RARE - Earnings call transcript Q1 2020 | Ultragenyx Pharmaceutical Inc.

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Operator

Ladies and gentlemen, thank you for standing by. And welcome to the Ultragenyx First Quarter 2020 Financial Results Conference Call. At this time, all participants are in a listen-only mode. Please be advised that today's conference may be recorded. [Operator Instructions]

to call like speaker to now Ms. today, would hand over the your I Keatley. Danielle ahead. go Please

Danielle Keatley

Good to first And our our update We've afternoon. website welcome a financial conference and financial issued can call corporate results, results at which for find release ultragenyx.com. the press quarter you XXXX. the of detailing Ultragenyx on Pharmaceutical

Danielle Senior I'm Keatley, Director of Investor Relations.

Chief Harris, Medical call are Officer Commercial Shalini Sharp, Executive Officer; Emil and Chief Bedrosian, Erik Chief this Financial Kakkis, on Camille me Chief Joining Officer. and Officer; President;

filed would with be like and filed in will investors SEC, on to Private XX, that on limited our XXXX, XXXX, remind XXXX XX-K filed report soon, our all will provisions the available annual the to forward-looking of our statements this not report forward-looking reports the include Form the I Safe Form the that call periodic types website within Securities on of of February in Act XX-Q on but Investors will which Litigation of section. Reform the statements be subsequent meaning including was as our Harbor identified quarterly

statement. uncertainties, beyond call actual These our date many this as risks differ views materially and could results our in are including and forward-looking involve projected of forward-looking of that any control. Please substantial that the only represent statements note those from

SEC. to differ as those the our of further well description periodic see to reports from the risks actual and materially a business, filed results could in For statements, relating that the uncertainties expressed risks as forward-looking with our cause

Emil. the turn to now I'll over call

Emil Kakkis

good call. joining and afternoon, and everyone, Thank for today's thank us on you, you Danielle,

COVID-XX their health and in our impact been despite on. precedence health focused The point on pandemic everyone by families over care society virtually at their This just patients else. we the good own everything first everyone truly their history quarter a unique when of Ultragenyx the highly had Ultragenyx, has providers. is a taking everything serve, felt going the of

effective pandemic, The has biopharma are this assays, facing and clear. the never are a we mobilized has seeing. ways, robust drugs, to and industry dramatic industry When its by expertise progress encouraged develop been the more need in vaccines we for biopharma

directly mRNA for COVID is vaccine, COVID-XX a a last announced mRNA virus-neutralizing COVID. it immune Although, is therapeutic response recently on and low positive invested Arcturus an who working COVID dose. supported not showing very They candidate in Ultragenyx mRNA preclinical inducing we vaccine that a partner vaccine based year, potential for at at Therapeutics developing their data important potent our

The Arcturus adaptive mRNA expression amplifies response from clinical induce better. vaccine forms We protein an RNA both immune technology unusual initiate expected which is summer. this because study, self-replicating to to stimulate inhaling to immune it their system also uses look forward and [bioreactor] data a that

they need For participating clinical is to pandemic, our our continuing as treatments studies well during to products Crysvita those support as in receive treat own we and that needed patients. challenging the the to are to ensure Mepsevii, with in do worked patient our continue time. our this whatever We've

established fraction had To-date commercial access. relatively any with of small our only issue a patients have

institutional time Fortunately, lessening the their via is is impact and more closings to receive home visiting of patients already of our for nurse. at at patients now. convenient than care established X% This be the potential Crysvita of want

Crysvita provide temporary need feasible. that option team the nurse the authorization and with not resolve. which as is number for few efforts of self-administration of helping site payers an work help our our our administration are our example where patients is One do care the of with teams is system need A doing that patients in-home small to the to of

enrollment there to on are prophylactic is closings. move the development collect safe That enrollment. the to DTXXXX On we the we to and resume to said, OTC clinical it institutional program for we those forward, they data able pause due treated. but are for steroid go cohort some once already gene and limitations We of open programs the the front, are our can therapy ready

Our most data earlier presentations program. recent Conference, Virtual well presented therapy from data at the gene and ASGCT as and as both GSDXa technology other and our OTC were about include program, stage

two has a patients the to For treat to GTXXXX and manner. program is the GeneTx clinical working on with to to will partner our and partner treatment. at access clinic Further depend receive in forward safe progressed GeneTx progress push who ASO Angelman, program continue sites our the enrolled

challenges, created has patients continue to COVID-XX our ensure remain FDA clinical continue While receiving and to and necessary U.S. programs with steps therapy, we a their commercial schedule of taking unique preclinical discussion are data. on our set generate our

is a I'll provide will call With doing reach. our that, expand what turn update and core business Erik, the commercial team strength who of to to his the global our highlighting

