TVTX - Earnings call transcript Q1 2021 | Travere Therapeutics, Inc.

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Operator

Hello and welcome to the Travere Therapeutics First Quarter Financial Results and Corporate Update. My name is Brandon and I'll be your operator for today. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions. [Operator Instructions] Please note this conference is being recorded. turn to now Cline. will I it Chris over And

You sir. may begin

Chris Cline

Chief Thank and Brandon. results Travere Noah Commercial call. call us. prepared and will you will Chief well. Thank by Officer, all welcome by for hope our and President our Dr. for Officer; Officer, Chief our for you the Vice remarks I Q&A Good Officer, the Dr. Rosenberg; update be joined Development financial Great. Today's Clague. XXXX Heerma, Dr. afternoon to be Medical Eric remain Eric Peter joining Senior us quarter Laura Bill Financial Therapeutics and Chief corporate session. will Rote, our led join you Executive first Research Dube. of

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

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

date disclaims of information, In made, XXXX. future our specifically circumstances. addition, represent any to Travere such to obligation any statements are the update statements or only as views X, And such events reflect forward-looking May statements

over now the call me Eric? Eric. turn Let to

Eric Dube

rare our to was position of enable will of key another the first year our quarter dedicated execute. strengthen to example disease the us And believe remain we We ability of community. that leadership organization's in the this to priorities

journey. rare on The Foundation that average programs for burden that diseases diseases than nephropathy. upwards X.X specialists the approximately rare study. year, the of is over U.S. see XX the was in with rare diagnosis Everylife targeting For direct faces their the years study pipeline billion that disease disease us community few have for potential decades the living need. like unmet costs to this centers patients a especially our to that IgA medical the study Overall in the rare find XXXX. innovation Earlier a national published significant on associated person due challenges first-in-class more a along FSGS rare it estimated given $XXX relevant the and for typically found limited the past takes with This developing nephrology diseases this economic and of diagnostic

is groups between encouraging. that at were This each treatment become treatment people in to For a February. FSGS reaching of data of experience The and if considered encouraged diseases. generally XX% will prognoses in Patients have end-stage in transplant kidney one compared to example, showed is patients been a that Study our relative FPRE safety generally journey, the glomerular approved. with It achieving time dialysis. worst interim year has We nephropathy primary upon FSGS the estimated resulted in the and DUPLEX the and function overall dependent This XX comparable, profile within by IgA of the translates we end-stage progressive have today to pursuing disease kidney of years. nephrotic sparsentan more for a long of standard FSGS become showed FSGS-related data sparsentan from treatment proteinuria developing we the and when new remain data reported that that kidney over X,XXX is disease reach with loss amongst irbesartan. the than of study is the greater persistent urgency also U.S. have analysis to proteinuria likelihood goal great in well-tolerated why The sparsentan diagnostic XX% which

engaging to the accelerated agents in for are of We discuss with process FSGS. regulatory potential approval submissions

broad As parallel, regulatory today, planned track sparsentan so community continues the on the Study be families. before the remain The a to the end update of PROTECT nephropathy continues advance in and to will a nephrology to year. do such, provide nearing enrollment. but and study patients their half completion position from and not our IgA we is of to In of from first receive of as support in

for proteinuria PROTECT consistency to throughout with, topline the ability to look this readout encouraged from reduce its has of which the year. meaningfully data proteinuria quarter we Importantly, And track remain remain from demonstrated development. sparsentan we on the third we a interim assessment the in by its

continue analyses in in additional reduction also in specifically published link of to see this eGFR. We that nephropathy clearly the trial-level with outcomes IgA space proteinuria beneficial benefit

Importantly results coming with looking PROTECT consistent these are to we forward those how soon. up designed have study are we our and

of the that address without literature excited molecule classical pegtibatinase, our nonproprietary continue new of HCU genetic no to of specifically cause for HCU. treatments also about TVT-XXX approved underlying suggests or control for development approximately are face to We XX% be to age adhering that and There and diet compliance the especially in homocystinuria name and inadequate The HCU. Available low-protein challenges difficult as often attack they XX% also a treatments with patients patients and patients age XX lead thromboembolism, of heart have which are age. XX by by stroke. can

treatment there an be more is to believe than and to need patients HCU opportunity option. U.S. X,XXX meaningful that in help estimated a Europe with the classical the and living of we effective So in

with working program encouraged advance classical make later disease-modifying for be potential updates the Our pegtibatinase, the first X/X look to We on additional to and therapy by continue Phase forward we to this the team study ongoing and ultimately the homocystinuria. providing patients to year. is investigators

experienced referrals organization ongoing continued first but deliver challenges position our our ability the In our also we underlying some our approved upon strength new consistently patients. in and disease commercial quarter, to patient rare to community Our leadership to pandemic is remains. the built the deliver of in therapies the business from

last treatments the we expertise new if us be and of did months COVID upon had January challenges in of see starts support growth achieve in patient new through over we fall, February This successful the guidance than we that provides increase with in the Importantly, while resilience can for patient anticipated year our slower consistent referrals through for delivering and as maintained the draw year. a and the mid-single-digit in approved. have to April. of a resurgence access And ultimately approved our and we meaningfully confidence result sparsentan and our full March

