RTRX - Earnings call transcript Q2 2019 | Travere Therapeutics, Inc.

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Operator

Good afternoon, ladies and gentlemen, and welcome to the Retrophin Inc.

Second Quarter 2019 Financial Results and Corporate Update Call. At this time, all participants are in a 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.

to conference like host, to now Mr. would turn over the your I Cline. Chris

sir. You may begin,

Chris Cline

Thanks, everyone, Call. Good Financial Rusty. afternoon, Results Quarter Retrophin's and XXXX Second Corporate Update and welcome to

Chief will our Today's be Clague. Financial Development Eric be Research Noah be Chief for call joined Officer, Rosenberg; Senior Dr. us of for our Dr. prepared and by Officer, led our Laura will remarks will the Vice Medical joining Eric President Executive also Q&A by and Dube. Rote, session. Officer, Bill Dr. Chief our the

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

They expressed results, achievements performance the implied assumptions and of are risks, unknown guarantees those and and statement. actual Forward-looking statements from that or not cause materially involve known may by differ performance. to uncertainties

disclaimer XX-K the press in the Risk Please see well Forms today, on the company's SEC. was with the and release XX-Q issued Factors our as section as earlier filed statement forward-looking that

specifically made, addition, X, of the or circumstances. are only forward-looking XXXX, statements as statements In such to statements and future represent views date update obligation information, events any our disclaims Retrophin such reflect any August to

With that, let me now turn the Eric? call over to Eric.

Eric Dube

exciting Retrophin. for It's time Chris. you, an afternoon, good Thank everyone. And

new and as to treatments to and innovative members options Core first-in-class focus and delivering execution team leadership patients Our treatment fosmetpantotenate sparsentan, developing on living an continue like on demonstrate is rare treatment. focus for to mission, our disease. potential with unwavering

unmet need an Patient meeting with a help to about challenges fosmetpantotenate and the to past from hearing team these therapy the honor significant several first and the potential fill has for the of Community. approved members to living recently patients the bring become the in to number and treatment. believe We've approved and effort of PKAN the working attending years give and treatment hope. of been need had for an a Community We families desperate A parents PKAN the directly I to

The report am remains patients for and PKAN everything Phase with on available to us I later pleased X/X ongoing share making to studies. that to this key track is results fosmetpantotenate top-line quarter. for obtain our

positioning While the to to in we our we remain MAA blinded move data, and planning ourselves results. positive quickly and success are of for NDA to in the submissions file XXXX, event

key access, the diseases. glomerular sound be the our launch in support activity. advanced In place leader position a medical progress to such as quarter. X Phase addition, also we rare in and And studies we're during with market marketing affairs readiness Sparsentan and Europe. making continue as launches roles now in two to U.S. build in have We our to ready

growing for like sparsentan's FSGS potential The nephropathy become conditions support new IgA our the underpinning to and position for treatment strength. standard is

Our lead FSGS open sites DUPLEX new study to continues globally. and

in DUPLEX enrollments through difficult trends DUET the times. summer we critical have we months recent not have at be months. forecasted As In increased in beginning project rate to during of study, FSGS Phase the learned our the X these enrollment can

Those on screening moved DUET, experience impact accelerate enrollment. positive already and initiatives our with a were initiatives have quickly this when prepared to to giving planned Importantly, starting arose. and we are

adjust XXXX. of remain half today, second to results of half for stand we our it appropriate the top-line team's from However, first believe based upon to of the guidance I end the point proteinuria in execute. would our ability confident be XXXX where to we

to is patients in We’ve any delay recognized treatment that getting this meaningful.

sense maintaining recruitment we far. study of with thus have to continuing the high are a we seen of act great quality while urgency So

enrollment for goal our year, our initiatives if possible. these accelerate Over the the balance of be timelines will ensure to

Our considerable study participation have are job drive leveraging team study. IgA by And DUPLEX we strong half doing execution the progressing sites. footprint The the proteinuria to nephropathy to remained forged of the a is we in is Phase top-line results, on-track X and of PROTECT seeing across in XXXX. good lead and nicely work and first

is therapies. Further disease rare development towards cardiovascular second abnormalities, research by Alagille for research Alagille and Development severe and characterized our Syndrome the approved or liver we efforts are entered CRADA, efforts a and potential Agreement of therapeutics Cooperative identification is the and a on debilitating during into quarter, Research no early and currently there Syndrome. which dedicating

the Center establishes NIHS our for collaboration, National to patient and we're research. Alliance, collaborating is NCATS This innovative with leading our this second the patient advocacy with it Sciences, Advancing of Syndrome and strategy foundation. communities scientists Translational working with Alagille and early advance In the CRADA Alagille leading

It to in talent, a these By look take approach probability new collaborations pathways. to you pooling the resources believe late programs. future progress. disciplined with updating also of stage our teams for best identifying as focused on remain allows We increasing to allows the our us operational treatment we this in success to forward clearly our

I'll exercise of option in program briefly discontinue We’ve our completed for and touch in to Phase program our the recently PKU. joint on acquire our the PKU. Censa, accordingly review CNSA-XXX decision, to to development decline to X

meet months. for treatment Pharmaceuticals. did Censa Censa not acquiring potential the be Retrophin’s their and over option a requirements XX last may drug ultimately very CNSA-XXX, a are for to for for we become team collaboration development thankful PKU, specific has it there While talented

