TVTX Travere Therapeutics

Chris Cline Vice President, Investor Relations and Corporate Communications
Eric Dube President and Chief Executive Officer
Noah Rosenberg Chief Medical Officer
Laura Clague Chief Financial Officer
William Rote Senior Vice President, Research and Development
Greg Harrison Barclays Bank PLC
Tim Lugo William Blair & Co.
Maury Raycroft Jefferies Group LLC
Michelle Gilson Canaccord Genuity Group Inc.
Christopher Marai Nomura Securities Co., Ltd.
Liisa Bayko JMP Securities LLC
Joseph Schwartz SVB Leerink LLC
Call transcript
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Good day, ladies and gentlemen, and welcome to the Retrophin First Quarter 2019 Financial Results and Corporate Update Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] As a reminder, this call is being recorded. I would now like to turn the call over to Chris Cline.

begin. may You

Chris Cline

Officer, Q&A Michelle. First you, Call. Chief everyone, welcome Dr. Chief Development and call Vice Senior be the Eric Great. and by joined XXXX Good Financial Retrophin's Executive our us President by Research Quarter be Update and Noah our Eric Results also to Dube. will will Thank led afternoon, Dr. and our Laura be Officer, for Today's our Financial Medical Bill the for Rote, Officer, will joining Clague, prepared Rosenberg; Chief session. remarks. Dr. of Corporate

begin, Private to that made are we Litigation XXXX. of Securities Harbor statements everyone matters during facts historical that Safe are forward-looking Reform Act Before statements I'd like not within regarding the this of call the remind provisions

not the see implied in Forward-looking actual our on today, may with the that performance. materially section Factors from Please was are of the Risk and as XX-Q, known those well statement. XX-K earlier that achievements differ They cause release forward-looking the uncertainties Forms risks, and filed to issued by the guarantees that statements press as company's disclaimer SEC. statement or are unknown assumptions results, and performance involve expressed

that, statements In such date to addition, as obligation reflect Retrophin me And Eric. statements information, made, turn let views May Eric? XXXX. now over update of to our future such or only any forward-looking represent the X, are the any With circumstances. statements specifically to call events disclaims

Eric Dube

everyone. Thank you, and afternoon, good Chris,

diseases. to people advance need with We rare for pipeline our unmet significant to continue address living

this of late-stage to positioned reach programs. the in the year next has execution Our milestones our key quarter each for us first

quarter remains for fosmetpantotenate this from treatment of of top-line on third year. Study FORT lead program readout for in the the Our track evaluating the pivotal a PKAN

dedication their further first from We've bring with We if great awaiting have in families We for the leaders. seen neurodegenerative the this patients commitment. and they work rare disease. efforts global commercial launch. are to this we the the potential our continued results, progress filings, living with the as preparation U.S. treatment and thankful our European product we a our FORT are and commercial during approved Study for medical as guiding positive eagerly both strengthened exciting team And fosmetpantotenate to time which well of participation and patients are as addition and

the potential the half pivotal our the remains of continues And Overall, therapy which IgA upon to during for year. sparsentan is of is for the nephropathy. the expect with diseases momentum XXXX. We first-in-class we FSGS, many site to as efforts and to we enthusiasm also for and progress our X rare to build sparsentan on continue come pleased represent will in X patients DUPLEX minute. two with on a will engagement in as to sparsentan's foundation a FSGS new standard strong year. two the bit But kidney positive the end throughout be the second track activation a Together, opportunity community. the these a programs going we an sparsentan potential for we our Investigator treatment and IgA Europe. U.S. are more impact program last and Phase plan. PROTECT at make FSGS within Study Phase about treatment have numbers XXX,XXX for us with the nephropathy. optimistic starting high evaluate Noah in make to disorders, second this to paradigm further remain similar for nephrology the build talk about deliver IgA our to after building The Study to In to readout, potentially nephropathy

inflection potential collaboration. a nearing also are with We CNSA-XXX point the

deliver of will months. progress PKU. in Censa the couple decision Phase we CNSA-XXX data remains the dataset. quarter And our option on with third is patients the X make pleased the Censa study in that call, an are of outcome. making. with the to completed at year. to decision on conducting evaluating assessment option, team last in randomization program And proof-of-concept evaluating track come thus, full expected the to We order Since this have next In the from understand top-line in our the of on looking is they the we been our to We the forward be

acquire. the to to the allow the data to plan on us outlook of as input be board to We assessing the hosting advisory align leading landscape. to X with we Phase have well the of That exercise expect all our competitive you Phase event the public. Study about been including at disclosure leverage decision context and Censa make will time refine know our in We also in input, an our of regulatory interactions, experts available give our worked during field the planned from would when of option This our in of to to quarter. us the of you into be will of as FDA asking I recently results the decision takes the we event in participating allow the to made the needs third account the exercise. collaboration X many full the to