Erik Harris

Emil. you, Thank

is times, extraordinary teams these day The work the ensuring extremely and care. medical I'm the commercial During on every focused continuity patients. the for of especially team patient grateful commercial do behalf on relentless of

important XXXX, Since base therapy. grown ensure clinical the benefits patient April Crysvita to is we significantly these continue that top priority patients have receiving of launching and in our the this

for go we of able of to social seeing patients to COVID-XX site a distancing, are number period who this are During treatment. care their not small of

team by for Our to working is includes these find patients, a home nurse. services hard health solutions which alternative

by about COVID-XX specialty and been of in worked handful a specific we provider the Emil that concern exposure. response payer mentioned, These aware allow healthcare the has pharmacy a to with As a where of to their have self-administration. instances patient exceptions for are initiated are

has demand COVID of year, for seen received In we this first through which Crysvita. to North nurse administration, through helped have minimize America strong disruptions. patients continued the than any More related of XX% at-home part Crysvita

would to expect, present receive some challenges a new Crysvita As distancing will start and for therapy. one diagnosis social patients

our the up come and of providers these with products. this educate and which care burdens pandemic additional team has is to health respectful new to on being safely innovative understanding are our put We required on ways providers,

national educate of caregivers. virtual a teams programs field and speaker audience Our are to patients conducting

We are providers prominent care also led KOL. will that hosting webinars by be for health national

Lastly, have providers, tools our use as and maintain our we contact manage of We their continue to start expanded resources new initiatives with types of they will with our actively to support generating health around believe these close care these digital suite patients XLH. forms. efforts

reimbursement patients number reimbursed to we on to treatment feedback demand steady receive agencies, In and America, meaningful regulatory Colombia North positive increasing continue also increase. continue and of patient for while discussions named-patient to continues Outside demand we see name with these support Argentina, and have of as the therapy.

to judicial we Brazil, successfully Crysvita the injunctions a of treated these number with with steady patients seen have In have a of has legal be for able the filing to Crysvita resulted in after growing This therapy. of negotiating review receive been number patients requests process. many who

had full that Earlier patient hold, year, Latin Based treated the our reimbursement to are pricing discussions demand, in XLH forced requests, are the situation shift and Ministry The and the the its with significant Brazil in seeking approval. patients we confident remains these America. Crysvita. Crysvita agency on reviewing with on allowing to for they increasing discussions a focus opportunity put COVID-XX positive interactions to but with we be obtain still of Health and new

of officials Crysvita successfully patients sales clinical granted of XLH for for data to in our Turkey, an Crysvita approval important more injunctions. believe strength being quarter of patients and named-patient continues therapy in will on receive is the Turkish demand we grow have based with reimbursement with XXXX. we first positive are discussions Similarly, part and had as the we for

to disorders significant patients and our UXXXX and by patients July long-chain benefit we a a need that approval studies for XX, patients, the are of treatment there UXXXX, acid a physicians substantial Based current the feedback oxidation from continuing number prepare can unmet and to Shifting potential PDUFA from clinical options. UXXXX are date we therapy who believe fatty XXXX. despite on from this for data with for of fills

to XXX centers many the of are estimated majority patients the of With the at metabolic newborn X,XXX of The the in LC-FAOD across diagnosed. the of screening country. seeing are patients for approximately U.S., major X,XXX advent already

efforts with which will relationships instrumental launch these be in of centers, the strong helped build metabolic Mepsevii UXXXX. commercial with and existing Our have Crysvita

leverage significant overlap the Given of the increase we to infrastructure will much our current our and with only modestly able be current force. commercial activities, field existing

potential Looking approvals two later forward, we have year. coming this

approved. therapies to We expect UXXXX while steadily patients significant be if for all launches and in are These with they build will over Crysvita few the important to options. bringing there time TIO, LC-FAOD interest

continued we our success. are than our rare plan fourth three collective utilize to for ensure experience our disease and to be what and We could third launches less in excited years

With results who to through walk financial for the the that, will I'd you over like call Shalini turn quarter. the to

Shalini Sharp

afternoon, Erik, and you, Thank everyone. good

of financial included $XX.X In Earlier today territories. will Crysvita three includes and XXXX, profit March we product $X.X $XX.X Ultragenyx a XX, the briefly million. summarize. was $XX.X update, total sales in and was ending the the that $XX.X Total million quarter revenue issued collaboration first months for XXXX in million. share a total million This which a territory million revenue in American press revenue of or release North of other net I regions

Pharma in XXXX revenue that Non-cash million. territory sales the in Recall million. of quarter of royalty UXXXX the Crysvita was to and $X.X was of million Royalty XXXX. first related this European revenue in product $X.X December were Mepsevii of for royalties the sold to region $X.X revenue was rates the the named-patient

$XX.X first $XXX IND for non-cash maintain which total acquire by paid GTX-XXX to their $XX company US Our were the our includes operating This million, of This expenses compensation. FDA. of the million was accepted also the XXXX. to option quarter after stock-based GeneTx was for includes million to

