Ultragenyx Pharmaceutical (RARE) 28 Oct 202020 Q3 Earnings call transcript

Company Profile

Participants

Joshua Higa	Investor Relations
Emil Kakkis	Chief Executive Officer and President
Camille Bedrosian	Chief Medical Officer
Erik Harris	Chief Commercial Officer
Mardi Dier	Chief Financial Officer
Shalini Sharp	Executive Vice President, Finance
Brendan Smith	Cowen
Chris Raymond	Piper Sandler
Maury Raycroft	Jefferies
Gena Wang	Barclays
Tazeen Ahmad	Bank of America
Samantha Semenkow	Citi
Cory Kasimov	JPMorgan
Salveen Richter	Goldman Sachs
Ken Shields	Wedbush Securities
Jeff Hung	Morgan Stanley
Joon Lee	Truist Securities
Arlinda Lee	Canaccord
Vincent Chen	Bernstein

Call transcript

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Ladies and gentlemen, thank you for standing by and welcome to the Ultragenyx Third Quarter 2020 Financial Results and Corporate Update. 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 host, Joshua Higa. Sir, please go ahead.

Joshua Higa

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

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

not beyond in of the are from those our filed and the Report filed that we Form today, will our that XX-K in date that Camille materially weeks Commercial and Officer call this statement. on and President; of Officer; for a any actual call. of the Shalini the section. Dier, only periodic Chief call Safe forward-looking These including of I results Litigation forward-looking end the substantial Financial Private many as in and website joined involve Please Vice Investors Finance, on XXXX, include differ subsequent Officer this that which be Officer; XXXX, in the President couple Medical this to meaning Form me the identified of on including, of Securities XXXX control. but like statements represent and the is projected Emil note will was Kakkis, statements limited could Mardi XX-Q uncertainties, available as within Chief investors remind this with available Joining views Q&A risks Executive our our that Sharp, Executive also at Erik types February quarterly the report Reform who company Chief ago. the Annual XX, filed statements provisions our are would and Bedrosian, on of Harris, all Chief Harbor Act to reports SEC, for will this our on meeting our scripted on of portion call the forward-looking forward-looking of be

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

Emil Kakkis

large Crysvita of our those supporting clinical which need, June. internally with on development underpins development. the updates our the tumor-reduced all team our focus in therapy progress this on the call in will continues throughout growth programs, in and afternoon, XLH commercial The strongly has stable COVID. rapidly today. on the a therapy gotten the Starting week, year the Commercial global approvals and is of This has productive encouraging. gene is to for rest We now also partner, GeneTx, the business of philosophy proud team with across of we to while LC-FAOD top brought number partnership team the the additional relationships unmet bolstered need the a teams perform programs have of to of new in part start have and which facets quarter, and and and that am have delivered source been now with March. speaks of a strong how those our and first all cylinders annual living call. for to Regular in provide at new working and been It’s in matches and Sankyo trials with those program a by patients execution. thank updates series and joining Good successfully which centers. announced to start guidance that all of you, provide the osteomalacia naive the the on its for strong we technology off gene on still jointly major I first two year growing program for our for are is launched products Dojolvi. impact diverse the products but Daiichi and been Angelman treatment in In quarter busy Duchenne prescribers with our team and the us and quarter of people despite the today’s Ultragenyx. a Crysvita the improving a patients on We’ve all in is our indication. firms company positive development weeks X to gene This Crysvita launch, fundamental everyone, my for received business. of relentless company, the physicians on therapy firing and

Moving gene therapy to we’re new or Duchenne Solid of strategic a Biosciences, DMD. collaboration with week, for that last developing Dystrophy, announced our treatment Muscular the we

this While technology Duchenne to gene programs far two other there advanced, opportunity Solid’s that with our and was are disease. are companies technology for unique this more combine to create next-generation a therapy a likeminded

the to Solid’s process. bringing high-quality its blood therapy flow Duchenne cell be could with and global and we ability excellent to this in our has coming We forward new We HeLa capsid, quarters. continue is and believe X,XXX-liter HeLa update with treatment muscles by the progress enhance with microdystrophin to for to produced will working on collaborating differentiated will We produces a best-in-class of Solid potential profile novel an out that the a achieve collaboration combine we will which forward coming to accessibility. seek look immune you manufacturing to rapidly

GeneTx. difficult of with is antisense Moving ongoing is program the patients study data for GTX-XXX the clinical key science to by first supported and a in announced of by the our other is patients treated have Dindot the the provided domains the X.X. score reach GTX-XXX Biotherapeutics. being of using we of are from in of to In X/X Clinical some Scott multiple devices augmentative on to Global substantial include their Angelman interim and oligonucleotide effects positive to communication time. with clinic Phase the syndrome, X for investigational product from the Dr. beginning others with with Angelman by GeneTx for now supported gestures Angelman, The lives shown patients is nonverbal based change entire going first from mean the endpoints of tailored yesterday, treated collaboration part CGI, extraordinary use the while words, Angelman changes signs, remarkable which study or scale, to of GTX-XXX. a all Impression the update, reports X patients and in characteristics communicating These the positive of improvements first

after reports to for on follow Not capabilities to and through learning night. the by due few reportedly tasks, Others families these changes, treatment name time. weeks commands, the just changes Some or independent to such patients part a and and months the in as some swim a unprecedented include after respond patients, adopted ability were first In of focus began but fork their few are all often have own, themselves other a these to continue profound, feed using dose. only sleep on they is the in to study. desire a rapidly indicated lowest the

extremity completely an and related protein Turning is the SAE, SAE to at of now safety, lower of the resolved. with serious event, local to all – have a X CSF highest elevation adverse now had which observed inflammation the patients believe or we believe weakness, meaningful We the in the was dose also GTX-XXX study, the strategy. at the with region manageable We and doses changes GTX-XXX. in This the responses. where of in of lower of lower clinically saw administration intrathecal in extremity is not administration impact doses weakness

to we agreed an the with And dosing and amendment filed so and once we expect get to resume enrollment FDA.

are hearing are and We the on in excited safety the as about very on patients’ December first these families about program provide results efficacy for we additional data time and We are to yesterday. additional seeing data FAST the what in are plan XXXX. patients at Summit who these the

prevalence. treatment are terms great diseases, leverage best two both the Looking Duchenne disease, aligned greatest of need with developing and for patients. – a where Angelman, strategy for company’s significant modalities create in are platforms of and we to is at various these and and unmet options developments, need our rare our very recent there therapies They will both

