Cymabay Therapeutics (CBAY) 15 May 232023 Q1 Earnings call transcript

Company Profile

Participants

Paul Quinlan	General Counsel
Sujal Shah	President and Chief Executive Officer
Charles McWherter	Chief Scientific Officer and President, Research and Development
Lewis Stuart	Chief Commercial Officer
Dan Menold	Vice President, Finance
Yasmeen Rahimi	Piper Sandler
Steve Seedhouse	Raymond James
Kristen Kluska	Cantor Fitzgerald
Patrick Dolezal	LifeSci Capital
Julian Harrison	BTIG
Andy Hsieh	William Blair
Jay Olson	Oppenheimer
Mayank Mamtani	B. Riley

Call transcript

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Operator

Good day, ladies and gentlemen, and welcome to CymaBay’s First Quarter 2023 Financial Results and Business Update Conference Call. At this time, all participants are in a listen-only mode.

Following the formal remarks today, we will open up 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.

would turn I to call time, Mr. this like the to over At Counsel CymaBay. Quinlan, at Paul General Mr. Quinlan, please proceed.

Paul Quinlan

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

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

Dan President me Menold, Commercial McWherter, Chief Chuck on Executive Chief Officer; Chief the Joining Finance. Stuart, Officer VP, call of and Shah, Officer; Lewis today and are Sujal R&D; Scientific

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

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

believes that actual outcomes, Although, assumptions, to such many whether and to due future are of subject those impact materially except events no supplement are update new obligation The statements company any results otherwise, forward-looking the by uncertainties as or forecast of applicable reasonable assumes required the result risks statements from in company to as could a factors. on and differ based information, expectations or reflected and law. forward-looking

factors statements. CymaBay’s forth recording and from that property for is written the is materially prohibited or of of without set risk results as forward-looking CymaBay to cautionary in cause those to call with anticipated set as any directed differ the statements filed CymaBay. in the annual consent factors expressly quarterly the rebroadcast press are in and conference actual today’s the This Participants the could well reports forth SEC release,

turn like I’d time, to to the over this call At Sujal.

Sujal Shah

an exciting today. patients PBC. joining liver Phase service important commitment program need have the patients rare to to five only worked world's with continents, advancing RESPONSE disease. year improved this for close patient across and before those with program Paul. the seladelpar In a be treatments, the autoimmune X continues for thank leading development for months you patient seladelpar of advocacy experts or from few in XXXX afternoon, of to and Thank care the embodies for results for cholangitis, of Conducted short CymaBay biliary study design Good us aspects in disease you, report we the their primary collaboration with with most ensure that groups our evaluates needs. we seladelpar

operating QX of our of develop early accomplishments through included fund in Pharmaceutical capital and Japan and we a The XXXX. commercialize raised to to will follow rights on Our Kaken offering. equity licensing seladelpar QX Company plan

Our our call key update will and in will and describe today activities, last share our progress, accomplishments the made expected ahead. months since catalyst

Shantharam week, announced of Last the Harish as Financial Chief we Officer. our appointment

financial planning various serving For focus spent building launch. to and Head commercial accounting Vice drug at setting biotech with Global and functions companies, as commercial years, President of Commercial Harish a last strategy particular Sciences for led almost Finance. XX years operations on Gilead companies financial prepare and XX in Harish

business he roles analytics, Gilead, development responsibility, at to driving commercial increasing and Prior served forecasting in Amgen. various of

operational becoming his fully transformation has to as embark CymaBay to as Harish multiple supported leadership brings well strong company. launches commercial course a the Over of his as on strategic a experience finance and capabilities commercial product biotech integrated we and his career,

patients to with impact our deliver on to We our are look thrilled forward on seladelpar his to have and team PBC. mission him to

cholestatic the addressing on around patients XX-year within particularly be treatments to burden, the disease, better industry important advocacy advancing clinical future update call, we and goals symptom and inflection to study leveraging inherent theme at effects a risk we that data we PBC of include groups, pruritus, participant advances had and experts, global meeting stakeholder treatment and with and collaborate communities. world Since In is with anniversary with inflammatory the world, are markers PBC. by April, the real included of in the point hand regulators, the opportunity Thanks an outcomes. normalization for assembled parameters treating understand so of knowledge concepts have long-term our treating on last group convinced to of of Emerging fatigue to many to the sponsor PBC continued that and PBC.

awareness improved and collective the of PBC. Novel investments and growing treatments increasing by community diagnosis is

is goal first quality with time treated both have progression the for therapies of patients while the improving the increase risk to potential Our of that to also the disease number reduce of life.

Chief brief of President McWherter, our I call ask Development, Today Lewis financials our Scientific VP and will Research of quarter. activities to for handing Chuck Menold, Chief Commercial before then a Officer will progress. our review summary pre-commercial Officer, the important our Dan planning Finance updates share Stuart, on and our first clinical provide to of to the

Chuck?

