RTRX - Earnings call transcript Q2 2018 | Travere Therapeutics, Inc.

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Operator

Good day, ladies and gentlemen. And welcome to the Retrophin, Incorporated Second Quarter 2018 Financial Results and Corporate Update. At this time, all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will be given at that time [Operator Instructions].

As a reminder, this conference call maybe recorded. to of conference Investor like Cline, to now President Vice Relations. would turn over the Chris I

You may begin.

Chris Cline

corporate Great. Thank second and Retrophin and welcome call. you, Nicole. Good quarter afternoon, financial everyone to update results

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

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

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

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

Steve Aselage

call. Thanks Chris. Good joining today's afternoon you on thank everyone and us for

potentially our trials pipeline in and clinical base enrolling pivotal quarter of solid had XXXX. milestones our another track us the to reached of candidates. the quarter of advance fourth execution and grow end significant team the keeps with achieve our We strategies business. Our execution during all XXXX our Most on product of by a programs on notably, three on to

from This opened pediatric safety event an successful important study. committee was was PKAN. with pivotal we and evaluation enabled study independent of for enrollment the the data by that Specifically, fosmetpantotenate monitoring FORT the in

and quarter half track have during meaningful in to the saw As expected, we inflection that remain available enrollment the we second XXXX. in on of

that FSGS expect the of sparsentan H April subpart with of patients We serve also to during submission. sparsentan both landmark interim The basis assessment progress treatment patients pleased need are initiation better in the efforts off enable the primary in IgA XXX on as that. and nephropathy and desperate the potential enroll kicked a made to will to we I the duplex of in for path study that are FSGS option, we providing am quarter.

the care continue the significant We optimistic also patients. in and to and be nephropathy made opportunity standard improve progress IgA to with of sparsentan for these

directly during trial very we finalization and enables to to are in initiation quickly a We’re for study us study that the are pivotal guidance to underway, of pleased this received IgA year. nephropathy. move into the moving fourth of quarter efforts enable regulatory that and Protocol the activation single quarter

this if nephropathy we for sparsentan, broadening IgA property and of newly in quarter will a regarding position granted issued got use to and come. a coverage first-in-class nephropathy during approved, of Lastly, expanding dosing FSGS FSGS. new as both a sparsentan us and covering the The intellectual as help therapy many the of our patent IgA for the sparsentan, years result patent

the for We’re also gaining further surrounding optimism research collaboration CNSA-XXX.

preliminary X the us study gave move of portions showed CNSA-XXX Phase encouraging both were results. the results ahead multiple These and dose our a partners of completed the ascending and quarter, X planned. of and proof-of-concept During as with Phase the confidence second single sense the to study

XXXX. and line I track That that quarter another to to impact option products. also in base We we business, to keeps team more patients’ his our exercise early continue of with approved from data to and see inform expect am lives to acquire in our in will pleased proof-of-concept our which the turned Neil few study next readout start decision on weeks, top the the CNSA. positively us growth

XXXX traction expect commercial to reach and million to $XXX We the of to of sales in million the our balance our guided range initiatives $XXX gain for net through product year.

been the to the my announcement was Before Bill Retrophin touch through to would short through ago. lead I release I walk transition. included an pipeline turn while retirement a to a to on press like brief has that our the call to take It in remarkable over a of update, moment honor truly upcoming

never tell can have earlier clearly you stronger. of business the been As from my fundamentals comments, the

we will made successor time to has I and Retrophin include new is the usual our my I'm and will has The internal guide great Company search business and is upon the transitioning to the years, confident several to external move ensure organization build team leader is time, that for Given that transition. can smooth will both now and person progress a and continue candidates, that Until ahead. identified forward. begin members Board it Company years over at the the our as past a for appropriate initiated we collective Retrophin success our the to until that and continue lead a