Now all from and last been instrumental in been over me on joining I of Shalini. contribution many wish We thanking Sharp turn that the way at start throughout the days us her Sharp company first New us early been before the ups by her Zealand. that Shalini call, to has friend, hand and I is the one her the financings to over in you call where time, RARE work that, remain here Mardi, with all to all certainly would the her valued has has not greatly I contribution just colleague with conference the Shalini with call. RARE She will want to from know like going a phone the and building the on the it forward, with forever. superb but downs call it CFO. could best to a of legacy and I company and for the today, and be And without to

Shalini Sharp

good that your here. company, its to so finance mission to new Mardi to I to last thank to I team, everybody. team, And ability patients and and would these and you the had to the to could opportunity the experience I’d to thank take you day. on call. quarterly I like Emil, you the all, past to our above rest introduce team also team this be its the investors analysts Well, and years. have for working I this Ultragenyx serve this much, more not afternoon Ultragenyx our CFO, Dier. of of join delighted extraordinary very all effectively you every like for Emil, mission proud the our am conference over with all for IT execute am executive that, our pleased of support thank I

turn Mardi, You to now quarter. the financials will excellent the walk you her over hands with and I’ll for in through call be to the

Mardi Dier

very has know team, that with a you, will make very excited been a next thank I am stage and during smooth rare patients building Shalini critical to helping diseases. a in Well, its growth already this part I be joining of Shalini Ultragenyx fantastic one transition. of

So Shalini. you, thank

revenue territories XXXX; million totaling XXXX. months of revenue issued This Crysvita first which period over from to million. XXXX. months or Total summarize. Total of totaled in Ultragenyx increase today, release XXXX, over and million, included $XX.X Mepsevii growth press representing was ending update, that $XX.X a XX% and X compared a same So X we for the a XX% XX, from a million, combined in revenue revenue the will Dojolvi includes I September company the for financial $XX.X $XXX.X growth XXX% XXXX briefly

of and territory Ultragenyx Crysvita revenue the share the in included was For net regions of $XX.X $X.X $X.X in related European North sales million. million royalty other in third revenue was of million. XX, million. was the revenue product product Crysvita revenue in collaboration the territory quarter for Mepsevii ended This in America the sales territory to September quarter million. XXXX, Total $XX.X $X.X the profit XXXX

revenues In product sales. the We there revenue modestly we was may do named not initial these Dojolvi approval, U.S. quarter over to growth. This expect patient from FDA sales time, worldwide expect significant includes $X.X receiving increase U.S. be million. but after and

recognized of agreement is completed million Daiichi. this XXXX. performed be revenue line, from as based will license recognized of to towards on with to March straight the work completion, will XXXX. this and also executed and majority not technology related end agreement We on $XX.X was be that recognized The Daiichi of progress the the of expect majority in we work be by to Sankyo collaboration They revenue the transfer

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

product over into We we development to clinical continue advance expect and preclinical R&D pivotal our costs studies. early candidates additional from increasing time as

as over quarters LC-FAOD Dojolvi increase Crysvita and We our expansion of for support the the we expect commercial modestly to launch for of and existing also SG&A programs the coming TIO.

of per diluted. consistent. basic remain of $XX.X of We This share, reported XXXX, the or basic loss $X.XX third quarter Arcturus in the a million third million expense This expect from per to the $XX.X in third on a for unrealized versus $XXX R&D the million adjustment the non-cash net quarter related $X.X fair future net we XXXX value to of loss quarter XXXX. diluted, of of on and includes the SG&A sale the fairly The In and for expense net also loss $X.XX to equity. liability investment royalties. loss includes split compares interest the million the or share, of

same million quarter $XX.X $XXX.X operations X for XXXX in first cash of an now period the $XXX.X who the Crysvita. net like I third guidance our the For million on and will were update available-for-sale in XXXX. Erik months compared used cash, ended for the commercial million range with and XXXX, to call the in to our on investments. equivalents performance an provide to turn quarter cash would for update We

Erik Harris

meaningful tumor-induced you, Dojolvi and Thank In to osteomalacia. continued Crysvita The acid long-chain for occupation patients revenue Mardi. with launched the growth. Crysvita for deliver team third disorders also fatty quarter,

product, third a meaningful in which well. of Our therapy across the I by the June on patients launched FDA the all U.S. in the to molecularly MPS Mepsevii, to pediatric was the of U.S. begin and Dojolvi with update with July to to confirmed was X,XXX by providing X,XXX approved treatment patients for Dojolvi an and continues diagnosis forms diseases. and provide XX adult ultra-rare very LC-FAOD launch, on would X is going like a

of XXX XX reimbursement made and start to received and from are patients XX approved. the we converted prior All is The end team to XX% of trial been more start many months few As of reimbursement of XX% approximately forms The in the therapy. reimbursed approximately have are patients front the on who naive the government. commercial with approximately prescribers. the XX than to patients compassionate have progress first clinical therapy. use being mix led commercial significant quarter, for all successfully after Dojolvi and has navigating payer commercial approximately XX% on This forms therapy unique third has

policies formal are during exception most guidelines. basis new-to-market Dojolvi but in put approving coverage place, an payers on Some their

their broad to continue time patients, take pharmacy has various work that label. LC-FAOD ensure closely and FDA payers its a does with to We commercial process the Dojolvi to establish policies. as and benefit, accessible indicated is government all payers Dojolvi by as coverage administration

along American to Dojolvi Dojolvi launch. granted Dojolvi incremental priority U.S. to build France important And as continue from Outside Health with more discussions a we are Anvisa expect commitment countries than Brazil These therapy. of Over patients the way being been Dojolvi in and now infrastructure The with for programs. through fewer and patient named gradually ongoing. attraction. added time, our after been regulators meaningful been submitted submitted Canada we has to to reiterate States, has to Italy United programs leveraging review. in the approval, gains the gain Looking the EU have able with forward, will U.S. launch like are other we even established commercial hires I XX support the this to access access European approval in to for would with to patients in the and our broad access. Latin revenue

these supported will relevant During requests discussions for and access countries. regulatory named be review patient to in continue phases, all

