Cymabay Therapeutics (CBAY) 11 Nov 212021 Q3 Earnings call transcript

Company Profile

Participants

Paul Quinlan	General Counsel
Sujal Shah	Chief Executive Officer
Dennis Kim	Chief Medical Officer
Chuck McWherter	Chief Scientific Officer
Steven Seedhouse	Raymond James
Patrick Dolezal	LifeSci Capital
Jay Olson	Oppenheimer
Sahil Kazmi	B. Riley Securities
Thomas Yip	HC Wainwright
Emily Bodnar	Cantor Fitzgerald
Mike Kratky	SVB Leerink

Call transcript

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Operator

Good day, ladies and gentlemen, and welcome to CymaBay’s Third Quarter 2021 Financial Results and Business Update Conference Call. At this time, all participants are in a 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.

turn over to Mr. like I’d the to call Now, at Paul Counsel Quinlan, General CymaBay. Mr. please Quinlan, proceed.

Paul Quinlan

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

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

Kim, Dr. Chief Scientific Officer; me call are today McWherter, Joining Officer. Sujal Shah, Dr. Chuck Dennis Chief on the Chief Medical Executive Officer; and

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

I’d relating Before everyone commercialization business statements expected begin, including remind we like including defined that plans lines XXXX. performance, forward-looking schedules, and made time to call clinical conference of the trial under future approvals, plans, prospects, anticipated future or for during to funding this events, and statements, Q&A Securities regulatory products of session CymaBay’s the are Act dates, Litigation Reform cash Private as enrollment repayment any runway, planning and

actual The obligation the today’s differ or the 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 believes of company factors This subject in 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 forecast to events, annual and information, forth 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 could except expectations risk quarterly set reflected the reasonable assumptions, those outcomes from are such are the based directed

Sujal Shah

Paul. Good today. you, you joining us Thank for and afternoon thank

marked the of manufacturing period inflammatory toward of in autoimmune have Since CymaBay at primary liver mission with PBC. our lives patients we improving and grown commercial of planning. operating cholangitis our objectives disease, The of and development, seladelpar significant another quarter rare team the achieving restarting in strengthened or have third quarter progress clinical development, achieved many this and plan our biliary

team for the Our in we any program PBC. completing drug is most is working towards extensive believe investigational development what

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for with patients give be an a those with discuss not seladelpar experience presentations Chuck us PBC. Before and time breadth appreciation the for ongoing Meeting, the Liver the many studies that Dennis is at a seladelpar good has and our confidence potential I treatment this familiar depth our PBC, upcoming has this given think in and to of option the and leading to

lay the discussing unmet needs three as see we for main seladelpar patients the me out what program, let Before with PBC.

normalization associated potentially many with offering These a improved that opportunity should as cholestasis the include in improved can transaminase, in patients of lab poor with of responses and achieve therapy commonly in liver tests goal been observed need for against outcomes activity evidenced of PBC. liver argued PBC. the and the phosphatase, the and histological response It progression ideal that especially tests to incomplete be biochemical an patients First, the therapies and disease, response alkaline bilirubin for with injury. patients biochemical levels have biomarkers be

PBC therapies Obeticholic approved only can improving treatment acceptability Acid symptoms many with associated burden pruritus. suffer with patients worsening provide A been well. significant that of have would this potentially not need addressed approved therapy second-line or has adherence Currently from therapy as lives, Second, the exacerbation to relief including of improve patient’s pruritus. symptom and and

of found in for that wide to a included cirrhosis treated of XXX Over without more. have A with seladelpar milligrams. we from cirrhosis, or these to emerged years of series primary endpoint, seladelpar remarkably and currently a also bilirubin in rapid XX and of of XXX sustained PBC. phase patients as two from pattern of response option studies with label phosphatase ranging and with been were available consistent achieving seladelpar XXXX, stage treatment to approval that the potentially open has encompass the patients of two X safe for spectrum has in be hypertension. spectrum we alkaline studies second-line leading majority levels and in the decreases up other XX XX% the which believe includes been dose from in participants than improvement may the used with a their important and across drugs and initiated line developed the of PBC compensated-cirrhotic significant optimal PBC stages three of A an including of XX% XX% in across development. range a placebo-controlled have alkaline on Lastly, disease has Further, Beginning of dose to Significant patients difference those ENHANCE phosphatase, second profile these treatment A that now more patients portal studies should normalize transaminases accelerated patients phase study. doses over non-cirrhotic treatment. a with be compensated milligram of those disease. explored

levels in which biochemical described and was in As pruritus milligram bile have XX on scale. efficacy the rating XX and a a improved lowering publication measured appears reported serum of recent fatigue comparison data with June, on of and of after in pruritus, Liver was our profiles In in finding three treatment. to with ENHANCE, that International, the This acid numerical journal effect subpopulations. similar weeks the sleep patients dose months the the after e-diary of while treatment using we consistent cirrhosis, were these safety safety collection in and pruritus analysis responses the without an pooled

phosphatase, Dennis? Phase and turn ask progress what I’d the Taken pruritus studies, appears profile be treatment on for made effects submission across to and and enrolling the studies. Three package. expected our together, and RESPONSE attention and the to is our cirrhotic ASSURE NDA confirm RESPONSE of the a bilirubin, encouraging well-tolerated we’ve long-term stages as alkaline to transaminases safe Dennis like our support this in to toward to non-cirrhotic and efforts ASSURE part

