Travere Therapeutics (TVTX) 8 May 222022 Q1 Earnings call transcript

Company Profile

Participants

Chris Cline	Senior Vice President-Investor Relations
Eric Dube	Chief Executive Officer
Jula Inrig	Chief Medical Officer
Peter Heerma	Chief Commercial Officer
Laura Clague	Chief Financial Officer
Bill Rote	Senior Vice President-Research & Development
Carter Gould	Barclays
Mary Kate Davis	Bank of America
Liisa Bayko	Evercore ISI
Maury Raycroft	Jefferies
Tim Lugo	William Blair
Laura Chico	Wedbush Securities
Ed Arce	H.C. Wainwright and Company

Call transcript

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Operator

Good day, and welcome to the Travere Therapeutics Conference Call. Today's conference is being recorded. At this time, I'd like to turn the conference over to the Senior Vice President of Investor Relations, Chris Cline. Please go ahead, sir.

Chris Cline

Great. Justin. you, Thank Good afternoon. First Update Therapeutics XXXX Results Travere Financial Quarter Corporate to Call. and welcome And for joining all you Thank us. will Chief Executive call Officer, Today's be Dr. led by our Dube. Eric will Dr. remarks the joined Eric by be for prepared Heerma, Jula Chief our Financial Laura Inrig, Officer, Commercial Dr. Medical Peter Chief our Clague; Officer; our Officer; and Chief join Bill Vice will Research Q&A the & of Senior for session. Rote, Development President us

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

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

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

Eric Dube

Thank XXXX. good our you, with afternoon. start Chris, and progress to I'm pleased very

application the our achieved accelerated pipeline drug our preparing first the the quarter, of the and during first-in-class If we launch potential of this the submission forward notice treatments if an sparsentan NDA early or later review, as our FDA nephropathy review. is for key sparsentan of approval quarter, in NDA rare of as and this for NDA, approved. approved. on-time of the for would of end advancing We to disease sparsentan the the During is granted we living notably of anticipate nephropathy. made our are priority significant potential on we for new priorities IgA Most is month people look IgA with as well as regarding accepted we in launches headway organization timeline acceptance receiving year from a

are additional prior providing to we eGFR before ongoing quarter, submit regulatory FDA study consistent the providing guidance, following DUPLEX the be We our will our updates an we this will incremental track on data with interactions, update also to from then. not be but

half positive further we following a potential people homocystinuria regulators exciting pivotal to an submit to X/X a Subpart for month. in we full which FSGS. submission we As recommends the alignment the a living for or potential on either combined H that FDA our submit approval will our for its SIMD look for study of we of Pharma, a European remain believe outcomes Vifor next case, of sparsentan treatment with I’m regulatory FDA would the MAA submit HCU. are with in our conditional In the is path Pegtibatinase coming the discussions at program. pending presentation the pegtibatinase, of data in COMPOSE new full FSGS ongoing under in Europe, the or supportive of the XXXX. And an last from U.S. accelerated also NDA until the classical continuing reception Together And pleased preparing to months that instead we submission represents reminder, subsequently nephropathy be expect of first of also partner, prepared of with study in approval we Phase wait to the with to completion approval progress IgA forward and year. that the design with

challenging of side the a environment. line quarter with was the for strong commercial and expectations in in business, the On performance our

year that sales, update, of for As net Thiola. in not our dynamics to overall we due many do XXXX, product generic of you we anticipate in the will outlined end during growth recall

consistent is Demand products performance guidance. quarter first with across quarter that Our all increased. in the

In demand the patient expected, of to But evolving business. to business organization continues cystinuria prepare to the and the levels the by portfolio overall in planning incredibly building fact, dynamics our the launch commercial I for and seeing despite strengths from return perform is and accounted are to team's end approved. ability be U.S. a as well. the for a expertise a put Notably current for fantastic as bioasset landscape to continue our shift we of our support revenue pre-pandemic in the to of net beginning This early the pressure competitive and the on in as year are Thiola and of impressed to our team sales upon the job challenges. these community maintain sparsentan continuity the of for continued Peter nephrology do for have in

I both perspective, kidney preparation confident if the standard from sparsentan and treatment to-date many rare the become in new result with a a patients currently rapid facing our a approved dialysis. As clinic to progress remain a can for disease that of commercial progression

XXXX extend incremental strengthened the convertible vast modest foundation majority balance resulted an the our the through maturity the to we convertible during transactions cash sheet. These our XXXX, refinancing us add able in in March. to notes being financial to of on Finally, transactions from quarter, enacted effectively the