Consistent with that and need. with our unmet areas our on mission delivering life of high of therapies, to programs we look focus changing focus pipeline will diversify patient additional further

teams very execution. the with continued I'm commercial base business, our pleased to Turning

products. quarter strong was net delivered driven treatment quarter growth. We by proof patients sales have our of all This of initiating product a year-over-year second another and primarily three new with

choice cystinuria there well are a THIOLA But patients patients. original steady. THIOLA’s great a been will we treatment for step we of challenges remain utility started forward cystinuria approval who know new the with summer in some this the face the that performance established. with in Notably, formulation. community, towards THIOLAs off EC, provide has

hour there from pill most Two oftentimes the remain challenges when a burden, to of medication one be can be times a that or original prescribed two can and potassium is common citrate, day. indicated particularly we to a that three concomitant high it were the many difficult also compliant after before taking taken are food that patients, meals, when hours patients formulation be heard with

should manage necessary cystinuria. is their be potential number the with provide THIOLA to indicated with food, EC Importantly, the freedom to welcome provides XXX THIOLA administration. a or And, patients EC for option reduce without of element milligram which taken to of tablets the

I'm first the as prescriptions EC underway, shipped Our and to end pleased were planned, launch the that report July. is at of THIOLA patients to

of brand new from making and of option team has available is the approval. within we prescribers encouraged are reception, weeks commercial excellent patients patients. done to getting Our early job, new by And this an the

simple. price EC which cystinuria, is a and do as clear formulation. choice are And providing access patients to original formulation both the is their ensuring same to THIOLA. approach available Our plans for of the and THIOLA managing forms therapy We're is remove at with their they believe original we of best the not have physicians we

current revisit who it is offers. discontinued there find that treatment THIOLA an formulation. and flexibility us likely convenience the brand additional that new that us THIOLA if prefer to demand over for would there's And to tells research market and our patients be from and have who they significant from the Importantly, patients may tells the years new opportunity benefit the also see may EC

the we're confident for in a franchise. outlook As the result,

uptake additional share patients. to the data about given engaging the gathering physicians we limited with is we giving the for more there quarters, to once While in recent very THIOLA coming and look have of EC, forward feedback more detail time launch,

team growth the the last and pleased into to confidence ultimately quarter. maximizing was portfolio, for This EC over launch has going driven were great us Regarding the strong organic new we with approved. and of asset the by starts which ongoing for our during recent several across shown if Overall, an ability our second the the potential years. period drive final sparsentan consistent THIOLA patient products performance, the commercial critical fosmetpantotenate gives

me Noah? the Let turn call on the pipeline. updates for not Noah, some for over

Noah Rosenberg

with Thank program. I'll fosmetpantotenate you, start Eric and good the afternoon.

kinase-associated therapy, evaluated Our or novel being substrate of PKAN. neurodegeneration, for pantothenate treatment the replacement

X the FDA. with We top-line FORT which our Phase Study, pivotal SPA under is to conducted being a or are trials readout nearing the agreement Assessment Protocol Special

treatment of The point Living enrolled Daily after its PKAN to measure XX will the FORT scale, or will study change endpoint show receiving in PKAN-ADL the study either three of with baseline evaluate patients of this changes difference patients power and treatment. between And placebo. a weeks in from primary XX activities The groups. PKAN in fosmetpantotenate or average PKAN-ADL the

measured will end study scale physicians. our be treatment patients clinically SPA looking change The a agreement, will a outlined each XX effect of from the change in point on at meaningful to X baseline the in be visit, score the weeks. through PKAN-ADL for and considered in As

to remain biometrics we our clinical to teams And are data, and the the continue blinded data the While our very pleased activities. study. our be we validation complete to conduct of CRO with working

and in validate initiated range score our on with the PKAN-ADL mentioned XX of as States, four we as with population, and characteristics seen upper the the are work their with to study the we outside the a baseline include patients well remain consistent done scale. As representative call, a what United in PKAN-ADL who last physician programs the have baseline

Also, expected we're overall is a X population confidence points support the the of looking the FORT. study at pairing us blinded design. These study range on basis to in within give variability of the the the key

double-blind visits. patients therapy than patients that are the FORT all who now portion label years. in some have continue point, entered this At extension study with of study more open for completed X and receiving the We the

Given the if to having filings, anticipate and data MAA robust continued set examine are support we our participation NDA label open a data positive. extension, FORT and in to the

Our development submission well positioned filings to materials, regulatory our are our submit team and we advancing XXXX. in is

over to shift sparsentan. now I'll

like During the focal discussions continue need recurring in Medical a thought all unmet in and there segmental second The our leaders quarter, key at of engage nephropathy. FSGS Congresses, glomerulosclerosis, ERA-EDTA. or that significant is and theme we with both physicians IgA is to

will eager for non-ISTs use. potential profile physicians non-immunosuppressive options explore -- treatment to long-term Specifically, are be or with [ph] that appropriate IS a

evaluate a as rare or to well how of to nephrologists and believe are that from therapy patient our and pivotal choice DUPLEX are sparsentan for sparsentan hear work, in continue potentially Phase position designed, treatment could We renal disease. as studies advisors a option X PROTECT and

study. quarter, second which sparsentan During is and the Monitoring studies. that first evaluating FSGS plan as Committee the report scheduled the to both had DUPLEX conduct with the with proceed we pleased DUPLEX pleased both continues to potential and remain I'm Data to protected DMC for recommended PROTECT. And and nephro our meeting enroll of

rate at the planned mentioned, of quickly in recent been of up we've months. enrollment Eric increase to did expected As But of move the not implementation able our pace act our to initiatives. some

renal recently centers partners the an study. accelerated registries For eligible pathology at activities example, we’ve [ph] reach with to for and patients’ broaden

