TVTX - Earnings call transcript Q2 2021 | Travere Therapeutics, Inc.

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Operator

Good afternoon, ladies and gentlemen, and welcome to the Travere Therapeutics Second Quarter 2021 Financial Results and Corporate Update Call. At this time, all participants are in a listen-only mode. Later, we will conduct question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Cline. Chris ahead. go Please

Chris Cline

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

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

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

reflect the the now statements information, views I’ll represent to addition, to XX, In forward-looking events any statements that, only date as or over With turn are And specifically future our update Eric. disclaims statements to such of Travere such circumstances. XXXX. July made, call Eric? any obligation

Dr. Eric Dube

priority living organization-wide to options to for our without rare FSGS a At treatment of on the decades of adequate with focus treatment we good People and and nephropathy, IgA potentially the an Chris, limited standard the highlighted and become if nephropathy IgA gone of order sparsentan approved. is have priority FSGS and new positioning disease innovation. the you, progression strengthen key pipeline a heart afternoon. our year, in community. this At outset the position as Thank in

way be Despite a that accelerated advancing proteinuria DUPLEX collective patients XXXX, this, our year. where of and or treatment the these with patients. are achieving we unmet FSGS, as interim the forefront build U.S., with a upon that have challenges dialogue is next A Type upcoming generated the interim from overcome. U.S. each As X we deliver could to come milestones, not this these continue but continuing accelerated of progress and well bit of largest by to first can of interventional for for the commitment detail that the this every interim in plan, to We more go look in share support package XXXX. adequate organization new a therapeutic need the pave Outside study DUET FSGS to standard data for we drives the an Noah discussions our pivotal of we Being approvable consistent meeting during result, the data the am help for efforts FDA were This regulatory Study the these FDA both, our Based results positive effort assessment conditional These in year. understanding forward significant in to-date. significant to Europe the our FSGS. transplant strong disorders kidney It our of but this must sparsentan the from quarter, we our authorization approval I progress data to from this heard pursue with in we on of submission potential constructive made of FSGS the year, our DUPLEX the scheduled submission subsequently as an day sparsentan marketing submitting provide inform to pleased believe with for remain will ultimately in anticipate confident that, we of for FSGS further evident new interactions, a in study Europe, thousands remain was controlled if EMA. according At ongoing as shortly, profile. dialysis. by half first to a end Study, half an sparsentan latest options sparsentan the be into that in upon application proof-of-concept sparsentan’s to generated of undeterred the to treatment data of year. approval Phase at

treatment common also cause is leading ultimately and new option sparsentan we an identification glomerulonephritis to patients of for If remain with between the IgA become there excited for IgA cause with and end-stage primary and of kidney and potential room the at patients important patient nephropathy. nephropathy approved, time. We to diagnosis XX,XXX about in over launch most the be the addressable greater XXX,XXX awareness through believe U.S. Europe, disease. increased for

in assessment month. nephropathy submissions next potential evaluating in well. on-track Our data first is with sparsentan as U.S. could the assessment IgA remain designed is next patients Europe, study proteinuria progressing Phase interim interim top-line for early the accelerated in which we XX-week and X year. nephropathy And as approval approval the result for IgA The from PROTECT support to

X/X made pipeline, see with data we remain on-track pegtibatinase are preliminary later encouraged study to the Phase the from progress this Finally, program, two in we by where year.

working in from history be study. leveraging lay the foundation pathway and our also ongoing natural HCU, the to both regulatory will We data by the X/X Phase for

to Lastly, like commercial our I’d portfolio. on touch

candidates, capabilities ability believe Travere’s deliver As in about for our talked current to we the if are have U.S. several we approved. product for commercialization last the years, strength strategic our

and and demonstrated rare patients liver to living have conditions. consistent treat a We with kidney new identify ability

role by the rebound impact year-over-year The important continued resilience, across products, in second growth quarter. speaks play to our all is first team’s of for following they We the our of quarter patients. another example as in the performance evidenced which the of pandemic the experienced

to time, but half the a recently that unfavorable that anticipate not second does we our cystinuria version approved. continue our community. to position patients, part announced to provide But what Looking we to some growth of ahead, the be. we an demand plan more and also we identifying that generic planning plans sales is product -- updates, as will building for estimate capabilities been market. our confidence Thiola this in the will learn This of At the a the original we has formulation business of year. the deliver sparsentan, and and committed time, evolution remain that of expect reliably we do to It continue underlying the if about to impact commercialization or in successfully impact for not our products, have net upon in scenario are of treating our on change