turn Noah updates for pipeline. the to Noah? call me the from over now Let

Noah Rosenberg

Thank Eric, everyone. good and you, afternoon,

continue sparsentan as to nephrology standard engagement We a the towards be of and developing with our FSGS community, for IgA encouraged treatment if new goal in work approved. nephropathy by the we

cause and FSGS there As recognition a indicated kidney Eric the Unfortunately, to be rise. referenced no medicines leading approved kidney a of earlier, believed end-stage prevalence disease that is are the is And on clear failure. incidents disorder. and subsequent there for is of FSGS this

patients about of result, to with provides and/or therapy, need aggressive unmet with dialysis a suppressive challenges each advance our are if sense options, daily they as, treatments with Even us such we to for immune come continue treatment As require go transplantation. people a program. often This urgency successful, these living. day, living significant FSGS as

in our broad from results team to continued from ongoing of the DUPLEX FSGS, pivotal of announcement and the Study the medical Following the has advocacy support, interim nephrology communities. sparsentan receive

kidney well-accepted mentioned, the nephrologists FSGS. in among link previously This by community lowering in is outcomes we primary nephrology managing As treatment sustained population. proteinuria is as proteinuria that driven in recognized this a improved reduction goal results amongst the

are believe, become reduction the treatment option important compared use that the the sparsentan base If can paradigm. first We an of development new a to demonstrating treatment we forefront proteinuria, available physicians their study currently at approved, treatments. with that of can pivotal as meaningful

pursue as the in for the we'll before provide earlier, process to approval submissions outlined Eric And our regulators. accelerated to we position As update, on of a first we year regulatory in our expect, end an the to continue of planned. plans half discussing the with that FSGS of be pathway, are

to we reached milestone study. DUPLEX interim the important we an of endpoint While achieving the phase still the complete Study, its need with proteinuria confirmatory

retention sites the study thus DUPLEX the blinded am eGFR maintain the engage endpoint to we of study. data investigators of XXXX. ensure in And continues half high-quality the throughout from two-year I track the and conduct, on to progress trial top-line patient Our far. team the very and remain first of confirmatory with pleased for endpoint,

IgA angiotensin the Notably, U.S. amongst opinion blocks also between nephropathy IgA and in key estimated like interim combined. the play inflammation, and continues, X in for believed a and Sparsentan, nephropathy. proteinuria and is end-stage affect selectively, to readout for Phase simultaneously areas disease. DUPLEX, receptors, leaders of kidney sparsentan roles Following PROTECT of results the is people of key FSGS, IgA nephropathy. Study it responsible and XXX,XXX Much IgA the to proteinuria and upcoming endothelin FSGS, in nephropathy. cause fibrosis enthusiasm XXX,XXX the be inflammation leading in to the Similar from fibrosis Europe the

studies a to its translates well. Importantly, been nephropathy interventions predicted in importance shown baseline, a the higher and the from reducing continues on has And pre-clinical and IgA amongst models reduced eGFR Inker, IgAN, to is IgA slope eGFR XX in ability between fast mesangial to correlation treatment reductions in In well-established that proteinuria effects effects target, generally eGFR, And has University, IgA proteinuria, and analysis of one sparsentan In Dr. proteinuria to on the nephropathy. strengthen it have that strengthen on as multiple nephropathy. prevent proteinuria slope. consistently this of found proliferation in treatment of as the publication, proteinuria a link treatment evaluated proteinuria progression with associated The and cell glomerular and the sclerosis in accurately effects outcomes. of further disease. Tufts recent improved others, early

some many treatment in Specifically, of the or consistent or day, treatment of least that X.X DUPLEX PROTECT at those of findings which our approximately at also benefit proteinuria are to years. there XX% would is in the patients enrolls reduction XX% effect with the the for PROTECT but are confirm one more. per DUPLEX slope publication ways two between included similar These more with gram compared differences two. to per notable an gram eGFR probability Study proteinuria with of Study. design cited in observed PROTECT

Elsewhere X.X is into reflective below heavily which greater both a be proteinuria one nephropathy wash more DUPLEX, show, patient Study designed are per reduction of proteinuria analysis with meet of the for publications randomization. in the after are to in as will we day. year. is to combined have And on XX on the with study per coming XX% baseline in recently sparsentan are in versus anticipate We proteinuric in up of to-date. no of IgA to tends the percent note, analysis and last track to the of expected therapy Enrollment FSGS integration assessment a While we of in gram progressive get in HCU. the for near-future. treatment dose there finally, these is of top-line patients trial-level disease. treatment. proteinuria randomizing that FPRE the over And weeks ACE/ARB strong. powered in PROTECT on threshold interim the patients XX% effect consistent be reduction is All data an compared IgA required nature, relative a the and fact in XX% program for is interim completed this remains quarter the successfully gram measured requires gram of the PROTECT nephropathy study. a the to report values reduction eGFR, years, Of pipeline, screening from that endpoint than gram we third pegtibatinase per The of closed proteinuria interim PROTECT in proteinuria to from confer max analyses from the A comprehensive relative benefit irbesartan to two irbesartan.

