RARE - Earnings call transcript Q1 2018 | Ultragenyx Pharmaceutical Inc.

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Operator

Good day, ladies and gentlemen, and welcome to the First Quarter 2018 Financial Results and Corporate Update Conference Call. [Operator Instructions].

As reminder, this conference is being recorded. I would now like to introduce your host for today's conference, Danielle Keatley.

You may begin.

Danielle Keatley

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

Investors this section. our substantial that and projected in results statements have These that and and With our and I'd Officer; Chief on statement. the release as XX-K you be only press Director Executive of am safe are issued our report annual will on on Form Officer Form risks financial XX, statements this not and of Private identified in call and types investors Senior that are all including our could uncertainties, many involve Dallas, Act to We which Jayson Corporate but the filed be harbor Financial Emil materially the which control. the date XXXX, Relations to Please Reform our of from results, Chief the call Officer. that Communications. Danielle those available will as forward-looking detailing Keatley, was of soon like within Commercial beyond Sharp, of subsequent can statements today Securities at forward-looking include XXXX Litigation views President; that'll XXXX, in website of on our February the on Shani with Investor the limited including remind ultragenyx.com. quarterly our website forward-looking our me XX-Q Chief SEC, periodic note reports I meaning differ of report actual find filed a represent any forward-looking filed Kakkis, provisions

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

Emil Kakkis

and highlight for the Crysvita joining XXXX us. pivotal and Europe. us of thank in been X, Dallas before hypophosphatemia were Shalini On I'll adult I'll Crysvita We feel Jayson and financial and or that our then discussing a broad for X-linked for approved and important and afternoon, an progress. of for To go to good you of on for launch spend results moment in update in importance has first some of of U.S. label, patients other The over year adults. time the of for April approval Danielle, the Thanks, commercialization a receive turning X quarter recent the we label, an treatment key over to to XLH pediatrics everyone, which our for beginning overview the with progress the in the FDA and is pediatric elements age launch. Crysvita and both value have older. pleased

indication First disease. XLH limitations is shown and X simple the data with year will pediatrics disease, the this adults. for older of broad for and with respect in indicated And is fact that important a of is decisions. all, type and This to lifelong or indication statement the we XLH thought modifiers reimbursement severity patients treatment the or text both around statement includes no that all be

and include assets pediatric includes data label pediatric for and phosphorus important improvement of both which also Second, as demonstrating serum well not by the oral rickets growth, expected, it phosphate are patients, disease particularly volume but improvement in therapy. are treated and in

data. achieved a really, clinical FDA's greatly phosphorus never the labeled, healing data these is serum effectively FDA of appreciate before We has result fractures any adults, included and in, showing for again the but strongly data been importance also of the in pseudo-fractures, a recognition significant Third, positive increase means and disease. of which

high now as entering disease announcing of patients supply I'm of patients. support. feedback to signs from only well days continue treated data after network urgent severe start with is we're for quality many underlying We approval, biopsy is active of this Crysvita XLH contains demonstrate study showing hear The just shows patient right over XLH of support The first patients in Crysvita The report end in for well adults severe this going Crysvita Their we pleased Crysvita high in treatment. how in disease approval, but XX,XXX with osteomalacia not to bone in label the and been osteomalacia enthusiasm osteomalacia. that bone approval substantial the our Crysvita has and mineralization in views, data shows pediatric for commercial study is significant their consistent severities among a the the The which has of adults need community. in community on want early a prevalence and with separate XX the video last thank of on effect significant just bone basis caregivers. Facebook and -- and receiving XLH. improvement to healing Lastly, launch as the chain the and post-approval disease was also important adults and week, forms at adult the positive bone since

evidence and X U.S. received the the older of authorization for Crysvita growing European to treatment and disease this of bone for adolescents years approval radiographic with with XLH we in skeletons. age addition conditional of year, earlier In marketing children

booked our a received Kirin, XX% and partner, royalty to in a Europe, Hakko reminder, As net of up we commercialized and Kyowa sales of sales.