Lastly for success. on are we this planning program,

came option. organic of to CNSA-XXX continues benefit consecutive we from performance business a the working continues first programs, patients indications pipeline move which with have last slightly partner base if cystinuria. impact nature patient team Thiola our Our make trigger the and new forward, are the marked appropriate the starts resources bile year-over-year to growth. another the these teams Outside period quickly we uneven treatment. in The acid of to and ensure the quarter to to our And ultra with field be same initiate portfolio positive patients and is continues the period in below to with in diagnosed which rare attributable receive steady. year,

gross coming As expectations resets to anticipate line back is experience insurance first quarter, moving normally business, discounts the in in by we we shift This Year. the driven into and New expected net in in with higher our was our the quarter year over with and projection full seen April the of have second Notably, past. in years we month to we started on remain a meet XXXX. levels we our strong for quarter growth normal track as

We are formulation on date also XX. new preparing the upcoming for PDUFA June the for of Thiola

to are Thiola and have for ensure and to to fortunate community experienced the been quarter. are raise positioned seamless years. has third the potentially educate look launch the access the in when well working opportunity cystinuria awareness We experience a last team that very the They improve X of with the to we

turn Noah Noah? a to now over call clinical me Let the update. for

Noah Rosenberg

our Thank program you, the our of and to which have strong start team's Eric, led a and good afternoon. I'm advancement three proud of the all year. of pivotal efforts,

our lead progress Study program for treatment PKAN X advances. Our fosmetpantotenate the as Phase make to FORT of solid continues

complete you As of December. of recall, will many enrollment in FORT

safety at top-line study majority quarter. the importantly, track third completed into the and readout XX-week trial. and remaining are So Eric in the a conduct with with there open-label data We in efficacy period mentioned, Most pleased have patients as point, of this progressing on double-blind a the the of patients remain deliver the pre-specified XX-week study. of and portion in

time. our with our readout. continue In be ensure submit and team validation add regulatory and high NDA to community do to team And the sharing PKAN a XXXX. MAA parallel, to package ensure, continues applications that that development to position prepare our in look results of the and forward all have to Our CRO we to necessary work biometrics you quality we'll a

want leaders to lack to the DUET X awareness sparsentan, possible. nephrology continues key We recently Phase to I continue ongoing Given meeting at of pleased and had trial as raising with to with significant able opportunity Nephrology current from the Australia. patients of the open-label and of make in the our PKAN. as rise be the position over well a in positive we're how for and unmet within will extension. Congress enthusiasm shift available awareness fosmetpantotenate quickly broadly Melbourne, with treatment We need the We results program now engage as to our Study were World the opinion of options living community. to as

IgA on of of the the to and DUET the move encouraging. and our therapy partial resulted readout online and safety remission showed look sustained interplanetary a in effect in showed year, appear impact have datasets during discussions of are a the over remains the weeks to Study pivotal FSGS evaluating sites leaders with meeting. in dual a and Study remain of balance from execution with from for the achieved focused initial many It able to there have I quarter. FSGS off the getting Study our pivotal programs. treatment was balance the PROTECT XX inflection of the to meaningful and for As through of continues period. meeting key approach thought patients the With an FSGS the at looking extension number pivotal enthusiasm three of nephropathy of blockade, track and DUPLEX recall, antiproteinuric We support of RAAS the we'll antagonism advance with inhibition progressive as that assessment leverage the treatment with other measures sharing to you forward half positive points sparsentan IgA teams also with utility nephropathy. to who more presented at milestones readout top-line data and the and endothelin The you, to advance been the may immunosuppressive in DUPLEX in X with second on FORT coming and trends one-on-one potential and sparsentan ongoing third Along efficacy footprint we sentiment be We've benefit running. the the Phase patients, like sparsentan and initiated increasing near-term top-line Through for open-label nephroprotective each of FSGS proteinuria. newly the the had XXXX. year to growing we're sparsentan of proportion the still on no them quickly in forward the

