TVTX Travere Therapeutics

Chris Cline IR
Steve Aselage CEO
Neil McFarlane COO
Laura Clague CFO
Bill Rote SVP and Head of Research and Development
Tim Lugo William Blair
Liisa Bayko JMP Securities
Dae Gon Leerink Partners
Gobind Singh BMO
Call transcript
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Good day, ladies and gentlemen, and welcome to the Retrophin, Inc.

First Quarter 2018 Financial Results and Corporate Update Conference Call. [Operator Instructions] I would now like to turn the conference over to your host, Mr. Chris Cline. Sir, you may begin.

Chris Cline

Great. Valerie. you, Thank and XXXX everyone for Retrophin's joining Good Results thank and Quarter Financial Corporate First afternoon, you Update Call.

the McFarlane, Laura Research and Joining Executive Senior Vice and on call Head Steve Rote, are me of and Aselage, Dr. Officer; Bill Neil Chief Chief Clague, Officer; Development. President Operating Financial Officer; today Chief

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

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

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

Steve Aselage

established year. of everyone, the important in X regulatory the into to bit is EMA Most feedback from will and directly Bill forward already and successful. to proud Chris. a the to this have today's shortly, and the to call. US to for our trial they interactions the group efforts announce be study we taking XXXX. positive obtain a FDA steps initiate accomplish much the Europe IgA that for but encouraging in both is the into sparsentan the Thanks as more I'm afternoon, an and as extremely move registration are to takeaway ability joining of that in Good detail path that returned supports go development recently This nephropathy. with and later research here pleased And Phase enable fronts. progress outcome single you thank us I'm on on team multiple if we most

Phase bill. and for of initiated his and great sparsentan this team a X Bill the and to DUPLEX credit deal for in pioneering April moving up the get deserve FSGS we study Additionally, early groundwork running. quickly laying of

second Looking registration forward, DUPLEX to additional on focused we are the of half targets the end and opening readout enabling reaching study enrollment of sites XXXX. interim in our point enable

we year. FSGS for for exciting achievements, progress with Given is having these start and patients rare in conditions nephropathy not IgA clearly are these to of nephrotic worldwide. the us, but two just sparsentan studies pleased The registrational prospect enrolling also

study reached pivotal in at safety also FORT with good our our We to progress monitoring fosmetpantotenate enrollment PKAN. to made review recently target pediatric interim enrollment. a for data open board planned adults We in enable

previously As enrollment this a will later guided. we pediatric begin quarter, we as anticipate result,

accelerating dose enrollment the as working development progressed into paradigm made potential portion study has of the multiple program I phases. first we dose quarter, the treatment forward and the In CNSA-XXX CNSA-XXX patients further remain and single to PKU. its with completed ascending for through improve about impact look have teams the study of positive Our the the ascending continues we FORT to balance and the optimistic year. the a on planned, Phase

early data expect We to the XXXX. available mid-year continue initiate by study becoming proof-of-concept to X Phase with

now net that meet on to the to from product new update. commercial the start team's turn $XXX over find in million also call development year $XXX portfolio research for and had I million Bill? range pleased patients Our will a of remain to can in Bill growth solid a to XXXX. to guided our track and with sales I am commercial the We for the our quarter, efforts benefit strong first therapies. with

Bill Rote

Thanks Steve.

Let me start sparsentan. by covering our most milestone related recent development to

both interactions week, the and FDA nephropathy. last completed IgA with program EMA of for As our on we positive

key similar, Steve our from is Phase As from directly single agencies feedback initiated X similar nephropathy In exactly same. the also in the numbers to study endpoints the be both to moving patient but expect EGFR. a conclusion trial recently IgA proteinuria mentioned, into evaluating expect we with We strokes, study. broad duration the to the DUPLEX would meetings study be supports and sparsentan that and the not

and path, to our running. that expect to in enrollment of timelines. finalize the more specifics incorporating initiate investigators protocol, with year. and fourth current a our to in to of up we study to the quarter important time, be working project our activity. all protocol this We harmonize protocol position currently guide team about the On Alongside our our would CRO study clinical get and feedback are At is we'll this closely the to share by efforts working