Charles McWherter

currently The countries than to XXX Sujal. this the XX progress long-term XXX studies more pivotal ASSURE than we RESPONSE made more pleased study you, X and and research Phase report in quarter seladelpar than patients of two global active with are in the with sites safety study participants. Thank in together I'm our PBC. and more

Safety quarterly studies by patient that directed The We continue review the most in can both study before with continue as any be RESPONSE retention. to Data the encouraged Monitoring Board recently changes. without

and both sites from partners to data our studies. with then the collaborating is complete RESPONSE team Our analyze to and

submit in to line is RESPONSE NDA as as third soon top feasible. quarter, to the an results share from and goal then Our

whom of are for pre-treatment approval. upper half one previously most of normal. XX despite placebo In by The the in and the is endpoint first-line late online achieved seladelpar ENHANCE, oral RESPONSE, peer month Nearly months, article ENHANCE months, normalization a having tolerated nearly for XX after persistent RESPONSE three alkaline the significant three results aim composite Our After ursodeoxycholic and months. acid. in observed results patients in of and PBC, phosphatase of and published to endpoints alkaline milligrams and the daily alkaline phosphatase of in elevations reports with are clinically phosphatase months in the months. achieve average these patients two of after in meaningful times bilirubin confirm patients after an the Hepatology. six limit phosphatase, at three if three last were composite review pruritus phosphatase endpoint taking controlled regulators bilirubin XX% on journal at results flagship is used ENHANCE alkaline alkaline The normalization AASLD having level taking,

The three severe the in article meaningful moderate testing compared with endpoints pre-specified further using PBC, had seladelpar taking reported disease first milligrams in for diary. pre-specified patients placebo to of and to placebo. with study their that improvements differences electronic intensity, significant XX treatment months those achieve of of compared using on pruritus daily with in baseline to a ENHANCE activity a patients and symptoms active clinically significant is markers itch, both

Once three an to be titled abstract validated serum biliary have the with Austria The associated Post-talk placebo be in ENHANCE with abstracts June and in target be correlated presence to Vienna, is Phase from the the University X eager patients results we'll Once Professor XXth. seladelpar a EASL Conference cholangitis. able presented treatment June, study presented Zurich. gathered ILXX EASL controlled with significant to in Kremer pathway a of from embargo the lifted in and again, share is decreases dermatological in at pruritus diseases. XXst ILXX to we the by insights will accepted pruritus primary in resulted randomized chronic in Andreas we've of

line of from Liver Characteristics Gideon Center X,XXX presented and considerable is since experience having titled biliary by CymaBay XXXX. therapy. acquired conducted cholangitis research has Profiles Patients A in University with to second Toronto in clinical the abstract Risk of clinical be need PBC primary of Professor Hirschfield Baseline second

data seladelpar at The titled are available abstracts sets. implicated RNA-SEQ results third pharmacology the progress established fibrosis CymaBay Pathways the using independent reductions be they mouse preclinical team, by liver Presented new represent and and is Congress. plex the of findings full from The search recorded. identified abstract of data research mechanistic mediated will are two the accepted in Novel these

At this to Lewis. over turn it time. I'll

Lewis Stuart

Chuck. you, Thank

During multi-stage our deliverables, launch flawless core milestones. themes Many and shared careful numerous our began projects We to we projects our January, the streams components while in quarterly of set support execution Beginning to infrastructure logistic and alignment groundwork course enabled initiated distribution have strategy. be within strategy. several human overall commercial for the to that key our channel a call, activating supply will from last seladelpar commercial corporate the go-to-market resources support lay of key I readiness launch. and company's that these legal, our work PBC with seladelpar solutions roadmap evaluate with readiness timelines commercial longer of organizational beginning finance, in all for to

primary to Our analytics by recently response encouraged further our strategies. were we value evidence finalized messaging profile, initiatives studies payer patient are our organizations ongoing and pivotal with continue generation. by results the research research We inform and to and which from strengthen data market and product payer continued HCP, their seladelpar's and QUS target

people emotional, related also deeper Patient PBC. of with insights healthcare were completed living and needs the revealing personal

use expected relative PBC attitudes, patient which interviewed In behaviors XXX can on addition, and then future management, gastroenterologists future for segmentation we their we efforts. hepatologists targeting and drivers to and

plans so for as launch excited to readiness the of leadership we're search scheduled XXXX. initiated for accelerate summer talent key to hire we Finally, second prepare commercial throughout roles have half months our the in our

over the call Dan. I'll turn now to

Dan Menold

Thank you, Lewis.

integrated final NDA during the CFO. initiatives commercial RESPONSE enhanced our strategic phase we prepare the and We As data readiness future work for parallel, for Harish team and towards transforming our experience on line others company results QX in underway preparing for strategic progress thereafter. made are as our we're further operational completing and new in operational analysis potential strengthened on XXXX, of top as leadership commercial to delivering biotech and QX Shantharam study, as treatment recruiting Overall, fully into the additional by launch to and execute and PBC. our submitting have efforts of we our launch a seladelpar to select focus begin year this patient commercial we on highlighted, team

cash, with we XXXX. $XXX.X sheet million strong cash a quarter as a March equivalents finished financial perspective, balance XXst, of totaling From investments and with the

offering. plan to on third quarter enhanced from following the reminder million Kaken $XX.X collaboration in QX position completion year a the a payment fund XXXX. $XX.X hand our of our we Overall, through from of as our believe license cash be operating that cash a March upfront the of will call, XXXX, of XXst, end public As we our equity from XXXX and million sufficient investments current receipt our and

turn operating of quarter now review to our I'll first results.

