RTRX - Earnings call transcript Q3 2017 | Travere Therapeutics, Inc.

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Operator

Good day, ladies and gentlemen, and welcome to the Retrophin Third Quarter 2017 Financial Results and Corporate Update. [Operator Instructions] As a reminder, this call may be recorded. I would now like to introduce your host for today's conference, Chris Cline. Please go ahead.

Chris Cline

Thank you, Tiana. thank and joining Corporate and XXXX Third everyone Results Financial Update afternoon, you Retrophin's for Quarter Good Call. A brief begin. note before we

release ago. some having to with provider so SEC press the minutes refer filed our technical XX about please X-K Our difficulties, is

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

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

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

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

Steve Aselage

all you the on us afternoon. call joining Good for Chris. Thank Thanks, today.

has make was positioned growth. Our meaningful to our fundamental and in that Retrophin disease third quarter community the accelerate to marked progress by impact a rare

most preclinical the initiation Phase of dosing with the was to-date, the most the made trial Phase to it our the the priority, from was that the marks could short development of remarkably period of I'm study We to quarter. notable of III pleased of in and beginning marketing commencement as top the pipeline the for significant during time, make developments gone in quarter milestone and in ultimately III our pipeline our we lead a development third We've dosing the in FORT continue Perhaps the of PKAN. strides fosmetpantotenate and perhaps clinical approval.

We the far. I'm recent with of of pleased a study, patient track four at efforts have treatment second the in in fosmetpantotenate are in CNS completion the half on continued we thus also data in for effects enrollment enroll XXXX. our from the with Annual data that publication of the with are continuing and of highly fosmetpantotenate. The be to of to patients seen remain clinical the with physician-initiated October consistent Meeting beneficial treated patients PKAN supportive

advancement the readout. from We shortly, support approval submitted Bill our our surrogate in will H also FDA night, detail but interim continued a more last FSGS. positive trial more III for work The agency Phase planned go upon endpoint in protocol we the has requested just for on into FSGS. received Subpart the of sparsentan statistical we feedback to

pleased as anticipation sites with Our we've requested continue our with to and plan very I is startup study. quickly of our activities FDA as parallel. the back the get to analysis the trial initiating in am in possible preparation made

rare patients use Additionally, diseases, were with example be Week and pleased ago, discovering supportive understanding has to Kidney help enhance able can These in NIH meaningful ASN division strengthen days to for NGLYX just and a public enter of put were first-in-class our Elsewhere as resolve work the of extension of we and patient few Patient This sparsentan toward research potential an FSGS. the NCATS, collaboration positive such the is NGLYX we a presented together NGLYX.org efforts Foundation. community, with potential the that open-label to important great sparsentan and advancement research the a with into of pipeline, may for potential significantly data and very data long-term at our sparsentan. a deficiency. how therapeutics advocates and

While revenue strong growth to with Operationally, our our our build we and and The track developments contributed we of the remain effort in team's execution. will we come focus efforts commercial the and out upon there future. all are the on to all we XXXX. in momentum reach had these to quarter, expect work looking is products, three forward across remain and XXXX pleased guidance early, another I

turn over Let to number Bill now of Bill? walk the updates a call through pipeline. to me the with

Bill Rote

Thanks, Steve.

two to with are be respectively. communities Both deliver us currently development approved conditions. we have needs unmet quarter, therapies first-in-class and are our serious for During these continued make third programs. for sparsentan to These patient upon fosmetpantotenate dependent to facing III the and PKAN FSGS first therapies the Phase potential progress the and

move diligently as both As possible. to them along such, we're working quickly as

of initiating quarter, dosing the increased during Regarding we clinical have FORT reach the to and our for enrollment. pleased fosmetpantotenate open and our program in milestone accelerate are PKAN, since efforts the to study additional very we sites

than progress for patients enable in a to commence. allow US, data and preparing the we is additional would to study, board participating and for sites enrollment initiating our safety Per to monitoring study protocol, have are during that more enrolling in in quarter. we readout sites XX the goal the initiation sites pediatric Our adult made Europe further

of to observed expect readout some a PKAN period short adult over been have that reach time. We relatively after patients

of to enrollment half As on Steve remain second we earlier, complete the pace during XXXX. mentioned