Dennis Kim

baseline seladelpar global of the Sujal. the three phase Thank I phosphatase the initiated first-line primary our biochemical ENHANCE. same study therapy. placebo-controlled, responder in we The rate outcome and intended will and RESPONSE evaluate PBC. baseline bilirubin, once quarter phosphatase the efficacy registration rate patient is PBC XX inadequate four of first rating daily endpoint to or for XX numerical RESPONSE value phase or with patients year with first same is from response the randomizing include patients treatment Ocaliva, to weeks randomization safety that initial evaluating as acid with in you add-on X-to-X moderate-to-severe secondary you, of second-line composite therapy three our seladelpar measure of is greater. placebo after global, and enroll to alternative key of phase The will endpoints from study April. milligrams, an a patients alkaline pruritus, intolerance ENHANCE a registration register normalization in and pruritus in reflected six in in weeks studied three for pruritus scale this to patients RESPONSE XX-week, XX as or a ursodeoxycholic that the also to remind to at we improvements oral Two in used only in in approved endpoint our an the study randomized, study XXX alkaline and months

the during our faced a entire Sujal to our tremendous in conducting operational experience As extensive global the face asset, COVID-XX continues of PBC has industry challenges trial be clinical pandemic. particularly studies in the highlighted,

review have prior We resourcing investigational have have for sites, times committees and site boards challenges increased including and which to required sites ethics screening. the longer approval previously timelines mentioned faced, contracting at and we activations headwinds with for

continue active of with screen year. on call, patients staff, and patient expected in our more is sites, had completed make to be to XX than driving one sharing XXX We XXXX. to be a have groups, average, the enrollment, in more patients such relationships before hitting PBC, last of sites investigators approximately to two rare range mass critical alternatives patients, in advocacy accelerated more sites countries. the first continues of XX building easier participate end in In wide enroll studies On per quarterly Years as an is XX key expect activated patients our than a countries half results from where activated to to of and disease we we sites Today, pay studies. sites which to we to it the enroll offer in to on with to of uncommon site not and our it off. previous activated this

to our not be to RESPONSE, screening than development and than than that the and RESPONSE, receive overlooked. and placed having providing drug of in physicians ASSURE enrolled. as payers, dedicated In ASSURE, already approval addition seladelpar knowledge with PBC that with long clinical persuasive accelerate. emphasis It our patients our chronic term conducted PBC site RESPONSE, patients in study making patients investigational Like patients in it studies But will sized the progress ASSURE does only enrollment to will and we activated in sites more seladelpar information continues treatment options. our that will decisions of source support significant they it practice is has more of a with rich completing as provide believe make, XX% is guidelines, regulators study in of view, more and enrolled XX this countries only is seladelpar. inform understandable XX sometimes investigators significantly In an has on also but opportunity patients in open to previous treatment ASSURE the to select seladelpar our is significantly like value for well long-term crucial as seladelpar study with with PBC experience. to activation

Let Chuck? me and abstracts featured call to hand to this at the Meeting The week. Chuck discuss that Liver be will upcoming over clinical presentations

Chuck McWherter

without Assistant the of patients minus and These seladelpar an UDCA The today that tolerated. well for analyses Digestive phase and Dennis. biomarker alkaline this in seladelpar of pleased two about presentation hypertension compensated is hypertension with extensive of a of with results is the respectively. part studies FDA’s and markers treated and Director XX% additional to presentation with of a in have The treatment cirrhosis. This for Florida examining recent well has and two the XX cholestasis years, X cirrhotic minus of Liver Hepatology XX hypertension, Center at to changes an a XX% understand a with in seladelpar AASLD to have was and Marlyn is safety stable. of in for evidence cirrhotic Shift either Patients patients in risk at our Mayo, with treatment this ongoing give to during will and factor the portal patients PBC. to portal reductions with of with after seladelpar made interpretation of tolerated. treatment safety for program presentation, in division the appeared mean Portal stress. in that our we’re years, this XX% for years after of add-on the hypertension Liver of Dr. and cholestasis comprise Professor tolerability with seladelpar of pooled led research, ways AST a expert In data, therapy in further the Meeting. support baseline only We will in commonly is transferase, of Southwestern associated liver associated An that Levy, you, for and Over Texas continued goal decompensation, in appropriate understanding treatment all safe a are and and program group. two sustained results efficacy year Program seladelpar compensated option to treatment. the revealing studies there estimated options the alkaline of half two discuss Mayo, believe profile. minus PBC Diseases, and Medicine, that Dallas, in percent of gamma-glutamyl label. from Transplant in injury, revision more of Texas. seladelpar seladelpar at albumin ALT University safe analysis Dr. were to the results and restriction adverse are be as Thank due Dr. and are narrowing portal for in in the drug who and we phosphatase an appeared in In efficacy seladelpar the phosphatase significant in patients. have in hypertension which with discuss Miami Fellowship transaminases Total oxidative than to Understanding oral patients of liver-related and an and cirrhosis events. patients cirrhosis investigator clinical effects long-term Portal and efficacy or Internal improved and the by Liver to PBC University inflammation cirrhosis prominent the its Again electronic to and hypertension change hypertension risk analysis cirrhotic non-cirrhotic unserved second XX% their in for in and examine patients to for to the compensated and that safety patients analysis, minus treatment with and safety platelets, will continues months bilirubin, duration. to year seladelpar seladelpar improvement poster three progression. PBC this report safety and with milligram commitment and currently remained improved Ocaliva development Cynthia of phase compensated upcoming progression was disease Director from portal three with us The milligram PBC. part highlight Continuing portal efficacy, the population. important in two Disease will at that an This comparable patients two efficacy the group

