Cymabay Therapeutics (CBAY) 10 Aug 232023 Q2 Earnings call transcript

Company Profile

Participants

Paul Quinlan	General Counsel
Sujal Shah	President and Chief Executive Officer
Harish Shantharam	Chief Financial Officer
Charles McWherter	Chief Scientific Officer and President of Research and Development
Lewis Stuart	Chief Commercial Officer
Yasmeen Rahimi	Piper Sandler
Steve Seedhouse	Raymond James
Kristen Kluska	Cantor Fitzgerald
Patrick Dolezal	LifeSci Capital
Julian Harrison	BTIG
Jay Olson	Oppenheimer
Ed Arce	H.C. Wainwright
Thomas Smith	Leerink Partners
Mayank Mamtani	B. Riley

Call transcript

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Operator

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

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

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

Paul Quinlan

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

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

Chief Joining Officer; R&D; Scientific Shantharam, Chief President of Officer. Chuck Lewis and the Chief Financial Officer today Officer; Sujal are call on McWherter, me Shah, Executive Commercial Stuart, Chief and Harish

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

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

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

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

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

Sujal Shah

Paul and afternoon. you, Thank good

top to when RESPONSE. line this confidently advancing key and milestone exciting expect a are we We is report fall, results towards what from

anticipate line seladelpar biliary X from then top as September. by releasing submit approval RESPONSE people Our global for regulatory we’ll PBC, with of of registration primary as study results feasible. And Phase to plan end in we cholangitis or soon the

includes the new program of an of important Our seladelpar development IDEAL, update planning and seladelpar. in of an our study for clinical announcement the PBC today activities for pre-commercial overview

a we setting new CFO, Shantaram commercial to get various me in and us strategy our commercial call. on years May driving with to functions experience Before this building XX Harish companies these focus updates, to financial operations and brings drug and financial welcome particular let in over planning for launch. joined of companies biotech Harish prepare accounting

leadership His biotech and have experience critical be PBC. well plan to we during a of as as filing growth we commercial his Phase for this the critical X finance company commercialization our study, integrated delighted capabilities transformation with strong Harish are will on a regulatory the for for seladelpar patients fully as as time of strategic to completion We operational and company. of commercial embark

treatment participants patients ongoing our currently for and the ASSURE execute to most with ENHANCE including study we is Phase ongoing the what progress our study, receiving seladelpar on in studies, ever the in the Turning to RESPONSE clinical robust XXX program with X study, program conducted with believe open-label X Between over in Phase continue largest PBC seladelpar our one the development is Phase with prior X XXX of seladelpar, currently development over studies. we PBC.

an advocacy Vienna either to centered for in patient normalization agent. and approved treatment sponsors, to or consistent goals EASL on for the industry first-line there patients that adjusting dialogue symptom in We treatment markers who the leaders, intolerant goals of the depth revisiting the the treatment have is inadequate theme include to improving potential seladelpar the in clinical gives consider thought clear, PBC confidence differentiated second-line Congress It us groups to include burden. ursodeoxycholic a At are PBC. a response to data offer among acid, in June, cholestatic that of discussion considerable need noted and inflammatory was and option

in continued we initiation have study announced but used normal progression. continue X.XX at normalization the level today, times identified a for been that be a trials ALP cut a will of lower compare for versus to to the effects in on In ALP’s population upper of of above limit of elevations persistent Earlier has IDEAL, PBC. several disease often clinical has off as the that EASL of who risk this below placebo patients normal, been publications,

guidelines recommending for included they not current clinical research. treatment not meet in and typically are second-line However, do

We potential believe potentially to be for population, neglected second-line that transformational treatment. IDEAL this the for expectations resetting has

recruited much be in higher than We previous ALP population levels given of and results alkaline IDEAL. are optimistic will normalization IDEAL seladelpar’s potential for a ENHANCE the to rates phosphatase had that of

excited timelines Chuck enrollment are today’s while data, more comment is actively is the this We provide it for us and first study on will during study call. Seladelpar too in this cholestatic that the and is that on on for for to recruiting now that and primary early total responsive registration the delpar class of liver. of it the and PPARδ efficacy composite including has on bilirubin Study drugs profile results in normalization. a markers suggests ALP of disease, targets endpoint ALP detail to-date differentiated

far be results confirm clinically or believe patients seladelpar If suggested in of Unique our today. that be reducing PBC, treating prior study RESPONSE in we we to to If is consistent could important care. key ongoing than in improvement the landscape aspirations PBC, advance seladelpar in to if treated is significant future of patient seladelpar’s a second-line those would for confirmed more the scene the of important approved IDEAL, pivotal that step that today, an are with in studies therapy are feature development this and itch. with by positive might in remake safety expectation have be the for quality life profile profile our those agents with able among pruritus forward in

good front, field operational progress PBC. continue make having affairs including medical organizational build, to the leaders our in members including we On experience, years and of both with

We in commercial the pre been line with assembling successful also will our have call. in for priorities. infrastructure more our talent later in Lewis commercial key provide details

provide let hand to the me that, Before call will Chuck, who over clinical on progress. our updates

Charles McWherter

the global patients we I’m you, X two this pleased quarter our in Thank in report with Sujal. progress of Phase studies seladelpar to made PBC.

