted associated patients. We the available. Last osteogenesis reduce BLA for has on agency We we offer imperfecta track aligned of meeting X FDA's a the in a the potential with file that benefit for fracture FDA to or for greatly setrusumab are treatment since the product of this the designation flexibility potentially the getting of have or of to participated the X age details years received appreciate recognized this thoughtful X, and FDA substantial UXXXX year. types month, UXXXX to OI breakthrough and to pleased or therapy on as these approach with the We're

qualify Before and the that it Mepsevii, bulk Dojolvi quarter for with does Europe the business, over in Dojolvi. In our it In medicines the Japan, for from reached second in the filing in acknowledge growing Crysvita country-by-country Middle access patient revenue responding we year also the to of about clinical already continuing navigating successfully but launched we team package unmet $XXX to Evkeeza to year same with around address are to and based At PMDA data. the conditional demand are Latin the America, we done a to important I Evkeeza. million in the reimbursement time, hand coming named over Turkey for for and for Mepsevii. recently a on Evkeeza current the generated agreement process East, commercial the talk Dojolvi need this has Crysvita to our I to this and Across have work Harris these patients. world teams bring Eric wanted

the expect mid-XXXX. file could the path profitability. disease in contribute building business in to to to key Japan, overall our ultimately rare X We are products company's These components J-NDA to

in and prescribers Finally, will with LC-FAOD. bone the from patients as foundation who and setrusumab have Mepsevii with who care Dojolvi and IIIA, for OI. in calling These XLH we're we treating therapies. errors of DTXXXX in error gaining centers commercializing prescribers commercial with We're a build for the U.S., to could health other leverage think treat be an we MPSX important about relationships Erik provide clearly same UXXXX commercial also update GSDIa our launch Crysvita financial products for also maintaining The our turn across of progress support patients on are our over Chief to UXXXX TIO. inborn We providers call to KKC's continue future and the metabolism on inborn the the at treat a in MPS gene to portfolio. our Officer, now or efforts to the Harris, progress firm of for contributors the while ahead. I'll years Commercial

Erik Harris

Across you, afternoon, grow the start I'll with meaningful is to States, our efforts. leading Crysvita X low patients more strong. for adult demand are penetration has given This adult or the where XX% continues continue X than by into in Crysvita there start in XXX global and In the prescription. prescribed continues XX% room Thank revenue quarter XXs where with the good U.S., to Emil, Crysvita, commercialization encouraging is were United approximately physicians. growing. partner our commercialization, and of suggests to years came the Kirin and everyone. prescribers is forms writing so from remain Approximately impressively community Kyowa the

quarter XXX America the since year largest full lead of to commercialization, the majority approximately in adding demand. Shifting patients drive XX to continues Am totaling to is therapy reimbursed America the of now strength in the given almost new Crysvita We launch. Latin successful underlying Brazil market Latin are confident patients Lat where and region. the projections revenue team delivered by our in another Crysvita our we our given U.S. revenue -- in on

and patient demand. increasing by Mexico, Argentina, increasing seeing however, are, countries We contributions driven from including Colombia

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

an in with start last currently Moving and U.S. the In experience region, this the patient significant writing sales patient XX of on Europe therapy. In revenue added from to continuing across quarter, launch a and of top prescription. With X reimbursed patients request. we received Germany, by of XX support on prescribers to approximately approximately pricing in patients XXXX, for the majority or therapy we of Evkeeza, more XXX and new Dojolvi. have LDL number new as to in where as and adding to reductions They our to driven steadily of reimbursed than including is with care in number from resulting the quarter named standard on and U.S., continue responding France, than Middle The negotiations. In major the the patients the of XX provide all commercialization forms close reimbursement MENA with forms adults. Japan, to few The levels for patients increase. their countries, and prescribers communities launch. East approximately in continues patients XXX we HoFH we of pediatric under on potential HoFH. Evkeeza. data continues XX the Dojolvi efforts region, and across territories, demand the region, France, clinical the pricing this peds we on the since continue of treated country-by-country the to compared Growth is Austria feedback in them The positive Middle physician showing within long-term the In named Italy, patients of outside been are adult to including approval EMEA East. I'll MPS countries more quarter. we launching start second other a third the EMEA therapy countries therapy is are requests between over have navigate XX% patient half split following receiving the with region. be but transformative new requests grow, our build the QX of comments the increasing The that from on result XX%

important remarks, for an that, just and Japan forward. With programs to As turn the for all to for share and results our the of in call is quarter our increasingly going not I'll revenue expected for the of his Emil but Howard opening financial more contributor guidance on mentioned Evkeeza to be details year.

