Cymabay Therapeutics (CBAY) 28 Feb 192018 Q4 Earnings call transcript

Company Profile

Participants

Dan Menold	Vice President-Finance
Sujal Shah	Chief Executive Officer
Pol Boudes	Chief Medical Officer
Chuck McWherter	Chief Scientific Officer
Yasmeen Rahimi	Roth Capital
Fang-Ke Huang	SunTrust Robinson Humphrey
Tyler Van Buren	Piper Jaffray
Michael Giaquinto	SVB Leerink
Jay Olson	Oppenheimer & Company
Ellie Merle	Cantor Fitzgerald
Steven Seedhouse	Raymond James
Ed Arce	H.C. Wainwright
Mayank Mamtani	B. Riley FBR
Patrick Dolezal	LifeSci

Call transcript

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Operator

Good day, ladies and gentlemen, and welcome to CymaBay’s Fourth Quarter and Year-End 2018 Financial Results Conference Call. At this time, all participants are in listen-only mode.

Following the formal remarks, we will open the call for your questions. Please be advised that the call will be recorded at the Company’s request. It is also being webcast live on the Investors section at the CymaBay website at www.cymabay.com.

to call like to would I turn over the Mr. Now at Vice President Menold, CymaBay. of Dan Finance Mr. Menold, you may proceed.

Dan Menold

to release operator, Hopefully XXXX will chance by you, financial now the Thank and update. you have good results and had our issued announcing we afternoon, business everyone. review year-end press a

our that Investors under tab. You can website, access release on the

provide and milestones me an and Officer. Pol Boudes, Dr. on Executive Chief Officer; Scientific financial our the Joining on open Q&A. review Chief Medical update call the Dr. Officer; position Shah, They programs McWherter, today for call will before are Sujal clinical up Chuck Chief and we upcoming

expected like call, the prospects, events future Before I’d to XXXX. we plans, conference everyone or plans, during including including that begin, forward-looking under session statements defined Act clinical statements Q&A as Reform of this CymaBay’s performance, made the are Private business remind to Litigation Securities relating

forth that based expressly forecasted factors CymaBay. actual to the and such to from forth this the could as believes the to CymaBay’s in any anticipated risks CymaBay, materially the uncertainties reflected assumptions, Company in the SEC impact is written risk results time, to I’d consent subject call and over those recording from of cause that conference reasonable future of as At This statements. set as and the the forward-looking outcomes differ many in or factors call differ upon result results is could rebroadcast without set annual the and Although statements today’s are or prohibited expectations supplement to the whether materially Sujal. are release turn to like The filed for of press Company in update quarterly by of those the to due forward-looking except reports with statement, well new and Participants property assumes statements obligation any forward-looking as otherwise, are directed factors. no events information, required law. or a applicable cautionary actual

Sujal Shah

It’s already everyone, at Good afternoon, and to start busy CymaBay. thank year us a the been joining us. for you for

focus During key the as disease. accomplishment upcoming to needs in liver meaningfully establish addressing we to increase most call the significant value today, future and for on the a patients have importantly CymaBay our milestones will and potential that leader on unmet with

As development I with achievements first ever thus for X registration the announced of data for potential in with radar months The CymaBay two CymaBay ended widely into its far this late is investors, significant of or year that inflammatory profile study two we the sometimes given X derisk Earlier of liver disease. additional made opportunity and time accomplishments. few us spend transform at treatment to presentation made AASLD’s the improved of XXXX the the speaking month, with patients XXXX with Meeting. and first advances primary appreciated. under have will major the one XXXX breaking and or consecutive Liver an We Phase hear cholangitis We seladelpar positioned the we underappreciated of Phase PBC. in third for a sentiment biliary in alternative seladelpar I and patients we more be initiated to promise where that our

proof-of-concept patients in schedule. we treating Phase completed Xb FDA enrollment study we seladelpar Therapy of with for nonalcoholic or steatohepatitis one of quarter Designation a the and patients Breakthrough First, PBC; with ahead announced seladelpar NASH, granted of second,

have already registration enrollment seladelpar PBC our also global ahead, X increase of we recent we expect study year key of Phase completing Phase interim and medical our in we in milestone and in for we to to shared the look events data X study pace XXXX, major end catalysts. completing ongoing By the As the for anticipate at PBC which meetings. ENHANCE,

in reach top the quarter. evaluation expect of opportunities of addition, data regarding Phase our from to to NASH seladelpar seladelpar. complete in decisions Xb study development In ongoing line other And to and finally, expand second the we announce we intend

our with start development us program in Let PBC.

is UDCA, As fourth in first PBC I line of seladelpar an global to inadequate Phase establish who treatment mentioned, study intolerant the X of efficacy safety This to, and XXXX. in ENHANCE our initiated we response have who study for patients, the in that current registration designed are PBC to the PBC. of seladelpar quarter is

with to seladelpar. support is of authorities submission to some goal to Pol? ask dossier registration the study elements health of obtain this of Our ENHANCE. Pol review I’ll global approval with the of key a

Pol Boudes

possibility ENHANCE Thank approximately being level at months. rate with endpoint dose or to The at the six XXX in from composite as with a than X.XXx after PBC achieves XX phosphatase clinical enrollment a patient randomized XX-week a milligram which as responder of receive than countries. less an you, least are of randomized seladelpar XX% decrease sites at upper more primary level the at XX defined to also normal is of of responder alkaline study a in target the weeks. XX with who Sujal. daily is Patients normal total milligram baseline a patients a bilirubin and over limits is doses or XX increase XXX placebo X placebo-controlled milligram of to either

