CBAY - Earnings call transcript Q3 2020 | Cymabay Therapeutics Inc

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Operator

Good day, ladies and gentlemen, and welcome to CymaBay's third-quarter 2020 financial results and business update conference call. [Operator instructions] 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.

Now I'd like to turn the call over to Mr. Dan Menold, vice president of finance at CymaBay. Mr. proceed. please Menold,

Dan Menold

update. and had operator, we everyone. hope and I announcing Thank chance good to you've business results financial a third-quarter release review that our you, XXXX afternoon, the issued press

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

Joining regulatory are Shah, for officer. recent provide update officer; call Sujal chief officer; and progress today chief on on program Chuck will seladelpar. McWherter, scientific the the and Klara me executive on Sujal Dr. Dickinson, development plans chief an

I our Before brief financials. of provide summary a

our open Following up call for prepared the remarks, we will Q&A.

to Before as approvals we of to begin, everyone Litigation and defined to data anticipated events statements or and made I'd including Securities Reform prospects, Act session, business like during call, this time runway relating line the plans, performance, Q&A are date forward-looking remind conference under release including regulatory statements plans, XXXX. cash Private future expected the CymaBay's that clinical

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

Sujal Shah

and thank afternoon, us. Good for you joining

keep We focus from a prepared on the remarks two highlights recent most with quarter. will key today's brief

progress patients program primary reinitiate late-stage biliary able development the ensuring cholangitis, with are responsibly our significant emphasis effectively and on cash operating for on continued second, plan. we efforts seladelpar or of to our core made PBC; with First, manage our to deliver an to our

agency's mid-July thorough of of program. quarter by lifting experts. quick seladelpar to XXXX with the liver on of the third After start review The the got a acceptance world-renowned FDA a and of clinical hold independent off panel a review the safety

full least later, of clinical XX XX, week phosphatase versus our response alkaline composite a on in Just XX% seladelpar on approval placebo-controlled or significant XX% measured positive at the baseline, when zero patients regulatory numerical on patients response measured NRS. revealed at evaluated PBC a provided XX endpoints benefit in secondary trial. were for pruritus a on reducing of to for severe and in an scale, patients XX XX versus weeks treatment who the seladelpar effect endpoint, treatment normalization by with a symptom on the rating with accepted milligrams our XX.X% XX key on placebo for primary pruritus, ENHANCE prior endpoint PBC. the Almost at and the moderate milligrams on meaningful top-line and nearly statistically demonstrated weeks XXX X placebo. patients to study XX-week key primary ENHANCE patients seladelpar we results announced XX a patients the two The completing Despite data that Phase significant period, of completed who results at halted having week from

study. to appeared be seladelpar In addition, and in this safe tolerated well

since PBC for four We over in seladelpar subset years a clinical two patients trials treated believe a with or studying XXXX, been at seladelpar in has of XXX seladelpar profile these is have with anti-pyritic burden. effect having XX biochemical activities and program from anti-inflammatory our which seladelpar longer. an dosed anti-cholestatic, completed We overall with safety pattern improvements This provide potential to and dose the date. good reduce progression to and suggests optimal severity disease to patients and having of of markers of in milligrams symptom emerged and

patients translate goal improved quality ultimate benefits outcomes these that many of life into is and with Our for PBC.

below efficacy to to once-daily inadequate defined a level to responder at allowed that dose placebo-controlled randomized alkaline randomization intolerance global properly in safety to Phase so of have it endpoints overpowered XX limit size an a design as still milligrams is halted endpoint, possible. restarting A to primary study who and RESPONSE, times seladelpar Phase development a is baseline the confidence is composite study. the of let or reinforce with results enrolling our early, phosphatase Its select key outcome normal next priority acid invaluable patients patients its response or least gave of and and X term in registration PBC. its an upper named ursodeoxycholic XX seladelpar They XX-week us they as near the study and patient RESPONSE from on was ENHANCE us evaluating of optimal as intended in who of optimize after our is oral measure is XXX is decrease Although to placebo. the rate The seladelpar X:X are X.XX total The quickly weeks. enroll it bilirubin. because has with a the the the study PBC. level XX% results normal, while confirm responder achieves us X

numerical successfully of same change rating the diary or for pruritus. Pruritus baseline electronic baseline assessed moderate ENHANCE. phosphatase outcomes Additional NRS the of of patients XX and alkaline used months compare a scale from are will of four efficacy rate normalization be with in key pruritus the to to six greater at daily and we weeks will in as using severe

two The same optimal study the secondary primary evaluate same the same XX-milligram of the will ENHANCE. in as targeted demonstrating including key markers size RESPONSE safety of in population, patient and overall on our efficacy the and dose evaluated study timely supporting seladelpar disease pruritus, the of reducing completing most with with Importantly, the endpoints fashion. along biochemical a enrolling on important goal balances and of and objectives,

As registration. enrollment. want the and quarter the dialogue we unknown caution toward as three with the key had compound also information our patients will the that to ENHANCE, the year is activities XX-week start-up also introduce own will XX-week continue patients provide a be ENHANCE. have and sponsor volunteer specific encourage We'll biopsies The in will have the Based I for by we We or we in will progress RESPONSE. progress a option RESPONSE guidance unrelated baseline and done will RESPONSE, on we these continue is commence pandemic develop impact of of years in and in we planned pleased COVID believe than biopsy incorporation to but The the additional RESPONSE next we assessment to progress. make that been at team offer PBC for support refined Phase will biopsy in as for internal safety difference safety of as issues. our am risks paired of to updates instead PBC may biopsy experience in study. made their FDA, liver posture of study the broader provide study of new start-up has part X required to to first further to in

long-term seladelpar In in addition Like RESPONSE, to study to RESPONSE, enroll who studies activities QX an have of including we collect participated to with will study have study X safety patients study. in intended study, the first expected ENHANCE, PBC ASSURE, begin open-label enrolling for seladelpar in this prior is data patients additional The support safety long-term our also patients and Phase initiated of study to start-up PBC, in open-label registration. XXXX. our prior

seladelpar, RESPONSE patients to PBC ASSURE. potentially with have opportunity future will the As also in enroll and they complete studies other

in these global ensure For studies, consideration each pandemic also procedures to COVID-XX. current the involving necessary of incorporate of to patient plan safety we