Laura? over for update. financial now turn me call Let the Laura to

Laura Clague

Thanks, Noah.

in $X is the the Selling, expenses On period the and X were quarter an $XX.X operational over of our support first of significant expenses we expenses and clinical adjusted million Phase of million for reported non-cash quarter, first three of acquired P&L period expenses expenses stock-based basis, and During for quarter $X.X development for we first compensation quarter depreciation to attributable million the increase XXXX same increase sales portfolio for GAAP non-GAAP $X.X in period. our After first adjustments in the the product the XXXX, to non-cash period quarter compensation efforts largely million for an portfolio during commercial including same the $XX.X our discontinued charge for net tax, product non-GAAP in included quarter and $XX.X adjusted R&D the the were net million. a company to XXXX of the increased $XX.X for over non-cash net stock-based the same basis, the for The in of XXXX. On loss grew and non-cash amortization. a million, income administrative We L-UDCA our to increase loss million million. quarter $XX $XX and consistent rights from general the fees. resulted for to of X% of development over first a net L-UDCA and million, adjusting $XX.X reported On $X.X the expenses the program, sparsentan, Relevant a attributable GAAP decision in and quarter. trial. the made legal XXXX. first headcount and quarter, of R&D support were the which XXXX for a expenses to growth SG&A quarter million, is further largely for million basis, our amortization. fosmetpantotenate higher a In first were

cash As our financial our XX, program, to continue pivotal we as XXXX. we million three foundation in advance cash with $XXX.X strong equivalents leverage and March of

to continue from increase the expenses current levels. For we modestly year, the expect to R&D of balance

of we increases as do said anticipate may be higher nature with should scale times. but for advanced expenses we be the as As uneven relatively year continuing stable for SG&A an and we've potential remainder clinical clinic. in readiness, in up material at manufacture modestly for our commercial studies the previously, the minor the there R&D

remarks. now closing turn back Eric Eric? the me Let his call for to

Eric Dube

organization. of and Thank coming the call a years, in ago, talked is a year-end new to pursuit the of to the report strong It evaluation our about be I'm in and months capabilities couple few you, clear in team on in became that our as Laura. our the confident pipeline that there patient and Retrophin a I'm were the pleased to years deliver my key On further of I fosmetpantotenate organization position sparsentan our leader. of order ahead, the assets the maintain to mission across in made. adjustments focus

mentioned, As during focus the the with pipeline. sharpen discontinued will we our our Laura program late-stage it quarter the L-UDCA on that belief advancing

We also launches and success will decision the ahead. allow made maximize the commercial focused reach in of transition a our years to and This to on upcoming to executive shift our many as patients team. the the to with therapies more product our team as new possible currently approved role

in evolve the and dedication leader this for reposition Neil's are a all departing result, continued rare the be last As Operating commitment has community. and the we as of Officer, mission company Chief us end of at support our the a over Neil And our McFarlane, patients to work years. grateful disease this will several and month. leadership, helped

greater growth looking are on growth for remain serve forward a entering offers our this of programs transform and our readout significantly It we period three pivotal are We is that Each next on our community. of near-term fosmetpantotenate rare quarter. number and to we And delivering sparsentan are and disease to ability us create year. center our to opportunity an the the milestones significant these clear of will patients for focused of organization. the to reach value

have for formulation opportunities of the the to cystinuria. the of PKU and the make options With treatment also quarter, improving with on current and the collaboration year third option we patients decision towards anticipated upcoming this in Thiola Censa new strides launch

to as this prepared current of to progress. well later are our we all year. we look We recognize you from milestones value on programs forward these reach And continued updating key our

it turn the up over questions. to call Chris? back Chris, open me Let for to

Chris Cline

can lines Thanks, go please. ahead Q&A, we for the open Eric. Michelle, and


comes [Operator Instructions] Our first question Meacham of Barclays. Geoff from

Your line is open.

