TVTX Travere Therapeutics

Chris Cline VP, IR
Eric Dube CEO
Dr. Noah Rosenberg CMO
Laura Clague CFO
Neil McFarlane COO
Dr. Bill Rote SVP, R&D
Tim Lugo William Blair
Maury Raycroft Jefferies
Joseph Schwartz SVB Leerink
Keith Tapper BMO Capital Markets
Michelle Gilson Canaccord Genuity
Lisa Bayko JMP Securities
Call transcript
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Good afternoon, ladies and gentlemen, and welcome to the Retrophin Incorporated Fourth Quarter and Full Year 2018 Financial Results and Corporate Update Conference Call. At this time, all participants are in listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to Chris Cline. begin. may you Sir,

Chris Cline

Eric President remarks. Tom. and Development everyone Results Full Today's by led Update you, Eric and Bill Chief Executive Retrophin's our Operating be will and welcome Fourth be and Call. joined by Dr. be will Noah Quarter Year Chief Medical Financial call for session. our Laura Good thank Chief and McFarlane, Officer. afternoon our Officer, to Corporate Rosenberg; Dube, Great, the XXXX Dr. and Clague, Neil Officer, our of Senior joining us Vice Rote, Chief will our for Research their prepared Q&A Officer; Financial

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

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

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

Eric Dube

and joining Thank us thank you, today. for all Chris, you

with bring I and and an a is an Aligned mission. future me can and month, since to shaping call our this be organization with objectives or their joining needs. opportunities mission pantothenate the an important I nephropathy development our on to impressive and FSGS rare like start has board As and last leading this innovative diseases to quarter, potential communities is unmet execute coming advance our ability our update severe will sparsentan be to living to further neurodegeneration in for start segmental IgA opportunities of glomerulosclerosis with we help programs and corporate Retrophin of patients focal to by or demonstrated to the key clinical patients. unwavering how first-in the saying rare I'm fosmetpantotenate pivotal class years performance. or and and disease my let to IgAN such quarter employees focus our them company these with treatment treatments number to Last important XXXX for for PKAN, to fourth for thank disorders. deliver honored how first Retrophin and kinase-associated on commend work want

X how we in without approved in know FORT We life Phase fosmetpantotenate, pleased patients December. treatment. enrollment is complete the difficult were to are for living an these For very Study who

a major dedication the So, line the the put which for of PKAN will track third potentially patients. milestone we're December see and in top for Study. quarter this enrollment be Completing encouraged a for to very on readout FORT their us year, in program to

NDA As designed which you assessment protocol filings. and we if generate a should is know with FDA, agreement positive the in alignment support study with and special data both MAA

put that XXXX. review Our a will we in for working in on high preparations, development are already together so quality teams submission package together be to position

progress making programs. on We good are also sparsentan our

quickly two to our effective need nephropathies, and therapies Given leadership programs in advancing we're establish working community. as possible rare as the rare pivotal the these treat for to nephrology the

sparsentan Phase FSGS. DUPLEX X efforts and of We advance our continue the to to enroll study

a second patients the top interim line positive We assessment the readout from in randomizing remains first the believe focus XXX proteinuria order our of enable on in FSGS Our to XXXX. half additional from allow us Europe. significant sparsentan. approval parallel, with readout the to result in our conditional to make for would we progress marketing continue and potentially In indication gain U.S. that

patient During Study key The nephropathy. quarter, dose represents the disease option. for in indicated first the of of milestone here initiating progression a we this for thousands a currently world. globally better understanding teams these improve programs, diseases potential developmental and patient goal a who with to rare IgA with end-stage class Phase people operationalizing fourth first-in help our around with pivotal X the of in PROTECT the IgAN short amazing achieved study sparsentan significant to we're facing a looking opportunity no hundreds renal job are Through treatment the two potentially additional to to did these of pivotal period an and treatment. lives the sites is of contribute forward to introduction

key us ongoing or phenylketonuria PKU. driver the is Another value with CNSA-XXX collaboration to advance the for of treatment Censa for