last that TVT-XXX Eric outlined acquired earlier, year. late As molecule this for our name assigned we is nonproprietary newly the

as grow, Our for develop disease-modifying first with therapy the and deliver time HCU potentially spend continues the more optimism, program. we to to potential

clinical forward. with and advance. of community X/X pharmacokinetic, we HCU We to the The their fortunate tolerability, directly and Phase to safety forward engage study And insights have to move pharmacodynamics assess to look the escalation the integrating optimize program, dose continues pegtibatinase, we to been effects investigators. as

have through one worked with pegtibatinase sites, enrollment that the COVID-related of in have the planned. now but new program such highest is opening as to cohort We currently patient final we some challenges site the

study dosing an at, Assuming with this dose and could have history ability parallel, later safety optimal how or the in provide from In we to if continue provide we us will this look are remain the the we additional determine, enhance if progressing on expect the cohort. to study. us ongoing the should year. schedule, an with to initial natural benefit program cohort we identified This

Peter. become to identifying, continue the call people with developing therapies to the program. regulatory Peter understanding We this in update. available. providing progress over insights changing efforts of to they for year pursuing our mission gaining with progression to pathway, I our establishing and a as expect the this additional to instrumental now life and with very the Overall, to I’ll disease. of We and our be rare be for beginning pleased clinical turn anticipate our and deeper commercial of HCU forward later clinical dialogue delivering HCU look for the regulators in

Peter Heerma

Noah. you, Thank

our year through potential challenging central period for for provided upon foundation organization has therapies for launches. through to commercial team has the The begun to great to and well XXXX. a performed motivation futures us and throughout prepare Our solid deliver approved build with to the have a

quarter, new our of three the products. first therapy for approved we all saw patient During initiate a

At year. of year-over-year of However, This increased a in COVID-XX related of over approximately sales from in $X product a in pandemic performance. the the is million who quarter quarter $X.X resulted patients onset revenue first of experienced net the have to expectations performance. we first above demands dynamics million product in last the year, last flat

start As expected, of year. mature not in the this this did

impact our the This a from industry-wide surprisingly, physicians. in is accelerated the momentum has two than we COVID-XX for in to have seen on And a fall patients cumulative demand referrals said, and beginning the did had referrals April. the see fewer trend and see effect COVID-XX our for of cases the year. first stronger start patient dynamic had in modestly has continues approved their That we we created important slower-than-expected that the visiting started to January and new Another XXXX, months when products. And March an of year. February patient now

account, are Also driven beginning encouraged with net gross present. higher the the experienced our strength underlying levels the sound the This during we of all anticipate we of for resets Year. by return position to quarter, remainder to we and year. we So we the our the the to of is this patients is believe previous a primarily treat are a identify year. to by factor, the business insurance years expected that the normal Taking in New and consistent this through in into new of in balance and

before, in to our expect growth mid-single-digit approved for Eric continue XXXX. As mentioned products we

continuing and is also ultimately sparsentan in prepare to approved. work launches are FSGS if We nephropathy for our IgA

take continue and to access We understanding our journey the strengthen will broad patient it therapy. what to to of provide

last place seen over to decades. the few do We that the innovation pieces are product space in a in the has to to continuing launch foundational limited work put a

infrastructure We preparations ultimately if generation delivering disease people be turn rare and will in to are expertise Laura for insight confident nephropathy us and to over Laura? to and the our that FSGS IgA with longstanding update. now call successful I'd financial ecology, approved. to the our our ongoing enable living established like sparsentan commercial

Laura Clague

Peter. you, Thank

HCU. period and same million increase a to and For $XX.X the which attributable XXXX, were for the of loss portfolio, we XXXX. the well R&D expenses stock-based of first cash, first net The is studies adjusting of first general amortization. period for in increased of million a the quarter was quarter non-GAAP XXXX. the increase the the of largely and non-cash basis million comparable is reported over R&D basis largely compensation of quarter a patient from increased for quarter $XX.X to After $XX.X depreciation sparsentan, as the basis, to $XX.X to million product basis, XXXX. of of first DUPLEX of initial enrollment expenses classical for million. were loss million. $XX.X pivotal compared XXXX for net adjusted and We growth were to in first Relevant $X.X of million amortization. compensation and were integration stock-based quarter as pegtibatinase and adjustments XXXX quarter in our and expense we sales investment $XX.X net attributable of our for launch On the compensation an in for income a for preparation quarter same the GAAP commercial in $XX.X the an The ongoing for and non-cash adjusted reported support GAAP On expenses in expenses the expenses non-cash first GAAP organization of XXXX. administrative first reported program million PROTECT On million advancement the XXXX. the first selling, quarter activities. included Significant consisted SG&A the On expenses of quarter $XX.X

from sparsentan, expect preparing to we to success confirmatory increase advance accounts studies the classical expected their respective balance HCU of support organization operating the ahead, endpoints. as It of DUPLEX quarter-to-quarter pegtibatinase will Looking year, ongoing occur of our for to for the our in to QX continue as expenses planned throughout This uneven and is which investing in launches. program commercial potential may PROTECT but the pivotal be levels. and two-year modestly we also the