XXXX. to sales We continue in sales make Dojolvi bulk expect to named of patient the up

The XX% existing therapy. leverage tumors. we has FGFXX-related between or the hypophosphatemia TIO, TIO having we X,XXX well, gone treatment second Moving next small there population infrastructure which be ensure important FDA physicians to by last XXX to tumor-induced Crysvita, able in launch relationships to in have as patients receive with to and a of estimate been approved indication U.S., the this unresectable successfully with the for the was and indication, able quarter patient our is osteomalacia, to this for

of broadly all been care patients to we more maintain have treatment. turning for Now, able on Crysvita about XLH, to for continuity just

in-person to to adapt recently. personal and by initiatives, more with meetings digital more the promotions continue situation to We shifting along COVID virtual limited resources

range being revenue of more we to of also patient quarter a $XXX increased. expect reimbursed and we on Latin territories. steadily time, patient second community for million start $XXX in increasing provide reimbursed America, by at meaningful and contribution established largest this evaluate of year, as impact to growing are the pre-COVID, beginning revised in range governments. Brazil, and bringing On to forms patients on the Recall Latin KOL team’s awareness continued our in and state Ultragenyx call, revenue million $XXX to Crysvita both the demand launch seeing to more and starting the the lot this patients. of range we Based million Over region progresses. Crysvita. of are America named growth to leading region’s to a of we a have as drive strong our end and are see number our we to a execute. $XXX of we market, that finding continue for the of of the maintained pandemic the guidance patients, and Argentina, to ability funded federal number injunctions treatment, granted million, a $XXX support million. the recent to Similarly, trends, are Colombia new In We raising success to the In lower

the is are impact allow range. still to to as the and turn this and uncertainty continue its an be to as we some be the treatment close update COVID place to put While will that, it within tactics long-term there revenue, confident believe I XLH call winter significant will a us clinical strategies rare in we TIO related enter peak, the the potential for the will over we have out gene has on and With blockbuster on Crysvita we the patients world. At who year therapy provide with Camille to Crysvita will around a programs. disease season

Camille Bedrosian

is progress our clinical program, the Starting for most OTC It clinically in the I and urea. program complications, that Thank metabolic an by good detoxify syndrome and hospitalizations coma. encouraging to of caused the of death. ongoing DTXXXX, and will programs. and you, Emil could on OTC deficiency, our treatment X/X from with Erik, responses ornithine sometimes DTXXXX. results update also review crises therapy other with Phase or our gene ammonia stage afternoon, provided Angelman everyone. neurological disorders OTC, common can transcarbamylase meaningful durable to And experience cycle with study in into inability Data patients urea demonstrate our result

improvements previous diets. patients medications alternative Importantly, their continued their discontinued these pathway, after and liberalized have

fourth cohort kilogram of ongoing X steroids. per the a DTXXXX, to genome using at For copies XxXX dose is prophylactic patients XXth the

XXXX. the We by of expect end data

start also and with planning X XXXX. study in to and discussions are plan We continuing FDA the the Phase X Phase

every long-term patients to study DTXXXX these Moving Results disease GSDIa lives glycogen patients reliance DTXXXX to that therapy, glucose far Phase death. levels at is and risk potentially, to steroids. the Safety these who on complications significant elevations cornstarch. showing avoid and requires disease hypoglycemia, improved these of reduction death. hours from are would cornstarch any metabolism type gene the that Prior been severe of a for storage to Ia. take acceptable reduction X cornstarch with by to life-threatening for date show so with managed transaminase put X is to X/X changing have has in patients less reactive

impact data, following cohorts is commercial to to steroids The copies per presents first the X preventing using the also fourth X of kilogram XxXX patients timing decision treatment addition than cohort cohort This genome not are a the in of regimen In this Phase X setting. the of reactive use monitoring prophylactic on rather the the XXth of value based the initiation same study. enrolling of will standardized and at for a steroid we longer-term dose level.

X with forward are planning DTXXXX. for study moving Phase We our

initiate we with finalization the to study first the in of of plan the XXXX. study meetings half design, Following the FDA and

call updates, Emil. I much. very turn you these to now the Thank With will back

Emil Kakkis

Thank you, Camille.

clinical strong that XXXX, the new business across unique have in on this of delivered in have a opportunities X commercial summary therapy launch. continues Erik’s are commercial simultaneously gene that a through with to XXXX and has products adding has we we pipeline a programs, On and position that, of top diseases, platform new growth Now phase very been our robust X results of also to be but in positive source partnerships.

are that, vital therapy, internally be program the platform the bring competitive which potent a that exciting moving severe disease. we that beyond larger supports for first to through a scalability that believe we populations. program, therapy that forward. large a in plan work patient able for molecule is issues includes larger gene And behind ahead. rare uniquely the but leverage will indications this manufacturing and have forward This disease we of Angelman gene we have that our other the one space to will is safety the newly the disease Wilson yesterday to Duchenne and up space, data advance a and we in large to announced this disease, which have HeLa Next we from And be is place product have

stage we across last disease large gone us, the have dedicated biotech and from a years, Over number continue a a balance revenue. our the diversified of of commercial strong clinical to rare all employees With we with business of will couple a facets growing our across opportunities of leader. ahead and executing globe sheet

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

Operator

Werber of [Operator first the of Yaron question Our from Instructions] line Cowen. comes

Your line is open.

Brendan Smith

is very this you the for Yaron. again the congrats on for questions on Brendan Thank taking progress. Hi, and much you

Just a on of I quick DMD. couple us, guess from first ones

patients? you And then really the quickly Thank just capsid versus prophylactic data there? from by the a I a matter of much. potentially you just ones here. little or the how year give process. it’s differentiated bit to very about therapy very As But we it’s you the a out if plan can get could cohorts. OTC the mentioned approach few sense on your steroid us kind data end, be obviously of mentioned, timing there of kind you from maybe the totally players maybe for other early you of know mechanistically, And wondering I already of in in just in are GSDIa your motion a understand gene really

Erik Harris

Solid. Duchenne differentiation. microdystrophin when aspects are the Well, look I of good. there several at from Very binding One is program, not the

excellent the X second which ability for many to and third very is system, we using the now has successfully these are is been a large-scale an with which High-quality And produce we’ve selling capsid in – achievable programs the AAV high-quality producer more skills do to combine that think I differentiated. both have and are And with approach. those a become to in companies not transaction have with U.S. forward We we something with X in opportunity bring special that accessible produce for profile. which space, can mammalian is a an areas, Duchenne approach program difficult and to think very we used, The AAV. immune the the the have patients features, globally, just bring

I that think, would how that’s, So I answer question.

