CBAY - Earnings call 3 2023 | Cymabay Therapeutics Inc

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Operator

Good day, ladies and gentlemen, and welcome to the CymaBay's Third Quarter 2023 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 would like to turn the call over to Mr. Paul Quinlan, General Counsel at CymaBay. Mr. proceed. please Quinlan,

Paul Quinlan

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

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

McWherter, open Lewis Chief on Chuck will Stuart, remarks, call Officer; Harish call Shantharam, Chief Officer.Following Executive the and our we Officer me are Sujal Commercial Scientific Joining Chief for Q&A. President Financial prepared Shah, the today Officer; Chief of R&D; and

we made Private rebroadcast reasonable conference runway time, actual and time from under believes our Reform are turn statements. begin, the expressly plans, for by quarterly clinical CymaBay's statements due in and events, on law.Participants including and based differ Sujal. of lines that as planning the prohibited the session I'd that well a assumes release materially future company outcomes applicable risks factors. could this set subject Before prospects, factors over and forward-looking to and anticipated forth differ like from to dates, to commercialization the are consent expectations is recording anticipated for CymaBay, including uncertainties property forecast risk to annual any of written to to I'd without filed company events during the with as regulatory reports data of This the no everyone forward-looking in reflected The factors like release remind in forth the or obligation materially as statements forward-looking approvals, relating CymaBay.At to expected new call statements set business any plans, Securities of call, in information, result results such many future required funding press and and performance, to this the SEC results and impact cash conference of otherwise, assumptions, XXXX.Although could are is supplement or the schedules, whether the those are Q&A those Litigation defined actual as the statements repayment update as CymaBay's statements today's or or that cause cautionary except directed forward-looking call the to Act

Sujal Shah

momentous to Paul, living with Thank of our third afternoon. update We multiple for us the you, midst and lives achievement CymaBay significant been year remarkable goal especially and a everyone listen has in improving at time the appreciate our busy earnings the of CymaBay, taking with good milestones, of period.XXXX the advancing people the a was quarter to of PBC. towards

advocacy who from have on community feedback with patient the patients its will the from we've results The First, reactions journey.The seladelpar Lewis received in of and and caregivers, II, without key resilience statistical but part and the and PBC. some demonstrates now disease that potential to ENHANCE pivotal significance our data related milestone members symptoms. possible on required the and clinical of family risk all all positive, groups not including at been their are RESPONSE would been the of this RESPONSE in III with secondary high perseverance progression Phase participated markers RESPONSE, of advocacy primary trials, our depth the here to trial the and patients commitment X for generated who by clinical partners, and reduce it and consistency set team met Reaching medical for endpoints Phase and focus, the to groups both treatment color PBC be later his have our patients with first-ever quarters investigators approved provide seladelpar of significantly this the CymaBay, the patient has remarks. very in from

designation for development disease PBC of in Our and the today regulatory with across the encouraged patients are PBC, those significantly patients, is treatment without that people broad a with team as actively applications spectrum high-quality approval PBC compensated that breakthrough cirrhosis in drug symptoms received pruritus that for regulatory a that with needs progression, inadequate of to unmet their while now UDCA FDA, review singular engaged seladelpar as to are FDA's or as possible, working and covers our for reducing granting soon of designation now compensated of who forward other a or lower seladelpar the very to completion FDA with The is only pruritus also including therapy that We cirrhosis. agencies compensated and includes treatments conduct cirrhosis. are provide the in for post living intolerant by with risk were responders including they working for revised focus of PBC submissions. MHRA a breakthrough believe has deliver with We cirrhosis.We look regulatory and EMA.

our for commitment designed our continue study, earlier program largest to of benefit to that recruiting development already in that we with we has the demonstrate third rich data X.XXx responders of unwavering of to studies be outcomes. work advancing the with design challenges will September, address limit global and attempts in interrogating PBC in and quarter long-term our risk placebo. deep, progression firm outcomes the to we study continue outcome have to Our treatment.In neglected trials for alkaline to regulators PBC. on dialogue phosphatase with duration commitment potentially for to believe sets placebo-controlled levels living relative and the between the will commitment. post-marketing also the negative assessing clinical seladelpar by inherent disease PVC a add compensated believe that study this The what PBC. gathered its with These regulatory We care announced group, and years cirrhosis, higher Leveraging PBC. of potential second-line to design in we AFFIRM, a is event-driven the first-line fixed initiation announced as treatment resetting this we also expectations targets transformational upper runs population, X patients serve and of study The in partial to who IDEAL unique outcome people study, evaluating normal,

of and place, scientific are with data community in and engaging with the on the We detail additional disease early call.We potential team understand fully a in our clinical comment have for actively during field are collaborations medical lines but have time today's to for start-up these initiated too us will results, seladelpar education to more community and studies of they now and enrollment majority U.S. seladelpar. on provide the Chuck on the

III We results pre-commercial have activities now and we'll us. planning progress be behind this also significant made these positive year Phase accelerating with with pivotal efforts,

patient are partners providers, of structure and enabling currently near-term commercial while lives.With now continue the over proud will distribution also PBC that, for In commercial health and call seladelpar are towards we people determining and our the supply in reach the and of date. distribution place and sheet, team and a U.S. field our effective achievements our live chain soon potentially front, extremely coverage We optimize will in our have manufacturing ready launch.On potential established care team preparation size to of strong we to in quality to for key activation have I'm a goal with channel balance its march better our the I deliverables With put longer Chuck. a to execute of summary, turn our to we on helping finalize