Eric? development, expected current ended Our million financial I cash to Notwithstanding business will the to his the over Eric this years. back to $XXX.X cash two hand is now total activity call operations remains and quarter with the support foundation support our closing equivalents. first in cash balance this next strong. beyond comments. We additional for

Eric Dube

strength core partners and for thank a like of their our our to for to our continued I remains all would members commitment Execution mission. Laura. you, and team Thank organization

the off well. started have year We

of in by sense listening need treatment is large part new that urgency is options. to the and driven performance the clear patients' perspectives created by for Our

We HCU. the the people nephropathy. will therapy first for continue new and leaders and IgA FSGS with in community to this our develop potential as nephrology channel deliver to living rare with will break of disease-modifying ground our pursuit it classical innovation further in first work we the bringing efforts continue And to decades in the drive to

Let to for over me Chris call now turn Chris? Q&A. the

Chris Cline

go Q&A? Thanks, Eric. please line and we can Brandon, the for open up Great. ahead

Operator

Yes. Thank you, sir.

ahead. Joseph have Leerink will We And begin Instructions] from the question-and-answer go [Operator now session. we Please Schwartz. SVB

Joseph Schwartz

of questions. All guess right. that that for just analysis Thank worked doing in one you literature ago a you of IgA really FSGS so taking hard variation my you'll use the and be first PROTECT a And in is with continuing out my – FDA. to your know I in much the hammer I commentary all insightful on that for referenced nephropathy. you supports the years lot the

if due just personnel to just I'm there the reasons from the of So reasons, given granted. or dealing question. unknown continuous you in the it people hearing wondering looking could between same my how now such for various wondering string a now with than establish some maybe and agency much like at ago, like look things long things analysis the us, at years seems is surprises as you're shifts differently commonality they plan personnel there FSGS? versus that for Thanks when just I'm you is might characterize and how

Eric Dube

Very Bill to few Joe. comments clear question. have then information. ask And and further Thanks provide I'll I'll a

don't any understand where And at IgA you the are I only for mentioned, to overall, certainly, with they recognizes certainly some we that, at want provide they that but the in further top comments that to support nephropathy I'd XX innovative longer-term have in as think how things link trial programs the from the agency you speak today I want of for of on weeks Bill, regulators, believe with as over grown. your literature to our say very years evolved eGFR how years, perspective? has literature. FDA the a on analyses. design we ago so do look looking that And So recent designed we proteinuria two and and were

Bill Rote

months. for members group on areas these There's has quite across question, seen thanks cardiorenal agency people they I've be little same We've years on statistical after and from almost way in the from and the of of a and that medical of that consistent. projects seconded and the the much at the Certainly, division, X.X shuffled then professionals to the turnover. But whole And specific in change return dealing very Joe. the table us the team through. a in other with pretty some people been for and COVID period been in tasks, the nine agency six positions to allocated review relatively been resulted for change some been leadership we've the way team. the the in within period the

Joseph Schwartz

I in the then you ever color. have. then mentioned do the of that what already Great. capabilities at a brand? helpful. have out to mentioned currently think – top if for that to US That's both be a sense super global And enhanced this actually, what the build Thanks build for think Can you outside US? starting on but parametrize presence you don't Laura, much commercialization could there, might us just of that you point And given the make you it

Eric Dube

-- thanks Certainly, Joe. our and

our this US Europe. or take Peter team that we whether and sparsentan under Our commercialize whether that the partner. for both ask activities prepare still bit a the about is is review that we the in in approach But plan do And undertaking to certainly talk to I'll to we commercialization. for is little own Europe

Peter Heerma

Thanks Eric. Yes.

with So to to part with regards latter Europe. question the the start of

So but different. need as equally the we in unmet Europe high the think are is Europe US, the dynamics in quite in

statement take good patient understanding commercialize part a little what as we does Well we we Eric of will ourselves. as disease we first And and With made the investments the question your as now We it of our and I [indiscernible]. display perspective. regards what the a the burden year public patient framework. are is if like year we disciplined we're sizing are making certain think a quite a in really the pleased is gain now said, to progress. So to what of what haven't on what requirement the And right we with well are population is right Europe focusing addressable get and continuing insight R&D value to generation that on doing the also we invest Day. of to during And is patients commercialization patient last bit journey in say, of the from what that field are ultimately force force population this gave looking mainly addressable field required. a

at is We both patients and efforts the distribution the that are the way is endpoint. as but we also underserved disease to really gain of patients also understanding educational look study conducted as model to because needed is FPRE what product as the physicians, I bring and to our think as best educational to well FSGS initiatives an well

Joseph Schwartz

Thanks for insights. again

Eric Dube

Thanks, Eric.