In at patients an their in of development Liver from around data this into and We enrolling for physicians. and raising RESPONSE. late-breaking drive oral for meeting formed Professor positive interest basis providing American from Toronto valuable Association the liver seladelpar by week, from awareness among the ENHANCE program addition have Gideon investigators from of sponsored in by in and November PBC the reinitiation excitement believe results we input announced the XX. ENHANCE featured on of potential Study Diseases Earlier has will considerations University design RESPONSE, delivered data patients to to the be the Hirschfield from the the the for presentation the ENHANCE

data patients of nonalcoholic again once presence X include significant a meeting, our from having to will poster Dr. this forward with or featuring which a by also looking of study are We seladelpar given Stephen in Harrison. at NASH, important Phase steatohepatitis, distinction,

we for As believe to while NASH. with to complementary a to for anti-fibrotic approach us metabolic others improving aspects will these or in meaningful data to a support discussed, collaborate mechanisms the potential that treating exploring continue previously of effects may seladelpar the NASH. combination offer opportunities platform we have anti-inflammatory presentation and with offer patients This partner disease have poster

in to in diabetes. factor Earlier assets and glucose is In levels completing agonists potential we of While Type preclinical assess a diabetes. to prevent models. pharmacology seladelpar our plans low in can evaluate in remains achieving seladelpar during GPRXXX stage prevent enhance development secretion Insulin-induced conduct were early with study in with to The evaluate Xa significant in Type recent of opportunities. shown able for of on the to indications hypoglycemia will glucose patients study proof response for MBX-XXXX, we diabetics is the secretion X today, control glucagon announced to enhance cause agonist insulin-induced clinical MBX-XXXX desired PBC, development whether and other morbidity. subjects significant GPRXXX and Phase studies, were to hypoglycemia the to and animal in a our glucagon hypoglycemia limiting focus continue other hypoglycemia our X

led of to most to individuals insulin-induced study Institute insulin be will diabetes. rights Translational complications Orlando, by treat fully in full retains opportunity Helmsley its CymaBay risk to hypoglycemia, the Trust. and for contribute funded and potential therapy appreciate While potentially AdventHealth Florida MBX-XXXX the to effort Research evaluate the at be and the We by will the this to life-threatening Charitable one of challenging for MBX-XXXX,

behind provide Dan for key of a financial quarter. significant of overall for brief highlights seladelpar In addition cost our to the is On and yet another efforts development note, complete I'll ask restart highlight from that our management quarter. Dan? momentum to the of to PBC, successful our key quarter a of summary the the third

Dan Menold

Sujal. Thank you,

cash in XXXX. XX, overall clearance plan, for XXXX. other expenditures our During short-term seladelpar while investments to our to our development totaled development of our is third manage which cash obtained cash We program and to complete to operating were necessary seladelpar fund quarters. expenditures clinical the with development cash of second-quarter from September third-quarter to significantly reinitiation million cash XXXX, our of down and PBC of previous the we Overall, efforts continue PBC. our studies commenced $XXX.X believe investigation, of the the in $X.X including our at work and to expense NDA-enabling led FDA's RESPONSE sufficient study we quarter full restart million line $X.X and plan management Overall, current in late-stage our cash, and seladelpar completed expenditures program successfully NASH the studies million, equivalents of into ASSURE

per ended share, termination three in the million to XXXX, nine share three September our a compared primarily September XX, was $XX.X a loss XXXX periods of expenses, nine September in XXXX, net in the per clinical decrease results. September operating in now or $XX.X Net of loss months to ended efforts expenses XX, share, $XX.X $X.XX and as placed FDA's ended Net hold to million Net million the the cost diluted that $XX.X for three compared XXXX. the million brief or per our the in for the XX, clinical per $X.XX of XXXX. due labor-related and XXXX. XXXX, Turning loss of review $X.XX $X.XX the nine trial including diluted were fourth diluted corresponding to was response reduction on seladelpar of a share compared months or months months and was the the net to early studies diluted program to of loss XX, in in the a of operating seladelpar our months quarter loss undertaken ended lower result or

Given clinical of subsequent our of we expected are the clinical and development the FDA's losses exploration cash expenditures restarted increase our and to the in programs seladelpar lifting and other development the restart our and opportunities, of program as hold clinical advance future activities. further

to like to which a remotely possible. coronavirus on on developments operating we pandemic, the for business provide brief continue not impact to financial to of I'd our Finally, employees, to ongoing has business our update ability impact current continue all with as environment. you operations conduct have global the condition allowed as activity significant To a these date, or plan. seamlessly had execute our our Due

patients safety actions guided monitor seladelpar employees, studies. development including to as be risks and commitment in where mitigate and PBC, in risks all associated our of to our pandemic the by to our to a closely our will their possible. to development Further, continue ensuring available well will of health our and enrolled will clinical business, clinical these continue as Sujal? we take We restarted continue to actions

Sujal Shah

Thank now you, questions. take Dan. Operator? to We're happy

Operator

[Operator the your from question. question first Rahimi proceed Instructions] Please line Piper Our with with Sandler. comes Yasmeen of

Yasmeen Rahimi

on on questions. quick two team, congratulations ASSURANCE. I the have amazing RESPONSE Hi, and accomplishment

helpful. at question, Sujal, quick And who of patients on and If if the percentage clarify RESPONSE? just were has the to can a thank be again would my to part the FDA clarification. one could The that that at on second biopsy months? have they previously do should first for that are eligible is ASSURANCE? a be XX for you requested then, get end comment on patients of very you questions. baseline to And they If you ENHANCE, us, taking of just that go Or

Sujal Shah

question patients chief as be biopsy ENHANCE, is and response your RESPONSE going Yes, the you seladelpar-naive discuss the maintaining second we of the With then, ensuring well. answer to know, important in the is respect sure. regulatory, Yasmeen. out as to questions, that Phase so our patients study that fundamentally, registration have And and of Thanks here, coming X to a start study for and I'll of have Klara first. I off integrity implementation can our Dickinson RESPONSE.