Let update. turn the to Jula Jula? clinical over a me now call for

Jula Inrig

report Thank advanced programs. first in all pleased and our to of afternoon. I'm quarter you, clinical the we Eric that good

treatment the desperate need the most long-term patients looking transplantation. the the submission with an seen proteinuria IgA approval non-immunosuppressive was in progression an the is to accelerated RAS IgA achievement dialysis many nephropathy sparsentan, nephropathy. with And need For novel treatments. notable Physicians and steroids attempt a treating for reduce nephropathy for quarter IgA there's for NDA avoid a during delay paradigm, If therapy of currently kidney This to become is a we've sparsentan at inhibitors Many what or are and their path It to limited foundational possible, years in patients for approved. the potential the to the sparsentan first the potentially ultimately non-immunosuppressive And treatment patients critical be physicians. submission nephropathy. to to effectiveness are has believe delivering the was a NDA pace at in to progressing for safety. indicated that disease. IgA available on end-stage the still are rapid we step and March for future. ability in treatments has

our IgA PROTECT receive accept from Given review the acceptance nephropathy the a we as requested and in to if a submission throughout data response submission, priority forward granted, year. is month, as review process. of on study. this would PDUFA potential need expect of significant unmet this of strength the look the early in working November and with and the review the We later approval timeline the we a to We priority action review the date FDA

Eric We as track of on as this our study our quarter last regulatory the for will requirements and update Consistent duplex meeting earlier, Type also meet the accelerated to additional our provide agency's FDA data provide with the A year, interactions. remain ongoing had since an approval. once FSGS plans, eGFR we the additional from outlined with eGFR data we've should

both IgAN and our continue respectively, their quickly on well and as European submit will in progress that as DUPLEX studies two an with year-to-date year. to remain completion joint PROTECT, I’m the FSGS for used a of submission track MAA partner, next We FSGS position Vifor and for large Pharma. NDA be to and

upon from invaluable in six program continuing on discussions first for constraints commercial planning, the XXXX. continue ad extension cohort. of with COMPOSE our patients sustained build the and we COMPOSE the set pegtibatinase first pegtibatinase for of while the designed to metabolic the the initial pivotal open-label including global and regulators HCU. our we data In as modifying Finally, methionine and access. the to five received continued phase advancing study pegtibatinase Phase a become development to the the from uniformly Throughout The April, as and at decreases in SIMD And positive plan is SIMD. proof-of-concept incorporate to pivotal feedback data manufacturing excited and strong We first meeting are phase in for The results be presentations held rapid board to conjunction patient and in approach the of HCU. for advancement supply cohorts mean leaders working cohorts engagement to five we're the and continuing XXX to had on homocysteine COMPOSE at demonstrate and about program with CMC enrollment ongoing levels pegtibatinase the potential treatment for detailed homocysteine micromole improve opportunity study next can and pivotal the activities homocystinuria. the In annual a disease we support potential insights of parallel, study the achieving potential in development. KOLs, the of into present through overall we're data, dysfunction scale below X/X for an

progress with these our providing of are We to on look we later year. additional this pleased updates forward initiatives, both and

the Let me Peter? now for commercial the Peter to over call update. turn

Peter Heerma

Thank you, Jula.

which see the potential encouraging as organization, We continue commercial closer execution is if first from sparsentan move particularly our of to to we great launch approved.

sales initiate again, at first did slight which since quarter number more efforts, engage the to compared decline our with same generic has of team's last prescribers a deposition is initiating the our regularly. Thiola, see The net of We continued quarter first and increased visiting business of driven we This of is and with planning. therapies Despite year. the to patients increase consistent is XXXX. access result new this year in in believe our affecting dynamics the patients the product highest new expectations by patients therapy, a period

in support of XX% driven pressure the patients the balance Thiola in year identifying primarily team's the year, first work As growth the acid to experienced that over continue continue organic on quarter, the Cholbam. community. we our new bile same to we will product the we execution year, period guided the by with last cystinuria Notably further expect and through of the beginning

the nature While Cholbam particularly see is bile to a even which we this believe of for rare with ultra a they products treat, often was the to acid our continuous conditions Cholbam, we quarter engagements. suite’s the due quality testament an strong performance

treat We year, expected expectations performance on the in generic is upcoming rare of growths patients identify bile launches within added our our and a Thiola. acid provides prepare sparsentan for it confidence it importantly, our of with XXXX, continue ability demonstrate to expect Furthermore, and our products if diseases. execution, Most we continues approved. impact start is the partially as and to in to planning to potential modest which to the offset

the of points team FSGS. three outset education. we the year, And our of move medical Advancing the helpful compelling will be provide treat to will expanding at ready our value the we commercial develop we to support I to pleased proposition our patients the on is working our launches physicians disease through deeper nephropathy with to those balance continue very understanding a leverage and and areas state they XXXX, so the to who if ahead approved. of with nephrology IgA As goal infrastructure sparsentan established payers we can progress. with key outline focus continue be to

financial have extensive enable we treatment we over leadership rare remain example, incredibly the high Altogether, experience. and update. I – to nephrology approved. for hired in the candidates confident standard strong with sparsentan sales already will to For Laura for if sparsentan. senior field position to be have a turn new ultimately quality nephrology, a Laura? call leadership to based that senior now become will roles support demands we our from rare And we in