now me the over for his to research call Let and Bill update. turn development

Bill Rote

focused force the currently perspective, made lead the program, X/X our I'll and the Pantothenate of Neurodegeneration during be in Phase research which and or a a quarter. study continue enrolling From proud with for PKAN. execution start clinic Steve. Kinase-Associated pivotal development on we I'm fosmetpantotenate, we've progress Thanks to is

planned pediatric are enrollment required After our fosmetpantotenate the reviewing we recommended adult data to study, to is continued safety profile us study planned for will as quarter, and and strengthens DMC study support the in patients after doing with to PKAN. great milestone of the patients pivotal trial review safety a corresponding enrollment of now have of the supported scheduled six work as or complete DMC's FORT enrolling safety of teams May, that the and continue line to enable steady in in Notably, that tolerability the of The study, around During XX. pediatric approved ultimately the the data pace second our early age the open first seeing in the of this expect XXXX, and to the readout clinical With for we medical the committee available half available XXXX. opening top year-end encouraging and monitoring enrollment and patients, continue the tolerability the for we're patients. in the the study treatment the key sites, pediatric and data of deliver second enrollment. clinical independent its for optimism of reached review completed

is successful to working the builds support both I registration Phase quarter, first-in-class pleased potential Since make that to candidate and study strides Segmental duplex and the with activities, activation initiated through am both few Phase initiation made X X in glomerulosclerosis beginning Focal and At study and our additional progress This a with sparsentan, FSGS which our We upon DUET nephropathy. product the Europe. we've in with of great sparsentan U.S. the far. second we IgA started continue ago, the is the for site been and also study study for pivotal FSGS. months thus the

to evaluate after XX-weeks sites of approximately with Our on modified XXX clinical interim observation. enabling to enrollment partial efficacy team's of patients the focus proteinuria the point, target reach FSGS, remain will initial end of remission

DUPLEX serve accelerated end proteinuria the subpart for of and successful of We point will this conditional analysis achievement the the and the as interim sparsentan in basis United approval marketing authorization expect in consideration States, of H Europe.

the point second in top remains track line this Importantly, readout half for the XXXX. end of on program of

At teams parallel We end and Alongside track progress support Phase site fourth with feedback protocol received, start. the great initiate initiate our our progress Europe. regulatory for trial to on And the the nephropathy. in us strong been settings our the U.S. feedback this activities. April, making to IgA to running we up and then, in received also remain in to of we've that Since align our clinical made year. CRO, of confirmed a regulatory trial single registration the working in quarter finalize study on in approach our begin trial the we a are the to initiation in efforts X pivotal

in As FSGS. our for be previously, we trial respects have the similar stated DUPLEX in we study to ongoing envision many

expect that in a and support will for U.S. conditional analysis submission interim an the approval proteinuria we based on Europe. Specifically,

As and timelines study we of stated data in for our last details update, we initiation. to the anticipate the giving study closer full

become we a for With pivotal first-in-class year, nephropathy. these clinic ultimately option two trials subsequently sparsentan, the running the of will and have treatment end and successful to approved, in IgA FSGS both if the by positioned

predictable PK of second a our X the observed on development humans In data for normal collaboration I'll hypothesis and dosing of volunteers our the program PKU. phenylketonuria, in the with an Lastly, is and for bioavailability has quarter, preclinical of or preliminary CNSA-XXX data in shown CNSA-XXX provide that review the its Phase completed update study, confirms studies. in the consistent

Next the and double is patients in patient be up of sites X coming partner been CNSA-XXX The the anticipate program commencement proof-of-concept the study Phase initiated the control proof-of-concept with PKU. we first of already be have crossover, will study in the will study, active dosing for our weeks. open randomized, label,

at XXXX. will current with early care. meaningful the we top the key of over looking standard the on maximum produce of We readout a and expectation Overall, treatment of to current and toward advancing clinically great forward look milestones. doses versus will that a two and track of care execution had CNSA-XXX dose CNSA-XXX of recommended optimistic expect study in of line quarter be We of the the benefit our programs the all remain standard upcoming