PBC. in of enrolling treatment and progress a this the regimen, represented of are ENHANCE the to derisk in of on Phase the X using differentiated by America time X the ENHANCE. and enrollment first measures same to in results for good the making helped same outcome in by Latin that Europe, across differentiated potential what Results very North key Liver was complete Phase are the as same the this strength regions; of that large compelling a we past treatment working at a November. using end for global – from duration XX Meeting Asia-Pacific, year. and profile America, are in ENHANCE Phase dose establishing study ongoing treatments seladelpar We We of patients in four to the initiated be the for have efficacy and the believe which X profile program in this weeks X and Phase includes believe we the tolerability

that anti-inflammatory positive busiest We weeks and meeting at this quality pruritus can All found treated results PBC in well with clinical effect, the last adversely may benefits potential our our ongoing with has and press or in seladelpar PBC of affect improved quality of The sustained importantly with we seladelpar also patients Very to On on most second inflammation and ENHANCE. it serve two additional the one companying fact XX provided in cirrhosis study unmet to patients a today, will of patients an Each needs of pruritus. on Phase with the believed overall these believe suggest baseline. compliance up their a foster be treatment effect seladelpar in forum pruritus to and subset life. confirm that efficacy number for that described X symptom on look common recap, in to and potential critical the in we the details PBC. seladelpar patients be and progression The To release Improved cholestasis we efficacy today, medical to external data from key website. was our evaluation AP while namely linked without of Liver in XX potentially PBC, as potential the tolerability better risk November based with is better the approved reduction of have and worsening of a the line itching, the in improving of patients tolerability. only and disease anti-cholestatic Sujal? in Meeting potential reduce greater of presentations improved all, patients from weeks. as surrogate for to patients. of in on at as better as life to contrast

Sujal Shah

of the I turn X PBC. examines this to Thank that clinical to and seladelpar for existing over The therapy for decision alternative of the a believe has as this substantially patients accomplishment agency with with designation to stage granted designation seladelpar medicine major expected the regulatory be for benefits PBC corresponding breakthrough for has in by been the PDC. therapy we prime greater breakthrough designation. this FDA early The Together rolling seladelpar opportunity designation importance In authorities facilitate the the priority review. program evidence for want well grant agency. efficiency, for potential or Seladelpar also determining and reflects for to organizational treatments programs improvement earlier The review are the an awarded We potential these substantial therapy. development eligibility you, seladelpar fast PBC. of medicines over path for senior by improved FDA of of approval as European include data the the treatment Phase managers month. announced existing FDA track on clinical existing that the Paul. to commitment designation enhance with dialogue involving priority provide guidance and and those the

over program. NASH the our to seladelpar an Xb study. to We of pleased on Chuck very study one were call update development Phase schedule. to completion turn our of to quarter provide I’ll in approximately Moving announce of enrollment ahead this

Chuck McWherter

greater placebo. a controlled biopsy, diagnosis receive were milligrams of X:X content randomized than randomized Thanks, confirmed comprehensive a subjects a milligrams, study. to proton dose either XX% fibrosis density have MRI-PDFF a into required milligrams patients fraction XX in have with fat X:X Sujal. using XXX this with method. ratio fat to once liver baseline NASH or been placebo daily, imaging, resonance Subjects magnetic total XX seladelpar double-blind biopsy A the to baseline of ranging Prior XX were or or to

or randomization of baseline X and NAFLD In activity Type with absence had histology evaluation addition, of were confirmed a liver this stratified the presence subjects mean score of liver stage subject the fibrosis of by well-established diabetes. The NASH X.X. at

relative subjects measures using of fibrosis improvement Important samples. baseline. fibrosis patients enrolled MRI-PDFF primary had XXX the is at in weeks. histological are paired or evaluation pathology outcome and at XX% Additionally, the at liver the efficacy biopsy of liver secondary XX NASH X/X reduction in by fat weeks The Stage XX approximately

an screening the Additional evaluation algorithms. elastography assessments include MRI-PDFF with using the multiple has XX machine X resonance pathology this proportion be and at than Phase liver would FX learning and NASH. Phase non-cirrhotic patients higher technologies, enrolled imaging seen weeks, of X planned are fibrosis markers, reflective study subjects and digital believe innovative population magnetic weeks in biochemical a in study. approach led been We noninvasive recent evaluations other The FX targeted in studies for to similar that registration and XX of

FX potential anti-fibrotic the chances distinguishing seladelpar greater NASH. in study this of patients of believe also the FX effects and fibrosis with with We a patients a proportion improves in

a diabetes, progression. with We of who also XX% risk population have a patients, significant for number of increased disease at NASH enrolled patients approximately are

concerns Going mechanism and Phase has pathways study, CB seladelpar LDL pathogenesis and that candidate included lower wanted believe correct Furthermore, and in obese like into ability development. and and evaluating for with benefit actions for decisions its lipids and dose to it promising triglyceride had a complete a Phase enrolling points regarding to with appropriate therapy. increasing to cholesterol, histological that NASH, the for cholesterol bile CB of progression. ranging potential therapy that We with Sujal? a in a about aspects outcome Suggesting involved confirm hypercholesterolemic this risks other raised we X a anti-inflammatory the the of foundational hepatocytes, NASH needed population, selection end profile. a could dose other for action. and disease coupled We including key lowers This Seladelpar commitment actions enable to X be constitute the potentially or seladelpar that of avoiding anti-fibrotic subjects. been measures lipotoxic believe seladelpar’s potential acids, to all

Sujal Shah

Thanks, Chuck.

make NASH, with to diseases inflammatory and forward work determine front high Seladelpar future course thought current rationale expand need opportunities to liver this other with includes clinical evaluating for progress our over development PBC in other unmet discussions leaders, important of chronic with assessment We the for the look sharing us valuation the in Beyond focus of we This this updates continue on to opportunities. year. for scientific to criteria unmet the need. of of development, and

on our XXXX. December expected Turning cash, fund our to equivalents, operating at existing plan Based the and is our current to XXXX. securities projection, totaled into million cash briefly financials, XX, cash $XXX.X current marketable