Ia risks we if this devastating is option programs, IND disease. hypoglycemic proof-of-concept DTXXXX, based gene in our virus seizures our severe of vector We adeno-associated and with a and type an progress for clear for There this enzyme severe therapy death. treatment therapy make must study the for or can patients. storage the now glycogen for disease deficiency missed, to continue April, in adhere with in these to clearance also that Patients forward with to lead need protocol, a strict our which received and can feeding and a hypoglycemia FDA treatment We addresses GSDIa. gene disease move

started off you III as number we'll report data X can X a progress our and important pipeline, well programs. Phase see, to from As from exciting year, catalysts our gene including continue and of across launches an as studies this our X therapy

study provide will the middle And the to expecting randomized from Phase For The data for III control as also Crysvita expected of active could from Crysvita, we're half XXXX. data vitamin comparing Europe, current and therapies. confirmatory D further Crysvita phosphate the study all we of therapy are second over in support pediatric in study and a XXXX. Phase serve the oral TIO, in believe the II of it benefit for patients in

opinion Committee from or for Turning In Medicinal CHMP Human mid-XXXX. Use MEPSEVII. anticipate for Europe, Products to an in the we

UXXXX movement For make III disorder expect In our filing XXXX. mid-XXXX the of decision in in data on GlutX in with Phase a NDA to the Phase feedback. FAOD after patients indication, II based second FAOD, potential half on DS expect data in we from we a FDA

the cohort been the DTXXXX, patients For treatment AAV the for higher-dose of our have gene already of OTC study. second enrolled in X deficiency, therapy

cohort, this patients choose at to have from we additional this to second to option higher year. DTXXXX same X to we'll move a second cohort data study in have from of the may the expect After half the the dose reviewing dose. data We in or

the With gene our this and from trial of our enroll therapy product treatment the of call XXXX. our IND already of data with the DTXXXX, over manufactured the been study in The first the this patients has financial to I/II to an I'd AAV Shani that, following GSDIa, turn we're expected second first results. quarter provide in clearance, of For of half cohort the overview expecting year. recent for our patient to Phase second

Shalini Sharp

shift and financial with briefly earlier that Emil, you, share afternoon, MEPSEVII first we share Thank per first per for million profitability We of the summarize. of due this review $XX.X quarter to to which Net diluted, million the in a priority net or the $XX.X primarily and XXXX compared the of the per everyone. for a year. will sold press and in quarter of issued or loss of $XXX million update, $X.XX of income first diluted was quarter a which XXXX. good $X.XX basic was January included XXXX basic for voucher, today sale The $X.XX release share, I

$XX.X in incur quarters used million in in the sale, on first to from basis with and expect over not future of PRV XXXX. million losses benefit annual year the years. was and $XX.X coming the quarter XXXX compared in We include does any this operations, quarter first an Cash the of which

from to amortization in restructuring million and stock-based subsidiaries, non-cash quarter, compensation expense which foreign recur. of remeasurement losses some currency not had resulting depreciation significant expected This our $XX.X in of including million is $X.X and foreign we of million, $X.X charges,

proportion expect expenses We to a increase continue to noncash compensation of expect XXXX. expenses stock our similar generally time. to be operating that to over is We

$X.X first the quarter total MEPSEVII revenue, also we offset revenue expenses Bayer Bayer. is Revenue a and modest on the million The sales million operating from from reported includes by of which license revenue, million of generally revenue research primarily XXXX, in includes incur collaboration. $XX.X amount to that from collaboration due of For and UXXXX. our performed work in we in product $X.X with agreement named-patient

research million of costs for and operating including $XX.X quarter of Our expenses $XXX.X were total the first million. development XXXX,

generally we Kyowa development Crysvita with share partner, Hakko cost reminder, XX-XX collaborative As Kirin. a our

Our and to research research will advance as and programs. our we preclinical clinical development translational continue increase cost

consistent of We R&D operating to to generally be XXXX. expect a proportion versus expenses attributable our SG&A with

business therapy balance sheet. and $XXX and expect cash, first relative includes to through in operating in million net MEPSEVII with increase We addition base equivalents raised proceeds quarter XXXX an the ended to on investments our equity the offering. sale some the we expenses $XXX.X due that gene the PRV We of cash the million the to with some modestly for business. increase This

believe sale. XX% the any be in U.S. in the At over be guidance we providing nor represent also for proceeds predict providing on of ago. since a initial PRV is assumptions significant receiving We to any Crysvita ultimately of on both the how launched guidance we will will will potential The opportunity do data impact a progressing Crysvita U.S. will adults. is and MEPSEVII market product We clinical sales financial Crysvita based we that point, and week difficult the it of launch just uptake. strength children this Ultragenyx factors well, anticipate potential various or not

possibility gain the our of call Chief continue Crysvita. to the concludes over I Commercial would will MEPSEVII the This quarters. both both like and turn MEPSEVII now and with my over progress guidance coming evaluate Officer, today, as Dallas, the launch we to remarks Jayson We for providing to revenue with to Crysvita experience discuss and

Jayson Dallas

happy launch The start forms including MEPSEVII. progressing are a that had and update in progress our feel MEPSEVII not well, for good on an to regular estimate patients. be the gives we with disclosing from no a is start newly we I'll new diagnosed patients forms receive to from confidence our are provide able previously numbers, in Though approved of the therapy. coming us world. who forms about in XXX start and patient an Shalini. to Thanks, continue We patient are basis, patients start developed option