Censa at partners year. Our the quarter made and in this considerable fourth the start progress

their had hypothesis the Molecular published significantly study online what than was is of the was program X standard in and that This Phase Journal the Just for Metabolism. important an care. of greater plasma BHX milestone in the CNSA-XXX Genetics recently from Censa support findings dosing seen team amount their with of the and derived

of to X proof-of-concept development. standard with As We to top continue thereafter. full versus remain follow on further understand a to data and package data with CNSA-XXX currently second of know, this deliver their that will a of in you Censa study partners year track many from they to running is care we pleased to evaluate be the our current Phase quarter line Retrophin

patients we unmet in to anticipate making FORT regulatory pipeline. quarter a chance from of pivotal successful portfolio. growth well The consecutive and had marked We as have to another data complete to fully acquire address program evaluate quarter period Censa continue product significant need to over has our requirements commercial other development. for the a option decision on the fourth the We after our also as pathway the believe this be period the potential for and possibly program a

fourth patients' play with to line in during acid quarter. continue nice role lives. products rebound important bile in performed the and Thiola approved the in expectations saw a we products an Our

teams growth commercial to and to an XXXX. the new Noah based improve on forward update FDA growth helped date. to Thiola assigned the second like have call the PDUFA the would organic look continuing approved, experience have new to the programs. the stage a review patient NDA Also in that in development for rate that we we and during of half formulation deliver now similar was and June to cystinuria year. for we treatment late may starts quarter, at XXth ability working If a sure by a I on for Our are solid we turn our the have launch the patients of make for over to accepted the

Dr. Noah Rosenberg

good everyone. Eric, and afternoon Thanks, to

is for approval SPA FDA, readout. As a if and commend met underway enabling to data XXXX Europe, we randomization XXX% year in upon trial the enrollment our and December, very to in our Most for in the enrollment the investigators the study a exciting programs PKAN. placebo-controlled continue want I based milestones is to the programs results. observation third to of I'll our announced will stage available safety of three Eric key completing for our reach FORT of support with and and in in expecting had clinical review the FORT U.S. we us under upon In the by year. we conducted X recently, and to independent clinical XX-week pivotal we the This pivotal fourth to in committee. review data our from mentioned, committee line target a the This quarter agreement positive the fosmetpantotenate of team monitoring developments. have top Study. this yearend. of the we start being for and and of recommended, we Phase trial be expected period, is schedule first the quarter Based our enrollment planned. generate both completion set fourth are our database all are be study lock that pre-specified as with for

sparsentan continuing to both progressing, fosmetpantotenate NDA and our our of partner of submit pleased enrolling rare nephrology in quickly If goal sharing leaders in DUPLEX development as PKAN IgA our closely as continues becoming community and first work progress often are continues the kidney The FSGS two spirit, end highest Phase In am towards stage potential to our we debilitating position our team successful, patients we FSGS class progress applications studies a make rare towards to to year. teams to first-in make the that forward after we this to all we disorders, interim possible. later therapy in to MAA and X look receive of our how to with are available working results becoming Sparsentan we indication and lead a to enable continues I which to community. renal XXX in you ensure Our to the of steady internal disease. to want also assessment. advance With Study out for the data subjects package. the toward have be data progressed pivotal with biometrics those with quality make necessary nephropathy, XXX the CRO

and these landmark The reception I'm study engaging we Our overall that this driving patients. community enrollment continue to study we its team meet open positive about now has XXX great job have has to done with pleased believe is and than investigators the after that been a more sites goals. screening excited

proteinuria in the readout next subsequently and second our months positive, of we H XX line our If expect Europe. filing the work NDA top half next over an Subpart will to during of expect would partial We U.S. endpoint of marketing the in conditional the on pursue FSGS the authorization year. remission enable

to nephropathy. for make our in sparsentan continued also progress IgA next indication We

Europe. with proteinuria-based utilizes consideration clinical endpoint of targeted the Phase As to Europe. Study December, the PROTECT authorization the for a sparsentan patient conditioning our study the our active-controlled X registration at dosing H the Eric of IgA PROTECT in Similar Study. U.S. after and support marketing the PROTECT ongoing to milestone nephropathy to in FSGS, Subpart The in in of X end for support submission pivotal of reached treatment of our trial U.S. mentioned, is weeks designed treatment sparsentan and Study Phase we first the XX in a

have are in three and we progressing rollout, our plan. study to completed the in global line early encouraged initiations with meetings investigator have still site While

additional patients year, be our proteinuria of half first building on goal For getting of assessment focused towards XXXX. we the expect study and line data of Based on study. ultimate XX top enrolling further world the the balance the awareness of in this our will of in sites move open around we from current the generate patients to XXX our week projections, XXX to