On the groups. dosing prophylactic both are patients we steroid in timing,

We but us prophylactic this And it to program Both programs will data interfere before we have have the to the done Phase but the approach will year. does shown the some helpful X. Phase some of good. on and patients to into it’s program, plan them – cohort start neither built initiation steroid X just with have steroid how looks

some patients. data So those We’ll provide continues data. prophylactic on both Phase year planning this in X steroid for the

Brendan Smith

Alright, great. very much. you Thank

Operator

Thank you.

Sandler. Our next question comes of Piper Chris from Raymond

Your question please.

Chris Raymond

Also disease. DMD I HeLa program you transduced establish surrounding program, as it that the as you the have clinically? on Hi, therapy And to – gene by us of specifically, hypothesis do sort on this that’s a can caused in actual any normally be have to Emil, long maybe looked where on to way address a because the damage efficiently takes aren’t guess Or you DMD thanks. you’ve I’m producer would thoughts the able cell closely. they sure muscles of maybe at there then just a that? also how that there a execution line? Thanks. on remind Is question is

Emil Kakkis

Sure.

is without control Muscle familiar sensory diseases. create It’s or of really, creates to capillary muscle So when of so difficult lot is that by a exercise. have way, products. a on swollen don’t big, designed flow. fluid molecules deliver and you particulates to you challenges that difficult, It’s muscles very and They delivering. And delivery just designed Duchenne, with large in way, by injury, general. the different even we’re very tight

not, can We’re are another of the is high to to we’re clone that won’t months do large approaching stable platform looking issue, highly that’s, X part that stable in about high-producing clones that that be approaches of and have hyper-reproducibility, quality of that stable reproducible improve power we it clone on damaged number – we – being ways incorporation liters, would X,XXX high aware a a The of I manufacturing we a identify a that. a originally The And at and the at But within certainly of we the to maintain automation improve ways the us issue and commercial or vector. process growth allows can way of any I that X,XXX-liter it. in help time. think producer story. have to to combination cell help That screen and we’re established, – survive approaches in clone. we can regard of and say created those a that long-term time, gives at and processes and using the standpoint, the we’ve general. us in it With create point generally, the more company challenge aware of muscle the produce lines, the titers this from has but high-quality, the at AAV then challenge actually scale. The identify and automated how to a to

Chris Raymond

you. thank Okay,

Operator

Thank you.

question of comes of line next Jefferies. the Our Maury from Raycroft

Your open. line is

Maury Raycroft

questions. taking for my Hi, thanks

or naive just you update treatment? Dojolvi, of and the bolus if naive getting a you represent about a age wondering those talk can demographics, can more patients for XX% the on natural of For comment including patients their and more patients patients, if

Emil Kakkis

or course, patients, interest it’s work. XX, a they now we about forms watch on believe this are the to across because drug there in it seeing early of ages a but number if of well. bolus, Well, which how exact generated we of to the and Difficulty] talking yet But out haven’t steady the really But forward. described that we been moves have are I newborns, the have we getting the broad And from response can in on physicians of a are what already start think spectrum. the there’s exact and prescribers, we’re put quarter think don’t expect and detail And is from on as product, it’s there of think going spectrum [Technical just a I general, the launch, bolus I would we wait to continue and obviously, in patients. it’s clearly and say we’re identified

Maury Raycroft

Got it.

in And guess and is for natural CGI Okay. CGI on besides history good data data efficacy to you’re is the IAS versus it lines, And or contextualize to there along Angelman, time range? these general convert age seeing then points out Angelman the what existing scale? the data I possible more the the based reconcile there, on

Emil Kakkis

Well, certainly, data in of on, When I some gained over they ground flat. you some provide try and time. can history much they to look They’re Angelman age. think, relatively they’re years I around patients, until, flat, X generally or for X there natural really change don’t at pretty you. And help from

nonverbal. So by become all the verbal. People are deletion that’s are are never way, And nonverbal. deletions patients something They changes we’re seeing seen. the not

talking, at that are regard the make domain different and creating in looking so to the score domains names, at the to score the that and any #X what a and CGI, the has their CGI family-reported life forms because responding or you patient. need in in and the and just highly communication something other physician’s all words becoming affects score With then learning particular involves could And each that verbal communication of the they is easing them unusual. is view. But fact with of in look problems – is instructions of the areas itself CGI, valuable means not have making are patients summation from global up, are which of patients And using people other unique was and CGI. their – It’s in the for strong each anything basically each really and you, effect other kind in But very with not domain. patients a a CGI. half disease, It’s and the unique between particular is on might general, think seeing, possible it far data that’s CGI. score, is on natural for for improved improved different with placebo problems just main do change compelling. say that much would Angelman passed be have explorer by some any in what and changes much any Angelman magnitude X.X, that very there we’re see inside I on which including and history I to absolutely families of the is that with based regardless

and So that CGI on we’ll the support information out more put of at with December FAST the the data. potency, and we’re the meeting details comfortable data

Maury Raycroft

for questions. Got helpful. it. my That’s taking really Thanks

Operator

you. Thank

Wang next from of Our question comes Barclays. Gena

question please. Your

Gena Wang

questions. my Thank you taking for

to to years, wanted are I with has looking been past it forward your you. And and X, Mardi, working journey. to to Shalini, you for X with the working my great First, wishes we say best next

sets two have I So of questions.

First is one the Dojolvi.

I regarding a know majority ex-U.S. now And patients let’s and DMD is my to assume adult? the the patients XX% right say, file the gene are on access question also, that of to many year. therapy. fair could main teen So you drug right within now it program? R&D adult, mentioned how X you’ve is second and teen on is And XX% patient

the can additional potency such what need FDA to give mean, the the preparation R&D, on you I including you – effi? do order – CMC, to And inspection also, you preparation in you additional So color as for need do for all start?

Emil Kakkis

questions. No, say to want quite a I few okay. Shalini? you That’s know Gena. don’t anything, if thanks, Well,

Shalini Sharp

enjoyed I just and you, well. working you Okay. and will Gena, all to as your you with thank team really

Emil Kakkis

good. Very

patients that very a dosing is Dojolvi, to have X, once old, first about X, flat it’s you then curve. with years years of X usage, to but get the drug So amount life X, X about of understand X the in increasing or thing the

is age from amounts to getting patients we which drug, adults are school of to so adjustment year. similar and estimated very XXX,XXX around gross compliance after of And for a net

matter if we’re want might young is but what you don’t going much many So be we that we of have, probably know in anything have Erik, not Dojolvi? else, on this point to at out patients this time. patients very on putting – issue I how delineation

Erik Harris

that. add to else Nothing to

Emil Kakkis

Alright.

question other many how you asked patients on patient ex-U.S.? the think were was I Yes. named

Gena Wang

it. That’s

Emil Kakkis

respond the to Yes, it’s requests a of but I France. of on FAOD the we than countries handful able because in patients, U.S., now. little XX Italy, a approval more patient less on think, the In are to patients, are the in French-HU program, named it’s in

although certainly So as U.S. Last significantly grow, question we dramatically. not Wilson. to The for we was that the revenue in we is grow, to IND potentially going launch. forward expect think, move on

So far, the program has gone...