X entering is now the important RESPONSE what are stage study. our Phase of We most

final we line closing by database results. are for and locking top visits study diligently and and are analysis last followed Our imminent unblinding cleaning activities out for

In in development the quality, activities, of to this, efficiency. all principles of and as our committed we integrity data are

with followed communication pivotal studies, to top then at approval experience medical complete by feasible. well members line With in-depth submit the for publication. the applications team a share to we end and results on timely, and our of having by regulatory positioned intend endpoints in are of as specified September We key many as conference pre a results in soon

Safety seladelpar is it study. The active further depth of has and than this XX research Currently countries more seladelpar patients over in sites underscored ASSURE taking by the XXX XXX XX mg. program long-term

the supporting regulatory and efficacy both symptoms a data We long-term And source of and PBC. sets will expect filings. disease to treatment, ASSURE safety impact our in contribute information a of be to seladelpar documenting important

both DSMB retention continue encouraged pleased most changes. are continue review the RESPONSE any the by by We report patient recent can be and that as that to in without before studies quarterly to the directed study

as the or stems important PPARδ of delpar. from profile confidence of clinically and both is profiles. regulate have in critical as important mechanism inflammation. PBC well we differentiated selective the set data seladelpar delpar To past the trials clinical agonist from underlying Seladelpar cholestasis Our including driving the robust understanding pathology liver, clinical in first pathways in our the and accumulated

the before call Given our some X XX% from who be more point open-label XX% that the this studies. end mg there three phosphatase at trying taking regulators recap mg among arm seladelpar months And this by bilirubin about XX in alkaline used I’d study just study, seladelpar and over briefly patients our program composite achieved may results are parts previously like for many months. ENHANCE is approval. to at Phase endpoint oral top prior daily other learn achieved line profile twelve our In saw to now our XX the last

three in one on average limit normal. nearly ENHANCE upper normalized levels after of these of addition, patients the phosphatase alkaline despite months, of levels X.X times In had pretreatment having three ALP

electronic quality life, mg placebo compared pre key using had a with moderate amongst in to of result diary. three With seladelpar to XX those itch, to patients intensity improvements daily significant taking months the severe specified pruritus for their respect baseline

on the expectations now in These over clinical me high results RESPONSE. working us achieve point outcomes to the of we the initiating the study have confidence opportunity also talk our hope that a seladelpar in few today’s on and range profile past call that on IDEAL, is been months. we about Perhaps set for to give

in me risk have normal that from the background beginning for a progression above those First, Study for at some of are than XXXX alkaline elevation disease shown context, ALP. any PBC normal publications let having greater Group you Global give patients of with levels phosphatase

by worse, These times risk benefit evidence treatment critical the than a of is gather could studies this UDCA To market in additional way the level patients of eligible of for agents. with to between greater spite to persistent research have that fail on suggests ALP has early with their population new UDCA patients typically it limit X.XX in of potential be they lead wait disease. may second-line paradigm, the progression make and treatment This would U.S. clinical of be Our evidence where normal. that in managed seladelpar. the XX,XXX IDEAL of the wouldn’t one from a to some upper

of a is one a with to Given randomize evaluated daily believe limit XX placebo randomized, population, endpoints one the X criteria The upper a plan levels normalization daily will IDEAL ALP this patients. using groundbreaking intolerant X.XX mg the weeks. between ALP Phase for XX All weeks. treatment that diagnosis planned times many be diary. prior of seladelpar IDEAL be of to placebo-controlled stable seladelpar taken will electronic unless endpoint oral for dose and similar and PBC with at secondary is It study patients study of situation and for two to overlooked exploratory XX a be primary patients studies other the the prurituswill UDCA, normal. is of to we eligibility XX

this at approximately study. X.XX The the times prevalence enrolling prior one studies seladelpar upper that of levels presented our in that we the between in and feasibility screen are regarding ALP poster, reported encouragement in a these June, of normal. us considerable patients In patients of failed because screened PBC we EASL limit half of provide of

the turn MBX-XXXX. over I discuss progress for a brief Phase I’ll proof XA seladelpar pre-commercial Lewis to on Before preparations study provide our call update of to the our for in pharmacology

a is funded by Leona a agonist hypoglycemia Charitable The B. As developed GPRXXX and fully in to and retaining for study MBX investigated MBX-XXXX, X minimize being its prevent discovered by Trust, type the is or all rights CymaBay potential reminder with Harry with to XXXX. Helmsley diabetes. patients the M. and CymaBay

clinical glucagon the In maximal and related treated the versus are of now results addition the fully The study to the safety curve, patients the MBX-XXXX the share area we we steps data end and study and will path. next Depending placebo. the tolerability, enrolled to regulatory determine expect glucagon under from to on the is endpoints and primary year. before release

will to Lewis. that, turn over I With it

Lewis Stuart

Thank you, Chuck.

Over continued scientific high to by tough engagement focus rigor its have CymaBay’s few commercial and vision is months model innovation. unwavering utilizing stakeholders, by driven that supported I PBC an a and last the on

and course kicked throughout of we half designed by brand, the support parallel, infrastructure. our the seladelpar first of XXXX, their and global journey. patients to as to foundation began with enhance established corporate support new programs developing been media a specialized providers building the well of partnership to PBC distribution ongoing In advocacy treatment During agencies and diligently We of highly have we commercial model their record marketing outreach CymaBay’s HCP all groups. off patient as programs designed

goals second whom commercial to in leaders fortunate marketing, quarter, the experience each with key an CymaBay’s in key During of we our with alignment potential recruit considerable begins seladelpar vision treatment. and role our corporate its and success resetting for as market accelerate build were in of access for science will commercial hire and come senior and the operations, essential have IDEAL we the PBC milestones. with commercial commercial achieving

and response, able a Our bilirubin. in total other complete of secondary treated less than of experience words, research normalization indicates XX% ALP UDCA patients that are to