Greg Harrison

for With assess CNSA-XXX Hi, asset when to or see reduction the this for minimum taking as us the the color whether Thanks give you be additional of you some is how question. on data? And see this a criteria we Geoff. making our there there going the asset, level on important Greg Harrison could to the as decision? should is thinking is decision regards any acquire bar fee other to you're you in that

Eric Dube

Greg. Yeah, thank you,

then add you So I'll share ask else. I'll to with my and thoughts Bill anything Rote

want the we're primarily in asset. the need clinical meets is to we going And the current this way product that established We in sure would the that essentially, looking that profile of to the do market CNSA-XXX a at So be make there be we deliver believe that on need still to unmet a do unmet market. assets not. able

at we requirements because and by also market, going look be But this to the this going we we're non-clinical pathway at the evolve likely able market certainly is at time, that given particularly is speed it PKU be to be a that So entrants There competitive which to dynamic. currently are new to that regulatory in clinical will bring how would pre-clinical what we looking also what's want looking the think be it or we'll market. profile. over asset phase. are to are; the But

of we that be that over key we addressable for before what a a efficacy are data. decision. about So Bill Those months looking little talk next now, what sure invite and make the the at we make will fee market areas be. will the at looking bit to want And three the understand we'll I'll to we reduction be couple

William Rote

you view program. whole it's said, Certainly, the of a I as holistic mean,

that know hit. need for a we with fee reduction, care. to off I qualitative established we've cut standard which numerical specific the more don't a over existing it's superiority But think view Specifically, clear of I of

those they for to categories, a them you see point non-responders, those And a significantly who weak response. where Those into So be two difference. who are where really a are push patients would see or responders? we'd improved the for you can want become responders,

Greg Harrison

- Thank the dynamic overall know evolving? you and just it's fit of the but could Great. don't if follow about competitive in you'd acquire and you I pricing, decide just in market there, PALYNZIQ with Kuvan kind asset maybe about you. if you if thought to and I how have see up, And, early, how the

Eric Dube

I generic all certainly, Yeah, And Kuvan. will mean, into would trends into this is Greg. But Thanks, that certainly, play again, on we're need. to we want pricing. we that continuing input what for look those not make thoughts would new our think of - delivering be on well the potential look sure like those we've pricing. as at pricing including asset the And unmet we potential importantly, our entrants, thoughts are that on decision. what And as to disclosed an most our specifically primary

Greg Harrison

much. so Thanks Great.


Our next question Blair. comes of William Lugo from Tim

is line Your open.

Tim Lugo

for Thanks the question.

do have And of how the maybe base upcoming the have the was you age follow-up of how population or and you medications? For a open-label the many it enrolled, study? patients into could rolled FORT that ranges portion heterogeneous line speak to sense concomitant Study,

Eric Dube

detail Yeah. Tim. the progress been your we've And to question. so share invite pleased Hey, bit far. both I'll Certainly, Noah on of a more with

Noah Rosenberg

Tim. Hey,

the adults overall, say, as the We are mentioned we're can a the blinded the emphasize terms conduct. one-third a entered study, we, have we I portion in reminder, remain by completed question, to about information. your retention the that and have giving the amount led say seen to two-thirds of we've representative see just of really So the patients demographics, open-label we pediatric. previously of first open-label. and part And open-label pleased blind the specific only We we we're as It's limited study. would the portion of numbers. study the remain double far now majority encouraged population. We But well. as But in of with the

in it's And now making in but instance, XXs. baseline - well. are then, what families, rating finally, tell share in demographics, And that possible. we can't some just patients locked. want published to PKAN to results isn't course, this for And for the of median we final the the fair think of the database can considered I we saw and it's validation and as specifics, the participating similar terms say the consistent study, which to that you other until was PKAN-ADL upper the data the is PKAN-ADL I thank of

of So and like expect that's design. feel able hopefully an right there, be effective I to we've reflective our assumptions show to it And to We initial patients enrolled what fosmet question does answered your do. the Tim. we

Tim Lugo

definitely. can the what maybe Yeah, pool and UPDRS the PKAN And us and you the are between Part II III? differences specific remind

Eric Dube

the Scale. validation really done was II, of UPDRS essentially of living so the were which is that Rating Unified - There XX mentioned Parkinson's with Yeah, Noah Part activities the Disease part lot daily the patients are in are what versus community or symptoms their that those are to understand caregivers. disease. we with that And PKAN the to Parkinson's was that the of study work did hallmarked that specific a

that's that changed can couple the that those speak of the And were items approach one from intent. So Noah to the and taken was to other.