We also results an month. with and upon is study innovative US Europe. the our Phase and from in X efforts positions our gain approval in recently the FSGS DUPLEX initiation X trial our potentially clinical of momentum DUPLEX last us promising to Phase DUET increased builds study, the that

urine XXX one to week enrollment is randomize of measuring expected either see will of after received taking We Achieving modified XX treatment is weeks. All of each steps notably reduction for irbesartan. the in and XXX than assessment total to patients from patients a sites for the with new remission across XX% and initiate sites receive sparsentan a opening FSGS around enroll creatinine to approximately active-control one estimated they've the patients patients. globe. in to proteinuria partial baseline. less the or to FSGS gram study provides are remission defined or proteinuria per greater as protein DUPLEX to excited of Most XXX of X.X obtaining partial modified equal design proteinuria approximately trial gram to interim ratio in an than

of in study. recall, significant you statistically the endpoint X group treatment in this treatment same which As group the irbesartan a saw the between we DUET many weeks and difference sparsentan and this after is of

primary in XXX be portion expect We endpoint subpart patients change DUPLEX is The our evaluate interim enrollment glomerular available the the in conditional which after study for to the proteinuria achievement measured or treatment. XXXX. analysis slope weeks XXX will endpoint, will and filtration of authorization top-line of accelerated expected the approximately assessment is in half second of basis sparsentan Europe. estimates confirmatory the and successful EGFR in United serve that estimated States the rate of of consideration approval interim on in data current marketing of H as of this from the our Based the

expected, Given have forward the in remission a a confidence our Phase we outcome degree of endpoint promising the X high results seen on look the and patient of DUPLEX and modified population study similar the partial pleased our forward study we to and key of in sparsentan we are by to sharing registration the by Overall, two the in milestones. the to coming to we end enroll made continuing towards quarters. advance look more XXXX, progress enabling in studies

in treatment pivotal Our study. of lead program for port fosmetpantotenate PKAN the continues to the progress

study and pediatric we the initial study reaching first initiating shortly As recently trigger the to mentioned an patient medical adult awareness review, safety month. enrollment and anticipate by of to milestone opening additional DSMB earlier, board monitoring that in the data resulting later study's meeting enrollment milestone enrollment. uptick we the met driving is this after The work sites great our the in teams clinical and Steve

we During also enrollment. on impact European quarter, the having first opened which already are centers study key encouraging an

read patients of enrollment remain Importantly, XXXX. a top-line year-end on all around which second track to in XXXX the complete we or half sometime out in early this would enable of study

I'm partners team ascending has I is sepiapterin further the I being difference CNSA our Phenylketonuria a collaboration completed orally this bioavailable Pharmaceuticals developed an PKU. portion The our the and very the pleased opportunity of we in with multiple This dose announced single is development minute Finally, which CNSA-XXX, will on advance agreement in ascending to initiated. conjunction the take form candidate, or between has a meaningful and through to option proprietary of to segments study that joint for touch with dose PKU patients. our make Phase for and January.

the track envision current crossover standard doses is in of study study a the versus with on in care Phase CNSA-XXX months. Importantly, multiple of initiate remains X The the proof-of-concept evaluating PKU. design to coming patients

the readout to X by XXXX. from expect Phase data continue study We early

into we've the advancement results our the acquire a then study As option quickly the we Phase pivotal from would delighted development Overall, positive, across previously, mentioned if the portfolio study I'm and year. to start X our XXXX. proof-of-concept with in choose moving CNSA, anticipate we exercise are to to

fourth a class put and end pivotal has performance enrolling for XXXX. programs of this starting molecules us on in X three the potentially year, Our Phase first studies by track

over now update. me for his call operational Let Neil turn the Neil?

