RARE - Earnings call transcript Q2 2021 | Ultragenyx Pharmaceutical Inc.

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Operator

Good day, and thank you for standing by. Welcome to the Second Quarter 2021 Financial Results and Corporate Update Conference Call. At this time, all participants are in a listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. [Operator Instructions] I would now like to hand the conference over to your first speaker today, Joshua Higa, Director of Investor Relations. you. Thank ahead. go Please

Joshua Higa

welcome call Ultragenyx XXXX. for and corporate results update conference the to afternoon, financial the and Good second quarter

have our release you press of Investor am issued financial Director We which results, Joshua detailing Higa, can at website our on Relations. ultragenyx.com. I find a

those on our which our section on reports Form be soon statements and our in filed and with many XX-K our subsequent call. was types will on flight of Chief our the as the views had Act Dier, I our would of Medical and me statements the Harris, this in Officer; today’s of like Private delay, projected any note Joining the available of will uncertainties, Kakkis, to Chief this the an date our Please control. all of forward-looking filed differ President; call forward-looking remind only forward-looking to and Commercial Reform the investors on that Litigation on XXXX, that SEC, identified the report from annual XX-Q filed quarterly today as call Officer on including include this Mardi that could Chief Erik Officer report statements be that XXXX, will Financial Camille unavoidable periodic the XX, Emil These Officer. call able Securities us Investors not provisions February involve Form website. but are forward-looking materially Safe and Executive not Bedrosian, represent is in risks Harbor meaning to, Chief within beyond limited statement. join substantial of including are results actual

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

Emil Kakkis

Josh, on a I and want to this the our Thanks, reflect we're everyone. approaching of of and period execution in quickly mid-year on progress second-half that programs year. our significant afternoon, good note clinical

are indications. approved to identify prolific have global company now rare diversified a the one disease pipelines and late-stage of four among and to products for terms with indications, deal clinical of rare testament both For our companies story, a newer transaction We effective one development of three do commercial forward disease modalities most therapy portfolios of XX those who the that a is we're in drive the years and to as rare company. disease. in gene great of to our our It science, ability work broadest

the two We gene DTXXXX are these and a Phase initiate now our Disease. X/X/X of seamless study this clinical GSDIa in X And trials of second-half to Phase four DTXXXX gene pivotal on UXXXX studies Wilson track in for over therapy target OTC, programs therapy and include year. Two, for respectively.

Finally, a in study pivotal imperfecta. for osteogenesis Phase X/X UXXXX

first GSDIII. X/X therapy for study our Phase begin UXXXX will and we Additionally, mRNA a

progress across to pediatric modified June, to team make May to during Canada previously clinical bolster patients data demands bodies in patient discussions back with the requested some GeneTx those followed syndrome first agreed naive Angelman of documentation U.S., to wrap a adjustments on They specific to at to in the in had green conduct, FDA protocol the based be progress the enrolling patients. logistical dose MHRA time adverse more the for get promising has non-clinical the closely. remaining data. to details. with the well both in UK’s clearance clinic announced the and Angelman again. in five approvals then before provide begin we on agency us the partners neurological the families. the agency the our Finally, by dosing verify approved retreating The patients. clinical our the to to now on XX,XXX in now patients details make I'll to as have on the summarize plan the of review and the with with as protocol the regions. from GeneTx FDA These study study GTX-XXX commercial over with regulatory GTX-XXX been treated resuming and and extremely discussion back Angelman partner the and our abundant hold need it occurred, simple protocol I'll We unmet We're asked both by lower clinical signs resume We'll our up to those further first the for on we assessments of in any has prior treating event high developed with working This and and get additional feedback will GTX-XXX and of dosing, us in to closely medical a order productive, In study moving forward the Those handing study. patients have before seen Health received dose based patients. U.S., administration continue and sites activity their program to safety serious the begin U.S. the

Angelman the two a all nine enthusiastic made three and improve first about May, up a U.S. a positive OTC the long-term Overall, plan to As showing on DTXXXX durable response either Phase nine Phase with we developed. expect Phase ASGCT known we are Similarly, franchise At deficiency from of steady conference that treatment. the GTX-XXX data all first be the some responders in the expect Shifting preliminary from to the GSDIa, we patients extending looking before, soon. based patients and patients the end to treatment who get Disease has six completed timing the their based or initiate We're dose, to with a The in pivotal this study studies positive X/X we startup total the and of getting and three going and GTX-XXX shorter cohorts to three the amount our forward following provide response with the data DTXXXX half know the announced longer-term today. through year. These data have years. treated, to for response UXXXX. by years of at therapy the from the gene Wilson update in treat requirements studies on everything maintain DTXXXX of patients in data to them we're treating we process we limited response patients. from study, mode or ex-U.S. of XXX% noted of pivotal three The the teams shared X enrolled. patients X/X for DTXXXX all across rate in we to cohorts

and for much programs preclinical platform latter extending neurodevelopmental the disease therapies, Muscular gene basis patient be manufacturing programs for populations. starting larger Dystrophy program next gene products, address for therapy proprietary quality our non-liver HeLa represent and of to Wilson's will generation Our and These two preclinical disorder. the CDKLX Ultragenyx a our Duchenne targeted and first commercial with our deficiency,

these in programs We will updates later year. provide this

antibody additional programs to mean initiate Beyond for year. or imperfecta track two One our this therapy OI. gene on osteogenesis clinical work, recently UXXXX Setrusumab, is we or licensed our

study bone in in bone This the bone Type decrease early enhancement substantial bones productive and in defamation life, to bone density antiresorptive of The that strength that manner that with fractures of spine potentially results and Based will with prevent the will anti-sclerostin Type already a review purely so stresses with that clinical patients improve OI. frequent generated showing mechanism for improve both or production by on density. OI, the adults as pediatric patients new science III on then we where of the example, partner's disappear initiate promising young in the will bone via needed, focus improvement adult making bisphosphonates. patient and bone comes repeated in fracture are Our OI non-clinical, IV fractures. have will mineral with our and right should clinical especially convinced actions, the and

Phase the end by are X/X the study the of with of initiation We a planning year. this population

UXXXX glycogen program for Type expected X/X license Therapeutics by study This our with is mRNA a Arcturus disease program year-end. storage first The also is III. for other program mRNA Phase to and begin enrolling