Laura Clague

Peter. you, Thank

revenue first of reported European product sales net partnership $XX.X revenue million total the compares in product net with approximately $XX.X Pharma. million our of for and consisting collaboration million, from the XXXX. period This in of $X in sales in reported we For XXXX, same to million quarter Vifor $XX.X

did us to beginning for As we in higher driven growth of typical has net the discounts coverage in year. quarter, past, experience changes the been in first by the insurance new years

for XXXX planning expenses first for for years our $XX.X non-cash are well million adjusting increased We is quarter for quarter After of million of the of basis, in and continue were the $XX the a of for $XX.X of XXXX the the quarter commercial first basis, discounts expected largely quarter. continue of we higher R&D and loss as largely the potential for we increase quarter income well non-GAAP On the from expenses expenses narrow attributable were $XX.X million anticipate the sparsentan as million IgA SG&A non-cash depreciation $XX.X for to foundational The including an run fourth adjustments for work, year, rate of attributable an Thiola a of HCU. build-out, at DUPLEX R&D million. the in As that XXXX quarter in our the $XX ongoing selling, On expenses reported were with loss as From be we commercial OpEx balance the This first million. included beginning headcount first launch quarter tax, ongoing Relevant SG&A through the we for first of as scaling to launch compared continue and The GAAP for CMC, XXXX. XXXX, the to year, non-cash and expenses level, and but as XXXX. studies basis, from PROTECT reported and an compensation first GAAP Significant pegtibatinase of adjusted the million net quarter for On expenses and pivotal of is quarter will compared preparations. of XXXX. pegtibatinase of a we increases the gross move field including Consistent stock-based expenses of XXXX. $XX.X a than quarter increase will and the to for to team than nephropathy. in stock-based compensation continue expect to the ongoing slightly in additions advancement net net perspective, the R&D a studies first of is of that program million business. into to a higher consisted GAAP our to previous were the study XXXX administrative prepare program. in to first the general adjusted $X.X the amortization. sparsentan will COMPOSE to representative amortization. basis, the On

million and approximately to the Importantly, March. in first cash XXXX $XX equivalents. facility, from refinancing convertible a and and transactions position be at-the-market beyond. in in our first execute from $XX financial We million quarter This the strong balance net in in convertible ended to prior affected in proceeds million we $XXX.X continue approximately the transaction cash to with our quarter includes

related but impact account, pegtibatinase, this XXXX. pay Thiola, factoring out. planned we still expect balance into the milestone We to further to on payments sparsentan, our being planned full pressure program achievements yet as for Taken of anticipate will in cash potential not pivotal is regulatory competitive into the for that support operations

Let his opening call back for hand Eric? Eric me over the now comments. to

Eric Dube

CFO Last XX has industry time her you, more can we disease so that thank Thank Laura. announced she dedication than would I contributions our in spend month, retire spending more decided years for unwavering after and than making biopharmaceutical rare Laura this Travere, family. opportunity the as a years into leader in to like to with in to seven to to of that her deserved much Travere community. Laura mission and take the

foundation. As strong financial Laura's a team finance an developed we a result and have of outstanding leadership,

standard that knowledge pleased new we strategic role for work to the our His of we ideally capital the Most if will and a for markets we I making the institutional August. to execution as sparsentan enter will him very goal in rare investment And community have new experience, very forward growth. to pleased treatment importantly, continuing CFO with Chris suited with am towards over disorders continued to this phase of closely be deep start are kidney We vision look role. the be Overall, progress approved. our taking year.

on the DUPLEX program during we approval FSGS. the pegtibatinase eGFR execution for sheet continue we have the data a strengthening regarding in And preparations the and for early launch from commercial to for of accelerated of track the we the Together the pathway FDA and planned be to time with XXXX. remain our advancement of the of balance well positioned throughout are continued as on NDA of as interactions furthered our our our balance this potential IgA quarter, year. end With submission nephropathy, our ready our

to the Chris? me turn Let Chris now for over call Q&A.