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

Dr. Noah Rosenberg

treatment remain FSGS X afternoon, planned. potential nephropathy. continue on We ongoing report as pivotal that and studies new pleased PROTECT living Thank you, with good sparsentan Eric, developing for and DUPLEX IgA Phase our focused and a as option people everyone. to And I’m to progress

completed planned, Data DMC sixth During meeting review. Monitoring the second recommended DUPLEX safety the based PROTECT. scheduled both, assess quarter, proceed both to The Committee its as or the studies their and in upon independent safety DMC

to after blinded in its in and to DUPLEX XXX FSGS, we the of continue pleased endpoint, is high-quality will be treatment, measure confirmatory study’s manner weeks which conduct. a continuing at with first eGFR Looking changes

will FSGS many announced of proteinuria February, in when that likelihood As the FPRE, time or tolerability DUPLEX endpoint. with in endpoint proteinuria And were we relative you the encouraging. comparable, compared of of the achieved which The at study remission study, is very greater overall, the showed and safety partial recall, treatment to the sparsentan between achieving of groups its analysis the irbesartan. in XX% resulted that data generally interim treatment a profile

working In progressing pathways ultimately FDA the with A and EMA collaboratively for well are Type upcoming independent are on meeting. the We our sparsentan. U.S., both deliver to submission preparations

Our objective the pre-NDA is this for earlier the from to meeting further our dialogue meeting year. held

agency accelerated the We the the goal with We FSGS year. dialogue U.S. with in forward ongoing the months. submission enabling and data for in update to eGFR providing our will additional constructive look an continuing in on next approval an providing study look to the coming of align

assigned we authorization EMA process for productive marketing sparsentan our quarter, co-rapporteur, Europe. second conditional and in representing had the a the the interaction with During rapporteur

a we result to of move application the by for year. of with a in CMA a are submit interaction, continuing upon that end to As these standard time ahead Europe. Based are our this planning plans review we discussions,

to generate U.S. in We utilize planned regulatory supplement the our proposing the are allow us expect XXXX submission. for a that to first eGFR the clock-stop we half submission with data of same potential review the standard to to

the in but could potential process first As is you XXXX. result this in of know, we anticipate different U.S., a approval the review the half EMA from

we support the complete are to are year-end. submission teams CMA progress application to Consistent with studies pediatric investigation earlier by parallel, study also Our meaningful third XX making initiating open-label and sparsentan on of in the approval working by ages to agreed we of year, diligently this pediatric the between that In in to potential children in enabling Europe. and CMA EPIC plan our quarter. X EMA was initiate our expect the the

IgA to Moving nephropathy.

options for And concerns population. completion the achieved needed, clinical study tolerability desperately milestone patients. approved and non-immunosuppressive of this given ahead significant of execution long-term and amongst IgA are indicated we PROTECT sparing schedule in our speaks in need quarter, unmet Achieving no There second of the Phase steroid sparsentan. treatment to enrollment X currently are During medicines of this patient teams nephropathy. the nephrologists our

evaluate the function the and IgA irbesartan, care to mean IgA utility is nephrologists from standard designed data of consistent The recognized continues the clinically irbesartan. outcomes compared treatment and ratio and generation effect tie assessment renal in will which the reduction, be Our reduction Phase proteinuria of for is proteinuria as of in endpoint, sparsentan receptor as function. sparsentan Proteinuria primary treatment and determine X of of in kidney to clearly to well-established PROTECT baseline supports meaningful primary is with to proteinuria nephropathy. reduction literature. between an on the to a by and the interim current pre-specified geometric angiotensin in XX nephropathy the study field PROTECT with its goal It weeks of patients blocker

the us the interim endpoint to DUPLEX assessment accelerated primary to believe Ultimately, be at at PROTECT that patients data its is meaningful is for proteinuria optimistic generate clinically therapy significant data, eGFR and that tolerated package regulators the number to will PROTECT this some sparsentan that to there ability time support on designs to our study anticipate statistically of interactions to no for at a approval. reduction DUPLEX potentially for dose of determining come the should may with Europe. that the submit note, that on a FDA Our data accelerated and package, U.S. we interim there significant anticipate have be Of least between XX available Study, prior demonstrate and in submissions goal patients the assessment. on the all to will lead ACE/ARB year trial into differences will randomization. EMA data developed irbesartan. the one least period. looking requires submission. all Similar of eGFR at we are weeks the And There to analysis, compared response Also, for meet compared regulatory interim are study support more including a of maximal a to an study approval we washout the the

blinded the on we from we remain patients a will but look We primary this out interim And and data able to the proteinuria and disclosure from need as anticipate track providing do mentioned, for limited data a As assessment be anticipate an reduction outcome you of we providing update for in basis to completion. month. continuing through assessment have interim endpoint we maintain do being of eGFR not PROTECT two for community. study the nephrology expect to Eric provide years, forward integrity We on to values. the a next on top-line baseline,

profile if have over forward cohort planned dosing reached of next to if with dose we pegtibatinase our have with Lastly, pegtibatinase opportunity program the benefits. providing top-line establish generated our or an program to patient before and of qualitative an to a a the the the development tolerability additional update. pleased We or now last profile PROTECT look highest We in appropriate Overall, to supporting Peter? understanding update data, quantitative readout gains call safety of for year-end. maximize advance of either and from study sparsentan potential the turn comprehensive remain further sparsentan’s Peter into for the pivotal to strong study determine cohort. We excellent month. progress. commercial of the the gaining currently to utility enrollment I’ll anticipate a the we treatment a more from I achieve interim the and pipeline, the FSGS,