focused on important the goals to remain will to new serve. options closer team treatment us meeting our potentially our delivering we near-term ahead, Looking development move to patients

generate starting excited the in We filings MAA and our coming to in fosmetpantotenate pipeline are with the especially and near multiple opportunity by future. the NDA years, advance

let Laura? now So, to turn over call me for the financial updates. Laura

Laura Clague

of for in our the loss fosmetpantotenate our for Birthday the were from for amortization. the expenses million Relevant as and full represents XXXX. and sparsentan, and administrative and stock-based the On fourth of This Thank a for levels. $XX.X $XX the million and expenses for to non-cash the the the an XXXX. sales increase year expenses expenses quarter support X% $X.X full XXXX. well you. $X.X clinical the for were included for you, and non-GAAP and full for a year XXXX. year XXXX. year non-cash $XXX.X a and the fourth million the depreciation consisted loss the $XX.X million of funding over year $XX million for of is quarter commercial full for product for quarter and higher non-GAAP Noah, Happy an net and quarter for We for GAAP On year XXXX. $X.X portfolio compensation million largely $XXX.X year XXXX. million product is CNSA-XXX SG&A we Significant spending attributable expenses million fourth increased reported were as over XXXX. The This largely full to million to to adjusted quarter million and the for million $XXX.X Net After unchanged and million $XXX.X basis, development basis, fourth R&D for fourth for general million fourth collaboration. from quarter compensation XXXX net $XX.X net expenses reported R&D a Selling, for amortization. were SG&A the the adjusted XXXX basis, of quarter million adjustments the fourth On $XX.X and efforts full million full non-cash million quarter a loss for for the increase GAAP and quarter $XXX.X full a year fourth $X.X our expenses, $XX.X stock-based the adjusting

December of $XXX.X increase very a XX, our As securities, marketable our levels. cash to we position had levels over in and million for and expense company Looking XXXX, we XXXX ahead, XXXX strong equivalents cash expect in financial operating

course investing advance product our activities in clinical increase such, our and R&D we XXXX. our ongoing are and we of materially We pivotal development programs, expense expect as over the as year-over-year to

stable invest throughout be we our trend We be quarters manufacturing of with increases that there will uneven While activities, in more year throughout expect capability. some launch year. anticipate runs the smaller the general timing product as may we the CRO the SG&A and or incur to upward

Lastly, of will year, you previous that our XXXX to in expect as our convertible of future third higher when in last result compared expense interest notes issued a should quarter year. the the be

Let to now turn me closing remarks. his the Eric? call Eric for back

Eric Dube

group me and closing, strong dedicated leaders build the the a meeting teams of with we capabilities two and at also to months Thank It upon Retrophin. our like clear you, where I get my to perspective gain our to talented an of for living potential time for I have have has that some employees, with patients. to initial built Company. value Laura. become future much continue growth. commercial rare the impressions pipeline organization I helping business inherited on foundation with and create spent to my share and of stakeholders initial tremendously from time with patients significant I areas community to additional disease In the can the spent first have sense strength of the to would

the pipeline treatments the to doing these possible. to will we know am of as to to going come will value I my therapies programs. quickly we from in creation near-term as patients priority innovative ahead, as background be our Looking everything continue our that one our such get delivering and are ensure number leverage can we to execution

the product days our seen evaluation to the of I the growth Study and our of with days With encouraged the on tenure, timelines, over and enrollment confidence leadership future. first of Simultaneously, the exciting is XX to execute company's for going and line. first I'm our ability all my of important the an this saw our deliver pivotal most to key part them As as year patients. ultimately front. and with be programs, this it you me on for reaching of XXX year, my What accelerate get further candidates first-in-class our the to working to our am Company's our of plans sparsentan milestones our organization will their the the team first for I've program by advancement gives finish be to FORT in I across and capabilities assessing our in the readout recently people. decision the CNSA-XXX completion expertise therapies our with planned in enrollment to readiness our

when efforts, strategic bringing market. uniquely are funding continue R&D to portfolio an positioned with to pursue We a new our that it execute advantage commercial a importantly and gives important more of source but therapies to will provide

new launches. in instrumental years our our expect strong teams We be will launching medical and three in in of at the therapies to least the capabilities and coming commercial successful indications four be

to to which diversify groundwork further a bringing with We you of well success treatment on rare to disease significant treatment require for needs potential our look as long-term we more as our deliver I IgAN for unmet as PKAN, additional first of therapies and continued continuing sustainability living the and disciplined and to execute to with sharing forward of development focus be execution people of on will improved all Retrophin's business with goals to a approved business. also FSGS approach PKU. laying our address the will a and patients

up call questions. can we Okay, open the for Chris, I think

Chris Cline

Tom, Q&A Great, can thanks, go Eric. for lines ahead open the please? and up we


first [Operator the of Blair. Tim Instructions] our comes William question from line And from Lugo