Howard Horn

$XX $XXX contributed Erik, represents XX% in $X Dojolvi today. by of we growth North million Thanks, the million contributed America, start million, million. contributed prior $XX briefly quarter. total third release year million, Turkey, We $XX good in America afternoon, $XX and $XX revenue and earlier including versus Europe. million from I'll summarizing and for that everyone. and which reported million. from $XX Mepsevii contributed our XXXX, financials quarter our million Evkeeza from press reported Latin the Crysvita

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

marketable million securities. the $XXX months quarter equivalents operations September and the cash XXXX. XX, for used for was As and $XX was million we X of XXXX, Net cash, $XXX in cash ended million September XX, had third in

million year. Shifting net be to cash for $XXX guidance. XXXX We expect to used revenue operations in around the

launch revenue of total $XXX across the between Crysvita million performance territories. year reflects announced trajectory our products, and and our We for and including the strong last are million in range that of This quarter. reaffirming our $XXX we globally Evkeeza of all

expect revenue upper of to $XXX $XXX million, includes range Crysvita, all to to revenue to Crysvita we of and be the forms of all regions For million our continue Ultragenyx. which towards end

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

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

Eric Crombez

good you, and Thank afternoon, everyone. Howard,

to new demonstrate III for In the press already we patients in the with patients treated week type today, programs. addition these through the treatment pipeline reduction These have Emil and that in has been of DTXXXX commented Phase control the therapy reached the the to cornstarch have with from XX. patients randomized Starting in disease clinical reduction earlier group DTXXXX the of with patients start gene study. who with and now our the crossover made importantly, our DTXXXX of originally we XX DTXXXX double is shared treated XX daily meaningful originally use crossover posttreatment DTXXXX. the the in III XX patients a XX% release glycogen are to In receive randomized This week also continued GlucoGene data placebo on, clinically across part have cornstarch to meaningful good advancement progress Phase requirement. Xa. our III time same at glucose of reduce at study the maintaining the point. Also, study while blinded weeks seen the mean storage Phase

study to blinded which patients design with that patients a the important, would of randomized. the without the Phase cornstarch reduction knowing III was physicians cautious of we and While and approach conservative take arm were anticipated

weeks. Now to enables of all UXXXX reduction cornstarch glucose complete, have for for cornstarch and in XX had seventh and times that year. followed an continued cohorts for data are tapering hyperglycemia. and/or or we August patients more dose for need into the particularly deep discontinued stage patients from appropriately, of titrate and the disease. the fasting across received treatment update study increased act the well Phase In that I/II/III patients these as completely the patient reinforces of this and unmet cutoff demonstrated the cautiousness in with zinc month, unblinding the Six sequential in to stage, full during of data dose-finding with included and values, XX cohorts copper to were and consequences as date of The which that the physicians enrolled clinical a metabolism. begun and to is treatment the hypoglycemia visibility this period patients DTXXXX X Last as with able metabolic them dosing direct initial rapidly all activity chelators and fear X patients at to patients the least showing of ingrained of provided Wilson demand. Shifting improvements blinded immediate

and ceruloplasmin We loading the [ the with the copper for the copper in seeing of copper excess body of ceruloplasm of excretion from of body. of the copper (sic) trafficking safe are establishment normal bile onto the for transport of the system into ] evidence pumping the

of an of before moderately and As closing current the a optimized allowing turn study. I'll care to the dose Phase immunomodulation with the I/II/III to plan some provide cohort the a discontinue standard for to back an selecting dose regimen next with pivotal to treatment call step, part now of enroll goal additional at Emil patients majority we the a increased of remarks.

Emil Kakkis

Eric. you, Thank

first quickly by our X marks rest this to catalysts growing I'll close advancing year. revenue, continued key the by year, the Over of clinical the we've delivering for programs. of our summarizing quarters hit clinical our

end Bayley initiate data succeed end For program at data Phase III we MDRI. at point is Neurology leader novel end we the our least update and science the of GTX-XXX to the show on of the supports FAST point are it, and will that been planning will from Based who key for powered This the expansion syndrome, data confident meetings. Child to days. The by that on patients on escalation primary and dose our track to XXX cognition own cohort for year. for the therapy also include and Phase a in Society scores study secondary III Angelman in are very share are clinical also update We're space. upcoming we have the this now that of well raw for the

early than less there Phase interim in early or p-value anticipated In In to is of p-value X.XXX. III order setrusumab, stringent at first UXXXX of separation at year-end and second we a of less mid-XXXX. and portion study a of the planned will to to the early spend extreme the very the a need X the we'll to meet the with a are there Orbit of interim, interim, end an second study end For the little be there order study than the little first the X.XX by XXXX set early alpha, threshold X groups and have to when analyses threshold be final several the therapy for the in stringent our or second months. quarter occur requires first of groups only we of the announced met. to interim collect clears patient results separate. In need would the all for that fourth line and be readout share analysis, the months the was have analysis clinical the analysis to time on visits more analysis. time is study a as the event couple patients months not Top event the for threshold, completes the at prepare a data would been data accelerated after XXXX XX that later we'll over threshold final the and complete an will In

for opening, the one also BLA working in mentioned I we're end As on multiple of around the UXXXX year. the submissions,

is Another for DTXXXX expecting in mid-XXXX.

options. As in any that late-stage largest express of real patients one want talented incredible potentially to first-ever is couple treatments to without next I think the Ultragenyx pipelines executing to final approved head new for month the industry. of years, team gratitude the launch It year, of in we of over the the we bringing can of the X my therapies

would on programs With the instructions. over let's just commercial a the questions. the provide to in decade please public Operator, we make move that, market. With Q&A approved currently programs on have, X your that