All these In the years, FORT more remain than City. experience from Recently, Child addition these parameters. of stable on study, for two described been United in have a an XX, clinical of annual receive patients fosmetpantotenate continue receiving in of Neurology data at to to fosmetpantotenate, patients' presentation update and was improvement XX of months treatment in new one fosmetpantotenate showed Kansas studies with Society's meeting therapy. all physician-initiated four the The patients and States. the to outside four

exhibited evidenced outcomes on persistent UPDRS persistent treatment served the studies. remains the daily encouraging functional the outcomes the our functioning, most or is with novel living on the The is PKAN-ADL the functional activities Also PKAN X-level PKAN-ADL primary X that reportedly was us, other X and of Gait, under from as thus FORT independence development benefit the is assessment the the as our improved basis for X-dimensional for converging of data such scale. scales, Part and the as UPDRS, the as agreement in reported study. the a encouraging consistent X Specifically, to with physician-initiated of of recently associated protocol being special especially of scale. patient's Part efficacy Part by utilized improvement in endpoint remained UPDRS also The presented and stable. a pivotal non-controlled Euroqol

begun the a lot order be Phase PKAN. the higher requested Subpart approval momentum standards H in III look move quarter steps I'm accelerated FDA together already our via behind to trial a preparation agency forward feedback necessary we we'll to not as proposed program, and treatment continue set I'll Subpart just the the submit to thereafter. to to constructive XXXX, for review, endpoint modeling protocol to for to of to forward in the again clinical some diligently As considered in on initiating you given H last see, can first-in-class parallel, is received in to look We've pathway. we CRO the furthering the sparsentan, pulling a obtain our Steve trial trial and engaging site for our analyses, investigational response, approval. advancement request gain and from our efficient qualify of now in This and next our mentioned, progress quantitative for our anticipation and night. seeing the surrogate start. activities worked pleased potential early In the with additional we're agency agency there preparation for the the an positive fosmetpantotenate the We FSGS. of is their candidate with this unique during

We II the new open-label also community had we Kidney from and Phase from nephrology in where data returned the great presented DUET just study. with extension ASN our engagement a Week

We were perceived Perhaps we safe we tolerated. kidney heard Week, to and greatest a XX physicians, the Regarding have patients participating treat still that at the the both sparsentan as that enthusiasm encouraged sparsentan Overall, very as with other to amongst well patients the we for during Kidney progressive see is date. see eGFR also today. to consistent generally from the reduction FSGS, new stable benefit in open-label diseases. physicians testament were to well period proteinuria the in presentation of who of extension and combined remained data, pleased community

of treatment of may orphan mechanism and readout considerable since all know, have have its to a you doing As dual the work of been action DUET, indications. nephrology of amount in understand utility where the sparsentan we unique positive additional

to us of sparsentan the nephropathies. While high-quality drove evaluation highly potential endothelin to utility an properties develop with a towards of multiple may have glomerular and significant been sparsentan analysis use nephropathy. to data had of those IgA anticipated. science to the has foundational quickly many efforts antagonism of data with more we anti-proteinuric to premise began Mechanistically, well access are indications, we our the work that continuously IgA which that have originally across very Key nephropathy, than translate of covering supportive IGAN, appeared of

FSGS, non-immunosuppressive who see this treatment few has agent advisers opinion options for physicians did patients. with like leaders, work also opportunity We and external validation and that key a great in for disease, a

So in we're advancing of nephropathy looking FSGS. alongside forward sparsentan IgA to