Gena Wang

For DMD.

Emil Kakkis

With DMD, yes. sorry, did you say? I’m

Gena Wang

both and Yes, Wilson for DMD. the

Emil Kakkis

that. Wilson, Wilson. And start you with for I Well, let’s Okay. added guess

runs drug product. scale, completed at lot a and well platform. the we Wilson, We of it making have in HeLa For made substance the

we going that there’s so and pieces somewhat methods other are of as we test no of our together. release some and for if work on than the with together We’re There requires track we’ve expectation program. end are overall, the year, analytical then some put And things coming should, is program. delayed. file more the in find But and long we And of the and ongoing. as problems well, a the at non-clinical gone IND, COVID-related that that’s working been the the plan should that, have be is going to of there has forth, phase labs ourselves lot on or shutdowns in clinical development process and those could

program, other a Duchenne, on So it’s the at stage. beginning with

which the are the the cell line We the and system, number process to in factors making also production HeLa of in design. other system, going of a testing producer we’re be is

get have forth, to scale-up process. so time. take will through the take months we that So some it which some to do will and Then

a manufacturing for able the get put us I said to more a and but wanted nonclinical in certainly clear, be be to frame, together. time work year haven’t to We it’s well

get want – So clinic, as good we’re with not very are in after the the immediate and an already at event. to it not it’s strategy coming We it and administration other immediate high-quality will production in treatment effect. a this system If come to that in right. optimize a an well as quality parties point it’s want nail we

Gena Wang

you. Thank

Emil Kakkis

Okay.

Operator

Thank you.

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

open. line Your is

Tazeen Ahmad

Thanks afternoon. FDA? And specifically, follow-up. on DTXXXX more for have get are wanted taking I for necessary you completed any XXX reaching just in your Emil, then good you both for supply the I programs? And Hi, and assays thoughts commercial have scale to a questions. potency where

Emil Kakkis

want the we’ve have program – that dealing an a They across I have entering in we’re X, the think potent Yes. that the see been it’s their us lot potency them sure And about Well, programs so of pretty assay the assay is all understanding on want part the managing talking how and clear this of with release. they Phase to thing. agency giving while, point with not programs. a we’re across process been as it’s for they potency make to a manufacturing new absolutely that each all and to for vector. to And

a a – have assays. We of number guidance on multiple matrix the allows for

multiple, the We have for looks have they protein those but those want story. that And definitely programs. at we’ll one in place active

Tazeen Ahmad

to And just little DTXXXX program? add can the decision you led to the steroid to on then a cohort bit Okay. a give background us more what prophylactic

Emil Kakkis

little of a would that, us bit and that a safety would would we we got the that initiation sort in any might better the bit – We inflammation on doing X, about the earlier of – easier it sense we the the was the the set from parent a there. help actually practically much. – little steroids we cohort decided moving earlier. trigger was We controlling DTXXXX a look steroids speaking, of going. commercial get in we the And for just, steroids earlier. inflammatory like used become plan It just it a setting was hadn’t talked ahead think it response Well, – and with in lot liver. looked at effect get at little any up the We process, as even

X, So but way the the well. learnings cohort looks And it’s as actually in it doesn’t it to our from it It seemed plan. the us. cohort, was prudent third with from that basically data, do affect

the long-term have durability product that argument can treated, will another patients you the us are the moving available approval as Phase durability. X of benefit, certainly will with is And patients we successfully in in become a X. have treat you they course, is ensure and to here treat and number be on while that other for having XX help tell enough the program. XX that, to piece an important far because that I we program, forward is process getting we could – the at patients going filing ahead – We treatment other know, of of that

extra having is few so helpful in a And that. probably

we’ll exploring So that. be

treat story it We’re to just it, commercial when to how already think enrolling in way patients and the the I in efficient adds in an setting. for you’re

Tazeen Ahmad

Okay, you. thank

Operator

you. Thank

from comes Citi. the Nochomovitz question of next Yigal Our of line

Your line is open.

Samantha Semenkow

for Yigal. on Samantha is our questions. This for very Thanks taking much

expectations or you new incremental in Just prepared first increase end patients the the that mentioned revision. the of your identification lower is that Is $X launch on the Crysvita on XLH the million TIO related that remarks? guidance to and for the to related

Emil Kakkis

TIO would guidance assume get put the the Well, that at the year did we of approved beginning out launch.

Crysvita So all it was for was we sales year. – the the put out for guidance

because Erik, if TheStreet. higher for of the predictions simply that the that was we’ve don’t end revenue the into lower guidance I tightening know, guidance, thoughts up our on the guiding a so provide? of And you part is other the guidance any moved had actually the want up you so to

Erik Harris

would providers patients find more pandemic. meetings in of a a I think trending levels, safety and opening are there to meetings but trending existing been direction. remarks we’re I my to build as on ways growth that’s yet back up and X% to have protocols. that seeing result innovative are and offices according say, live so far, in while the virtual continuing just allowed just we’re earlier, patients Yes. COVID And And And educate of the than patients pre-COVID not rates to less impacted seeing toward the they’re result of in are through is clinic. more team right

encouraged are we the we seeing. So that are by rebound

Emil Kakkis

Great. Thanks, Erik.

the the bottom continued, line, remove don’t – up tighten us the the not base really, mean, of the I end to bottom I guidance So of and the our focus has kept in has launch range. maintenance and able we’re off TIO, COVID game of and

I good news for Crysvita the think that’s franchise.