Operator

[Operator Sandler. Instructions] with question Our comes Raymond first Piper Chris from

Christopher Raymond

maybe patients? And to rollout, means, all model be, about a this suited Congrats what chelators there? for more I guys from a you're maybe us envisioning this explain the the this you're using binomial therapy? maybe, what only on are that little a the progress. bit is this first controlled targeted follow-up color those a X -- commercial program, guess would little Maybe on on well on as Would also bit patient patients on doing or Emil, most who Wilson negative ideal that what are setrusumab. is just you gene that give questions. for Or Can regression zinc? you for be severe then copper

Emil Kakkis

Very good.

the into All You really wanted right. that me go Chris? of to math

patients of XX% better XX% start meds who there are talked of addressable Wilson of or not very the Let's highest, may about stable most be several there's are, XX,XXX, of the question. of that are a Wilson's patients could probably the addressable. well the can't well the with who's about most or There's We've and controlled, controlled addressable. most XX,XXX about really out tolerate the the they not population, about a program XX,XXX. certainly segment may Among but remaining, patients of do be -- than population that a population some times

wouldn't getting that ones who we we not necessarily are tolerating will the are copper all having are think that be clinical So treatment. patients assume patients people those Wilson symptoms that might the combination management not but optimal of benefit addressable, and from are do

fraction it our more negative years the has that XX very that to potential it's -- Given analysis, Head event-driven best large P. a market. With for than do biometric who K. for a anyone, makes has disease binomial and disease setrusumab, population statistician at is Genzyme highly was FDA a the is done a the agreed Biometrics, is programs of of basic large experienced total rare still model regard and a probably believes way to. an model to Tandon,

for looking like way factors the the -- math in on some model. rate But able model to things and to explanation the at control look go event other baseline probably are rates patients. will can for it's that or provide events be different that to through we pretty would difficult, and being for me be fracture age between at or probably different We best but investors

while in Orbit study. well is explain the can study succeed setrusumab it, powered I say is can't I the very So what to

Operator

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

Tazeen Ahmad

the would be to to has those calculation first is this given success Emil, commercial That's a past likelihood continue on the the there one reads, lines clear. that eventually the interim, time that time secondly, about of very question. of if would depending for study at when with of stop you've which stops, that the the at then time made if you've the point interim first provided that OI, would trial? On the And assuming significantly different become successful study it is question line the

Emil Kakkis

understand. I No,

with of will all, to have how first on think It In the doesn't powerful. -- lines we whether So first or the that not fast the not they be the the any few first do a or dramatically impact has outcome. whether months separate kids hits could separate, at interim less words, other be but time X.XX. months one it for at hit more giving not later. few just occur help interim the But fractures than the X.XXX and of could a to separate. second how

So it doesn't efficacy really affect determination be. what the would

in should if doing fast speaks at the the it I to that interim and for efficacy we toward of the think drug. goal filing. think the end level try possible program to And don't how speaks they question It first as it was threshold, quickly But all we to are. as head to Our they a variable how the of separate be. just of hit

the with line, time help year, time, -- interims the to commercialization shorten the or difference with at the put go that to obviously, year reason there's So or the end towards to there. potentially IAX of the with in XX a regard month the

substantial between first first some interim collect interim, a data and the will months the is from extra We there final. the number of but

year. The occurred, everyone great interim, because the differential a be the had interim will when have second will secondary not as

any -- have So to information. have to further means don't that wait we collect wouldn't we to

not months BLA will long second after the It of filing. few interim. only probably a in be as terms the be will after one So

look a company that. the The how in parts cash of of few carefully months because represents so is time of amount just argue do very one was of as you the the company, substantial and disease a worry there. can each the And value dependent -- -- months value highly terms many month on the at achieve spend program affects you in value value of execution. story, rare So we important understanding few value a not about to a how here, why most NPV success

that's a very get why by promptly So way, things present we as hard on months, we done big a net the press has impact value, few to can. and as

Operator

next with JPMorgan. Our Rama from question comes Anupam

Malcolm Kuno

This is actually Malcolm Kuno on Anupam. for

Angelman at to what focus we with should the regard So coming near-term on near-term the updates medical conferences? data

Emil Kakkis

III patients on provide on of And the you to going the day we'll have more focus that's trial. the Well, reason the through to data XXX. doing allow bit been group, see a longer length Okay. will day so XXX little long-term Phase it is how expansion the

what fact the the your So affect also XXX the or will give the like you of to population. We're one looks to that is you transformation analysis, not problem. view raw it if a power. going It help doesn't show day you for matter day at as the as of really that doesn't a it other GSV use

use So was people's of we'll or raw help number that provide FDA's request. about are from which on requested think GSV we the we a questions that will settle the we based investors as information, planning whether scores

safety a on couple talk also of there forward. sort III the in we'll things, So -- overall going are of plan and we'll Phase update

be won't minds and step Phase be at, but update, massive completed. nice getting is up, which Phase are our it will heading So III running where it forward a III, to a