ID and patient penetration even are our In a turning with in testament had helps It’s both XXXX, America Now commercial to XLH. major patients initial another patient with over progress helped the entered Crysvita efforts That line from outreach reimbursement and trajectory its our approval we America, fourth with much in and In physician on significant a to supporting their America. particularly programs, XXXX. how with the solid patients The is as on expanded adult Latin efforts. started North QX population North from team's a by America, increased drive driven quarter Latin uptake growth driver, broader it is once in continued was we compliant revenue volume, with regimens. revenue, Crysvita strong globe. in in continues on have year our staying the increases XLH

of happy number across shift comment treatment. Crysvita this happen. making to than a at of notes paradigm multi-generational generations Crysvita, how lives the get changed. We have a be much have to on XLH families forefront also better their there's families nothing We're from and is now in

strong on followed year. market our launch has by has a the been for been Our launch successful which with Crysvita about now Dojolvi,

securing strong in Eric, progress needed start was Our will how the I'll team established management great momentum, shift a identifying commercial turn quarter. shows new LC-FAOD, and start call that coverage, forms. also new the represent facilitating tells over of now LC-FAOD. reimbursement much elaborate a option our to paradigm in for to, us The on the Dojolvi patients,

Erik Harris

afternoon, you, Emil, good Thank and everyone.

times. the Before dedication details the for to all what are uncertain for like get their and quarter, work of commercial performance teams I I'd of the during still into thank the

challenges new While second quarter have XXXX, ability overcome and the first most had of environment versus this shown within strong business. adapt Crysvita states another an over to the of conducting the quarter X% XXXX. quarter of and teams XX% reopened, growing have

Within patterns, the not the by but in normalized. the Crysvita this second grew is quarter-over-quarter, ordering underlying Latin XX% declined driven quarter of prior the North American reflective versus America revenue uneven as revenue reimbursement is and demand. an seasonal region, quarter, effects

of we we number in are Given $XXX of demand make community we XX% adults continue revenue endocrinology Crysvita. have with launch, on patients bone into previously the the $XXX has Ultragenyx the inroads clinics and so Today, to to in has to States, received on in as XXXX guidance Early million. portion year, the the metabolic are provided and finding the total treated first-half the of too United made and affirm seen patients. million continuing execution major the where strong we and we beyond of the for the territories have adult range, adults patients more centers, up more of therapy significantly, grown Crysvita. who In

We team has are the split at on a by driven clinics. done Crysvita reach harder in therapy, mainly XX/XX work to now community adults approximately and small, the between ped the

teams this last leverage growing across over year, to the to with Latin confident we that Crysvita are see TIO expand and find the made and continue As to upon for we could Crysvita. continue Shifting XLH demand investments benefit were from quickly more will who region. patients America, we

and programs, this in continue this meantime the who of Brazil, significantly the as reimbursed to reimbursement, therapy. had named on full more orders time. of In received XLH Health patients to regulatory and But for for granted we been in of base Latin American access countries recently pediatric on can and receive in In full we but take patient As are expand the through and year age process been negotiating patients from Patients patient final receive the to injunctions continues some therapy one therapy. have reimbursement Ministry patients in the is of adult to patients injunction streamline approval main support of branded older. treatment cells. Colombia, will approvals to receive gain to stages typical, the an

with in for now Turning by in Recall the which February to estimated treatment long of patients U.S. XXXX. FDA X,XXX the was an acids Dojolvi, June approved disorders X,XXX the Canada or XXXX, are in fatty oxidation Health there and to LC-FAOD LC-FAOD. chain by

to this XXX unique end approximately the patients approximately on second has This seeing to support prevalent we the of completed significant physician speak launch. approximately from of half and we Dojolvi, the one are have of from from at XXX written payers. to and than As forms start forms. represents strong reimburse into XXX population come therapy, received community start prescribers the with penetration prescription. have of quarter, more the continues having patient commercial interests point the than more These This led broad

June great to U.S. broad navigating patients forms As completed a with to providing payers with insurance of Crysvita so access are who LC-FAODs from there Dojolvi similar the patients reimburse are the start see time launch. a programs. point their in patients quickly is to of XXXX, Outside saw, end at the many continues successfully and The and number in we through therapy of the coverage LC-FAODs. outpace named patient for U.S. early policies. It received have what of

are the with discussions regulators who about from to process to all could agency. with In of hopeful we benefit Health, had work with and Brazil, positive We full this patients continuing review regional Dojolvi. are treat gain to Ministry the approval have

of teams were will and leveraging of the Looking that to developed the forward many field second-half tactics last commercial year. the continue the year,

have to financial Crysvita, to We call Joshua, Mepsevii. that, the will back Dojolvi share new find and this results. how With environment to learned and and adapt to, turn benefit could the from patients, continue who I'll to more more

Joshua Higa

press European Erik. quarter briefly earlier million. that the XX, $XX.X was related We revenue issued share million, American was release today North Crysvita $XX.X royalty Company $X.X for $X.X update, of million Crysvita will I $XX.X territories and revenue June of financial the to totaled million XXXX including other in million. a the included a the Thanks, sales Ultragenyx product territory in ending regions. net which summarize. Total sales from revenue in profit territory,

patterns reminder, a in the ordering in Ministry demand from received a will region. first that In large order from As Latin Health vary we the to America Brazil's quarter quarter-to-quarter. stocking patient of XXXX, support

will But Dojolvi uneven time, revenue We million. are demand. through by continue strong reimbursement process. the ordering over driven work will the the for this $XX.X patterns region was quarter from near-term we grow to underlying be in confident as revenue

As second we of these million. so describes far. first discussed quarter revenue be for Dojolvi providing Erik Mepsevii are the success launch. have metrics we not revenue in $X.X noted XXXX before, the will we the for guidance We believe better seeing quarters was

We expect revenues modestly time. may these over increase

partnership Second significantly, the rest year, also HeLa as with transfer includes tech Technologies. quarter transfer recognized Sankyo, part related around our XXXX strategic $XX.X revenue to Daiichi and as to PCL activities the million this revenue the our taper HEKXXX tech Through a of manufacturing will of come close.