Neil McFarlane

focus XXXX started meeting the pipeline operational serve. with and across continuing commercial Thanks we of progress portfolio, to the on We patients the disease Bill. rare needs while

grow in through of the the next typical impact usual quarter, range anticipate commercial more the to another portfolio. $XXX for we few quarter for XX% turned last years. growth performance the experienced we in This importantly, discounts. to quarter, and strong of over continues growth the gross Perhaps sales, net the track all organic expectations XXXX. beginning valued seen our in similar levels to product execution, to products. the our this $XX and year. our with in $XXX first our of first We drove which quarter commercial to we've new team us of three guided in was from a year to contributor Thiola patient first keeps resulted than additions moving quarter higher Our remain most line what great a meet in our net sales million to in note over on net million product million of Of back

provide stone improve to brand Working identification to for and important in more Coupling foreseeable track filing launch NDA to with our friendly the be Once and identify treated able ongoing our potential an formulation to and growth. at style cystinuria XXXX. opportunity Our offer further this us patient the year free. made gives color component patients is under remain on should new franchise our have conjunction I'm for cystinuria Thiola we the the to newly the of of products patients efforts to milestone, to diagnosed efforts pleased dosing cystine the underpin in an we future of for progress grow optimization efforts we start physicians best help on Thiola more ultimately can An that reach chance expectations Overall, development. currently for patients being the continue continues and remain the confident; we with later subsequent steady year. approval future. partner, with in

sponsored atypical our the acid we remain by use genetic testing symptom-based Cholbam, programs. screening bile and encouraged the Regarding of panel

previously, mentioned we've to can are As integral spectrum understanding bile patients diagnosis of synthesis disorders and Cholbam and acid when benefit further disorders. these tools with Zellweger

expect further physicians on of in to Chenodal, important to as value to study support these believe as population. ultra year. for helps the ongoing prevalence continue regard educate growth throughout In knowledge see We will it patient rare we patients we the about well as provides initiatives continue diagnosis CTX, our this

continue active the through we Finally, to business to I'll could to explore Laura? value the turn organization. call and our now to utilize disciplined add walk remain our that assets to in financials. our approach over development Laura initiatives

Laura Clague

Neil. Thanks

expenses million, to rules, notable year. of reported first we non-cash product first adjusting the with a quarter GAAP In During After made non-GAAP to million sales to a quarter, accounting commercial loss of $XX.X net We accordance $XX.X reported the change of for new million. P&L one grew loss start XXXX. new $X.X of from net our net million. $XX.X for our portfolio the we

quarter adjusted will such million funding quarter the derivative $X.X of fosmetpantotenate $XX.X in recognize in higher first included the GAAP CNSA-XXX. and general for Relevant due administrative well the R&D fair We compensation On activities in first largely increase an to no associated for quarter initiatives to non-clinical is longer increases operational value as of and related to The same statement. with compared The R&D the basis, XXXX marketing last million and related On basis, and expenses growth for period sparsentan development year headcount were same expenses expenses stock-based to as over the the for expenses Selling, first clinical the expenses program, XXXX. of the expenses of primarily on first commercial income $XX.X due products. our higher the were $XX.X changes a non-cash instruments period quarter. increase to to and million support million XXXX. amortization. as in is were

adjusted million disease $X.X National over commitment In Children's our incurred charge of next SG&A addition, consisted the first depreciation six million expenses Rare On to rare basis non-cash approximately for to million one-time for the the years Retrophin and network adjustments at million for a the $X quarter compensation, create Institute. quarter. an were Disease non-GAAP present $XX stock-based and SG&A $X.X the value amortization. Significant of related the of

XXXX, payment provided we non to XXst, securities. milestones the March call, during on use in $XXX.X a equivalents amount related to of covered million that cash and upfront payment and aggregate to due related CNSA, to The guidance and initiation DUPLEX $XX of cumulative we quarter. LIGAN As payments upon cash several payment Cholbam study. a million sales, the these approximately milestone included was marketable the first with and incurred the In operational alignment last cash milestone our had of amount

of anticipate at further than lower over three Steve expected turn back the to programs, our we in pivotal comments. now used we as the balance we his levels call the first closing Steve? advance quarter. I'll to While be throughout we experienced cash for