RESPONSE decision in the to could placebo the have of to to enroll Of from option. into theory, study. for ENHANCE a actually well fundamentally their behalf patients allow patients course, we physicians, on We patients as allow as made those

going is to patients. So new fundamentally, RESPONSE be

position of globally. of your mentioned in a able now in comment, to PBC the into chronic and an sites, biopsy least safety and at some remarks, moved I enrolling have of inflammatory PSC was overall XXX-plus As we XX-plus this study. a data reviewing of has experience diseases, the PBC confident countries, interest agency we'll in in question in just certainly high as a a across know, level, assessment. having you we prepared very part address to of in an as tremendous make really NASH number so over to amount studies remain just from in PBC ENHANCE first a patients I'll be get And new

agency that not overall and of given do to you incredibly about we study is aware and require how a feel part we talk you experience? is in studies. all patients biopsy clinical this development challenging our our talk a that quite be confident. and practice, this Klara, in medical it about little to think ensure fundamentally about the that to we bit would But we really bit harmful to biopsy can I ability get little study get think how want for

Klara Dickinson

you, Yes, Sujal. thank

to a we're that on pretty the approximately had that at biopsy, ENHANCE in in the we of to XX% addressable to study. to look it get of number can allow adequate course And submission patients of having for the so for in RESPONSE an confident and randomized about consent to patients requirement go study, patients did caring a study, that had the meet over So the histology needs to the asking changes in consent we biopsy. also FDA

Yasmeen Rahimi

Thank into answering right question. back jump you the so queue. I'll much for my

Sujal Shah

Yasmeen. you, Thank

Operator

Our with line the proceed of Raymond your Seedhouse next with Please question. question from Steve comes James.

Steve Seedhouse

follow just on the to I afternoon, biopsy. up want good thank Hey, you.

is to FDA, biopsies from So the you just standpoint use example? not there for from this that glean a a able might I requirement, XX-week has an been be that for could label claim, guess, given efficacy you think you that anything for

Chuck McWherter

great Right. That's a question.

it's think I we're little into territory a unchartered bit here.

So, I optimistic. think one can be always

I stretch. things controlled, to the you us that And see but for think wasn't the It kind want think, of disease the be those feels progression that kind an small that went are period be of be of If certainly histology. of biliary It inflammation we a set. not scored that it there would later think for about collect likely what's would the signal. time, on an a with of that and like nature over small in you on population, disease would of it's peribiliary slow three fibrosis, an a efficacy to and given to view of we're opportunity lot times, do that you some enrolling a response would likelihood I And that of could looking period that upside. on responses. from a UC of would at longer the to that a study get reveal but in a years. out expectation know see and helps just number. years any a be always there's they histology more possibility then, patients set, about with I'll Bowlus sub did bit after think Davis three And some a biopsy they I and there, Chris had XX point published may Intercept It's

Steve Seedhouse

per on think, like from support is former there any or was more mentioned on Yes, reverse didn't to intended, that requirement if of getting if but requirement or helpful. a I'm registration, it that study just patients data That's sound back I an just patient thanks. wondering FDA you inquiry like that's se. ASSURE, it's means And drug? then,

Klara Dickinson

definite Well, to database the a longer safety I not on for call it's is robust mandate of you there a drug probably of the requirement provides as for collection a NDA. period have of I think overall that a most safety think But know, a time.

patients months that beyond exposures, more for ideal can especially the have the XX we be have FDA. of would treatment, longer So,

treatment today XX to NDA than them to FDA So in Intercept believe, commitment still treatment. our longer And have allow to be to also durations and patients that addition I they that's with treatment probably beyond to to months had think collect, longer the exposure of on out three just to they allow back come of small would also patients, for because filing to, I of Ocaliva, three years. subset were data think,

So we the will stronger the be. data the application, have can more in the filing

Sujal Shah

are And Steve, protocol, although with I'll just we, had in still a remind you new as course, long-term development of we study. that ENHANCE, when an ongoing ASSURE

open-label elect return that allow ENHANCE long-term future that over we enroll study patients study. and of As in you patients study to as XXX is will after RESPONSE. X know, about studies, for XX had those that the effectively as XXX in weeks This of Phase including well our out patients,

Steve Seedhouse

for Yes, me. Last thanks Sujal. one

have is Just or had think diabetes rights the for program, Type you X a an for this to had while while. I a guys asset

stage? overall CymaBay for So it sort this what of wanted of starting that your to program just on strategic I the reflect in just might and thinking understand reboot timing new

Chuck McWherter

glucose-dependent homegrown targeting X in and What's is target that's we came effort interesting a out expressed were Type about pancreas it's diabetes. Steve. the a medicinal beta the of chemistry very that that time, you you case, thank compound Yes, at hormones Yes, and glucose-dependent cells a insulin stimulates regulatory of other one and glucagon for and really in manner, it in a in control the as levels. manner that know, counter secretion which, alpha glucose regulatory, in cells. And

identified So that science out came and that emergent is actually Merck that of Yale that.