Firstly, satisfaction required completion from while transfer associated $XX.X associated Kaken, a all XXXX, was our agreement. payment of upfront received deferred revenue of tech with the activities March contractually and as the of million pending company future with other performance obligations collaboration XXst,

in development offset impact to and due enrollment periods additional XXXX XXXX by studies offsetting lower As and of personnel for for our to quarters ended were response mid-XXXX, $XX.X respectively. were expenses higher $XX.X the development in expenses, support hire and continue other and XXXX, compensation line employee operating the costs March million, corresponding completion development XXst, of as XXXX activities. clinical we clinical CNC primarily and Research expenses to XXst, our to following research quarter and March ended in for compared million

As research we and future. our development we development in late seladelpar expect continue in the to PBC, overall increase expenses to and progress stage

corresponding were quarters Turning million $X.X March million, respectively. the our XXXX for These briefly of March $X.X in review ended administrative and XXXX now personnel quarter expenses. corporate infrastructure the administrative and than continue the General were and for general and expand XXXX period to growth. expenses to higher as we ended XXst, and XXst, our of XXXX administrative add costs in a support

equivalents quarter to or as for period XXXX continue ended was seladelpar loss share, and million offset by for corresponding loss an $X.XX and execute in in Overall, March was $X.XX our Net PBC. the of expenses plans in operating pre-commercialization Overall, the increase development per due $XX.X and XXXX and an primarily March interest XXXX to in than income the million we increase XXst, for our $XX.X higher XXXX increased future general each respectively. expect and and earned and to XXst, net our the quarters cash we administrative investments. expenses partially on ended

hand to me Let now call the back Sujal.

Sujal Shah

Dan. you, Thank

coming the the the us excited While about made we we thus milestones are we in even more the are thrilled with far progress months. have of ahead in year,

awareness team improving Our into raising the laser of may seladelpar reducing unique dive characteristics to a impact an forward seladelpar mechanism, at of and on how course, progression all frontier at upcoming new life, the have to remains when in both treatment of updates meetings, includes more objective to goals. aim sharing focused results the plan quality We medical end top on third we a we patients with quarter. the of PBC, RESPONSE to seladelpar lead share of the risk deeper and from you with delivering disease the of line and that look

We're now Operator? happy questions. to take

Operator

will Instructions] We the now [Operator begin session. question-and-answer

comes with Sandler. Piper Please question Yasmeen Rahimi first Today's from proceed.

Yasmeen Rahimi

data we in great team, readout all the afternoon for thank and quick for the Two you. you upcoming very updates, and the quarter. Good about so third much are excited questions

studies you we to junction to know towards of expansion in of phosphatase. be working what at have the would learn spoken PBC regards additional that the you in studies for seladelpar about is, about on one opportunity? patients -- first normalization should past have I who The with conducting expect alkaline

there? any So commentary

And then in to post-marketing of regards the us to report be the requirements third color you time Appreciate any it the both is, out thank on time to the them. after and line you at second for questions, whether question color quarter, top expect commitments will or premature taking and likely data more answer for that is when approval? in

Sujal Shah

Yasmeen. questions, the Appreciate

So I'll some context. provide

patients committed and not path and over RESPONSE since advancing the continue I to around of current we remain ASSURE, PBC. about describing X the as course very for studies with continue trying with we be really to been really long-term first, thinking to advance but to guidelines, seladelpar Phase study our think, treatment committed treatment just we've XXXX, the the development ongoing PBC paradigm

studies advantages think across as we'd started studies we've with historically, we through data better certainly objectives patients first disease clinical advance that care spectrum where around PBC construct those to to and as patients. for have we'll continue we of can steps So then and I committed and seladelpar studies like may we're elucidate additional done think to of begin. thinking those But for the sets the getting those what is studies do details in share the to commit

around continue So additional think with about this clinical throughout of course look seladelpar forward to year trials as updates we patients. the sharing more to you across

in as post-marketing conditional most commitments, to respect With pathway this call you've on as highlighted obviously is think and the Subpart I know approval PBC, accelerated H approval.

with steps study. with long-term challenges course We tell dialogue will have PBC. placebo the a these for to dissimilar you had commitment that of our regulators. you somewhat for we work, as much a updates novel make disease feedback and from not we've we'll over patients in the as in and to way, in running forward the such already can this a that your to We some first think, share years with key of of months. I feedback I approaching more commitment inherent I again have rare in coming controlled given for regulators post-marketing several back just studies described here, what question, a And that are exist committing

to The file commitment we to -- approval. pathway, as for some and that make that study path course, progress in we around post-marketing of is fact, the study in study look have in which initiated, one regulatory have

for tuned it's us coming in and And both the we'll have I'd much these much so areas. to months, near the of on very share stay in more term say