Charles McWherter

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with There and composite analyze of voluntary X.X rating adjudication enrolled studies. the in can safety from you, were patient world-renowned results study. in patients of X-year key with and the ALP The our baseline patients panel and with oral were significant. included serious to patients. ALP was itch, intensity seladelpar measured primary in phosphatase patients XX seladelpar normalizing a and highly We at week, than expert beyond to with PBC, share for Alimentary meaningful experience the generally placebo have depth in across Dr. arms. panel An electronic numerical Gideon ALP. the ] liver previous pruritus the a regulatory we including Boston achieved a first submissions normal publication, forward of from are years on in during adverse The now and from long-term improvements additional patients scale. of using events highly the high results prespecified XX% XX% of of present comparable meaningful data trial we placebo reduction these biopsies of the subjects.While Pharmacology RESPONSE from composite announced study open-label delivered advent showed sharing patient met for reduction across mean receiving the in study review treatment.In publication both subset time more all and to also Treatment-emergent seladelpar treatment [ response and had on To was no safety strong significant follow-up. concluded safety of primary more ALP The X will seladelpar treatment-related more balanced safety XX report consistent events there late-breaking liver XX months serious baseline more groups response paired pruritus blinded with Treatment adverse of ALT reported events, to completed next clinically between cholestatic significant no seladelpar and Overall, significant and diary, and for versus we the points an & Last multiple in were patients had discontinuations Monday.There's endpoints disease, the with supporting levels XX% program these We requiring that reporting. cholestatic than and for significance. with endpoint, remind Liver statistical our biopsy Meeting findings with look continuing by the of disease normalization presentation seen Therapeutics, RESPONSE. reductions important after course all intended any the collected. alkaline endpoint of expert in effect be were data seladelpar endpoints, the pathologists Hirschfield,

seladelpar long-term has daily ASSURE, as more should than our patients more You study, taking to second safety come XXX currently XX milligrams. expect

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significant liver-related liver a placebo-controlled key conducting survival long-term survival, we develop Additional overall and time outcomes outcome of for regulators hospitalization backdrop had interactions the few in years, study many and events.Over past transplant-free include study. a with ethical known serious operational the in to challenges

in daily seladelpar for studies reasonable committed PBC.Now blinded people in during diabetes XX II days, showed in dosed X-period studied XX hyperinsulinemic compare of dosing MBX-XXXX comparative us seladelpar washout Subjects the subjects either while type conducted was which and with relative In glucose-dependent the volunteers meeting can objective XX. with commitments Phase of placebo. treatment safety establish enabled with by these period with Phase subjects to population in and and hypoglycemia healthy diabetes.The study for a and outcomes periods engagement hypoglycemic it an health days of subjects IIa X to on occasion type found period to and assess on within MBX-XXXX clamps crossover randomly subjects during cirrhotic of contrast, glucagon select with or increases double-blind The counterregulatory of the to was secretion which a X the change of efficacy placebo GLP-X diabetes. diabetes patients among ended insulin-induced or me XX demonstrated were fully study Our gears in time data purposes in type before a this a to followed to design during with placebo-controlled of MBX-XXXX increase daily glucagon to proof secretion was our living Primary provide prioritizing treatment day levels on frame. study. treatment, and and shift MBX-XXXX, single of placebo across aside We MBX-XXXX versus X in by remain pharmacology release Further, of the diabetes Phase responses opposite regulatory target in ENHANCE X whether in III effect type starting X was scientific with XX MBX-XXXX. there X have were consisted Each with glucagon effect to with This hypoglycemia. placebo. on MBX-XXXX type no enhance glucagon that by X been completed. with insulin-induced X, update hypoglycemia has days clamp, let

results his to diabetes like the further action benefit While pharmacology questions studies disappointing, type The and CymaBay demonstrate MBX-XXXX, answered, thank that is planning the were needed MBX-XXXX.We a the study not executed well-designed but without scientific X pharmacodynamic any population. would with demonstrating to and demonstrated and Harry of well and to of Inc. for Orinda, team California, Health. for and executed in a well-designed Charitable Institute study the as Helmsley M. AdventHealth support Richard Leona ProSciento Pratley conducting their Trust, at as and a grant Dr. through Research Florida B. study Advent

to and the to grateful also the it team type our I moment think X PBC-related medical are early in turn is call dissemination and We Lewis, I their mention impact healthy their an for of regional expanded to study.Before patients participation the KOL conferences. volunteers participation and the deployment on diabetes with field in medical data engagement, the opportune

further affairs medical hand the needs and liver be stellar with patient Boston scientific forward exchange to call the look rare patients remain a core who the interface data. featured of to and medical week that play the in today team Lewis. advocates questions I'll for key This CymaBay gathered a next seladelpar interact community awareness we team key we other diseases in raising prominently.With the meeting where have also and unmet as and that, at over facilitating PBC understanding being expertise addressing and will will of and regarding with the role our in We PBC

Lewis Stuart

to leading Throughout market on top are leaders have our alignment and team. line to Chuck. leadership quarter commercial commercial up with response progress in commercial We all been launch and the you, operations the expansion our of access, schedule. added focused in marketing, on recently realizing results, senior that we while infrastructure readiness projects commercial Thank

in including with for to each field-based an in CymaBay hiring launches.The payers in other experience, comes of where role arrival with pharma team in average with commercial leadership XX has more emerging Our the central deliverables. our successes the been with positions, over build-out capabilities of of played to product large biopharma, they access recent lead leaders years' of by support in staff parallel introductory commercial building beginning begin excited commercial our team. pharmacy and of meetings scheduling our deployment the followed key the network We're specialty key market a patient