Our of with a reduce not and being to treatment research improve suggests there is patients an proportion biochemical second-line today. for treated opportunity response significant symptoms

study potential initiation for the in excited target with utility this expanding and IDEAL the seladelpar We’re of very population. its

call Harish Harish? I’ll over now discuss to the to hand results. our financial

Harish Shantharam

Thank everyone. you, Lewis, and afternoon, good

The one CymaBay. second productive was another for quarter

by highlighted made related year everyone pre-commercial As the for towards regulatory we progress meeting significant and objectives planning. before me, to key our development,

In with the are XX, quarter and terms through finished of operating strong as call the CymaBay’s XXXX. of the In prior plan update we million of investments a line totaling sheet on believe quarter cash that with June financials, equivalents investment fund cash XXXX. with third cash, balance we hand and sufficient to $XXX.X

seladelpar and initial by second transfer will collaboration and $XX companies clinical remains revenue Kaken to the in technology the recognized data obligations. completion to performance million commercialize development with develop XXXX completion CMC upon recognize of million of Off paid the associated of $X.X We be quarter of upfront Kaken in the $XX.X the following payment and delivery Japan. deferred million ongoing license

our research expenses, for for As and Quakers $XX.X million operating XXXX and and were $XX.X June XXXX their and respectively. the million XX, expenses development

to ended the and XXXX the activities. expense costs were June primarily were driven and period studies regulatory support Development for by higher and higher quarters in XXXX than clinical Research our corresponding XX, personnel

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

in pre of million the we’re to $XX.X General now by period and June and Turning initiation ended general and XXXX higher $X.X expenses. were XXXX expenses administrative These affairs were of XXXX review for XX, quarters primarily to commercial medical costs the for XX, XXXX the corresponding respectively. million organization build than administrative and quarters driven briefly and spend ended commercial a planning. June related and and

the increase and ended cent XXXX quarters Overall, the of million loss XXXX March was the quarter for period continue higher an revenue, collaboration pre $X.XX seladelpar $XX.XX our Overall, increase XX, and Net in or development XXXX XXXX in primarily for XX, in due expenses for as future than commercialization million each operating by lower the operating in we was our expect expenses. to plans per respectively. and net to loss offset execute $X.X to partially we one and ended share corresponding June PBC.

to Sujal. hand the me back Let call

Sujal Shah

top focused have that PBC. patients we PBC result at tireless opportunity the thought dedication their of Harish. our you, their team their this support. come I’d look from forward our work to patient improving their caregivers remain sharing It our these like goal this step and related RESPONSE laser community one able towards partnerships to journey our Thank and lives been September. to our with to the for thank for leaders for take and to is of here CymaBay of on efforts. updates The results the We objective investigators, and of to line in end closer before investors

take to Operator? happy questions. now We’re

Operator

Thank the Please of to Rahimi you. Yasmeen Sandler. [Operator go Instructions] Our ahead. line first with Piper question going

YasmeenRahimi

and updates. much on so the Congrats you quick team IDEAL. you. Two questions thank afternoon for for Good

the the I short at directed fall is to current are then squeeze end that. under which And the question study And going so the a commercial many one percentage second top as that X first the PBC ideal data? guys is, calls to know that somewhere The weeks line of have about I couple population? like is, but patients mini investors may lot then -- are a why would you think IDEAL between IDEAL side if especially question into wondering I in that what is before, on one are we X.XX, now time the the spoken of market

Sujal Shah

for just as Phase well around PBC is of ENHANCE to to as in the an is important All Yas. highlight X the in questions, really success. right, for thanks experience think Sujal. start timing with I prior course timing off I’ll back with the respect predicated development IDEAL on This we the dates conducted calls, our work prior of through we’ve that and XXXX to as have me study, expectation our extensive

important sets a be as respect with the to how think for, I also really months expectations planning data experts, to market it’s it’s research very could about additional for we thoughtful meeting highlight many describe in with and to making broader PBC. that for learn treatment conducting around reset might the potential population that from benefits in we treatment patient setting me of

a possible. as think do timing really success I set So to much and with quickly as perspective for has being a the to of our able deliver planning data from

been setting, have disease As the remain evaluated who you with second-line in living we not to-date higher imagine, day. our described at risk believe that patients progression, really a of we might that patients these patients every the

to set knowledge is for collecting we’re forward your excited additional benefits we our and about to on provide to potential may as second around excited off these we more Lewis With So can now as to question, invite just that this X context. patients. for which attention respect population data support and have I’ll to press some maybe that quickly start

launch in submissions and upper UDCA, We third continue we a the twelve normal. have times of suggests that his Lewis continue the is are bucket this entire team of that UDCA. just least U.S. UDCA beyond RESPONSE the actually one data a months treatment limit a that if might I readiness to Post on remain be market and taking almost of patients, as that population X.XX on from those be it patients to research are and of population research that look you we regulatory particularly at at the to amount that that between top line will could could towards imagine planning conduct conduct significant perspective. then as date market of all Early a advance commercial that

we’re Lewis, population. know you I a about to bit of to a if want to this talk don’t get better sense continuing little what do

to experience UDCA, these a treatment complete in earlier We we XX% evaluate comments actually my response. through really. patients of less than continue about How of their as I patients flow mentioned those

So we of in incomplete patients on that of limit upper XX,XXX those course out and of and are patients above to tend and course course described, partial segment Sujal as the into of are responders times then which be these X.XX normal as responders. the

criteria second-line patients. poise XX,XXX to the as course at normal, times of based on about of the limit defined XX,XXX that’s we’ve And market upper X.XX

continue at continue experienced not too. patients population and So those those but liver look from exactly symptom to course will we standpoint both understand are the complete responders this between think We also really of that. to a how health just that of management

partial XX,XXX Of probably to course, about are continue that responders patients there are pruritus. experience

that’s it continue we’re that at how looking to but now, at So we’ll data. look

YasmeenRahimi

could you, or queue the that jump I’ll when And I Thank to will September? then of ask and the data day just back is And team. want saying end thank into of sure be is by coming last it option does to and now everyone just September? me regards response, that Sure. my to day come the questions. timing clear it of end between of for mean make any in two, allowing you the on you’re September,

Lewis Stuart

release the we been end the know, of has QX. data before would you top Yes, line expectation that always

appropriate follow-up visits, and cleaning locking highlight we quality the to out we I want close as importantly, have integrity visits, think data final that database. we and

an as top it expectation that leave the simply in having line September. would sometime will I come results think I

YasmeenRahimi

much luck. and you of so Thank best

Lewis Stuart

Thank you.