Peter Heerma

Noah. you, Thank

We the same for from with commercial the half period are underlying resulted very the strengths the provided lost the in of our organic in approximately in XX% performance second in first which over ground to up some X% organization business and of quarter, approximately These pleased year the us our last expectations. make from line with year. our was quarter, organic growth, growth first

-- compliance all the patient second strong During continue products. to we strong patient focus see quarter, demand and across approved

resets. with our of gross deductions We at the which net insurance beginning year also occur experienced normally the each of normalization

for see Thiola through foreseeable new the generic important initiate future. original the continue second during hepatologists, Thiola we anticipate able being treat formulation there We EC. the this the entered the half we and version that will therapy demand acid portfolio gross to experienced on an of Regarding of the quarter. we new to during minimal mentioned in to Eric the further for expect currently the year. patient our balance Thiola pediatric unfavorable impact Bile be continue and impact continued patients year. is this, we as from an Despite and the to And our identify earlier, quarter, market product, revenues providing anticipate treatment choice of

of to approval Albireo’s recent of pediatric see subtypes first pruritus approved hepatology were the we treatment Bylvay, the all pleased the PFIC. space, in Within

new part of help with people co-promotion we options to Albireo, forward As treatment look hepatic living with limited our established needed for rare leveraging this these our agreement expertise to much deliver condition.

optimistic the proud year. opportunities with work We to am this of also in with on first engage our commercial Cholbam. will I our provide specialists launch that of treat are that Overall, those additional progress half

treating strengthening maintained have identifying further new focus and our We on and capabilities. patients

most call ready by organization for turn continue work early We over goal successful to financial to and nephropathy year. first upon to pre-commercialization in U.S. IgA the FSGS like as now in to launch the I’d as sparsentan our next foundational the building with update. the Laura? of support Laura

Laura Clague

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

expenses, of activity by and increases of development launches primarily clinical in programs sparsentan. driven potential expect support modest year, balance of and gradual the continued we For operating to our the

support Our financial foundation strong. to this remains activity

balance final as to investments neutral For the the the hand as at total use, Not the Eric? into over the million Eric early will now of quarter expected sparsentan’s operations as of cash our our receiving of and of I cash second XXXX. tax comments. nearly generic CARES launch activities well back quarter, a prelaunch to Thiola on to pegtibatinase consideration which is development, Act, refunds cash in additional $XXX.X including we takes were offset business into result the support the related of end operating version program. impact his current advancing This our for closing call potential use. and

Dr. Eric Dube

I’m bring Thank support will Europe in with the supporting have you, submitting the We to of in our balance treatment of priorities half incredibly position organization. next a application remain proud and which this year. year, us results a who remain the opportunity we FSGS IgA to our nephropathy. to an generated our by month, pipeline of ahead Laura. innovation we further as in patients the In to new on first options. end Furthermore, growing year, studies and as us decades PROTECT the sparsentan to the our the that living sparsentan and sparsentan we patients upon that ahead. exciting this of of rest this CMA for that can build by sparsentan to and our will execution deliver Through be confident will desperately potential potential evidence IgA patients we the to the remain first for value enable the of need our coming landmark new continuing non-immunosuppressive focused in excited for the nephropathy, with team’s patients of continued path look path treatment evidence with our we U.S., and in standard deliver forward ultimately to about FSGS patients have

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

Chris Cline

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

Operator

from your comes question with And Schwartz [Operator SVB Joseph first Instructions] Leerink.

Joseph Schwartz

about an the the any was have you for Thiola patterns that do you from do XX expectation a converted EC us better has expect much on changes hoping the Thiola that EC? -- generic impact sales model much. franchise first-gen first approval the commentary Or will impact of that gain in back can generation the understanding decrease recent how branded to so Thiola switch Thanks from the unfavorable prescription actually from first you in color to expect generic far? the a I just patients Is your offer to for your franchise Thiola already everyone. formulation? you Hi, help so unfavorable formulation. of to on And of your could next the detected you’ve gen? that And over that months revenue any a

Dr. Eric Dube

to thanks us Joe, for time, questions. that there’s actually generics between we will little EC. since bit been do evolve. how a Thiola talk the that’s for Thiola just and I’ll as of so much saying available. this place, to your around split what That very question out that erosion little it’s believe start that And I’ll we’ve project evolve reliably by little been And Peter seen and impact our very things the uptick take as about ask difficult might time. that really given business might the said, at well