CymaBay link press webcast of live call events will who an The a issued attend KOL two a contained will presentations release in XX, Eastern this and also Levy access these website. Information webcast section those to available to Time. at hosting the X:XXpm be Liver were and the today on of post-AASLD the Mayo is Meeting, Monday, of be we on doctors unable archived For we November with

placebo a reviewed improve earlier, in seladelpar correlated paper a specific co-authors pruritus. appeared disturbance Liver the is set the acid over include three and its The weeks using peer improvements scores has the XX pruritus of independent Pruritus also effect pruritus PBC weeks, this was in the reported improvements agreement pruritus measures by International experts mitigated decreases as of serum and through that reported reported cholestasis bile significant controlled, but different two fatigue in It serum with not questionnaire. bile and published XX using improvement Sujal weeks. acids. questionnaires. assessed persistence is limitations August in XX leading of study with in in This baseline that over between patient paper correlated questionnaires and intensity As and the six levels mentioned sleep XX by to different

confirm RESPONSE our seek CymaBay can Once them publishing remains this from You to sharing committed our study to a provided will clinical confirmation results and study. studies. further pruritus publication to The the important again, these Sujal? we link effects and ENHANCE of in find website. on

Sujal Shah

you, Thank Chuck.

let the of key me we call quarter. for review financials the some our third open questions, for Before

$X.XX million ended we per respectively. diluted and Net share, and $XX.X ended million, to As Net months clinical the in million or during comparable $X.XX which $X.XX half enrollment in diluted medical primarily months the and particular, September employees, largely nine two the and incurred increases global XX, during of million of due loss trials members PBC per Net and in XX, resumption and late periods was as our months grew and XXXX and XXXX, key recruited development loss operating $XX.X were were than ended loss with affairs. our of successfully the in regulatory ASSURE, respectively. higher September activities of or our clinical September XXXX. seladelpar for was XXXX by the expenses, XXXX for nine increases the XXXX headcount $X.XX PBC. following XX, second three team was in XX, driven cost $XX.X clinical RESPONSE XX associated three In XXXX $XX.X share September and clinical active to stage

development cash execute XX, in we with million. XXXX, ended to our our operating in the plans expenses clinical of $XXX.X expect as equivalents, We We cash, PBC. short-term continue and September increase to completed quarter XXXX, on investments

the transaction. XXXX up in for installment we agreement, this million As of will with to months risk enrollment million $XX in installment development quarter, $XX capital February week. into received we existing operating available XXXX. approximately non-dilutive We seladelpar which our we of program Furthermore, third agreement early over installment We last million first be strategic will PBC. development received million completion from third development the call, of the million RESPONSE the that receive of sufficient the our Under of we million on the options under cash fund second Abingworth plan from together add the to in extend provide to the funding $XXX PBC, and $XX with of receive the six additional execution discussed the support to Abingworth our and to expect of to seladelpar investments, would runway. quarter funding development in the sharing believe prior fund Abingworth, terms financing $XX a agreement July, will signed of our $XX current further during our upon

to happy questions. now We’re take Operator?

Operator

question Sandler, please Rahimi first comes proceed Yasmeen question. with Our your with from Piper Instructions] [Operator

Unidentified Participant

Hi, Jessie quarter on and questions for team. This Yas. is on your just [ph] Seladelpar. on Congrats two have we

The can first track XHXX, X-to-XXX? study is you on we now are how on pushed timeline completion, a that that for RESPONSE you or us scale of is enrollment one know enrollment will tell please this to remain back the confident that

Sujal Shah

for precision, the smiling question. to game of position Thank first active the and evolving the in quarterly us you, way. of your here pandemic made let because a the into of put Jessie so We half knew on call situations enrolling really we but next me that giving keep year, prior I wish call. our that I’m and in the kind last globally pandemic announcement with sites our on question this address I was that quarterly

make to continue progress. significant We

we’re accelerations, seeing increases in are We seeing screenings.