Operator? XX-month XX-K up the to refer overview For I XX, question. I’d open to results of the December you a detailed our like XXXX. to for SEC the call operating press filed and the today. release period to ending with that, With will our

Operator

you. Thank

be now a question-and-answer you. will session. conducting Thank We [Operator Instructions]

from proceed with Rahimi question. Our Roth first line Please Yasmeen Capital. your the question comes of with

Yasmeen Rahimi

you question, Question patients. my in in you taking are And PBC attention us our maybe are one EASL study, for caught on XX give where presenting on of what update you’ll progress. the hepatic Phase at in the conducting continued study an mentioned you regard? at the thanks then weeks. is, past X and what congrats a team, Hi preview impaired was maybe the Can that that was And gatekeeper? survival be transplant also had data

So can little you. Thank you on regard? us give color bit a that

Sujal Shah

first Yes, question. to turn and the it Chuck then answer sure. over the I’ll Yasmeen, I’ll second. to for and one thanks address the Maybe Pol

PBC with ultimately respect think I to our study…

Chuck McWherter

Yes. This is Chuck.

an with developed Bettina being overall presented risk EASL, and this terms that’s score. led is Hirschfield, collaboration from GLOBE Gideon really what at yes, they’ve a Hansen So, what��s in of

done really reduction characteristics based then happens in biochemical was take baseline ended upon what in what patients their the analysis And the GLOBE and of the risk so to population treatment, enrolled evaluate was score who using parameters.

the I reconfirmed we that improved gratifyingly This score. already sophisticated and the of transplant-free – underscored use you know – methodology risk by linked what expect, using would al-phos was most survival. see the reduction to think more you what was is GLOBE

Sujal Shah

risk And what we population, in a the is to in overall question, better advance is a better benefit and to recognized exposure, the a understanding there have then your patient have of understanding of that swap profile that PBC need particularly broader also with first population. patients and in disease Yasmeen, answer

prior able was particular, in only is we though continue you that there the impaired development, study particularly in not the included was exposure in that even acid, actually have through their obeticholic overall understanding in that population. benefit Phase PBC again, a is not to As the know, population fact with on patients sponsor, population being measure X hepatic label. design agency and a conversations, that desire, And profile to what for with right thinking and but risk their

to the we continue So regulatory that dialogue have that’s a with agency.

Yasmeen Rahimi

question. Thank you, my taking team, for

Sujal Shah

Thank you.

Operator

The next SunTrust question. proceed your the line is Nash Humphrey. Edward from Robinson Please with of with question

Fang-Ke Huang

expectations second for your this is about additional Huang Edward And Thank on year? question. in you question Thank to And you our data some given taking ask that in of we you. One This X you us what Phase you for is of one can on in talk NASH. the PBC. And can you can Fang-Ke data Nash. ongoing second quick study see internal terms present question do my give your quarter, trial? want in – that

Sujal Shah

Yes, for the question. sure. Thanks

So as X to and a series study recent medical had know, PBC start has ongoing just progressed. with the off, at study, as meetings presentations we’ve respect you Phase to that of

by study entire an significant. at or early from that a before on study, have through provide be it’s in been that and study effectively treatment our reviewed year, upcoming treatment the in Phase patients been year fulsome fact, somewhere the patients very of year, come have in XX-week coming patients year, in and in with in patients would this we now the this as rolled publication our robust XXX is long-term think to able way data over update to roll would will of dataset that we to be period A so this is of long-term in end that of Sometime a ultimately And middle And just the year all the randomized, study, medical which past, dataset fulsome the through eligible peer least the into the meetings. goal the little continue next Phase dataset a that this this study will the it’s for the XX This we in To to encouraged. very expect this of X ongoing either middle a all an year. share that to bit be assure the in XX-week entire period. ENHANCE, through the north with be X population practice extension, that’s extension.

a now efficacy this weeks. of collect got number which beyond data XX and to we significant study, safety we continue for patients So in

of about we and by it And little of to context the NASH. continue thoughts the ongoing whereas So a perhaps, to encouraged Pol, that some turn respect to to talk extremely Chuck be I’ll study. bit with

Chuck McWherter

Obviously. Yes.

So I guess recent for Phase from X us results the benchmark similarly is position dataset.

data, thing’s fat where really the issue is about one So relative extent the NGM a data to certain reduction. medical key

and NASH reduction, improvement relative XX%, absolute of the That’s fibrosis linkage look achieve example, histological was fat. the characteristic for finding reduction NASH the So and result has reduction, proportion with meta-placebo key increasingly well. And in in we is fat that patients importantly, in improvement overall. fat who associated XX% about XX% at been been key as that the a the

So clear would be down in reduction to XX% profile competitive hepatic relative for XX% us, sign we a fat. reduction somewhere think maybe in maybe between of a

Fang-Ke Huang

helpful. Thank much. you Really so

Sujal Shah

you. Thank

Operator

line of with question Tyler from proceed next question. Our with Jaffray. the Van Buren Please is Piper your

Tyler Van Buren

Hey, on good congratulations afternoon, the and designation. breakthrough guys,

follow-up but over bit. to And with In need respect to see clinical that comparison mentioned get substantial therapies a is maybe have and wanted in evidences, results to presented order that And and on measures pruritis. involved release alkaline existing little end of you phosphatase the on any the analysis. other the forth it, one point. OCA on in least, at mentioned, Just improvement call is been you back that to particular, and release, imagine you in there in that

there get it to to designation. breakthrough curious thoughts your relates the Just as