Noah Rosenberg

PKAN-ADL, the such on sitting - independently weren't in the there each. felt these item were Tim, of [ph], I freezing. these which overall, just They which two II. so, the I is a out look X want UPDRS any scale change they're be separate from chair. Part that the of that also or if a population. remember, that is There relevant getting items relevant point domains XX was were to when four way designed There was to essentially you at was tremor removed, mention think of [UKPDS] will was scale that were are and And one clinical are that terms effect. which a problem And point the designed, scales, domains. There adapted Yeah, added,

even recall keep effect. a changes modest as patients, it's changes. to you with So PKAN-ADL compassionate - significant clinically But in the I using important in double-digit the think II meaningful we signal mind. the Again, would that UKPDS saw use

Tim Lugo

around around if all you Could that in, more around are support? to Thank I retention, kind I comfort Great. could the interest of hearing sneak issue KOL And for talk is especially KOL the support, that it the what's be maybe support? be [WCN] and just mention that [ph], did and that that you interest [ph] Is action] if leading more - you it which [mechanism prior? holistically just class may comfort with one fluid was you of apologize. an may Or what's details. But

Eric Dube

things I think, there feeding into are that are a few that.

to and in the think that of in well got that on we're is, some I are number one DUET proven. earlier tolerated long-term product we the to continue to are - coming demonstrate little a that as we've data, that fact less the other see folks opposed

presented lot you there on So I studies [CRIDNS] two think if at large were emphasis that [ph]. that a specifically there safety. I that that more meeting is long-term that and think, recall, also there durability and SONAR outcome were

these Things in there's I nephropathy risk like together that there is were double risk, reduce diabetic disease. what looking drugs serum or these hard But syndrome studies than And actually and to population end-stage prove or taken renal that we're slightly studies. in nephropathy outcomes. think is at. for both and different nephrotic residual residual demonstrated outcome population improve potential this creatinine the

potentially down credibility for potential and mechanisms of well provide long-term as utilized then think on ERA, concept overall road different I'll even and more also the So credence in SONAR of combining those the that improving outcomes. adds it background the add nephroprotection, it which of both - compound, terms ACEs support finally, inhibition, to or rationale and for was was RAAS as in I just and you the ARBs the while data, of

Tim Lugo

hear. great the for Thanks detail. to It's all


of question Raycroft comes Jefferies. from next Maury Our

Your open. line is

Maury Raycroft

the good progress. everyone, on congrats afternoon and Hi,

First study. question is on DUPLEX for sparsentan X the Phase

Can how XXX to out best comment eGFR could way eGFR? play on FPRE, for on approval there. succeed try power Assuming you understand expectations slope weeks? for What's at that to the and your you differences accelerated

Eric Dube

Yes. Maury. Hey,

So to Noah, one. this you want take

William Rote

this Well, Bill. is

going I that, off care, of lose a the we expecting powered We're the that. population best or think each patients those that expectations in that that, even is are see to and five of eGFR at to treatment statistically to end mils delta absent of FSGS three standard significant of year.

haven't powering revealed helpful? you what specific what can that it. that give But around or of size is We the is. effect the generalities some that Is

Maury Raycroft

That's helpful. how that play XX-week, proteinuria relationship I guess point, And the I insight and get would eGFR some also we guess? at so between at about into or greater eGFR confident out

William Rote

is So at not weeks that there time show point, it's XX if difference. to clear enough the at

of the that in So in a two one endpoint The two and a weeks at diverse. years longer to lines leads those for to XX why longer then of for variability you the confirmatory effect looking reason primary have two size out study why build running the you and the combination you proteinuria at and eGFR. that years that's the point need it's review way we're study such at

Eric Dube

Bill. Thanks,

long-term renal really renal outcomes. or that that curves weeks show X.X remission survival partial have I as at partial a think I XX better endpoint which as proteinuria look really on that for whereby used reduction XX% complete was which the FSGS or of thing well It's at of is the other greater to below. on the done is in those baseline that endpoint based proteinuria level is that remission of composite endpoint, or proteinuria, a actually health, is would add modeling

But a function. which can you that think longer-term of believe based was renal basis or looking would DUET, could weeks. we I endpoint, large so predictive at then be XX for And a show on that be

Maury Raycroft

label then provide Thiola, as your quickly went maybe on I'm company for the any the the Got package it. just focus as shift if And opportunities, filing to second to to what that, pipeline on wondering, then current strategic with in color extensions more commercial the Thiola? helpful. - any in you question the the to thoughts Very more And can on NDA?