Peter Heerma

for be generic Thanks, knew Eric. at that And we Joe, facing could Yes. competition. mean, time, certain question. I thanks we your a

and of year, patients scenario. services we patients we In for have to both those have well So patients. treatment are been identifying in for already as that as those our new in this allowing performance, that pleased retain continued respect, with the first been very having the planning half

but second the Eric’s point, in early it’s far. there the be so that impact an have mean It we to limited won’t year, seen doesn’t of half days. So impact

as the regards XX% to understand with have the So, may but than have most Right generic see. will those and in the have on the that with the we with be several better identifying in the are question but in how ability we like formulary the in we years, we formulation on have less understanding adopting original play to a well that as to product. to I to I we sale original in we our patients the your last better of now, providing formulation still patients Also built support think the new infrastructure version, patients for new how dynamics and remain confident are out. regards formulary get dynamics said, rather that that the regards over

and seen, we be a additional be. So the be it’s just what time impact give impact may the will to we perspective will closely monitoring and later point, at

Joseph Schwartz

helpful. the super talk this you occurs also meeting to possible? yet? Will the And could order Type for investors I if or interactions FDA That’s you’re preparing was it been about update in upcoming make scheduled as has after you your your as with wondering persuasive minutes? how receive meeting And you A

Dr. Eric Dube

was I on mute. that. about Sorry

yet have Joe, the and has So occurred. not we do meeting it yes, schedule,

So the is preparation. talk we the that I’ll bit need unmet understands reinforce, believe FDA and ask Bill this say certainly within I a we space. to high about What can

much, very are talk Bill So goals how they’re preparing. and about will our what

Dr. Bill Rote

patients. Certainly, and details forward. Eric. one briefing going and study, your a work the a the agency runs efforts around the ask meeting to moving made each are The look you plans discussions, for process that us just of first proposal give around out from the of specifics analysis was of the next is the the would preparation out book that begins preserving with detailed agency integrity that the into this positions study our information How at years us of specific the give of ongoing pre-NDA and of the statistical in our half for an submission about outlining specifically, given year. second the of with eGFR Coming providing and how of the two

the that’s bulk the the forward of been this with discussions and those quarter. preparation, agency we to So look having

Dr. Eric Dube

the receive the that of once interactions minutes, to these we and we alignment on full sure written public thinking make disclosure question, the Joe, will out to a close in other your meeting. provide to understand plan on Thanks, just after Bill. And we’re just FDA’s part regulatory

Operator

Carter next And from your with Gould question Barclays. comes

Justin Burns

for on Carter. This is Justin,

to you of the with you’re First of reads maintain patients that substantial to have amount study, integrity need of one but of PROTECT, it understanding the that the worth to data Can sort a changed eGFR year’s going given month. when on just going freedom at all discussions thinking you’re potentially next those sort of And you’re your qualitative ongoing around have with give messaging out you that to eGFR has characterize trends? with much FDA. how if

Dr. Eric Dube

Sure.

we’re update sure understand any seeing make as be the as on that we’re based they provides our much are say thanks that as be to question. the going trial. that’s to the and may But to what ongoing DUPLEX as helpful alignment that for FDA so the how well the the then provide we practice, possible aiming I’d view data disclosure guidance Duplex Justin, from on So that bit to integrity from quite be the our type we’ve And disclosure consistent though, the the on of in able interim. maintenance may trial. of since some interactions we status FDA on learned of a just with with believe that and qualitative to ultimately, I be of

Justin Burns

based stoppage next should that filing? was meeting one Or Okay. Awesome. review was lot there filing occurring DUPLEX, additional And a them that in you share or one that of on then be of sort information the to -- around year? we ahead the with plan of that first. that EU that any you’d quick mid- decision sort Is shared that be just given

Dr. Eric Dube

I over turn So, Bill. will that to question

Dr. Bill Rote

Yes.

that eGFR our seeing share FDA the aware appropriately. half package the stoppage FDA. additional the next year. to, that of made data And and So additionally, there’s a to in was The provided with times in clock first discussions period interested of the as eGFR the that progress the data of also same EMA and we plans interim out saw we you to the data, them alluded EMA

procedurally, the -- that time. can at information that review add information data, those point we We’re those in -- to

Justin Burns

the Great. progress. on And congrats

Operator

Your with Bayko from ISI. Evercore question comes next Liisa

Liisa Bayko

you that on are generic And patents Thiola us scenario? you of you quarter. through implications what Good a walk that? Can for if Can do EC? get comment there any possibility the

Dr. Noah Rosenberg

we don’t certainly of There imminently. not Why possibility we’re take is Lisa? Thiola such to one, activity generic I something that but It’s possibility. a will it Sure. a there a again, aware monitor, that is EC. that is continue But for

We office may exclusivity patent, patent in is are patent existing with been -- we continued EC and then Thiola I if would point, of allow of that the that for for of continue that coverage engaging the that, granted, not certainly granted, to given process upside be At any business. suggest as that type an this has newer considering to formulation. yet that

Liisa Bayko

period crossed a will people that FSGS, for help of how was Okay, about on that having PROTECT, see over. X-week coming point period proxy placebo, then and just study, for And was to my is an understand great. of the standard And the ask, that, it’s sort I wanted up. treatment the a and granted for then data really there something time in ahead tolerated I to view, of weeks which excited there XX of an like was maximum care,

of run-in took So what Because took about observation there eGFR, that you which dip to think are recover. we actually -- you to talk make how in vis-à-vis period? the sort was about look about it that? a pretty Can of lengthy weeks maybe XX like

Dr. Eric Dube

I’m think, that make I on And Sure. you’re clear sure eGFR DUET acute Lisa, X-week is to study. the referring just the from question, on dip crossover the that and

Liisa Bayko

Yes.