With responding approval, now potential our from to look other authorization with turn expanding year. this are Europe we inpatient the Crysvita. in U.S. programs we the the requests efforts I'll forward countries, into later to marketing to and now

field diagnostic with X,XXX patient the numbers Our efforts our with new adults full XX diagnosis confidence continue X,XXX and with continue patient give in liaisons us out Their on to patients children living of XLH. in activities prevalence U.S. in around XLH. focused about force finding

ultra-care and Prior reactions and positive has XX based who the continued in promotion these And noted for pricing active responsible with on Crysvita. the have Our these of to Early about presently commercial uptake approval, we post began are payer our efforts have approval. liaisons optimistic have field we payer assumptions cautiously physicians conversations. been are XLH. offices we begun patients of force managing discussions,

underway, complete. PDLs to sharing great to are the But As value X adults, we community, a incoming to to the coming reflect positive efforts will forward excitement are X that start for and general look from forms will off progress payer and Between discussions the believe that the penetration and these and on launch ultimately do we feedback to Crysvita. expect in details the take reimbursement reminder, patients XLH of both months true new amongst the find, the quarters. a additional we continue start the children

Emil Kakkis

Jayson. you, Thank

and important MEPSEVII. two recent can Crysvita are for transformation a The and We stage year thought on know We critical with an and you approach of they we development intensely see, once. As to need. a a assure and transition only company tremendous assure the deep this happens is we to from these care made this focused amount extremely therapies transition the receive a approvals ultimately, commercial execution, progress stage the launch of launches. with successful and commercial patients

we're study clinical lead III programs, In year, addition X our data from data year movement around activities, end throughout I and disorders you clarity the have in our advancing of the to deficiency updating on regulatory look By our programs. therapy translational progress. UXXXX GlutX launch the to gene Phase our we expect for our the UXXXX and for passage syndrome forward to FAOD. clinical

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

Operator

Instructions]. [Operator

Schmidt from is question first from Company. Cowen and Our Eric

Eric Schmidt

phenotype so patients? different benefits launch? to so the they surprised about that adopting on reimbursement Jayson to And you to mentioned early or have them you months. good for therapy type I up these either bad? in Anything What's guess, in days launch. do to these get expect allows X the What of, the you of or early again that soon Crysvita that then take Maybe insurance

Jayson Dallas

Yes. Eric. Thank you,

So has coverage. the patient we've identified groups that population broad in our pretty insurance

So we're private of bunch some Medicaid Medicare bunch patients of and seeing payers. patients, a a

takes we discussions, that we're to spent time. get can what P&T speaking said, launch, course, I just to lot them As the advance of payers committee we simply of of a the time to have but in doing and

then patients running to programs We the get of drug insurance approving provide bridge form free gap once the between their for and the therapy on to and we physician the patients start approved reimbursement to X the are being that. go by

takes X But normal So getting early the we're drug for we're develop programs. going policies. That's by to launch. using just the through why of some payers time patients free on processes those it

in far We and what days, early have we've the seen so a few policies seen. with come happy we're out very

We great. in a early very seen a label, that's that that's anything very, haven't diversion major and from it's But regard. days

Eric Schmidt

Okay. forms, -- And those of approval start drug, number will of number have metrics of patients providing of reimbursement sort be patients number who company on the like types indicators?

Jayson Dallas

course the able Eric, kind of couple next be I of think, over the of that start to quarters, providing we'll information.

Operator

Evercore Our ISI. next question Breazzano Steven is from from

Steven Breazzano

And expect in the the the efficacy gene cohort, the on do think therapy, some based first where you GSDI for you do alpha On dose curve are dose see preclinical program, on you to these patients? of models?

Emil Kakkis

although because efficacy. dose first-in-man Well, the range we do think low the we a situation, could see want gene of always potential a to of for a at type did that one-and-done because experiment. we it's is potentially low have it's efficacy, But end therapy had

as know see range as normal think low might translation sufficient animal we from impact. the we from that be something and on at other what So patient we an have of of perhaps sources lower based dose the -- the GSDI from this end the that to could effect. models, data And an X%

expect see So impact. could we we an do

to we optimal what get the But second effective in expectation may we optimal lower to to go to the cohort our for or was dosing to of was animal is range it However, end models. the the need get impact. on based higher think the dose,

Operator

from Cory question is next from Kasimov JPMorgan. Our

Cory Kasimov

started only it's weeks. at been or are I'm and about product one's of have also in of and how point reps But headwinds preliminary what outside talk what's aware curious stage physicians the in as with gotten discussed? on a see maybe I the approval to your kind recognizing the first payer the you of And typical have main Crysvita guess, few there I be already level follow-up. do that you hurdles this dealing like. tailwinds one this or it at you'll the is and awareness label the And