of new was In cell aspect us. alpha fact, the to something glucagon

experience. really that biomarkers, X glucose, that was meeting was represented, came opportunity that Type in to I think, an had some study studies That but us had it five a Phase And previous Xa to a considerable been leverage been showed commercial some on effects the so effects a where it well on diabetes as clinical compound really not profile. in significant way

being had exciting represented sitting pipeline, it so in So been really really to and not and our us this opportunity. advanced, significant a really

an toxicology CMC science. As bit the our disease little autoimmune you It's was diabetes as And in nonclinical a terms program, as like PSC. know, well Type like of PBC, wheelhouse X of terms is in in program. advanced the also program the

having intriguing to really retain able effects to capital-efficient, can moderate committed based meaning collaborate glucagon Helmsley glucose us to show then a no to continuous and an in very also an severe very upon this raises rights monitoring the and experienced, have imagine with this study, we'll the that hypoglycemic we data. If who history area set So, create possibility we'll very Type capital at in to glucose for on look looking the in TRI, data that secretion, we're one in a a opportunity diabetics manage have episodes. possibility blood and of using X

that we have example, interactions. beyond a opportunity You the placebo-controlled that. commercial pretty But about. severity those right where There's us, for efforts the of events. regulatory far compare that obvious use were it then, really of of run the whole for landscape, you and new of excited the is in a of the And And the an can you understanding a pretty host down number company to There's road unmet was blossoming that have a study, to, need. technology kind would something now. all

Sujal Shah

this moving forward, say from CymaBay. things capital perspective, a I'll from happen without these ongoing, program and Steve, don't Discussions any to overnight. advance timing ultimately And are they materialize again, just into

continue a us core the focus excited if out-license important group data, And we're of this we to keep we in provides PBC. without this question think support monetize an some on I patients. ultimately, to or seladelpar see And effort and our develop so program program either to as for just in absolutely indication some with to meaningful optionality,

Steve Seedhouse

Thanks much for so questions. the good. Very

Sujal Shah

you. Thank

Operator

the comes question next Our proceed of question. Alethia your with Fitzgerald. Young from with line Cantor Please

Unidentified Analyst

taking for for thanks question. Alethia. This my Hi, is on Emily

you've kind also, about and additional spoken could combinations on thought Type you make this combinations comment could what sense? study information what you will before Thank the of of in comment maybe next for NASH. be Have you any might testing of maybe about then more week? you. If seladelpar kind from ENHANCE shared And on

Chuck McWherter

Yes, study. the Thank so to first Yes, I'll combination Emily. glad take questions, NASH those you. start with

to some XX% XX% NASH the NASH, subjects we Just in fibrosis have on the of are to dose group. AASLD improvement we one of the that of versus the placebo and data greater XX reiterate at the histological XX-week placebo X% going study, the to or in compared XX% saw milligram the be looking group, and a saw the presenting in We at improvement, poster. stage resolved

breakout So as for for mechanisms. signal improvement opportunity that the to a profile. looking anti-fibrotic NASH, an complementary for quite us, in combine a really intriguing though, suggested, a and, therapy That as with well NASH

in be we're row to to for at or in with years and a come fat us. composite able insulin reduction and gratified metabolic to contribute Other general we hepatic your resistance resistance, that that to mind now second weight be previously of improvement even valuable. ones be think strict to mind, agonist, that with this NASH minimal our effects. exciting very with things question the hepatic loss about AASLD, do. back the on AASLD, would under to recently might whether now all Then as the with a very data either really effects which need presentation, metabolic pruritus as late-breaker combining fibrosis. shown well in that could time normalization, on resolution very course, NASH in sponsors mechanisms, the in So, with effects to anti-fibrotic One GLP-X strong effect a looking receptor either we mentioned we've moving Sujal rates effect mechanisms at shared, have insulin as response wonder that and like embargo respect come We're the or several

not more very have much to So, data. to you opportunity just present that, really to say to than but we're able the that assure excited the

We as I information to I at really subsets of terms to of the to to think a lot is publication and patients. from to as various study. think very and find commitment parameters not that all it's for that of we of only produce that stakeholders. subsets data a garner look we'll quickly set going responded are the patients going our have interest. be to continue way of certainly important a think hope can work informative to And but course, high-quality really in share We that's ENHANCE the

CymaBay as soon may It it as all think eager investigators who in I data. get course, encourage and are enrollment. we will ability are to certainly interested can impact the out, it have that those they an of very be see to

Unidentified Analyst

you. Thank helpful. that's Great,

Operator

proceed Please the question. next of with your Olson Jay Oppenheimer. question Our line from with comes

Jay Olson

question, Hey, and the the progress. on taking for congrats all thanks

Thank they of then, separately, with their to convened way strategic from -- And hypoglycemia about on that diabetic that the histology you a X panel to a plan You Can data CymaBay? a diabetics. had those where you GPRXXX you've question into just that development that got Type through your you you fits of the something talk vision that of Or Can Is commercialization? all future take you any fit Or experts treat seladelpar future PBC? into how would partner? how anecdotal comment that molecule use review to CymaBay? agonist to is does experts that about you. to patients feedback and into would your for seladelpar. maybe plan strategic for prevent on the

Sujal Shah

first. questions, question here the for Jay. I'll the Thanks again second address

in for is as premature think ways far fact, Again, study liver partner might pure think, development. could, particularly the today. future we excited goal monetize itself. this be you MBX-XXXX. the pipeline us continue know, have overall I gives to to keep in us the these to excited mentioned, do potentially it about efforts One well. is optionality program contribution I focused groups and and asset. it's we're what right chronic is to time really It at molecule the to But too which as the really which inflammatory as from Helmsley our as our of this one to us our with in speculate think create exists to either to out-license on we've overall Foundation with and And fund our value broaden allow opportunity to diseases