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

Neil McFarlane

Bill. Thanks,

Thiola the and formulation These our Thiola cystinuria identify formulation half commercial we additional which team's progress I’m expected trend report patient the in with track $XX.X by to new grow year. product new for steady a made application, new we the also of portfolio, physicians remain help for to that effective product our the covering million gives quarter. Thiola anticipate us the on progress Our growing in continued alongside sales business. of growth and pleased second the newly friendly partner, Notably, net more in our throughout report growth. combined XXXX. we with in pleased to and of across the I'm diagnosed as quarter. of continued formulation remain efforts the our the patent during an product patients organic resulted confidence of the in balance NDA team for continues that initiatives of brand the advancement filing to filing of dedicated undertreated its this approved

believe and of longevity the the the a great the patient continue formulation new the improve to with if to patent brand the experience give improve to help opportunity We will product us will granted.

the to XXXX new experience, improvement and patient the already Given should plan is be we a for product, transition underway. in expected there the believe seamless launch

relative products last the bioavailability period also Our to grew same year.

seen growth the the quarters, of and While to in not with is was experienced diseases with portfolio uneven populations. this uncommon small patient rare as compared portion previously we’ve previous

We last of continue to throughout XXXX the will balance growth expect year. as we saw accelerate

and to therapies continue support gain Importantly, Cholbam patients new our both see initiatives we to continue initiate traction. Cholbam to

I'm continued our bile utilizing to disorders. physicians The to trial We’re disease programs manifestation Overall, confidence and seeing physicians evaluate sponsor’s panel involvement from atypical the therapy grow and who've had us with the genetic symptom interest Cholbam this Combining continue foreseeable the the Cholbam with effects. EFT trail we've the in gives screening in pleased program had based in year. start for allows for potential liver initiate growth will testing of future. spectrum our revenue patients to the for increased patients hepatic we with programs acid Zellweger beneficial

the initiatives earlier, dedicated continue that our in to growth of the accelerate will half teams’ Laura? now Thiola call guided And to $XXX and I'll we to are with over efforts for to Laura reach million we $XXX walk of will through expect turn Steve our the Cholbam mentioned XXXX. the We expect year. million financials. second as driving range the and full

Laura Clague

Neil. Thanks

second for commercial legal the administrative expenses $XX.X for second $XX.X million quarter, the to basis, second non-GAAP of reported quarter Relevant million, $X.X GAAP loss non-GAAP and of second XXXX. an portfolio primarily a period quarter million $XX.X of million. of $XX.X quarter from lower expenses the We for loss On adjusting our net amortization. programs, million $X.X stock-based On second quarter. due same After the the of decrease higher expenses $XX.X to the same a as for development On The in for and were for second increase related expenses the sparsentan is over our general adjusted quarter. product adjustments second we net depreciation $XX.X stock-based non-GAAP the net During to last sales grew reported largely non-cash in of The to adjusted the and an GAAP expenses million is million support XXXX. pivotal well R&D million. $X.X basis, non-cash R&D expenses $XX.X fosmetpantotenate consisted and expenses CNSA-XXX. to million funding Selling, as were for period to XXXX Significant the were compared SG&A basis quarter for fees. of compensation were of year amortization. and million due included XXXX. compensation, quarter

of had As securities. June $XXX.X XXXX, equivalents cash marketable and XX, we in million and cash

key in the quarter slight with to used XXXX, of programs R&D to our we steady expect potential clinic. our compared to some increases remain and operating to for second we expenses Looking the cash as relatively advance balance continue levels the

back for over Steve the Let to comments. me call closing turn Steve? his now

Steve Aselage

Laura. Thanks,

opportunities mentioned I had organization creation. Company of all of short value three running due for time, end pivotal the dedicated the of diverse that we've built we've our to our position XXXX. In commercial offers fundamentals sustainable business And an advanced a success a programs never and in a of to earlier, of have period employees, efforts the pipeline stronger. As our building growing been of by tremendous remarkably

All treatment diseases with around patients first-in-class significant have for of of to the thousands world. rare the offer which opportunity

value focused vision of inflection and our towards a will growth. our becoming the rare preeminent our community. led key of the advances disease We on upcoming remain will and that pipeline next the by of will execution demonstrate be transformative This points phase member reaching of

the question. now over call me turn Chris back Let to for

Chris Cline

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

Operator

Thank you [Operator Instructions].