Chris Cline

Great. Thanks Eric.

ahead lines go and up Q&A? for the we can Justin, open please

Operator

Joseph today with from question And SVB Schwartz Securities. Instructions] comes [Operator our Absolutely. first

Unidentified Analyst

all. today recent Will Hi, in thank Joe This congrats progress. and questions taking a on our and for for you is

quarter? in would IgAN here, So a of early of addressable overall terms approach something like large one it we're higher segments the something with fourth market, patient population but the question the be in for the for curious other Is that who there's Thank to physicians with those the you. proteinuria relatively as are launch or us you are there obviously adopters target important sparsentan. in company potential

Eric Dube

thoughts assume and Jula great to start Peter, that your ask their maybe sparsentan. on and for uptake maybe thanks add question. Jula can I for might will where perspective. can Peter for you early give we you the Well, then

Peter Heerma

IgA spoken see market. the Yes, absolutely. Well, about we how we've nephropathy

we market is what think about sparsentan. lungs at to is XXX,XXX I the for total way like patient are XXX,XXX about patients, the the population addressable thinking

indicated have nephrologist XX,XXX the proteinuria progression. nephropathy have by through And the high rapid those that confirmed that are seen XX,XXX, the patients and seen diagnosis path that's patient on are and We are that biopsy, levels. are that are those to about of consistent IgA as a

on that initially. So are be the patients will those we focused

Unidentified Analyst

with Great. just follow one another report inhibitor, you. up. another And in an recently in ETA We if saw sponsor Thank also some sneak this IgA quick could space interim I data nephropathy.

competitive how of kind thinking potentially landscape about forward wondering moving market? players this multiple just the with you're So in

Eric Dube

positioned I you her early think on but approved. if up that Jula ask sparsentan there where important maybe that. data, I'll it's caveat well And and Jula, you to perspective pick very an will Sure. can interim give on are be those believe

Jula Inrig

patients risk current for very and new seeking believe ACE remain Yes, and treatment high at ultimately we IgA with the treatment out nephrologists treatment that is or will unmet be nephropathy there we need which have, remains options of options inhibitors ARBs. a significant

believe with PROTECT development XX% a so become yes, foundational we significant in we number with of we are foundational a therapy therapies other the that reduction proteinuria, that with And therapy will sparsentan in in therapy. And sparsentan it's saw approved. sparsentan there will if proteinuria reduction believe that be

the additive can potentially And be some niche of that. to other therapies

Unidentified Analyst

in again you congrats Thank progress. and Excellent.

Eric Dube

Thanks.

Operator

will question our And Carter Barclays. Gould with next come from

Carter Gould

go going launch? eGFR congrats we we're potential the plans and other I and since shown new the part the of afternoon, characteristics guess, of luck I It's terms data. other secondary the Laura FSGS, in question that additional been baseline and a are publish got anytime close understanding providing best soon. eGFR see you. Chris as there to of any – to still Thank or conversation? share to any any there Good be that role. you'll data we the then Are around not and on PROTECT to the first maybe next guess, days haven't beyond seen of data then round data is line to top in sort data already analysis XXX of or from that FDA kind before you've And to

Eric Dube

questions. for thanks the Carter, Yes,

the was endpoint trial at really of as an in discussed And I the the repeatedly proteinuria, integrity in risk I and beyond FDA. is of that until interim we've answer analysis completed focus year. past, the are think it half that's study the to those unblinding, for any could that of point the additional of think on area increase we potentially data the next primary of which the the blind both integrity first prioritize and going to managing is

company to interim study we're this we FDA we're a complete that with with data that to eGFR. we not that we analysis as actually not the the for everything minimal based have to and carefully set possible compromise FDA to And for point. compromise quarter, meeting any and such, final may making later anything that make unblinding ability So plans do additional within possible data the also any the or at eGFR do alpha analyses to this beyond We that would sure have analysis. to as are robust on maintain publish additional the do balancing sure and see, very that beyond doing on as could with update like want what sites our we look

So of have additional for at this beyond or disclosure have data we point, additional we analysis what to-date. plans do not

Carter Gould

All right. Thank you.

Eric Dube

Thank you.

Operator

you. Thank

of will America. question Harrison next Bank Greg come Our from with

Mary Kate Davis

the question. the are Good Mary you This Thank you. afternoon. expectations your Thank approved, IgAN ramp of is if in regard the and a what your Greg. what for so sparsentan maybe of to up and just question IgAN, regarding Maybe are expectations launch uptake on on initial for Kate reimbursement? much in sparsentan potential for our and taking cadence

Eric Dube

in expectations goal a that Kate, for that. Peter does can for questions. you those that, is more those. thanks standard order it bit and early is as little launch and us have to talk focus on strong new Mary that make to in talking do And as team, Peter that sparsentan bit area within about imagine, right. access populations. IgAN a team early I'll we is a and the become The but broad the And ask is after about need his have working very of his to we FSGS of within our terms so, sure to

Peter Heerma

Yes, very Mary Thank good. you, Kate.

seeing own early is of well community, I physician research, see the lot a external very as claim too very action large as those specifics patients mode well the we being not sparsentan therapy point, target of opportunity, of Jula’s consistent trend because the it's earlier treated that that consistently to among are we the would to research about think very how mechanistic foundational syndicated physicians to see. what research a excited uptake, like that the feel remain our to like done. we to as investor give but in actually see So Also that as

very question think see presented can to recognize getting well. about to KDIGO is [indiscernible], like interest with They the the the last there other the a thinking unmet they was really sparsentan With that new guidelines far and payers interactions need, with fall. So need, and position are regards with the pleased so we're we've response unmet high that's new that your payers treatment. also also we play, access, with with modalities regards innovative and as from had we