Dr. Eric Dube

Okay, great.

So to want this take question? Noah, do you

Dr. Noah Rosenberg

that different three had just sense reminding great design It’s we in was DUET Lisa. I’m doses. question, Sure. that the a

we So XXX. at and XXX started XXX,

ERA the having I ACE/ARB may angiotensin in tolerated that of have blockade. going on across right And So show those then a we -- here, coming provide some to the addition impacted ability more and patients into think right may believe, way lower doses with into dip. maximally again, clear top of of the irbesartan,

to So, answer question. your it’s design a slightly different

Liisa Bayko

how Sorry, the quite link. dose didn’t relate? make the does I

Dr. Eric Dube

sorry, so blockade into Well, in sparsentan DUET, period. going from not XXX of -- angiotensin to say, let’s a was from of dose, higher they It the just doses sparsentan remember XXX went going -- crossover irbesartan. it’s when to matter or from up

is study saying I [indiscernible] an there’s coming saying into we’re DUET variability -- you’re the so, there to what the in as well So, addition think, as impact of patients Noah, across going the the tolerated. different purely sparsentan looking and -- the introduce levels, more and of have do dose on you’re blockade what that can endothelin top to variability variability at

Liisa Bayko

As were XXX. might and XXX I you remember, have gone the doses to and you then XXX started like

that out and effect, that that probably So I right? guess, some time, lengthened took hemodynamic

Dr. Eric Dube

Yes.

I saying. I’m what think so. That’s

Liisa Bayko

then Okay. for just last And question me.

you get the to going going think us of just looking but For dose could for you’re looking about the there? a understanding XXX, to you If another be to highest you don’t are what in as you data? a What dose looking exactly give cohort. kind are product That’s for be much, determine at? we to mentioned

Dr. Eric Dube

Sure. Thanks, talk really question program to to I’ll excited evaluate what that about Lisa, we about because this bit X/X. in we’re as Phase that planning Bill And a for well. ask are

Dr. Bill Rote

first-in-human for and Certainly. course, safety of With study, X/X and any the this is, tolerability. Phase molecule. is study this Primary

homocysteine that, changing is basis -- What from we’re These the patients on homocysteine you many, these low is Almost in methionine. in response measurements, patients those readout question really dose-dependent I are also in markers. an and looking concentration a to by Beyond overtime pegtibatinase. seeing biochemical the and looking certainly your plasma. the think of repeated very on are heart high within of troubled administrations and are very at efficacy there

term, looking studies, patient can the what is term, the at would is short variability the in in that we enough of for that very allowed the homocysteine adhere the lower And of in that a the patients forced point things patients one diet can plasma plasma also aspect. as patients These or ability that of from homocysteine And some overtime. between very to in if perspective an and huge these the able so quite life to durability part, lower individuals their population degree might and methionine their for protein within diet. looking of at -- benefit to, and adults. the that’s important is to long to response and in difficult children a response high levels to what be us to predict ameliorated regardless but in that to of there’s be in eat these where normalizing there of we’re the liberalization whether going clinical they’re well even low quality very piece that in be poor at patient as is part that of we’re mix are the future homocysteine Ultimately, lowering unpalatable the is, be

Liisa Bayko

Great. Okay. really That’s helpful.

Operator

Your next with Blair. Tim from question comes William Lugo

Tim Lugo

following the a of on health homocysteine endpoints data up much function lead in -- there by will and can the how is some a much data cognitive the going at. of looking ocular you’re just give -- how to the maybe what of Is year-end, us of guess, you sense kind maybe the to natural suggests? history I pegtibatinase drop drops expected And

Dr. Eric Dube

I’ll Tim. there’s patients, data I high Sure, XXX turn the Bill I’ll But of is considered help there. over. is work linking but mean been we’re give under patients, there which a link that doing challenging for levels have trying national and some is a about get of that threshold to to then the XX understanding link. ask talk history these our bolstering in and Bill really really study early level on incredibly for the homocysteine to many or what