Emil Kakkis

let one. Jayson that I'll answer

Jayson Dallas

Yes.

weeks, see endocrinology they're come adult in we forms of treat offices encouraging always very bunch actually start I these tomorrow, disease first as other see. result is offices bone of many presenting the the the X from it kinds for We Cory, early They're we've that we we've days in so remembering, the patients who think, of So the pretty endocrinology being said an specialties been they're that and not and in pediatric disease. that's very however, metabolic launch. in, of starting patients offices the the a disease very, the -- only in offers days, the of are who additional bone expect getting to diseases, in in high, their adult awareness complications that And that are is presenting that so complexity metabolic for up. and in picking the approved physicians said, population early That the treat for to us docs. that

a of in of so got get treaters we metabolic work to that little on getting that work bit got referred And of terms into therapy. can has since approval. do extra we've so offices folks And those them they started bone disease the

but we're that treated So it's for get disease. patients very early again, this by the to days that, desire have for encouraged

Cory Kasimov

And That's bit FAOD is, it all? is data as the Okay. little confidence how basis is then collection Phase file your now, going is early potential helpful. and this if perhaps through speculative, follow-up process a I the question overall II patient-by-patient in a recognize on at that but you're the and to dataset evolving

Emil Kakkis

had the -- of Well, and were the certainly improvement majority on or in symptoms feedback improved in sense a large to from a symptoms there and major in improvement doctor's We also that opinion, gotten they clinical improvement. events either UXXXX. majority had the a based attribute clinical and patients, on was certainly base always case-by-case doctor. We we've each if the see

a think consistency so they respond, of patients we And has make at FDA by our still response. them. have this that certainly, their but and We it fact do we're what don't look the real own it to I UXXXX to all feel going So what result. certainly, confirms these patients expectation them. the not hospital, our -- we the of and the overall, supports I following there. better plan and to by and mid-year changes a that to still we're, see get to to the hospitalization that is case. and the until so with We'll discussion But that's we already believe put Cory, that we It show provide did, them physician encouraged take think, a top effect. is that, encouraged reducing making days data at in still determination, largely, line certainly to have

Operator

Our is of Ahmad from Tazeen next from America. question Bank

Tazeen Ahmad

on is Mine GlutX.

So you've to you the disorder be of would coming the half successful 'XX. What III movement result a study got Phase second from data consider up in trial there?

Emil Kakkis

in so remember we wouldn't effect We heterogeneous profound look they a let's group X of the had controlled always in necessarily found expect in a not that in there's managed carefully the -- heterogeneity to like because Well, neurology, Phase event, randomized it's neurology. were reduction X in setting. be is disorder patients, very now II by a study. physician, same We of study, XX% that back in movement which the lot quite it had a something what to patients in

respond. fraction would of the expect So would we what see would patients we'd some to be like thereby

even want XX% some result. threshold. a particularly significant XX% a a very We I might a would We statistically result still see who less to would have mix patients have to haven't reduction I it patients and be overall be would would meaningful, nice who set and certainly since of say expect result,

at if some XX% to in expect And have like XX% but would I a So of -- look studies you would some reduction a study. seizures, to look seizure just I'd response, look this in all seizure see at good really a a you at heterogeneity lot effect. So right? show overall patients

and that's would of the kind Some it look. patients have way effect, no

way and how patients of looking of too see might get meaningful at with wouldn't data mean be its value syndrome. I clinically patients good GlutX would the I'd So up really another for have terms look the hung in many effect, on deficiency effect.

Tazeen Ahmad

a a Okay. follow-up the your or that are for then entire that, And population you to expecting subset? GlutX applicable would across drug be

Emil Kakkis

but -- at That's I above most that. that GlutX criteria, be looking effect who We're that other patients we're things school the -- movement we're at majority Well, truth we're have inclusion disorder, where there, disorders we're are looking movement to think focused age from to the talking distinguished. some by in particular so the on speaking is and seems degree. of The have that of phenomenon. not patients the

require fraction problems, disorder those who So substantial the population and the would But study patients. seizures look good patients be able we point, that patients would it's looking include well those it earlier have with were a movement would pretty we disorders. see effect enroll to movement I not indicated which of at we this we But did at have seizures show point. think specifically, doesn't only, and the at for would more -- for this it think

Operator

question next Raycroft from from Jefferies. is Maury Our

Maurice Raycroft

question First on XXX. is

So that to noticed -- hypoglycemic clinical to was event measures. shows was of first is time secondary posted trial ClinicalTrials.gov. outcome it the one we posted the it And to

would the approval. if endpoint for wondering eventual be Just would use you FDA this