Sujal Shah

Great absolutely. question. Yes,

something in that with secondary upside continue think and a Certainly, setting acid. that’s designation breakthrough And patients that’s not the submitted reductions. only and with quite FDA, of look an validated them that designation of we’ve will that designation when package can ladder the you is device, outcome. our a particular, to where tremendous seladelpar in by that patients is PBC. EMA to dataset potential important improvement is potential that available far, the relative agency around there placebo endpoint in that obeticholic will the we in review have to study ENHANCE ENHANCE and on specifically data think looking AP, respect seladelpar And an of robustness an key for around proportion PRIME the a tolerability other relative that X be we additional overall to the treatments that with by the – when able seladelpar today. daily experiencing opportunity key the to agencies compared information by thus the in the and certainly, itch to profile for now, pruritus study around believe we’ll But AP the far in of validate we for reductions believe but I we the that accomplishment with substantial at normalization to agency those as differentiated the have applied improvement provide I is for The So with supported the are review. of at what with and I that’s that we a to recognition in PRO. in differentiator And delivered think, a ENHANCE, seeing Phase to-date thus handheld see treatment with

Tyler Van Buren

Obviously, related what of with you the people we to of endpoints to a I results any things there. be reduction fairly second But that respect would about respect how analyzing the or lot we fat, about lipids with the effects, terms reductions those other and of numerical think of just of various X impressive think to statistically on would And should meaningful Great. be and that antifibrotic result acids there’s aware are quarter. to types in bile of mentioned result Phase the on think potential see? guys lipotoxic focus spoke will coming MRI-PDFF and very next been those anti-inflammatory and and in And that effects, measures? how the is specifically, terms significant and in previously, sort you question about NASH data

Chuck McWherter

Tyler. Thanks, This Chuck. is

we some additional it going that’s so of I think really thing liver already But fat, is as at what study be the another think literature impressive And sharing will thus line, the collected. some feature, quite one and I transaminase richness We’ve in the transaminases endpoints top data of have the anticipate really of correlation this features will to important and it speaking that’s or improvement. and data, be. the between is that supports histological be discussed

was another but measure the quality just the the NASH baseline enrolled score, think not the we of of transaminases. population I

XX. So mean was ALT a baseline had XX, AST of

potential another would point So I examine what treatment the interest of that be. think to be will effect

In web collect at practice, take you addition, if analyzed. time biomarkers will those. look us clean, through a when as we to It weeks. you will, information been have XX a principal understood, other number mentioned, will And we appropriately releasing it’s some a of or of

patch of rational work ability we inflammation – all, biomarkers would take fibrosis, data the to But being as more some time think and just a try fulsome of present I part around fat, – but lipids, of bile mechanism and those medical a anticipate could be linked action package markers, really on the conference. of at in through analyzed, wet its features at And fibrosis. some those all hepatocytes to in that our their effects acid some will would be connect together that of the look hypothesis will seek all transaminase, to and foundational you seladelpar of liver of terms about some to

Tyler Van Buren

of the causing that mechanism. as in that is with with – to debate think particular you’ve you that just proportion drugs the higher follow reduction the specifically And and it? a would seen in What in some also for follow seladelpar suggest there fat, there up you’re had other anything if Because it’s is We’ve transaminase to ALTs? case, just in respect datasets to seeing extent ALTs, seen reducing are preclinically to I ALTs. of increased had see a respect ALTs related fat. some some that up increase I a elevation with the that’s whether in an you to but won’t is do then

Chuck McWherter

Well, XX highly XXX weeks. much. These I any a studied in be we’ve little see patients with mixed subjects XXX We milligram really patients fatty signal milligram LDL are study me. acid our dyslipidemia, baseline, cholesterol elevated and fatty in excuse with transaminases. eight Tyler, triglyceride, in in and over liver – where presumptive for they’ll disease, didn’t to can’t you help

early I that the and think I there not anything stat be the would point ones not just mechanisms speculating, not many datas – for a there’s that If nothing that have first-generation is I’m linked, compounds PHR for to is I’d I that lower but aware that lipids there NASH, label signal. in that can development mechanism did be. certainly in there’s necessarily really

Pol Boudes

Yes, it’s Pol.

decrease signal the the transaminase think for and we inflammation And the us, very liver. in in increasing see I we that I really we transaminase PBC in think have Because impressive PBC. So data interesting. are a see

is which see think a where a I to transaminase. linked population expecting we also see the population So in because NASH you that is as the are inflammation

Chuck, what you know I was think about now and think to probably it’s related – I talking TRβ the listening I agonist. I are to

synthesis bile in where of the But reason an side. generation especially type agonist. that of that know saw increase compound in previous is other transaminase. and we they see the the effect an general, effective I transaminase. I acid you be increasing the is in would there the for fact, anti-inflammatory decrease you in is triglycerides, former would that One in are one what of TRβ one thing could In with is actually say, And thing, that say

Chuck McWherter

Yes.

Tyler Van Buren

taking Thanks questions. it. the for Got

Operator

next Please question is with from the your of SVB Leerink. Pasha question. Sarraf The line with proceed

Michael Giaquinto

Yes. Thanks for Hi, question. taking my Pasha. this for is Michael on

have of you you away, of of So which couple approved PBC in in how discuss disease PBC between can in and scenario for a dynamics your we pricing and pricing thinking seladelpar years strategy but should NASH? about we terms NASH, be both potentially in obviously, rare still a