with tremendous XXXX, fully enrolling we XXX That a XX confidence of you in overall a successful know, study us patients. global just degree and I got others were of took course, as ability pre-pandemic, to enroll. study in phase ENHANCE our to three execute. at months we’ve under And think So

execute currently, of relationships. you of that But pandemic course, sites enrolling disease, This type over are that really to timelines. know, offset RESPONSE XX patients inadequate challenges now three smaller XXX of elevated active our of So although population we’re countries. mentioned, targeting think the to have rare firmly is we’re execution I is response in the as patient across comes the levels hopeful that and enrolling the at alkaline we believe core That’s UDCA, a XX largely. to and Jessie, these after the from we fewer study We continue phosphatase timeline confidence, patients the that with needed here. on effectively the we’re we’re phase

even that most so that’s outclass regulatory And but the required from below is screen this elevated pre-pandemic, are failure threshold approval. significant still patients whose population really for

much talked since we that’s of for XXXX study, as and execute sites at there medical study. Chuck we think more how and patients drive that’s patients Now, with a the seladelpar to about, week. very confidence. progressed community growing benefit the the this at three about continue experience and on the Those at really I The that a in lot when phase AASLD data of datasets potential excitement excited really our where bit we end underlie initial we we for mentioned, this is we’re our that that to see as share

timelines to So a we expect significant and exponential to curves, We active from screenings, we now tracking to not and a critical with mass straight see we’re of really we continue that to typically to get see active. all really did conversion eventually to different how are from Enrollment enrollment. as sites, expect opposed progress lines, ENHANCE. as sites

So described, all our this exists. programming executing, our most as we of confident and to course, enrolled again. that we on of focus, the think ability depth of And, we’re done is getting before, it responsibly PBC we’ve do in really

Unidentified Participant

you I for question. more Thank the one color have Sujal.

done, looking that recent CymaBay at [indiscernible] market for the we has year. $XXX,XXX the that approved a know So was research

tell So pricing? work what see front the been note, value and guys obviously on for you the that on us payers done activity can periodic and has a payer

Sujal Shah

for well. Jessie, thanks that question as

actually for bit doing the of seladelpar. work population thinking quite a about patient been addressable we’ve So,

rough a different phase as the for anti It experience. And very very a with current than our a mentioned it anti-cholestatic an sets call for children indications, a significant effects has course, life. cholestatic their Of young you approved quality the course, where of priced treatment the well of from only and benefit. more setting three anti-pruritic a patients offer data estimate, and is during PBC Ultimately, has PBC, ability the think highlighted pruritus meaningful burden course. we set second a pediatric year greater on our significant alternatives. of the impact the currently line And benefit. in drug phase even that’s data that significant rare as today, that Chuck of inhibitors, in potentially robust effect, at benefit, ultra and seladelpar Certainly, demonstrates IBAT XX,XXX these two as starts includes diseases inflammatory about and Dennis to of

we we seeing targeted our as in day. key commercial confidence quite pricing to that that about improve Obviously, at the will deliver the out in We’re patients execute how opportunity going to community, think to our work and community, market ability about with research, confident of our the we’re there the And the to actually be that’s continue we datasets. do actual around but continue right in I with driver. the medical value pricing. and upon, the continue have underlie again, to we our payer the really what’s do That’s ability to really would there’s the thinking end for today that and scenario think

their Obeticholic You unmet think for risk reduce to this last that in And need run $XXX saw, Acid, patients, underscores year. need the million Obeticholic revenue now Acid progression. for for north week rate of a on disease significant really the year perhaps a estimates of and I estimates unmet increases

value that do as continue datasets continue think to and but we it’s that we quite work early, and to continue still about confident in of emboldened per and we that progress off patient builds and mature. commercial, to our as So it pricing will we be opportunity really

Unidentified Participant

for thank weekend I’ll so jump Hey, much. to all guys me we’re the at AASLD. back and this in see you That’s queue. excited you

Sujal Shah

Jessie. you, Thank

Operator

question please your question. next Steven from with with James, comes Our Raymond Seedhouse proceed

Steven Seedhouse

taking for Thanks afternoon. the questions. good Hi,

Just be long-term in data were if that or hoping on the Liver Monday? event, that’s Meeting could data I how was The presentation sustained included you pruritus seladelpar. your at and for will if comment improvements on

Chuck McWherter

is Thank for question. Sure. you that This Chuck.

and this as basically, response safety. as cholestatic well liver focused We’re particular tests, so presentation both on around markers, biochemistry in inflammatory biochemical

of the So I very I visits, pruritus with but, through of within a right shows fairly we decreases study to be then all XX the of won’t study quickly, as this four on part that it mentioned sustains the about story out have, weeks that earlier, do call, think, out weeks. data, impressive XX weeks. And price

opportunity would going stability, So that a presenting month we’re pruritus in XX I from way to abate, that the the think reason particular effects weeks. any be all given on the that say, don’t but data not this there’s through

Sujal Shah

The Liver only on came there, significant a relations publication Chuck symptoms I’d is, in just bile as the information burden patients, depth thing of as of Steve, out amount add content. symptom that as other those for International has to the disturbance, pruritus, overall sleep well had acid mentioned, fatigue, of

confidence around specifically these sustained effectively there hypothesize should to that we acids a lead think if effects acids, bile I benefit on so the a that And are on symptom bile the those burdens. that and limiting

Steven Seedhouse

Yep.

the thanks. Okay. there. Well, color for Thanks

I from even first that seem just launch it you everything some enrollment well, in to half like The is you would with goes other on use $XX etc, before, dollars use happening ask million if and want certainly Abingworth. I tranche those thing ‘XX mean, probably could point. final that could at money

draw be would potential what the there? you factors reasons or So your down why wouldn’t on option