Operator

ahead. Raymond Steve Please next is from Seedhouse question go from Our James.

Steve Seedhouse

quarter on think is basis screen these than will back from and this very the just like prior it like your questions. and failures, the rates dynamic. despite your then enroll believe, XXX/XXX I published study? to scratch mean, -- sort Do X.XX you. actually it this Um, IDEAL. of data essentially is patients in taking ALP. and of of two failed identify population. prevalent fact some in for One for Hi. care having you on the any starting of limit Thanks was this given clinical upper so say. easy levels I the patients times I steadily much sense ALP the let’s quarter Thank of and similar pruritus if are screen the have of And range this less and go of in? so in something screened in enrolling Does like you a and Rapidly normal I second it be a data, would think studies over that failure EASL just patients the days some on have of investigators or that population be question or it’s with ALP,

Sujal Shah

for Steve thanks Yeah, that question.

occurred already investigators involved potentially strategy and I patients that IDEAL, screened has would back Go to a patients to think identified to us of would qualify. you’ve to. who be want may course know who’ve who in

that know of we attention course a competing that never you for say a we I I that lever take pull. is no So comment would when comments, experience clinical always upon and based research though it’s take couple say will something would population it this I of a right. you course of trials takes there’s that we look lightly. do, our add it’s

first find of we out to alkaline what study example sponsor. with level where them. this forward might phosphatase This step and accrue the said patients to might want know we’re Is to we a benefit is

forward have to do I So opportunity very. we an think move

must We’re about ready protocol that yet experts I this the idea any then we off as mark to we when of as potential the pretty our the the but Conceived feedback developed not now, say the communicate basically and well because extremely just we’re is from investigators excited consulted first. timelines enthusiastic. and who we’re

for tuned stay So, that.

conclusion those that in can exactly is with was pruritus draw our generally history It’s and -- this a medical answer to it about that I’ve see one we because do to patient saying, methodology with research itching at what is hour I used don’t you in clinical been with less collect of presented poster diary think at a of in mentioned. a greater, EASEL, perspective electronic from look recall terms the pruritus look with common. at it’s that’s and because you of question you study, but In the wouldn’t I the an intensity significant we a XX same Pruritus population. hard can But quite values. just setting is just a really a before, one the we yes, vexed that very want limitations of it’s if

And doesn’t strong can patients in work there’s in fact which phosphatase, the generally bilirubin, if it connection drug find a disease alkaline in it UDCA published for look you’ll not literature, good many levels is lowers you of between that speaking a effect and phosphatase that has and fact activity, affect affect pruritus alkaline a pruritus. with it on

while the disease with is consequence activity. lockstep of disease, travel a level doesn’t pruritus the of it So in

So examine seladelpar, aspect pruritus. appears cholestatic as to this we at interesting markers as least another which have is initially us for well as on effects

Steve Seedhouse

Perfect. Thank you.

Operator

go Please from Cantor Fitzgerald. with next Kristen is question Kluska ahead. Our

Kristen Kluska

quality address life I Thanks your treatments. for major questions. know that symptom taking being are issue burden of prepared my to a about looking you in with everyone. new and Hi, remarks talked physicians really

pruritus, in the huge talk upcoming a what specifically unmet as win So would you given the in need addressing characterize you readout? about here can

Sujal Shah

question, to off happy that Yes, Kristen. answer start and

the treatment only mentioned to PBC. think shown patients in to I recognize first-line important with Chuck it’s been never first existing with as really UDCA, existing reduce treatment, that here pruritus

the of of benefit of acid to itch. of has quality or real element you’ve second-line life only treatment one the worsen course for on improving got as demonstrate existing has quality life yet first-line from U.S. shown in a obeticholic treatment cause been that to And that patients, so, perspective,

it’s we’ve when even XD we patients PBC NRS benefits in at or the cytokine well as seladelpar XX to-date and three reductions the Phase studies of XX points the So of as seladelpar And quite and or itch itch respect seeing with patient on on seladelpar, and saw with pruritus then the we VAS generated four from at been we mg our have the Aisle look that reductions at even placebo. fundamentally the EASL ENHANCE greater seladelpar, looking benefits the whether real. the patients responder totality associated at on questionnaire -- that’s reported course X evidence we pruritus it with itch, of in for correlate inflammatory with with believe scores on to XX scores scale in is from that rates versus and placebo-controlled shared with reductions evidence that those with all reducing

And so suggestive of to win. an think improvement. really to differentiate which our to we don’t but is aspiration a Clearly, we’ve itch the you bit with treatment, when worsen about see second-line or this opportunity existing cause from obviously there’s seen date what seladelpar, is

our significance improvement we’ll is continue statistical so And the measure expectation that see degree see this in in we that to to RESPONSE.

get actually position patients to in And thinking as it the well. key for potentially secondary treating about PBC stronger go to to approval much opportunities regulators seladelpar’s endpoint of course, puts us a in itch

patients there’s an opportunity alternative a a quality has life. opportunity the in think on exists that improve take we to RESPONSE new today, to based of real So first in confirmed that really seladelpar if the time that’s of treatment for really to profile dialogue