XXXX. we is IgA being clinic in nephropathy plan on related in Our details more and the development to established strategy providing

agreement better as will and was as a to patients. to and the meaningful in research sharing Foundation, molecules small small the took high effort screening steps of NGLYX.org, cradle quarter be many molecule, throughput National Finally, Advocacy of excited during of this to and milestone focus in quarter. the announced cooperative biology expand we developed pipeline, the and with earlier, development we identify look our Health, XXXX. NGLYX are our Overall, that understand advance the an Steve on potential mentioned The Patient to deficiency development ahead NCATS forward therapeutics we and about for Institute assays of

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

Neil McFarlane

continued the and of in growth Bill. deliver pleased commercial products therapies of million resulting our $XX.X to $XXX living net portfolio full turned another $XXX rare quarter, three of track in diseases. to year-over-year remain This year strong Operationally, meet product our million million life-changing keeps our us we in sales Thanks, on quarter. capabilities for in grow, and the with business the to we with All XXXX. third to people XX% of to guidance progression

one and our see to two some the starting Cholbam. are have of we've changes commercial teams, we now early one supporting team. focused recently, We Thiola of footprint of enhance some made the talked supporting the As to believe we benefits about

team remains Regarding Thiola, The high patients, those cystinuria who for are demand remains and strong. compliance to continues therapy. find on

the marketing We of of Pharmacal, XXXX. entered agreement Mission manufacturers as grow into that future the takes represents helping This our original in out we the can exclusive patients partners' Thiola extension an have well reaching U.S. which Thiola, our to and to our confidence also rights Canada extension at brand just continue more stone-free. and for as becoming them licensing a with confidence our chance the by foreseeable

made on formulation our a of endeavor to also We new headway Thiola. develop

get previously, next that stated acid spectrum Zellweger dedicated Cholbam, we're look for with fatal to I'll impact expanded hepatic replacement closer year. and more a diseases, of therapy synthesis disorders. seeing sharing significant that earlier amount two some we over and the I on as and bile we Cholbam. we NDA shift to benefit an forward filing been has now bile teams, mentioned from our potentially that As to acid

growth of that may bile patient clarify screen helped the already Cholbam. to screening see patients and of be candidate continue efficiently indicate sponsored results leading patients, number use acid symptom-based panel, testing that have our genetic can for testing a the great starting We atypical cholestatic This and comprehensive program therapy. a in has benefited genetic from strategy further a to

gathering and a remains for continue bilateral CTX in raising our prevalent enroll tool idiopathic we for information of awareness to with diagnosis. related to new cataracts. Regarding This Chenodal, study valuable earlier effort patients individuals

Finally, We finish cross business and diversify to remains development and to more you forward the we the if engage. of business, to one continue core updating or to opportunities when to line. strategy look these our find

turn the the over through you walk to to me Laura Let financials. call

Laura Clague

Thanks, Neil.

company's to third related for in share and We to an adjusted compensation $X.X general expenses resulting the over were sparsentan. the non-GAAP $X.X on basis, million loss changes quarter, million. third were the expenses million company's non-GAAP quarter income to stock-based expenses quarter amortization. During sales the increase net quarter of non-GAAP expense expense portfolio growth. for XX% of year adjustments $XX.X administrative period quarter basis noncash commercial for quarter from was is of from of higher in $XX.X $XX.X year-over-year of million reported to million change $XX.X commercial noncash liability $X.X included the adjusting for fosmetpantotenate last million. of million the GAAP due consisted R&D GAAP and The Significant depreciation On reported R&D our $XX.X a million. the third loss XXXX. and third noncash the and third tax quarter headcount million due operational XXXX. million. adjustments basis, related adjusted Significant and basis $XX.X product for the million. net quarter After to clinical for primarily derivative and on same an net On third expenses, the for were of Selling, grew GAAP included benefit increase stock-based $X.X a we the to year-over-year related and support The Relevant third SG&A and $X.X amortization. for XXXX. a expenses in income an operating million of compensation and adjustments price quarter adjustment the the nonclinical $XX noncash to a of is

fund are year-end XXXX, strong pipeline of positioned and and products to we the clinic. and a million progress As We securities. marketable cash as through $XXX.X September equivalents our they XX, well sheet cash in had balance approach with

our we As time, closing do expenses rise III I'll trials. past, our to in we his the Phase as stated over expect turn have to in we initiate R&D, Steve? in advanced Steve call for operating now back predominantly comments. an the over enrollment