$XXX Our were million. R&D costs

over We expect advancing to early moderately candidates into increase from development and we clinical R&D early time cost study. as preclinical product our continue pivotal

Our were $XX.X QX in costs million. SG&A

over potential to our increase we support We expect and new time programs as launches. moderately product commercial SG&A

XXXX, of produced Our not a $X.X our million inventory manufacturers that with negative of meet cost our quarter did the batches that which of Mepsevii a standards. agreed million credit of first $X.X to the includes of quarter one during for to previously due was was quality sales

As related a million or any during had year future the these reserve batches to issue this We anticipate XX, a at reserves reminder, XXXX. interruptions $X.X recorded this December supply company for particular the time. do not of ended previously

first the non-cash a million to on interest $X.X million from in the quarter of related the of in investment on fair March and loss includes loss expense gains value or the liability $X.X securities adjustments compared XXXX, $XX.X future of The Arcturus unrealized XXXX a net sale equity Net ended royalties. quarter loss basic with was XXXX. share first per $X.XX and or basic for of and share the for million $X.XX diluted, million quarter $XXX XX, a the per diluted the

In million Daiichi sale and were We payment. These Sankyo the This the used excludes the of operations with quarter cash purchase $XX.X closed in available $XXX common million was March partnership in Daiichi from investments. stock manufacturing Sankyo cash upfront includes of in in XXXX $XX of million for first million. strategic quarter XXXX. the ended the equivalents from receivable XXXX, which but $XXX.X funds April cash, received

Moving to our guidance for XXXX.

guidance that the year. the maintaining beginning shared currently range are of the at we We

in America, Specifically, that our that the million to expected in include with the million. exclude $XXX North transaction of and announced to America be monetized Ultragenyx Pharma royalty and and was Latin Royalty as was this revenue was completed Turkey, EU XXXX. Those Crysvita between is territories December in territories $XXX

exceed expect decrease pace in in net expense our We used to also maintain to cash growth greater of therefore of expenditures. cash operations, net a significantly as capital our the continue burn, pace than revenue growth well and as of the includes XX% which projection

at they impact if pandemic when guidance COVID-XX the As become updates look business, to will the including range we'll continue and material. evolve our for provide we the potential or

that, this like Camille, of program. recent call turn regulatory will some over update to who I'd With interactions our provide on clinical an to and

Camille Bedrosian

everyone afternoon. a you, good I Thank too Shalini. wish

long MCT Starting or a program. two programs chain on disorders screening current with devastating currently regulatory LC-FAOD, we'll our UXXXX significant update our provide fatty for and will an oxidation newborn even acids chain the with and Angelman I disease review morbidities with oil. despite discuss Syndrome use under progress of

review currently XXXX. July expect The new review the to application the of track PDUFA The decision and we on by is set continues a date. date drug FDA reviewing XX, and PDUFA process have

have required not UXXXX clinical Importantly, do of FDA's inspections completed. believe this point, anticipated all of any At review. the and for manufacturing been for inspections additional completion that would we successfully be

Brazil oil that doing and the Canada. is on application in treatment by know EU on marketing experience, are LC-FAOD authorities we regulatory authorization our MCT In even discussions seeking chain continue in addition the are regulatory with we and to many patients not US, management current the authorities under our options. Based progress with and in review new well with

seen significant requests of the have through access interest for and We named-patient program. our number this compassionate

rare Moving Erik and I the moving patients of with options. current to progress TIO, treatment or leaving are for Crysvita second closer be am medicine surgically Crysvita debilitating that that have cannot no commercial patients discussed the for to this disease tumors our other for a approximately tumor-induced half approval previously osteomalacia a of for with which encouraged potential them with TIO. we removed treatment

on BLA the FDA approved of the be disease date would patients tumor. for review a of this our is XX, by decision June The PDUFA supplemental devastating and an first setting unresectable for XXXX. This the treatment track with of

with be required inspections anticipate completion their FDA continue conversations do we would of We that and for review. the not

our will affects no to approved touch on Angelman for treatment XX,XXX advance disorder Biotherapeutics there options and not patients is neurodegenerative. a Syndrome. approximately and is GTX-XXX, antisense I The a GeneTx treatment neurodevelopmental worldwide, program now disease oligonucleotide that neurologic of today. with devastating disease are the Angelman an

of So specific that potent that We GeneTx reverse is developed the the ataxia, seizures, oligonucleotide an manifestations, antisense believe directly there the very and has at cognitive many a disease. approach potential impairment, can team include sleep and which speech dysfunction. target differentiated and to disease

clinic the efficiently. and moving have they addition, the into a job program rapidly In of tremendous done

cohort first dose continued for the Phase institutional and will conduct. I/II to patients the study been has continues escalation, cohorts on continued the enrolled progress of The through two depend safe status study