Kristen Kluska

about hiring Thank after anticipate you. you additional RESPONSE? talk And what can you plans

Sujal Shah

to regulatory medical specific additional host only us for a commercial for step think we commercial access, say commercial affairs and organization can as company key with to-date data I marketing, continue house affairs again commercial. that remarks, particularly preparing folks preparing within out and field we’ve to started ourselves ultimately Well, post in the in reverts way folks the hires mentioned build see and our the launch same line approval with app now well market we’ve the as post a operations, here both of the within as done internally top some data, internally and by our of both out in prepared also positive up you’ll medical those with result

launch gear if Of closer supportive course Again, roles we’ll here in helping would we’re as the regulatory company. to us later activity well in successful all force prepare in continued RESPONSE but the submissions. a as come ourselves commercial towards field within have from itself for approval timeline perspective,

Kristen Kluska

and the the Okay, all heading thank best into readout. you

Sujal Shah

Kristen. Thank you,

Operator

next Patrick Capital. with is question ahead. LifeSci Our from Dolezal Please go

Patrick Dolezal

for thanks the taking Hi, question.

in any it’s focused. above may more of some Could you of obviously this the Thank you How as guys relates speak it I if to or there? thinking just marketing I’m inform might any there? couple in implications have that support limit to study on so there if success that And kind of a elk like of upper a kind potential more I normal So is design X patients of kind just IDEAL, efforts? And about study past just then you. were may. primary, mean X.XX curious this label study fast helped are were studies the variability that expansion

Sujal Shah

you. you registry question the we studies, Patrick. a data screening phosphatase, distributions we in where that appreciate at I potential effort study. course the label where is the think work that much of we clinical central thank but also ultimately a our thinking. question. for it strategy was the We know accessing not had only efforts, looked very for very of expansion is very would road, down a your oh, Yes, to Initially, Because develop to could our distribution. the done but look worked known at concerted alkaline the internally we in we’ve hard

of not getting concerns some around have So to you of where of range phosphatase lead I imbalances around that astray I we’ve at intending distributed to any alkaline very you screen design, that’s going there are or think And representation registries. that’s got the populations the think something the what kind might you’re is, availability we’re and prevalence comfortable study. and patients in both across the

the intent With label gather to think respect first expansion So just success. up to very feedback as fact And both share We would committees them are course seen. evidence little in this convinced we Then agency. set IRB’s with the what’s really had the submitted to that of that to results way. guess would the question, than is say our data then of other that, we’ve really we’re the ethics we we in very well are for as expect supportive from course community. I just we the effects medical I a see protocol those to and that

sure focus locked we those is The as and interactions cadence stage I in issues ideal high regulatory expansion basically at think the label get of a that now address conversations. at you very on imagine indication point and would around first the this RESPONSE might we our time. right making and later is down

Lewis Stuart

for The treating from and intolerant only label the based responders either inadequate Patrick with to know registration as study are have idea that population indication or part we’re benefit other I’d would thing may serving to patients opportunity on The add UDCA excited be a patient IDEAL category to And RESPONSE of a label. that is the RESPONSE have as for to an support patients course who consideration. this approval and perhaps for think learn an I seladelpar. around broader key you of initial is and in that’s how

Patrick Dolezal

makes appreciate the I sense. Definitely thoughts.

Operator

Please Our next with is question from Harrison ahead. Julian go BTIG.

Julian Harrison

the to On terms of one at to forward Ideal, times limit clinical and like of my curious taking normal second-line see questions looking I’m what of would yet for payers if thank Hi, regarding any really the of patients reimbursement in month. the start therapy? data have for between upper X.XX next response from feedback they and you date you

Sujal Shah

IDEAL, design future. us. about input time potential the how and we bit set very Julian. guidelines from that Chuck data the yet carefully in thinking treatment front the when -- thought think we mentioned, think that’s about don’t the input in question, and we like HCP’s payers. may But influence would this field, benefits Certainly spent in about a a gathering the the good from a leaders population, considerable of actually body work evidence creating yet as That’s in of of support We the have

with think things that front of future. some that I updates on of So of will getting all to continue work in feedback into provide the are these from payers, inclusive us,

Julian Harrison

now pursue then as second-line initially Okay, not there that is are but supported treatment Thanks. by a got and beyond additional have patients for therapy, seeking all these with change at response those RESPONSE? That’s prevailing incomplete why on approved to helpful. it. And yet reason to IDEAL gone not seladelpar could UDCA is

Sujal Shah

question. a good That’s

existing a as first itself improving clinical reasons you cirrhotics of say from of good dictates which of only we’ve studied HCP’s normal treatment patients non or key of with again, And parameters, market alkaline some likely variety lipid patients meaningful that degree research, that upper the exist think of think, early using the it’s different the be -- composite primary bring time the limit some of a to we’ve very I about if response course burden here proportion meaningful to all patients patients below should safety successful of as treatment. go I would profile to-date. cirrhotics as over actually to both the current for we’re across I phosphatase, times X.XX are pruritus which think rate second-line believe symptom willing a even fully and second-line to-date element good on the to normalizing again our on well compensated based on seen endpoint seladelpar to-date

believe. are overall second-line to be When profile treatment certainly today yet treatment. moving you think that on There’ll not about that we more second-line patients

excited additional that And considered hesitant those been perhaps of so or about alternatives been second line patients haven’t for yet. perhaps have having for treatment as