Your line open. now is

Tim Lugo

program, you program CNSA-XXX on with forward be with bring the in meeting I some And Eric co-developments guess, upcoming path want of dependent acquisition X in your the data you Phase also of not it to I end like go for but just up a regulatory noted readout, but So, mean would deciding house? before Does are sounds maybe for the -- some sort that I the may any an have think agency about commentary, may to guess there path forward. comment you mechanisms program, in where forward? that the

Eric Dube

and look forward Censa really what steering at be eye would I we committee are are comments the we for X, and working a portion we ensure the the Company decision how I like can of quickly at acquire Phase to make on given to the the that path a on sure competitive Certainly, really path my sure make totality the we informed want look able the bring are and equity landscape. closely the through based but acquisition. that the sizable joint are making with not would data to the that keeping FDA just product this evolving to for most we of using about keen looking the continued that and decision that

best way been decision I in I if value the in as but possible Company's using So, patient sure the that to that equity significantly deliver holders. I'm sure value as past, make to for not am want well that how certainly made changes resources would've the

Tim Lugo

deficiency be in know of outside you I about guess, Investigator that's I indications, the thoughts also that but be PKU or you gastroparesis? led at at will any study its there maybe over around maybe whether BHX thinking all your acquire is or ongoing will XXX impact or not? you with, already And other looking and

Eric Dube

available Censa complete program. be going at we at we so, data PKU made any from Yes, package time the looking are the that is for data to the that get

primary until of if We want will are make very certainly those to positive would we that decision completed number but see completed we or where we data the data. wouldn't As BHX like able patients, be there to bring PKU are know, are programs wait before be excited studies a you I a to those on data deficiency.

Tim Lugo

one maybe last question. And

you are mentioned You assessing timelines.

For still DUPLEX, that half would patient birthday and any mention I you is XXXth answer be for won't of guess, on question. it you track you're did second the And be this say enrolled year? can't in I Noah, you asking to I your because the

Eric Dube

on as Noah, you continued of acceleration Tim. as to his speak have birthday we I'll a be add move From everything see see on forward we and trials track screening anything gift. to So, want with you behalf, I else that and continue seen, as often enrollment.

Dr. Noah Rosenberg

well, I think it you've captured Eric.


question from Raycroft of Jefferies. Maury Our comes next the line from

line open. Your now is

Maury Raycroft

on potentially any on that AEs is able anticipate penetration if specifically X readout? for be Censa-XXX in we if And efficacy. neuro if a readout, and Could question neuro on Phase you're measure, benefit with first standard differences for based looking My you're I'm to the now on we looking -- the wondering a benefit from blood-brain barrier benefit. will

Eric Dube

I'm to Bill. pass to going that over

Dr. Bill Rote

the is study your question to I concept protocol. Phase short assessment does in think neurocognitive X have the any not that answer

phenylalanine. looking by and plasma primary efficacy really safety it's efficacy, driven So, at

adverse of if to events, understand a differential between events blood-brain CNSA-XXX your penetration. adverse with So vis-à-vis care see the question would around and piece the standard second on it of able in I be differential we the

becoming of pick be eye in my don't something enough the a based no, traditionally patients would later number in the that certainly out would is think but in a patients I I would look study, a likely small you probably ratio on studies. just that study. XX at a to that, and to study in we expect probably signal-to-noise large that subtle, are answer larger the have going

Maury Raycroft

readout, are the the we not blood-brain ability to going a sense get good this on So, benefit probably based with to penetrate drug's barrier. of

Dr. Bill Rote

we It's really to of looking going at. fee be really reductions going evaluation to what be are an

Maury Raycroft

you stat As of well? far can differences the endpoints sig are look XX% reduction on primary on as at we endpoint, any that and rates? looking some and -- should Should anticipate for that you as XX% the assessment with potentially XX% the we secondary where stats