We excellence. know of centers reach or population. from experience sites academic are of broader us efforts significant our referral there a allow patients to DUET, numbers that with outside of These

We with confidence are pleased us outcome deliver very promising and of in these for have seeing the from we potential and increasing a study, be positive study efforts. of new a result gives Most a treatment to FSGS. enrolled as we This the right standard the momentum and the are importantly, patients getting conduct that the continue DUPLEX. to

of Study, PROTECT remains X potential IgA sparsentan Our which track. is on and evaluating the phase nephropathy

the obtained has the allowed engagement us Our to we and drive we build an and done seeing DUPLEX the strong while clinical to trends. in practices enrollment that leveraging PROTECT job knowledge teams develop footprint out study. of and exceptional are we rolling have best This tailored have translate

turn call financial now update. Laura, for to me over Let a Laura? the

Laura Clague

you, Thank Noah.

grew from for portfolio keeps strong sales our on period the second increase an X% performance $XX.X year. guided million, in to the the product net XXXX. track growth full This us over During quarter, same our reach commercial to

non-GAAP On income tax, basis net to million evaluating quarter X XXXX. product million. of and for support The After of a attributable for GAAP reported our non-cash our clinical for same trials million expenses loss and period a expenses three GAAP loss higher a expenses largely $XX.X the efforts, of net were to we’ve second including over reported and adjusting second the increase R&D development $XX.X $XX.X the fosmetpantotenate sparsentan. in quarter We’ve XXXX. Phase is XXXX

R&D expenses basis, second for compensation adjusted an for non-cash the On million of $XX.X second million expenses Relevant the included quarter stock were $X.X and quarter. based amortization.

attributable the growth over professional the administrative is and and were for to our in general quarter operational largely Selling, XXXX increase headcount and expenses same $XX of fees. increased the period million, support

CEO, were settlement our fees the outstanding arbitration to during including due the was the founding dispute fees Company legal completed materially matters, to pertaining between higher and quarter. the and that Professional litigation quarter of its during

On million in quarter were non-GAAP expenses basis depreciation for the $X.X Significant consisted compensation amortization. an non-cash $XX.X of adjusted and for the and adjustments SG&A stock second million. quarter based

sheet remains balance strong. Our

converted the us a During our to XXXX quarter of This outstanding. last issued were were $XX convertible that shares in notes to to roughly notes leaves convertible second $XXX remaining million XXXX million common September.

to XXth we of support of As cash equivalents June ongoing million development XXXX $XXX.X effort. and have cash

we modest manufacturing readiness, of upward the to For expenses X programs R&D Phase remainder but result XXXX we quarter, current continue three scaling we levels this a our trend as material for may overall, anticipate the our by a as resulting commercial of anticipate throughout vary year. from in advance clinical and

Our should the quarter, variability first level seen XXXX. maybe base return of throughout but professional to fees continued expense the in there balance SG&A in

quarter. As decision write-off announced associated the investment of a of program, the $XX third during long-term a approximate on anticipate result we million an recently CNSA-XXX the

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

Eric Dube

strengthen in Thank first also and you, of to half year the the good made organization. Laura. progress continue We our

-- as mission at than be During Board our to our life-changing development Biopharmaceutical that delivering we disease. XX board living develop manufacturing brings were to further of to time Sandra more years joins welcome a people Directors. the product therapies us rare to to with Sandra quarter, Poole pivotal and to experience, and pleased of invaluable will we the

fosmetpantotenate XXXX, in and FORT to the of readout priority X successful NDA We're data balance submissions which for execution. the be focus would on study treatment MAA approved our quarter, For forward make first the to our will to support upcoming if potentially looking Phase later PKAN. the from

on program, sparsentan, have the potential launch of of recent focused on of treatment patients fosmetpantotenate achieving focused we a with to enrollment make And two our executing approval IgA the that with THIOLA this become franchise accelerating has are are the treatment growth Beyond and choice our to in and nephropathy. cystinuria. new standard FSGS we EC, continue for studies that for pivotal to successful the

now it back me over to Let for Chris, the open Chris? turn to up call questions.

Chris Cline

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

Operator

Maury comes of Our Instructions] [Operator question line first from the Raycroft from Jefferies.

open. is line Your

Maury Raycroft

taking afternoon, Good questions. everyone. thanks Hi, my and for

First question is for Noah.

acceleration activities to example enrollment of registries at one you the an of So mentioned is initiative enhance taken in FSGS.

made can Just were any initiatives of comment wondering if on enrollment? you the other potentially enhance to that

Noah Rosenberg

great Yeah. Thanks Maury, question.