in Additional SEC. and details filed the on this revenue recognition can annual be our found quarterly with reports

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

other number to we and activities get gene ready clinic. pre-clinical the advance most year As into UXXXX, expect program, X/X our to the pivotal study costs a as GSDIII, three UXXXX next programs support we in increase of reminder, for the advanced we clinical and OI, versus Phase this our studies, therapy the a study mRNA X/X XXXX, R&D Phase as

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

with for same ended For value as and six net million, a June share the compared a XXXX. decrease in of ‘XX. the the used in includes was the license Daiichi agreement, was commercial cash marketable million gain We we call the to Sankyo investments basic the The operations for XXXX commercial and securities. million, in securities, This per compared XXXX clinical equivalents net for diluted. puts XXXX of $XX $XXX.X $XX in in period loss million in ended to ended used $XXX.X collaboration to for Net net or the cash in fair per Camille. development Net the closing net XXXX, the strong quarter million $XXX turn June to quarter the the first-half to as same loss $XXX.X in $X.X position us agreements. that related key of to Mereo or milestones in approximately of million and XX, strategies. first-half from capital million, received QX quarter of share. $X.XX cash, million ‘XX executing months cash cash now our I'll $XX upfront in equity $XX.X continue share the million XXXX, $X.XX and and reach and period collaboration the second payment included operating income compares compared a includes of $X.XX license This per of XX, second over

Camille Bedrosian

spectrum on will update with GeneTx of Thank in disorder, is substantial afternoon. Last that a for efficacy surprisingly too five study reported of important wish syndrome. devastating data developed positive Angelman treatment good the multiple from our being patients. in we indicated of syndrome my early an X/X year, a improvements the everyone domains. you, domains Angelman providing focus Josh. a multiple the broad disease I with first manifestations in genetic GTX-XXX, And that on GTX-XXX affect remarks all treated Today, patients partner I neuro Phase

given have least may Trendelenburg patients help reach flush artificial safety we event. local observed of time, down it aged for milligrams complete as magna a as and ASO U.S., the the should Furthermore, us productive an GTX-XXX. where productive light the a cisterna associated regulators, Regulatory and Healthcare reported eight individual has begin exploration both to also reduce have discussions a In of with we to doses Canada, and a have extensive the plan allow and that Medicines first help data an reviewed we the enrollment with SAE. through brain. XX two clinical UK, or of patients tests at two circulation that The the Careful that positioning on MHRA discussed SAE body, including had SAE, Outside as a one after and and this and run CSF shared agencies, all the FDA. range to of resolved adverse the be we confirm agencies U.S., treating regulatory serious regulatory to is capped with dosing At to titration resume of new plan. begin administered a domains, United without data of we cohorts and doses milligrams non-clinical This the in lower the the and to great One the been been plan resolution plan milligrams contact have non-clinical Agency, in old, escalation start or extensive five had discussions at had Since but extremity the event and single doses two the Products study. activity, paused our dose for the has number agencies at dose. States. clinical have X.X of our for dosing administration clinical Dose X.X within then, two all below the with fully had the causing affected the patients. head with in in the first patients We tilted to cohort dosing is both evaluated Health patients distributed patients fully is nature to higher FDA, presentation years and a resolved. five or of at the and with dosing in agreed titration dose in under eight SAE, repeat substantially will diluted, well is our modified and other parallel green XX-years. with the basis, weakness

dose specific agreement submitted naive asked this strategy treat resume have we we on protocol a be on A neurological Following in to added on feedback received the we meeting, that before patients. we the amendment. few a and dose agency a assessments amendment, additional with agency to The dosing administration protocol a U.S. Based we believe the Type

with in looking to the generated team these U.S. dose Angelman the phase to now in have dose next this the make safely regimens, GeneTx this changes inquiries We We inform confirm and as Emil. patients study. will the move you. forward back update, The Thank and of with patients families With maintenance we to to can call children received we the and GTX-XXX will will the from resuming data development. enroll treatment of will numerous I loading syndrome and their look turn

Emil Kakkis

Thank you, Camille.

key for close upcoming a I'll Ultragenyx. Before milestones out, reminder we provide quick of the

dose summary quarter later For this in some of data by year. the and we will patient and the syndrome, GTX-XXX Angelman end Canada provide

to to We also resume request. expect adapted the the U.S. study in FDA’s

DTXXXX the are final have patient released three globe our in dosing around gene underway all and we DTXXXX, begin finding operational completed studies in and use and UXXXX, pivotal studies, sites for identified For been products the the tests before manufactured therapy efforts are patients. as and

UXXXX by pivotal the in with study imperfect, we and initiate adult For osteogenesis pediatric final on study that patient, reached year-end. green FDA a

UXXXX a and move study. GSDIII, our to mRNA we tend in X/X Phase with the first therapy For clinic

us can and you in coming as from over will As at as multiple as areas readouts the over let's instructions. all see, that, couple your in pivotal XX-months, This important provide disease least questions. fronts, next with study programs to years. Q&A Operator, With early on move the we're data various as please set executing four trials the clinical well for late-stage well therapeutic clinical and commercial. up on modes

Operator

Definitely.

Maury is with from Raycroft question first Your Jefferies.

Your line is open.

Maury Raycroft

questions. Hi, for my thanks taking

additional wondering much Angelman. are follow So what year? use you how up how to that FDA you many if could this report neuro the and for can later can on patients And and was assessments is asking to have I you elaborate book you

Emil Kakkis

logistical But agreed we're on you we've certain training really the things. detail. careful do are are just kind some They us when also they the with we'll the action of just of find of be you also more necessary. electrophysiology, is. triggers to And want exams. what deliberate is, and will like have want, when of these a it doing want part, neuro who our imaging what what we're person little They Well, Sure. documenting assessments just how the the of on sort what

put But it's to little logistical So detail. we taking big unfortunate it's fundamentally, it's think together, It's we'll confident finally out. But what get I think pieces the this. I we're there. call figured have we're do that management why I'd a time

the Canada. on ethics the gone they've get The already treated in how and it a sites of some patients, through many depends UK, have the committee. Now inquiring, of how bit many little patients in patients

it's a hard So for us bit last number, receive we to the of say, patients I patients We of end but to before, it's a more that we'll have little have patients. cohorts, of the we The end by at the few few said year. most the few some steps. the through probably would, by treated two exact a one have a data the in the And doses, expect first of logistical we’d on year. least should dose think

we'll be with put program. right but in extensive amount of do direction won't of limited. the have, moving will we we least we be it believe are data. the sense So it'll do whatever out It But us an give a

having if load is we'll three and the the end patients clinical on we will we for get low adverse effects before, these read begin Our that the events, without that year. so they four confidence that them, start doses before so give a get of the or to saw seeing

Maury Raycroft

Great.