Yasmeen Rahimi

queue. much. I'll jump so the you Thank into

Sujal Shah

you. Thank

Operator

The with Please next Raymond comes Steve Seedhouse sir. James. question from proceed,

Steve Seedhouse

two together just taking one's them ask for because questions. Thanks I quick. first afternoon. I'll Good pretty actually, the had the Hi.

relative some including to been of improvement, could or delta why doing, pruritus PPARs pruritus the you just you've potentially at on mechanistic seems presentation work the more EASL, first Pan so mechanistically improve explain Just PPAR specifically to fibroids. much

of for And failures then about there were X.XX. of than screen of reiterated those details one ENHANCE on the confirmed the patients study screen sort was the and paper publication, or of al-phos on half second less majority were that based the is the

from big would threshold. in I'm patients if normal, that interpretation -- think Thank a suggest that if limit So of but market. theory And demand middle to ground a you. sort above biochemical are below upper it of seems be is those And correct to from there's get patients you of the that substantial of that's that you that. ultimately wondering upside group when

Charles McWherter

in fashion price treatment months confirm in label the Steve, in of that understanding And you is mechanism And controlled in with to quite the and a remained This committed with interested study our seeking six with thank certainly then XX We've week Yeah. three for after endpoint that of open we're for as that seen improvement we've first study. the months. reported the diary. in itch placebo RESPONSE to clinically at a in question. course, patients ENHANCE significant electronic secondary Chuck.

I here. and a think liver eager share this long-term say used Really much or commitment used you be and we And mechanism. the you're distinguished the sharing explain lifted. able down We're disease, a the at only And able mechanistic as to additional aspects features not to that's more really del tracking fully the the details EASL just to, about agents mechanistic committed to PPAR available to the of have that quite symptoms that is approved how some embargo point. once PPAR other from but really of to del to also of not off-label. idea kind either of are trying alluded right improving

Sujal Shah

question. answer And I'll second the Steve,

that over carry Gideon Professor As all prepared And patients have we've roughly have al-phos thousand been are upper true trying targeted Chuck of you're and And presented is at above of detail those the those be in characteristics and those abstracts of for is the better al-phos levels around correct, remarks, it of in somewhere vast X.XX other does of half fact of fact, the benefits of with patients at times in that because patients, of the is the to Hirschfield, across this normal much be had in of And screened failed patients precursors, upper that's to one by some much is doing limited baseline one normal. the that fact mentioned point. have. work the understand fact half between profiles patients line may studies. the a we those potential majority for This X.XX. in we're they're EASL screen that'll risk

And there's domain now, others the global course of in potential over the alkaline published really benefits much specifically more from work progression as in the study pointing work group, towards around as disease of normalization PBC PBC. the phosphatase well

so studied benefits the fact, in sponsors of we're work trials, really group that this recognize seladelpar to trying historically that in in populations And patient well. other and as potential or it CymaBay be not broader really clinical we've of follow

significant And potentially And burden. may month. reduction patients, so see biochemical much we more in and course, in that think are there many think there's from of talk about benefit us course, these of symptom here. opportunity you'll coming We the that more are things the of normalization from again, a some

Steve Seedhouse

much. so Thanks

Sujal Shah

Thank you.

Operator

Please from proceed. Cantor Kristen The Kluska question next Fitzgerald. comes with

Kristen Kluska

questions forward to these Hi. Good and taking afternoon. upcoming data for that some share. you're of to going Thanks looking my

of deep sounds into some work pathways done more the to data, that different So are a mechanism heading you've going the year, upcoming these like in lot relative and it past impacted. into the understanding

that while thinking other to mechanism. tell that going can So focus, further autoimmunity are laser again, recognize in to you and population how the to understand directions, patient help PBC, you. wanted I comorbidities just future what you you're this any indications linked I And to the ask about

Sujal Shah

Kristen. Thank Yeah. you,

to disease to others, with with that antibody. autoimmunity, comorbidities antigen. mitochondrial like is respect an is think and auto auto related to autoimmunity Sjogren's remember autoimmune really I the terms basically by of restricted Related related organ commonality PDAC have PBC a disease, antigen comorbidities described the dominant the which respect that in don't to predominantly to

AIH, statins. be terms which low-hanging is from whether sure There autoimmune of there's I'm does that in some So abstract have overlap, PBC. to common fruit them dyslipidemia, presented seladelpar And well. cholesterol, them the they so carry many that that our lipid could there. And on future we therapy the reasonable which comorbidities, did hepatitis total far are half PBC more interest. therapy, that an we a LDL perspective, effect. other About of studies, going of particular in found course, a not a cholesterol. cholesterol, found recent an on cholesterol remember disease But have triglycerides to lowered elevations and we that LDL And with third half PBC and than of of would strong elevations. in be lipid that something area more patients of in presence as background lowering in And in that total both are of that than a be

an that's common that population look area the patient So this additional in we have with benefit continue at a comorbidity. to and