Sujal Shah

in interest we programs and It’s funding multiple the and we we in the discretion fund we the it’s the we and financing as co-development an of continued XX going to from completion ways final is to I’ve are to show to about more company company. our the like about day, at investor. opportunities want mentioned that contain a continue solely have end Steve, forward, the Abingworth think the We the enrollment. CymaBay great investors ourselves, XXX we lens financing of I our in past, of that events, company absolutely, we avenues I’d And this, think ourselves value of clinical say Fundamentally, have broadly. up question. to programs the to continue we million course, and And data quite the for option. mentioned, as have as at our seladelpar think this that the the option in to to of right decisions wearing we optionality progress. have just as remain finance actually of various existing of those have that value At grateful way and maintain million driving the the for our to

So you’re absolutely right.

benefit at information great the a is based available ultimately just it that us think that to decision tranche. additional that time. on have I We’ll make for best

Steven Seedhouse

it. Appreciate Sujal. Thanks, Great.

Sujal Shah

Thank you.

Operator

next Our with question question. from LifeSci Capital, proceed comes Patrick Dolezal your please with

Patrick Dolezal

the for thanks taking question. Hi,

use the in provide for you the answer AASLD compensated if cirrhotics enrolled could the to it to So of potential And the patient opportunity for de-compensated into just subset seladelpar changes label within the portal And market evidence context so, be can this of if of Ocaliva, this if hypertension, kind presentation mean of backdrop relates there? reminder will with for you then cirrhotic the question as could population, what RESPONSE? thanks. would you same as regarding

Chuck McWherter

the of the I’ll for portal cirrhosis patients inclusion are comes as with criteria perspective. Yes, step for first to considerations. the have in compensated thrombocytopenia, from portal and RESPONSE so market part. well some hypertension, take and the as expected splenomegaly And allow clinical let patients evidence for those, Sujal hypertension ENHANCE I’ll in would in those some

of probably remind but somewhere you ENHANCE, so in two have well had XX% phase of hypertension. and And that as a to evidence them portal as had a south patients enrolled just representation, about we XX% little of compensated XXX expect to cirrhosis an would comparable

in right now it enroll So, there’s that RESPONSE. to no believe be and shown we same in reason the population remains to

is feel And so it’s something important. we very that

that are You consequences, a significant And much And peritonitis, higher of progress with that’s event an like want [ph] for that safe, drug no risk tracheal your portal compensated downhill SIDS down to can patients either is a patient an much you de-compensation important going as they types basically [ph] risk at events, the slope. portal with mentioned, dire once you’re have if you’re that, bleed, those you be develops, to so things. and know on or for hypertension, with hypertension, have a cirrhosis then consideration. I

studies. that where They’re just higher that de-compensation the a our commercial unfortunately. much progression for let I much I’ll in could excluded are representation patients risk but about reverse, low. it not progression, currently situation think is they’re Sujal say more therapeutic reality less and intervention imagine decompensation, of the terms becomes you’d at is In it’s to population, their call challenging have And let’s a in much talk that

Sujal Shah

in hypertension, I and are with the Yeah, it meant that of percentage be important and maybe those of when so, treating to disease point or some of but we for may you about Chuck RESPONSE even continue XX% at is percentages that XX% there’s Patrick, with a population as though patients a cirrhosis again, additional expecting, here our think study only I patient comments do work much us you’re ongoing some more could patient this time portal spectrum similar even course, a smaller know to evaluate that in physician, think, is progress.

advanced who more safety comfort even be as importantly that physicians or shows in a patients And a efficacy, both patients dataset so cusp perhaps in cirrhotics compensated on as the are becoming of it later think, the having disease, cirrhotic. stage population, drives have level for I well that

the more to a safety. it given set in so it’s a greater population, safety overall pertains population, spectrum, efficacy and drives data And I think can it confidence even in that have broad when you fact that as the

Patrick Dolezal

again. Thanks helpful. Super

Sujal Shah

Thank you.

Operator

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

Jay Olson

separately, to then treatment questions. [ph]? update redefine taken additional efficacy Oh, the hey, of thanks normalization thanks for in be about for or at have steps on of may about as be seladelpar treatment Can for and potentially preclinically? combo Thank you PBC, talk you potential about the to bezafibrate combinations that considering any is the since And you what talk OCA you. alphas the taking PBC, may can looked an Intercept plus working

Chuck McWherter

Thank question. Yeah. Jay, that Thank you. you, for

Sujal Shah

initially, – think just Jay, respect I asking Yeah, steps to with the additional about development you’re

Chuck McWherter

And yeah, yeah. normalization, yeah,

than which a a is a its So give academic where an large of improvement from more the alkaline outcomes. consortium and emerging XXXX little of very of a coming made on registry to they this have color, phosphatase just that Global access Study is normalization Group, to research that’s you has bit PBC patient connection patients, from to

as benefits the benefit be normalization patient, measures the argued, academic have of, normalization. but ongoing comes turn say, from alkaline what community let’s can from And a so look lowering would these phosphatase to best become to call from discussions below previous addition response. of X.XX it ideal Every in from the we their an

the And so and community. endpoints we would but of disease area discussion, academic as continued expect this for patients progression, to be it a sponsors, also both from monitoring in measuring

practice at be be discussion address ideal there’s about the I normalization goal. to should guidelines should think whether whether afloat looked considering that already