Capital of first Our question BMO Do Kim from comes Markets.

now is line open. Your

Do Kim

in on XXX. findings CNSA it safety Could of study a and how question compares healthy Cholbam? Phase First you X the volunteers about little talk more the to the bit

Bill Rote

pretty top don't adverse that both nothing the was that at data event on profile, a groups agent. and benign looked the line clinical either study we were We’ve recollection My have report. out stood in

Do Kim

will running it be And it, be for who Phase CNSA? X, or will the you

Bill Rote

that for CNSA the runs they responsible development. program,

Do Kim

And getting and Could the size that And to population incremental what us remind patient? market? where penetrated far into in challenges Thiola. you’ve how for the the is are

Steve Aselage

really steering that with If both talks the can while collaborative running Retrophin the everyone to CNSA question to a effort in it’s to-date. have study, I Thiola, It’s feels again And comfortable clinical CNSA. make back Neil working senior two want we relationship go is a before mention we’re between from the sure companies about joint to very CNSA been great moving been that just committee people that a and a direction with.

question want mentioning. give didn’t it to was I Neil but to interrupt, Thiola? about that I to worth going So am thought answer your to now I

Neil McFarlane

a is our terms portion there large point, Thiola. that believe X,XXX we of formulation in be we approximately at with there cystinuria and with this our new we, patients that still feel address of of are there we’ll today market, those the So that is out patients penetration where of

Operator

Thank you.

comes from the of Leerink Joseph Our line question Schwartz next of Partners.

now line open. Your is

Unidentified Analyst

questions. Dig is thanks on in for This for our dialing Joe, taking

or I So speaking, on XX me, enrollment know three PKAN broadly pediatric in Bill, what study? sounded breakdown of been between the from patient the remind month patients one only adult been? program. enrollment so, pretty can specifically a us you how estimated And you and is the about the FORT upbeat. the pediatric first it’s and

terms progression on follow I’ve And get And much of is wanted pediatric between difference, terms got disease the the in and Just there the enrollment progress to some couple up. there? a then of in patients? of Thanks. progression, adult commentary in

Bill Rote

does pediatric, adult study not protocol and FORT the it’s between division. on protocols, breakdown silent a specify that the In

So predominantly right you we can now, as been have we’ve because enrolling. imagine, adults, that's who

getting opening beginning subjects we’ve the enthusiasm enroll study. we’re in as a to stated, and pediatric and started. With been it's just you But patients, had good pediatric month to the just

it patients is just when working to around they want it And school of and some a calendars that. has at stuff and of lot start -- like scheduling been

a the was much presents and and way PKAN question between really and progression make there's the think kids. around a the there clear on distinction heterogeneity And adults I too second to in that. is difference that disease progresses Your

a between of progression deal I is there rates patient-to-patient. think difference that of great the

really is So I there don't a I know really that that specific it's think known it's difference there, case-by-case.

Unidentified Analyst

And the I clinical to on we’re guess program, on while moving DUPLEX.