Kristen Kluska

agenda, Thank group, be anniversary and it it therapies -- much to appears is you then to conference change include global could very for the going yourself. new about XX-year suggesting how how such at written, that. that landscape And and as was PBC the looking study

heading a share to of again. that surprise bigger you data such away walked so there's that the So these important. with meeting either leaders, want a since felt was that will out focus you it general thought you anything that of Thank in to that came you group just or or be that if much something anything into was was wondering, intimate you

Sujal Shah

much was groups and in you were as energy thought leaders patient participating there surrounding attendance that first approved I field. It in going as in And since drug acid. tell advocacy ursodeoxycholic that appreciate to XX tremendous was regulators field, that's in a years, the on was this a there's those more of ago sense event. the in when that pioneered years course much PBC, [indiscernible] number sponsors, Kristen. it. simply work Thanks of There's for I'd the a been really come the well palpable the actually question,

to around among in real when amount think studies, these being future symptom date, patients. clinical significantly. with PBC and to about normalization. enthusiasm goals profile perspective, of and excitement Those of the to our the seen improving patients we've we've really ongoing. ongoing and seladelpar was to of of some hope to around confirm as response mentioned, From to cholestatic we treatment ALT, opportunities, life. themes, treat our broader profile inflammatory are as things in in during we we think markers was disease of alkaline discussion that key two-day likely set normalization burden discussions of the think of expand as as meetings relieving the for lot more these markers we potentially There one of quality us for discussion And much a patients all progression, the like AST. are was the focused the a I well space addressable are disease That there's of respect tremendous patient well, certainly There of course, population, phosphatase, of as see that

treatment don't published on be do that and in to improvements why area. shared data there in in reductions that continued these and hard to changing we've you have alternatives some what able the focus hard now the have discussion areas, that around discussions to researchers It's patients. when actually with Again, have you with benefit the might in this and field of have it's fatigue. in some efficacy We with seladelpar and pruritus, should the be see

theme and patients, of just but health was patients days, a a for say. look of of time. at key a the at these as to paper, treating improving patient actually international -- of fatigue the clinical of sense and pruritus, I would really continuing is just longevity And life course, disease, liver which again the another our not better symptom plaguing improving, those two you but If also just the whole during liver and get quality same all beyond into go this

great So we're seladelpar just think successful. if able as advance patients themes about be care really to help for where might we

potential meetings, with relative of to of setting last the demonstrating leveraging alternatives the data lot in a thing treatment mention And around of then benefit sets PBC. the a real I'll and talk world course, long-term

we've of with to our been that just grateful and will a work as we've we've articulated continue as the great continue really overall. in had relationships folks group working have we're Really since globally, PBC efforts. developed the to so as We've And you group with relationships closely XXXX. we with know,

Operator

Capital. next comes with Patrick LifeSci Our proceed. from Please Dolezal question

Patrick Dolezal

thanks for taking question. Hi, the

with perhaps on you but could even of improving horizon, thoughts levels the the al-phos, the So some competitor pruritus and provide of not surrounding data within importance PBC. just context transaminase

And the the Curious at metric six RESPONSE I versus this looking is know at pruritus was time on months. point. then endpoint, what six Thanks. in calculus XX-month there determining the

Charles McWherter

you for Thank Yeah. that.

and potential a liver risk that to back There outcomes. and its the in is effects of So which and with I inflammation is to associated was terms injury progression, to followed inflammation biopsy ALT of are respect in the we in paper two is mechanism relationship mechanism of harness stage the its to fibrosis extension other hearkening markers levels so showed the disease a on seladelpar for to and histological with important to fact think ENHANCE both del del PPAR in and remain factor XXXX, that a really and itself elevated to ALT study that study. well related study the the its progression. fact led that years, as in our This as the risk open-label significant study reductions PPAR interested ALT lends for risk in

short of up ALT half, study, half to a extension patients three And normalized the had the in somewhat decreases ENHANCE of XX%. which those patients in of those we two-year saw For in had who is ALT over their elevated study of example, months. ALT baseline, at

it that reduction on see injury we which of to the seladelpar So is future injury, will of translate the to liver to biliary fibrosis effects we're the up, and of liver hopeful is in a kind think cholestasis, of injury related outcomes. consequence wrap inflammation, the that into

Sujal Shah

And the question. I'll take Patrick, then second

pre-specified time well. the RESPONSE six in You versus point endpoint, little pruritus as baseline, was original for bit months decision to in a have endpoint ENHANCE that that at the asked the about actually

since Of course, was have analysis didn't to unblinded that when modified six was prematurely. study that patients stopped before many we plan that study out statistical we months

months. look think When our Liver you data, study, the Paper but course, years out pruritus that long-term X, the strong a benefit, of and demonstrating in Paper our prior reduction out the we to again point effects even And as year We uh, but out of six Phase our that point at consistently sustained one look for and not look time Journal ENHANCE, continued six around and studies. is to out time six-month International and to sustained data in the really from potential of three Hepatology if patients, X we you to months for even Phase at again, benefit. and two sustained most at think see clinical of months just

to that in months six a the run-in I at seven-day of taken the average days will as highlight patients series endpoint as period again, measure the in taken readings itself is get before six-month up that a the those leading is seven reading, which versus seladelpar. baseline

on as seen has a historically. it's show we the as in potential believe benefit what well that to So time which sustainability of point again, benefit based the that the seladelpar we've

Patrick Dolezal

so much. Thanks sense. Makes

Sujal Shah

Thank you.