Eric Dube

Thank Yeah. you.

the new much of certainly about sure the formulation So want we've that next time disclosed the Thiola, that approval, is we - not make will month. at which of to share we

I was of we'll challenges serve you and the to with the from heard current very formulation much certainly can the more, that so And we patients tight on the tell share the but some what package formulation. that we

So month then. share eve next the date of and again, certainly we're PDUFA at the on the we'll end of more

further expansion, out products that for of cetera. of But in label continue management, evidence, that look more for point. something this do continue and something to decided not to any our to we that's et we that In at disclose develop have lifecycle how it's terms we

Maury Raycroft

very question. Thanks you for Thank the much. Okay. taking


from Our next question comes Canaccord Michelle of Gilson Genuity.

Your line is open.

Michelle Gilson

There Hi, you atrasentan, a thank elaborate data comments. of bit for just Congress maybe indications could IgA were versus a talk hoping guess, touch more selective question. World endothelin you presented taking about Nephrology was I I see my and FSGS on, nephropathy on is ideal you the from nephropathy which for this sparsentan and mechanism? you diabetic why A read-through to just antagonist. Could receptor any are more

Noah Rosenberg

here. Noah it's Michelle, Yeah,

population other then tends with to and atrasentanm, there population heart sicker higher little of I a think we're there rates failure complications. and nephropathy, this So of in are it's what seeing - be diabetic

those the with the to data was these cases confer carefully properties addition, for we've that in managed actually diuretics, no more patients. of diuretics outcomes. population. in So was treatment worse significantly some that compound, terms at fluid seen confidence managed that any but was context DUET A FSGS see compound us heart may of our heart look some the actually We you was of gives actually a favorable properties But With and lower in even lot failure it in in know, failures. signals heartening that the retention and that as in with of with even

for safety proceed. overall So solid gives data very to basis us us from standpoint I think a that a good

foundation addition In DUET all data before. to the from to the - that terms in solid of we efficacy have alluded the was overall that

Michelle Gilson

Okay. Thank you.


question next of Nomura. comes from Marai Our Christopher

line is Your open.

Christopher Marai

Thanks question. afternoon. the good taking for Hey,

us could remind the you should primarily the one. you UPDRS one there - the fosmet. this if or on onto a regarding patient then we And further PKAN-ADL. single mapped in versus two, maybe wondering, just if X elaborate is the Phase Just reports - was those could Number in are studies, you. in that in open-label or of PKAN-ADL to follow-up. that results drug? you'll on how other really components Thank have and And for that then that think one the number mapping case exploring one be on on I on included if the couple actually the changed dose the you individual

Eric Dube

All right.

would So you like to those questions. Bill, take

William Rote

Phase The Sure. a milligrams Study is three currently XXX times day. X

was I that up how XXX does times those from milligrams question think individuals XXX compassionate with to three setting day, in a use they per the day. range milligrams compared your

in we're at higher we as Phase those had X,XXX doses. X went Study, milligrams administration, In high in even as single ballpark. that and pretty tolerability So reasonable

The PKAN. mapping from PKAN-ADL. question the individuals other and occur saw, that scale only really effect bridge that freezing the to the UPDRS of in removed and compassionate over components aspect don't and measured were your draw and which with comparing in we patients trying The to was were use the tremors,

ability PKAN-ADL, change, So you the chair those assessed should you be independently can that's patients, when to get in one components a more it net sit unassisted. out is not a go which of you've neutral had to and

difference. be would that only domain was So the new and

UPDRS in depends patient the is So that's either patient scales, they is there one where PKAN-ADL than of The reported physicians reported, or on the one diverge. more reported matchup some and difference the settings. caregiver that's much

your So I question. you helps hope that with

Christopher Marai

bit we what that versus the that one I I PKAN-ADL, that's one more meaningful about? Absolutely. a just you any then And be expect Thanks. be, in effect file scale And the Thank not, regards thinking would were pediatrics more see just on, And suppose, you about thinking on treatment an might and the to then be adults, if NDA one expectation relevant you. to you other versus group should with to clinically population? other? the clinically suppose,

Eric Dube

I'll address. Yeah. just

rating. again first how X question domain the works PKAN-ADL again emphasize to and around X is the is So XX the points just X to whole powering scale

would a we changes meaningful. on Bill to. low-single-digit we double-digit while see did as that or placebo so PKAN be Again, the alluded would anticipate these improvement And scale

I be subpopulations. here, it's a changes, the we with significant you power would small is subpopulations, disease really and these regard but in question a values studies to So we is answer modest it patients, fairly those to as P show expect the rare see group to clinically think now, do aren't to

think, meaningful favorably. in I in again quite disease even point we're totality changes we of to the important these would how treatment it's and in there no modest And upon understand disease something really and every would data looked believe and pediatric that be approved a small population at and where is So look the patients. be improving data would be


Our Bayko comes from Securities. Liisa next question JMP of

Your line is open.