Samantha Semenkow

you be pivotal you Do this little able get advance of, quickly how Okay, great. gene into then DMD you. think do clinic, sort safety from advance and you will a you XXX faster a some than to have can this for maybe Thank the of follow-up other construct a data the trial? generated And into bit you think use your that just the programs? to, once question, you XXX therapy

Emil Kakkis

but more quite truth think be safety will in dosing understand from dosing Duchenne, be I safety is the programs, importantly, the different. those differences. able quite the would is profile, I Well, we the But The to effect. think range

So than we can there’s less little a upon. leverage bit

is might be would – would think manufacturing HeLa-produced, give HemA some which program, helpful, the the us some particularly I us advantage. provide

necessary alone. So I it programs say don’t they think is those would

through well approach with randomized us design take more the the phase which design would as and I think we to trial, dose would as seamless design well able advantage a achieve phase the likely range going the the being in biggest to being involved and – need to are as Wilson. which understanding take endpoints endpoints the through we as able dosing is clinical biomarker come to and rapidly,

measures of I I type and been settle That CBER think Peter predict line clinic. the is the X Marks a without then dosing of know just by that when shorten think it promoted enough be get would about and having make, you we you want the into know the Phase ways can right kind the and endpoints to the which you possible has to design, can gap. disease and after time a one of plan move to we the

Samantha Semenkow

question. Thanks great. our Okay, taking for

Operator

Thank you.

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

question Your please.

Cory Kasimov

good afternoon. Hey

from hope Two it in so Zealand. well. enjoying as time questions for are Shalini to off, your is me First New you hear great

of look follow-up kind the would not if like you Wilson’s there? duration in prophylactic that? an you And comment evaluating And of can UXXXX and X that and look into might how amend us take a that the are on just mean initial just clinical does necessary second Phase insight just steroid regimen steroid about DTXXXX that impact with cohort give X, prophylactic what early thinking of my build the you some follow-up you on your new is First now start GSDXa. then question into protocol are for would and the timing Phase a to studies

Emil Kakkis

Okay.

disease, is is question therapy the which around clinical first Wilson studies. UXXXX, gene for the the So

Our design. we stay plan with cohort than and exploring will will the – a the but do then X in X one information, clinical to that go be we will larger did in be what And there that be significant is others, it studies design dose. straight the seamless groups dose will into essentially Phase

we because on pivotal up seamless approvals we shot. we – so design. therefore, getting where going operate go endpoint are of set can And – think primary Duchenne. design, straight right one are through a a of single program and history on is doing biomarker-based go endpoint to dosing approval on seeking is the that the we reason And the you and to in doable is through be is The possible primary that in a agreement

will The help drug from FDA design expectation will very probably year our Phase there’s close plan haven’t cohorts extension core we fully that we the safety have up make some early need? program. is our But the time. it. The study that clear would is. XXX, time will time for point with is of patients they XX-week study. end a to We efficacy sure we sufficient length of the I study the determination. How durability is to X amount more than the in exactly to dose the component look, and So the to need a nailed or much But But discuss what plan want down based blinded agency data length, on in at think having to on patients.

first we allow can cornstarch question, are So that effect weeks good primarily the the we sufficient see with get the XXX that prophylactic keep down that, to reduction. is steroid to inflammation and to in us few looking

know steroids weeks data order few So us. of for need in we are the a working only how to

soon baseline, in in what as like was treatment. from third which cohort, with been of we steroids really what the only it as they meant were looking So have from a that’s did for. we weeks couple Last change that the time

starting so bit a now earlier. we And just are little

So from earlier. bit know. it’s a week, with as happens as see issues really little we It GSDI not to have that doesn’t it is we the is first just that And look that It are. fundamentally any different we And will long safe. where the patients.

proposed forward. the back best steroid If then it’s submit steroid could that, and the we I regimens to the think and the away way. is but issue we doubt that though yes, in will, I with amend we protocol new had from plan that way an probably Our cohort, protocol the

Cory Kasimov

Okay. Thanks, Emil.

Operator

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

Your question please.

Salveen Richter

break the Emil, Good pipeline, just where working if joining afternoon, optimizing great you Ultragenyx. been HeLa with the you could I Shalini. particularly with third-generation was platform how congratulations us wondering with stand into intend Wilson? with you and this to layer and It’s process? update Mardi, on to to this manufacturing enjoy regard HeLa your you create And

Emil Kakkis

Right.

the produced process. program So version the Wilson call the of is X.X with HeLa, we

which at already we point some X.X don’t providing are from some cross it, time However, presented need improvements, making, for which Those need are enough be real big for certainly productivity higher could we But at And will changes version, have, in X.X to even I HeLa several already enable have at changes at we Duchenne program the additional we than the more many product. every run. X.X over very ASGCT. the right changes, of to The but to productivity scale up therefore, patients productive much versions is that particularly treat head fold, X,XXX-liter produce clinic because version. or which and we level the this we that win enough reach And to will valuable to with now get productivity, created and the product time. really the for point, patent ability where levels think, really a those were you that. can that will with is HeLa higher

coming play into looking X.X done with We a existing cell HeLa existing though, can making can X.X the process line. producer version. The actually and at any version are line it HeLa really X.X take there. we turn be into So

go could back. we certainly So

well. We for can Wilson, other as do through programs we haven’t that the it done go and

the being it look as a Going of we the HeLa And human chance the to for would finishing version forward, we us will to see use. Duchenne but give right are development still version, X.X optimize that.

Salveen Richter

Right. Thank you.

Operator

ahead. question Please comes next go Chico Our of Securities. from Wedbush Laura

Ken Shields

on Hi, taking for this Chico. Shields is Ken my Laura Thanks for question.

So Ultragenyx has drew capital financing transactions. one for the benefited think to Mardi, recent strategy and what And allocation should opportunity? you how and about from we

should anticipate as we look ahead? what So we

Mardi Dier

question. Yes, heard easy pretty it’s the great, think on lot and Thanks And to about you I Ultragenyx, a the great. it decision a for join call.

them to this honored team but achieve way part guided be I the has the have of to next for to a such pretty help company that. I’m the in phase point, planned team now and their great get growth. they think certainly to of And

– the deal, I company. at together, allocation, at balance company fantastic non-equity it’s has the pretty capital brought talked the of done X well about of same today, today, in in bringing our course, the $XXX And cash which were the that have terms over we then, darn frankly, In Royalty to million Royalty and dilutive in capital, into be of to particularly end deals nature year you close last last deal cash look the Pharma December. And think a the balance number. Daiichi here, job, last if the

fantastic. So that’s

the company of we takes our that announce, strong sort cash recently. are course, cash a in XXXX. and forward, to growth really the going business that is But we deal want that I deal have a on highlight So position. us path. But base of We into Solid did with

deals purchase like quite look we So highlight well. that the to GeneTx option solid. I want as And

to the $XXX that program, Phase data. Angelman after period time X option of have the For million which some company purchase after for we

and we we So the to steps. but are well, very position, great we as next forward look active are in