And advance even potential the looks to the CymaBay so other what has beyond really like continue in to programs. seladelpar, future

to on with And potential the so continue seladelpar the we in value look build to forward, heels PBC. success in opportunity fact, of to

might think premature CymaBay any I into a capital from proceeds At bit at require study or time. any to So, around we make of point today, this this in doesn't advancing least as of do. what decision fundamentally use it's

gives some so at I field. then, time. gain again, we've it think set the data asked a initially capital the with point without And of own the ability you burning our to And us, that in experts around this a question in done work

in drug-induced it in And outcome liver at you experienced evidence, pathologists of both agreed NASH you make as seladelpar a the is was And holds, miss histological we any that findings together something, clinical me areas it conclusion, can in no know there, with came had pulled injury. number really by accepted. that here. The I into experts or world-renowned that and of process, hepatologists, caused lifted it's seladelpar we liver a clinic have if out one as well That's so of And or injury NASH. original advance well correct the with, particularly of of which of liver well is, frankly, as sort included the in continue NASH study. the FDA that study the we I'll the comments. involved panel injury, this investigate couple in be of course, the particularly to also expert dialogue in the of that stage and biochemical a as drug-induced clinical, field PBC to that all back fact, And in

of up offer the this successful, this confidence was that, in of even And view given in through studies, to I'd seladelpar that in safety beyond. the review, how so experts discussed use we are change in work would and profile the really in even fact, no greater there's area. seladelpar also date, if clinical we have we've done,

been folks which And do tolerated we area have so, fact drug disease, and really excites we that's in is biochemical markers meaningful on what of really as well and a PBC date safe the of believe impacts I one to some for well that seladelpar think the as of the and and strength out to through believe, a data if ultimately, that coming NDA us particularly on an the of continue data in puts set, symptom set, advance to launch. ENHANCE, of RESPONSE, to position filing successful, burden. again, that And we totality there's

in opportunity be And alternative seladelpar, remain, these choice of treatment many so as we significantly PBC, may not patient perhaps, we to second-line only very of experts population the I profile actually encouraged for the in a of expand be by setting able do, the have think, to PBC. behind a but addressable which

Jay Olson

the taking questions. for you thank helpful, Super

Sujal Shah

Thank you.

Operator

line the proceed Riley. from of with Mayank comes Please next question. Mamtani with Our question your B.

Mayank Mamtani

taking biopsy and come my the question. have congrats on to progress. recent Thanks all I back the for on question,

XX% in the said of treatment feasible had biopsy was to Was that, there duration? treatment kind short the a not obviously, given ENHANCE. that a you So, that biopsy? Or follow-up

Sujal Shah

for biopsy subsequent histological really not see benefits or an been with from have had into study Yes, you wouldn't that two that to originally, around for similar have changes. biopsy in whether volunteered follow-up a perspective, those occurred baseline long-term that around three treatment. very years And to idea Intercept's question, good been a envisioned ENHANCE, could actually to Again, Mayank. to second into the assessment until is any would what trying a have glean being POISE the extension, the exploratory

paired on during volunteer the to X PBC XX baseline would agency's given to supplement forward, view weeks going the studies that safety Phase Here, get would look biopsy for at that patients picture any submission. be of the an those now overall really we to a NDA

anything which given that that the ENHANCE experience, ensued not least a just agency, wasn't PIs requirement least was from was us investigation follow-up case, described, a investigators that PBC. were setting seen biopsy. expected be own observed or decided in at So, from prior the what for And year, to end in really to post subset or two it our patients post at have it three, pathologist, as there be of the from there but however, and about decided seen of a those or dozen wouldn't last their themselves, in well as the in that the

that So a set be sample of really least but small do know those had at we're of certainly volunteered. there significance, from comforted by we that element. to We wouldn't this expect anything

Mayank Mamtani

the getting and final Got a break also sites and about you're a the looking-forward I for pandemic similar of you have it. but today, question, but and little that? lines how Again, RESPONSE giving the also review a a comments we against up for study couple of have in elements the on like understand about also think guidepost. getting should IRB guidance enrollment as time then, running And we a RESPONSE? bit. think not ENHANCE Any you

Sujal Shah

Yes, no, you. thank

question. good really Another

had in enrolled ENHANCE just with XX randomized months. for we about So, reference, XXX patients

with in Of competition dealing which course, as today. pandemic, PBC additional were the time we that we patients weren't are nor was with dealing for we a

smaller a is what will no be perhaps to ourselves at key study a precise you've targeted with COVID-XX question heard The course, RESPONSE, a for for challenges what is And in there's but very to these So, respect with set particularly of as about two least the the predict time was year's enroll what a to pandemic impact enrollment, study, on will and challenging we've although be today, target smaller frame. impossible us than it's those ENHANCE. study of factors.

first target projection of in study look and as a the end forward to looking a year. in enrollment of randomization to as next initiating would we're the by next quarter this complete So, year

Mayank Mamtani

second Great. comprehensive are broader, you more time would just the on question. population, a PBC part last update PBC, all have soon? How or And if a advanced you any of second question, more first-line that maybe thinking about at

Sujal Shah

my off, another start add to members and I'll I'll team miss. good so ask here Yes, question. anything may that I

are, many we have or have there internally, and very broader We of number a years ourselves well we to continue may, ideas of And the use. different as constructive around that in lot with in idea in worked thoughts additional here PBC of with the for a fact, experts that fact, support now. for supportive allow as studies dialogue

efficacious, of And well-tolerated alkaline even historical treatment limit have as at patient so normal, of mention, approval that in second-line be thing RESPONSE the the plan. But of a example, a like a where qualify and a elevated that the operating is And or Phase to for seladelpar a it may running position study an in course, some as study are the to of alternative, will can with study the registration about treatment some have we and second-line focus, study and say -- these open this not for believe necessarily really talked Mayank, These these one we to that we've registration patient phosphatase, then, our we us Subpart patient a Today, is the upper And are do of up for to our calls. X.X additional get times X potential this RESPONSE But by degree we final a or Historically, number of remains would approval. those still long-term idea on studies potential approved. X.X alongside ensure get the and has a you capital conducted potential can progression. as a with work. nevertheless, safe and core I higher it be treatment. opportunities that the risk successful thoughts. with believe of continue that gets to study as supported approval execute H