Okay. Thanks questions. taking my for

Operator

The with Bank Tazeen of question next Ahmad from is America.

open. is line Your

Tazeen Ahmad

on questions. another Angelman, for Hi, taking good Emil, one if afternoon. so may. much I my Thanks

patients discussed? to previous the is for have Is in pending for up? approval patients. made that redosing you're the that comment prepared going And be what new start protocol you that for you agency then something your you has remarks, do separately have to not would that that I follow the a the would Or, add? So asked to

Emil Kakkis

local from, now of their we I seen they'd we us it's But patients. believe works. don't higher response. knowledge, have what data is haven't right patients we retreating really a to perfect and thing. a kind don't it's said so that the see type perfect knowledge effect, no cellular what What do if have perfect that to reaction response, we have rediscuss we mechanism. will there's is. the with just no don't the what and want could There's local Yeah, that suggests mechanism to And chemical have we because safe irritation we knowledge But antibody then and do and get contact of treated have view think and doses it's

that, into nothing we're dealing of and patients' timeline certainly clean to it's So lower dose same do. way. our That about alone the it's saw think to After a what it we'll history now can overall mainly about low look get dose. in with way before. don't naive But going have we the changes at we patients we know exposure, back retraining of the we this

I think just who get patients retreated. can were is get those we we and the to for that really tough data need. excited It so want

getting them for we as time more disappointing going work For them. overall to treated them wait to feel do it have as soon them, and to for is but won't can. the affect we We're program. But

Tazeen Ahmad

follow And that case, safe Okay. and that? for? it And asked is information relative a asking if the the to is say then Canada as the what FDA additional UK to that's for what's prompting up,

Emil Kakkis

of safety monitoring same. checklist is Canada form, It's say, a actually there a doing detailed do there and let's the we around actual the and we're evaluations and want regarding much using et the sort question and operational we is It's same. we're of the have more some how other cetera. that them, provide They UK here. doing method things in Well, same in structure verification rigor of details training,

the It's us same making assessments. like things, really safety really. do they're extra So not it's

who neurologists the question makes of great we do very think we're is But by better of much not. that careful really can be we having care they're I you course, will. do, I patients conservative. So that just want I to what but then carefully. think about are it. or insights how And And think going great they And, documentation being assurance you can careful take to be

great If job. good you people, have they'll do a

So that's the difference.

Tazeen Ahmad

Okay. Thank you.

Operator

Your next Wang Gena with question from is Barclays.

open. line is Your

Gena Wang

question Thank have you. Angelman regarding clinical I endpoints. one

include Violin would another feedback. and some feedback recommended? such regarding on as much DMD. rely or parents' you Baileys has doctor suggests then, doctors for measurements CGI that's what the question Some What And Phase quickly [ph], X/X limitations, too other

you are R&D this wondering, the year? Just filing on track second-half for

Emil Kakkis

Did say DMD? you

Gena Wang

Yeah. DMD.

Emil Kakkis

the Alright. with physically that. up the involve they're well, giving -- evaluations Alright, because GeneTx administer a Well, part. other patient not on in you, by then administered our we'll things story But what the the that CGI quite there's patient. on psychologists trial the the themselves, caregiver story, complete do or and not whatever measures And we also has by been people. the Bailey's so doing follow watching dependent they're does feedback telling ones were investigator the there's like have tests, patient

really three complimentary already right. and that you. different data All the reporting we've So have data supportive. shown all parties in we the That's

measures In have Delta Active that the [ph], things. showed Mayo like we EEG right, which or other the power, addition, measures, objective

that other saw so that they had a them Chicago just So sure doing the view. from things of for parents the what about why a videos the want make physician, way, depends is so their they're confirmation words, shooting the independent point, had I not a things by But to patient our because the would things. swayed too. we I would multiple patients family's we this number by at have there's videos think out observers, to they of he I on methods and we read appreciate also fact excited different of data are speaking kids, by sees make say and also independent. that and their CGI know were be done, these I said actually sure PI

patient evidence what report, So clear, she they things. like evidence rather than doing, be video also to saw video were just objective of of just

and seeing things are confident real, we're are that meaningful. we're So, the

later Now at on we street to about put by not technology our scale this an year our about using the to out is Dystrophy, information be is able focused would we're the our that program. the well, it's potentially of never we're using this Duchenne X.X said on lay filing in update on production program. system. We've point Muscular we in expectation let's and large going said out non-clinical not HeLa It's said year time, At about to a would talk but what large to you we're data commercial Duchenne is on system, end we that this out the that year. system where our IND going very update we scale manufacturing on the on But a creating We're year. methodology put HeLa that. IND and timeline

Gena Wang

Thank Okay. you.

Operator

next question Cowen. Yaron from with Werber The is

open. is line Your

Unidentified Analyst

This on the questions. the Brendan taking is for Thanks Congrats Yaron. progress. for on

close can on doses. give Just I us. us for of first guess I one was the things color line, know Angelman. the Canada if to you to UK kind a bit see from timing pretty more little said the I like the to same keep is hoping on just maybe an a you study. quick

Just tell the can and you structure Canada, about get the kind Thanks. wanted to left you in patients some to for just wanting and any UK you're if administration see to that. what's same scheme those in urine see FDA, getting in if dosing we And if also drug of that set in design different the on with might a maybe you or see of it's thinking data. mentioned us if the if UK, using you're it then wanted

Emil Kakkis

Okay.