First, let say to-date. confident new cognitive with very in enrollment, the pleased are we've regarding timelines. And study the been me our that, in we delay

ensuring main is thing the focus study. So, a high on that quality we

that, of And to up, clinical doing challenges the to open acceleration begin as was is enrollment rate sites these fast not pick know, the types traction. a as in pulling for the you trials in of a I forward. you team continue as tactics DUPLEX more of And and shift for lot this just these give our timelines. to summer want be recognizing months, expected to our we to plan early and caused credit as which gain

and leadership to the build patient Our the relationships key to pathology of FSGS in registries the study identification to centers groups, question academic built centers, and allowed has drive DUET excellence. to leverage your get outside key of through and with us

we're in enrollment, DUET reach databases just I of that, see want a have meaningful So, starting to significant found and being foundational able re-emphasize our that. on to sort we impact geographic these to can that work

Maury Raycroft

there to sites, additional and add may you some into anticipated? Can seeing than more it. be that sites have Got any granularity you may you're sites are underperforming what at provide

Eric Dube

Eric. comment, let Yeah, Maury, this me is

the what Just based the Noah really pleased studies the type terms engagement, of mentioned, number the on of the with receiving and that patients in we're of of the we're study. the -- conduct into both site

concerning I think would there. be so that And there's anything not that

lowest trial if continue the areas things, step was see rare leader disease space. as the think and that Nephrology lowest we one over I back, the had among was nephrology. activity therapy other glomerular of the few glomerular the decades take clinical last one that within a the to we've

that trial were so, how sure academic just and regional to sites, building seen. that where U.S. we broader, the nephrology we're we And academic the have make do foundation, sites hundred in [ph] the link majority those patients really but to X,XXX of few not clinical and community the -- over centers

lot connectivity ongoing, building this of tactics that And have planned, is into and that I capability have Noah continue that we sites registry does, is area. to And mentioned sites that increasing think we have we build clinical what to a the and been so help productivity. within to nephrology the we really on what think focusing that the make and trial that sure the a build I

Maury Raycroft

it. question a just then Got That's really helpful. on And THIOLA.

the that formulation. for to who at So patients point back the come one THIOLA you potentially new mentioned could have the some drug discontinued of

So as can first to discontinued pill if for lower burden I'm you the the would patients why And say in or else wondering place. any can they have about be primarily just that? coming if you provide specifics what would the back

if then with your could USPTO new patent if for potentially there? support the approval your that help conversation formulation, And

Eric Dube

this of Yeah, any wouldn't me I specific early physicians from Other and response with, for THIOLA so types what the seeing. very the of profile patients. start on from knew the of provide informed potential challenges let by just positive on we're we're of formulation through say, what there first EC details THIOLA patients. heard We we've was much able very and current and that patients, years than, prior last first seeing demand couple it's seeing significant development all, of be that over terms be going patients. the the we're in a and the and to would in a I'd based

to that uptake we of sure areas there awareness cystinuria three and the continue of to that build focus are And make the focus And so, our education really, EC. around for is have potential treatment we and THIOLA options.

of drive think them a of to patients for the physicians therapy about because The profile. them allow first EC THIOLA new and and to to choice is, course, as to continue to their

or The of see second, without pill that the may the different and is choice with current for patients food food them, to given work this burden. aspects

And reengaging we reasons EC a stops make the want discontinued. that reasons, they why THIOLA right the some therapy them. who those patients, with is of and regard patients reengage to whether your sure for therapy to for number around question they or with choice are We that know for THIOLA

that We patients these reasons. importantly, much is also our on not to But for know make that want And patients. putting just of about are and we talked host very whole why that, it up be we may that a goal patients any sure their therapy barriers discontinue these therapy and that's optimized. most for

as And think I'm And to number particularly being growth who of of the patients in of to but the EC, continue XXXX. able to therapy, patients. we're just I that as THIOLA this prescriptions, pleased of reengaging on those number based a including shipped we for back have franchise patients confident outlook a far stopped in say,

meeting we're so we market patients. pleased these a expected for THIOLA And see, research, EC would is that to have really as need from

mentioned with filed would coverage question for a undergoing about US have EC. THIOLA say whether your And That periods those to has when is regard can't of the as type are receive really we was Office. there know, We we review Patent pending, patent with it's and any that patent, that review. iterative. you Now been

the access another to And EC and bring and our for as so THIOLA ensuring remains support for community choice patients. education to these cystinuria focus

Maury Raycroft

helpful. I'll That's it. you in Got very Thank hop And much. back very queue. the

Eric Dube

can we go Rusty, next to the and Mark. go Thanks ahead, please. question,

Operator

from Sure, Michelle Gilson sir, the from Canaccord question comes of our next line Genuity.

open. is line Your

Michelle Gilson

should could I analysis are results, kinds what expecting be us a little of thank the we wondering Phase was top-line release? tell if just you taking and question. a from X about planned bit Hi, for more you my around what FORT

the commercial in view past there talked from you've at the different sub-domains certain regulatory the or as of domains looking a score, think perspective? are I about then you more And that PKAN-ADL important

Eric Dube

more Eric, is this to me Michelle, provide I'll bit little let me let ask Thanks, Noah detail. a and start

this I for and well PKAN-ADL, very as primary our period. XX think And and will as as is data around first weeks tolerability of the priority release, we've double sometime our quarter. analysis much foremost, safety the mentioned, the blind endpoint be