Fast granted will this designation FDA. was GTX-XXX and with this patients therapy facilitate potential of the expedited This Track designation the disease. month, this review by devastating Earlier for development help

preliminary track COVID-XX study continue we this first site we potential half progress on the in to data and pandemic data the good collection, and of team the expect making While the impact activation is XXXX. of from

see, side treatment moved today. program. tremendous therapies Furthermore, regulatory continues you you has As have who Angelman have the in are who do the options not clinic and and on making the are new bringing months, we do the coming adequate job progress two important a to team the for to waiting could can indications into Syndrome and program this patients advancing significant approvals

call will I that, to With now turn the Emil. over

Emil Kakkis

you, Thank Camille.

and ornithine our discuss for limits excellent is urea into cycle deficiency up move therapy OTC can your some deficiency DTX-XXX, patients deteriorate hospitalizations, programs quickly ability body's to gene Starting or we'll will causing with coma metabolic and and neurological disorder to gene then crisis, an death. detoxify ammonia these questions. that I full in cases our deficits, and therapy transcarbamylase the the treatment OTC. of urea

of responses At patients dose and are we first seeing year, from and the believe adequate from responded scavenger we study. diet. all from this off patients' beginning to In reported up three effects. their the response cohort this In positive the nine and six longer-term have study is data of of dose patients and we achieved two and data have of the therapeutic importantly our come OTC level cohort cohorts medications X higher their liberalize consistent X ammonia three total

updated and Gene ASGCT to ASGCT next We And discuss three after from an GSDXa data consider call cohorts patients plan data presentation hold investor appear will this American week's and OTC and we Society to these first Cell both responders complete metabolic update. patients or Conference of conference to of be and cured. the study these the the Therapy present

waiting consistent expression. fourth risk to cohort the through and for we well the same the steroids. X, more at the reactive patients planned to are the than using rather We've these this COVID-XX program, institutions, fourth but expression, potentially using Due cohort steroid closure of identified especially cohort. patient patients as those dose OTC to the to steroids as level prophylactic pandemic, we as enhance enroll dose potential For provide

of data we in cohort significant this delays the this expect At to point, XXXX related second COVID-XX. still further from barring half

the meetings first regulatory the cohorts and alone. based our meeting edge planning simultaneously three and III III also Phase ANDA FDA to with for we the study expect are Phase an meet We on

on endpoint. XXXX. clinical FDA FDA, Based ammonia and expect enrolling conversations they have first will a a considers ongoing our in with endpoint based the Phase of be that primary expecting we is ammonia other products The validated The ammonia. approved begin III study on to half currently

four in to it to have But every gene they up. or type in disease hypoglycemia. the DTXXXX, glycogen levels life-threatening and miss their to of not Moving parents death, complications and storage cornstarch with fear disease Xa, these glucose Patients and which hours, dose. patients long-term does three cornstarch to a take our severe single sometimes keep today can its address GSDIa live leads if

Today, decrease we've patients cohorts a per demonstrating shown required meaningful clinical cornstarch. to the in gene levels, the substantial with a copies data dose two kilo including and XeXX response from of XeXX dose at the therapy first all six

Specific takeover discussions Phase in dose our of the three FDA plan same about December the patients and with III at study.

visit. from share occur the COVID-XX, we as data cornstarch visits ASGCT due Well, data which not the will the the patient that did cohort to well did meeting We not to to place, week. we be the reduction at at should available the some confirmatory able site site require expect of as cohort, later from share if next data take

glucose be could delays. year by depending there in with Phase for at the end study third share and whether begin the on In the Phase any position continuous on the will was II Depending a the these glucose cohort, this are from data confirmatory we III introduced continuous available conducted further we the home. to monitoring expected to into of insights protocol FDA, those addition, monitoring able ANDA be meeting

our and At technology, of we the producer for believe HeLa AAV is of their partnership Daiichi XXXX part and cell is them with our particular, therapy XXXX non-exclusive Daiichi the strategic mammalian March, entered liters including Sankyo in this HeLa into vision the system manufacturing manufacturing culture. the building a end gene platform program scalable license key our gene interest we therapy new a granted their of most partnership is shows suspension Sankyo a line in platform. that

a and in Daiichi in Sankyo investment of million made payment upfront an cash equity million $XXX $XX Ultragenyx.

addition, In PCL, transfer million form. and HeLa, with upon the pay will HEKXXX Daiichi Sankyo completion technology of the $XX

As of on either also AAV single-digit royalties is and to look well point technology time. as, over I net non-inclusive will manufacturing this other sales around products out our system. collaborations we that build

major to our will coming the progress, move continue then milestones recap that in we briefly I'll months can drive our the to and Q&A.

continued we to good $XXX America performance $XXX believe of expansion in our and achieve on track and time. reach America, between America, to over million Canada both and US we're Turkey Crysvita, the will This North still in Turkey. revenue million driven Latin by be and Latin For

seen the support to time. far We'll impact from continue COVID-XX, special during this have does patients efforts we and our minimal crisis established so continue impact could business. the new While and patients