Dr. Bill Rote

good, cetera. really at literature in and to a need they’re is dislocation, that that there treated you that that’s question it’s in development, part that the prevent down And think, teens. guidelines it’s below is and taken difficult program -- cognitive get of clinical see because In these thrombosis of XXX differences in know lower the the is order but element going occurred to not and diagnosed a in homocysteine most reduction of see that certainly the are treating for cognitive already how the much scores, But comparison, changes those not do is question, certain have to normal forward. to Thanks see in homocysteine, investigation untreated and the you’re they’re the And a are retrospective micromolar, to that been treatment until huge that place try of individuals. what’s Tim, the about studies patients clinical changes we IQ rates don’t know here. et historical future The probably a in I that in treated already asking in off drop today do than older. individuals Yes. because reverse. patient but lens anything better events past essentially there’s out question, challenging look do and going have and are you We have rates

to that benefit what study. aspects on be lower? we among if And the working the and all is lower what’s history from on able of we’ll them kind and there natural keep based data and the are So, it drive so, it

Tim Lugo

submission to guess data same is -- broadly and up Or data just, the seems the the is just changes the a the interim than of the Or about more took effectively guess, of timing with interactions clock the to with timing what this the I the just line there It function like Was more merely Or the take EMA, back sparsentan, EMA submission. I EMA in during Okay. of following of occurs differently can FDA? in you merely I was the you the which it the kind is any you’ll as guess, FDA? XXXX? the And this of -- the is looking going the a presentation? bit would stoppage, discuss at it filing? that

Dr. Eric Dube

one? do Bill, take want to you this

Dr. Bill Rote

Certainly.

saw different the same the full think presented can processes. both They question, I agencies it of answer package. saw different was the data have They first the the way, same and easily. part I cut, data your interim but essentially

FDA we proposal the as provided for them And advantage their information they knowing made data the to as team supplementary our a be where EMA of about that And made discussions data would our with it of so they or that so during to different. the -- and thinking that to next proposal half informed then with We the completely -- feasible outcome filing were have the the so the on known the additional first FDA. the those well had process, align intentions stop. shaped of us in year. review are the to had clock the process, And

Tim Lugo

I’d the I, guess, thoughts competitive love your larger II data we there? can PROTECT. Phase have kind to I ahead recently that around quickly hear And understood. relatively players Okay, the seen, to of from of I some out guess,

Dr. Eric Dube

Noah ask Sure. thoughts give development. other to in I’ll treatments the on some

Dr. Noah Rosenberg

Yes.

your I [Technical so think in ongoing deal studies number really this now about Difficulty]. question, what’s exciting a is there’s and of

Sorry, there’s studies number on. a of going

out, there’s one, work and Number think key the number immunosuppressive is is the ongoing you of being two steroids that’s still pointed an camp, So some lower most done points. dose studies, currently as of are that -- even a of realm. I ISP within the

in there sparsentan one still I concerns of of non-immune strengths issues. opportunity a think so think suppressive for therapy sparsentan being and potential treatments therapy immunosuppressive steroid tolerability because those there’s the And I is, for an long-term huge have and safety non-immunosuppressive the

-- that think borne I future out the long a So, be safety term. definitely from in will those standpoint

is I ability standard think be sparsentan to of a really profile and the with really -- treatment. efficacy has its safety care

main the think us. so And that’s for I

why, incrementally. used we and don’t is it we’ve sparsentan therapy reason see seeing piece any immunesupressive of use still of that sparsentan, other and top effect The an of on seen

So, used of sparsentan a why reason well be top complementary as see in a treatments. on manner could those we don’t

approach. our change really doesn’t it So

each really we the mechanisms but good for ultimately far pretty in We’re and these be lead again, patients, different think hopefully will it’s other. here complementary to

Operator

Greg of America. comes from question Harrison Bank next with Your

Greg Harrison

Another one on pegtibatinase.

as the that talk it and your asset? size deals see get but comes into of the kind now portfolio of on consider? your when in can fits just to to this focus of how future wanted account into considerations get a would BD you thoughts was commercial it what and happen I bit what to And wanted what if may for sense far know Just BD little activity. sparsentan potential you’d about your you the future as you take

Dr. Eric Dube

Sure. for so Greg. Thanks question. the much

homocystinuria So we are plasma about U.S. with that, patients currently look underestimates at there’s commercial -- regard real that we potential that the patients the we go just the many and And states. are of undiagnosed in the that years think them to And X,XXX help that’s of opportunity to for of methodology X,XXX, the these that that’s addressable. given homocystinuria, if as times, believe Europe, go in used, these because newborn screening undetected. patients, many having part despite often

will availability think that, and so and increased awareness increase. that we And only potential of through clinical trials therapeutics the

rare to footprint. that nice currently And relationships but approved. focus CHOLBAM with therapeutically, plasma example diagnostic think diagnosed treated at early We to in where pegtibatinase serves at our we focused the our very we specialties If as Europe the patients pediatric team is business, geneticist be hepatic are nicely leverage oftentimes, that with tertiary CHOLBAM, rare disease. So think there’s that will in it under similar And be and And about disease have and that looking different think centers around homocystinuria we going odyssey and these are real we’re might late-stage going has disease. a opportunity efforts fits strong or in of if commercialization where that rare U.S. that very there. through oftentimes bile be the stay care a care acid within treat a the to and it’s helping for navigating It’s very clinicians that, into development, think they portfolio. is a capabilities across that and diagnose of disorders we on a we disease and renal, we’re development rare our going to continue business rare to able metabolic, patients. how

point. be specifics look but it’s size more evolve able to that to can going provide continue that be terms will we of that In to something at, of time. certainly at not something this It’s deal, I’d over

Greg Harrison

Okay. That’s helpful.