Operator

comes with next BTIG. The proceed. from Julian question Please Harrison

Julian Harrison

and question Thank my progress. the congrats for taking Hi. you on

you to NDA a the factors can getting Assuming submission there? remind timeline will be the of of successful what from RESPONSE, result us

Charles McWherter

Julian. Certainly, Yeah.

post feasible file data of quickly course, as the objective, top to line readout. Our as is

our submission. successful think is to the end day, seladelpar RESPONSE, at in get and confident goal the regulatory first around I of

to Our quickly goal seladelpar the is get hands patients as as of possible. on

bit again, But see studies along continued have way, rolling submission ongoing. So the are clinical out. more in once the rolling clarity data here filing filing, readout us even We've comes specific of I the first to think to overall, work as NDA and look the about as having then you'll Europe. clear. little think path commitments urgency and timelines is processes do that a We and provide around intentions we ongoing

is all provide geared and see little specificity but the resource to this internally, data, line get going and top RESPONSE. in our procedures more fundamentally, you're we So towards successful focus as is if processes a urgency to us there bit

Julian Harrison

very Thanks much. Great. Okay.

Charles McWherter

Thank you.

Operator

Hsieh comes with Blair. proceed. The from question William Andy next Please

Andy Hsieh

role Harish, you. evil question. that very your is you my about taking to forward talked and for abstracts intriguing. congrats with working on look Thanks Great. new The

So question, maybe respectively. but a Chuck one for Lewis, and two-parter

say, dependent? call, cytokine, curious basis or that, ILXX serum your on based characterize level. on terms generally on is I'm can producing in I the right speaking, In that you context depends would type a itch that's cell the view, global just actually which it of So

normal panel have as is a curious you And think tracking ILXX basically forward, independently. if to going of developed the that's is I'm and something be part second cytokine or a you. on testing. it'll kind this about Thank that part just included of

Charles McWherter

Andy, for question. you. Thank you that so Thank much, Yeah.

a the disease you been think at literature, I look look or recent in if find settings, in implicated paper or well. if ILXX has it itch you potentially the cholestatic variety to of disease you'll be even as dermatological different that whether in uremic,

to because say It's in you've an really position thank that. interesting not of I'm embargo. origin, stay type cell just the We a an the to more in that on just have question respect important tuned. you yet, and to I that for so would June. on say that identified, might comment With

of a offer ILXX measured, to there of highly panel, that. part a are being ability measure vendors terms to specialized it's no, that's cytokine although -- normal who In offer

in relief something that some represent documented not view a first the I reported that by with symptom, think it is advance, biomarker our It's keenly sign, associated of course, would but and a of of. it's and patient. having type we're its aware is

Operator

Ed comes proceed. Wainwright. with next Arce The Please from question H.C.

Unidentified Analyst

afternoon a couple asking is Ed. for Hi, Thomas This questions everyone. good

study, pruritus So seladelpar question, headline and the [indiscernible] against do specifically proposition in positive, on envision -- endpoint, composite first how how you ALP from the measurement well? RESPONSE normalization assuming perhaps as the also and the

Sujal Shah

Appreciate question, Yeah. the Thomas.

see course, we we've studies greater proportion we think our to a if in greater profile response normalizing, proportion as clinical also a meeting caveats I reduction mentioned, so patients course, from a think there's of to head far with seladelpar's you've as seen demonstrates opposed phosphatase, across percent head. of that, of the studies, patients And comparing greater of that the all endpoint. inherent primary challenges in composite alkaline the

phosphatase clearly, alkaline than as to patients, patients. otherwise potentially positioning it line position get of treatment have in to one actual in seladelpar ability be potential terms to field offers a think And originally for for entire but goal normalization. I think of will the more potentially the patients, has thought is the of we treatment. setting. And which an one to a broader might in mentioned, a in that second advance used line we a continue the actual patient think of which preferred second as ability course, population areas we've goal, to just not I Again, the

really treatment today pruritus impact, again, acid. real shown pruritus benefit as mentioned, with The treatments first we've is have line significant there that on ursodeoxycholic changer game clearly are a no reducing because

relieve Although, been never to itch, it's been to never worsen shown cause itch or demonstrated it's either.

is we for or without greater we've statistically with we that data one And think benefit in we versus in significant. severe saw placebo. significant we as is overall patients well point moderate score patients relative the to of reconfirm NRS symptoms seladelpar itch, the successful saying the is reduction that overall to improvement of assuming think the that compelling. is physicians ability proportion patients so And that the in those forward. a seeing this clearly think the for we're four quite I story goes that again generated move data response that This to to ENHANCE a Having date to significant in

Unidentified Analyst

so both example, how candidates so are clinic, do for perhaps, much detail. you mentioned Thank in and look for you measurements at as the of ALP any both, Yeah. against candidates other as then well? And other pruritus PBC normalization, I

Sujal Shah

Yeah.