In addition, small be indication in a If to this TIO, treat to but preferably phosphate very over population, in Crysvita one for believe need procedures will adopted was we urgent treatment. therapy. expand we're looking patient approved

XX, the track PDUFA date decision For July expect UXXXX, is the of XXXX. our by review and NDA on

able launch be preparation to approval. in currently are We following efforts begin to or immediately

many share positive our next we've the responses at for shown both programs will programs, GSDXa, ASGCT on two we from the and both in week gene OTC Conference. data programs For therapy

or HeLa does the the to to program be pandemic provided of delays expected continues also the disease system by and clinic. will an is rare utilize second IND progress cause company's for program activity. program manufacturing in the XXXX manufacturing not any clinical This non-clinical end COVID-XX the Our

advance GTX-XXX XXXX. first in provide to ASO expect of for dose Angelman cohort program data half the we Syndrome in preliminary planned, and cohort also continues the first additional to

and even make the on business can a strong during built leveraging progress non-traditional As financing. challenging front, and resilient these see, we to significant traditional We've business continue development commercial, you and times. clinical

are we will to in therapies impact COVID-XX, see patients delivering continue we well-positioned the to we While important need. believe

stop I'll here.

your Operator, move for the Let's please you question. to the provide call? can instructions portion Q&A of

Operator

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

is line open. Your

Yaron Werber

you at the couple monitoring. in you in like So then, a from - Emil, glucose all to the is won't are are basically or - OTC thinking of How just sounds and as looking we're reconfirm conference. potentially now updates better? get terms of the on going secondly it we And get you a data it that able GSDXa GSDXa, get to for to questions prior data week hospitalized mentioned, the can can patients the obviously, data, be on have now

expect we can have and I that follow-up? at might data meeting sure quick what So a

Emil Kakkis

to Sure, going Yaron actual obviously, I'm not the pre-run data.

that OTC is the we're you the tell from you've I'll what that seen. cohort that expecting So, already is

So that it be more data from will cohort.

cohort before, announced will as earlier X. on cohort just as presented The done is we that on new data that X data be GSDXa So not existing dose been and Cohort the further the the Cohort so the that's a same year. new in

But it think amount they But able the later data street. on and will the new the not significant a data, we patients a present of Cohort have be the some story data to though we because connect. where of could have enough we to not to points, X three all will be

what that's So expecting. we're

Cohort an X Cohort of extension for should but be new some more data. So, X,

Yaron Werber

sort will it of the going programs monitoring as into I you still Phase the have probably might Phase that like hopefully questions you meeting, not going are, sort both that dosing? II the patients steroids in as Okay. the well. end about done sort you're planning where get move point cohort guess III you're my and in the glucose you But going GSDXa to of in the to III, by enhance and you point, be follow-up of of more fourth the FDA And prophy it have like to pre-Phase of three looks OTC, then and sounds to talk or will at

you but that the the at want data us generate III, more to make the suited Help product don't about, you'll patients. these same of can even better for time, Phase even think hold maybe kind

to is do you. Thank So, the thought III potentially thinking or dosing latest about are Phase the lines? to then protocol on later change you a and additional with move

Emil Kakkis

would OTC go understand, has our than expectation I and reactive go programs with will I for enough reactive prophylactic with the rather program say we you steroids. the steroids Phase thank data that Yes, to prefer steroids enough work better been probably prophylactic Yaron. III We mean, design than There we and arm in general.

We have that. enough safety suggest information I'll

So patient We we have testing administration drug rather authorities than the our arm trial the design, endpoint. the difference a few plan I like the the the size would the it to that related what issue. forward about design trial, randomized issue confirm the the primary nine talking in is well controlled we're regulatory put works to would design, steroids hand with though patients. can and the prophylactic call The size, of discussion approach. still we that with data a by But begin

talked work prophylactic with the can through, through we that discussion steroids that think have can the is we All agency. the the be and and only toggle

get conversation for we've we OTC. process start, but will the of we can prophylactic So in steroids, before done begin we actually regulatory a

For GSDXa, at are these looking we data.

agency Phase further a the conduct the with to we about out and both cohort III. after in out, while information put our nine-patient come next talk we'll the call in design and But the We will it to program. can the so our on cases, make steps expectations we're data ahead call

Operator

next question Gena Our of from comes Wang Barclays.

open. is Your line

Unidentified Analyst

name [indiscernible]. I'm is My - David.

on on So question Do my the COVID-XX. you first see any manufacturing impact incremental is specifically?