So, near-term. certainly a it is

are that. and abstracts, of a reason then also is September, able We there's couple at which not [ph], NDS some and in conferences, on in timing October, another to including conference the those depending acceptance of looking meet for we're

early with think on regard domains, as in first the I families focus of that heard experience as that and really some I'll I with well the start with comments, to publications conference patients PKAN. what from I my mentioned

most PKAN debilitating, caregivers, oftentimes patients time difficult these that that part I face stay from are Some of with majority between or these and of the symptoms require the we families And the and patients make giving. care or oftentimes symptoms full much are or patients most understand require writing emerged, or such these that knowing see of it walking difficulties. connected patients that or giving more are debilitating care that communicate to the some speech, that their think is and as symptoms first what very

we've I we think likely the least as what double-blind would period, we thinking versus then what that's three difference the improvements really is in terms at study, hope our over those to believe about powered see domains. these. in to reflect ensuring, one of focus than see more we're point placebo And, of which a in

if you, to you provide. over would analysis there's additional to or see Noah, if I'd to like like comments, any turn it

Noah Rosenberg

Eric. Sure,

just provide a But there. think I depth a quite of add things. a I'll you couple bit

populations populations, or patients. the the what highlight the you kind of be pediatric there. just analysis Important Michelle, effect with would pediatric, third to about asked about of see think, analysis, to a sub we important I the of I mean, reveal subgroup one clearly

at are it There's classic in progression PKAN those age these Look disease. the see typical of know, get heterogeneity, there understand of disease. presentation for of to in fairly traditional the risk important progression, is that as a and you’re seen really onset, factors groups also studies. some you But

got in So plans analyze populations. we've place those to already

what I'll And really domains, we've the got touch us. change upon I the a said, upon three just then mean as SPA, point waterfront, far for covers Eric as regulatory FDA, agreed with not mainly with

And the and others mentioned, domains are patient aspiration really can’t have some of those about these hospitalized that has key carefully show can swallow, at think but look sure of look pneumonia, the they potentially are make too, there, But but couple to compelling because six things Eric modification of and we to we we're swelling that to and potential always there at, death. have because Obviously, reasons. want might, walking, kinds disease pneumonia, unlikely, they also months. there's be follow-up not in are going and

and a ability will patients. Eric Speech, this more say talk just, are independent, to better important really caregiver I even able give little patient I be when last thing the being of the articulation, families, these bit are as to is, to break finally, And to patients, a even dress to themselves. ability

any of point have back so a them. by again, meaningfulness, for on was caregivers, scale, it domains change there’s patients, a to And been would one and PKAN-ADL those the clinical designed physicians,

Michelle Gilson

you've the a PKAN prepare to patient for about Okay, and then in efforts. of what launch And, population worldwide identification lot well. terms a little for as can doing you talked you're about talk bit

the So outside the rest thinking how access you're world the about of U.S.?

Eric Dube

Michelle. you, thank Yeah,

U.S. we So linked certainly, focusing been and of are first, has to on where and Europe, these efforts, the our patients many care. are

of number that centers or have clinicians. seen to trials our those clinical with centers is have in to specialty or patients identify by the looked engage of efforts of Some in excellence, those been

in mentioned the those where what of patients that we are space says some number patients have refine to are through identified What been of that's literature country-by-country. there where about sure is XXXX And XXX the And this the context that worldwide. make we've patients doing efforts. really over currently, and epidemiology we're in understand that the to previously they of,

sure and who We are is this experience patient the making are clinicians also treating journey what we identify, the that to and diagnosis.

in oftentimes proper in in know an disease, and number challenge the identifying the diagnosis. years that biggest patients rare is takes between the with it We and ultra-rare disease, symptom particularly of onset,

make close that that we And gap. really journey. patient understanding that's And want so by can we to sure that

some front. once think And really have the on to I that we we've do mentioned in also made we in of do move quickly make that can before, that what approval. sure key progress I we're very good part that place so is roles those trying team we gain

Michelle Gilson

Okay, thank you.

Eric Dube

Thanks, Michelle.

Operator

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

is open. line Your

Tim Lugo

Thanks for taking the question.

legacy the maybe much THIOLA or formulation prior to you could Going patients who maybe provide how think EC. and discontinued some formulation how back you the to some to expect the of patients about the convert EC? you of quantification tried growth numbers and Can this be to long on base Also, business?

Eric Dube

for Tim, Thanks, the Yes. question.

many the access So we other been introduction foreseeable therapies been awareness serve for the rare treatment do so available. focus having market the despite continue grow THIOLA increasing Cystinuria, new in why options to that's has the with to under of expect and diseases ours. like future, especially education EC. has of a And central

THIOLA patients XX% total the a of the number those to treated time of from about around was acquiring increase about to XXX since number answer And we've that question XXXX. ago, we’ve of seen that being than of rights patients, therapy. five THIOLA the your number period And years over with patients to of seen more have discontinued

there those treatment led the patients are the the still formulation treatment. even untreated. utility patients as of with And just of but majority we for to cystinuria patients beyond these not despite discontinue that And THIOLA’s challenges overcome, have many that are with to know diagnosis, that, choice. that that's existing And

continue commitment education options to efforts our a furthering the And and making THIOLA to challenges. EC awareness our through is addresses about goal available, by this Our community those treatment of available. which number

their optimally and they're have samples patients bonafide for quarter. can't the a make a once to a really samples want upon treatment, so treated, the choice opportunity THIOLA of build whether to to also cystinuria at generic, some with work point, during provide that that requesters. treated, policy community have the an say, there's our providing with and sure We we're provide is I that third in to we the process we patient And

terms we some EC treatment new they've in know and foreseeable potential education and the future treated and the to THIOLA years. EC the patients remain we the lived What continue result of option this, address the to future. growth that uptake, -- in is treatment as our we're that grow I focused for a will of patients for of number challenges And access, commitment to say of the in with the help to to THIOLA over bringing confident of

we So of what its optimistic this disease treatment, and launch. think, and this signs early of heard early all-in-all, I it in we're reinforces about the on, the challenges