Operator

comes Your Barclays. from next with Wang question Gena

Huidong Wang

I was FAST the you set? more more same data setrusumab and the question previous Will did study. meeting provide at bit last XXXX. one line. the the I Maybe likely regarding time Phase remember data And the a quickly III CNS beginning questions. just regarding clarity little of actually follow have time mean I

some the up is patient And to specifically interim, to III beginning that picking second will you the you Phase XQ and comment when already also range more not, Is happen? And regarding X clarity If could baseline characteristics 'XX. have this age XX? because of you to of at is and will then share Now the year-end between XX 'XX, definitive, XXXX. XX on also, and event's rate XX, attack baseline breakdown very XX patients, trial point? of at the

Emil Kakkis

a lift. Great. That's quite

the question. for you thank So

on child the will data the and be be not the from There meeting will FAST for not on differences timing regard be -- early collecting, is specific be interim we're later, between the the would XXXX. then the first was clarity not on from the it's cut few set. always same data And beginning a that data X. With based being to middle the end that of a more those saying based for XXXX, said peds, months should little fractures. but So months. the we're neurology timing, Angelman so We year, few it a

the was weren't intending we was related challenged, were to So trigger, about we having a talked in where but change just anything. we none the number It being But we ago, the of number change did hit just a because we operationally fracture it fractures. time time change timings as to line so But of estimated -- when fractures. is. the long certain

have so patients that that's closer those said more our to has is would out We'd where or date in but X/X, population We not X when characteristics more to half but we to III bring the II expect as expect and what we're data. more III we data time. about type data, and difference. III the there What patients, in was to at characteristics, will put put haven't out out we'll IV Phase the yet, the type to baseline releasing that Usually appropriate Phase was fractures. we have bring we Phase until opposed

peds a the the the the spread groups the majority to of put have It age we is It study haven't than now, in fracture expect across ped either. before. But study breakout for in rate trend are what We patients -- age is that III saw enrolled age we right higher in groups. Phase the then with primarily range. study the

the we have we -- randomization for where populations the We are in good stand. that sure make That's stratifying groups. questions. we're similar both in to Thanks

Operator

Our from with next Salveen comes Sachs. Richter Goldman question

Unknown Analyst

on Salveen. progress. is This on for Congrats the ] Lydia [

confidence on to we the want this set regulatory data Stage disease, Just expect what data and would this from guess commercial from I additional when in to Wilson's could X And cohort? gain see see to outlook? you

Emil Kakkis

the Thank you question. for

long up yet X, get at and we on So fourth the It we Because of haven't that the treatment, Wilson's we're depends exactly time months, take get wait start put to but a enroll in will hoping so to when X would the accepted. how patients the get be cohort, set least those year weeks going get But it It will in have line early most until to to get to come. takes there. we how of we on data XX on. next year. that expect protocol depend the study so specific will or of we it quickly

it have enroll U.S., primarily the takes We data. their then are the that and doing how in cohort, all settled get that X fact fast we it accepted and get to that and to

off in of until patients. the a majority all. patients, say, we this improve believe in And standard of a want as we and we mean to patients see potency higher And we higher to at care makes have a point, activity so need that make it sense it's hand, a we in to Phase important at consistent improve fraction a of viable, in and see year. What going want a standard work didn't majority will way worthwhile. So I we And simple of gene have we would we think if not potency III to take because make hope potency. to see profoundly good part a next to is modulation, that care therapy to We it distinct dose. to while to want patients of to of invest we're looking is, for reduction

want fraction looking We in changing a completely for. what we're large that's biology we're see patients. to that So really of

Operator

comes And from our Maury next Raycroft Jefferies. question with

Maurice Raycroft

progress. Congrats on the

chances or second updates for wondering key around follow-up a interim the less II help success just of you'll patients maybe clarify you have are updates. data XX-month the that the ASBMR fracture that new follow-up, setrusumab, interim rates Angelman at what just data and from first have. update you can And learnings Phase For triangulate in with you if quick if

Emil Kakkis

Okay.

the and separation a from no the learned in period can lead fact, separation, to update degree time. of XX-month very we over what think setrusumab these have fractures of profound that significant of can a I patients having that patients So was, on majority

we The patients improvement thing bone learned in dramatic other is mineral the that particularly have younger density. a

We threshold that or we a gave stringent in be, think in early. because us months strong large, the quickly in and that that separating separate they confidence expect for what the one. effect place X interim We the the how I within if the would first are effect we putting could if X size So could is groups set is for more very just first know sure. and then don't learned

second less confidence can also the we that. that time It to that then gave finish modestly so. But us the The of lower shorten one, enrollment. can line patients do number us confidence data we and gave

and So can. With everything learned so that we that promptly we've far tells Angelman, want on, are seen going strong a as effect are what we day to the those reach focused as things we patients XXX. that primarily and us we on to have we expect know. regard we And through to

is data the are to important need point. path on on program. Phase stages have larger of a We'll for whole III, where randomized The study in patients people piece are in up focusing Phase telling be information -- We where really a XXX XXX most the minds will gain. will day really become and series about we different on III, we for through day main our setting at people this the focus of

Phase help set what the focus it So people look II up Phase is, was see that's would what how on and like based our III the data. update

Operator

comes Lee from question next Joon with Truist Securities. Our

Mehdi Goudarzi

on Mahdi is Joon. This for

on study given patients X XX that some what So to Angelman, are mutation for this end this cognition on and the Bayley-X the study? please provide included? diverse optimal Aurora type color aged be could from you the point So be And the would study? for one should

Emil Kakkis

Right.