Emil Kakkis

chain so for far, to intact. we challenge Yes, manufacturing have supply seen our be not a appear

we disease program seeing the and program. at which that the cushion. third commercial on three products our for rare we're on us manufacturing to are inventory as UXXXX had all products would also We time, for substantial of for the be necessary our gives any But impact lot the one hand a do this their not

chain. with we're good the issue having any So all far supply is not and

Unidentified Analyst

those on what GSDIa, approval question initial is III? wondering was so thoughts hypoglycemia for next potential some for potential designs on The I endpoints, are Phase trial and example, so potential

Emil Kakkis

design I That's GSDXa expectation. the both XX-patient OTC part first, for can have discussing a start our the randomized controlled design last around and with been trial the and total. that we probably is

we gotten still of the but the of ahead not have have and basic and several kind that that hypoglycemia is the an be We important. with if that design. on show endpoint of the The GSDX primary go do one can would that on it's us ideas for agreement with and one original certainly discussed options certainly HCS of FDA, we we

community. understood good is part real hypoglycemia setting, that agency the is or by reduction what is to in ability glucose The seeing well their or how in is looking also control. medical more glucose usage the like, their door look as good We're at opens about GSDX that the cornstarch control glucose up requirement a the another possible continuous with monitoring of world

that primary. so to And through plan, develop can work story just a we not

secondary other and dramatically, where what can glucose patients metabolome situation improved achieve will endpoints characterize we believe benefits. of glucose metabolic an good control cornstarch the But have is their reduce

ways will of be, we'll about the our Cohort after lot a little more whatever endpoint a we but talk data. there's X to present think we So go, it

Operator

Our at next question comes Jefferies. Raycroft from Maury

Your line is open.

Maury Raycroft

I had that's say challenge generally hypoglycemia you GSDXa impact just and one wondering for - this cohort, to if well, as expansion time cornstarch, reduce the an modified had the in if cohort? if can if

Emil Kakkis

to we discuss want see don't it. data. will done But I it and Well, on that have we

less we obvious. seen pretty it's it, you've pretty that's starch right, from glucose less natural think get then I gain

how quantity glucose might So we does will But impact be from you presenting much they expect, that data it. cornstarch shortly. get as

Maury Raycroft

if you glucose sort could expect data from cohorts the comparison that the cohort that I'm in wondering a expansion the some dose-response and monitoring And get continuous compared then, initial for should we able were with update. be of to

Emil Kakkis

glucose - the the well. monitoring We won’t at and later after look in on getting very be earlier but putting patients will we course, before able to be

look quality fall consistent are and hypoglycemic So stable is on good we to can is we what common based limitation. the like, But what's and the one - it that how cornstarch for see will common look their control, hour compared treatment. how XX is variability is of in - they how be events, know a

So I improved control think exactly. the we in glucose interpret of dose it level metabolic patients, us will help that can for confirm the whether those has

the require determination one just think I dose more test. than would

multiple think in this have the we to look are XeXX doses and two that from program it I point is only XeXX. There at at parameters.

going have the we data based exact provide we to the sure is those already. would is not be cohort I'm more dose that XeXX will assume CGM doses, clarity. But on two that Our expectation between a potent

but I forward. help we components confirm do the what will it will - think be us going think one I of do

Operator

question next Stifel. Adam from Walsh Our of comes

open. Your line of

Unidentified Analyst

This very A Crysvita TIO, Zhang] I you get for which for Adam. on the hope on FDA will soon. [Edwin approval quick question is

First those many on this tumors mainly Crysvita is targeting patients that how of are Thanks. whose cannot TIO patients estimates? course your TIO removed and tumor be benign, surgically typically based on in category

Emil Kakkis

you. thank Sure

little have tumors a of are the There have make bone mesenchymal do majority that few in a associated. and of FGFXX. FGFXX malignant talking had tumors making we problems the been So, little But source patients are tumors the vast that tumors patients majority general, do benign. about the benign are small who have of you bit

other to remove have hard treatment. that tumor, the that X,XXX We means secondly, and non-resectable be for half place TIO don't have have of had first be sometimes do, we candidates numbers on precise and without believed would X,XXX that have it's a in United to around to or but found tumors more them it's causing is a States, the likely good tumor in round in can harm. you you it has Non-resectable

And in option better situation a long you can a Crysvita of a immediately where so in run. be lot might the end ways there's up whether or

So about assume I it's at point the would this half time. population of in total

very in So use a requests did we smaller is cases access. treat, why but program relative much before very to there and in is for urgency high the to and we TIO it's XLH devastating population compassionate getting is a many so which severe

Operator

from Our question Wedbush. of next comes Chico Laura

Your line is open.