be it mean, within UK is be or it in of treat some of would weeks could UK UK track, a much deal We of on patients different. terms the patient have each getting other, Canada there's treated, from the expect enrolled. community to I So great enthusiasm won't comparisons UK so that

enroll. able six they're to expect treated. be then X.X patients of All that So, additional and till dose, right, six them treated. two and study a once couple and remember then the treated, below will get the patients site's have open, And first will we to

patients. we'd UK expect some So that's,

dosing, U.S. specific fact, Now doing protocol protocol, and to what have you're not because have But Canada the will about will has also be monthly U.S. will asking the UK in U.S., made simpler the in the requests. the dose. FDA is methodology different identical

U.S. We will is monthly we're But bit bit little different. have different understanding a little strategy be with it in them dosing administration so method the on the that about use but far a FDA’s going similarly, It based is the dose input. to

Unidentified Analyst

Thank you. great. Okay,

Operator

Your next with Truist Joon is Securities. from question Lee

Your open. line is

Joon Lee

modified some the on CureVac. views interested unmodified given to signals questions GSDIII. if the non mRNA on our have set question UXXXX Curious BioNTech and updates. mRNA. given headwinds by taking versus of hear I for the and potential the modified We're use Arcturus thanks precedent a Hi, your UXXXX the uses Moderna, for

to that immunity. as And have innate to trigger a RNA contributed they also might point urethane think some KOLs CureVac and seem weak data for potential stranded to out double

are And on what you any to to concerns Thank data so kind from you. on collect there? of hoping biomarkers this assuage are that? of And sort curious your initial what views that

Emil Kakkis

Yeah.

for of very they We So, is the immunity we the response. do we innate test them. to we they're monocytes having not design. that peripheral very carefully patients to double mRNA And significant stimulation actually determine the we issue induce don't blood things purify to amounts actually think cells And of several important. assure mRNAs immunity how of mRNA to innate in design our white actually use prepare stranded

mRNA. nucleotides. on modified We're used using have We

have haven't the all think we But that. which But stimulating details cells, not on translated product. from handle in we enzyme white we is just gives that terms So know to make that substantial the of be a us of we mRNA efficiently. issue. And the human can is they're we much our sure disclosed mRNA as made particular know expression how is

with think, just whole issue frankly. the RNAs, So the the vaccines, to COVID I regard complex more than are

cleared body, themselves, weeks the that metabolizable -- couple a which and factors using a XX% of developed, et So can't is from The Those the other lipids. I cetera. aspects are occurs we're that other used, within unlike think the that LMP matter. lot there's being like of is one novel some lipids that a [indiscernible]

expression. It gives one. early appropriate releases very a and it's us this So better

LMP this quality, factor we of So optimizing prepare mRNA needed. we But think as the in the a in and works. lot nucleotides modified purify two how is work of use how we a do

Joon Lee

Thank you.

Operator

Salveen Richter next with question Sachs. Goldman from Your is

line open. Your is

Unidentified Analyst

Elizabeth thank types Angelman for solution the question discussed taking is A our on Hi, question. other expanding of of the genetic sort include previously, to program the severe Salveen. most good you've outside patients. based afternoon, on This for you and program, so know I

terms or future wondering and progress the a consideration? of of this if trials it's in would work Just how more

Emil Kakkis

the deletion genetic other in the mechanistic the think if type, deserve to it the types be treated think definitely it other works understanding. will ASO on and work We based types in the we

the Our deletions plan conducting. right now is patients to for focus XX the program we're of on

in is difficulty on at they patients et have to the the types. in most improvement language is because which deletion a of of mixture main we a However, patients words, pivotal probably communication we and that more are varying reason one would the are put second main you any Therefore, would study to severe. communication, The a other who study. genetic study type create degrees of cetera. focus would place look pivotal randomized skills, in if It interested have just things be in see clear

is, we'll main So something ways study of going not program the doing you those other some put types, on creating types place the but them with trouble are But body to as and that's is of can other focus big for the to well. forget in we the in great. create heterogeneity one deal

do wouldn't So that. we

Unidentified Analyst

you. Thank

Operator

Joseph from with SVB is question next Leerink. Schwartz Your

open. is line Your

Joseph Schwartz

a Crysvita. noticed Thanks questions on very much. We couple

self-administer in we the to point we expect if do similar any Europe And the see in the First, future? impact patients that we to what you change this wondering, a label take foreseeable in you do any? could therapy. updated was And place to allow have at were U.S. noticed think

Emil Kakkis

actually the Thank to of allow we self-ministration U.S. which the temporary FDA authorization is you, in going But pandemic. the from on right already, now, have Joe. because

drugs point submitted something special problems. the study. haven't about for done factor the or do The administration We we to change drug is the have very might at what you similar subcutaneous this is that human injections very any other

certainly has U.S. deal systems, different product B the it complexity, whether have home because be medical not added. hospital It could the or product. product, we though part a it a becomes to in or So with product, a

that in patients in run, are there's doing time, this we at direction. self-administration. would And complexities I So the in long the think in certainly point some U.S. move our but

supported drug, had getting that inject think the one compliance front we and We I issue. nurse in include you. home to your home comes of a to haven't know class things big up And just important that a right is do of the

at the patients their plus So getting percent are home already. of XX stuff

So self-ministration at up have don't house. someone of show is the benefit your you

want as to lifelong a this is an you're sure since make it's On the that your injection to as plus get side, there's a possible, because on lot going time we more confidence But convenient it's treatment. appointment.

home the look temporary but of the patients we'll in for are So working some self-administration long well it, now the getting the are so far, at on are run, it nurses and doing authorization.