of bit we had a developed. haven't with to I being much in other think, Thomas, experience from as PBC question of date, agents harder we've significant number dataset to times, rich we've seen answer data because a date. to a just that's Obviously, mentioned as

that and around impacts even of think idea a the answer have much I coming today, question obviously the harder we'll better pruritus. so months in but And to al-phops, other on agents on

data I harder date. experience, to to think not relative digest demonstrating to to those seladelpar, is our to where significant effects others more think I been it but compare yet there's much given

Unidentified Analyst

Got us, from commercial. this question it. one's And one perhaps more

your small you. [indiscernible] other Japan, with should how markets in Thank we partnership at others? So US look and

Sujal Shah

another It's good question. Yeah.

make decision seladelpar to so that we in data, have side We either dialogue we're to to or of approval, today. the that with of thereafter. do as capitalized any submit continue not specific and will get I the think the well EMEA. to ongoing in to where to with as the Phase We're for as going with RESPONSE MHRA third-party evaluate have is partners. other regulatory tell ourselves and bring us urgency And opportunities in both geographies sort I X we simply well as companies least We think other in Europe study well allow to points some a UK US constantly. you at would as

of to think seladelpar I we're clinical terms progress continue in our of patients best think the are fundamentally being and conduct to hands going able through So make in of that decisions get to the we studies the us broadly.

Unidentified Analyst

taking it. RESPONSE line the next you Thank questions. for our forward Got looking again quarter. to top Very

Sujal Shah

Thank you.

Operator

Please with Our next Jay proceed. question Oppenheimer. Olson from is

Jay Olson

you thank of team. you've including the all congrats publication received on ENHANCE Can for to talk X And any your Phase following Harish in feedback the the about you that questions. progress, results taking the addition of Hey, physician Hepatology?

Charles McWherter

Jay. Yeah. Yeah. Thank you,

uptake. been it's quite a gratifying. So been I general, of There's in think lot

that than have higher number before. fact, I jumping an I've up In of it's at a application anything already far looks citations seen and the --

level there's interest. I a So think of

think placebo normalization at that's methodology both placebo before. timeframe. three statistical large registration that's that done something of a that endpoints, is with And RESPONSE, the at that's it's been that not We've very lot a fact finally those related closely something to the looking a at and at albeit is driven part in I first think and combined design that with shorter I looking of the composite a by fact controlled, it's garnered for control pruritus, particular interest. looked

looked if terms Of a including course, of article, And of patients. itch. was used -- We that's got a that, biochemistry, agent a very to the of supportive attention. also We into had disease. of profile to had profile, at at course, profile think addition are People something safety of this an see addition in you you a measures in looking that other supportive different ALT for also we safe thus we've is cirrhotic analysis in subset the the that analysis can stages other that something lot looking across with which is I looking at look number overall then, in seen be population the far. really liver

Jay Olson

helpful. Phase points expecting for then or you'll launch? seladelpar That's about any Thank be elated elafibranor of read Great. X talk across you. study when And super differentiation can top you the

Charles McWherter

We would cells, hepatocytes and I know [indiscernible] think found cuff PPAR seladelpar focus macrophages, I in in is important unique for what we about del because think on cholangiocytes that and is mechanism. mechanism cells. activation that to PPAR be delta

homeostasis, and quite injury in you markers something bile mechanism or bilirubin markers GGT of inflammation liver that's at markers, if that so really acid I phosphatase, the well And clinical at. of think of ALT, it alkaline improvements improvement look the clinical cholestasis profile, both does itself, be whether

we to well in in so directly but had have an as yet preclinical in did PBC look as We not models. opportunity fibrosis, yet NASH

we liver on and stiffness long-term. ASSURE a rationale on with their looking to to fibrosis RESPONSE, So measurements effects think long-term but easier stiffness where more there, are the impacts appropriately probably there's detect in confirm study, we're probably through that the over both liver underlying in

Jay Olson

Thank Great. super very you That's helpful. much.

Sujal Shah

you. Thank

Operator

Mamtani The with Riley. Mayank from Please next question proceed. B. comes

Mayank Mamtani

questions taking Thanks on the Good afternoon, congrats our progress. and team. for

fibro also Phase And us of have which quick submission, anything hear, improvement a remind the check with on a be the that chat of more you have good you patients in notes. that. past, where So follow could there's ups. It's a for you the to but if And study box NDA would are then from new just maybe on you've learned RESPONSE. I if hepatic cohort impressive couple had liver the you've X [indiscernible] patients you comment treated. in biopsy small the you're handling piggybacking NASH Curious, and had prior in how the impairment of

Sujal Shah

Yeah. think a but PBC. setting program is in histology very significant that's it's about for the about yet the we pretty Well, baseline something effects on patients. a put opportunity PBC It's to of thorough going share not a and this the scientifically the done driven biopsy It's and lot standard-of-care be excited really normally I'm that, exciting, to evaluation. have very to Not in in evaluate to liver both intention Yeah. examine our intervention What thank population. you to question. we together. that not

Of course, that findings the will to the be to plan FDA, submitted there. publish also we but