Operator

Bank From Greg go Please Harrison. we America, of have ahead.

Greg Harrison

data get Hey, could study from Question there conference opportunity guys. Thanks see is Study. updates for DUPLEX taking periodic And before that is eGFR? there this be additional accelerated that to an for planned you presentations approval? to be an particularly the question. were medical Will the something or on

Eric Dube

with you additional and also don't why potential to Noah, So that take one presentations? any data regard

Noah Rosenberg

a That's question. great Yes.

see additional data, study the to now to have minimal -- had the to in know you plans for but data publish really desire we to right ongoing We've and as recognize the further. to the protect data endpoint confirmatory continue we no overall got provide two-year of So we terms DUPLEX, integrity. we and

we'll wouldn't introduce and to we potential update be bias that evaluate to get opportunities continue regulators. you there. We as acceptable That's

Eric Dube

about say, study to you, is we're study no think I how to making maintain do the conduct Yes. a blind, for little we're to that to about what the want Thank The that and doing DUPLEX the additional data, point. this that to Noah bit share the sure unblinding through plan which is end that as additional Noah. that approach exactly taking at I and maintain addition which Greg, in we important there thing any utmost is said, would only the have is us. to with a we

very have working filings, on to And studies. we we planned small there until regulatory that support the that nothing data is so a is there's that beyond team but the else those complete

Greg Harrison

is far makes consider, I your then the could but you're other just get as it. thinking can't access, Got one. about a I give especially provide this patients wanted how thinking combination for you you'd be are That know value little just know And also latest but maybe early broad one to talked And pricing other talk specifics about as and as sparsentan's will maybe with one used other versus providing if factors sense. balancing -- that to likely the treatments pricing. you've in eventually you it the about I therapies approved. we

get to So wanted thoughts just I your there?

Eric Dube

Sure.

make sure access we can that our goal too I to say that talk think So what I have broad innovation. it is early for to is to us specifically about this but pricing, is

And his waiting combine to a have these to be does the potential that want barrier therapies. So for with and patients been that that may includes make that we know many we become sure patients and are not sure team to other anything Peter, very he add? as making payers. say on and for Peter earlier for I mentioned much on focused that the concern can framework that else a access. you'd But want value

Peter Heerma

I think at you all. mentioned it

it think like if costs FSGS about $XX comprises a in XXX,XXX patients the associated about take and good about US step back I billion. and is it's disease to think you say the

patient IgA they are kidney you then And that at are are expensive society. fast that and and disease the nephropathy these population. almost FSGS progression for if of end-stage patients So among disproportionately most patients they look represented in

in the value a right like base come a with there's good solid -- up work I now. respect and doing that that for think we're to that's proposition

Greg Harrison

Super Great. Thanks for question. taking helpful. the

Eric Dube

Greg. Thanks,

Operator

From have ahead. Barclays, Please go Carter Gould. we

Unidentified Analyst

on for the congrats Yes. on for Justin all progress. This Thanks and our Hi. questions is Carter. taking

Can Just confirm what us remind given to from patients, drive enrolled Sort the milligram could how that gives XXX confidence on process dose that should will a be still XXX interim you sufficient of it possibly you of benefit? that's we the expect that just to the exceed level and PROTECT. or quickly decision-making to read us around the dose? inform data the couple of that then be you had a the selection number? And

Eric Dube

or Justin, thanks the the is one third XXX And to is And answer enrollment in for on the will I'll track be gets it quarter patient what visit. mentioned turn planned the questions. was time. it to dosing analysis complete as And XX-week XXXth be in depending talk this And Bill when about that and over second provide on because that plus PROTECT. we've that the got I'll to enrollment analysis at I to the we're able of year. minus

Bill Rote

for Thanks us the the FSGS data milligrams Justin. population. show right dose DUET that XXX We now is the believe from question, the from also DUPLEX that for and

As dose and high XXX a When both study you of we top milligrams the used the were of had PK/PD milligrams response at in and are the XXX the curves degree both sigmoidal curve. they and we a looked largely results. also Essentially, XXX the at recall DUET saw overlapping XXX overlap.

the So difference XXX and XXX. have while the effective a larger between you difference two, between numeric isn't doubling

and a plasma highly diseases and a comes IgA present highly has FSGS, a is other protein circulation. sparsentan are differently. albumin FSGS, With it's in they with degree nephrotic nephropathy different that of proteinuric proteinuria. of bound to Starting Specifically high and and disease and loss proteins they

is dose FSGS lower nephrotic proteinuria to to potential is designed you're proteinuria XXX protein, The that where patients patients lose in loss. IgA nephropathy sparsentan. the compensate you when less. of losing is much for Contrast also So rate that milligram

doses those for that that our where the there and confidence dose patients. nephropathy see to right And dose for sigmoidal us event. the curve need so have That fall response a the to XXX with -- we don't two is along on higher IgA give the modeling the compensate dose

Unidentified Analyst

Okay. much you so there. Thank for color your

Bill Rote

Sure.