Sujal Shah

Acid. for almost a if The the is bit, phase think the their have second incomplete or bezafibrate sense plus your XX% on inadequate or them, Obeticholic they And the response itch. from that OCA an OCA one of I’ll patients, Jay. worsening Acid patient and data Obeticholic on I is part have makes that Study actually here strategy, POISE We know question address of a three those based of a initially can

patient bezafibrate or importantly, markers disease as well that And of think symptoms when markers as strategy. can just alleviate at the well significant just of look perspective, sound on we either itch. pruritus, our a plus a so adding I of meet on on bezafibrate seladelpar to gain greater around those top proportion of seladelpar, biochemical fact, UDCA, needs From response think both we as in as disease UDCA biochemical of the progression data is

of as in through perhaps, benefit robust that data about, talked this can most data for also our alternative believe perspective, initial set demonstrate set be patient population, those get may to the treatment compelling most our we we’ve a first from choice, is if data the use may and even no above whose Chuck phosphatase, There’s not of be from alkaline elevated fact who mentioned, patients X.XX a that still normalization. benefit, further there in down in is disease progression. normal line, outclass at upper times decrease with as risk could point elevated, still even think priority which I can broader one second limit question preferred much some set, line So and that

point time may to. that something in that at stay is we think that patient combinations for close thinking continue through population will to what I be

many setting. to seladelpar We’ve Jay, patients benefit. any combinations se, Our as done pre yet that seladelpar hands in per not the will get goal or is date, clinically of to this of with type in

Jay Olson

Thank great. liver other portal about potential disease? utility one of in squeeze follow to you. to portal or about perhaps either PBC comment secondary could your if hypertension. patients in reducing hypertension, forms And Could Okay, in with I of up maybe question you talk the seladelpar maybe

Chuck McWherter

qualify thoughts sketch that begin can we data. it, wouldn’t being as any Maybe to I we somewhat some to have try I because with out don’t have speculative don’t

well showed two cases, It Paris, not they in there done patients portal it was a was first of versus Jay, One PBC group placebo their of are portal -- on and invasive wedge in that improve the subset in UDCA came they could The which very interesting, UDCA in patients with invasive sense very so that vein was artery patients hypertension. of fact, actually about study a that where PBC hepatic significant it’s study, a and in opposed of treatment portal the both a a as did is out study in number and in some XX, and the most in measured they study and in hypertension. progression examined was hepatic be also again, pressures measured hypertension. halted actually the it today. were UDCA that a improved patients actually of who the portal There looked publication years that as used the measures effects hypertension portal with that studied that compelling that for datasets as at hypertension, to that are Montreal very and

I the an gives this it So an point, portal where a it’s bit example, intervention affected actually one, at hypertension. of think of but an extrapolation

that think I us be for interesting something to that’s So would monitor.

wouldn’t examining in this evidence, hypertension. surrogates for probably be the would say Of way it do course, study clinical we -- what same clinical did, portal would we other you

Jay Olson

for Great. you questions. the That’s Thank much very helpful. taking all

Chuck McWherter

Jay. Thank you,

Operator

with Our please next your question question. from is proceed Mayank with B. Riley, Mamtani

Sahil Kazmi

to Thanks Hey, that the medical a can plan on population, there last our compensated starting up one of related the in taking afternoon. narrowed team. relationships This Sahil the patient in you abstract answered, AASLD, and of cirrhotics Can that be education for the particularly sort talk and about on you the but of Good them and coming one following on see for patients. of you’ve A community non-cirrhotic established cirrhotic maybe to just similar results questions. is how Mayank. particularly some number of both real sort fact of label you’re at been with the given the the Ocaliva. kind an campaign Kazmi benefit the

Sujal Shah

with that that his our for to you ask thank Dennis giving of questions, I’ll PIs some you Chief give and Yeah, patients he Medical some Maybe perspectives of Officer, as experience. Sahil. as interacts

Dennis Kim

something of particular that patients forces cirrhosis, our has the our patients investigators not Sure, they our those already, we on and don’t getting that thanks RESPONSE been at engage one into point Sujal. that Yeah, option compensated this patients in in for that already It’s investigators particular point. appreciate seladelpar is with involved interact principal which and lost advantage patients. in actually of driving really have has much principal study ASSURE, PIs or as an

demonstrate have able we TBC. although show So patients we study. agreement ASSURE, educational the with -- in as in as uncontrolled that roll is in hope well, for be an ASSURE making this to data FDA, able certainly label, assuming an to be And our that with are as out, to sell RESPONSE, point, that we we ballpark will in

Sahil Kazmi

thank Thanks taking Great, my the question. presentation. for you. look to I forward

Dennis Kim

Perfect.