I So we are good think making progress.

think are very from very from getting proposition, pleased with we a more to I both building as so but physicians payer we think the as the community. so we're well I far, come, value are solid feedback

Eric Dube

the Yes, I thing that understanding for Peter, thank the recognize think mentioned what that conservative to we by nephrologists only information clinical that's very to make I is right, and much And that. to the approved. of you the Mary mechanism how are nature of evidence would these and to add the very that for them Kate, best that patients certainly planning Peter decision on part treatment. much once is focused a And we're give help the prepare and tools

Mary Kate Davis

you. Thank Great.

Eric Dube

you. Thank

Operator

question Bayko Liisa And our next Evercore from ISI. comes with

Liisa Bayko

– the well your in in affect is, Europe? MAA And the for submission subsequent FSGS my congrats progress. decision FDA First how question the data and eGFR

Eric Dube

question. that for Thanks

what with to may there timelines yet that. not to we provide focus that changed that But our affect. FDA on of to not we after make for to be us with our place, position plans MAA are may in around of change FDA in year. FSGS plans before see it's for track this our The be – understand middle submission we be have sure MAA. what any any met to-date. And we helps We've we in combined of timeline the provides anything FDA point, the our – and statement at and I that in that the mentioned that of think think I changed don't would before our this have the is So nothing has plans information an going that through

Liisa Bayko

one if add and into And your how you your I it color portfolio? does some more fit can is with on question can what collaboration rationale your you. like, Thank get PharmaKrysto

Eric Dube

Well, an or about is well asset rare believe pre-IND. potential asset is Sure. opportunity. program asset that suited our It this an nephrology This expertise. it early or is pleased really is is we're that within that we and

IND And nephrology next development. at years, excited so see much to certainly when because have of we that for us for another it that that, the point. we we're point, not And discussing an is do to the information continuing to this support but it's potential progress we we're more We'll share get community. something over rare opportunity the for couple and

Liisa Bayko

Great. Thank you.

Eric Dube

Thank you.

Operator

Raycroft with Jefferies. next And our Maury from question comes

Maury Raycroft

progress. thanks and taking on the for my congrats Hi, questions

You disclosed characteristics abstract. the ERA conference in PROTECT from IgAN baseline patient study some

Just should wondering abstract what what's your talk the you FDA what can if to determine cholestasis, help and contrasting look with contextualize going the disclosed drug on Tarpeyo to is and about focused with like? in ultimately be comparing maybe label

Eric Dube

may I'll Sure. which data ask FDA looking really share take for. Jula to the ask be the baseline providing for is to context what I'll Bill first question, then and

Jula Inrig

Sure.

a novel FSGS. in independently. the largest interventional populations. So testing is important to They're patient therapy representative at we are PROTECT and and we planned that is of IgAN both excited share our characteristics baseline And the think trials are ERA really from what DUPLEX both studies both

community share excited the this are And so nephrology to we populations unique. are and the data each of with

the and populations abstracts that representatively. patient you when through look can that we those cover And representative you so see

Bill Rote

for Maury. the question. take question, Thanks I'll the second

risk, I think versus a at point, budesonide. the on impacts it's if the sparsentan And benefit you heart the speculation which and contrast I is of labeling, potential compare where that look at this is of it but think eventual course, that high that reviewers a profile risk have long-term such a the where tolerability therapy of will with it's class, steroid you see added start therapies than with is very different upon. we any would be what other sparsentan degree the of

around agency broader was provided the to than I experience with what Tarpeyo. label has be would would you what of get that there Certainly of my study. premise So the you a hope been most based

in to and that need going it's that the risk So benefit see in to I’m wait the to based review, position confident has come again, we'll down very but on and sparsentan really arena.

Maury Raycroft

Got And say to some this on tough helpful. baseline on it. be there That's will or proteinuria do at characteristics you point? restrictions based think

Eric Dube

say point. to difficult very this at It's

Maury Raycroft

Okay.

Okay, maybe last quick question.

dual gotten potential first-in-class so a you getting an talk you've AdCom ARB what Just and ERA sparsentan and since chances an remind about feedback for AdCom is on of blocker. Can on far?

Eric Dube

you that? to Bill, Sure. would like take

Bill Rote

picture. certainly package expect we committee. but it our the We meetings would was during know for And have request ask no don't directly, what the their type the we we Certainly, it's could we'd with clear it's where that, AdCom. agency change. an that data, a normally prepared don't of question having and advisory answer an to not pre-NDA an committee ask be does, advisory if very them Strong anticipate now, we

we're in confident no, their to one. that don't So intend they first answer have

Maury Raycroft

Got it.

for Thanks Okay. my questions. taking

Eric Dube

Thanks, Maury.