Operator

From Jefferies, have Raycroft. ahead. we Please Maury go

Maury Raycroft

can an clarify follow-up everyone. can then thanks not for on ongoing And of and you're for related I Hi, in on to taking regulatory update, a comment regulatory pre-NDA dialogue, scenarios you're a formal and the the forth a meeting you you mentioned you is for or now but engagement? know back preparation Congrats meeting scheduled if if the wondering providing I'm and had from my a engagement outcomes initial potential progress XQ? questions. ahead you as

Eric Dube

able previously much EMA to scenarios questions. this where regulatory the the But your it's We'll quarter. might not updates quarter. is regard provide an as don't be our on would we -- those later dates guided of be. engaging and provide our to interactions that provide for typical to I position be we're track second And to so we're practice probably are we've to on And able those to question by speculate and thanks we business to a in this any of our able your on end interactions. be on process around say with with FDA that both in for implications interactions update Maury, specific and

Maury Raycroft

Fair enough. Understood.

maybe Okay. And PROTECT. then and one question IgAN other on just

line it's can you whether with and expect else think to you Can fully baseline, mentioned do IgAN? it's or or if proteinuria improvement how higher patients that remind And you expectations? at with anything in proteinuria characteristics in you You about comment on baseline proteinuria baseline greater in me enrolled. close if on range

Eric Dube

you Sure. like that would one? Noah, to take

Noah Rosenberg

Sure. Maury. question, Great

So that as in mind currently we Study the approach keeping that as blinded. is the take far PROTECT

all through is is -- high -- blinded So data our groups sure data factors. is the with quality reflecting that any analysis doing making both together that for really baseline probably we're consistent expectations are analyses, the going

And much So can there Maury I characteristics. that's was part say think baseline to I as question? another as the about your

Maury Raycroft

Yes.

them expectations of improvement? benefit with your magnitude patients proteinuria to higher do Just a greater expect or have you baseline, for at

Noah Rosenberg

we see below did range, we slightly the lower exist as to reduction saw And to Yes. And right? you in [indiscernible] as level due in expect first higher proteinuria a it right add but far product diminished -- lower the IgAN above and was lower question, -- slightly a reduction in just and Yes. also would in slightly group DUET I'll the DUPLEX compared group. the proteinuria net

So FSGS. but a there expect that, remember might we're than in are we IgAN less in that proteinuric population having

know probably predicted, we but sure. August and of that we'll much wouldn't data impact have an back So as the again that have in for had we'll

Maury Raycroft

it. Got

my Okay. questions. for Thanks taking

Operator

go we ISI From Please ahead. Bayko. Liisa have Evercore

Liisa Bayko

approval? want going based make to want it's accelerated phrase is because -- for clarify, to are going approval you have, you to Just file to Hi. I an Yes, your file just it, way sure that your I that you expectation expectation accelerated the clarify, just is data you're that the just to on correct?

Eric Dube

we to Hi, obviously, to that's planning to prior be data going that and with sure that be accelerated Liisa. and our the that's approach why think, But, we'll submitting, base the that's subject provide Yes, reviewing closely very so writing ongoing want case an regulators have so we that the later engaging and that. the FDA, understand And with updates on interactions so their NDA we're -- this accelerated and the them able to expectations currently approval aligned with quarter. we package to something is for that for we're I approval. make also

Liisa Bayko

like, using -- for you then, gives And separately of data, using to for accelerated that of domain, -- of review -- proteinuria? or public an the kind nephropathy, of with idea notwithstanding file that confidence this base what's what you you Okay. maybe leads communicated that you can in what review maybe but IgA kind and the case? that's FDA just Can approval, kind of, for the believe what's you kind FSGS can been proteinuria

Eric Dube

And I'll take to one. ask that Yes, certainly. Bill

Bill Rote

and In renal faster. story slowing [ph] growing renal a we the age. And literature body [Indiscernible] these of lower is decline with GFR of in the term. of confer is in all we reductions Inker's as to coming proteinuria a lab a the the all are and function. preservation consistent that is out another showing others, In with reductions that Certainly. in have Throost there year-by-year Dr. specifically of domain of diseases function of of long If us, start Michigan a links glomerular public that proteinuria work out patients we rate

able shown been and you're over has to eGFR that's expected these patients, to the in proteinuria If time. reduce in to confer reduction a

the question, discussion used an the at the reductions the as around element two-year to predict surrogacy being the well of -- your eGFR in think in answer with proteinuria at to EMA we've the I in endpoint interim design, agency with other as trial confirmatory Europe of endpoint. been

we've that both in accelerated with of with approval. for the that had what's this are could regulators, domain used dialogue public is gives So a confidence us the the and be aligned combination path they that

Eric Dube

how the FDA, And programs have sponsors, I of designed obviously proteinuria. other who engaged in is consistency that think in Liisa is there use with their the aspect other

think I while So the we received that speak consistency, in the I programs, can't reassuring that we've we've and is received. to other think approach that guidance

Liisa Bayko

you. Thank Okay.

Eric Dube

Thanks, Liisa.