That's we've to in dialogue the in Type study. agreement be would it the agency have of patients. continued that to study we So more of with also and you advanced course, gain advance, have would on a which experience an outcome

sets And of continue advances. so sets those data in believe some will generate are, fact, program to data as that we the

Mayank Mamtani

questions, to Got Thanks my for I Sujal. it. And forward look taking updates. future

Operator

the with comes from with of question your next Capital. Patrick LifeSci question. Our Please line proceed Dolezal

Patrick Dolezal

us XX curious the if arm mg X was Hi, rationale ENHANCE? the risk? thanks question. for with or mg you safety potential used thinking dose how pruritus with to my Or just the incorporating moving strategy mg about the avert you're taking versus was it? exclusively titration for provide to used forward XX some that Can titration initially Obviously, in

Chuck McWherter

that we point, Intercept is the Thanks you use as that know, for exploring Chuck. We that have that didn't worsening. experience question. fact, arm were with the of have drug, That our we of never is the this to Patrick, was, Yes, the in strategy. having dose Hi, level utility titration actually, now. different because designed because risk associated the really options at avoid was pruritus wealth

we've results. just As taken open-label you years. past the XX-week two well extension where study we've know, with we've that, completed now top-line And an patients released beyond the then,

things we've speaking you and at month of been As know, of a couple ENHANCE from emerged. today three,

superior pruritus. correspondence GGT ALP, response, and mgs on XX the weeks, ALT, biochemical just durable to pattern responses biochemical and the at well all, as where but XX XX between rapid of a was First importantly, as consistent weeks; X the mgs. Not

XX. It decrease a against tolerated to interest. units but come this idea data actually that's baseline then, moderate study, go significant of five. those six of itches, in ENHANCE to know, on is the having also you differentiator. statistically whereas in the a consideration the So, as had X.XX NRS of at script of physicians over not a important we just back the last the is potentially patients, severe stat get It's of so just sense didn't dose think we the X Many a then, start commercial endpoints all really shines And sig convenience never I Ocaliva dose an mg. the the and makes across that have dose. well is And effective for very clearly that tolerated. has know single to to patients on we to it and adjust well from

have, that that's story. disappointment to the deliver our as efficacy often doesn't colleagues, it the and So, like dose commercial safe the a have job you medication as as really lower where long hear set notion it forget it from part you to can and all you'd does long it's and the as avoids of this real-world

not the do that lower more not be reason But population, we're X for a part XX data compelling of to actually available We the have X the a a there's have the of that's dose mg lot might core most picture. patients, of the prescribed is where at on of the say in I and drug. really mg, advanced think So, really to mg, for market, least the think core patients. I positioning

Patrick Dolezal

the perhaps which imagine kind sense. Makes is going that Obviously, Or on be X.XX of alkaline going the levels. is phosphatase one more of bit? to bar do this you kind current toward treatment. And normalization, moving like, bar is just to years? imagine you of continue you're sounds field initiation then, Sujal, Can multiple forward it alluding so the a in to for

Chuck McWherter

an message opportunity that develop that highest you with is first reason from patients more to that. these that to How experts a think at medical Yes. least label normalization? to differentiating appropriate do this hear data. as see, And the we along regulators swell is this risk I patient of actually But think I of all, There's then, approval, then, and that's more Why that's the can the as at to normalize. from something wouldn't regulatory medical that that think build point. with that to a completely clinical you're biochemical probably consistently agree data also that KOLs, give a and ability every gets a better, I epidemiology. linear lower gives is that's unclear alignment feature, opinion that the this factors. into relationship on in experts. achievable, begin from translated hurdle supporting And to you opportunity want there's there's And the message getting for drug log something really is to going

agree. that's So, to remains but I that would do trend, be seen, think I a

Patrick Dolezal

you. thank Great,

Chuck McWherter

you. Thank

Operator

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

Unidentified Analyst

Hi, Thomas a you've questions good the clinic. in the This Congratulations placed on Yip asking everyone. back of afternoon, Ed. couple is for progress

also in for the major you versus primary ENHANCE mg Can size over and XX focus the the RESPONSE than dose? question distinctions patient PBC. study some First other new go smaller on the

Sujal Shah

RESPONSE Yes, than really for ENHANCE happy than that's Thomas, you similar the thank question. more There's is with to. different. much

ENHANCE really highlighted biopsy in we here at patients you and Of collect the The used of well. a registration endpoints. X:X the opposed same are as at had trying assessment, with of X:X:X biopsy to in bilirubin, on secondary in patient entry. here really really looking XX-milligram Outside key course, is the of safety as that and two randomization we're we dose. a paired focusing biomarkers as subset data, for isolating to used been phosphatase, ENHANCE, as well disease endpoint criteria the that the population, at same same looking we entry We're really that's for exact on alkaline same primary validated as data

see optimal study. to to on, to expect you the So, patient in same I'd derisk want same is that highlight would what RESPONSE focus in case, frankly, endpoint, this that dose, everything we population in with same really fact,

patients. our across up XXX we study the and seen well same hopefully we've X and in Phase feel as data with of as very that to in execute even see study So, the get ENHANCE, ability and kind the running over confident consistent

Unidentified Analyst

do you Perhaps a IP would tell how maintain? your for indication, structure? NASH if lot us input of the or still then, partnership to the makes is if can for Yes, look that be much sense. you And what so, plan partnership? to And scientific want

Sujal Shah

Yes.