Tim Lugo

to for Thank if what perhaps in remind the squeeze Maybe you placebo Noah, Can you on me you -- for the can thorough more response PKAN. expectations remind study? answer. I are in one me could this

Noah Rosenberg

a great that's Tim. question, Yes,

XX the in recall, expect months X you we week if meaningful So, a placebo or change the arm over don't period.

disease what spontaneous PKAN limited, is interactions natural years, improvement. know by over in would research typically While do the not been call we our from characterized history the has I

but presentation. depending on expected So to pretty rates, decline varying at debilitated sick these patients, are disease they're

is the types these is and see usually you of be sustained. these on would of work context placebo unlikely how can severe UPDRS in pretty Parkinson's, some low improvement, given think So, are, in while about improvement patients some effect, we that there that it's scales

arm emphasize, population, I to on Part recent UPDRS study, a XX want II And X over this studying months. declined X placebo points to another just was there

think important I look the study powering seeing as most answer assumptions. that at the supports And, really question. the the thesis. So Tim, on that hope variability within that basis, our part, I we helps we're overall blinded

Tim Lugo

forward looking results. We're Thanks to for helpful. the Very answer. the

Noah Rosenberg

Tim. Thanks Awesome.

Operator

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

is line Your open.

Joseph Schwartz

much. very Thanks

September, couple a wondering announced. third of the I the in you more fourth accepted heard the we one the Do you quarter? us is not insight data the if in to be think meeting meetings, you expect any release were actually advance when a expect quarter. do data might meeting? any data event, if think the data Or a we So was we mentioned at give -- I in into medical top-line of slip in press the quarter, can just into might the which if that FORT in should for should you fourth

Noah Rosenberg

Yes, thank you, Joe.

an reviewed more hope with I'd and we peer high communication announcement, timing, I hear going work obviously that, for after a you top-line be journal. the we’ll quarter. beyond very release. tell within here will in medical that and to on getting we're complete the very So to And press or given will quickly anything sure quickly say in therapy data, us real that you'll the shortly space mentioned Certainly, conference to that we And third want from the third unblind make unmet quarter. unblind very work regard to to have we and haven't we the specific once need,

Joseph Schwartz

that hopefully And your for on use from any your now Do thoughts about have has leverage work, it PKAN? US what Okay, know able outside approved. US. plans then you order THIOLA the you been commercial outside addressing maybe was the to and footprint to your good the in cystinuria, we feel footprint wondering, for FDA you're that establish PKAN inside bringing it US, a EC luck. given to and I

Noah Rosenberg

can and outside in question, right reach want effective we how work going and a about really U.S. Yes, And, looking Joe. having first have of what's the of that I make the mean, U.S. is outside sure in But what think do to to is region, what foremost model to worked make would patients. it's a model centers, we country not certainly, to is in U.S. Europe. we at a able for these every work that we're have great we have the an do that that in sure we the right distribution effectively work

at some will the within to be building, infrastructure through business be we'll the U.S. look I company. fosmet leverage And, the launch development think, our able foundation the activities. of portfolio, our like outside for U.S. to whether, that growth much Things we we're current And the really of of future for the or, have

Joseph Schwartz

you. thank Great,

Operator

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

line Your is open.

Christopher Marai

Hey, good taking question. afternoon. Thanks the for

program there, if your based elaborate on competitive was landscape wondering, I was of could regarding more little the PKU Just emerging the you specifically. decision a that maybe bit on sort very data or

that was out trial you one FORT to blinded data follow-up. comment make it that perhaps any then data had a -- on So other perhaps decision? had make of you're I've seeing, or the helped confidence And -- got the helps increased you other, you or like, that could

Noah Rosenberg

Chris, thanks the Sure. Yes. for questions.

in let with we blinded, is exclusive, is remains the sure make and independent we FORT confidence have me and the start FORT made I'd which Censa mutually around disciplined we around we we're that the each first of decision in programs very question, to our say have last me Let and decisions. study, study company greater that want and, a confidence. that

want program the to was to a I with CNSA joint remind Pharmaceuticals. that regard Censa development With everyone programs, you this and

so, really And in comments position not we on a or data. to the disclose are

a say what don't PKU, of we're to in on that Unfortunately, there would go program. to treatment, since in we’ve into position own I the So in a really the PKU. assessment detail unmet level the an be despite recognized our we not need data, form is, continues

the maybe requirements in forward with And it potential while our treatment become there didn't of a acquisition to for for ultimately pharmaceuticals. option, terms moving CNSA-XXX, meet

decision we’ve to That's Censa. And to just really able we've going be make the we're sure that, not want to for so any seen. the that recognized disclose data that I of just