Liisa Bayko

expect you about I period. XX-week a the to and know thanks of a my background Thanks. question. about want endpoint, Would much ask how about little natural just you when taking the Hi, looking over bit of for change? that Study at history, more kind FORT as sort or thought the

Eric Dube

that us is reduction hard of flares worsening, know stepwise, what's steady a We ton can study. this modest of do been we effect XX maximal say studied. we're don't our see - term. months sure have see with state. to just that while worsening a effect I'm - folks see are to talked well answer weeks, a think their We rare It's is there and power the we We sorry, disease. at to hasn't with that gauge of experts helped I'll able the weeks, And you that disease knowledge patients XX the not worsening don't about this at to that going based that's XX question. on that think a But in What maximal population. see of that or to clinically Yeah. we're meaningful best maybe referred are a occasionally so be disease we position it's episodic. to that typically stepwise to background heard effect. going

Liisa Bayko

time, you how could going going I it over versus it's frequently week XX. way you're then, not questionnaire, baseline the And remind you're is Great. measuring, time. know through of correct? to kind us Okay. over It's The be because

Noah Rosenberg

that the note in frequently important measurement, throughout And think that's various Yeah, data. of the time-points. measurements the at the to robustness we I have way the study

And including XX, which week study. from the consider week data-points, X, change week are not we the the and it's XX XX, baseline. the just So week all week time-points measurements, X, in

Liisa Bayko

thanks a All right, lot.

Eric Dube

Thanks, Liisa.


Schwartz Our Leerink. from SVB of Joseph next question comes

open. line is Your

Joseph Schwartz

very Thanks Great. much.

that to So successful? continuing be, went seen that on order you that quote of can What you provide variability be trial assumptions if fosmet theme, trial. for the the degree I was powering in wondering based on expect to on the separation any information into needs unquote, the

Eric Dube

thanks, Yeah, Bill, this? do want take you Joe. to

William Rote


what and the low-single-digits the it between what need separation I is, to think the We specific powering haven't Noah mentioned previously, far groups. see two was as but as revealed - we is

Joseph Schwartz

patients us sounds could patients to label are over tell be you eligible tracking like opted who forward study these Can need greater you have of longer they'll term separation extension participate for and percentage the it the time. maybe in Even open what Okay. study?

William Rote

a the good patients opted open-label the function. long-term to collect double-blind a amount have out to that's to and good extension. data continue really cross-over And of think from long-term that I into point, have that assess Yeah, we all information Joe. will

Joseph Schwartz


the FDA's well to And you you of on you yet. don't talk can do reveal to Thiola, thanks. gen you think for about then, robustness understand But you quite I review? - - how the satisfy Okay, can and will generated but product, demand of the next the the want the attributes NDA that data that as support the Thiola,

William Rote

the And, so that we for that that. currently believe specific we in we're not process on that we're But filing. obviously, we've Yeah, track disclosed with had. because the engagements And We be that we month. we we're support we've next want just the labeling. with final believe to yet still on submitted discussion that cautious. FDA, approval what don't will But have

Joseph Schwartz

Okay, envision fruit. bears any have don't of great. pipeline or And you presence, much how your I so change for could a was of wondering one me, rest world I then, this of last all might if you one could, currently

William Rote

Joe. thanks, Yeah,

that is So, certainly, something that we are actively looking at.

process a particularly head over and in quarters, our building We of that fosmetpantotenate are last the for has of in expansion of got preparation We've team building European of couple Europe the organization. been out a the launch. part

would outside specifically And for we're looking is what But looking and different nephropathy rates with models particularly U.S., be know that Europe unmet the We are us not best need. and high, disclosed of of in is IgA that there the at engaged have for Europe. U.S. outside sparsentan, and where what in actively of value. and delivering we we've FSGS that model

Joseph Schwartz

good. again. Sounds Thanks


to like back Cline for any the to call further over no I'd are There questions. turn Chris remarks. closing

Chris Cline

Michelle. for future updating in you Thank Thank Great. you, progress. the our and the quarter first concludes joining. everybody for near This you call forward our look to we on


Ladies the and today's disconnect. great day. participating conclude a conference. you Everyone, your may you in And gentlemen, does have program. for all thank This