Operator

with next question comes Your Canaccord. Michelle from Gilson

Michelle Gilson

I one clarifying question. guess

or really FDA discussed guess, the next interim mechanism guys a more your look follow-up. from then have in to an interaction, reliance showed greater Am EMA on So, first that that the I I I I this I they your what this, half you surrogate there guys as correctly guess, may was you a the EMA, in was in difference like second and guess, if it And to that sounds have I proteinuria approval like you eGFR other, for guess, allow I conditional, gathering of them? file, some around as don’t willingness a you comfort discussion I the with guess, key year. it know, Or data between, option. it’s interim do or is the take driver behind the EMA’s that

Dr. Eric Dube

you to like Bill, would Okay. take that?

Dr. Bill Rote

Certainly.

same I but take through of two agency go they think the see to did same processes. function and taking Ultimately, independent we -- supplementary to strategy because their were the they’re that processes, it’s the the had next year. responses data. performing the aware they it’s data compare agencies the different they and the and difficult eGFR the proposing are benefit They with being or of review

allowable. under might that what how put was was so how key be we there. difference, point, the you process mechanics work biggest And data really difference do the That at review of types it in done with was into what’s that of and the really around reviews and that permissible, and that’s can the additional when that

Michelle Gilson

factors? you that wondering Got you this confident other the from or profile the would anything either a I’m seeing to it. would safety you really next I prevent And really going the if looking guess, you be you there’s of tonight. dose? could And probably -- you based lot target I that TVT-XXX, that seem effective just I that some there are on blinded expect dose higher the on achieve dose or is pegtibatinase in in are clarify, dose go-forward product you’re wanted interest And for? or if data -- the then, guess, been I know to is

Dr. Eric Dube

take don’t why that? you Bill, Michelle. Thanks,

Dr. Bill Rote

at we’re interim issues. that to are look dose data in at current the that be look Well, this of aggregate. At do cohort, data the safety have there we completion the look observing and we at blinded study will study, a point, an safety is dose-limiting blinded concerns no this the but

Now, full I say to having a of from that not access position the data.

by that’s there’s qualifier that believe where the there to going be all dose, lie. limits or So, that’s likely but dose data not next the be to do able determined but analyze we’re biochemical and We our on it’s going the to forward. a this data really when

later to able at on a forward. this be should you qualitative give how we least that update looks going year So,

Michelle Gilson

could little Can you on the I perspective to dose more, or next go-forward? I some provide guess dose are that so why just a out confident into probably figure be why? that if trying you’re the going bit dig I’m this to

Dr. Eric Dube

it’s say -- wouldn’t confidence as it that the I read I Well, be would it dose. will

well I and we’re the I blinded making. on think expect hope. what laid cohort dose enrolled, would seeing that criteria confident decision now think we’re The it’s and out. was design a we -- basis, as in latest have We Bill fully that we’re mentioned,

execute I for we make sure to how we continue want dose or and this So, think us, it’s the do make right higher. if to go is we the right as whether decision evaluate we

everything that the And on see drove that this. that’s characterize thinking is data of a the way think and very our that the hypothesis we program. development So, I preclinical would I lot with really consistent the

Michelle Gilson

on Congrats quarter. the

Operator

next Your Raycroft with comes Maury Jefferies. from question

Maury Raycroft

their the RAS you’re progress. patients they on and treatment. from III, I was washout without enrolling optimized for so switched IgAN wondering irbesartan over to study, the Congrats inhibitor the for Phase

irbesartan PROTECT what when in And how will latest secondly, So irbesartan based their in more UPC III worsening talk patients then have about can you starting Phase these or are is your outperforming on are if eGFR the they DUPLEX? stable? perform in on expectations

Dr. Eric Dube

pass over thanks Noah, Maurice, for much I’ll you. the to this questions. one so

Dr. Noah Rosenberg

Yes.

patients question, dose Maurice, So in I correctly, ARB come required when PROTECT, treatment. or and maximally ACE these first if heard PROTECT to on they’re in, you in tolerated I about of be the was the so washout think

bit. you of there, bit us be may have the the optimal a could the currently on don’t top so an it’s are or to board, really is idea when they [indiscernible] addition just to the which maximally, there’s tolerated the we maximum so between not of is the But which the what washout also little optimally presumption of with And blinded, then a patients. right, a to to those on we’re know -- switch doses ACE/ARB that difference whereas those potential not show happens in angiotension ERA help a and irbesartan, lack confounder have

the What So point washout. was second, second I think the just first around again? that’s you the question repeat the can