Steve Aselage

Laura. you, Thank

third from tell made quarter. remarks, clear can strides you some the As team's we the in

disciplined remains strong. and business base Our

a years the ahead. continue positive We growth that the to on have outlook in provide can

our on importantly, and we the now, maximize FSGS remain As PKAN, potential pipeline and advance focused our of priorities nephropathy. to in IgA

move first-in-class simultaneously questions. we creating now while to will and patients key diversifying year the turn need, to close the operationally the and advance financially, are call and back up milestones lines therapies we into and going for our durable the long-term positioned, to to open Chris that As XXXX, in both well growth. reach I'm our over business

Chris Cline

questions? up now Thanks, open Steve. Tiana, can we the lines for

Operator

Partners. And Instructions] from question Schwartz [Operator comes Joseph first from Leerink our

Dae Gon

Dae I'll and is back in a for Gon I Joe. This one just had dialing the hop quick then question follow-up then queue. and in

the at XQ. rebounds just be products, So looking that sales to into for over of some your three XQ a affected that by quarter in sort first going tends that I somehow seasonality noticed commercial years the

going new you you commercial do trend forward? And can talked maybe Bill. question, two little either this So the think pattern? or for about your a And about bit then teams. talk that How Steve that might you second impact strategy

III guess, this on forward more color maybe can more little the was of initiating looking a exactly at narrow I update you trial window within your get requesting? XXXX on the What said FDA XXXX, guidance, you a in to put And little wanted design. a Just all? to sparsentan Phase

Steve Aselage

want to the start you QX. Neil, with

Neil McFarlane

Sure.

you QX been ICD-XX on wasn't that in I regards have terms start a change teams, The - tools the codes, terms to QX, forces deliver an dedication question them testing was? to EGL of in and sales forces in sales yes, what both was of XX% And as along So previously, you terms in focus doesn't allow necessarily split in the dedicated the they've where to QX. as we much that QX teams is of terms mean, versus in found the is there in provided you with between of there see sales enough were. programs. the allowed So have change co-pays teams. get separation and that well of and QX I'll XX/XX effort. The the to commercial the from gross-to-net insurances what the that with that with question of

Steve Aselage

is This Steve. Sure.

on the your to But FDA comfortable FSGS the bases, proteinuria comments. access interim tie confirmatory I through time you data and feedback improvements he think had of if put would anywhere months to last registries, just FDA we data that. the part on when together over second Bill in like have to to step make long-term make available anything proteinuria me really after a Let haven't to so in their from The to to sparse. ability comfortable night, patient the on efforts I'll then assessment, of would my add way the that will, aware got question, last in been can eGFR ask the endpoint. from you're well would lot six our renal been work We a approval forecast outcomes them that data we has that the

put have has think, attempting for together. different FDA and come those fair to modeling. two some together, amount We back of information tie of a I asked type

going we're are for effort, through sort ourselves, can should next the is Right going less take. on need long to now, assess And going have help or hours. to new, the or than XX do that we some how whether over that's very, of whether we request weeks we've some to the with handle that exactly We very months, that. as outside had we course it

there is before around bit we So hopefully, to to that add need our on to as anything to respond able to going much that we make arms that? quickly. what further, we're effectively do comment bear be a you little want to too with want Bill, sure have and us we

Bill Rote

Thanks. think was answer. that comprehensive No, I a

Operator

from next from And BMO Capital comes Kim question Markets. Do our

Do Kim

do you'll do Or studies and more that literature the think you've find what accommodate first At do regarding in able is you'll look, FSGS? have follow-up a with the what be the requesting. on databases to requests? that you the analysis Just FDA FDA collected to to think order