Emil Kakkis

the that as extreme it to endpoint relevance because clinical has considering We population. are key this

happen. go that disease One patients middle who during there this of of even the die, of the hypoglycemic are can that is severe the have dangers night and that well and great they controlled are

relevance able that's I And we without a it and hypoglycemia, them one going being don't its so because I of to think And tolerate through clinically think think importance. drop benefit. of going be understands the FDA night the to a a to to is hard having convince meaningful glucose

the probably be is might that what primary. So

injury therefore, the look the see course, the look about at. we hypoglycemia patients, of injury in is look efficacy. potentially why the thing and of driver things and that the to it's metabolic -- that at -- secondary of other but be some another term what all will storage because liver so would, that's signs would the part liver We We and biggest of kills effects, how complications, disease liver probably other the causes long

Maurice Raycroft

next week XXX, should great. for then any for be we the ASGCT expect for that update on comments And what would update

Emil Kakkis

shouldn't still going in about first cohort. I think are be cohort, be to -- the progress. anything the presented second is data which There

Maurice Raycroft

it. Got

follow-up just probably. then So additional

Emil Kakkis

Yes.

Operator

from question is Adam from next Walsh Stifel. Our

Adam Walsh

more First you one patient then, that into difficult from Crysvita, your start the would us that, going on And you to peds identify there been possible, and kind the the and in of post requests convert I getting and in some for adults onto that talked noticed you you're terms comments about approval breakdown launch and pediatrics, requests? adults both patient in early surprised requests? population start be and this talk of drug. Are harder, launch, had start maybe give the the adult if adult could

Jayson Dallas

Yes.

diagnosis are but having I adult this adults who So that's definitive of for of and have with population that complications. overall always of for population endocrinologists the the the who the rather said of not perhaps adult the seeing centers a a of we're we're being some diagnosis adult think bulk a by and metabolic don't there surprised a XLH, population XLH are the early. requests managed that treated come proportion bone of in We

diagnosis patients and have this is sitting, therapy. for been who the group of waiting there have So

and And adults broader So start referral but to we the a more bit even challenging adult get we population expect would into see that longer to as that do. to of pediatric days, come in in, will to numbers patients the bit patterns happen. us and equal those get trying little take a numbers gets we as early roughly or

Adam Walsh

I UXXXX in and correctly was were in between this differences follow-up. Can looked differences, there then if the study disorder Emil, that X you I the study, our refresh are one any, memory the III. proof-of-concept endpoint some ago, a the recall if then just on what at studies? III Phase In the and endpoint in the was while And just Phase Understood. movement -- the minor

Emil Kakkis

movement they proof-of-concept disabling disabling with for disorders, living option you more disabling We activities the the was symptoms. as look we type. movement we our at is the request all one things. looked interfere any study, the a we with is of the offered the whatever program, In as were it broader That the overall. at was to that include daily and when discussions same FDA, the range disorders, a in ones of that that restricted doing the reduction opposed of set restricted data analysis, the or but proof-of-concept of In

observing. don't of but changed we So that effect just the think change the -- at, we're degree doesn't we it looking of number events it were

more So disabling than not we're all disorder. possible comfortable with the of limited set movement disorder -- movement rather

Operator

question Laura from Chico next Our is Raymond James. from

Laura Chico

X just program. the out on the actually there for that therapy opportunity, mouse. an in question feasible think wondering if is a a just a represent I'm at DS and in One publication AAV-based product. But describing whether There's interesting disorder that role this more you're as GlutX you DS came knock-out an gene gene therapy It's data looking just approach. curious, might preclinical

Emil Kakkis

it. we gene looked -- at that certainly now technology, a we we've have Well, certainly therapy

that the wherever deficiency a barrier I is mind, GlutX and less deficiency normal, blood-brain the of is. syndrome, transporter, GlutX classic syndrome, other in type. a GlutX say, you it's transporter because gives places That that's complicated defect in there's you it's also XX% gives disease XX% abnormal, whereas classic the non-classical type, dominant. severe severe autosomal GlutX is Keep think in in but a

in have close impact endothelium. really to get GlutX, big So means order barrier that blood-brain to that the like to need you on a problem the with XXX% on

the the few in cells a barrier You of expressing. can't blood-brain percent just have

doing high amount. You need a high to have a fraction

clinical significant go. in to the has blood-brain a complicating would body is occur. the feel to there a So just be barrier. this it's places It's challenge. way I Plus, where other like uptake not

gene brain, there's trying a easier to the more membranes we the be more small of lot Of potentially think across membranes neuronal and which can across expedient to superior right be therapy way to course, diffuse of a this will and and point, many apply we apply. to At a blood-brain energy things. to comprehensive that substrate would people the that molecule barrier think distribute