Operator

ahead. Tim have we go Please Blair William Lugo. From

John Boyle

provide Hi, you thinking guys. was you Tim. could And This on I about John a on color Thanks to FSGS. responding payers for our just sparsentan if commentary some going discussions the maybe if are provide Thanks. on payer how data? proteinuria questions. more in interim for guys specifically, taking how are could the bit wondering, is some for and

Eric Dube

take this your John, And Peter, thanks questions. one? you to like for would

Peter Heerma

question. that for Thanks Yes.

ultimately is informative think conversation into have work end-stage of on the of kind relationship progression disease. payers. kidney to that just proteinuria translates the disease reduction delay And Bill reduction with outlining, and I some that done of how we I of think, the like it builds have conversations those was between

do of of work of to kind think disease. is of understanding into ultimately in on XX that patient payers work the now are that's FSGS to presenting, end-stage and level over for Day interested base reduction data reduction Barratt remember we XX% proteinuria how Dr. an proteinuria hard kidney years in the very may and endpoints And disease. nephropathy R&D right You in gain I to translation in what the the similar issue, was where progression are IgA delay actually translates delay in about

John Boyle

so for right. All color. that Thanks much

Operator

Genuity we Please go ahead. have Canaccord Gilson. Michelle From

Unidentified Analyst

This Congrats is quarter. for on [Indiscernible] Michael Michelle. on the

of DUPLEX FSGS for the regulators. before filing you with PROTECT it And to filing come what anticipate the specifically, any data some agency's view I then, or IgAN? accepting data little other do questions and for Michelle the FDA engaging about these would a guess the rare the you like more see complete to seems read-through the interim sort glomerular So Gilson, disease, do more of, in be broadly, the for diseases? you package applications after for I are guess recently would

Eric Dube

provide of before later a thanks after the both our the we provide And so track this your we're in for the regard that. and comment to on data quarter timing the that with we or in an position much to occur filing and the we'll questions. whether to interactions first PROTECT Michael, update on of today your quarter. on from then third regulatory on be would PROTECT interim, sequencing -- wouldn't But are

we be is couple believe So read-through in provide that prepared I regulatory And progress kidney much regard we certainly, can filings, with certainly on that, of anyway recent very my wouldn't therapy. regard greater an conclusions and really to may detail closer I to so in patients our a With we make steps programs, each interactions to to recognition given that what something several that shared other potentially between very that One regulators need, approved I would we're other on different disorders. of remarks, significant in clearly rare is and program unmet of soon. things. say, for based draw understand But those there comparisons to our by this programs. need have just encouraged it's want and can't is

to the Our focus we're of to continue mentioned, And as regulators. engaging is I preparation. in the trials NDA our process with and continue

update be So, provide we'll soon. to able an

Unidentified Analyst

Thank Excellent. you.

Just pegtibatinase. to or named now switching switching newly TVT-XXX actually gears more just one

potential there due looks delay COVID. to is a So, like it

COVID Perhaps is other Is on or anything you there You sites. mentioned an that any way haven't issues sites affecting impact discussed? trial? open so much. opening with Thank already the new you else

Eric Dube

your like on that? the provide for would to thoughts Bill, question. Thanks you Great.

Bill Rote

as from Certainly. COVID The you of been trial note. has not impact free

where be is last might potentially dynamic patients. have that COVID. would in with -- depends And on it as of question times past been There site of during have the hospitals a that trial depends are which some This all throughout are with somebody lives year not these centers, clinical timing. to think something I of and placebo, the an anybody on specific XX been except be example. that's exclusion children's open your for our some in on it the list the specific months as certain sites, for

are and individualized seeing the it's but very as around the center-by-center schedules. these these We've to visit the of COVID responded come by at ease have month-to-month. some changing We restrictions looking the rates and nation, of down protocol

samples data visit to or collect For into in study progress patients using miss example, or come order and forward the key the in a momentum home as points. also, the to that key samples to having opposed nurses hospital to not go the to to maintain the

Unidentified Analyst

Excellent. That’s Thank for all me. you.

Eric Dube

Thanks,

Operator

go Securities, we Chico. ahead. Wedbush have Please Laura From

Laura Chico

circle much this very know was you with for taking mentioned the I if Apologies question. already wanted Inker just on to Thanks I back the one asked, but publication. PROTECT.

paper of the one they Inker of the in the limitations trial that the examined. was So, data mentioned heterogeneity

Thanks. assumptions the And powering. how PROTECT and around maybe so, to you've Study talk address I was just you wondering could if your with to that tried statistical also

Eric Dube

you I'll Thank you, this Laura. Bill, to take ask one.