Dr. Bill Rote

XX%. phenylalanine, I be I I start breaks changes think agents whether see on should those a that should statistical less it won't I than when to you get be than consistent groups, seeing than the see down come to in of endpoint. think statistical but the the between XX% overall fee down buckets It's the reduction, going XX% into expect percent significant. these game. in differences less to will trend Certainly, to overall do we less think difficult across numbers you reduction different be

Maury Raycroft

FSGS, general any And to on can you regions up are seeing types where mentioned your in sites the and you're or that strategy the shape any on? enrolling a based patients provide are on could and comment how U.S. you got whether x-U.S. of that willing that the you have trends just enrollment you XXX the so I and on located running. then perspectives question you're sites guess

Eric Dube

do that? take Noah, want to you

Dr. Noah Rosenberg

question over to with The XXX -- said just have sites of also has as team We and great for screening Eric's engaging -- generating so now investigators Yes, us. done a we job running. up into back activity.

but it's aligning while will to. monitor attention question pay and so look we suggests, or And too our definitely our patients in monitoring protocol. probably It's analysis something differences, early pre-specified the real-time to for a demographics still we with formal your haven't regional to patient done coming on


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

is Your line open. now

Joseph Schwartz

questions. of couple a just So

I the heard correct. Thiola line to Let's on in about were be growth, if the Eric, commercial. roughly talking projection start you with you with the sales year-over-year XXXX

said commence, X.X and half you So that second PDUFA 'XX. approved should mean, you launch in just if have I date the at of a the midyear Thiola wondering,

terms benefit Given a Can just through a us that remember of if roughly then growth delivers, conservative? Thiola correctly baseline, I getting walk higher creatinine the greater X.X gram. per the of inclusion. there? Study, the DUPLEX to than behind and in equal ratio slightly And urine That’s what four are year-over-year I being guess through or logic than you attributes, DUET was just X% grams you over sort of inherence sparsentan protein or looking X.X of of

So analysis whether complete modified in just wondering, off you standpoint, what remind remission your can represent commercial or does does that what roughly be you a cut populations? into extent guess in to XX,XXX proteinuria conducted DUET, us And patient remission, subgroup outcome I that gram baseline UPCR it'd from reduction? proportion impact X.X partial

Eric Dube

Actually, with baseline? at why question? UPC do to second don’t we talk about you want Noah, start the the

Dr. Noah Rosenberg

probably I that you analyzed seen are done don’t referring X believe UPCR. that overall of specifically total So, improvement think that was to meaningful. difference have patient's cut I that And regardless X.X clinically think we've to, we I but baseline directional that a not is for what we've

So we significant would difference. expect any

Eric Dube

going rate with will benefit, And to last I'm to the commercialized Neil. about and growth regard FORT projections, that the plus With growth commercial expected to across we overall specifically three over year. for with expecting growth turn we potential that our saw that Thiola regard products are that X% be the the more consistent

Neil McFarlane

in consistent date whole the to continue we We've actually product. be that to new previous through with those, So XXXX of friendly said, when it's about three four the things. will PDUFA in two attributes you of we more One, the rate. until consistently about line that growth Eric and I our of said which talk accumulation, as attributes the business described patient want talked last our four

see that our there our and fact I still XXXX. is with if QX see And we there Thiola, on maybe guidance else matter adds patient line So, might've in stop I'll anything missed. that of early absolutely is new for

Joseph Schwartz

would on just fairly given that now be assume compliance adds patient benefit the wondering new would given the the side probably better growth Yes, pill be and and burden it might I no, cracking. of guess the that’s pill and retention higher low consistent, higher. the I is

see guess the one Can look can an maybe enough BD I we way guess or thinking two is going two PDUFA elaborate we ask how I just which scenarios approaching you and where strategies kind kind how of alternatives. or unfold. in-house answer the more I So, you is forward? becomes you you June is one your forgo it? Censa-XXX and the about point, If what on is your got see can to question sufficient forward. though Eric, and have you just are date And option product scenarios of for going scenario two the BD develop

what I that open Just is considering? terms thinking beyond Company focus you new you the spot Or given to sparsentan? the at wheelhouse? a neurodegeneration is will in that nuero of organ evaluation, sweets continue be the will or guess you PKAN a that cutting system are that nephropathy with have

Eric Dube

overall want that unmet to we to the to With in look regard rare need. approach, have a disease continue development high areas at

we potential accelerate to strong rare decision manage Noah, Censa, disciplined regardless on we to be of what With on the one Censa. decisions. about data of us we Censa. disease readout potentially sure those areas expertise will able that While need we're there we unmet areas, we business you have first else very and that development in in business like how broadly active class And very believe of opportunistic every is or anything and be for a that are be in we want therapeutic to of in case looking to potentially make want to add in where would being to the better certain ultimate be our decisions continuing including development?