Operator

with proceed please question from HC your with Arce next Ed question. is Wainwright, Our

Thomas Yip

questions asking Ed. This Hello, for Yip everyone. is Thomas

patients ASSURE question percentage so and of what in progress the you past with what stage tell so screening the enrolled can on study and number clinical first far. is approximately regarding RESPONSE the congratulations have target sites the far? trial, RESPONSE Perhaps First, sites of us

Sujal Shah

Yeah, thanks for the question, Thomas.

in of north target here idea that was same. is the we a had the ENHANCE, XXX actually sites, So

and from than we’re fact us patients we doesn’t in sites stop In we fact, you, uncommon fully targeted the enroll finding where benefit enrollment contribute setting, this the study more but not of tell in we places ultimately targeting can in completes get may really I and can not some that actually patients. sites by ENHANCE type believe can sites and activated time

clinicaltrials.gov, got sites a active now. we’ve today, sit north we that XXX it’s can on see of So number where even are you

ultimately We that’s we year. this testament ability And expect enrolled. to end think getting more gives that our fully conduct really to between fully a confidence the important now even around -- study a be around of the study active us and global

I can many constant don’t around thus Chuck, up the that have this, and in Dennis, we myself, basis you enrolled tell patients at actually of specific here a granularity weekly try organization And find operations with identify had far. regions clinical not those sites So provide down the all others daily, with CymaBay, we sites interact on if sites around might have they’re resourcing and have have whether around world. to internal to the are challenges meetings really challenges, referrals of sites geographic for some to in the sites. opportunities we

screening. in in activated bring soon in that other cases, sites are cases there may in they are have are some question come few patient a there’s So that before patients sites and weeks no a clearly there that after where for

not cases, of and saw anchors contributors clinical that on in that a work really centers our in of to again, two may before, the some just broad for disease, that I’ll in effort a paint done we a really who help studies this that XXXX. disease our with much effort. won’t different been XXXX you. patients of at the is of rare continue a things said we’ve understanding and patient so we’ve physician here done this good know the and can end will sites I picture have day, before, rare CRO see experience it’s emphasize. the prior types a PBC So a It’s I but But

Thomas Yip

is where longest after [ph]? Understood, and treatment RESPONSE. us for the to patients top Perhaps long ASSURE one thanks. what among ASSURE for duration Thanks detail about the line report now tell added open responses is readout label for in you term. right approximately data ASSURE the the plan Can question

Sujal Shah

I and may I’ll maybe additional missed. here my Dennis, jump that conclusion and so, Yeah, have color in at start provide please off any

ASSURE, So I it year, say this Thomas, February was to want this actually year. really earlier off kicked of

patients continued previous to board, been three study seladelpar, our phase that sites study. phase as ENHANCE previous And on is come on so our as two studies, that previous for studies, with so had well as

is -- had AASLD, restart bit that a as development there’s to many paused of of through two as prior study, treatment. for what’s have we’ve paused study in even three a important our So actually those patients, our because of and long-term had a prior but long-term years you’ll most been treatment, years of we see, think of at we XX about patients subset out patients I

say those that were a treatment, markers a the disease, we that in of I’d to or continuous benefit terms dozen little three had so out much probably of across for So smaller even patients a consistent subset. and over biochemical see continue patients, to years we

feed a to on this long-term to good presentation XX patients database. focusing subset safety years. we’re continue that contribute to in So really ASSURE But and two out

efficacy the will patient study, around ashore by safety will and time of gather completion file to only additional we have the not We continue our of out at course, the NDA but RESPONSE benefit. data of long-term

depth adds a our currently data breadth study in programs we very are we valuable set. to its that really kind overall for for of think the today it’s program. remarks, those really mentioned as incredibly only in So development And and seladelpar prepared PBC, it the and

Dennis Kim

database and patients but to the that earning to the program, drug the only of setting. an continue trust not open adding in also label take Yeah, also seladelpar

principal I help us accumulate drug, will in think having run. with long investigators clinical As only well the the experience as

Chuck McWherter

add Thomas. maybe other one just thing, I’d

aspects about of of operational kind treatment. ASSURE you’re the at length getting of You’re asking when

patients had distinction ASSURE ASSURE already point back study. operating out prior in to is RESPONSE, to at a are Just really where we sites to key going a between and and be

for patients, in interest, not it’s confirming of searching getting scheduled enrolling. and a just So matter it’s contacting and them their them, going

and is get easier. a it’s into back ASSURE frankly, to ability process different the bit So little patients

Thomas Yip

of one understood, final Okay, perhaps, there. up follow RESPONSE the once

off patients -- into So there of at [ph] positive course, back Is a a treatment. getting plan after you look mentioned the ASSURE to study

Sujal Shah

we think there. I lost Thomas

Dennis Kim

Yeah, don’t hear. I

Operator

him Please meantime, I’d see get Young introduce with Cantor if back. her proceed. can in But I to for I Okay. Fitzgerald can question. the Alethia like

Emily Bodnar

you is served Emily conversations your for better based do patients maybe treating it and this for Thanks population? on our taking fit I’m that benefit Hi, the not getting you. are into characterize patients, or just how physicians currently questions. curious could PBC and who how you Thank seladelpar and on opportunity for Alethia. think be they’re with do from taking Ocaliva