Steve Aselage

you, Laura. Thank

As had great start we year. you tell to a the can

be in have completion our will to the study top-line for which including formulation and top of registration expected corresponding positive On help results number CNSA-XXX, or of from Thiola. that rest the XXXX, commercial initiation meeting key programs and and pivotal will and half filing highlight we our Initiation value value sparsentan enrollment We growth In these of for X/X the confidence support efforts. portfolio, second to that, expect new our of early proof-of-concept near pipeline support the of the continued of Europe. to years top-line in ahead. we potential the for for research an fourth future, late the of in the initiatives and the results study by the products, study throughout the in in US if early strength and a our IgA that development and XXXX nephropathy XXXX, of Phase on NDA lot have events shareholders. of meaningful XXXX We for and in our a patients expected Phase would and XXXX, focused quarter, with the of revenue remain X drive create

Let me now turn Chris. the to call back Chris? over

Chris Cline

for ahead go and open we Q&A the can Valerie, up line please. Steve. Thanks,


of first from Instructions] Our Blair. comes William [Operator question Lugo Tim

Your line is open.

Tim Lugo

a for nephropathy taking number market. question. Sure, IgA some one around market success the how some If unlike FSGS the sorry of will in immune are does in the approved come guess be the pipeline. the stayed compounds A about that, where quick thanks of pipeline I there modulators time? there other from market? through similar the How on sparsentan a might the compounds or competitive

Steve Aselage

is think to considered sparsentan depress that the many system. are how now immune programs that But know immune clinic market. to sparsentan hard it's to right an not of not the Sure, else suppressant. I actually in or going the development being anything It's for what it make from differentiates does

come we category. with. that's falls profile the think inhibitors being to all doesn't able would to it complement come immune give ability position, market types being which think suppressants, to of into product not the developed Everything that is Different most our the the but an calcineurin immune side-effect where a inhibitors, create go team is in commercial else suppressants suppressant sparsentan So front-line I immune with with immunosuppressant value. an positions that effective we

Tim Lugo

more Duplex talk in nephropathy the two broadly obviously but what I you differences and specifics be go you. between want into you the I broadly study? to will the variation year, thank later can programs? about the at Understood, maybe guess the talk least IgA you And between know

Bill Rote


that of cohort IgA mean with of think to as going different. what differently, have it's I I they high doesn't think than patients proteinuria as The level a with a are easier patient see them of you're more present FSGS. similar as to nephropathy populations

but So on impacts followed looking a an be analysis interim at based confirmatory it proteinuria of we'll based that end EGFR. we still that slope on point expect by

Tim Lugo

lot you X X and for as one this have last clinically next three off development, one business your potentially maybe year, and plate Phase Phase obviously Okay kicking with a on another looking you at you products? looking broadly about at Are looking what you're you marketed guess earlier platform Are Are stage development? talk products? I year. technologies? you business on at can doing

Steve Aselage

of Sure. all see to can We stages be creation that at the maximum where point in development programs look year but the focus to timeline. we at with three there should two value

scientific So about What with I be how through the to we is system. look put illustrative deal we we of truly tend we looking are picky think We platforms or CNSA our at things. very the unproven what technology did approaches. at

when trial right We lot plate our the to R&D our we with is in quickly everything didn't right being but with it going like gave X. without hit that head sepiapterin able a to was own CESA else those two feel sparsentan in talented a very group now research has. what has But team moving doing be worlds, the that given us Phase we themselves, research internal a studies you their forward do You on could program to X ourselves, the the distracting and of on said continue clinical was realistic funded the pulling fourth our Phase deal best group both team. thought which the of structure so people

or opportunities additional a in that team X core to our their distract task from type category, programs So find that Phase of at here don't a Retrophin are look at X if at Phase try hand. we we'll


you. Thank

Securities. JMP from question Liisa of next Our comes Bayko

open. is line Your

Liisa Bayko

things us or obtaining currently terms patients? for marketed in of new net the maybe question. that. highlights any trends give on Gross taking to products like thanks Hi, you Could