In on in treatment. play PBC the goals role and payers access affairs landscape our team addition, a for will central new educating medical in market supporting emerging

team force work initiated support Our has platforms commercial integration sales well of and standing sales operations of market intelligence, other size structure such planning, and data essential the components. to readiness as monitoring launch performance as the as up analytics and

efforts, The encouraged treatments unique milestone itch their those highlighted a statistically of key they to advocates opinion as PBC as PBC, be data response pleased pivotal consistently with of positivity which burden as symptom will advance approved both leaders the has has was by confidence the may are in and biochemical Overall, of life.Given pre-commercial target potential key seladelpar impressions biochemistries treatment response the believe only regarding that reduction. both given play the of to normalization since we've so and across positive patients pruritus. and With the early alongside top liver planning and we I improve would second-line living reduces major to spectrum the both relief. first for study seladelpar utility advocates, of that, that the symptoms prove clinical also the the and We set position ALP choice benefit over and to it of properties significant a our leaders in expressed normalizes the from on made on quality thought profile the only demonstrated differentiator, product seladelpar's for broad key welcomed patient that caregivers pruritus their key progress offering line agent reporting seladelpar, are has particularly accelerated and of This profile.Many both expressed the hand of whom and improvements potential a role excitement PBC delpar class, the with and of a very be lead of a for rate symptomatic to PDC. results. impact its the far Patient inability Harish. the

Harish Shantharam

Thank you, Lewis, good everyone. afternoon, and

activities and earlier, milestones stated quarter trial very as our RESPONSE third X AFFIRM to continue was achieved upsized strengthen Phase delivering sheet.Operationally, from III our and brisk we trials, forward planning clinical preparation move as a pre-commercial CymaBay significant as raise for line IDEAL seladelpar new by and capital initiating successful to related a the strong top one executing As well at an space. NDA results to all balance

September terms In with with of expenses. by discount XX, million balance a strong The our totaling the quarter after and from aided other were the $XXX.X equivalents finished equity we deducting offering and in public as investments financials, XXXX, offering cash, sheet offering September. proceeds million underwriting net upsized of cash $XXX.X the

and to first this for clinical investments Research of fund September ended was CymaBay's primarily quarters that into our the The the inflow driven count development million believe to operating sufficient from on and and cash AFFIRM higher respectively. IDEAL initiation million, hand half are activities cash by Following XXXX. the $XX XXXX $XX.X and related The XXXX expenses we was and studies. and increase offering, XX, expense of employee

in in respectively. and commercialization increase medical million September by were and commercial $X.X administrative general a now $XX.X of As development These to primarily in The we in related and increase future.Turning ended driven for expenses. and the spend for the expect XX, preparation quarters of we to and research expenses expenses in seladelpar XXXX and and PBC, affairs administrative of in general organization late-stage progress seladelpar review costs development briefly PBC. our the XXXX overall continue to million, potential were build

we due XX, to net expenses. ended We efforts in respectively.Net continue loss of XXXX September in execute ramp each the the fourth quarter further we for share and XXXX, the to and million increase million and up commercialization higher accelerate as me in to our expect hand an XXXX for precommercial was planning and operating efforts. ended XX, $XX.X or Overall, operating our $X.XX PBC.With the primarily XXXX, these quarter $XX.X our call in than loss September further Overall, for was development that, in we expect the $X.XX corresponding the to per as period expenses potential let increase plans seladelpar future back to quarters Sujal.

Sujal Shah

Thank you, Harish.

Let me room the the of here close this CymaBay. the enthusiasm throughout some colleagues the both portion out of in team by relaying and call of

We improving are milestones positive PBC. are the primary the lives our and to with of our and for on diligently seladelpar eager momentum of people near-term continue goal working

We are happy to now take questions. Operator?

Operator

Our [Operator Sandler. question Instructions] Yasmeen first with Piper comes Rahimi from

Unknown Analyst

speaking This Yas. Jungu for ] is [

X. have We

standard provide get you you NDA And on any receiving review? review soon your versus submitted? thoughts how could priority could First,

Sujal Shah

that take to happy Yes, question.

III months think, roughly as data key mentioned, to X pivotal behind Phase we file expectation unreasonable I So company as us. X be of our sole an focus and to assume to the It submission. for now wouldn't with course, regulatory this inside response is,

be of of So a the gives some by certainly what indication you standard next line. might early time part year

course, of as we quickly to focus, can is towards drive as Our filing.

and both benefits to breakthrough not -- that fully second-line therapy being as seeing compensated population, designation setting quality with the designation in with cirrhotics. differentiation now in we effects a This seladelpar quickly for pre-submission seen hand to and pruritus the in UDCA relative at patients some a a really really then the of seek particular and to opportunity date, existing deliver designation concluding our treatments revised to reductions other And treat that treatments meetings just but us resources filing indication, label potentially able as non-cirrhotics affords as improvements, are submitted of geared in filing as therapy therapy or Obviously, towards with regulators that believe breakthrough the in in possible. development. to added reflecting to internally, broader these so also ability well inadequate the and responders an the the we've in data the reflects processes, a intolerant all patient breakthrough revised now getting are

So as of review. is But therapy well as course, Chuck designation encourages a around the as agency. have up priority breakthrough course again, mentioned, look to of the to still rolling we will, these request forward. revised NDA us submission That paths