Ken Shields

an you one competitive so a syndrome, for publication you potential IND editing just do for I Angelman a CRISPR-based there and could just maybe strategy? that think how data, an potential more, interim was came recent landscape the the gene light if versus about think Thank in on out. therapy you. And apply disease? your wondering of And ASO

Emil Kakkis

a there for Thank people there. you. And very of fortunately, Well, lot is there of interesting right for patients, lot working the on a now. Angelman Angelman strategies are disease, out strategies

across superior total that the now state. potential. small in think then So or approach. and in all we can brain, which disease to And in particularly it work a we really a important in or is We ASO, of therapy very the neurons. than work ASO is well getting because a even communications. the varies, CRISPR-type Whereas like in SMA, neuronal it wide ray by whole fraction brain humans something be even that disease this complex may neurons – has in not primate the to gene they felt the likely ASO while The just distributes was still can of induces gene more ticked have certainly way, particularly therapy expression – in

expression to an only And not it’s be mind, the gene that extremely actually the cells are neurons neurons the I desire. impact brain in getting think have imprinted. you going editing challenging, and enough in imprinted, is adequate are to the other effective keep

gene non-neuronal want do. you – if you is that you to not are may actually cells, be correct what So

our was beat. which hard strategy. looked paternal on complicated. does – so, It I fashion, expression it’s neurons. I more be that is than the all, there. think the variety there ASOs would having turned in chromosome, still biology it that’s regulated best to ASOs occur say, of out to It So was And view a it are there to Even wide in allows us. a was and going at

superior and Biogen the was, which in down that I how were Roche, the in expertise. oligo, it superior regulation evolved the picked basically their involved RNA knock felt very potent understanding substantially others the understanding we to lot is firms because regarding more the had Ionis, and and laboratory. think, science, program capable we is of that GeneTx tried of RNA there’s So both more get to than they and a with We a think,

strategy, excited strategies. and going oligo. it to are we I best about still with And match, hard to be think it’s I think, So the actually will be that other

Ken Shields

Okay, much. thank you very

Operator

Stanley. ahead. Jeff Our comes question from Please Hung go Morgan next of

Jeff Hung

best the for wishes Thanks taking question Shalini. and to

growth Crysvita Just flat to clarify implies in on quarter, midpoint am the I Ultragenyx right, territories. the sales the fourth of sequential guidance, looking for range at this approximately if

due outstanding to uncertainty You mentioned COVID.

So driver that the the main Thanks. fourth thinking it are for there that quarter? other or is is factors conservatism be about or potential we any should

Emil Kakkis

the like discussed ongoing to probably on there guidance maybe what What is I relates want out the going forward are lot to factors the about there? were? touch about widely are Erik, COVID guidance you Well, is Is a the think what changes surge really happening? and how the of how that Jeff? the question, risks which do to right,

Jeff Hung

Yes.

Erik Harris

Yes.

of to great the But that we’re as the will by As still a I market open uncertainty rebound to levels. encouraged is fully when pre-COVID stated, are there we deal seeing.

with challenges – some the think creates it – as we So in you into and year forecasting about move out XXXX. as close move

Jeff Hung

Alright. Thank you.

Erik Harris

Thank you.

Operator

Thank you.

question next of Lee from Joon comes Truist Securities. Our

open. is line Your

Joon Lee

Hi, the for quarter questions. Yet use taking to thanks that the not IGG treat albumin call patients, GTX-XXX Angelman’s, for said and to strong yesterday, my I of altered. on you implies congrats on IVIg response. On some the believe, were which IgG you

about potential as appear the us was what is, is, reaction, But the little MRI, response to So based And question my on – little Thank assess very be spread new about then you. actually? a the to functions? a Just to the call, patient patient, the inflammation my might can to was bit localized. levels the maybe the trying of second you in more of extent bit the question spread just of I you more tell the what other, nature any in described, greatest

Emil Kakkis

Sure.

more was just regard It was the no localized, use based, symptoms. we lumbosacral, other steroids a in clear IVIg Immunoglobulin having region. as effect. extent, lumbosacral but steroids just thought the for does or rational any were fecal situation would was that that lumbar it smallest patients bigger actually the using conditions, work. done reactively IVIg the known looking was them there but not – parallel IVIg completeness, It and and the So indicate That’s of the actually based we patient we on was almost was an actually and it for some there’s evidence not largest because are not point. to was We It’s only. not that have including With where is this the said It that really used. patients. extended at to hemological to But in the region in basis.

The up levels actually was it not levels. So thoracic to include and higher it was region for smaller. biggest did lumbar the

the the part. the Trendelenburg, don’t only spread. up We Without be up would little it’s where think region we patient would to it sits after think more bottom if a fill patients, where it small appears immediately than fill administration. So drug it it a big

mixing action now that the and long goes on reaches dilute. And region, So which the both think top, in enough, out efficiently more and is with north of dosing local fit in from the help we The and it a thing mix flush any and it to and wash the over spinal help movement. after is adding And we there there So there move magna, cord will effect should you with drug are– stuff drug CSF the we cisterna have mix what almost we flows, one think once out – pumping additional a change promote cup artificial the the moves CSF all the tip think when hopefully and of to produce the would place. the mix are things. sit drug which lumbosacral Trendelenburg, that the will the pour if the up we the not

research And commonly not So that’s intrathecal actions are a believe distribution. why other and used in the been drugs. thing. have new XX we I alter It’s years. personally doing can therapy intrathecal these we and for Trendelenburg

very you will why an we this, it do And alter. issue. manage it’s become things very at familiar not area These an and simple new confident I’m that’s we’re are quite So them. can – with can

Joon Lee

follow-up just Trendelenburg? question, that with led or other And ASO there inflammation reactions was therapies a were that some resolved to localized

Emil Kakkis

meningeal CSF Well, clearly there other cause ASO irritation are therapies protein. that elevated and

the in – have have that. ones multiple shown clinic So

that true. definitely So is

Trendelenburg speak and intrathecal can’t is clinical to toward do. solution used in people or needs know, situation. So I brain where the but can a it of to in they as do the therapies way any the to take don’t judgment But and other widely move be Trendelenburg work. what drug don’t I the heavier it used

Joon Lee

you. thank Great,

Operator

you. Thank

Canaccord. comes of Arlinda question next from Our Lee

Your question please.