Christopher Marai

just daily study, then wondering benefit something that of burden, any guess study, the about had endpoint I And that's it. well the respect FORT primary extension, the some patients on to have and highlighted maybe been open with two past label you drug for years, there's Got was compliance had Thanks. be if secondarily details that perhaps pill thing you that you share would kind of interest? on three anecdotes be that could any or I times the of could

Eric Dube

Thank you, Chris.

say able data are label really extension. are receive double-blind that that to XX and treatment. to the be placebo to sure in we gather able position be real then to of longer-term any in in we weeks to that to remains including about period. treatment that blind, the active want I the be patients two anecdotes, to going the I that respect open extension blinded Let safety and efficacy the open will to first objective make me secondly, we and switch patients able fold, and mean remind study a everyone not the but those to in label after foremost of fosmet talk

a remains we will certainly look so that and double-blind the quality explore but analysis our on And foremost of to first high period. and readout priority

the remains So, anecdotes of focus we I will analysis for that save our on think top-line. later,

The seen that dropout will and is with switched set that we that that at. support study. a that a I we have cetera, encouraging of Whether I rate But look pill it's quality remains data all be that to this something patients have will burden double-blind we think remains high to able the study, analysis say low at regard anything robust think be thing to compliance, to will be certain I conquer completed other the overall et can around period over.

Christopher Marai

not two EU and file the require. to some you it. The be maybe potential you're thought around how opting at of and at last Thank assets And your part. would if maybe into license then one that looking you time same given and question, there potential? then that explain Got you roughly, program, to other one partner you earlier could then NDA of into kind time process Maybe license expect you. look do just the an CNSA -- MAA at

Eric Dube

thanks, Sure, yeah, Chris.

to NDA close are will simultaneous but to those be won't They with first certainly as regard that they make and sure submitted possible. closed. the want so as Okay, MAA be submission, we

I think a provide after in be to they that timing we’ll cetera. readout et position better

go-to With this Europe where partner, regard that on and a one commercial be market to model able potential our this will to we own. believe or I our in model do is

referral the we excellence with takes in pattern the disease we for We rare patients having a to focus regard that of enough opportunity operating that it what start is of reasonable have of and model relationships of that lot us in in believe PKAN that have to the for strong the building and approach centers understanding Europe. with identifying

by will distribution, but be Retrophin. have driven access really to we et cetera, market the partners Certainly, and overall regard with will model

and to with the front of then, of as that agreement development. PKU, refill joint of so final the active to your question pipeline at around Censa want program make then completion us, X with within completion look development, And continuing both business the fosmet we Phase we and we do are in what's our the development business sure very we in be the

partner need. have development focus and also We an launch want only such late believe there that for this strong and focused, is we our as is makes a stage in ultra-rare we to we company continue to where attractive on unmet disease that a think rare capabilities future stay high getting We stronger that foundation assets.

So stay nothing tuned, that. but beyond specific

Christopher Marai

considered molecule? in queue. anything as traditionally on you jump so small just back then Thank biologics Sorry, one molecules that, as And small one be will quick well I'll much.

Eric Dube

Yeah, thanks, Chris.

we've say we the So think is beyond at to are I the off being table nothing say we or that small really manufacturing look got by capabilities or and certainly that advance molecule this would I a and therapy. open sure understand within the molecule. would want I as to point, opportunity begin development, required make whether to that to diseases it's we see are what logic genetic we for commercialization, small

also our help making in this where addition is to Board will of think I us the informed even more Sandra So, choices.

Christopher Marai

you. Thank

Operator

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

Your line is open.

Liisa Bayko

recently the off probably comment, looking maybe you that for for more question. just did Syndrome, kind kind the of do. are on a partnership stage Could just just of you're Hi, stage investment looking assets? the you pipeline? the early you kind last terms of that of of know later to is comment see you just you of need could a indicative in where more I stage Perhaps, Alagille if

Noah Rosenberg

Yeah. Thanks, Liisa.

that's asset have want an fit Phase stage, And two one sure move within is we going we to that we've early portfolio, make We our late have and X. X, to within I think will say, through fit certainly look what as to our as I'd capabilities. Phase asset in now we got

say something I Phase certainly would pipeline, X, want we've will just We X, in in don't something shut to that example. got off because to we look but that for is fill Phase

would So, saying and asset probably be going sure reflective the that what the of of to be assess deal involved. of we make We'll long have structure that a we're stage risk that a development. regard are, to way risks of opportunistic, with the

Liisa Bayko

can you about some What kind which of a looking EMA, and Okay. its of bit to short the a totality, terms PKAN, course endpoint, of maybe be what data confidence that be for FDA you'd the you also flexibility data in there's about of give brainer. you like, And then how no would a on of stats primary anticipate indication, do and things be key signal? clear in hitting the just thinking the drug little this would you're shifting talk to

Noah Rosenberg

point our focus his difference the That I'll I positive on a be we turn versus likely, would Bill, not mean, get that. which approach, said, main so of reason, we terms to over to so is a to regulatory the for some views placebo. have meets Yeah, SPA, obviously, it study that that do ensure three see

William Rote

at we differentiated point, down come [ph], that to they view see, what to placebo. the How on that Atlantic is of think both -- then a sides you and Certainly, signal significant how with from how it. the the effect and I going durable is relative is, that it's the that, discussion to see around the agencies how

to flexibility come prior was clinical from trials, severe was was no U.S. borderline with you Certainly, existing that in especially in regulatory have and therapies. demonstrated and results where regulatory agencies have approvals, to allowed where such there cases the there European bear, diseases, both situations that drug

you're with partnering really to are agencies that space, when that, the and think trying I find solutions. you in