Dr. Noah Rosenberg

go just you I'll maybe neurodegeneration. Yes, terms and of talked your about specific to point in

be in continued As PKAN we have execute footprint able our are have that be we we that our to today, commercially will area. going we leveraging in program,

be disciplined look development can that late the to able both our to We that and today our that to current value we we diversify externally footprint continuing portfolio, commercially anywhere to but capabilities areas are see want actually from our can stage leverage. add we


Do [Operator from Instructions] the Capital from BMO of question Markets. next line comes Our Kim

now is line Your open.

Keith Tapper

just for Tapper is Do. a This quick I had Keith question. on

either might enrollment to from signs the or is to announced You in that seeing anything you're compassionate measurable of completion like of you that for expecting context enrolled analysis? from patient terms Protocol just study. would look example of Should wanted or or approved protocol, FORT finally And so PKAN? a Special willing form you agreement. have thirdly, Assessment anything of in arrangement for be age I know was progression evidence FORT anecdotal be from in the Is to I what physicians what to we the if with there impact the And our subgroup on patients in up. And similar in assume like of that? EMA? under the disease safe terms far used it then follow benefit comment or patients more one the of it the or mentioned terms then

Eric Dube

I'm that turn going to to over your answer questions. Noah to

Dr. Noah Rosenberg

so your first of just to question. part address, first Yes, your

in and all tracking and old these looks is protocol, like is all the can have meeting that the up that’s we on the There in if seeing, our are we the to between are the say we got to-date founding about wind well much with not is hypothesis with say we study what based really a had they set. month data. population study. with in obviously, into large that seeing blinded want but what we show terms unblinded I with pediatric the groups It are seen is correct. were we an we overall of clear enough of overall between We information we've representative question rolled our the effect, we In the patients demographics our to data cohorts of line can dedication to just and a lot had our thirds maybe being add, the DMC your from FORT to the in two expectations.

that planned a encouraging So, gave very us was they as proceed so sign.

conduct you subgroup analysis study, result. this analyses, a In not terms the of will we so huge probably remember if subgroup is pediatric

significance. looking you are necessarily when I think the have studies in statistical not size you for

benefit any we in results really some don't program updates And those and trends for looking are have populations. top You line there. anticipate we until

handle of want spot terms that in Bill one. you Just to

Dr. Bill Rote

We have the in approval, on agreement if positive. single that we study alignment study design and EU, to in have the but the don’t support have respond a U.S. to similar

So, same effectively, up. set the

Keith Tapper

on patient in of strategy new commercialization transitioning to want you patients And intensifying any recapturing likely there ramping generic you on are us Or the of entrance or friendly that formulation? patients you pill because are can that Thiola. of to you be formulation that take Is Is competition? competition to comment at the terms you looking at remind terms seeing anything then, in terms perhaps would could follow update there just present? burden? in more And up more the of up that lost the or

Eric Dube

but seen to generic on to regard with detail don't that generic nothing Keith, commercialization to I a predict would access to information over that’s there, more is have we is on So to there anything. What continued we that not We it bit maybe that like suggest on and question. out with going a monitor all turn to to landscape. maybe would we Neil horizon. obviously the everything do that give near And a see term

Neil McFarlane

give little to. that probably you your one in could I to bit question flavor specific on more a regards

new creating friendly our to We new to for friendly formulation, will patient conversion not patients be formulation. the barriers any of

discontinued their persistency. done what better our I the providers compliance have that partnership be able their are and and have think planning the for out hub to others them ensure -- heard. to the whatever physicians ability and contact listen what physicians is that may know the they And and those for actually are patients go and product to with understand service to have and we to to patients new to to who a that we reason, and be to needs do we've a we've we've that got are formulation continued is looking attributes what able let

So that's what do. we'll

new this to to we to to plan to barriers entry don't have the for addition patients that product, formulation. able raise convert being any the In to


Our Canaccord Gilson Genuity. the Michelle from of next line question comes from

Your now line is open.