Unknown Analyst

our Liver then seladelpar's what profile? hope product at some data are The establish And for key you best-in-class share to points second second-line Meeting question, to

Sujal Shah

information you Yes. think ask be overview give that this to week we end some Perhaps Monday. Chuck and towards I'll the we of additional of could as helpful look presentation the

Charles McWherter

Yes. question. Well, thank you for that

we to delegates quite far. finally found think we're and with what I be able committee share to unveil excited of the to the to so meeting the

baseline balanced, the study good in we've very really course, the our quite view. described demographics a already and population of Of well-conducted

primary course, key We will of sharing, secondary be and our endpoints.

clinically then, response course, So the register normalization. significant change used in that's with in at X. the previously Ocaliva to of as as And itch composite well to itch month patients

it about endpoints think I be those that's right in through I XX, will is to interested think that something to see quite study think we consistency month the so visits, I we way think share. folks would the look across

in measures at And the of like quite the the improvements composites, disease and importantly, the safely in efficacy. of to composites, that normalizations look symptoms. reduce can a and keenly of in population the interest markers looking think is for. symptoms, risk on And of and effect like. then of vital. aware components of other just excited not really course, the think It's focus activity of on as that's a about we effects of markers that whole Additional are to on broad I I chemistry, we're magnitude And we having the liver array used drugs enough think also something be information the transaminases on to safety, magnitude things

eventually So publication. is something forward meeting at to and look that we both a in reporting sharing additional safety

important I think all quite stakeholders development. that's in for the

Operator

Raymond of Seedhouse James. Steven from comes question next Our

Steven Seedhouse

just had I lines X questioning. of

any include Europe expectations sense aligned Or the they're curious expectation differentiated are to in on they're of U.S. still? Curious and of for designation label And is label Europe your that those diverging your quality if it's you in just for? whether have versus then separately, regulators it in the differentiated would looking that if cirrhotics I'm their a you in portend outcomes terms seems of data specifically. data get would a language similar breakthrough pruritus U.S. that First, sense the basically

Sujal Shah

Great question, I Steve, and then appreciate that.

bilirubin that the as important terms for today. are the second-line correlate and think I only been alkaline obviously need I used bar endpoint in to recognize of think first, outcomes. a of it's composite to Europe in the those regulators demonstrate surrogates and actually course U.S. has in improving that existing the And aligned approving in phosphatase treatment to regulatory

need of misalignment of demonstrate data data, different fact in study demonstrate think around think to views agencies in design, the sets, are regulators there's for for don't our I think So attempt these example. any course X-year, that sponsors from we, aligned European and some U.S. sort end, the course outcome X-year to there And that between an make outcomes. I as surrogates to certainly, correlated need regulators to and benefit. the of be I us are can But perspective, these

in think regulators outcomes in the inherent both challenges data in U.S. and I of the also generating some long-term recognize PBC. here in Europe

well. others are right the are to conducted certainly that are on data.There efforts we we relative both real-world being outcomes around And have design, and a AFFIRM generate both. parallel at to think with these right and as on folks the also think we very I generating paths novel with alongside data think real-world been be to in past closely to been today. others are looking should that executing CymaBay And study those data that at have understand of and so the generating is activities. evidence conducted challenges But Executing the real-world these evidence alongside that in sets open dialogue effectively I

is In terms U.S. BTD obviously, respect the with FDA-specific of Designation, designation. your second Breakthrough to Therapy a question

U.S. be the outside as have in not what some to designation at would to sets We mirrors around But or regulators label early intention us will really the I will will But specific have to EMA. provide. as the have to U.S. we for look BTD. look that's the whether It well. a the certainly think to little an outside U.S. will come in bit data prime view the regulators in indication that think our with to the how we the that outside I expect it's things conclusion U.S. these

Steven Seedhouse

And helpful. wanted I ask Very AFFIRM. separately just on Okay. also to

if your and course powering complete will, just And let you comfort then enrolling if primary endpoint which also on in the an First, benefit of is is it wanted I non-cirrhotics in outcomes ensure cirrhotics, study, and confirm adequate FDA that there? an comment period to since just in that will that hopefully the study success of AFFIRM successful. of completing is of time comfortable you inferring

Sujal Shah

the B with interrogated rates. And questions. to come Great Pugh to Child duration and the I'll bit up compensated between Pugh a that cirrhotics, and a patients Yes. fixed populations talk Child X-year event sets Chuck the A we've study, data ask of perhaps assumptions around little really about

again the a design. think you se. about wouldn't Chuck, approached detail the can we probably study But we bit little go per in talk of how I maybe, the into far too powering

Charles McWherter

Yes.