So that Ionis you're the about talking is right? study,

Joshua Higa

about I Emil, study, think the Aurora open-label talking study. No, the he's

Emil Kakkis

out to of said, talk what represent I'm main he quite sorry, there. year study, The is the the didn't let's X patients which XX% study. main Aurora Aspire. I deletion about type, understand Aspire, study, XX olds with the so Yes,

within the to who demonstrate as study. trial. We'll younger similar those who include other but deletion idea effect program like of Aurora, UPD, have genetic the a we've missense, the to in the supportive have this open-label X types have a will that's also -- deletion range. safety patients is in type or are would And that a they range and are the to the deletion ICD, in For patients the setting in efficacy patients Angelman. older XX the randomized at This have study seen that an take labeling are the be ones look where deletion

the drug, The We study So believe will in to -- effect the to idea age these study. some on and one large genetics the pockets cause them in these population cover a chance of will enough treated. prove Aurora randomized is other trial extend and and of the they is comparable the the safety and to to get Aurora get subpopulations. try various help verify efficacy

Operator

Werber Yaron from with TD comes next Cowen. Our question

Yaron Werber

on transfer a a decided a committee, becomes that ratio. to XXX. and of questions. you and complete So Emil, to would to the the around just have events COGS that totally facilities which when hazard is study they just on Maybe to based filing you Can to I tech now -- later? And the positive, on know, first and, hazard secondly, Z the Or us when p-value, of and in next couple statistics couple let's be that obviously the is a further you you question. margin one do new to least then not hit say, understand it I facility? have until a really just fracture at and At at anything committee finish the got a and on And the ratio rates terminate hopefully think you you're to say you time study, on can and own your assessments. whole the mid going they the share the to need because first with us anything goes let Y, initially I this will independent Did year? you hits setrusumab, scaling based certain recommend X, you the

Emil Kakkis

Thanks.

The of ratio data certain locked. locked. any we part not of other visits. little the and the partly final collect data the detail a hazard at out interim, is we all will a study more put not the it So because or locked, not have the There's will be time and bit -- to database

So with the database out put any set. to not locked, we don't data want an data of incomplete

allow entire not is to a and before to -- will the the that point lock -- that we by see end from database hit, the locked. interim we want study endanger we'll but data the database wouldn't that's want releasing the fully So interim us primary to

why will out the we -- that bit in move to that and tech we're though, should tech delay a -- to and little fact way want of big transfer, works be to investors hits, enough transfer a be that occurred. the track has it the that's So there'll drug give data. confidence ahead. Regard before on we The put

running are in We PPQ lots plant. the

the XX%. that plant will is probably around Our running expectation in them save us

margins out We plant our does and by structured a contract program. own and over our be the But cost plant, own running haven't so the improvement costs. our plant manufacturer. reduce an will outright, in put old We COGS substantially yet it

manufacturer I process because think, there. almost be process. internal team to an But at consistency because team The and and develop transfer Woburn will the tech actually like our ability And it's Woburn the that control the developed ourselves. assure we've to from easy -- our great. transfer the didn't our facility contract is like the execution, It's

and of other plant with the COGS business but we because leverage margin we to for to structure lot will XXX making improve the a which in out programs going think work to be therapy, in in investment we successful That's success cost only will gene the better. our ability gene not is important have forward. -- and this hand, going the help a way So therapy why of made

Operator

from comes Citi. question Yigal Nochomovitz with next our And

Yigal Nochomovitz

Maybe for the reduction us that treatment specific to appear you gave DTXXXX, I in the on period. that primary XX-week the with it were but doesn't just missed regard it, cornstarch for patients the

we the the up way to and received information? have I the And know catch ones crossovers. it You that that up that they should the thinking given they I'm is gene about catch would think therapy would that just were

Emil Kakkis

Yes.

during the during XX-week to phase, is primary So had blinded that said in and better. we look the so getting the the are glucose now going able values, at -- what treatment group down, they're period they're XX% their a reduction getting better they now

and XX% what versus if the commenting the what know are that was glucose that that fact you We point that XX rate your about in on DTXXXX are at you a XX% you're values know is, and group. for time mainly cross over by hitting have getting the treatment reduction you double the you of weeks,

they sky a that notice blind means they when they're low, really we in that don't they for were know if values, -- can people the patients double on. The glucose high. what it shooting saying the while realize They they feel to just Yigal, then, hypoglycemia, that reason necessarily. What hyperglycemia, tell reduction we're they're real have if by knowing you So would an And really you ago, didn't don't hour have were see you bad. their high. if cornstarch you're don't is running this,

feel also only doses lower and because lower will -- come transgene induce they're as It's to allows thing and lows. The glucose high is reducing higher So stimulate are their when the the can they that's your actually the as exercise. understand allows to the you to the insulin, transgene like to will important transgene then down or when almost smarter that, you starch glucose, expression. be monitoring need they have more trouble glucagon them therapy the it and know gene The thing them starch proactive. level

bit. little a it exercise to need You

a inducing bring lowering to expression, you allowing cornstarch it more driving of effect glucose synergistic down. cornstarch, become So will down,

the treated down, knowledge this their without original now makes people to direct -- ground gain blinding expect glucoses. having explains and manage very with but bit what's we it and a to coming for this happening we'd little think So patients the start disease intermediate that hard

in that commercially dependence it on -- thought way apply it the that So it is improve by is. fact applied on to the cornstarch if will too can encouraged low we're be we fear a and quite their important dying glucoses. and suddenly to it their we be would it in what And just think do investors you of patients reducing in going the particularly lot see