Laura Chico

there. you that know already remind more to the two just of us, dose program are couple you going achieve instrument patients I a brain. patients to just brain regions UbXa interested consistent with maybe pretty entirety I proportion on your across the thoughts there be the and first the guess I we could the certain to anti-epileptic are wondering marine saw important in if then believe And GTX-XXX, what on model in But drugs? that, of expression currently Angelman related of distribution, the are

Emil Kakkis

variety can a a to the So follow-up net the don't deep probably first say with very know you for DSOs wide UbXa brain is that we the on we monkey, work, a from the on you gene. discussion, I data going done regions the what need to getting all would of know that. I call to tie inducing following, regard they of have conference to in because but do into neural part, transcription and

regional treated So distribution we cell so throughout I'd can is brain the into and separately. know all to effect broad an that. the that is reasons feel regions go not comfortable it whatever rather there growth go get But we deeper action of the uptake for treatment and need we pretty

significant head, asked part, fraction. substantial but is know have I is - off Camille, you that if on a knowledge this it's percent know with top second all it's I fraction, my don't certainly of not seizures. The thoughts? don't any of the I and remember them. number to have Camille

Camille Bedrosian

all Yes. proportion, but Thank pathognomonic not have is Thank an the abnormal I question. know disorder. which if you, Exact patients either, that quite is you Emil. I don't do the for what do know most of EEG,

Operator

Yigal from comes of question Citi. next Nochomovitz Our

Your open. line is

Samantha Semenkow

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

it's of speak you what the other Daiichi in particular wonder specifically platform manufacturing you similar in deals companies makes it's can we I doing and unique first to interested deal, non-exclusive, so space. the in terms scalability in can the in And manufacturing On expect also interesting be others with versus the future?

Emil Kakkis

wouldn't or basic large because do buying the first, thing into that do support deal very to Daiichi, interested another do could reality to with have that's triple that scale. transaction many we we fundamental two. it's in do I in Well, transfer very partner answer but can we the doing important part is tech another deals. second The We hard is are is, they're do which other yes, the

getting be and scaling the create scaling to not liters to will do XXX to fact to essentially XXX, at XXX, continue means go plasma that it and the mix which linear besides get XXXX don't can You reliably XXXX, quite dose the difficult is is cost up.

add clonal are entire whole So basically of and suspension, can create you has take manufacturing cells system and AAV start this we start reliable just into very it production a to healthy and is so cell an machine the are grow because culture and AAV. line infect the the the growing culture adenovirus just small efficient growing a XXXX-liter would doing XXXX-liter HeLa into the which if tank cells the trends HeLa system then fully strong amounts, information production AAV vaccine you're the very fast-growing is guide they and making culture, like growers

stable potentially scale main a the grow HeLa reactor believe side, you the out large scale. just that's the efficiently, after we fact, the scale at and than largest then, And create go for are wouldn't is efficient be scale. exact transaction if which system but So idea could there it's XXXX-liter large we larger, use triple and we disposable could going more more can to that's to right talking healthy the we cells is in XX,XXX-liter doing situation [indiscernible] scale, very be even a colon so about because in the it way

induce be to that cost to without going other true triple be AAV. to being not of grow and plasmids able substantially AAV just for to more reagents these inexpensive material and is the the a make do is simply use large do be a to effective That just transaction cells production. truth more up such to It's in very way anti-virus going efficient, scale that

natural provides functions, right helper higher that's of the flow a reaction of the quality in made the quality begin with very source to with vector out similar product product is that system, to a is with. better situation AAV with adenovirus nano biological which the made the in The the ratio of

more get product. effective a the and you quality scale a cost in of less larger reaction, larger material So

seeing, The is million. are manufacturing get and especially people scale factor So that more are to to think $X cost think programs world key therapy ones start as million some every a we $X AAV or I can gene and needing and more of going to dose. those the commercial the higher become factors take quantity don't because I

do I these how and going have point I And think all think So world some have quality scale diseases of the going and to it. we're we and something at to this. is about manufacturing is think need we to

Samantha Semenkow

that of on the much just all transfer, about completing the And Thank typical can for the timeline you speak detail. you tech is? very for

Emil Kakkis

to them manufacturing a XX-month lines all moving their they're that most Well, us, idea They tech how continued create people for period over that support an period to of to the and an their going basically have we're do and a period transfer convert to a it PCL and once teach there in it's of period, of to use. is is of although go will and own to own them we help Japan. XX-month expected over expected, come five-year The plant their to around. but about course, do Japan, all

Samantha Semenkow

could. just I And more one if

a into on the into paused steroid Phase How the need With second weeks half. dosing up, can while currently that wind prophylactic III patients assuming this before therapy prophylactic you advance you're confident further the method? cohort steroids the the you dose before using you will bit headwind continues can many

Emil Kakkis

this. Well, we parallel be with the will III in doing advancing Phase plan

know and whether but being So are of well I'm what Phase XX it's first patient this steroids, to they've saying a but gives before first we like for probably so, well we in, up in. will UHS working is will able how data. allow we But have us to doing II patient can think kind that's Obviously, we got the is significant say to keep of patients you to kind do enough plan weeks more a timeframe. enough the until to

prophylactic reassure is to no – able be it our I that that steroids said getting and is our and confirmation proposal to go would there What is doing FDA issue plan to is be that happening with way. nothing unusual this just with that there ourselves

at we to As the getting the long safe, least. well, we XX potency whether go that weeks in a data potency sense and weeks XX will timeframe know first get like it's we know collect what looks from as we're probably better

Operator

Kasimov comes Cory of question JPMorgan. from next Our

is line Your open.