Julian Harrison

Okay very much. great. Thanks

Operator

next with Olson Jay Please from Oppenheimer. is ahead. Our go question

Jay Olson

with you that could talk X.XX? above limit the and And patients the normal? Hi, normalization the in of of questions. and between seen to about taking you’ve the upper rate on of the for extrapolate you progress normalization potentially population thank we with ENHANCE seladelpar for trial rate how congrats ALP ALP a patients with of one Can X.XX,

Sujal Shah

I’ll the question. at ENHANCE you Jay, highlight thank and least Well, maybe for additional here some give commentary.

patients you’ve upper are highlighted. population across mg, three the XX started of each times of units X.XX course normal The that saw ALP. arm. as enrolled patients around you was limit about the I of it we in months, data Again seen ENHANCE with baseline XXX liter per at as these XX% above normalize ENHANCE think in

of in months, that a bit this to normalized striking I be has the But around X course take and three baseline in obviously despite our for largely we patients to when from for you see the you are and published out think population, enrolled So placebo potential difficult of into these will that that roughly X.XX phosphatase think And ultimately range. That’s XX I above that patients normal the compare times we’ve phospholine us to these ideal at we’re and in to extrapolate patients here our literature data who X think that in the I for influence. limit the normal. back is shared are becomes of normal the guide at at alkaline below upper bring reductions the remember, percent about months being UDCA as these enrolling baseline. levels least fully twelve we -- of lower on levels parameter are

seladelpar expect So this I been versus see we’d dictate we’ve reductions with seen ultimately of to terms because see of that’s placebo, continue And a will dependent data XX% patient potentially that set with think measure to baseline what in we it’s ALP. on think in IDEAL. population these what I out not the

Charles McWherter

Yes.

X.Xx is just this X.XXx -- would say is thanks course, normal. if fact the upper That’s that -- per in and normal, below liter reference of upper a the our you it some normal. the encouraging is But response, the to Maybe the Not it’s if units of imputed as course, question. of -- you’re take wouldn’t XXX are normalized. take and you patients Chuck, a limit expect -- know add that Jay, average baselines are at of is I just right that’s sets kind limit you average, be for going an top to to actually Just, you’re I’ll just if actually range the going range, limited drop you which that’s of reduction a that average upper around they’re you end an at get to the guess, on distribution non-responders. to XX% out, get of XX%

Jay Olson

Okay, great. with who couple what could. on I of if be? And of a are based off-label ] the X the than patients know X.XXx [ if Do research? I and response super between if treatment you And receiving sort would market currently follow-ups, helpful. normal so, your That’s had zipper are OCA

Sujal Shah

another believe are. these Jay. Yes. really We good It’s question, don’t patients

very a we patients. In what of not fact, off-label is particularly therapy, believe proportion the significant U.S. in

UDCA And so I their wherever are maintained as the effectively. patients that alternative they think these just best left those and levels fundamentally, are are at ALP lands, on

population. this this perhaps what’s exciting additional to demonstrate patient about So some is benefit opportunity for an

Jay Olson

over you in just ASSURE Okay. the study? if And I what could into Can more question. of talk about sneak Great. in rolled percent one have patients RESPONSE

Sujal Shah

Yes.

XX% actually long-term when across studies numbers specific roll prior over provided our we’ve the seen significant But haven’t proportion long-term historically. patients we’ve Jay. of ongoing had into extensions We there, extension historically,

studies, nearing So now came which RESPONSE. ASSURE of rolled many has as over now which have is into over we’ve prior course, completion, mentioned, of but in has many from XXX of fact, patients RESPONSE

from a it’s high experience just number, So clinical date. on based to the patient studies our

Jay Olson

Thank I much. questions. you so Great. appreciate the taking you

Sujal Shah

you. Thank

Operator

Our ahead. next William go [indiscernible] question from Blair. Please is with

Unidentified Analyst

our questions. Thanks for taking

of quick ones. So a just couple

well? IDEAL longer-term eventually follow-up regarding ASSURE study, the over to basically, these patients any rolled as For or plans be will

Charles McWherter

Yes.

put But the in the out. wouldn’t ASSURE. beginning roll over into that I patients haven’t we’re we we guess study, currently, roll an So at place opportunity of and for those to necessarily

commercial. then into out, up would things And transition an option potentially be how it could they Another extension on open to depending play of even IDEAL.

have to So we’ll see.

We’re good I now. an just a for just yet. but question, at start have don’t It’s the answer you

Unidentified Analyst

this opportunities, we Got it. combination forward, perhaps hear That’s on since fair. your curious like rates, triplet of about going expansion theme potentially there? are with to thoughts X a just therapies maybe happy on And thoughts your

Sujal Shah

Yes.

number forward think in I paths the we think about treatment there PBC. are a as landscape for of treatment broader

second-line a that even elevated if and have Of [indiscernible]. require be a ultimately after may highly a successful, we’re who out say patients there some Seladelpar will third agent agent UDCA, course, as

additional that got as team So think we any to haven’t management Andy, you cycle hopefully, tell about think we’ve life but continues RESPONSE. specific studies post or success, yet committed can a there, thoughtfully I we strategies to about

Unidentified Analyst

have sure, And can’t basically Got months? be it. months, IDEAL, on Got to it patients is right? So just requirement three it. make XX to can’t want so six I the be would to be months, have XX it months, be UDCA for for

Charles McWherter

That’s right.

that, as a something enroll intolerant to course, of UDCA. traditionally We want more couldn’t not practice. line We’re be treatment. would that be mention that’s standard second to I population the following can a they being though, criticized

take So and X had between phosphatase not be they’ve would X.XX, simply levels before tried UDCA, tolerate can’t eligible. they also but it to if alkaline they of and able it

Unidentified Analyst

so helpful. very Thank that’s you it, much. Got

Charles McWherter

Thank you.