that, than improvements also to to very specifically, seeing rights LDL, disease, patients and I and would think party it indications. where be not of think Type Chuck the date. seen looking target a triglycerides. what split we seladelpar construct studies bit from talked targets that PPAR an an have with the we a that and talked there's different for one with interesting and be some see an sponsors believe we complementary even think The of about to would is in that's question. opportunity we're another the to with we already about could, a good we delta anti-fibrotic promising across very combine be anti-inflammatory do of fundamentally, effect, fact seladelpar along those We've have response an other which significant to other certainly with we combined opportunity little program assets. in And we've fact, for like a And drive elements in greater metabolic very

fatty we much I'd or sets with argument And start meaningful own. the that more are reduction responses yearning drive seen field Of data modification total course, in to not see you NASH. times responses four really targets of an placebo acid five three, that although other to to somewhat liver is drive entire is the we driving that combine level the see fat therein oxidation. opportunity lifestyle and know toward I kinds toward is the on to make or lies on with their seen think

to with forward absolutely, or in own whether progresses. not dialogue So will NASH we this be how dependent our respect process, on move

think that the on for nice AASLD us continue reengage back platform and broadly PBC. medical really to be us I discussions. if to in to again, gives these these community will, It's able of operating you then with opportunity, data dialogue getting the a in internal platform, kinds in the clinic to a while focus maintaining, our share

some the to before And think to complementary to perspective, sort to relinquishing work from right we brings program. has which do development resources of rights obviously, here the asset, have third-party about optimal us again, any an the but one so bear, it's think in I allows overall

Unidentified Analyst

Any about be that endpoint new expect on sharing Any with be? last one talk. your details And likely? it last details for the you should primary Phase the How you to think large XXXX study? Thank we on perhaps partnership. Xa will question

Chuck McWherter

a of Yes. is pharmacology proof study. This

the study, control. basically, service can internal own So patient each crossover where design is a their

once in studies we'll the where results hand. share would clamp So, do opportunity release side, think partnership we'll to I so of long so in additional TRI the will diabetics, details. either small look with opposite they a would kind to to we and and in is receive over that collaborator, It's cross don't forward a details have those have to we very in or something our it it's think the study. these relatively phase I take placebo they experienced active then enroll.

Unidentified Analyst

questions. for And to Sounds taking a you good. my again very busy we XXXX. Thank forward look

Chuck McWherter

Thank you.

Operator

Our with question. next question line Please with Smith comes Leerink. SVB from the proceed your Thomas of

Thomas Smith

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

pair the requirement. I patients to to of better design, is so, trial RESPONSE to possible a guess, couple it What the in I trying RESPONSE are clarify, agreeing biopsy? on you mentioned proportion paired just of you the biopsy for entry then, if patients biopsy. into XX% without patients the And ENHANCE to assuming in volunteered for to Just consent understand guess, will RESPONSE? this gain

Sujal Shah

They're good Yes. Thomas. questions,

So, not a to have it paired first patients agree mandatory baseline of or in to to for biopsy is all, RESPONSE.

we're XX-week minimal paired to wouldn't if that to biopsy, in a frankly, as why possible, it's partially get try our some experience threshold, course, absolutely encourage will. able commitment been is Our of the to as voluntary It's to patients of to based you many on without XX%. I We, regulators when argue agree. there's ENHANCE fully setting prior

course, So, biopsy having disease to sure and of their we even and a believe have as of that, and to set no practice, discussions by safety allow additional able this meet patients patients not to this the to it's in medical end appropriate of make to as get value, data encouraged the can we'll we with agency of assessment. our to be to day, although are of There's the subset stage many additional we in some question of at the want there that is understand the progression. But part study. encourage review them

to we the sufficient for study. set patients looking more our has of allows successful be to so the an agency again. evaluate robust an be And for assessment again, think it even simply that file our involved data we to part should ensure to at time want And in the a NDA do would RESPONSE

Thomas Smith

Okay, really helpful got question Sujal, just color. other it. And maybe PBC. then, that's on one Thanks

design you study, actively have also trivison any about how their just thinking maybe sense RESPONSE? compare their trials.gov. competitor who's overlap trial PBC of You that their in then, clinical the a might and Phase X into for you're sites amount posted and how versus And impact Can they've out enrolling enrollment? there

Sujal Shah

Yes, I'm happy to. no

I RESPONSE mirrors in did think what what I'd fundamentally we ENHANCE. first say is

we've parameters design and X:X Certainly out having randomization, designed really detail, ongoing making had done really the in of I'd terms study reflects, to sort that study are even it's dose think as design. before clinic placebo. comparison RESPONSE their is we've we've agency experience in we the any So, they've really simply dialogue in with we've what what of single the key on already talked the GENFIT say which also about put a in trials, versus ENHANCE. had, observed

those So for did in as same two same well the in in Both and elafibranor, fact, endpoints in again, common. what populations, patient things RESPONSE ENHANCE are mirror shared that we the both secondary had well. seladelpar, as as with really key ENHANCE. study primary we Same

Thomas Smith

it. Okay Sujal, insights. appreciate the great, got I Thanks

Sujal Shah

me. Yes, sorry. Chuck, just reminded

that would overlap, some we NASH. bit would of studies about centers be seeing sites, common asked sites. specific are patients with don't conducted there that We You expect GENFIT's we at in a little overlap PBC, also our very common had as know that

XX-plus was fact plus think I XXX anchored across ENHANCE in that countries are the sites. we around

experience more in more significant than And with of in the PBC, I experience at in Phase X. global and think so, the been our least PBC setting elafibranor has

of us. I a in of think PBC, seladelpar our with from experience significant have lot number I key perspective, with that's and think So, of the for setting we sites really

Thomas Smith

much, Okay the great, taking you Sujal. Appreciate thanks questions. very

Sujal Shah

thank Yes, you.