multiple then, phase. with we was on shared once, forward. their more protocol started Of in going discuss confirm with got FDA. did course, to years meetings input back that the alignment a had consider and of course, could preplanning outcomes. And source various move to several for very their face-to-face one after we the before we we settle than of And to approaches drafted, input discussions this the rich design and We had was it the

level including in to in A of of over also but years. quality liver-related of Pugh create that the which In the liver were fortunate element, patients design has rare we with effort this B. the thousands of particular different and only Europe data on in disease not The including in collaboration in has address severity been of Child investigators Pugh Steve, to characterization several international called the death transplant, and Study patients really database Group both there's the Global data PBC events terms needed of America, an disease, collected matured North between Child

us incidence see based natural in and risk to to, only the CymaBay you that So the with history that prevalence up with studies estimate collaborate to patient rate of a expect and expect surrogate. rates, have the not their around we we were allowed as upon what on prior taking reduction power with to to group allude into to based event seen understanding to we population develop able we an consideration of also that but design of events some what be come would a in thoughts

have in. the for example, composite, you factor that So surrogates on could and know the we effects

agents needle, asking benefited. participate So we longer-term to not for we Child balance the don't a population cases, the Child Pugh of be -- There's I nothing a situation. label. limitation particular are every the treatment use Pugh population that might they're placebo-controlled many think a used advanced they that which in options, in really, In that between practical A that patients on but in have available could other especially study, and have there's in phrase, guess in thread every we've B. might country are this approved aspects

to many, placebo they They scientific many think be Having collect the X:X not -- a of to and tell a you in patient It's then developing an try really, evidence, an really I to placebo. And duration. many opportunity, it's a a account in in a go. the into and many keeping typically some take benefit. placebo-controlled no So X enroll mind. a have years to it's There's go than events, but -- standard, on of the more and balance a it's other these a way just years, gold go is get they to event-driven duration. active which patient, cases, also total opportunity fixed ratio number could would trials nice

Sujal Shah

cirrhotics at believe know chilling that that to population. the least about to And have you broader end in out. We Steven, confirmed I it do outcome just and pertain noncirrhotics. regulators asked the cirrhotic will population

Operator

next with Our Kluska Kristen Cantor. comes from question

Kristen Kluska

so, unique that are see if larger the observations the you with then to analysis other patients when of Given do number can and could itch, study? differentiated planning RESPONSE work we IL-XX around And shown data? more And to you you've in that you further benefits? understand N there your conduct driving that a follow, any have to now are you're what's second, trying

Sujal Shah

I sets I -- Yes, to question with answer ask out the additional a about and talk the talk and little questions, itch respect I appreciate ASSURE. I Perhaps IL-XX, of can second to can coming data Chuck bit can more Kristen.

Charles McWherter

Yes. Kristen. you that for question, Thank

we molecular itch acid Of far understanding What's that course, to in that levels be IL-XX which bile symptoms, we're higher we've and I meeting trying why to in to And in or -- you And as at at and into an understand poster up And response. that explanation. seen. IL-XX improvements terms including to correlated PBC we at itch a really known a to protogenic evidence understanding seladelpar that confirm commitment quite driver least level, interested the cellular in of in Vienna will meeting, impacting a baseline course which in EASL dermatological the it delving always, that elsewhere. of in meaning had we the well of we're up we -- seen as molecule, levels with itch on probably we've and share have might at liver saw to cells develop the us and the expect as particular has publications. can of patients so where diseases. we've XX-fold alluding what medical molecular in to interest convincing And the you're IL-XX as can and have in levels be seen do follow mechanism, so think our

tuned. stay So

be assured in area. this continued a have we You that interest can

Sujal Shah

Kristen, -- to great of number going then, sorry, patients asked ASSURE. say asked taking some of great you about seladelpar, you I are today north that some just in And questions XXX

we're on treating to in patients or course understand I'll It this even continue post-marketing very generate our the commitment how life. also of of patients patients data quickly to data in commitment III set AFFIRM. earlier, helps translate or and doesn't end to And with tell is just advantage Phase the symptoms course enrolling intention better patients our normal response of they better that to you the Of quality our us absolutely and IDEAL treating to study. that understanding impacts PBC

to data milestone very flow so IDEAL be to serve come. and sets And ASSURE, both for we rich news years to expect for and generation

a option be data for and to continue support for seladelpar, we gaining to sets successful for very at this generate be the meaningful differentiated that option patients. we'll potential to hope a as launching Even approval and

Operator

comes Jubinville next Cory Capital. from Our question with LifeSci

Cory Jubinville

on within might placebo We little in existing from us. regard in likely couple bit seladelpar the of be this patients a time tackling feasibility challenges upon may a A some of to on that firm, you approved availability assuming the frame. keeping touched how that therapies. But emerge, earlier specifically do plan study

Sujal Shah

question that question. I'll share are a long-term great a there's few then Clearly, addition. setting. comments challenging in this Chuck invite Maybe we in no make and outcome That's anything mentioned, to studies, as

X:X a cirrhotics, Pugh really Child have of will rates to Cory, to that, that specifically We Pugh assumptions event study. part of drives unique hope getting duration feasibility patients Child and X-year to A target patients so in greater ultimately, compensated with think That's B. in one again, treatment. that more participate. a firm be by a fed randomization, likelihood is what's being to the Chuck I around fixed respect factor, mentioned on

asked course, placebo. you Of about specifically

I'd more limited Of limited patients. course, this look where challenge. more for the execute anchor things think options with. certainly patient they is are a already, is of And treatment begin alternatives for There in are I a those ours on what say, that this with ultimately to We'll study areas available be may this to population with regulators. respect we to commitment

Cory Jubinville

FDA's for to And driven the attain was thoughts more improvement that love to the great submitted needle the in it'd response? Got on this it. done moved of in to obviously pruritus in there? biomarker that of you've what previous data symptom a package to And about Was as in FDA hear seladelpar, primarily IL-XX work expansion by more the to quite for designation breakthrough be hear terms studies. bit about regard expansion. really a the Would

Sujal Shah

Yes.