Joseph Schwartz

to sales helpful Thanks. quantify appreciable color. but the adoption impact on holding an use expect visa Crysvita has back potential gain need you And see label for approval? to the That's from patients system Brazil Okay. to end then degree? the Brazilian there Can access to Crysvita to you'd

Emil Kakkis

seeing, approval Brazil. reimbursement. organization we're regular get it more determines the about talking actually we Brazil the of for would Health pattern getting ordering over Ministry but lumpy the we government as injunctions and orders reimbursement is it's that getting and that within With formal Well, time. getting we approval, growing we're What are have of it today that are orders is there help

final And that that is that's near that help once So expect would further. boost we'd completed, stages. the Brazil

the as your have with question? logistical to that the wouldn't much doing what we approach. So challenges Is of of you're injunction answer

Joseph Schwartz

there just Yeah, it's from? system back I was current how much how sales? helpful. boost much the get quantify has been wondering, to you way could holding Or, is any a

Emil Kakkis

patient there's know on be? a color have any I know. drugs. would Erik, we do many on have that you lot I I of think don’t get what want sense to for so don't that

do process? holding the how in And sense? many So back rough it's sense I demand, for net any mean, are you definitely the navigated many have Erik, something. how actually patients

Erik Harris

job are injunctions I to we've have as stated, been receiving good a magnitude And have reimburse Yeah, those patients to seeking sought say patients reimbursement many received injunctions. injunctions. have therapy. and threefold would of and identifying are done have received patients two that the of about versus that many identified of and

next just did and thing out, Brazil. stage, complete sometime Year. one what supporting I a clinical piggyback receive to reimbursement. the step formal establishing in upside the And We're stated some guidelines, once we which on process, to first quarter, the want is there's New to that in we through that in approval do just in receive opinion So, positive which point we working is reimbursement the final expect that Emil

Joseph Schwartz

Very helpful. again. Thanks

Operator

Your with Morgan. next from JP Kasimov is question Cory

is line Your open.

Cory Kasimov

has that that On something Angelman, afternoon, retreatment? the until go this good just Hey, in seemed about the very strategy Because wondering the was like in question. the triggered recently. U.S. taking Thanks change this new for plan concern FDA guys. it guess direction? I for the before conveyed for forward

Emil Kakkis

did as pretty Type A determining that who concerned might different, of we our never that clear therefore, they And, patient have they'd have reaction more established would a reaction that be was it opposed meeting to sensitized, trouble make had them. patient just with again, the the sense. Well, were been had had had a safety the they one that and have

new going data an to mind. change on understanding There's that that. back in us their So felt they hold of A want to terms helped on, safer they what's start with and patient, its no

prefer new We right. severe cause think data to reaction, a would think, more can't we them things on mechanism that we have have, that we exactly that everything way, the something have we have they might prove based no but regarding don't that

beginning. agency so with safe it to expose the they wants in that who without regard And to proof, play the

have that's just So, There came story. that the they us disclosed A isn't new that out, meeting. new to It's Type change. that at would the example, anything made finally for something

Cory Kasimov

said going the up? be And higher doses you you're doses and be and expecting going you're sense. dosing the different than UK. Canada the to in U.S., way here, you just given treated lower try to up that it's to wanted Makes some Okay. clear use it start Or, it I would to patients? to with your Is work then

Emil Kakkis

treatment we're to No, get FDA over the that little four load patients still think doses to think sufficiently bit be effect. start lower. But going to that will a We it's I wants more a the cautious. range a in

you what we so, went to do to with along have And get going.

Cory Kasimov

No, absolutely.

it. Thanks lot. Appreciate Okay. a

Operator

with Your is Nochomovitz question from Yigal Citi. next

Your line is open.

Unidentified Analyst

our taking Wilson couple this Yigal. a Carly Hi, disease. had on is We a Thanks for questions. on for

for on as be an focus could As far benefit if Phase if escalation from in is your you're earlier disease? part able patients? on newly expectation will magnitude target terms then Just later what for patients profile initial this of of of capture be advantage study? X/X/X the with curious see versus when focused the is you. UXXXX to their course patient disease? point diagnosed dose at And recruiting Or this patients there treating of for some program the established you you could the secondly, trial, the think we in potentially the data Thank with the

Emil Kakkis

Yes, thank you.

we result. result. a not the Does in and the urine, are then in able it long drug, bringing to if would matter that flow urinary would will difference copper don't many other cleaner would cleaner though, we the not naïve and routed copper so gone if the believe that the doing plasma greatly the to body. So treating is right. going though The it And patients, very oozing is the had copper changing longer on using culated. liver determine will removed what there's they has into give we us patients all that the the of thesis So, to would would or long they've disagree I sense? it so established with even run, few it in think up, time. And the out established and patients, truth key patients key the will bile be It's can pathway it the look I is long be is into changed already we're rule But make you happening. it take bile patients before bring can that a be who've who've allow copper by been the they're no copper being or into at do since therapy, doing mood and see really patients of a we urine, do the naive ending won't early on way. more up show a the In letters if take time letter. a problem established patients be the

Wilson, change that properly taking not of just in everyone problems their you're But I can in think also the happening, therapy the people's that course, both. gene well. view patients it's what's copper. distributed That's excess copper symptomatic might seeing fix believes think we as part and possible the it's earlier it's being

we the focus can study conduct to that's the ability So, and the way on them. based measure our

by set when patients data, that convenient I'm we Now be XXX% help certain. able come up relatively of in the XXXX. we little data but out a the but I it expect and will dosing regard easier. data recruiting sites will will running, so products make say Delta have we're enrolled get think to with to when But specifically It put to getting bit ready, out, can't end are be depends execution, the a up. on virus And easy on quickly. patients, will sites we be not think, I that could very I protocol

Unidentified Analyst

Thank That's much. helpful. great. Okay, so you very

Operator

is Your Wedbush. question Laura next with from Chico

Your open. line is

Laura Chico

for taking question. the very Thanks much

you another how I'm the then me Biogen. respect past the step in take program we in there's the a them level. and past, the many back, in patients on Roche UK wondering With spoken the It differences up through getting I are you've commented you think you've the data and Angelman, do Are about key versus patients just there clear for Angelman might experience? in prior time elements if understand as kind potential differ trying what how the three to then to we're Thanks. range, about be still aspects the to clinical focusing just And of state at more this in around think logical on on the that or and ASL which If how also starting just think between potency be Just a could this one kind points four GTX-XXX response dosing is actually program, needing steady to like and of doses. all going underway if a of the to follow two going I'm to evolving, I differentiation Canada, the here? quick targeted titration guess differ? we up. I guess in especially get might clinical these agents getting be setting strategies? isn't trials from should

Emil Kakkis

Okay.