Unidentified Analyst

Turner This is on Cory. for

able you timeframe. curious functional working an with frequently endpoints? with, to XXX-day study. of I'm physicians that the to endpoints what how point, Thanks. And your the interactive functional those improvement confidence and the be Angelman just what's to do as improvements from they are meaningful are on demonstrate Just what assessing baseline deem or in

Emil Kakkis

the in but question ahead. I Camille Very the that's good, very know will I frequency high answer should level. a a all But – answer be don't program I'll up point, but changes certainly have this we long go general, question. trials, at your Angelman detailed if in the on Camille endpoint will at clinical on every

Camille Bedrosian

also. developmental and because as domains for month of of large there number Right patients you patients the every thank these across experience are way. a Yes sure, to assessments well, and thank well are the number question along you. abnormalities evaluated And debilitating a

So a are of there assessments. number

they profoundly imagine previously, inability well. what In dysfunction, and terms discussed the as able degrees abnormalities for speak sleep of and being difficulty is that falling, of patients leads most not balance to seizures can meaningful walking as to you as communicate to the varying was

seeing to but what and discuss and learning what able So study, now, be go the in terms this forward, we'll in also human in we described is study of in we're as abnormality right we're meaningful. first this

Emil Kakkis

endpoint as in have of further do that closer expectations what like multi-system is, threshold it's and program endpoints precise complex. I the having through actually this, to depth will you what about get the would more along talking to how All we provide are meaningful but add, we the disorder meaningful. data, mean

that we still to learn, you. Thank to has noted providing analysis as So it. and Camille we look detail as the more and data get need to closer forward the about we interpretation

Operator

Securities. of Bayko JMP question from Liisa Our next comes

open. is line Your

Jon Wolleben

question. on Wolleben taking for Jon for Liisa. Thanks is This the

launch. Just on the XXX

that launch You those there Can doctors potentially and targeting are about kind hearing over metabolic change XXX you're time? how strategy or might at mentioned that discuss looking you're your you that and centers at. how you're first of what

Emil Kakkis

start let and I'll Sure, finish. then Erik

We metabolic familiar with are very centers. the XXX

our MPS. them Cuzan, already GSDX for are program, I've GSDXa OTC program, seen for for them We're program. seeing them seeing Naglazyme. We for [Proterazyme],

simply maybe So are been group we’ve number there Erik clinical trials be talk those to triple let involved we product using lot we've launch with about strategy the know of of there a centers with them, a in but among [indiscernible] But it's and are that I'll center. but will very We a metabolic segment these a seen well. simulators. of key

Erik Harris

thanks, Emil. Yes

are already stated, we the relationships. As a also majority those and is at we those of And and the there at then centers have centers high awareness where around the patients metabolic strong presence strong country XXX. XXX across

also the prescribe in therapy, So, and we our of for be expect transitioning going to those that commercial other patients onto primary focus patients is at some trial. trails didn't launch trial to the those up end XXX

and infrastructure team launch support XX to to small our incremental the efficient the to between five we'll So relatively current somewhere with – we be hires. small leverage think able

Operator

Thank you.

next Vincent of from Bernstein. Chen question comes Our

line open. Your is

Unidentified Analyst

could provide disease that Brian It's indication. going Chelation program. this you how any if large a and you also This my Wilson largely just, understanding thinking for about what is I you're patients Vincent. guess I the might controlled on well therapy one speaking after. color is on wondering on Wilson very just disease be was pretty is by

population there? guess I a about wondering you're just I'm patient target So thinking how

Emil Kakkis

stage patients right at that Yes programs that there. on clinical thank if We the submit they for on now the reference ill clearly that data right is have later. you. are stable drug. ASGTC clinical population data have significant, the at strategy being are who patients and and want not studied out relatively we be Wilson look will are you It early still The a in we coming There their very, that will at need Wilson some you patient establish first will study now expand have significant design clearly to symptoms. the patient safety because we then acutely the

call at regulatory with out when may put a for their not that really you yet therapy progression on or gene key are having because Wilson haven't symptoms additional clearly, the those focus But on haven't substantial disease be are doing and having leaders quite is but discussed need We the well some XX,XXX authorities. of still we may look there fraction patients Chelation. still details plus in despite

will So there fraction and focus in patients a of is I ultimately think on program the there significant those.

Operator

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

Danielle Keatley

and at Thank ir@ultragenyx.com. you will us for by have you and this soon available Thank questions, contact replay concludes our call additional be if for please today. phone or A joining you us.

Operator

Thank Thank participating. you. for today's this concludes gentlemen, you Ladies conference. and

may disconnect. You all

Ultragenyx Pharmaceutical (RARE) 6 May 202020 Q1 Earnings call transcript