Operator

the question. from line Derek proceed Archila comes Please question next Stifel. Our from with of your

Derek Archila

questions, for progress. guys, the Thanks on and the congrats Great. taking

us. two Just from

Ocaliva you the have trials? with part Has anything of to decent size on partnering. Phase provide if is safety did on as it a could works previous deal? while? How issues PBC quite the First, FDA upfront X at some the been important just been question fund that's And the just of the secure to help in the to on this biopsy in it follow Thanks. to require Or for on something thoughts NASH part program do in wondering move a PBC the PBC? a to then, up

Sujal Shah

the question, Thank Derek. for Yes. you

days that quite to as we've in thought worked something still and even PBC in not was that NASH for of for with with has understanding So, work that, first the space in development, from thought how the Even of with a they've this patients PSC. the that about in the our thought this or acid cholestatic leaders time. a of from few a fact, agency long done is biopsy, from therapies globally, is disease respect was obeticholic dissimilar development we've consideration, fact again all,

I think what's unique part it's medical PBC of to is really not that practice. here

novel to it potential to clinical alternatives patient a from in any the have with seen for the that before hindering always we're for recent study designation, and think, treatment overall PBC. underscoring they've something more be certainly for even I acid every new this perspective patients challenging agency's So, And PBC. PBC of with well obeticholic challenges, of therapy But gratified in breakthrough is really would I see here to even recognize challenges support fundamentally, course, think considered safety time, that quite agencies any implement some again, for development. quite without the perhaps

you wouldn't of to able that them observing they argue tell going to data biopsy safety at may what doesn't to assessment. put necessarily in a additional level forward see provide of of I some be be or Now position least wanting some now some

they're a in of as patient see very but think again, safety. that overall they logical recognize wanting to, that's ensure agency, a development regulatory that I position

then, diseases. course, chronic isn't can don't threshold. influenced to think, you even say, open say NASH, I explore always I the forgetting not recent specific sort of and some there they've any that other thought Irrespective, is So, the it be or would, advancement, the sort program the haven't whether the something these details I'd about I that we'll this advance simply long dialogue. we of of of important, them. very any in that I arrangement if to similar can't any set I of understanding, don't, but is near-term continue some would something opportunity tell sorry. in inflammatory seladelpar seladelpar NASH, in are the to again a liver events our to point-in-time the we a can establish here, not I'm you progress end Again At without Derek since continue second strategy just of quite PBC, to tell if I perhaps liver rare meaningful our day, that question, question. And But Oh, for think orphan significant it's for fundamental necessarily there's that we cholestatic very significant. at in PSC and opportunity disease to think be

Derek Archila

Got the progress. thanks Congrats it, okay on guys. again

Sujal Shah

Thank you, Derek.

Operator

Sandler. Rahimi with question. Piper from proceed Our follow-up is next from question Yasmeen the a of line your Please question

Yasmeen Rahimi

team, sorry and Hey for question. the additional last

So, just us what in thank now exists sort you enrollment. of the time just the investigators, could sites. of many expect began line you more us already on you process, you for to you as for turned terms of have But go of when providing data two have that versus sentiment years maybe help if you the ago color you

helpful, you the just like with on are to found the of believe summarize tricks study? time or with even from you or key that that this help sort enrollment ENHANCE would three of additional going me part is data maybe sentiment that being So, just be sort to physicians simply just of four lines, new

Chuck McWherter

add if start have Yasmeen. I'll you, Klara a miss I some she might anything. does, comments take then maybe and Thank if to

alluded least kind to or several you considerations. important at think I of So touched on

all, is help. sites in think outreach. now the We all dosed really of also And of very study. both data the the They're not I just strong XXX setting active been important. value rare CRO. having and can placebo-controlled on the We with help that really having years, our and finally, But by in enroll the team, KOLs, data PBC, the we First with having disease beyond hands-on weeks XX kind non-cirrhotics, by are concordance we and label over a global very of who data having with in at two very investigators, patients having believe world. really experience We've compensated to our even CRO, with talk received, a cirrhotics data enthusiastically open-label weeks very clinical folks with style. investigators a data, effective. relationship and close at the and bread-and-butter our is It's open partnership XX pruritus work data only over in but showing sets

it be just to So really the makes easier conversations have. talk much those so They're to data, calls it coming about to to next. arrange able discuss just what's easy

before, failures. The avoid to sites are those who how second things we screen patients that tactical thing to train I'd find to so of this really That's been how point and the going to we help know X that. internal recruitment having That's to to about ENHANCE we're and been qualify, through is were there's share kind leverage develop. as able to to we knowledge second and into move and through we've able able Phase improve, point. through the even And program, that

to bring a activated, story. slowed so other more performed asking maybe we to. those? every well. kind know knowing the well. we maybe we we of quickly how us the they sites where had didn't kind is are have But countries move staff many the lot down have well, regulatory had we It not didn't and line we point those back did sites know we which and a with who can that then, third a can distracted the time patients, bit sometimes of before just to support that the for success to to through? know back where in What it's like? sites to And were How of What's now avoid reasons. go so on think to, We were not of go I issues they questions maybe and was it they a get back. that know relationship like for We enrolled of that little variety front regulators reasons,

my follow you meaning. If

all invaluable. the experiences are kinds those of think are So, just I that

But but only kind I that some I it going forward. position really, to them think of of us Some help us significant we challenges Sujal can intangible, acknowledged respect and against with they competitive. are are think, COVID,

Yasmeen Rahimi

Thank the you, for detail. Chuck. very granular That was excellent absolutely

Chuck McWherter

you, Yasmeen. Thank

Operator

like Shah further closing hand Mr. to no the questions. to call are back for There remarks. I'd

Sujal Shah

significant a to provide our forward weeks we opportunities the we'll to as significant other seladelpar, today. I'll our programs you again And to getting make in presence looking move a once say AASLD. all Well, the at development stride, experience clinical as amount on patients once look in momentum. thank that having continuing positive are seladelpar programs, our joining back of we months hit hands for well you we just again we're updates closer in us of and to we as to forward as And progress as ahead.

like that As our work thank possible. we'd all those make you. always, to finally Thank

Operator

today’s Ladies you Thank for and gentlemen, does teleconference. your participation. this conclude

You and may day. have lines your disconnect this wonderful time at a

Cymabay Therapeutics (CBAY) 7 Nov 202020 Q3 Earnings call transcript