Sujal Shah

you’ve of thus great in a this Yeah, And Emily. of the again the at think it’s please look, with conclusion question far. Dennis, any once remarks, jump experience had I

benefit fairly moment that’s actually that those the putting and course, with actually are unreasonable physician itch, think in really just it’s for that I have but challenges further for treatment that aside of a providing not move a are to that biochemical Ocaliva challenges. RESPONSE known experiencing of them are well experiencing patient without patients, off benefit

prior there that, without of an on treatment either beyond to biochemical and we’ve biochemical also the key off-label of Now, are three experience actually on that we fairly datasets issues. really itch, on either have patients know improved two nevertheless an fibrates markers response. actually if seen the from inadequate of studies have Ocaliva that’s patients and of that even one exacerbation an you look But do And or itch at phase come have number benefit disease. Ocaliva seladelpar that the significant phase or patients

number had where Obeticholic with comparable And are patient’s prior efficacy had those example, we’ve a as the as so we we with patients at abstract a with treatment those experience patients seladelpar Acid alternatives see seen [indiscernible], that before. very data. of in overall tolerability or that tried an as that Those for well actually fibrates demonstrated effect haven’t patients,

have wash have those three fact, we intolerant come into to RESPONSE, but we phase So that into RESPONSE, who days, and as patients they in current off before they actually enroll is treatment PIs our do look move XX study. in this can, see for anecdotal do enrolling emphasize they to those patients, off patients to responders wash see are anecdotally, inadequate Acid I’ll but physicians Obeticholic

Dennis Kim

I me patient the and recent in for decision mentioned but with he something interactions saying made have to down a clinicians me -- actively the is the the disturbance that some have road, pruritus sleep a that to taught providers. that pruritus, pruritus, been will later patient add or clinician study. their of wouldn’t with Ocaliva that and very that fact, told on and that Yeah, he that learned clinician by in interesting, better may physician did, had my the the maybe be about talk patient their candidate one don’t fatigue the got and a Ocaliva placed healthcare would patient which have something known, never This physician place perhaps response on patients

this from to it’s profile as aspect So on that of has. as a Ocaliva it and we saying physicians long need patients can way seladelpar it’s how a educate very well that particular the of be divergent

Operator

Okay.

question. your proceed is question with with next Smith Our from Leerink, Tom please SVB

Mike Kratky

Tom. Mike Thanks is question. the This on for everyone. for taking Hi,

Just curious, RESPONSE aligned receive point to see. certain FDA biopsies patients biopsy enroll which after as liver patients is the to are a real a liver what can do just to only study willing are of follow amount scenario that you a have the to that opted the sense portion three phase in in And have expecting of a you just up, receive the biopsy? a to be able of with

Sujal Shah

Mike, part of did. the I get didn’t second question maybe Chuck quite the but

question. address first at least first your Let me

a a are outset. had that we of biopsy patients of say ENHANCE course, volunteering least again the which we for will that patients XX% we in our at So number at I not recognize for biopsy. That’s would volunteer report. study,

the study, we this we’re so of biopsy disease, learn patients their effort for to we’ve and benefit much we’re patients when their responses we confident the emphasize a conscious fundamentally, progression because RESPONSE, of about into need been for Going of further even did more us, to disease that we ENHANCE. it’s enrolled as going the for stage to in get volunteer

specifically in look numbers well the our we to but regulators as to enrolling we’re confident quite conversations, So patients study, to obligation as report well to as we’re those we don’t as complete those I things but track continue RESPONSE. think in

Chuck McWherter

to don’t it to just And just, said. Sujal with the But, to the personally of suppose any deliver question, to think your an turned I’ll reinforce anywhere be issue, out believe there’s we that’s problem might I going was but I come basically we threshold I if we’re that need close to second it, requiring to regulators. meeting just your what theoretically the part we’ll we situation could consider to going that pass, now. that think thought I right there take approach or don’t not

Mike Kratky

Got a first two phase then top it. maybe to That’s results something one, XXXX? just still before more year of sponsored see are study really half helpful. end terms the Thanks. could or And we that quick of investigator is in likely MBX-XXXX, line early come

Sujal Shah

Yeah, so good question, Mike.

to study complete expect ultimately year. sometime next have We that data and

TRI. Charitable again, once Trust study the by AdventHealth Florida, fully in Helmsley single CRO conducted being that’s by a that’s So a site funded

of with to study them the We are, continues and progress. course, contact close in

would are study that set year. novel from potential next data, quite who do come We’re at for hypoglycemia. patients that But excited one something about some to point with the data potential type as completed think, I suffering fundamentally, opportunity diabetes, is

Mike Kratky

very Thanks much. it. Got

Sujal Shah

Okay. Thank you.

Operator

the and the closing over the we turn to gentlemen, for I would remarks. session to call question-and-answer back reached Sujal Shah end have like Ladies and of

Sujal Shah

of program appreciation believe walk alternative you with a broad and what executing hope I seladelpar be our development to Thank away treatment with an PBC. around the our focus, today, population patients impactful the is we operator. to opportunity a leading, you, PBC excitement robust in today has for most

and in three of do are our all and confident to patience two again. efforts PBC for and in phase it ability We studies global on execution. We the is past phase the in this our is have once know enrolled and now multiple focus need

once this meaningful We to to more believe differentiate data you and Thank setting The to the share continue again. have PBC. presence also at Liver the in week the end Meeting have excited be of seladelpar will couldn’t of another we

Operator

conference. This today’s concludes

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

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