Just anticipate begin open an how much is sites how you wanted study? And along DUPLEX? you to if comment an of once those much the trial you nephropathy number of on I your see DUET? between there DUPLEX overlap overlap do lines, can And guess, and IgA for

Bill Rote

We're not, continuing patient giving the numbers. progress make point, site numbers which or at pace that's But with specifics forward. to happy this moving at certainly, and

we’re as a investigator open are but one significant it compound. their and DUPLEX in because the DUET nephropathy and dealing last world it's or folks, are label a talk that of out limit have DUET. of the like DUPLEX. matter number the much meeting the one of with don't competitive do how very I happy into or is in was for DUPLEX, it We sites either to do and If number site, It's one impact interesting in for are times lot got part of I also two the down that We a sit it they trials. very ones between are for for the IgA the was sites breakdown how as study they And reach much significant co-coordinators, there other. And does in the PIs a they they the the a enrollers studies, at were what their study? overlap And that individual next significant with up had they've enthusiasm that that no on with that The one, patients think to those study. once the sites comes sites. pretty the sign can't really the anticipate that rollover trial we capacity know the enthusiasm with We're that. both. to specific the for ability at does in

I've hope your answered question. I So

Unidentified Analyst

question on commercial the Neil current last my And is side. for

forward, And I exactly guess NDA of that. if the your fruit? that's Thiola the we experience a of with you hanging take-on to discontinuation go would X.X? patients So call amongst guess you I low comment wanted if X.X, the looking deem much them far. guess rates do in What's Can it get half your commercial the filing? who back and I general of you I remaining to Thiola for terms expect second launch of on recapture so commercial can 'XX just the to how

Neil McFarlane

part is your file. So of able the that what first to remaining question to was be

the working relevant parts up partner the to We’re that right cleaning to the and need be to with literature finalizing file in pull our done the now able of trigger. be

an for So correctly out formulation having through out that the to discontinue and as we’ll there, regards in the areas that it’d regard. other there pointed that on patients to the are that and whether you pill that's of be whether patients that's through be waiting it’d And in stomach product. all take a [indiscernible], new empty product number are

will what on to But those on and that the for one benefit still be today, a our be that go that be back patients would new therapy for new bringing readdress able better It longer patient the exposure on tell allow formulation. address half who hopeful formulation been from those to reason what have could and allow who for therapy working premature friendly probably able off clearly to them and look keep feel to We’re patients that to experience we’ll and or with me to we us and numbers therapy you we've those that patient have areas we to patients like came in patients. patients those we another.

Operator

Thank you.

Lisa Our comes line next of Securities. Bayko of JMP the question from

Your line now open. is

Lisa Bayko

what about to more Just we a in PKU. cautious follow the what say, really little be this what deal? bit and just on would a about is you your talk your thinking up objective a amount should drug the Can of study,

Bill Rote

clinical robust bioavailability. actually same so people extend and And with paradigm that There's sepiapterin PKU. to that greater that, the the barrier full forms responders frank PKU around a responder brain ultimately effect cross partial have BHX, that believe that to that to relative on also is those its the CNSA-XXX BHX. should tissue should treated, of relevant BHX CNSA-XXX. is is that full the table with addition standard the patients more efficacy ability amount the which reason care opposed much to of dosing the those can higher The availability, in you be of more of patients The of translate bioavailability higher much larger with vast about are a the a are bioavailability if or blood improved that for as bioavailability number. But have that we a currently current -- really are to And so fully XX to in which of tissue the much lot that really majority compound percentage of basis pool robust is leaves category, on response. should the -- hypothesis much

significant in see standard-of-care. Now fee reduction over question, to looking with for? compound we the the heart We are very CNSA of what your should the improvement

and easily not fee looking patients reduction incremental an very PKU. for We’re in a improvement and significant with observed increase quantified those for

Lisa Bayko

if and loosening there seeing et about Can business, talk to of maybe you talk that for can about any you. know you trying been cetera. any your I of discussion FDA your amongst then those generics to impact anything? some you’re there And opportunities Thank compounds. some about restrictions, has commercial with portfolio, for on look maybe these And trends

Steve Aselage

to our deal or seen a impacts is lower anything drug business. cost. great that haven’t directly There opportunities obviously options of discussion We particular about

the our products or our appropriately treat as years. taken those direct populations any ability government that at, happen degree products. either priced of are worth products have the had having a a be not can't three convey. what to I that over guess mentioning benefit currently they We we we think price for they in looking historically. see increase of can is might have say But we which what our on feel that not we and And for we size grow to the continue anything on it’s