Michelle Gilson

if hoping also And bit fosmetpantotenate then little are TID and you I patient XXX are expecting you about the And reduce of to how their seeing a to was selected you LFT little and talk managing, any on you need your dose? are a protocol? bit in the you how just dosing FORT could elevations dose?

Dr. Noah Rosenberg

the terms even of in effect doses. are tolerated want lower at use do dose. We the to I'll actually maximally maximum the who dose, selection patients compassionate of saw in benefit. the pick that Yes, we to get just correlate who back

in seen lab we -- question, In and have LFTs, are closely are are regarding following signal any follow looking LFTs. as the specifically terms to safety specifically potential not and with we aware any increases for concern LFT any or of abnormalities we at very you a we

Michelle Gilson

during have patients I of the compassionate the think study? in sick might use gotten one

Dr. Noah Rosenberg

there transient Yes, was to transaminases compassionate There -- folds -- the one the patient when elevation yes, one I'm to well, cut in was fine two use saw patient the three on the correct. and a referring was improved in that dose to was doing drug. that is

your that’s to -- point picking Michelle in as up. have we So, that and diligent usual, point that again, very

an We interest had everything and again, we specifically, as was are event recent LFTs. clear. meeting are following of DMC and adverse again anything seeing not or And a concerning AEOI we

usual. as Michelle question, great thanks, So

Michelle Gilson

PKAN Maybe one enrolling? if and more any hearing in conversation your I anecdotally are they study now uptick you're based this KOLs investigators if guess study, guess on on and seeing from I your diagnostics with just

Eric Dube


Neil McFarlane

we had on additional we as U.S. of patients of had identifying identified. patients, they across And call, study Europe have that have both are may have around awareness across that the on answer done came work than newly that the seen more in a we've some can patients The activity last of we assets we the you I originally maybe and our some once aware. the are absolutely with medical mentioned and flurry have our sites the Yes, diagnosed right. of clinical ground to affairs and

answer So, yes, to is that question. it


Christopher line from from Instinet. question next Marai comes of Nomura the Our

is line Your now open.

Unidentified Analyst

This is Alan on for Christopher.

Just one question on the PKAN.

the For And I and be the age readout disease onset And will the this more relevance readout between its and you onset? year, can segmenting have maybe progression or on a of expectations? got of age by correlation the format a elaborate little you follow-up. presentation to of

Dr. Noah Rosenberg

terms question in first it's here. of your so Yes, -- Noah

communicate in presented meeting. get the readout, an information the study actual timing that, on appropriate expect at it the to while that any retaining outside will ability we a relevant to depend minimum, key is of that primary safety endpoint In at data, we medical provide really of and terms aspect. know To

was so age So, pediatric around And the of be. One understood effect the would two there other don't question what top an predictions, but understanding line in terms a think data. that think the and quickly, is are that’s onset of populations. move may there of then terms little more I in that pediatric I actual potentially drug well is

know don't pediatric, that's think those sampling And we so nice a have are, third well I So, if of fairly clear. with again represented. population two-thirds a I patients

So, can that say. is much as I

Unidentified Analyst

And how for use physician the the interest patient or compassionate on is asset?

Dr. Noah Rosenberg

It's very high.

a and level get the there's moving we do stage up running, the from FORT long-term interest. use program where internally working compassionate into patients We hard to now are to we in are a and definitely best over the our but of high just open-label, obviously very are


question Securities. next line from comes Bayko Our Instructions] JMP the from [Operator Lisa of

is line open. now Your

Lisa Bayko

of but been quick majority one my just new one. have vast The answered, questions

a data For a tell the go-no-go? it’s with us? us Censa you Will that? expect program, how could level set should communicated? full what we expect around we of be Or disclosure share to Or that just data that

Eric Dube

cover to Bill ask I'll that.

Dr. Bill Rote

where know, presenting partnered the Censa you -- this and be our will we is we program with partners As where will working a Pharmaceuticals data to determine will be presented.

then decision had we an you let We point line had to we have data that. we And -- when underlying top have have do haven’t or So, don't haven’t completion. know. get down, yet and nailed around that we that need discussion to we where we the make evaluate its that, will


[Operator to to Cline. Instructions] at the There are conference this turn no I would Chris like over time. further questions Mr.

Chris Cline

in progress. you near Great, for updating And our you call. future thank us Tom. We joining today everybody forward on look for the thank you our to


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

disconnect. now may You