I think I comment. can

data fact in from as that was well as subset data X data designation fact, before the therapy came and recently extension substantial Phase before breakthrough of really II published, generated initial ENHANCE. X-year Our we certainly in set a year the

Phase much And revised the long-term II well of additional so came think our study. effects on anchoring prior the the from designation seladelpar therapy pruritus I breakthrough our around ENHANCE in sets at looking prior extension of as both as data

included in fact cirrhotics. studies All also of compensated these

So And the we it revised benefits. pertains therefore, continue mature. to we're therapy think that the data given by only potential that breakthrough to sets revision designation largely as additional the indication, to label, the never the came again, although sets guaranteed, data these encouraged from continue reinforce

Cory Jubinville

a of I for your also you're guess on assumptions has about? do a post potential great range Excellent. ongoing if And number thinking to us. or sales opportunity really the efforts about last you're to for presented changed speak one have from pre-commercialization how Can you more Last some And commercialization reps that response? would thinking love market or bit that the data. range you I hear a seladelpar? year note more you about strategy targeting,

Charles McWherter

the Thanks question. for Yes.

I think would really I medical perspective, affairs from deployment. start our precommercial with a

readout think on critical right top accounts the time I line and engage and the hoped KOLs would be here we in U.S. able of some we to where at schedule this coming with we're of being in stage out

nicely think I so very are things there. progressing And

the being relates patients. and payers on our the think to market as begin really deploy really them to team supporting guidance these that really to to really hope and ] early goals for quarter moving we to We in it year, into I here access treatment now opportunity next XXX payers. Fedemal helping begin really [ on understand based begin able update and PBC process and engage this

opportunity. target of last X,XXX tell around you XX going folks. initial couple Our that get typically reach XX of gastroenterology process, of I and is would force force hepatology a between lot to I X,XXX sales us to kind we This think generally deployment. sales And to somewhere speaking, the so. this to somewhere speaking, between us where would of here and XX% or to see audience is that, of we've somewhere going started months generally allow XX% the over representatives analysis the a the is

So see kind on now right how some of analysis, that very but that's would that some early at point. of we terms of of this in

Operator

comes with UBS. question Merle next from Our Eli

Unknown Analyst

or preparations It's ex is [ not are with Sam U.S. the strategy U.S. Eli. in on ex you'd thinking Earlier, for ] commercialization progress. whether filing What that and of kind your you seek latest mentioned around a your partner?

Sujal Shah

for would U.S. still as time. the always both seladelpar We've allow study as a I Phase us think period well we're in had been we've Sam, III of some register place, in as confidence pivotal same that in as the to RESPONSE Europe. potentially

in capabilities as And so from in working so a time to same closest can complete that quickly as filings evaluate and that going to one continue parallel and have in the another. parallel, submit to these at so to forward we already infrastructure do continuing ourselves, to potential possible, strategy interest We're we're Europe. in with licensors move proximity fact,

think to these open too staged strategic the the parallel, for think good our that not continue and evaluate decision you'll we But is potential broader the keep to launch for can alternatives I of us company we for today each own. us opportunities think in of on a us commercial balance make sheet the with see news quickly. have, I inclusive about

we're alternatives near think a term. are we where each evaluate in to these of fortunate continue So can position I in to be the

Operator

Oppenheimer & Co. Olson next question with Jay from Our comes

Jay Olson

Congrats the this update. providing for thanks and progress on

that liver about results do ahead approved? landscape, compare look will treatment to those and you we you'd elated And like compare results for they the to elafibranor meeting, become RESPONSE can contrast to to assuming drugs? the the into maybe study and investors you where As X talk how just the think both the fit

Sujal Shah

for of good seen comment That's the of little a question, Jay. line little really we've ELATIVE. because challenging really to top for elafibranor results out us so a bit It's

week fact. shared from from data date an I week today, obviously a we'll an yesterday data to share around think to we've anchor look expanded that when the and in set a look we from RESPONSE

been PBC be much on around response broader anchored placebo-controlled today we are already reducing PBC We're This to is need data quite other set not that in remain proved I settings. with to PBC. significance risk also in an with statistical normalization fact is think are was not patients appears other insignificant and Clearly, that's and biochemistries where an patients challenging global PBC something milestone. the the our experience progression. with which at yet really opportunity agents a It's treat have be distinguished potentially of in something had to to which for ELATIVE on hit something itch reductions now patients normalize fact rates, treatment where think alternatives. the at seladelpar the really therefore is did significance on elafibranor heard second not of results we differentiated the is line we in from they top to that This that respect that from study hit itch. already statistical patients we least study anchored that physicians driving

between look seladelpar. mechanistic profiles we'll of differences as broadly think I There are well. more and safety at these to X elafibranor and the agents look compare

a differentiating ALT liver We are continue this this to that while and inflammatory with continue seladelpar liver Remember, ALT disease. part well. data we is and is differentiation date, chronic we also and think anti-inflammatory. see fibrosing transaminases impacts inflammation, And existing both meeting to seladelpar as approval, endpoint at the to and to anticholestatic primary towards think seeing are we'll on with look of the sets on not be the well-positioned for

anti-inflammatory safety seladelpar. believe well-positions also that again, and So profile now we a anti-cholestatic, antipruritic with

Operator

Our Blair. next question comes William from Andy with Hsieh

Andy Hsieh

to the the kind that this surprises in potential perhaps any the sort undertake that the share as derisk I will that deep of risk? you One field. prevent approach have curious DILI. if to do has have emulates of I'm regulatory seen investigation can in-house X. just And likely you're to taking a sponsor FDA with we

Sujal Shah

Happy Chuck, here. to like some if to answer the question, Andy. you'd Maybe, give color