Steve Aselage

really a I going can think to we that. we're time of little before bit take answer

our literally we less release I than you XX request, with accurate our this with a said, call have sure had we bit hours As make on that good XX that hours and providing spent adjusting press of are the have to and information. comments

tell sure make we So if of you bit time give us we little would a is can what that accurate. so you

Do Kim

are FDA design to from you get the And us? what the disclose wait is the after until going confirmatory resubmitting to feedback you design to

Steve Aselage

we updated you think to clear I it's we're how attempt exactly it's keep will they're going going to to until for hard project things to communicate further. as And when me progress. progress,

We that to as we extent and updated be to try will possible as the transparent you keep can.

Do Kim

you question accumulation be to taking in on to we be treatment changes during able the scans that? observe RE-XXX. a to FORT the brain Should MRI for see iron And this will study? period And

Bill Rote

is This Bill.

that measure looking imaging, think at that the effects going we is mechanism And easily necessarily data our of is or positive the hallmarks point but MRI suggests whether be of of in that simply this that, ways we going patients. that's can PKAN won't parallel be aren't the imaging. absent that, disease. have in impact unclear one and these We there's we causative to it's eye-of-a-tiger We in study. scans something that diagnostic of on don't FORT the function patients, is at taking iron other sign that The occurring deposition with that to

Operator

our from And Liisa question comes next Bayko JMP from Instructions] [Operator Securities.

Liisa Bayko

analysis context Does want. confirm to be FDA a just does confirmatory So comfortable your will that eGFR request? sense you that still the make of of feel endpoint? the any Do new

Bill Rote

the endpoint pretty I don't from confirmatory standpoint. standard. see That's anything changing

Liisa Bayko

as eGFR And endpoint? confirmatory a

Bill Rote

Yes.

Liisa Bayko

of eGFR. terms how measuring think about in then you And

changes slow should role maybe sort do eGFR you think in crossover because how Just about stabilization. - know anticipate ad stability component it's can of Or [indiscernible]? me, explain a for to more I in to it's or to you longer-term the able a being Do you of move to of having distinguish it? we

Bill Rote

of measures. any eGFR duration studies variability you is and think the that repeated longer in there around And measurements. with get Well, I

of the I'm study Beyond at into specifics stage. to get going that, this the not around design

Liisa Bayko

you to of versus would placebo to - trying just understand it arm, that historical stable it's was for was reasonable I I would long have you compared time, an periods be how ongoing asking that's would control? that what about. show Or

Bill Rote

an No, be there control likely would active arm.

controlled and If surrogate. at gives a good with that irbesartan you look were DUET, we you

Liisa Bayko

- crossover for have be arm a the would won't of We of a time. control longer there period

Bill Rote

Correct.

Liisa Bayko

FDA parameters then, mean, qualify taking some might And trying at Just request be of this we a point. can maybe I to you how what the is kind onerous around get delay [indiscernible]?

Steve Aselage

I say it's to little a too think Yes, right now. Liisa, early bit

and really the I reasonable. agency. it us that seems think point. we've what comfortable that long get And to it's how giving an they've don't hours together, to going through you back sort that to asked had, in and XX work than out quality this want for less at I We take to pull the on control feel estimate

confident until on and quick work already. be pretty we that deeper, that we have here exactly. But area We've team. good a the respond and guys feel get build tearing as we've quickly. We as of on yes, it's can. as as the got with we it some is move able it, say me will spent We'll to it hard fast We'll into learned we'll I already can time we've apart. to what some hopefully Bill's And work team on knowledge who for already

Liisa Bayko

the IgA nephropathies? through And of then the can some just for evidence you go maybe