Operator

on with moving Blair. Lugo Tim to William And

Tim Lugo

to for Laura Phase X And interaction? you congratulations about as six us chain question complete other or meeting, on need Thanks the is to these do cohort issues? the can I the the formulation, six of FDA they you the guess used on career, five? questions also before different talk on in how the Are remind new the Can as impacting pegtibatinase. a next global role. the you cohorts than your Chris end supply Briefly some well and formulation

Eric Dube

Sure. will to over those hand Tim, I Bill.

Bill Rote

Certainly. Thanks Tim.

biologics they're much so I in space, endemic we're not With to specific within seeing I of the manufacturer. think think manufacturing challenges the all they're pegtibatinase, that but that

manufacturing lot And into combination of of a have an the space. You full manage to was contract chain of vaccine world. manufacturer the already inserted a a supply COVID flexing around that

to made and obtain, more materials two slots it consumed of did raw and a manufacturing consumables. lot challenging up them It So that took things. and

that had been and we've those made areas with happy progress challenges we're we've have where those. working through the So the

stakeholders challenges for we've progress manufacturing continue important let know on the to side, that but been us are navigating. We make these it's that to

was using of half recall formulation, the liquid formulation X the low formulation Cohort This stability be X. that used for a temperatures. that'll first reasons, question at cohort second a is X, very the on frozen lyophilized through difference So was Cohorts your that it for

trial on to and clinical also patients and it much the on and supply going both but by the sponsor So the shipments formulation, our the the material material us collaborators, as the overall handling study sites. makes of and lyophilized easier the

Eric Dube

Thanks Bill.

bearing of question ones on those Tim, I that pace. they we yet in with supply continue working good it's thing And these making team progress of the through challenges is And the is think Bill and add, team his I expect one to just FDA. made believe I new. interactions timing we've that would shared any are be. that Bill's has our think challenges we don't progress the not again, on interactions, on and good We've These at would with of that those only that all past. great are are

Tim Lugo

other and everyone's seems I is seeing some are additional what Okay. overall assets, broader good And in there question pegtibatinase of around aware position your bringing thinking to giving could there. kind a that of. ask weakness we're sure a be market, cash out current the which assets change wide in kind transaction? of maybe Does I'm your

Eric Dube

it's Yes, I'll I'll start and further. anything a great then certainly provide Laura question, Tim. ask to

very companies know lack at and about that in that we Certainly or look capability out want a we there marketplace. That are to and move dilution, have any mindful be disease to sparsentan of have of may there successful a within that are continue to very be use of the there. out potential said, our there to and weakness We get We assets cash cash before their expertise opportunities the there. launch forward. either sensitive also is particularly lot rare

not one of there that landscape. diligence our all We're launch successful need that remains the number our down in monitoring Laura, of goal is want doing to sparsentan. you'd So invest anything going to add? that, but else to, slow we and to it's

Laura Clague

it you we on to Difficulty] XXXX, [Technical any your that undervalued our days. of point, Eric, I additional Difficulty] well, BD a point cash these do to to more work, cash, we on as little do the believe No, completing really call seem be is position mentioned course, covered our is but think Eric’s really [Technical

good never this you planned know there But there. could So a out nothing fit right point. at be

Eric Dube

Yes.

cash it's but going where we're any the use little a an of business reflection to is, our activities. with in and this but very going investment, think I to cash early, little would only very good it's continue, disciplined important I renal rare of development example the add and thing a within space. responsible Again, PharmaKrysto the in area could be be other we're Tim is

Tim Lugo

Great. you. Thank

Eric Dube

Thanks, Tim.

Operator

Gilson Michelle with from come will question Canaccord next our And Genuity.

Unidentified Analyst

on from Michael couple questions pegtibatinase. the Hello, this just is a on Michelle. quarter, Congrats more on

engagement versus path based regarding biomarker forward outcomes How regulators need endpoints? clinical the So and pegtibatinase? your with moving perhaps what to is secondary functional a forward has with been you on would what on show any

Eric Dube

how's and going to anything Jula, regulators engagement secondary the on, you're specialist Great. the the take that and from there's if that Bill, you biomarker, hearing question Thank else with on add can you, maybe you'd want maybe questions. for Michael, endpoints community. on the

Bill Rote

the Certainly, going path The well. is Michael. thanks questions, for forward

agency program, get on is design, in point essentially the enough for endpoint. clinical clinical trial dialogue And use how the et are specific are a to when biomarker what CMC efforts package, a with endpoints, as question those where an various endpoint homocystine guidance your on versus of working that, to total endpoint ideal we the biomarker around around and the We and a underway. as And appropriate completed is update iterative disease we've an the aspects This of we're agency qualify for cetera. of that. following we'll overall with their

on of mismatch it you trials use just think disease There's in what this take to because space, be be time longer I pivotal clinical would clinical place what would duration a than wanted the scale in these challenging endpoint of the which a are very required. changes are. if traditional

biomarker. is but homocysteine So ideal is as is using XXs, there setting a early and there the an precedent it a total that approved precedent of was all really drug from

to clinical So to we've those we'll around question you interactions, once achieve endpoints. the want if secondary optimistic completed able be that that take Jula, we're