Sujal Shah

sense still in at important seladelpar able expect the we certainly, planning us, view ENHANCE so be for completed. make over hard put XXXX, specific only Yes, it a the thanks have scenario that potential are And of which course different some consideration. to for to that It’s with developments between we also into as for Mike. believe PBC do now number a will question, share have early that to NASH. And together it’s answer. the we me factors be point be But somewhat of you the some and we and to not of in input our but we’ll of development anticipating for will

and to validating various strategies and are dynamic far. So things look expand the hopefully we well responder that and of vis-à-vis addressable input to of differentiate about profile reductions, learn pricing to be from the also maturation a We’ll profile, thus to continue normalization respect effects key all, significantly pruritus. thinking confirm overall outbox with perspective These believe respect we’ll tolerability the overall the we to seladelpar PBC. will the and rate efficacy continue the first on from ENHANCE validate even composite And rate as as at positioning, seladelpar in what found key ENHANCE and population. seladelpar to a with the

think population. consideration I expand treatment, as potentially there’s PBC. significantly that addressable we the put treatment another That’s think need a in pricing dynamics. that an about is more and in And we opportunity and tolerated a line with to you’re for second will better for significant there – that today more think better seeing treatment, treatment – efficacious the overall

reflective continuous current the the over treatment trajectory the continues of – couple environment, bit of progress also years. how of is and all that to course the are learned how a today, course, be overall will we progress earlier the Of that about of meaning that and competitive to

on your patient and of question substantially the important think NASH alternatives As retention how there connecting with recognize about well tolerated key ability it are population. a PBC. a respect the as keys finally, of it’s as the unmet things overall, think Fundamentally, And I in this will NASH. treatment underscore respect improved in safety, of terms of the grow respect are, And that well will it. overall Not get key to, input with need just benefits treatment patient develop current the first treatment more for goal in and high in development to at PBC, the another but to alternatives. for alternative, significant as base patients its we pharmacoeconomic efficacious that better that a us, will be how to need tolerability, differentiated a think successful how ultimately be, to We’ll population agents I profile, really root only other to be think, the the there we population. with our a to is treatment in our that

NASH we’re around about X NASH, Phase for still questions development also population. the the there’s in between and progress we As profile post and positioning to drive us the NASH if Seladelpar what in believe doses course in Seladelpar what will another right key input into the In thinking PBC. the successful differentiations in forward carry end, That’s we is some X of NASH. data in we NASH the would of with Phase

that address if in resolution effects on that’s gives fibrosis. what a niche going and it’s the hepatic importantly pricing think reduction, broader see I And based of pharmacoeconomic it’s could more some if fat of on population, then on reflective sense be of the the that in broader population population. only but to effects benefit to the So, we course not a of come where of NASH course me

to lot assets. fundamentally Okay. support to get also differential. treatments in these the inputs development, I’ve we’ll through of true think able patient clinical we these goal all take into our but be not to is only of you when pricing end account about given different a And full things

Michael Giaquinto

much. very Thanks helpful. Very

Operator

Next your Jay Company. Oppenheimer question line is from question. the Please from state & Olson

Jay Olson

questions. on my for Hey guys and the congrats thanks all taking progress

be you can for efficacy, that And the line much the to How touched how NASH upon the Phase guess around can earlier, this you that You line? tolerability from results expectations to provide regards but in X you specific, Study, your and top addition outlined safety study? with I in sort information us. of top from

Sujal Shah

Yeah, I progress’s study remaining this patients point if to important weeks course, XX blinded. out of it’s to think that

we blinds we is and patient from the that as at that this you findings. for we’ve least think can me group important for Similarly it’s keep to seen what from now a we medical have development This confident. safety see is to that and I which very monitoring the the study executing to important, all basis as tell we continue at on data be perspective, as to continue a be tell mean, so respect be daily is blind is to you enrolled, done we a will support clinical the the for top is report for So, in today to measures study study planned. as population. also respect typical I think the line be a the without jeopardizing for us perspective What share line fundamental top transparent, I with able

Jay Olson

great. broadening a made with to Thank wanted development regards line what PBC? the you. additional earlier just you on Seladelpar with first population in potentially And you’re you follow-up and I some on to comments of consider doing studies, for PBC. Would thought study to Okay, then in pursue leaders

Sujal Shah

and think target in Fundamentally in fact largely that well. for the add I Pol as adequate second some is tolerated perhaps are color maybe patients patients, a UDCA, can Well, PBC generic treatment treatment, alternative of well I’ll UDCA. to here line start the number responders off

And course, to there risk of we key the patients about responders UDCA. that start at disease of the those so are think technically in bit progression, to remain that really a little for need population unmet

benefit is even further anti-inflammatory believe from AP, the normal, AP through of benefit one the preclinical them efficacious, in potential below reduction to for seladelpar, drop experience in that potential well-tolerated limit X.XX on to a be upper – of expand times measures nevertheless treatment brings that population well as alternative upper elevated of that above but limit continues think we I into alternative would treatment of it as normal. some and development the And continues from from So safe symptoms an further this remain patient disease. the that alternative

treatment opportunities the even think alternative. population the there therapy we expand are of frontline thinking So outside about to

Jay Olson

taking for question. Thanks the Great.

Sujal Shah

Thank you, Jay.

Operator

your The next Please with question. Merle from line Cantor the Fitzgerald. comes question with of proceed Ellie

Ellie Merle

much question. so Hey my taking guys, thanks for

I endpoints. PPAR but NASH First – FX? I trial delta, greater guess, might FX take on activity to clinical relative FX that FX your the there just and harder just is guess, in and NASH mechanistically versus in what’s context I whether of terms of, anything drive and FX for agents, patients specifically in NASH or guess, on be also patient? easier FX I resolution patients clinically guess, mechanistic in will to first in generally achieve

Chuck McWherter

Chuck. Thank for This Yes. is question, you the Ellie.

two that is at about or is injury, in other somewhat cut we oxidation, strong If looking really the to pushing lipid that sponsors reduction extracellular. function would acid be I of drive fat I it don’t a the at matrix that seen of necessarily degrees burning think feel the I’m have of droplets response. fatty that we which it mitochondria, stage different internal differences what depending extracellular fat breaking upon oxidizing like hepatocyte, out taken different that sure any is a and things it, is liver matrix into not I fat environment. those haven’t think saw connection. down. and simplistically basically steatosis Maybe has