Yigal Nochomovitz

XX-week ones at from the beginning in where in very driving I from the therapy will guess total is data were a originally Okay. the of what become there I point that the was share structure. cost terms time you'll on

Emil Kakkis

second in out -- point with feel going Sure, we We we'll put XX-week all right updated scientific here. year through they're like that But you. data when complete Yes, meeting they're But in we the said, We'd so get data. they're we're on the group get at. we'll data should comfortable through particular get out formal they I thought the out of piece just put this time. where to a put data their a show way. we to titrating to further, as everyone

Yigal Nochomovitz

R&D too specific and to terms path ago in the the at a corporate just and York, It a you recall in year year. cash New profitability. the wasn't sort And numbers the just more I picture of the profitability. slide of Day a on as qualitative far a exact then over as had

over revised have any around Japan that. that now to little So comment million you that I million burning time a in further mentioned you're bank, cash accelerate I'm know $XXX could on you $XXX if the and the wondering a the year in launch just thoughts profitability. could

Emil Kakkis

us. the other And programs detail. it X Yes. will the and we a path little I'll we've let in help starting including about ones the good put But help we're to Well, for make. same Analyst Day, the the approved before continue to and more of will forth go contributions new launches, product at Howard on feel to example, is the launching that. portfolio, growing through product Evkeeza,

about maybe, talk probability. So can -- you touch to the Howard, path

Howard Horn

will by have XXX said we have question. XXXX. that that the is XXX to to. profitable of or Yigal, the did cash a that plan the monetization June we we fund the the for we we GAAP like [ with feel What in us our PRVs Glad operating and have quarter cash of things and thanks to gets coming end ] from

end of that what underlying that pathway. And in that the about a revenue. So is launches but the the to P&L., you if And a logic 'XX. year our again, that's translation by that's spending, Add we on enormous bit of the of of level programs that about from we sort see a commercial grow get profitability, ago of quarter before the -- to then all little said the an then rest amount. you out not the how kind continue of

Operator

from Ed Wainwright. question H.C. comes next with Arce Our

Wing Yip

a couple Thomas Yip of Ed. asking questions for is This

a first, III Angelman the GTX-XXX for Phase Aspire study. question syndrome, for So

this you was So question that in XX-week or step development we we the design duration? it And you XX then from that can time discussed next April called preliminary period. program in point second Can of primary incorporate discuss for expect days significance discuss status can and it XXX, efficacy progress, XXXX? this what

Emil Kakkis

the depending the we in the file. where go data year, will final end to months but the finish have year. finish to to the Phase on about Orbit and will we'd We'll filed, out the all get to which as BLA Okay. after XXX Cosmic in getting it be XXXX to help be And our take the year is we the the to of go that III. at filing we and next interims come study well as study expect a few assessment, sometime expectation, unless

really drivers. it's more question. question the Orbit heard GTX-XXX to things the a data others sure will someone I the So I or from clearly. and Aspire, be from wasn't done, Eric studies and the few Maybe but are other quite those heard Cosmic. Regard is be primary need that to really and package There some X biopsies

Eric Crombez

I period obviously, to assessments think start we we without we there think go that develop, and these that the long. letting question I a study the was And to skills the want or we follow-up think, with be to why too did. -- detect to the selected new children importance sorry, chance the able I give the have gain on question on

also we not lot data of I/II Phase the on a key on primary duration. have secondaries that based that end presented just the So so point confidence but at far, we

Emil Kakkis

Yes.

time day we going more see, it if there I that's you more you your there some are little separation. worth that weeks. And things that went faster gain think but moved felt question? and shorter, would like a than why XX were took we to that XXX, things Is

Wing Yip

it. that Yes, was

the understand data duration to wanted of measurement. the Just

that helps. So

Operator

comes Baird. next from Our question Jack Allen with

Jack Allen

a Congratulations to what lot but we course of was around some I analysis interim the the context provide the of team been quarter. and made I over call, on the on know more hoping progress the could just there's the analysis. discussion interim the step on triggers setrusumab and back

there there to is stone to about think time of interim Or when previously accrue? set incoming the relates was it to are as interims determine timing the be still made I I just Emil the an that a those to understand, accrued by data in interims fracture comment want And needs estimation analysis? internally of to rate.