Jula Inrig

disease progressive would as very the time. endpoints would those our in and mentioned, some patients develop Bill As years they be slowly collecting is of we many over but secondary be studies, in

many, of And endpoints effects for eventually homocysteine course the or short-term. able would goal So over many their a collected as of liberalize be perspective, to and events part would lowering such be those thrombotic direct years ocular not as their diet. over clinical their patients, from

that the time. of over and that eventually so the some be goal long-term, we'd to would information And to over ultimate capture able be hope

Unidentified Analyst

Excellent. Thank you.

How look an period? – IP how Just pegtibatinase. exclusivity And do you the on one quick thank long does anticipate one you. more

Eric Dube

that would have patent simplest do biologic I orphan the a be for disease approach pegtibatinase, think for for and Yes, the robust most family so but product. a we exclusivity granted period

you years XX So for plus can years that, and then assume in Europe. XX

Unidentified Analyst

Excellent. you. Thank

Eric Dube

you. Thank

Operator

come question And from Securities. Laura Wedbush with Chico our next will

Laura Chico

to, slope taking very focusing FSGS, of so for in FSGS I on study Good just in defined change population, endpoints our proteinuria. eGFR question. and first pivotal competitors your announced in much genetically the have but you a one evening. pursue Thank they'll single a they're the

with comment of more a sparsentan So elaborate terms made treatment. about Thank a and follow changing little bit a curious comment of well you a perhaps IgAN. earlier in wondering, a I'm I'm kind for foundational could development other the actually robustness as you or perhaps landscape broad to what features the foundational my FSGS is you be in if over sparsentan as maybe versus surrogate question serving of could component you. make MOAs regulatory on And suited just measure Jula see then time. up Eric how FPRE for lens, the specific you

Eric Dube

Certainly I'll that. take

of Jula of joining of anything company, of the the term proteinuria renal certainly is foundational I'll FPRE, element a and proteinuria the measure FPRE. renal add lot outcomes. to long-term involved and of survival her patients It largest But foundational the registries longer tying First ask before robust to in correlated work, is work with that on eGFR. because measures FSGS using reduction of is further, really a some robust

which function. a of high There eGFR in is else of a Jula, treating think to the making it's the profile in you about know measuring over program also to anything a is FPRE bar another focus strong is FSGS very as of foundational patients patients. want and in a are fundamentally I we was why it same these development. But that that of clinical is so then chose being It component therapy? subset and with important kind think aligned how of And sparsentan well nephrologists long-term, FPRE regulators. proteinuria, we confident for as on add which the proteinuria that

Jula Inrig

how aligned at a of is Yes. with which FPRE we which think in is remission. you're more looking FSGS, disease

of can – a getting a certain of measure and magnitude is and reduction. You patients proteinuria threshold to and a it

and or which and the to protecting time dialysis it that. is the strongest delaying And has So as captures correlation Eric measure, with function it said, kidney transplantation. eGFR preserving

it So has correlation greatest there. the

so reduction consistent proteinuria, of measure with of trial. a the ultimately other it it's However, is

for data we greatest And have so just endpoint. the that

we little believe sparsentan answer why then me foundational a be therapy. Let about further can bit

receptor sparsentan molecule. So itself a blocks in single and angiotensin endothelin

that combined the of and proteinuria benefit reduce is a you the single are so molecule. getting And mechanism in you

in And so proteinuria magnitude profile of with addition you get the the to reduction. that safety

in with from And significant blocker so on essentially is we've reduction an demonstrated to know their over effective of beneficial you patients an which angiotensin proteinuria. can proteinuria, or a we therapy receptor therapy reducing safe a and switch ACE inhibitor

be foundational therapy believe that's so far effective. we can why it And and so it's safe it's

Eric Dube

want add? Peter, anything to you'd And

Peter Heerma

our mechanistic therapies – allows itself they're hear need. that a very we What approach from also great add-on comfortable think it's it for in Jula, I that no, think physicians with the Yes, explanation. is because

speaking gives early to this are that add. allowing confidence with, what treatments see paradigm, the to that's treatment in physicians like needed additional think the frontline for modalities we I they if

Laura Chico

Chris much both very and Laura, the you congrats on to Thank and transition.

Eric Dube

Thank Laura. you,

Laura Clague

Thank you.