Neil McFarlane

give here. to an products on quarter Lisa, Yes. update. quarter-over- three Happy -- basis. continue have to to organic growth hi, Happy All you a Neil

Our on XX% We our gross numbers. our back we to stabilizing that run approximately net impact call, to XX% anticipate the as year-over-year versus XX% quarterly in of year's our gross in QX is to higher previous net. to discussed rate

Lars Brorson

what great, as Thank about penetration those think you. et thank talk IgA How areas? so collect cetera kind Okay, get to position And and the can for of nephropathy? to of FSGS those you about benefit you. for you both then pharmcoeconomic good market kind x-US can you in dating

Steve Aselage

question of a of that and ASN Sure, transplant; financial the patients can good a to benefit is that we life, in only think patients that there's it's quality early but that or was presented do delay we to that have data health do do systems need modeling and essential one things I the it's into at of in but substantial shows improvement significant a if care going that NEPTUNE very XXXX. the dialysis not the

delays think prices We'll loss between part Europe. in savings that of correlation showed model goes clear systems. on into impact, proteinuria kidney I a the come both make that of that that and up US And with in will in the impact could of setting reduction to process and benefits cost cost be function. sparsentan the the


Thank you.

next Partners. of Leerink Joseph comes Our from Schwartz question

is line Your open.

Dae Gon

Congrats Hey, the Dae our questions. for on Gon dialing guys. This taking progress. in for is Thanks Joe. all

me. from couple So

the enrollment second trial? comment with how you trial wondering progressing start can I PKU. is on sites And you Let's then have how is And DUET question CNSA-XXXand currently to many qualitatively patients? enrolling FSGS. on compared if was

have your colleagues recently potential didn't what our the there three you see expedite light UK Coast so sites X in commentary, trials as designation pursuing points there the at site If EU coming hearken was XXXX? spoke recruiting be that PKU clinical going you timeline, the if Phase mentioned so lastly to that later first more UK development XQ up what for the a well online. therapy could gene your of listed? EU RMAT trial, emerges on look the EU are as an since sites to I Thanks. after this And forward? update PKAN patients. of early on in sites are proof-of-concept are data showed and any the needs And West on UK back in on site were April So we one website opening also that of that be then some are we comment continue and that consideration to you in candidate up XX to going Can done the sites CNSA-XXX

Bill Rote

it. I'll you're Okay. once, start and Bill. that take of all This challenging remember is to at me but we'll

for patients. more number FSGS the beginning just open the for off end the DUPLEX site that by with the kick We're June. will than So now are and sites first study up and the currently of XX at that enrollment to be double first April study sites of and that

and in rate With develop and on of CRO to structured sites total a recall that So more with plan idea entirely too focused we're to not on it's enrollment a have move. enough XXX. this the sites, aggressive but that a and sites gives more some and suffice to to you DUET, different enough that's pace much team that's the number comparison capable is on making patients say early of we are much

So nothing coming enrollment in like DUET should months. look on the

PKAN a the to logical European order. Steve question skipping We're up on opening I'll sites As PKU. come the back will. in that I know your or --and I'm for

up until open opening Some shortly. we got will specific of hasn't up through made Those it the be sites sense so them DSMB. are and pediatric to the early

while been of Now some the to specific rating a but there was personnel completed. get It the of specific on around took site that's the all now. open and them that UK training some it's of site scales, and for

Steve Aselage

the of if And I of correctly open believe this sites in there'll European so the recall up another month May. five be I course or

than therapies. a to think a somewhere gene PKU it in some of regard US, think point. I quickly I at company but So virtually and where type development is therapy, at medical slower very as moving we're this gene gone point disease and looking genetic at Europe has the With every

early, are more some developed. are them of Some

can and solve assume don't just Retrophin, to is company back going If you you're that therapy like everything. gene think a sit I