Laura Chico

sorry And I I'm one On follow-up. just missed DTXXXX, if this.

you have update second an kind of still the on progressing the the of an for do IND 'XX? milestones half curious, planning towards Just Are on in program? you

Emil Kakkis

Yes. general, we're been well, we'll to we get described. IND filed with just do what and them not is on In progressing with to new But as as get we announce we are did track in progress, they've when GSDI. DTXXXX, they the play when we'll that cleared in

Operator

Raymond from with question Our is Piper Jaffray. next Chris

Christopher Raymond

I the with Crysvita. dosing just want to dynamics understand

to but is there. step-up With I the as And there's guys of And go with you XX-milligram calling as increments, and in price Shalini, have weight-based a I out, step dosing big guess. to for XX decent have example, drug between round sort patient. know XX.X-kilogram the cost in, you the I a variability to from XX-kilogram do of bit milligram, coming a is for a there's net in we XX for guided amount a of function, guess, nearest peds there's this treatment the to peds label adults. said obviously, and patient, I -- in a a a But guys to works weight in math, that for It know, I XX, dose.

you So $XX,XXX. a I'm the by you up step of peds, I as weight, we curious, about as guess, $XXX,XXX for guided look see that function at to could just kind have

variability? should maybe built-in that XX% is a much to plus guess, like discount you of sizable talk Or be there can I we just is So here? thinking about that how

Emil Kakkis

Chris. Sure,

trial of basically in therefore, be point is is average It's of step be so range and give range. you to average middle But Yes, price that milligrams there -- two the XX their the also step data out, so I $XXX,XXX. steps. patients cost the to an and I XX, piece. of we XX per XX put to those think at we phosphate We doctors function and in the of between were would would that the there in going would you to going up a looking normal XX kind get the out kind in is the the step, are took those that doses. trying to low and of in what and XX the an the or give averaged be like we and

And the If see rough their may a that to dosing with and goal you average, so low, and that was have up including kid's $XXX,XXX, bit you real-world there need as little an may give what in increase they're does be and estimate they'll the look a be. whether adjustment number doing. The they or of gross-to-net the on a step you for to giving you you your are practical of the result what to kind well at kind average place, compliance of step a the But how of each of dose. up certain go individual more a an basis, cost. questions starting would their is give not, kid calculation lot and about

Christopher Raymond

especially of see step-up in conclusion -- the And in next as then get a to is so should on, you et go goes heavier logical these if, by, maybe kids just corresponding I revenue peds, if follow time the years per getting are cetera, patient. sort guess, up,

Emil Kakkis

would would as dose they expectation our That be grow. that increasing be they the

Operator

Partners. Ha is from Leerink Our Dae from question Gon next

Dae Gon Helen Ashton

So one follow-up and or a quick Jayson Emil. one for then

are market, to Brazilian rare us your you -- some there? approaching the talk access estate this contemplate launching about of physicians follow-up drug, metabolic there. Can regards guys to nephrologists? Crysvita and to this the so can of extent and versus with First specialties begin administering And is, your Crysvita bone IP specifically, regards about the to their is you to disease you and geneticists, launch, and specialists you of with talk -- process endocrinologists then as the a medicines different how

Emil Kakkis

take I'll let Jayson that.

Jayson Dallas

and of first office. Crysvita our regards with all, that treated will get patients the So endocrinology majority to prescribing, an anticipation is of in vast

the treating patients for for patients being majority That's the have patients history treating comfortable disease. maybe we patients centers specialties, for complications what to adult the clinical in other kind see nephrologists some the the to do with the or specialties disease. docs So geneticists bone identify of that endocrine clear some them that endocrinologists, metabolic disease, expectation. specific pediatric them and bone a moment. at not But them are disease. population, treating They in managed in expect those them their endocrinologists are in be for we and we're metabolic be bone underlying as want the these We refer disease. population, And that That's would terms it's relatively of do seeing who metabolic pediatric the not being managing treated feel to would other do own. to a of the see the adult of expect various on complications our into vast interest of the do managed presently get in the where for

that's So have, that's it to that unfold. we and expect the we how plan

specific that have In responding question U.S., your the start approval we Brazil. we requests in able to in Brazil, named-patient are now for terms around to of therapies

actually MEPSEVII in filed have We Brazil.

anticipate other will is more be countries, there'll Latin America, and IP estate, that. we very around for be and no be So in filing question these approval. that complex about we the will to going we And IP in say then obviously,