Operator

go Our Arce next Ed question Please from with ahead. and Company. H.C. Wainwright is

Ed Arce

Great. Thanks for on taking progress study. congrats my the questions with \ continued and

label I that know mentioned IDEAL, is to goal I Regarding though, wanted be ask also on which with would of pruritus data collected generated and if and your a and of label follow-up. two expansion you Obviously, quality this regarding you’ve to a call there. life new trials. then a on Yes, on have already. know measures further you expansion clearly I I this consider reason fatigue, data

Sujal Shah

of Yes. as well. a I ours focus absolutely say certainly that’s

Seladelpar’s of we we we to both effects data invest well continue the to that hope RESPONSE, as we particularly life to around on As overall. continue gather will as data PBC-XX some as that of and These from these be symptoms in areas fatigue. of better generate pruritus on quality understand questionnaire disease

Ed Arce

that you that. some data including supporting had data, of important it mechanistic with initially label. the and Great. having sense you rationale to as the of the some additional this would IL-XX how wanted have future to ask a the pruritus, at be from Rationale have regard part of to of that I least agency underlying And now if and on with inclusion

Charles McWherter

and you scientific which think a it. And, been don’t how speak understood take IL-XX are intensely about the to Hopefully, not think they to is Well, wouldn’t know of interested is thank we’re for that. they mechanism and for pruritus mechanism, going regulators. pretty course, it, of want about mentioning perspective. cholestatic I Yes. about for really very I decades. thinking the

about PBC, you only I nothing think for patient subjective, symptom. it’s different for many measure only as but can not pruritus, can you tell course, a that, diseases of that the

intriguing. could a that considered be something to potentially moving biomarker is Now really

look experiencing pruritus patient, the measure, up is of I what be that I just measured people having as can point. if be can FDA appropriate a can to we going at? ways to I well day, start starting think would question, that you line is analysis something the really coming methodology very used data that the also a And that that with your something were kind they’re of would we what’s think I of have key something patient-reported data be also them, going respond, now the end quite fluctuates -- I measure not I I’m think, that the to with At we validated. what’s a confirms something or only I how interesting. sensitivity know suggest something to that outcome, to have supports robust? that the but I it. independent Do negotiated bit, a with additional there’s have quite pruritus measure feel

I something increased community least think will the wait what they’re think some have laboratory else that’s or to we’ll and just I going do, to see. having biochemically patient-reported But medical a outcome. focus related, on confidence have let at first it’s because regulators that

Ed Arce

to realize qualitatively just Great. I if describe had your could to I Harish, year sense of think one, you this one in remarks last degree overall this any and build-out prepared preparations, an and mentioned expectation but subjectively next then is the or launch the to increase. increase I Right. related OpEx is And all of there commercial into for Thanks. for year.

Charles McWherter

leaders other infrastructure in think closer some on bring in the medical are our the we’re side which commercial commercial lift the to for on of Ed had more mostly Sujal MSLs in say, the the bring in on We’ve only mean, as side the key I the our and -- on when field and the will thing would I accelerate. about, main, next we’ll talked bringing added then I and then would -- add, data we as year both will well will which the teams, be approval. be commercial now, side will I think I building

level we’re support best and support beyond sure possible any needed that, goal to be, the guidance resources of in infrastructure not launch. support having to specific So what to we’re that have quantitative I would we’re required that’s I’m make give the have the sure to on is the making spend but that’s and think launch position the we to that a

Sujal Shah

Ed, I to cash think think what’s next there, takes as having you us QX with least that can end guidance at runway estimates. year, of you helpful about the some that the help of

Ed Arce

thank Great, you.

Operator

Our go is Thomas with question ahead. Please from Smith next Leerink Partners.

Thomas Smith

progress. afternoon. my Hey me Thanks taking the the guys. questions add congrats for let all Good on and

to what guess, explicitly the agent clinician I that’s of seem to within Just just able you’ve follow-up been based your the like versus that able claim on elafibranor will Ipsen I’m an a market recent top seen whether on benefit be you research, from explicit line, doesn’t wondering, we’ve it there. quantify all pruritus. labeled toward that prescribing labeled, trend like preference has benefit a have agent having data equal, all for things on able with label? been incremental of improvement quantify an isn’t benefit, being the to an plane but explicitly the suggesting pruritus a

Lewis Stuart

utilization very increase I we a qualitatively, specific that you, expectations significant and the that give can have there’s don’t question a but based pruritus impact see can having overall claim. on We for no tell you there’s I in

burden that around aren’t those. not tools really that with of really providers fact to that by patients just symptom think And have us, had really how the address I tell so expectations their couple addressed providers. succinctly the being course, the

only and will and coming that to want been treatment, disease. that this setting, think -- their have paradigm played patients the interested about not And I new you to but we course throughout as there really, address in providers utilize with for be in to are issue they they’ve the think wanting that Seladelpar that able certainly be saying, to going of a

an that for we strong qualitatively preference extremely provides agent I is what that benefit. think So see an

Thomas Smith

NDA helpful. for on and elaborate undertake the the try that was submission of you bit line between now some to close just the things wondering to data time a of That’s to pre-NDA can kind of it. I then help top if you from could And the activities, sort Got little maybe submission? to preparation prepare try gap

Charles McWherter

question, It’s Thomas. great a

and situation manufacturing. our that doing alluding to. that is in are of a the be what successful are of lot to things lot progress wants complete space. anybody There exactly You nonclinical made in industry our in who imagine We’ve you’re a can

We studies completed that closed are have out. clinical prior and

we can assemble assembled written have studies that approaching ongoing information to We into lot has which of documentation a for all that have completion be consistent. made the we are QC

those as of our around in All being most built teams are We the activity really important place. this efforts the company. in

have expertise, the We have us and additional succeed. supported consultants who experience in like space with that up that this set can to

you So I of the ready as to what is that really think I this as to what you’re drop everyone advance the work, prepare do you in situation. speak. you can in, you’re And as getting in so that’s think at much data so much that does

Thomas Smith

Thanks Got the taking question. it. helpful. That’s again for

Operator

B. Mamtani Mayank Our next Please is question Securities. with ahead. from Riley go

Mayank Mamtani

for our good questions and team. the taking Hi on afternoon Thanks congrats progress.