Lisa Bayko

everything I And final line on And Is on enrollment very but It the you’re with FORT know saying end. looks it everything saying end. still that much. still around second just is then towards Thank there? the track more you’re year in question. still completion track for study? like half before, this you so me. Is that’s with

Steve Aselage

takes we've a been it or think the early year. saying somewhere I year-end, either while this year that for late by next around

enrollment uptick the seen a have nice review. since in DSMB We

wrap-up continue year it enrollment second XXXX. early half this late we So to have of year will data believe next or and we’ll by

Operator

you. Thank

William of from Tim Lugo of Our the comes next line Blair. question

is now open. line Your

Unidentified Analyst

especially with in to identifying by you of on France. Any guess, The underestimating places, PKAN. or underestimating size can your a you is the the Street and not I context much? is guys population, start -- PKAN whether me wanted such terms patients little as are I sites if new idea as give at how Street of The and

Bill Rote

is answer is here how -- really conversant don't to that. know a we because we I'm some different the me know true patient accurate or number know we And epidemiology in prevalence. of past, now certain high are, level estimates in mapping of patients in Street’s we do much the regions. tough did I think The how It's true the when that incidents for found the What

seeing FORT are up some opened original patients there mapping. a pockets seeing. stage. is we I that the study that didn't are you What those to numbers trial, very not common we position in disease and to it’s of that sites therapies But number open it’s regions, patient that in we're more by the but rare identified those unexpected. the we don't of with make actual are think at when and see As when study, a patients available, patients estimates call, we're early that's translates this we’re encouraged to, in and And

Steve Aselage

we've get are a just group put people more about and knew And there centers of science small in on eyes patients visibility, on and to better previously. were we feet we've we France As prior mapping did in patient we getting in those Europe. getting liaisons feedback gotten some when the that invariably, We ground that opportunities. medical

we going can't to So say we we’ll think estimate how find wishes in really for future. on that continuing any mentioning worth in to probably go, to I successor company I and work going am to disappear and not high continue intentionally way can thank And well your want it's retirement, patients. to you do my support new the with I also but the

So I'm going to capacity. be different hanging around for a a while just on

Unidentified Analyst

on seat the in Board holding long-term? on to planning you your Are

Steve Aselage

for that that the that plan. how and we’ll works out, to plan but have currently see is a happen We

Unidentified Analyst

guess impressed medical that data, you the or similar on and And Can will Phase something then give sense a sharing PKU. of conference quickly X you were how I with be some us your you can we comments, at And higher times that on based four showing to can Cholbam. bioavailability compared in least to is humans the Any context I you this later drug guess, infer five share? year. at

Bill Rote

will I the data CNSA-XXX don't know publication future. what in the be plan for near

we in from was was was be mirrored effects, the how certainly I as to of preclinical we And studies see, seen a profiles translated saw amount standard-of-care, As what increasing which of was that it's much and exactly standpoint. negotiated that think wanted has for to greater and I we what bioavailability am, both than know, miserable and which a best needs the PK what predictable enthusiastic program BHX saw pretty you to on significantly as and plasma organizations. qualitative partner

Unidentified Analyst

any share the you on frequency. Can details dosing

Bill Rote

dose single One study study. was a

dosed stealing seven the was for days. other one were study. multiple The They dose

Operator

I’m no this questions And showing time. at further

Chris Cline

you, future. Great, the Nicole. on to thank call. progress we updating Thank This you concludes our today, our us look you joining and for in forward near all

Operator

conclude does a you Ladies and program participation disconnect. day. thank have today’s That today’s may gentlemen, all conference. for and great Everyone, you in

Travere Therapeutics (TVTX) 26 Jul 182018 Q2 Earnings call transcript