Bill Rote

expansion sparsentan with it's that of the inflammatory mechanism kidney's of mechanism in in across to is really injury, question applicable. its is injury so reducing where but where is much response in not the value shows pathology. sparsentan's The response the in the the glomerulonephropathy And prevention

at directly reduction that pretty outlined there the time - in clear extension proteinuria we've disease. paper in looked end-stage from Anchor databases is Group to and actually by to between relationship a a study linked databases renal So Tufts

is that in databases a for that going and at data. So looking we've some think been the strong there We've there. of we trial looking at rationale been

Steve Aselage

to add said. maybe what Bill thing could I one If

relationship proteinuria huge think in really patients hard the to seen FSGS aren't with us of because you've I outcomes tie work there numbers long-term between accessible databases.

that attractions the parameters two the of the long-term IgA large and together, benefit. easier databases. And accessible to it's of are those are One patients, there the proteinuria of numbers much fact tie there is

Operator

Lugo And Tim William Blair. from our next question comes from

Ashiq Mubarack

on Mubarack Tim. Ashiq for is This

were it integrating just Just and if on plan old I to bit. was a Thiola, might what how What about and the line your hearing you new between with us your set? little the And time the you you I patients. and the differences like remind on up new was wondering questions sparsentan to switch formulation what the formulation you be that could wondering ASN, - physicians switch and from formulation commercially. the a about particularly existing mentioned, related the data expectations generally specific for then are that what's

Neil McFarlane

Thiola about We going said before people our regards a we've a a you in talked to formulation. different new just little basically I'm to that got about are this formulation. couple to bit answer, going talk be to patient-friendly what We've Ashiq. and to going

what We of additional to game. Mission into five in for out is extended haven't it XXXX to Pharmacal the get our point. is I'll What to this that any reiterate agreement do point years this with gone also at we've at an regards us depth

I'll it hand to that development to confidence to us gives Bill these today over the be moving that forward. other some questions. as represent our formulation new will able answer well Thiola of patients as add to So

Bill Rote

data. some question of the remarks were at think was, the the what ASN I So sparsentan regarding

I the interest think of the study. there in duration was

remaining that flat, crossed that was of on the in and the only the which is on I patients sparsentan small well in see a proteinuria were open-label number in think It's open-label there on seen there were in reduction we double-blind that both the and a to over to as comparator in about fact that's durability is the the were the seen encouraging. that's remain XX effect of what period. in remarks period sparsentan then remaining months remarkable out Also, that expectation - irbesartan as the those that to patients, a continuous and the number GFR the cases, trial are certainly associated want in that that are repeated We normal what have you still but with going the data study the have are certainly fatigue and of trial. go. study. extension, even pretty best the participating and is with in solid. patients The visits rigors was the it open-label with long-term with in set that in of XX direction you a in patients numbers

that's to us. very So encouraging

Ashiq Mubarack

make that's Okay, a sense. of lot

a Regarding physician your the specific, relationship did hear endpoints? chronic proteinuria feedback to of questions about And was and with response what you regarding the them? lot

Bill Rote

think between I the Well that. disease challenge that the end-stage relationship we're established within is firmly renal hearing becoming across and not proteinuria and community nephrology

of in side attractive are physicians with trial specific space going do investigators, the we and a these treating very very that distinguished lot very powerful, work there's And they things when effect that talk renal immunosuppressant. because it's while the the on. a But of is sparsentan immunosuppressants an we lot that's hear also to One not profile. to come the there a is in days of activity

are frankly, quite them, renotoxic. of some And

So know that's deal that with, what one than has has adverse no to the on horizon with comment a this I events that benign, are physician amlodipine, day. that I more have how that potential managed had easily safety profile different with. is agent that every a to and one work they use

encouraged much are that can safety. be of a with operates space chronically they a this degree to tool used higher So in that have

Operator

Cline turn back call further to like remarks. Chris any now would to I the And for

Chris Cline

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

Operator

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

Travere Therapeutics (TVTX) 11 Nov 172017 Q3 Earnings call transcript