Operator

question from from Arlinda Genuity. next Canaccord Our Lee is

Arlinda Lee

I I expanded to after the the for program. dataset, you one had had guess, on I'm initial some for go on whether the that wondering cohort you And follow-up that. guys to a second looking in XXX -- are maybe the be next Shalini should or in or since dose forward already then level. not decide efficacy what one

Emil Kakkis

Okay.

go obviously may cohort we we're dose forward consistency safety. give what patients it across in that's limited, patients, next better to level got We question? kind cohort, sample or we'd to as all looking the of that we of around depend get the to a for. it looking the level. would degree We of but patients. a X as second well like whether see only second XXX% of what that the DTXXXX, but XXX. what to kind With how the many patients And see takes with the a that's effect normal the looking little of we're on For normal makes reconsider to reliably But to X, of of X. be think a effect, those that -- And in ureagenesis Cohort ureagenesis we're looking patients to for X for,

Arlinda Lee

this do fluctuate On the you bit R&D cost, little think a more? good for a Or maybe to this is forward? might Shalini, base go

Shalini Sharp

this and expenses. to Thanks, what year we've XXXX, operating for Arlinda. of that XXXX, we so is proportion said in similar operating expect it XX% be was R&D expenses a approximately as of to

at the any guidance of quarter-to-quarter, a So for full doing We're you sense. year, can kind use but from gives you hopefully, that least guidance. not that

Operator

is Our JMP next Bayko question Securities. from from Liisa

Liisa Bayko

Just thinking drug, of numbers maybe with with then too, your respect kind to learning sort patient you're about market you're helpful. speak MPS of there? this about X, be now you out of you're to what how to and And in terms learning can many of would sort out as what going comments gross and of any on patients there net, are

Emil Kakkis

be some are everyone. if diagnosed. are be we Well, we who off, that I there X patients for found always think And not reason or had MPS a get number then the couple might fall assumption originally and there would patients hundred but -- diagnosed expect lost identified, is more properly that there you patients the than that

start which drug's a of We we'll patients now the during diagnosed and new it's some few patients. seeing think that start do that's as pick time. have diagnosed that possible getting available, be in starts were People oddity curiosity. get start seen running just patients older this interval, MPS more an to We pattern seen it the it. to all up would become to before it over or opposed now attention. I've launches It paying important Labs

of seeing more that we're So now.

and two that not then situation, could might XXs significant wasn't the also be in the where might diagnosis be intermediate bone young or their are areas happen sometimes patients in I where milder have think in currently. they that growth disease hydropic teens the the made babies' always where diagnosed

So probably area, we diagnosed doesn't that we're number. say overall don't and it's we It some couple fair starting We that Right that's new hundred. expect evidence a see to around patients different. prediction to any our still to change have in think that now continue.

Liisa Bayko

as And Like, many today? are how today, many start? a approximate guess, do I you how identified

Emil Kakkis

Yes.

identified because we out it and also -- really we you constantly changing, information, always deduplicate. We haven't because put haven't can't it's can't carry

So number That it's where and I why everyone will found. projection has That's have if as we don't our commercializing. find to have, say been that's in the case. of XXX were kind the we that. we're never areas a we

Operator

from Wang Our next Barclays. question Gena is from

Gena Wang

in you key for movement trend? disabling any disorder. the secondary if primary movement to frequency you can us hitting placebo And assumption addition remind positive also drug approval, have and endpoint, endpoints show events Wondering for arm? of for the one Just on to

Emil Kakkis

Okay.

around like doing The out to that really specifics So haven't movement few. put having the say into It's What requirement to study. disorder. XX period. X-month a in I events the we though during X there the more can only the get period. you We're pilot is a around X-week study, were is

think the majority see. patients for a you seeing pilot disabling. are really. in kind reasonable disabling the And estimate that of more with those what We so of are might many of were of is of than I range, that a study Majority though lot around kind XX events

helpful So you. that's hopefully, to

Gena Wang

endpoints, to trend? have to you primary approval, secondary any positive Yes. addition And also for the endpoints in specific show

Emil Kakkis

primary. have at one. the we And look on We what the to you any endpoint it core data secondary is the to what requirements more the -- of a how the authority, primary is it's course, picked they're meaningful to think we would determine specific kind this point any a don't I there end you endpoint. secondary with of because in requirement primary point, to for clinical able may endpoints, you need particular going data be secondary study at to actual of but so no discuss regulatory though, and We the is get, compelling single for specific day, a see this like as have at you The It's but to have it there's requirement, but thing. data -- main I say the any the is, file. don't look supported regardless what pivotal -- of a more