Arlinda Lee

going Dojolvi, on taking have my you on on the quick the what regulatory a if you can then in you appetite provide to more program discussions X.X. you additional that one and are indications your about into? HeLa additional I Hi, enhance thank looking update am is you for Maybe And you going version the interested for talk HeLa mentioned collaborations are involved questions. maybe about efficiencies, there. on with wondering in would how you agencies? And now think further in improvements you an the can and be

Emil Kakkis

Thank you.

– Brazil, on have filed to priority review. in filed under addition. We Dojolvi in have in we update, Canada So we regulatory, just

In sessions. get X because we a And ongoing are and we then pediatric so are those play. we having that in are the are investigational Europe, having plan, to need discussions

no yet for filing So plan Europe.

reg where So the that’s are agencies. we in

sales many HeLa approval, and very really And do With to With the the X.X, alterations, requests. fulfill are requests help is substantial generate places some we patient new version and named U.S. important. in the though, can we where can more productivity come which those to regard us.

looking and owning we with Daiichi I ground. Wadsworth value. the ones Sam lot Sankyo. we continuing its of, kind possible, monoclonal kind days agreement as which gaining whoever creation next of the is, of would are did of And indications monoclonal team field early is led or the adding happened that an ending controlled ow in partnering tuning, technology the the and building would additional for of on and for whether large-scale, things the collaborations antibodies that so are started, cost-efficient believe are to system provide there and we platform of changes in antibodies course, these by for a up And develop what the think we

open deal of arrangements. which of with largest we more those and became other look at platform, we just – indications. extensive deal looking particularly knowledgeable another The And as an of and another on of products production putting and advantage licensed our which But high-dose and AAV are the knowledge have high a Or involves help for to of lot available We of was mammalian where high-quality a their to indications. We a system do is it’s tech deals at system we similar large-scale distinct transfer companies the and also which story are kind bring And would certainly in about deals, created particular as especially the product area advantage, HeLa could of where of manufacture, to microdystrophin. the avenues. coming solid spectrum, variety we we work the so play. could if fourth, it one Duchenne We productivity technology supply technology – make tighter take that that. and then a as pretty would unique, ourselves the a particular as could Duchenne in that are pick to type the be effort. with we to Daiichi Sankyo for third would lot other a muscle and for scalable available up and partner us

platform. So versions I the guarantee in and we will further it. are Sam looking of at ask those, investment continually for

Arlinda Lee

Great. Thank you much. very

Operator

Thanks.

question of Bernstein. next comes Chen Vincent Our from

Your question please.

Vincent Chen

and potentially certain I much. RNA complement-related what the use would have provide color you some Angelman’s, be on related the a an effects with couple above that wondering hypothesis? I around causes is is what simply I related your seen inflammation of the be AAVX less molecules to the may likely previously, for collaboration, solid be DMD. observations Or longer curious half-life think be vector that capsid effect certain think common Angelman’s this Is you is you so more that DMD a is this you RNA do on was be what to you applications? something have mechanism questions then your Starting could thinking the a if of this for program think saw science potential to dose? are And and or or seen findings certain side to a dose support and Thank intrathecally what RNA these and using to susceptible? would like something likely

Emil Kakkis

Sure.

So in very and of from of help lead drug. to ASOs the from cells to how apply Angelman paper various Crooke has the Ionis story, proteins inflammation, is that, some showing its mechanism ASO All of And mechanism the combine culture. the distinctive of showing probably we very the action Stan elegant think, can the kinds published certain toxicity. toxicity and

ASOs toxicity positive that so as idea And is of oligos. many different well-established a in class pretty

do down with issue what something nonhuman widely up. lower without done the in oligo time in cell really is the and too of We it’s local and a getting What an now relating the are So primate. in our not know we and contact see taken the It than to this inflammation has concentration human, being are in know toxicity believe local CSF present much those why not with tissues. humans, a we do in very also primates. that oligo don’t see primarily locally and we That’s we brain that data to nonhuman of the high you locally. we the we is with supports looked drug. observed. been very the to the in have concentrations But And anything do mechanism that and protein way mechanisms in problem. in non-primates, is a They the same issue concentration problem The in associated Trendelenburg as The of it’s applied hadn’t doing for think RNA. meningeal has not new.

hasn’t having And but caused are this so we take action, it therefore, primate. to in nonhuman problem

number the that a from toxicity. laboratories its there. in oligos higher We high vitro at know of know we work by localized concentration can So concentration cause is

simple in manner, and in the local to inflammation. lowering think a we the level just So exposure that reduce trying

the to much related the I think type drug it’s in a something is actual action drug a fundamental that’s not is applied locally – to toxicity too toxicity completely manageable just of space. related that and local

what from does we very it have very will get drug we I therefore, managed. something differentiated and confident and So feel the feel, manageable,

which that and This compliment had Pfizer Now complement Pfizer time may with program, longer particular appears be the have problem, this extent AAVX, this to – be know is not lasts – The with regard that due factor. to the have and Sarepta activation. Duchenne, clearly program exposure have to and both antibody-related little AAVX-related program appears activation, could disclosed, be longer. they have to vector Solid’s quicker, and we a bit to a

to big another Duchenne I and think it we muscle. circulation And something to work. with approach, deals upside had. standpoint, from a like create another an best-in-class a combined we AAVX good treating product. We Duchenne. for It can platform to be kind choice another for better be current they very that is potential winners, get there figure and And good indication. an well, We type it’s will goal a was Duchenne and it and do some very a I option it’s I got Solid, lot patients. have ammonium also learnings the deal confident we win. for have have could that way that gives that variant in this technology out microdystrophin – could combine deal and is think the us of and there to very combine And we its the a provide and feel them doing that its lot done they product. think to issues X the clearance room really good There of immune that them a a for have the good and could and has. potential faster therefore, together good being a is room way common here profile of has that’s and a which the best and have with think distros option of past. a work Solid They the optimum put intelligent fine profile their from out of gives delivers has with know This

else, can but better. think we be are I behind We everyone

Vincent Chen

for Great. taking the on the Thanks Congrats progress. questions.

Emil Kakkis

you. Thank

Operator

Sir? Thank back would Joshua closing the call this to turn like Higa At for time, I to you. remarks. over

Joshua Higa

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

Operator

for participating. thank and gentlemen, you Ladies

now You may disconnect.

We use cookies on this site to provide a more responsive and personalized service. Continuing to browse, clicking I Agree, or closing this banner indicates agreement. See our Cookie Policy for more information.