Dr. Eric Dube

or to on PROTECT, think criteria despite go ACE before but being UPC max just I their just risk. The going are is at eGFR. on these that, I is we based considered does add. to and presumably important was aspect reflect which Sorry, patients other tolerated around They’re they still irbesartan really that their ABR, the inclusion/exclusion question was the for

controlled patients guidelines be treatment by considered are that community. nephrology not here these So would by the well

would background fully that function in expect what be about would of just this disease. understand there renal a as over time, a we decline So, their continued we

was we that into to in and question point irbesartan. then can thought So go the important sorry, around expect what an Noah, Maurice’s mention, to I

Dr. Noah Rosenberg

So yes, what’s expected in irbesartan.

if look difference. controlled on blocking a with setting, the So to proteinuria XX% or literature that across the front, in about reduction you recent the within X% at a would most I say, the angiotensin seeing up we’re analog XX% studies, but in teens showed

to similar dose. I is is that or so we’ve studies think optimized to And see piece the -- and we’ve something dose the here, possible irbesartan that again, the the maximally but other somewhere think close it’s important X% between I tolerated that which got we’ll got

on able ultimately of of really thinking goal. to we be be less ERA top test we’re That’s angiotensin hypothesis that there hope the there difference So, would the and a II ultimately.

Maury Raycroft

it. Got That’s helpful.

Dr. Eric Dube

There accounted Maurice. we all -- covered... in thing, that are have scenarios and more one we’ve our for those plants, all add I’ll And

Operator

with comes Securities. question Chico from Wedbush Laura next Your

Laura Chico

frequently first I we’ve guess one, gotten investors. from one this

wanted far learned we’ve that from clarify. the so So, contrasting what and others competitor, just provided your It’s versus from to like [indiscernible]. eGFR around around I disclosures you DUPLEX

towards what around see of eGFR FDA’s also a to, I’m guess, And in then your you then filing do from quick to surrogate their disclosure I own So with kind respect reads follow-up. but strategy? commitment the have wondering IgAN endpoints, I

Dr. Eric Dube

Sure. thanks I’ll one. first, take trial much I’d Laura, are so for question. different. this the the say, designs

on IgAN treatment completion the phase the of companies analyses based those disclosure in other those trial. while the eGFR so are from have of And data disclosed trials,

DUPLEX studies so PROTECT same ongoing way they are. And that in and not the blinded are

so And there’s of way the certainly slightly think a our just disclosure that that, ongoing different regulators. trial. has been with from engagement managing I by And informed data of an continued

potential going foremost, or cannot positive that’s and been We sure. for to confidence building them. the to incrementally confidence FDA the with within I’m lacking have decades. I building. that it regard read space certainly top and just for it’s speak be innovation mean, not is any Reflecting results certainly see through, for quite that great conversation not, with first had to incrementally the Whether the nephrologists, how we Now, the

the in that that improve also out that’s eGFR to data of come this improving that proteinuria over interventions space are All show continue time.

what hearing So we’re from community. the certainly nephrology that’s

that data we’ll think I answers work will sparsentan question. that package Hopefully, and we’re continue that optimistic demonstrate regulators. on with to with your

Laura Chico

A going a similar interim on have would didn’t to to if but be after on that, one -- I in for this broader, of filing It sparsentan to where is your if does, And just meeting. the assessment. perhaps question have Would you guess, if still had you, to I the Eric. understanding seek following a if And level away have and kind higher more work a out an agencies accelerated right then pursue I follow-up, accelerated an you bit look completed? cannot strategy IgAN, related Type FSGS filing guess you strategic it stand you’re approval to

Dr. Eric Dube

for the Yes. you Thank question.

challenges reason, face, laid FDA Type that the this just when A our confirmatory the cannot continue and that think data scenario alignment trial undeterred the we out. we through I think going that the we’re next meeting, have to to I said whatever endpoint, out in some with we’re month. for I PROTECT we’ll in you If certainly gain extends on

to patient community we’re how we’ll but de-tour the from but let or blinded, of we’re to similar them of committed going see we we of to these programs to any is continue and types are, not these and space. accelerated programs need aspects the how so clear interim, approval, the important We’re for with pathway the once if us. hear the see to opportunity I have able that be conversations accelerated

Operator

back I’m time. would turn to conference showing no now further I to like Chris Cline. the question at this

Chris Cline

assessment great Thanks, we next thank you forward We line Great. study all have from Thank results again look the PROTECT joining when you to I the for a Jerome, today. proteinuria with hope interim and top month. rest and have of all us you. speaking you week.

Operator

and this gentlemen, you day. conference. for today’s a participation, and Thank have your Ladies wonderful concludes

may You disconnect. all

Travere Therapeutics (TVTX) 31 Jul 212021 Q2 Earnings call transcript