Emil Kakkis

Yes.

was events lot So challenging, where It plan line, the left that move to they're a setup unknowns events do set when or interims on they a occurred. on based because now team. the originally, but the up operationally the and it based the on was back time of could to

set that So the fractures the we line just just to XX% those time when hit, XX%, be estimate and we'd decided to X. expect those down, and laid at

fractures. type expect the will the we'd after fact true the to the we of original historical the would and that, that at that, estimating make rate, than best, we because Because numbers, for what Therefore on saw the to their be fracture is confident have fracture based are be faster is been -- of XX% we example, that estimation III that We rate fine believe fracture be, IV used and faster probably from rate the will hit, because estimates. but going than we're what

in more on the any our So line choice, the forth we which set just described. Was on just they're presentation and time current corporate this? but is there time an put operational now, what we line it's color, Eric,

Eric Crombez

that's great. No, think I

Jack Allen

did going potential And of how onset just time for That's the I one follow-up? Can guess when guess did you really in. operationally -- you rather the that were it I yes. interested estimates? fracture that's estimating very the those helpful. than make off setrusumab you about the ask fracture all Maybe I to I'm brief in when relates think making thinking as rate change

Emil Kakkis

months, in or number onset number we and of few by less. -- the gaining so fractures X declined relatively much looking after that had the months, time of X and looked really about data. the much, estimate, by started The XX-month X It the after at months

our X fact take is within separation And that was probably so in months. happening

people osteoporosis, that with know occurs separation the anti-sclerostin. around for months Now a X

it not it's you we've to children what that be that's go faster. when learned. might surprising So so But

answer for an part that? the Now Erik, do have second you of

Eric Crombez

Well, Phase younger Orbit's guess, in the based we changes? to decided II/III, Yes. the when was part, set. this, we seeing original I these really patients. we on it I we make mean When the second really only had results started adult originally designed remember a data

to seeing really profound much were overall study. we recalculate Phase and design part younger II/III the effects III that that these earlier So able once of started we really power of in and children,

Emil Kakkis

wait and would during event-driven it do than was the from But made started It's he think versus think upfront that just rather to interim still the we same it right interim. actual the the matters. see. Is time-driven about decision year decided event. operational were change I don't you be to sometime an thinking It what we essentially we -- easier was when I it asking?

Joshua Higa

think next Operator, to can question. the move I on we

fracture we're was Phase operational looking III not there that caused we that underlying rate, not is had that also information it's of so that that the at think I from change. the sort blinded some

Operator

question Our comes Schwartz next Leerink Partners. with from Joe

Joseph Schwartz

be follow-up the I on needed interim different that helpful. and was a you And also in of talk that very range if order could II setrusumab. questions hit have might Orbit, compares of a calculating couple in how Cosmic. sig I on size did have be effect wondering, Phase you then effect the first, at size then you're a separations to to little in how analyses bit Orbit, on would I about Great. stat more how

Emil Kakkis

a assumed XX% X.X rate fracture and a in Well, we reduction rate fracture the estimate. for of powering

However, higher based not of that doing on could be rate fractures the for interims, happening, interims a the events choice the reduction. fracture was concept there and more

there power to if earlier, just more are right, events the result so defined. it detect And fractures, that more improves because

the effect used and was and both rate, the of power was fracture So size XX% in the X.X, what design.

the Given that or to binomial, pretty XX%, similar closer could we've I be that was good position we which study. rate the fracture higher reduction with comfortable in how we're the designed feel in think

that's with to sort effect So what happened the regard size. of there

Now...

Joseph Schwartz

sorry, Okay. go sorry, then what kind Emil, ahead. And --

Emil Kakkis

I'm ahead. go No, ready.

Joseph Schwartz

Okay.

there So for setrusumab nuances in you you assume to hope calculated of treatment points Are any Cosmic effect how do terms of versus a powering in Cosmic, to your relative terms bisphosphonates? do the in end see what of kind are the What arm? Orbit? in

Emil Kakkis

majority -- were those vast about had bisphosphonates Well, something mind those bones. and their of part are Orbit patients the patients The in bisphosphonates. in Phase II been keep is Orbit the on

the head occur that when in tailing reduction, we their in really have That's head Orbit. similar actually the setrusumab a the XX% of expect Cosmic, terms everyone looking some not will, So XX% of on right? But similar differential right? bisphosphonates is we'd with because with effect in bisphosphonate fracture bisphosphonates, at them like that's if in be Orbit clear, top of you they rate. had in to really It we're -- to an so even so is in to bisphosphonates, be expectation our went might already off bones. There reduction be like on upfront, add-on, just

in than XX- It be be in assumptions which is higher, XX-patient Now why higher. range our fold to little can the the partly the now. rate study But is fracture XXX. right rather much several kids could that's

Operator

next Hung Morgan comes Our with question from Jeffrey Stanley.