know the the the end. for It's mechanism is on this So the special, region at prime different. is algo competition, five we patented

down I the that think production of Dr. Dindo [ph] potent antisense in is the induction. of of has message, from shown far level message the parts more than in and antisense of terms algo other knocking

to each levels you to to have across of mRNA I I brain. I have ASL brain RNA shown small whole could regions. and a to enough down, far significant that the antisense down drug, varying penetration. induction can the get want the message. we all of And high potency that goes that of the And do mean, the want with and bring relatively amount potency effect across we've think think But you

toxicities, we're lot will of the effects if effects. sometimes you've that other greatly that reduce think So, will it's are will to have added do systemic down? them well then ASO challenges. saw becoming neurological knock the algos a there I dose very single and the well range, efficacy having ASLs low potency distributing achieve that possibility double are how And therapeutic of I an the digit how you potential seen The able digit in be able are difficulty all successfully to a all believe of the question we side and drugs window. because programs, have of common, see, lot they or

our that's certainly, expectation. So

then patients, titration the each first right, on two going dosing the we're treating story, tight set in four. four and in and group, and So we're of add to

the the we'll patients at. XX do be see try get and information the dose are XX, total. seem we've be that we're where and said use they get efficacy it a efficacy, we'll say the read starting and to the it'll get patients dose a that it take based XX it's we'll the on sense Those titration during So of we once before, dose would on idea little makes information, the XX bit to on regimen, you? little have patients into XX Does they forward. standardized next have dose dosing and We'll make achieved expanded optimal sense? to then those we're look more it getting an Once how titrate going help XX learn that And going and getting of use starting from that or is a that a cohort individuals and That patients. in But as that we good and translate to way try now would at if XX group. right? to standardize to their a safety dose So what dose.

Laura Chico

Got it.

Okay. Thank you, Emil.

Operator

Dae Ha is question Your next Gon Stifel. from with

open. is Your line

Dae Gon Ha

in Good congrats on question and for thanks Great. progress. one the then Angelman squeezing afternoon, XXX, on Maybe Emil. quick all one and and me on

for my measures clin benefit, glucose control the wondering starts Will using during in variability as have you a of on CGMs, the have going there questions of included? confident in validations the to XXX, the any you. that of well Angelman on variability the protocol you I one that terms just might when occur in Thank just on that of was make the centralized CGM UK, And trials the curious, vote a reading but other benefits if entails to study? backups sure for that side your a given the one malfunctioning endpoints case have lot answered as any side you significant then some one CGI CGI back curious is issue. which So in Canada, been is are

Emil Kakkis

Sure.

the not So they're there's do an centralized, the endpoints. of terms and doing will could CGI the sites centrally be there, on because video. various into turned a patient a everything The trained to it's of really physician be in no opinion valuing way

We'll be do and For able specific do they CGI, tests. best the do individual to our and domains, our try have CGIs train that ones it. one for answer, team global train team individual these there's there's to which the to a to sides then we'll to questions for

for We that also tests are as measures. by CGI, example, number being some other scoring basic have a administered that have well mechanisms, not trained psychologists, are and right, objective professionally as of

So CGI the we to calls. all won't make be relying on

rest to other as at underpinning, give what will the of an should be wishful just PI you CGI hoping something the to really want CGI without we there data the look things. think having the underpinning there's feeling If better, I gestalt before, of if If a happening. presented right. suspicious, they're data show better. you be in endpoints noted, But we else would when right. any they're thinking someone's this But

we So objectivity manage of we supporting confident, by the feel question using data. can the

time controls, and Now, will monitored, actually them do the have we're calculations for continuously while we going how to CGM regard they're of with take XXX to doing. windows sample

when it. of see probe sampled if, for and the effectively they could example, would they So probe, time had calibrate a the take period and defect, we fell the off stopped and we working,

correctly period. during will it correctly, period of periods take calibrate That for months makes is calibrated to on windows sampling and continuously we'll expected to a closely. the But be months, it assessment that as in the yes, going where more stuff of you and sample use operating they're And verified happen have Because to data going are able and be periods. is to them we'll that intervals watch for to carefully So put we're measure time, all we'll a more sense. use going receiving. if that

too. the of they also talking and devices out locally, us been the disaster should against that about you're in collect So digital too. I'd the we've thinking that, help kind point And then about. cloud collect protect

and doesn't If the that shift device So But do we a it's patient a anything, could or probe from having actually stop double malfunction actually collected. it. something.

from have of trouble. can you the a far. to feel get digitally, connect getting include Very to to the a are patients, without good. pretty the data So work of all good source of bigger probably But having levels lot lot questions. physical parts had just of problem but experience not comfortable. which We're we good the the so We so on we've this patient,

Next question.

Operator

Okay.

Your is Bayko next Evercore with question Liisa from ISI.

Your line is open.

Liisa Bayko

taking for question. Thanks my Hi.

though the to starting U.S., you're to up so back Just still we're Angelman just program. to level? XX clarify, the in the Even lower, can you milligram escalate

Emil Kakkis

and whatever have the lower we dose, titrate then enough safety lower doses establish and how then drug if do, to of we'll we to I taking control apply of an elsewhere, said, start baby But, them the FDA than site there, will happens treat safety a see up get not forward more comfort. U.S. we come get with effect. plan. We'll doing U.S. UK data at us we'll some several as together, a all which, will then, in one the get And kind But up. we're give and have them and should kind we that steps sides data, U.S., still the on tested, show with future, we’ll the

it for patients, those have still to our very know are think get going. ability that agency way I doing anything. for is to to don't enough conservative, working it's drug it's I but typical that it's will know on affect fairly and the we them. delay our just to And safe them. to But we will being something board think It going titrate is

Liisa Bayko

Okay, see. I

that's Okay. helpful. you, Thank

because determined, And kind other. dose it basically XX so for is you’d of is or max up the there's there -- set no not for -- the

Emil Kakkis

other agreed XX at authorities low to U.S., that. But going multiple the in is agreed to max. the in FDA And not with we're we we're For starting dose, we XX a ex-U.S., the that titrating times. is based a on we're because the set max and haven't repeat

some get data. So we'll start and getting started

Liisa Bayko

a in you the GSDIa are I'm on just last I and know sufficient file? just file primary thinking back is XX OTC. safety therapy gene Okay. can endpoints And then, weeks. that that? of your XX greater and really and to to thing your programs you is Do a reach BioMarin on need or you efficacy? to endpoint Thanks. kind longer-term Or when file that database