It’s bad fat there reduce or inflammation tissue So fibrogenesis. actually injury just fat. continuing really – ugly causing is which the anticipate fat so to driving – the goal and but actually is not driving

stage risk feel we linked that recognize least should to adverse like strongly all F Having to event a across stage. is liver progression NASH I that, liver is assumption. complications, be neutral, for So at at least F said resolution baseline fairly

the ultimately fat so resolve outcomes. advanced think study to focus even And where is where on the there. have key the is liver disease as payers are resolution, you’re concerned, at we that potential far reduction to long-term the And more That likely there are looking of NASH focused have leaders a I and and least benefit with as thought fibrosis study interest. degree a would going to have a

so of stage, there of general, have cardiovascular risk as terms in could board And benefit them really in potential liver course terms decreasing the of PB aspect irrespective well. a of the – lipids that’s that’s across in

large in that stage. So that measure, again be think independent I would of

Ellie Merle

need Thanks. sites. running already helpful. could versus what XXX And setup. characterize, then to still you just enrollment question are the the trial very PBC sites proportion That’s guess, the for it. on ones trial think guess, up quick got you I a and Got Thanks. of Phase I mentioned to that X, I if

Chuck McWherter

over space site robust are across our And largely enrollment in four we PBC. be come all enrollment both strength of we what to XX tell and orphan initiated And Yes. the featured field enrollment medical The Sure, these progress ultimately things really the with not as we meetings are different from this in a how breakthrough continue sites. only Ellie. of sites the last can are at site We and tools, been set study well end and of the all completing mid-stage progressing. has believe therefore on thought specific initiation, PRIME. globally you rare an it’s key effort. the powerful we don’t typically to as countries comment XXX We’ve held with in respective disease support very EMA think but the respect Phase across leaders global X with data our and be in and most ultimately and to that about and have FDA to the regions. out in year with continents. things investigator across pleased of to on the of I to key you, meetings we These very as respect how continue board, over because number continue pointed challenged the

we’re patients studies, and opened very are today to progressing with once leverage into these be that sites to truly drive are how able things our to are the tools Now continue these pleased

Ellie Merle

Got it. Thanks.

Chuck McWherter

you. Thank

Operator

The Please of Seedhouse, question. proceed with next question the your Steven comes from line Raymond James.

Steven Seedhouse

in Hi, X NASH reading good one the have study. protocol question regarding afternoon. Thank biopsies I clarification for Phase you. your

that the for Our could paired just describe protocol? baseline are and that together i.e. the you scrambled the using? same and biopsies Or studies ongoing you’re all by Phase post X treatment you are paired? together, as any and use there read one previous each the patient or protocol pathologist? if other And not biopsies Maybe similar then

Pol Boudes

Maybe Pol I I can take – maybe that. can its –

is biopsy. field way criteria pathology. pathologies And details key that And to not also easy best are And for it’s would to liver for I actually technology I it remind on system. haven’t done, the recon can use we the reading biopsies you We read on say that’s one that using the of actually two radar of biopsy. we’re the are we biopsy. of are reading will the say actually well run I that, the extremely think quality the communicated suffice in known the possible scoring the of we of call. But inventor we so to actually. our think us communicated having who I NASH is, NASH

a very robust that So feel they confident have very we we technology own.

So, far everything looks very good.

Steven Seedhouse

more of NASH just patient I of are – most And overlapping mean, less these these enrolling or trials Okay. the on trials enrollment populations.

right study trial lists? the getting right to more a to that I’m playing able and scheduled, the function just NASH obviously, about which your enrolling? different of access enroll centers, ahead patients You’re in role educated in actually trial’s of curious, Or is a mechanisms choosing of

Pol Boudes

think a I important factor. of one The combination mentioning, you’re very certainly one. it’s a it’s

really when us of what I grow well. kind with been think before, has didn’t you campaigns work you the worked also to into well study want done what what recruitment and

network somebody was organized investigators. well were of very So with, investigators like and extremely Harrison working an I really think also amazing Stephen efficient as a we’re

way also the that’s prior clinical Then success. in also, And really on for the to to is think that. that also proficient channel based do this a answer you trial. And right to possible the to I study. example that’s patients thing in patient think is last is very kind I a of be are want able there So again that

Steven Seedhouse

you. Thank Great.

Operator

the from comes proceed Ed Arce with line question of next Wainwright. question. your with The Please H.C.

Ed Arce

my Hey a Sujal, add the on one. designation. That’s And Chuck, are you? breakthrough Thanks big questions. how taking Pol therapy for my congrats I’ll

I But asked. out further little just to next upcoming NASH your the been drill on read already had Phase questions couple X study bit wanted So in quarter.

me. for here there So

could PDFF? on few considerations XX% times. is First, of powering novel key that that the Second study a endpoints if just the readouts you around relative particular on to threshold discuss the mentioned like you’ve the in secondary

decided obviously, how NASH? could on three complete And just data the if could milligrams, XX go a how lot. just to I that that go review XX but the question in a over Thanks If study milligrams those more especially, these is, you’ve of the doses. and and terms XXX milligrams, this prior just may dose in you of the of final you discussed compound seen. is you given over ranging this understanding we know support before, then

Chuck McWherter

Ed, dose it’s and to Chuck. with I’m going ranging. start selection dose the the off

you work remind or you And phosphatase and we only a of is of the in acid with a which have but attractive to reductions, end a in know trip a also two depression, really very reductions cost a in had treatment wide select range doses. go really to we alpha of helped degree forward, milligrams to study. if feature the to bile reduction called the prior which we’ve our transaminase development XX effect to attention effect themselves not having from drug to folks many that able kind diseases, linked dose is settled for with very that's milligrams on the is in Just PBC, times decide to particular, in if because PDC, range up. wide to you in XX a range explored particular, it’s At where the want low the strong end shared top very wanted be

at remind And nearly XXX these in reduction comparable mixed we a XX% treated milligrams, a just lot syndrome. XX weeks milligrams. saw to XX% in that in XXX obese, XXX the that were study, of study in subjects who treatment were top at end, reductions and X we almost prior on you, subjects cholesterol. the trig, have triglyceride, a or LDL metabolic And between are high are So for had LDL dyslipidemia, done XX have the effects

a fatty type end safe lot at certainly their then the the disease log just a of to the really of some end fill an one are well tolerated, XX the not population XX -- the it could effect, that to really risk potential NASH, it the to So have see is patients share of of the that likely the kind of to quite that fact added and because milligrams that as of is drug are liver top reduction. kind see we seems the kind see body albeit, top of was These didn't features, is, it's a and like range. the milligrams increase think the out signal milligrams transaminase, XX over and time range

Pol Boudes

the think of I power question the other this about was study.