Charles McWherter

Yes.

tried algorithm toxicity. scrutiny that's that's And drug severe disease. today Of employed. there's employing be risk adjustment it, every an for I pattern in There's there's drug-induced would liver risk there's that toxicity. for the implemented to about of published course, and well. hepatic the paper believe sponsor it's setting close a the a that and algorithm consensus very a evaluates course, always one development, to of the liver important that guidance FDA true specific in And for plays any we follow we as follow how the

program setting We of studied it's we've across really think our me milligrams a XX profile. I to a lower that we has as feel encouraging in good that really say reported seladelpar really disease, where liver for the that had solid really or we've previously, experience.

as So enzymes. of other to course, liver But together we this putting any there's -- we regulators. well methodology, regulatory for that analyze that level the any not a that do as way point. and changes, report of and an part to that's in use example, protocol-specified at And of concern we FDA all have accepted we're submitting package

Andy Hsieh

being about kind think patients, Wave you some opinion about patients you X, speaking think perhaps as And it. obviously, penetrate. discontinued OCA-treated commercialization, there with and appears to terms of the stickiness as in Got to key in of Wave easiest be leaders, and X some

various convincing into currently care Andy, one frequencies communicate of in you to may stickiness very what's As OCA, overcome there have good them you question, second-line that there. where be potentially have kind either setting a first only better inertia and of here pruritus, the treatment, I'd high say providers think is strategy of And that I a expansion really it's out you about option approved a think all. concept health having alternatives. on with but different the when of

and most have you that described, to there on for patients already first second I been are the many call think important is out, line thing as have me discontinued.

the on is In number in fact, remained is U.S. here greater line that those even that approved acid we the only than think have second obeticholic

well. And still many opportunity patients are those so not been have as I there's think to yet patients. more treatment there serve on an second-line that clearly Overall,

has that think very have may some current you significant fact I and in issues switch treatment. completely any with the not expect tolerability adequate to to over having you. agree We change treatment We may opportunity the before patients do PBC. But really even remain that are should they're alternatives. occur. from second-line so of seladelpar seek other an given. ultimately Certainly, on the that in do that the the paradigm opportunity Those response, contemplate of is patients treatment we again, And being think actually setting I

respect And actually an is a to second-line efficacy in what to date XX up-titrate us, we're we've are about improving Pruritus seladelpar, reduce patients actually X significant don't with always issue. that treatment, know talked to with tolerability something most that as excited to parameter, quality ability We with the the existing actually And has for the effects lives pruritus the seen so of profile. ultimately, patients. for milligrams. is on about been for pruritus from example, efficacy

treatment. see different a most going you're view the approach and think giving optimal I again, to very patients So to

believe right to we're process the advantages really significant may through And to so regulatory offer get because now, we is focus patients do our successful. if seladelpar

Operator

Arce comes from Our next Wainwright. with H.C. Ant question

Unknown Analyst

questions. Really our [ questions ] It's Ant. Thomas asking appreciate taking for

the major rate first half estimate. behind what be launch that runway the in table, on fixed the cash U.S. you preparation figure next wondering what of incremental believe and cash in then if is, factor the would XXXX the as the spend is ballpark not, some assumptions And Does second run months. XX question in just EU So mentioned. are I launch

Sujal Shah

Yes. Can line again? about Were estimates? the time related repeat costs you know something to or asking launch. question you I you first asked

Unknown Analyst

U.S. estimate, Cost our to incremental months spend perhaps related in next launch. XX the

Sujal Shah

to to sheet and the Harish ask balance the questions costs. both I'll of Yes. related answer

Harish Shantharam

see line launch what affairs way board, our Actually, Yes. in all side question, account we investments PDUFA other medical we're come commercial more we're rep the organization. we'll now The cash team actually a that looks piece, I the sales to, in sense that takes have will we to efforts, -- on date runway like. Lewis data of and commercialization accelerate. our on actually, closer time once The into on our of building would a were probably that that that have the alluded the answer

uncommon, So probably it's X, by months X not before.

our kind assumption baked runway. cash that's -- I in so is into how our would So of that

you assume now team run when board. so commercialization the launch the would the our you premarket to question, have efforts and full a of on to And build included second part get runway. having our get higher and up commercialization your and it EU have rate the planning between now a that as And we more cash will post then -- now is we we end all we of not -- full-scale in would

filings again, not commercialization we're progress making some still full base are that respect I commercial We is estimate. -- in with say early understanding regulatory and efforts, wouldn't a to but

Operator

Our comes from next B. Mayank Securities. question Mamtani with Riley

Unknown Analyst

] William for on [ actually Mayank. is This

EU? just Ocaliva into couple read in of So that were here. any if a for there your to was the follow-ups We anything curious indication review the investigation Article XX has

Sujal Shah

the what review to don't for respect affect domain would date, is of, of that We with seladelpar. process everyone forward our know with XX anything frankly. discussions we're public and this aware there's To that No, believe we Article outside little from knows that nothing the Ocaliva.