FDA how that the for to patients, forward. path. going about going what's if that be we're the that best we at where thinking have do not we to we want So, to certainly point develop and but be, And have to there, talk situation are we and

Liisa Bayko

one on recently, a and there wondering you literature, more was could Okay, then we about I on patients calls feeling there if that? that on you comment report there could the was how you're hosted great. like on just And report of was patient a comment that, thinking then KOL numbers, out

Eric Dube

see comment could that, approval prevalence effective Yeah, I is and ultra-rare then certainly that. be diseases say do that that on there's There Liisa, increased Eric. be I'll cautious and this other an We would with rare that, therapy, there one. you want is on numbers. to

prepare we're work the And part disease. that the for launch of epidemiology doing update within this is to so, to some of

hasn't there know been We including much around PKAN. that [ph] as NBIAs

we're it's have point, that XXXX around so, us this share literature at have to we'll only updated, worldwide that be But patients. work prevalence continuing whether And And once been this, on really that. certainly completed of it’s focus anything and the recently we worldwide. the which would led to XXXX

Liisa Bayko

then Thank final XQ? for fully question, the that is in write-down option taken the you. Okay, final just And And that's be it the question. Censa will my great.

Eric Dube

Liisa. Thanks

Laura Clague

Eric. will QX. once Hi, Liisa. Yeah, write-down sorry, Yeah, at take that full in we

Liisa Bayko

Thank you.

Operator

from Gena of Our last line Wang question Barclays. the from comes

open. Your line is

Gena Wang

regarding for questions. questions my Thank I have a few you taking program. also PKAN

progression the year? terms, onset First, the just More wondering disease what natural then like of for history adult at patients in is course patients? pediatric and PKAN-ADL the the data and domains affected and for

Eric Dube

last domains is PKAN to you, understand in conducted natural the natural across have don't Where of Gena within symptom for evolving the with question. was about I we the been what XX caregivers we've PKAN-ADL, Thank recognition of those, really has patients our validate would -- is, burden We disease. with understanding done PKAN. the PKAN-ADL. history a which of within that to that and of published focusing over say some the couple work an years efforts the the have this greater Liisa been history your their

a scale symptomatology was just that similar for UPDRS based given disease in of the on the condition. Parkinson's And background, PKAN-ADL

disease. we is is know What progressive this PKAN a that about do

of this disease. for does pediatric specifically patients. mentioned rapid You earlier more of confer And progression age we know an a that onset

validation a to And we say in have And the that broader broad PKAN do to to one is FORT population six, study, data of well as validated enroll we PKAN-ADL within to safety patients age ensure use wanted that the clinical older. in also the things data the as the was support community. and the that study, and

this a assess one -- here we that the nice we'll we the with believe the study able what is placebo validity prior understand arm think in progression to So be I no condition. patients, the that that pediatric for and have mentioned, for to

Gena Wang

Okay.

Eric Dube

that helping disease. inform I quite think bit within answer, Gena, I a that this really long there's know to but we're

Gena Wang

Thank you.

try the So around question pediatric they patients like like fit stimulus patients, to another course? between baseline adult would like, asking different, scores them way and of would balance be very or you the is

Eric Dube

of a make we it's look, to disease. question, sure we we included have their that understand, data within headline group once broad age certainly part within sample study. that where the PKAN-ADL Yes, for great overall And we’ll the and why one scale, FORT the patients the that was of validation reason are

consistent how as will assumptions see as overall the the Noah baseline double-blind, adult, our we to the power well but think and in where with conduct what varies Whether that sample, the both seeing the or is of consistent study. variability assess, I our we scores, be by as we assumptions. in the And able in with we're mentioned, pediatric

Gena Wang

you… Just wondering if

Noah Rosenberg

that important patients of your us that's it why that, third I was Eric, emphasize, the in to population. to for pediatric Just point think get the really

So independently look analysis cohort. do we an that at can there,

of it's pediatric expected may current also, may quickly. younger more quicker. progress at proven. actually that [indiscernible] And [ph] been therefore But the recent onset those they in that evidence levels, be think suggests can that less to progress hasn't and the the population I related there earlier age groups, be that

being certainly looking both and that forward we're status to to the excited speak And about But to by KOL have really fact adult are and a the in and able we encouraged study we're soon. going our pediatric this population.

shortly. they benefit believe see potential there that there's and I think we'll

Gena Wang

on you your assumption. I'm Okay. that like if share, based you can wondering mentioned just

for is on patients assumption So for analysis and have a patient? subgroup lastly, And adult pre-specify do and you pediatric adult patients? your pediatric then also what patient

Noah Rosenberg

plan. pediatric So we're we the analysis will comfortable where analysis in fiscal with of we pre-specified a And now group. be in with terms are doing our assumptions our

Gena Wang

Thank great. you. Okay,

Eric Dube

Gena. Thanks,

Operator

this like call now no time. to to the questions There would are at Cline. turn I Chris back

Chris Cline

Rusty. again forward to Great. Thank update. with our here you speaking all. Thank quarter soon. look you, We This second you concludes

Operator

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

disconnect. all may You

Travere Therapeutics (TVTX) 6 Aug 192019 Q2 Earnings call transcript