Emil Kakkis

Yes, question. it's good

will some have key both the the give durability. cohorts of that exposed, thing sense that that been think will have I XX programs in earlier is us patients from we here

so far, Now, looks durable. it

challenge decline I the had the FDA is understand think direction. BioMarin with they and trying activity the some to was

much than Hemophilia hemophilia. are patients disease Our rather XXX,XXX it. in has smaller

to XX,XXX eight have diseases Our patients one-tenth.

to compared population size of the more. our So is substantially actually trial size the

taking a sample. we're bigger So

In we we it's to ages very safety have would patients X/X, of now package. too. patient other randomized situations might XX will on the that to different or remember, studies But example add not likely believe And regulatory get have the treated, with the for size the us. other additional these right addition we treated add XX trial, that we Phase some are patient the had authority we the debate of And the frankly, numbers fine. XX any

it's all XX you XX set pretty a patients unusual it's disease That's products. So crossovers, treated. rare not normal have or one. treated, an will safety patients

therapy. gene For

it's good. think I

Liisa Bayko

Emil. Thanks, Okay.

Operator

Thanks, Lisa.

Operator

Your question Stanley. next from is Jeff with Hung Morgan

Jeff Hung

Thanks for taking the already asked. mine question. Most were of

recent to particularly Any that increase then, variant indicated back since in identification that update given had COVID? because on recently levels. come you So and additional pre-pandemic patient data level the Delta Thanks.

Emil Kakkis

pretty seems going still it well. be Yeah, to

I people to have just think the adapted it's new world.

And still are responding virtual lot done. a things so getting of and we're and work doing people

if sure something know to seen And anything Delta. don't and I'm from delta about this at say Erik we've or not that Camille I ID patient have point. So

Erik Harris

would is consistent And last about adult the identification any variant the that add thus the Delta early. seen more Only but good than of thing pediatric very been other thing add patients, that pretty the we're have that it's finding I quarters. to has couple patient is and from not impact far, patients

Camille Bedrosian

in well Right. bit, teams patients of would Emil’s virtually This as to point, disease working as the agree more And that Camille, multigenerational way about the and a as aspects understanding quite finding been have the with that. is I well.

Jeff Hung

Thank you, guys.

Erik Harris

you, right, Thank next Jeff. All question.

Operator

with The Baird. Joel next question is Beatty from

open. is line Your

Joel Beatty

Hi, taking the question. thanks for

I reason ask studies, For preclinical Angelman, lower dose in may that And, difficult reduce make extremity to part avoid the weakness? will that the it help of seen for. or you're wasn't changes administration confidence that making test it in more you since what is the gives

Emil Kakkis

based pretty the but non-clinical say, know on actually there's think that, therapeutic Certainly, is I I two window studies, would goes we Joe, things low.

in see in range, we we what digit efficacy based on the animal. the that single we're would know seeing So,

the course, seem potency, early What problem stay there's so we far have So that there's a the all. it at they lower extremities that could didn't patients, fact, low lower confidence improving us a the the got effect, event. doses still and to gives And prior would expect. patients right, their the therapeutic we in only few get the to know were in we is among

that they not their talking on not actually uphill, down only grass, effect, they're function fall was and able not were people a an that weren't about, they they're kids down, falling having to So walking actually down. walk adverse were lot. things lower These one the of extremity first falling

stop for So right, of because when parents notice, they kind falling down it's them. normal

think we're dose those we improvement the that seeing effects. an So early even before

at why a happening event we're lower lower better they having doses. getting not you're I at safety effects confident the pretty So that's because be wouldn't that lower think extremity if doses,

I two sitting a those the drug concentration taking delivery of features and and effect. causing combination menstruation. chemical get to in us the will believe down optimize at that think flush the of believe sure we it's lower locally, high Trendelenburg we're approach ex-U.S. So will appropriate not a make the And doses put position safety

Joel Beatty

Thank Got it. you.

Operator

Lee Truist question last with Joon And is your from Securities.

Your line is open.

Joon Lee

the follow-up question. Thanks for taking

of Because any about? no sources curious that tertiary reaction, on may [ph] antibodies, you what that said ruled concerned an or sewage like is So self-complimentary it's of out the sort the But worried innate unlikely of may trigger a be just which be to like what that formation immune clarify you. just FDA to have is thank Emil, their saw So, or structures FDA GTX-XXX cells you adaptive dosing. you XP that implying immune of responses?

Emil Kakkis

everything is Yeah. inflammation, seeing we systemic. Thank like, you. value, only the From there's we're thing localized nothing

been talking gets animal land in is a systemic to it and So in it's about, any just drug have absorbed says, structure up going can everywhere. we localized necessarily you, significant wouldn't become kidney should at concentrations from circulation because the as the and you're prove the -- It the phenomenon. thing absorbed,

we’ve story. I So of otherwise. lot a effect such work really this don't and localized why you're done about you there's think a on anything immunity really having So, that And fundamental problem? it if would highly have innate fits that

Joon Lee

itself, the local. Actually, don't agree it's brain see just with I you you, in because any effect

goes. end like So it's wherever see it of [indiscernible] the not you

Emil Kakkis

where Right, applied. you first it is only the see place

know be cord not, happening shouldn't where is. which If by that localized way, up So did is It work just it it's it highly concentration even the nucleotide below targeting drug it dependent. the drug does L-X were, the T-XX It some went why you it's if detailed they a the to thing, very a but there. it was wherever showing doesn't. area, not we on to

a like to thing. what keep such very The we to get see managed. other a part you this is below if We specificity phenomenon, a look just it. we'll threshold. the me. of That's the [indiscernible] the looks stay And part it's localized need wouldn't think. potent. So I think, the I to dependent, That's you need at cause concentration just weird And lot so data, why, nonspecific toxicity

Joon Lee

Thanks Thank for follow-up. the you.

Emil Kakkis

Great.

Operator

remarks. this to for additional the concludes conference turn for will Higa closing back session the that And Q&A or call. call now I Joshua

Joshua Higa

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

Operator

today's concludes this gentlemen, and conference Ladies call.

well. and Stay day. now safe Have disconnect. You may good a

Ultragenyx Pharmaceutical (RARE) 3 Aug 212021 Q2 Earnings call transcript