I something receiving here. say team energy of tuned. it's It's the So from a lot would stay just

In impairment. that traditional studies in cirrhotic or etiology recruits patients say to hepatic first already regardless terms B, of and child C we do hepatic the have impairment of either completed normals just patients, compared which impairment study, Pugh of A, with

completed that's to So been and FDA. submitted

I ongoing. think B In conducting that C. this I addition, say includes PBC patients in study the impairment that hypertension, the we're and with we're of That pleased where is that A it's portal study cause patients is well. proceeding can

relationship to dose more a to exposure first it be that any liver dose all severe with to and predominantly there's whether a establishes the support study adjustment disease needed stage. identify of or of It's expect the submission. able We to

And then a also at short response, secondly, we're treatment looking over although duration.

those share be results So when study to we completed. the able is expect to

Mayank Mamtani

and combination PPAR biochemical I discussion response, other but And complete on that sequencing been maybe a of response and complete of for There's quick endpoint of, around seladelpar, modeling just internally how also have approaches endpoint. done as stacks biochemical the transaminase del work say, not biochemical an example, using just out up do follow some it. discussion that up PPAR. the against, increased markers. using force. which del includes, view on was of Has a you Got to internal monotherapy trying

Charles McWherter

that Yeah. Well, thank for you question.

that in well relatively That study other was, than considering. was -- New ENHANCE study only not eligibility or was The well for increasingly conducted was studies, well a a Of were liver even in example, in England is goal that example [indiscernible] is, [indiscernible]. what's of course, course, study, the at course, on lower or criteria study for Journal challenging, for it's with been POISE, posted conceived and conducted, it looked ClinicalTrials.gov the in field but published RESPONSE seen the carefully or of that's XXXX. that normalizing biochemistry published the been

little it's easier consequently, a achieve So bit setting. a that biochemical to in normalization

normalize Some parameters to say. I than are would easier others,

a go either dual in With if therapy a respect how line In particular familiar at OKA think reductions. play the indicating is additional failed or mechanism red to has interesting case, therapy fibroid you study that significant a receive therapy. might think probably patients paper, the was very normalization, fibroid had that on that in dual second our this and but into they triple potential XX% population, about the therapy, who on had look I and On I with, which outlast that average, with patients added [ph] fibroid. you're triple or it therapy select a failed reduction who've to then OKA, then visa with

than agonist. and think Phase If del OKA, I consistent ENHANCE more and with on group, reduction. is line inverse me, hopefully of you outlast fibro it's second open-label get X, add potentially PPAR only who and we'll really of kinds that you an what an at It the therapy, have effect says to those we've mechanism RESPONSE patients in the a XX% even mechanisms, vibrates, those or seen look and lowering the you see failed FXR in seem to

raises effect what start you needed? a if Would start with with? you it has So future drugs, multiple a that the would or the approved reverse. one add then greater you and question That's drug have state really in the lesser where

it for. us agonist. for drug the improve you it needs, there's symptoms if would you really course, yes, for think some, liver then could And reach that suggests agent an patient I might FXR biochemistry, the look first example, be would to for improved of something a left that through then

Mayank Mamtani

condition, question an make like label, study taking you on pruritus Yep. inhibitors, to on think to curious, the Or Are was for That on study, thinking RESPONSE the primary have of sufficient question. itch as I Thanks that like doing do crossover providers sure sense. that the you final target beyond also do makes is the strategy label? that? the the be a currently [indiscernible] And get do generally, have effort the to our design. going additional you is mechanisms

Charles McWherter

question. Yeah. Thanks for that

and where for the was our Of well of conversations advance methodology upon including RESPONSE, course, for as ENHANCE method statistical the we with indication to the discuss in desire used. measuring and be our of both as the the design study based with endpoint FDA, close endpoint regulators, and

outcomes XX% of design first These a from add a and more have to nested course, and moderate we are XX% in at sense is, it's since at pruritus, our mount a perspective, think difficult as feature, it large makes could to it's common have are related and to I well, design composite because it's call to look that study itch normalization. severe of all, you endpoint INR disease, factors it studies, patients baseline. a or rare studies, in the of history where,

symptom And a lot a sense. get so looking relief where makes the in we case, endpoint secondary of for that

a the the to get the sense patient they patient, that biochemistry, I the as a cholestatic has that's with as risk outcomes, finally, our that makes and need what best opportunity sense if well most for colitis, factors as for then once treat feeling makes prognostic again, is be for related to an improved symptoms to that used have And single the improved agents agent. respect in that physician, you to most think might gives for other agent

Mayank Mamtani

it. Got

Operator

showing question-and-answer the turn now further At are in our would to for I back Sujal we questioners to closing any remarks. this like conference this over Shah And time, no queue. concludes the session.

Sujal Shah

us you today. for once again all Thank joining

updates been you. look and data with coming RESPONSE forward the third throughout data we've the start the a sharing a additional in of us from with and the We Thank the end year clinical year. month. much As June. at times, to to Top of at fast you mentioned few it's ahead more in regulatory line EASL quarter,

Operator

concluded. attending and for today's may is now disconnect. presentation you conference Thank you The now

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