Bill Rote

Sure.

trials I to community challenge is of draw paper in that rare lot not a unusual aren't there think from. the in the disease the with Inker clinical

publication, a cardiovascular they pull you're a and to studies space, to Inker an the that the doing meta-analysis XX exclusion whittle it in XX case criteria you're matter. the down really going out be took If too to In studies them would or reason with IgA in leave nephropathy sample different that, that group basically the small those set. this Taking treatment and all for studies, together. interventional them might a of

was input was heterogeneous result end group. the So, fairly the a

the I correlation measure the results in when to it well analysis regression line. that of start of look at of and also the in but think the they potential cited consistent uncertainty as a message weakness result one the greater the a coming you and draw -- is,

by look other you we internally as renal the others preservation in linked well message, to work of as proteinuria signal that of the and at that consistency done If the registry in have overall the with analysis have the and data with been reductions data clear are function. done that it's

the think paper the is don't concern. So, Inker something heterogeneity of that in I cited

the I but really was an I their clear. data, it think app input it was of quite output think description message in

Eric Dube

the to around Bill, And question powering?

Bill Rote

relative XX% proteinuria, endpoint. the powering, clinically to importantly, analysis in what believe more powered In or predicts -- clinically a preservation relevant be and reduction to And proteinuria, interim that we're eGFR in of a in reduction very slope. eGFR on show XX% reduction gives loss us we is relevant at a

that with so I patients that's study first really to When the that our similar piece we the is how So mention. study of the heterogeneity powered. baseline with control other talk have we criteria heterogeneity, would entrance about the even are inclusion/exclusion guess around that I that And study, on designed group we your get question characteristics. on pretty to a

Eric Dube

add That's else great. that anything there on Noah, you to is that wanted one?

Noah Rosenberg

Yes.

inclusion/exclusion have Phase specific I think to opportunity trial with setting the with I we here patients criteria. agree when everything study, of Bill that a clinical take you're X think I in randomized said, and the

irbesartan UPC different having that studies than So standardized enriches are standards of results. have interpretation the care variable. control ensuring stringent like backgrounds an things we critical In greater that and to having on is a doing they're of and one, the us

and every So of it's providing population, and UPC again patient by that a care, by more dose for I standard enriching with getting standard irbesartan think little uniform.

I pretty in think we feel like good we're shape.

able were similar across shift heterogeneous population, to you were the going in it we into diverse the population. a after If was we effect. we compare felt DUPLEX, much numbers more And show

patients are is smaller studies to XXX lot ability. And that that the studies. so us the A really I study out size point cited having Bill also gives of of important think those

[Technical confident we'll well. are why address very we be analysis the Then And some feel to it. statistical think also are those Difficulty] able so robust as I

those think the of some I are keys. So

Eric Dube

Thanks, Laura.

Operator

Capital have From BMO we Please Kim. go Markets, ahead. Do

Do Kim

in reduction potentially X endpoint, the you what Hi your What the they Are turn? the duration. that? when Phase question. that the wanted over these the a treatment interested go they my are you hoping drove I of or interactions. interim and that aspects like eGFR during everyone. is end FDA a Phase confirmed approvable that proteinuria specifically Thanks they was ended but for just had requesting longer or for meeting X of looking for company up data study taking could expectations is FDA there Back safety other

Eric Dube

Do, this have we one let expectation which on me that provide further take proteinuria in submission statistically in Bill is a a comment. The reduction any FSGS we February. regulatory meaningful ask and significant I'll for clinically to provided

and trial on many them data, at because mentioned, of be in the going And why As we're safety approval. see in And be we a the on I engaging charting totality they're very the so and design, accelerated sharing need of new they evidence. is we're confirmatory that use to for this that think we with endpoint to can we path them certainly, in will with much the believe proteinuria ways eGFR. this what align looking

I point say from provide to much anything that's I of terms to as his this think would able in as expectations, at but be further ask Bill I'll perspective.

Bill Rote

Eric, I that agree with everything you say.

we're eGFR meets the clear of of color would sure the add see we want two submission surrogate the is engaged is accelerated of that only to to agency this of agency to the that that the think at to why currently totality to the put the of approval forward significant endpoint with would in evidence see their be making preservation the enough a situation addition part Then that provides a I endpoint. that based in or of picture with data, year reduction that being I need the to going proteinuria in prediction that's off needs.

Do Kim

from Great. Could is program? color clinical quarter-over-quarter That's the of Is R&D in provide primarily helpful. expenses? it the studies, some or additional Laura. addition question on HCU all from a step-up the the for you And broadly it

Laura Clague

a of Yeah, all combination of really Do. it's Hi, that.

So yes, you're correct.

the endpoints then quarter continue just here And that those and XXXX. it's we've addition, as And in eGFR pegtibatinase DUPLEX first full at far in study continuing As that that studies on had this PROTECT. program their is for the the recall program Travere. progression to

there's So that continued so investment. continued requires monitoring assessment

So it mentioned. is across that those programs you

Do Kim

questions. Thanks Perfect. for taking my

Eric Dube

Do. Thanks,

Laura Clague

Yes.

Operator

at We questions have further this time. no

Chris Cline

for the progress forward look first future. concludes you you of Thank to today. the in all us Great. and Thank week. rest our joining you, providing We have call This a great updates thank near you quarter. the Brandon, our on for and

Operator

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

disconnect. You may now

Travere Therapeutics (TVTX) 9 May 212021 Q1 Earnings call transcript