Michael Riad

Jeff on Riad This for is Hung. Michael

include for in heparan is given the sole in it expect the like UXXXX case? data I in have other follow-up. Or just in sulfate markers assume maybe like is For and association to HS biomarker their BLA? to do downstream a NfL more reductions measurements you safe Bayley, Sanfilippo, like

Emil Kakkis

heparan as Well, Yes. presented, we have sulfate. we

probably And also which which these elevated have also NfL important, are we or pathologically We have data, also data. we gangliosides, brain and volume have then decreased.

way all from to chain support the value all a helps have through the beginning what we So biology the Bayley. that way is the that efficacy

Michael Riad

to as similar in That's setrusumab if that like first winding analysis? follow-up, a like a fracture have like Orbit, Orbit a enough you Orbit detected you sufficient clear would at with the then have enough like for analysis, treatment like And helpful. -- effect would the interim to like representative back see have interim separation seen been

Emil Kakkis

think probably I fracture if X.X then is the the rate XX%, less. Well, interim much of and first hitting

a us what interim is the has to fracture those if interim. asking? hitting if higher, fracture Is The opportunity an is you're first chance the greater, would first of reduction the give synergize and and that hit

Michael Riad

No, no, that's very helpful.

Operator

from comes Kristen next with Cantor Our question Fitzgerald. Kluska

Kristen Kluska

placebo thoughts readouts. We hoping diverse get had setrusumab, On color know studies bit more about was the X a little to I that bisphosphonate arm. around the

there you to about are figure? clinic? that being placebo the trial if more how this possibility the developed protocol can requires inactive that some XX% that in on quite you And could So then them context give see fractures to to because is we us used patients any go

Emil Kakkis

Yes.

better, fractures of reduction. reasons And with fracture kind. and which reduction too, better, an which studies XX% reduction micro is -- and people probably fracture they was successful. of there at than were less were of dealing a of is And that them to are make why one we're there like estimate it, X about using feeling them the that they that of were randomized So had feel something in the X failed aware patients of X did bisphosphonates. look Three successful,

XX% So was compelling, if at of were own of the on the the Phase data data, are patients. those not on reduction but you bisphosphonates, that II majority top look our which really

between what -- we substantial able to So a the pretty X. be should it's difference see clear

elected have them which placebo Now would anymore normally study, about on addition, some not be impact they getting bones be bisphosphonates the period the study, might the they're so year. you in And over the home. staying opens if in of the the of weaning, the that in talk and of the in most to the arm accidents know a them will But during by their up It's going goes of are patients having we to the alone, up. at one placebo-controlled and know every clinic clinic forth fractures month study. They come incident reasons back coming fractures.

us their opportunity fractures, to to between difference more the actually them. we'd So would which actually detect give the clinical increase expect activity

So data want but because if the placebo they they get and Phase so even on because cross strong, decided over opportunity. got else, before doctors, the in drug -- anyone the that realized patient II to would in were the they the they they want

difference why we were is felt people to real that's not to to suddenly it. So and big well go like get going be as want be part to this enrollment crank to able of a up too

Operator

with next Gon question Dae Our comes from Ha Stifel.

Dae Gon Ha

manufacturing to about questions I the setrusumab. wanted to, have line Emil, take wanted I a for of different today. angle to that call the ask setrusumab dominated

actually to at. that or success of it Crysvita, us to the plant over when track as maybe it activity it more or was, if it I can a have what remind transitioned XXXX, Just comes approved? And a regulatory royalty-based guess, scale comes burosumab similarly, you drug is when getting precedent Does some in as U.S. of commercialization record

hiring force be So spree a ready #X? done? need massive get you are standpoint, the need that hit sales interim just Would wondering there how to a to would from to if you were

Emil Kakkis

Okay.

the batch already for are Mepsevii, contract X,XXX-liter for licensed a so use and it's it's they that And manufacturing So tank traditional approved. is method. setrusumab, third currently operating scale. we already at manufacturer a a on Germany type in It

that. on very we're So comfortable

some addition the start another manufacturer and market, and in working we have at get size of that to Given expect the the would product we on our our had obviously view since to to of point, that study. knew we'd we

our as But to They So product second to well while we moved actually roles. and are become salespeople we manufacturer part as other have situation team experience. within have. places had are have we think get been support we currently have partner, April people a have in still going. since the in in with the the other filled have We set up regard in we KKC. U.S., still to it that crossover we're work launch manufacturer -- org, the and and need patient approved. With we does helping And the the them. can, the the field it royalty-based, XX but XXXX, and some confident services well around, has if are the within people They we'll knowledge, of our

would no need -- established to significant hire there's launch its value the the doubt, we we're additional be I real it's think people of So lot that evidence value but program think be and of will that will And that launching, point, that, base product if to the people, that potential, I investment of going given to product. a sense. make high of the at but

us and have If launch who Erik, expectations that effective don't think meet we have if else, anything we you enough a plan I allow established and will needs. know that about launch. want really on to will our our and design efficient really be base, I

Erik Harris

leadership that place we covered had on the I about I right led think of successfully it still The just commercialization everything. and add the in you have would that Crysvita only is trajectory. it, team our thing

So we'll be ready.

Emil Kakkis

but great that, up, got of be of shape -- regional services take managers, a in the head, Our head team involved the got we've our patient we'll good to team North off. hub, a the we've lot American set

Operator

And ladies and I'll gentlemen, we now to remarks. have reached session. end Higa back floor the the closing the for Joshua question-and-answer of hand

Joshua Higa

you. Thank

for able be Sorry, responses. than to to weren't us. This questions. more follow-up happy today's we free you the all Thank facilitate call. help reach get joining Feel to out. concludes I'd to

Operator

you. now may parties disconnect. Thank All

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