Unknown Analyst

one that What is table or release. I'm do any included Okay. really included show then part the on be extra you we since follow-up. improvement Just color. on your Appreciate info think this a sure is the will that needing quick that. adverse be label? Should differentiating on safety attribute pruritus? line to also you wasn't And when focus of is event top full should disclosed

Sujal Shah

Yes.

was observed comparable arm observed milligrams I treatment what were XX the in response to the we was think in placebo. that with we at statements top safety line seladelpar in the made

what studies. that consistent previous fact with We in we've safety also the was made a in statement seen

a seladelpar, Phase north of the I of think of are out out XXX fairly which that's published data set, and X II ENHANCE to in to significant randomized years actually the study plus all patients, domain. the it patients which went a that then XX for XXX year,

set a there's already. seladelpar for published rich the think very interrogate I to safety domain so data And in

observed now the with profile that published And is very response we've what multiple safety on in we'd so in consistent studies.

out some look of data should week, of share so again, think that we'll confidence, And I garner more response. of that and level to towards next

AEs pruritus course of respect with their of the about to XX% You asked disease. PBC specifically with perhaps in experience up pruritus. Again, remember, can patients to

not NRS The PROs, to assess observe So course, PBC-XX setting of the uncommon or reducing you more the as clinical have in domains. we've in of are tool pruritis prior pruritus. whether incorporated an as studies our as itch well not AEs that ENHANCE XD of important on including impact well tool to other response study, as itch, actually

best of you the not reducing gives really itch. tools you whether these interrogating have impact think I sense on or an

next all sharing more are we look think collectively this to these I forward of data week. insightful, So and of

Unknown Analyst

again. Congratulations

Operator

Smith Thomas next Leerink Our comes question Partners. from with

Thomas Smith

And or Just have you in could to to your Europe to valuable the and EMA favorable designation. therapy perhaps revised be think plans the breakthrough of more you similar scope clarify, that there? securing the designation is regulatory something path reimbursement U.S., the now wanted either in that access or prime a engage the their around conversation

Sujal Shah

Chuck, comments? you some like would to make opening

Charles McWherter

Yes.

Thomas. So thank logical A very question. you,

to of and regulatory that for application I opening seladelpar's the submitting again then -- rather the once Prime. have think is on at path meetings is than our up having you now kinds presubmission our focus Prime the or EU products not respect the are unique. but oncology is very actually are diseases. -- more prime-designated of Europe -- products of position Most more types with serious Prime in different in other

to So we we're benefits access us the rapporteur, designation, from to frequency and have interactions a of close provides come interactions. and a regulatory with it us think with the quality that with prime able

they're thing. same we and I kind U.S. but going towards people do that think They process the same thing. that that though just that even I guiding breakthrough have be would semblance, the you I in repeat did of the exactly same it's think So thinking prime think the not we're to some not are logical designation

is can. soon thing we get us So submission most into review share the towards and as to forward we do think important as move data to sets for regulatory the

had patients As than the XXX is it's that strengths one seladelpar. our with of -- think program the with PBC Sujal more dosed I earlier, we've of mentioned

than more have as we ongoing speak. We XXX

size of with of then that treated the and that around in the Now and the the cirrhosis pruritus -- is X that effects is consistency have placebo-controlled the and that data an totality open-label also study namely features the it's sets came something revision, the pruritus opportunity. patients we've studies,

response. the more experiences in the subpopulations. than studies other have got supplement We just those We've to

Thomas Smith

could know Got from guidance just any biopsy sneak was it. just any I on data paired That's in liver biopsies with here. based AFFIRM follow-up if if cirrhotic response helpful. patient And paired liver study some I the were wondering you the in the a in optional I the quick collection conducting or if you there's FDA. requirement compensated collected population. trial of

Sujal Shah

Chuck?

Charles McWherter

Yes.

disease feature characterize collected -- just once studies, not to unique most we've -- them, is So think, on of program. we for histology started we we differentiating be all to we response, -- with commitment, and we've continued status that a seladelpar of able the

ASSURE. collect collecting paired also biopsies liver will actually biopsies So AFFIRM. in we've be in we And

they're Now optional.

their it's a those and to making. requirement understand the effort interested histology are that's in happen willing in not care standard liver So part important and for be of PBC. because their think for help might to it's status of an patients gain who we an responses, understanding us not to treatment what of But

Operator

at There are further questions this time. no

to the concludes for I closing company that to would floor like session. So turn over our Q&A back the comments. management

Sujal Shah

Thank you, operator.

we've pivotal accelerating our in activities as is patients positive As as to to each team hand, Phase with now of date quickly get seladelpar necessary discussed III the possible. to data

have to liver as for of living potential we in in have as our months people the professionals to discussed prominently seladelpar the liver CymaBay of excited and IDEAL been week. a partners improve studies, for at meeting as PBC to the in few to presence be Hirschfield began in Thank here as most others all struggle health the valued with significant we fortunate as here with we which This Congress people and of of the their we and our reduce ago, earlier joining. to we of for well have to later present life. in company. since XX. Gideon expect care III sincere investigators not seladelpar be and treat share more meet to they us the has more to ongoing need XXXX.And of have and will AFFIRM, were potential and Dr. much PBC presentation continues We again the and progress featured development to years symptoms treat commitment normalization be be our the our to we RESPONSE results with remarks, treatment advance to treat behalf Their as RESPONSE, starting detailed November today's late-breaker quality ASSURE, we've that the driven Monday, about alternative us the at Q&A expect of again to a daily Chuck work in inspire you the Phase session, a you summary of ahead. study every stories do fortunate many years. today us meeting in again, continues remind patient of with to advocates, on weeks And all many beyond this our PBC Boston mentioned on that with several and more to They share us at Just could mentioned with Again, day. focused, in his be we

Operator

concludes today's This teleconference.

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

Cymabay Therapeutics (CBAY) 7 Nov 232023 Q3 Earnings call transcript