So we've study powered the on MRI-PDFF. the

in out than because actually that's As that's out. well difference power a study reminder, calculation, power a and the the the rate drop more so for XX% this expected actually fraction. outcome have potential the also taking drop account is fat And is we of the we to in very included powered change relative XX%

Sujal Shah

other it biomarkers I'm question? leave sorry, any on in measures portion your did we out Ed, Was the study? additional and of

Ed Arce

package entire decide got number of the and you you've XX-week the I ultimately how already, mentioned full details of of maybe more you fill a some you've sort that them, and quite previously. sort are of but around wanted to Phase you're the the will data, there's get of once just how that design? a X novel little secondary The endpoints how on out thinking of help

Sujal Shah

that’s great a Yes, question.

additional I’ll thoughts take it. on can an Pol with fill in first maybe So it the and half,

MRI-PDFF, have MRI we only elastography. have not we right, You’re

of have perspective we biomarkers have inflammation a mutli the edema at line, liver can which And is linked parametric commonly will to for reflective of look in which key then methodology totality of stage matters. have is to is not building help really corrective inflammation addition in knowledge essentially think we’ll is, a this liver, in In where only NASH. a In used number we’ll been you different make us histology what we’re lot that is ultimately measures But recognize the I what have base, bile process, an which that before. acids decisions.

dose somewhat theoretically and have, differences efficacy the fibrosis parameters we for say, So just different Do to you look it something seladelpar and respect effective see you they fibrosis different one may you kind on have differently has put sure want, want for that one help you mechanism would do to in fibrosis more be X, that to NASH maybe dataset may example, we and doses you broad want do dose and you you we understand. know to for mean Phase all together. focus fat. a to to a little support with inflammation that that’s for I those make marker And what you and align couldn’t make different have covered. then understand or you us may somewhat the of of in That’s resolution see or Or want decision. on want

help the or higher Phase the the patients, those degree of So acid bile have all – plan could of characteristics to versus as this the that patients that higher some really markers lower. package responses lower have X have fibrosis of built that baseline for have about data as versus subsets, well who of think those

scenarios but I’m scientist, through it’s really rich hypotheses. a highly just little, little, alternative a and the at a not as think and about excited set So, point, data this speculative but to

set kind quite totality get trade-offs committed we’re a be exactly really it but to don’t we out, of the be that decisions it’ll a make of facing. I very scientific of some how that to know turn would intrigued that might know we So, help taking I around view data the

Pol Boudes

a very should delta, the data seems it that given interesting Yes, eliminating. PPAR very specific highly Yes. such be you’re mechanistically

Ed Arce

Exactly. Thanks

Pol Boudes

you much. so Thank

Operator

FBR. from is from Please your state of question. Mamtani question Riley next B. The Mayank the line

Mayank Mamtani

taking On progress. to the very questions Hi need, first, discussed. seladelpar just mechanism PBC how curious of everyone. in out, all Thanks the points my designation, follow-up. like the markets strong I new and much that the in a has so also, – could could indications Phase new you in My about generally are for PBC the think being relative follow-ups role X And NASH talk combinations or congrats are play did in but as pointed Phase then breakthrough analog FDA. data, many on different acid aware to with the a that question already we’re unmet with the you consideration potential I also you mechanisms other maybe X a about also maybe much. lot And review then go. Thanks that in talked And you pursued have studies. are the you bile

Sujal Shah

Sure. Thanks question. for the

is to respect determination mechanism-specific the breakthrough, much with benefit. potential evident not overall for profile so to as So substantial the it and is overall a improvement in see

agency significant we’ve into the normal quite the robust only normal, times phosphatase the X.X seen drop as of experiencing was that upper Ocaliva. alkaline not differentiated Phase decreases nature concurred but of the So, I X proportion of development X.X, below patients think our fundamentally, from also and the range in limit

that the really more respect a different is the well only So, determine designation with being prime respect has as I’ll highlight it seladelpar for compound to anything was just PBC effects that breakthrough treatment with today. actual to as than mechanism.

Mayank Mamtani

Great.

Sujal Shah

thinking in could and is we on talk on focused Maybe, study believe of setting. how NASH, I’ll that little about seladelpar, is, treatment alternative I about particular, and think question well in the combinations about the – a its be of the what given we while fibrotic have nature it work. in We Chuck – particularly think disease. in turn over the this other do which was of we’re bit more that addressing some as I maybe, overall other Chuck’s your impacts as and think a profile think seladelpar, I’ll metabolic your Phase there foundational long I with understanding of potential very Xb bit elements group, to setting a I completing discussed, to been and inflammatory since combinations think the And we

Chuck McWherter

Yes.

our has against various kind So to features of where the or either scientific trying could disease focus we’ve gap. it the a understand fill or been in reinforce of enhance might our rationale, mechanism been actions –

or NASH on in recently characteristics features that focusing been an look that NASH-TAG a these, we think meeting And of as for models presented we some in coming have The biochemical metabolic, those Keystone studying pathology agents where selecting had various or alone in molecule, in really therapies additional and plasma fibrosis. In have obese lead look have been NASH you and we’ve some context, that activities will as either for presentation up mouse liver the of had can anti-fibrotic at both anti-inflammatory and example, of at well and benchmarking lead the at we and we Liver observations. at – interesting combination. presentation we at should and EASL, these have Meeting, we’ve at both I So,