Operator

Kim Sandler. on Do Moving to with Piper

Unidentified Analyst

I that. is thinking Thank FSGS, you Hi, Kim. about on you you if different any could your approval indications taking dose of times could you dosing how color the Skyler of be are around for two when this or Do both question. thoughts for provide the around levels IgAN was my the get curious Thank pricing wondering IgAN. in you you. and I'm FSGS consider if

Eric Dube

Thank for question. the Sure. Skyler, you,

of talk we be vision lot dosing. pricing will given label of it's early specifics doing that of reimbursement to the sparsentan, the difference and sparsentan broad that there's his to for access about around are more to are bit and price we that sparsentan that achieve talk ensure in sparsentan to the ultimately is I'll sure That to illness strategy from that's the little but said, aspect potential a So team our economic I'd work foundational benefit is way able of approach the Peter us populations being health overall how and make difference, burden for of largely, the within to say therapy. to ask able the the about pricing. patients a assess our thinking

Peter Heerma

I think it's covered well majority you've said, Eric, there. I the think

more disease. progressive I different FSGS and also seen a as generally states it's disease think is

account is we of from taken, it those to have is dose a a Eric before aspects make perspective, all pricing pricing disease we you point a well. Your well take to as burden decision. consideration to into different take like there's point, also that And difference there's the but

IgAN and initial I think we there will already. competitor Our be launch in have a

So on I that think data flexibility come now. that that allows you the more right on are some based with we gathering to

Unidentified Analyst

Makes sense. you. Thank

Eric Dube

Skyler. Thanks

Operator

will question And our Wainwright H.C. with from Company. come and Arce next Ed

Ed Arce

but you for me, this, just for to wanted firstly thanks both to congrats slightly my Chris. Hi, pricing, taking let Laura on questions. add approach. Couple questions I And take my know I different me mentioned and a

agent, to As the get you decision that have and the up. on you you still in ultimately, take how see follow a and and a pricing specifically have the of of to non-immunosuppressant your value view context proposition general of label about in think but the but decision then process context course, I wanted in the being

Eric Dube

for like question, you, that? Peter, to the great. Okay, Ed, Thank take you would

Peter Heerma

why so important of excited path. your allows see had that's it increase about a Yes, them interim that again to And think steroid in you I versus both earlier, right. substantial cautions exciting this the where to is And quite of see high, are allows use regards a efficacy. And because steroid last use. those going data light sparsentan question, the the most as think aspect of reluctance the with we and action. from is And well KDIGO was on with I the that's of as more community we XX% think to physicians reluctance I think XX% the patients. I is physicians steroids prescriber mentioning the the to I in shown without referring this that mode Also higher patients part, guidelines physicians. treat effectively And where

to value you we your to progressed for expensive price a building are become want patients that disease towards and to new because the proposition the point, in amongst into standard potential broad we project and The have that are particular, care. most have consideration earlier this dialysis So think we of those kidney take to has society. the payers access patients patients

we patients, have I we think the upon. a progressing we can have especially so And that rapid strong of for build burden disease

Ed Arce

expense thinking have also when presumably PDUFA to may And activities you and Great, you pending you I then But that. wanted me, structure be – and through get for approval. that now leaving for your readiness take your November through of expectations there, the date. the thank rest what the a year are not on commercial excuse ongoing only

Eric Dube

question, Great Yes. Ed.

our let Jula’s to for a first, say launch think our work and that what plan And one Peter lot doing ready in approval. of for I day success is launch readiness. and driving the be me is to in of overall are that's team that largely approach

wait in until recognize our continued We of other you mentioned, our of increase be there the SG&A throughout you the that one. that So from a may of launch not for investing day just expansion that. in be, a infrastructure, companies lot year, de-risk will Laura commercial also in as from but different where kind

anything for looks add? later planning continue to that this that and like you provide year. updates Laura, preparing we'll and what want on else success how We and to are for Peter we're think launch

Peter Heerma

I well, it Eric. covered think you No,

organization building in and I existing shown upon is think patients. year-over-year, that has are identifying treating that new are that we successful an they

aspects. IgA the approach think point and focused I educate really make mechanistic burden FSGS. are the understand in angiotensin making market new antagonism dual One the disease sure to that look that payers the with and comfortable sure endothelin of are nephropathy in we like, I that expense think like? you're three how that educating making does particular, and we they on that of to physicians is

of sparsentan to we those research, own nice and also our well both as And treat. third value awareness is the intention see parameters, received. research. as is the And proposition well the increasing one external our So also progress,

Ed Arce

Thanks the questions. for Fantastic. taking

Eric Dube

Thank you, Ed.

Operator

That to I'll does conclude turn or conference the for any remarks. back the you closing over question-and-answer session. now additional

Eric Dube

all Justin. rest updated And We evening. speaking joining soon. of quarter thank you today. great concludes update. a to throughout and the first again This you, Thank our look Have your you for forward year you us keeping with

Operator

day. today's you conclude does that an for We do Have And conference. your excellent you. Thank participation. thank

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