Operator

Bernstein. from Chen Vincent from is question next Our

Vincent Chen

response sense while did rise what's patient is On two well, overall, late rather X if gene the be a and response the makes might -- three DTXXXX week with treated you likely and patient patients why rise? sustained for to not. XX relative therapy, robust And to your dose robust the real showed patients you what over XXX% late in a so? the in seen explain makes not? think What so, What actually X effect? And

Emil Kakkis

talked is before but we've for, patient largest not of kilos. the AAV reason distinctive the vector XXX patient the about that thing for Well, the we X proven dose reason one because one the the have was received total

size he almost X. So of twice patient the was

that -- is is there factor. one So

and why haven't so or of is. out in that liver other And seemed level. got enzymes I on were some point initially. the criteria when rose lot They came that bounce strong we're response, explain of the the then steroids that their above it's there off, also some it that looked to patient to With other pretty back. on, immunological there don't of regard. And I their like any just patient-to-patient very is put variability, -- regard response the think there to therapy steroids because was a was depression threshold would We seen -- gene steroids clear unusual when for programs, that

it back back to XXX% was shooting the suppressed that So have then the that the -- bring X- where back the range, and were at the it liver's and XXs it because it or down was to steroids to may that in the genomes removal our came impression steroids right something of now hit that it present XXX% of or then or is activity went released use In XX-week up. and expression of range.

saw actually that applied. So similar weeks were was steroids early on XX what was what at before to think we you

one a seeing be But think now has can't steroid the maintained substantial, so right be and we patient could it's event it during say -- sure. doing transitional well. of let's a the -- and we well, that. causing But one that's you're is seems that experience is effect So been do now treatment

Operator

question next Our Yigal from from Citi. Nochomovitz is

Yang Huang

on Yang Yigal. is This for

quick just one. So

you you UXXXX. and MEPSEVII has $X.X much Your revenue how MEPSEVII guiding from is where press revenue million from release XXX? from product and And say Can then UXXXX? in for are how much

Emil Kakkis

ahead, Go Shani.

Shalini Sharp

question. the Thank you for

the So that you amount named-patient is Hopefully, the a ex-U.S. revenue small to attributable named-patient to very sales. MEPSEVII, can vast is sales of for UXXXX and is tell And I that's majority attributable helpful. UXXXX. revenue

Operator

from Our Morgan for from is Harrison next question Stanley. Matthew

Ishmael Asante

This is Ishmael for Matthew. on

could the off-label usage additional So on some on if one supports on access also in the you TIO and and with data on data the opportunity therapeutic really XXX. Can how details of likelihood -- Crysvita drive us the XXX. market remind and Could the range? within are you opportunity talk the on other within preclinical to especially good dosage more about the you the are evaluating context

Emil Kakkis

Okay.

So improvements and that's we for is quality to did relatively which bone only It's saw symptoms. maybe through in have point. certainly we're TIO, and as biopsy tumors. TIO well thousand data having weeks, what bone which with out, not additional data opportunity. the at we're a put what ready, certainly, we've TIO looking this XX we and at couple data similar osteomalacia half we say is have and about phosphate but is TIO The The patients, TIO for not most, smaller is non-resectable label, a certainly, TIO promoting at opportunity as some

so looking the in XX,XXX we're it's patient, XLH. it's fraction range X,XXX relatively of small a So the total of for

the to have -- to a the data BLA talk we patients both the model on supplemental were to data about it agency with for additional those XXX, would mouse With collect to is product. take mice regard in expectation a probably Our Mouse model. studied of what achieve of expression the file in euglycemia. models, preclinical but dose glycogen detail the model in a both in provides of the exact a able was improvement of the dog in on small more dog vector, amount terms to the significant particularly and and storage relatively and

upper to that been of We presented, end mouse that, XeXX the going deep we the titration think middle rather XX, range the the at through have the study, some than range. -- XX do already the the range of starting to range, we within in encompasses to data's of the expect which that but we're the that of

in right and mouse think, the what which we could the that we lower elsewhere. where we dose we mouse, are in end, increasing based efficacy. is the first think given is the what's to to and translating it range compared really expect we're So higher should I of would the on XeXX, go said, to human, That see need seen the middle at dose been

that's hopefully, So helpful.

Operator

questions. to to Danielle showing for I'm would the call over At I no remarks. further this Keatley turn time, closing like back

Danielle Keatley

shortly. this concludes and our call A available today. you, replay will Thank be

have us. additional please at you you us for or phone contact joining Thank If ir@ultragenyx.com. questions, by

Operator

concludes Ladies in participation you for your and thank today's gentlemen, program. conference. This the

disconnect. now may You

Ultragenyx Pharmaceutical (RARE) 7 May 182018 Q1 Earnings call transcript