For very interestingly, you looking to Pathology. then loss, and fibrosis, model agonist, if found mix. the did, we of was that reduction, work effect mouse at able mechanism at on has Pathology, NASH fill a both fat looking example, all there minimal in to we comparing a liraglutide, terms on NASH really the will, two into the together, had some add added were also it in anti-fibrogenesis the that the well has receptor components of on in seladelpar in NASH weight that component this But liraglutide effect study gap GLP-X some data human which combination, in those and effect

those So, NASH, aspect, in stage that. to some to drugs some you a that’s two. we state let’s continue marketed as anti-inflammatory think drug agent. at anti-fibrotic time, as the are have to rationale, at same combination think that either has add available combination presentations continued about other redirected the at has that the metabolic one you’ll well be where even say a look development that do begins potential We’ve with late can but as or and that’s with EASL something to That’s that to the I see combinations not potential some additive of therapy, marketed of or to compounds to be the strong

Mayank Mamtani

U.S. And is any you. international NASH the again, Thank but versus in helpful. also and you sites, Maybe the this program? missed that specifically in the super is responses sites? there that thinking the been studies NASH populations, this there’s mentioned there different the overlap any then international. have PBC part And PBC placebo had and and there That’s for I different floor, patient baseline, generally overlap great. on maybe, there see about are in Great, the NASH, of you at also

through the thinking how to just So, about think response placebo there.

Pol Boudes

So, I can part first take of the question. the

overlap be study, tend People specialized that to resigned to overlap actually. PBC NASH study and more So, study. between there into it’s lab big is not some NASH

there is that a of So little bit an overlap.

Chuck McWherter

the response placebo trying conferences least I at entire guess, course, or range sponsors responses. the the placebo I that of are and – portions question and regulators of think of podium keenly major you to showing presentation aware of conferences been issue, question a main address there’s that KOL

X. effective this leverage be enroll to case, practicality in our to U.S. thoughtful selected very, efficient, experience really, a think lot a that sites that in we study, I really sites is and syndicate is the to I to around high And then are was placebo – subjects is in high only think have like of our degree population of strategy with our to able of NASH proportion of your that and NASH, that just have I a X.X, favor of very the question our a in kind and high phase you the would response, coming mean inflammation. think

and would vagaries the think F-X and baseline is this imagine and to an a subject bit of latter a for fibrosis. than AST around, likely patients see that be between a F-X is move some whereas and earlier kind I less effect. I And little gives and easier to to leader the it I more in the advanced proportion time in not XX reassurance of stage ALT with something XX the that’s put data let’s difference and some so less the there’s easier FX It’s leader be could And FX bit variation necessarily of think forth little a particular, back to fibrosis baseline fibrosis. So, to theoretically placebo way, importantly, respectively. F-X, or think having

the of the vital speed to and that study is of us. So this I practicality of and quality think a which of size in terms

I think placebo we’ve minimize we about potential measure try can done just to every any reasonable to effect.

Mayank Mamtani

Sounds it. Appreciate great. guys. Thank you

Pol Boudes

Thank you.

Operator

with with question line LifeSci. next Please go question. The ahead your the Dolezal Patrick of from is

Patrick Dolezal

seladelpar Hi, on just one for plan these patients me. provide Can in separately? and NASH data diabetic guidance from whether you versus just speak you of to the to potential non-diabetic analyze populations

Sujal Shah

with that anti-diabetic risk And may some NASH, because such progression diabetes a also of component. see, treatment factor in and earlier The they the that’s as of have entirety this a to overall an we ultimately are is Yes, we what baseline co-morbidity known we for uncomfortable the it’s I might various that think examine population because associated subsets certainly drugs handicapping or be the course. the risk little fact top-line alluded higher at particular of I aspect, results, of at to population I a with of as speculating we’ll registered stratify of I’m look diabetes medications. importantly, NASH to a place parameters. would baseline think, respect and important did in but

a I makes of that sense. examining think that lot

be serious about for we’re really at be. differences parameters that would more another to our potential – I’m that happening or at that’s of say, the really a of X, target least a in type to up informed that of expect diabetic we us population that take one say. Patrick, take does course, the way sure would phase we look expect, something this glycemic data successful analysis there on strategy not position to what will as to certainly are effects sure. a shape terms we by data. that but least that’s So, give patients including at think and a we’d be having ends some I sensitivity study in – us the for insights to size potential parameters about any half of of collect made that always if push in we’ve you us would that Does our we the early should ultimately ultimate can

Patrick Dolezal

Thank Great. you.

Pol Boudes

Thank you, Patrick.

Operator

you. management At Thank I remarks. floor for back time, to the turn this closing will

Sujal Shah

once today. all you for joining Well, thank us again,

patients, world We that to for space. caregivers, objective company really to opportunity substantially seladelpar core the do firmly PBC, we to to bring our in the the an renowned treatment have about at opportunity that in we thought continue are have their believe an focused But improve leaders here on to passionate we with patient. work extremely patients. remain we

and the we of patients with well. programs see approaching seladelpar treatment these disease hardened set And believe see isn’t alternative both the previously designation support compelling that action, the compound of truly particularly, today. FDA the given seladelpar’s development quickly we look a believe year. plans this a seladelpar mechanism are as well we with as positioning fundamentally to providing as to as foundational of updates and potential granting prime the the expand forward NASH, a for you. that in We on to more there that being potential as the and of be seladelpar for certainly this EMA breakthrough studied excited to to data unique in coming Thank We’re

Operator

conference. today’s This concludes

time. disconnect participation. for your at Thank lines may You this your you

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