Some we've of got very can of going those opportunity if unmet aren't. programs feel of that within We we're a the PKU to work, like need type proof-of-concept are address some them community. a significant straightforward, correct

has the years it one but that from as proved, whenever So product clinical when the gene one has the years address gene we're here, to that XX now, at PKU trials up, to a not a and and gets now the if second PADUFA and from competitive in for going comes coming worry we therapy is XX therapy finished now. about moment out, looking there's have already date as that something that we're right landscape

Dae Gon

thinking Great. could rare asking if was If could Tim squeeze at sort in Retrophin one and these of look If disorders guess I along I of on tag then you one I fulfilling indications Thiola going and in rare in neurological of Lugo thinking now of fosmetpantotenate CNSA-XXX we going what or question, kidney sparsentan been Retrophin is and buckets Steve, on where where in of typically more thus you earlier. has BD forward opportunities either far? are end not BD about

Steve Aselage

both. pretty for we morbidity, specifically we've clear and or We by mortality that the being be specialists. nephrology, to spots used in to hundreds people four look for sweet that thousands are significant willing with commercial a for the would build have or because on therapeutics of we opportunity. diseases look we but like therapeutics bring worked our Certainly or deal neurology, desire that would in We that the that significant focus guidelines those are We with ability early have hepatology no able intention and unmet need is almost areas years actually to outside specialists organization. right address last or

the that CNSA program now. and fits I think really But opportunities see still opportunities. mean it doing types of illustrative. them we think we And partnered like with we're what of of to this a hadn't those open been we're well. is niche kind other until metabolic doing of metabolic time in up disease. if last that I the on We that's we capable


you. Thank

Our next question [Gobind comes BMO. of from Singh]

open. is line Your

Gobind Singh

Do Hi, hi all updates. guys. I Kim. for am wonderful on the on of Congrats

with I up you could here. Just nephropathy starting two help if guess trial me questions the IgA

the only the mentioned there's going be difference patient real You to numbers. probably

of understand other that into if operating going numbers then might expenses with forward or indication understand the or be maybe wondering the like could that here acquisition not possibly us the present? in it your how trial that you --whether how differences sounds might help you they X Phase Just you FSGS. higher baseline patient maybe four? model and Just be us than proteinuria feeding depending even help of can And upcoming on to whether in three going have results it

Bill Rote

Sure. versus take at is the question first part proteinuria of which IgA that I'll entry for FSGS.

larger IgA, higher gram a to FSGS, you're marked of get per frank translate the comes gram is more translate reason that it X.X you likely the criteria frank patients. have percentage it's with in doesn't lower a about much nephrotic into can why to to it they when variability. per is lot with lower gram patients The entry proteinuria anywhere proteinuria in patient With gram from For studies, correct a up XX-XX grams be is that much of and the level of seen number lower. reduction between a you

So when smaller as change, And well measure. at looking a here you're to a it's you the looking at you're the that powering study, in effect we're while the variability absolute as going the look side of I'll if hand for in translate on a and And helpful. on an doesn't off financial a it less answer patients, necessarily number into population, that's Laura so larger to variable that. for,

Laura Clague


for R&D trials see above expect need arise where pivotal but incrementally can quarter see from mentioned molecule. three run or to slightly and relatively so As are the rise our levels rate, CNSA-XXX decline we as next far currently. current you with may to fourth as a flat, SG&A we remain R&D also expect levels, you planning


Thank no further closing you. showing Cline this time. I'm questions over the remarks. back for at call to I'll any turn

Chris Cline

quarters. forward call. Thank joining for you everybody you Great. updating Thank We Valerie. our coming look the to you, in


does that conclude for wonderful your today's gentlemen, and Ladies you. And Thank day. participation. have Thank you conference.

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