Harish team see to great the I’m So join here.

even an that being your broad of is spectrum, the the of sort trials complete in in and comment, know also the for going is Chuck, talked and about. EASL biochemical response ad just maybe at top runway to endpoint, I you solidify down. in mind is if which obviously population, cirrhosis outcomes, I IDEAL, that past guys the Any board you do on I’m maybe you about big obviously, are that setting, [indiscernible] that in liver beyond get IDEAL very study And Harish, label. trials about about work So talked if that guidance. you’ve crossover in additional of pruritus sure what looks runway could, any talked as

follow-up. love a on that. your thoughts quick have would hear So to I And

Harish Shantharam

That’s with the about, I which will that is key IDEAL something Mayank, have we start enrolled as expenses, we about recruit the on course, We in let is spoke patients R&D -- that’s me patients. ASSURE, of listed on you talk over trial ones some there. besides first clinical right? expand think this study that long-term as question and study. the where XXX of actually the we currently this

we’ll study and outcome of have NDA part is we And our be is this filing. initiating -- that then other will the one the

we to up. we ramp that have and see so need that enrolling So activated will

I are lens So hit from three a out those clinical from and spend trials we that would it ones trial main an say that there. would main [indiscernible] would are -- the the it perspective those

the other know to the don’t part question? you of I if Chuck, want add on

Charles McWherter

Yes.

But you’ve others, very or thanks seen as recent The from So normalization total well appealing biochemical as parameters normalization of the question, biochemical us it validation the any something you same the receive course, that’s it’s are of both not regulators the that for idea. from not about. presentations, call of as means. there is and level it’s is talked to by actually wanted true certainly if a remission,

the true Registry to PBC the of from Group various that And you about Registry, the a elevated global the work group the not In linked really particular, measuring been coming Are how much weeds, is what just parameters know from without key that we bilirubin and independent injury, they travel our the really ALT, to I some inflammation the community to study these powerful of too -- understand often debate that there, think fibrosis. don’t thing. liver to What are of of ones as well transaminases just especially the think quantitatively, in far phosphatase And the Dutch do Italian issue some the reflects the covariants or out but ongoing PBC we’re move one. they together? work extent that same getting medical whether from is terms will beginning alkaline as appealing I it’s U.K. yet National there’s happened. concept the has qualitatively, into from relates

PBC but also like with NASH, histological diseases follow-up. in liver evidence other In using long-term

accepted ALP strongest X, of the We to to bilirubin produce is those data the outcome still suggest have are Clearly, sets parameter. believe already evidence near-term also important medical we’re believe, are community. ALT the they going to by going one at the at degree look least, needs to an studies. that and most I get from we although regulators, So that level embrace, think, a would

a biochemical Fibroscan or attention also Beyond transient mention normalization, like noninvasive of would measures moving elastography. I think using I that is liver lot stiffness towards

lot merit. think has I a that of

an It’s of work clinical for of [indiscernible] other terms follow is and by say, Some used and our it I medical important as I judgments being long-term diseases, used, of potentially studies. the practice. serial suggesting really include is towards used to the this use follow we lead out progressing, to patients. in clinicians PBC Many around cirrhosis. is the in to make But in be show like to be coming this -- should is being long-term how going going this patients way patient’s measure in Christoph importantly, actually, up especially research will think disease decisions are

Mayank Mamtani

there? the another data, score, what are questions about then NRS study readout, asked, but seeing if have appreciate And color comment expectation not for response I And full you your as helpful. you’re just expecting comprehensive discontinuation we obviously Very know, on you there. RESPONSE, seen rate? on after could the on placebo the also

Sujal Shah

less rates XX%. discontinuation seen I that think rates, respect than to Thank you, we’ve historically, been have Mayank. With

be continue X-year patient to ENHANCE our study. RESPONSE. our that would that study And in had Phase II we XXX anchor on So significant even though with of experience, prior patients obviously, our terminated Again, north randomizing from XXX early. expectation numbers very into been patients

So we I the around discontinuation. think see similar expectation we continue that have to very expectations

to I think itself. data have all is placebo the with in NRS, again, ENHANCE respect With to we respect

will with saw point NRS data best an and the as we patients. into that’s We the or was have, today that where we here more drop greater the baseline have placebo treatment obviously, in only NRS expectation well and And came the X.X. placebo our study number in as this X so of arm, arms with the set significant patients of X.X

XX% expect see response coming as X least or to to NRS roughly at with into continue of greater patients an We of our well.

our that’s date. historical we experience. So the It’s really that data have point only to

Mayank Mamtani

the taking Thank our Sujal. and you for set. appreciate questions I best the data you, all

Sujal Shah

you. Thank

Operator

Our from ahead. Olson next Please question is a Jay go Oppenheimer. follow-up with

Jay Olson

for followup. taking the thanks Oh

is to PBC like early treating increased IDEAL aggressively, study step the the one treatment. on and it seems closer line emphasis With

you Seladelpar latest on thoughts please your for in the pursuit of if first-line wondering indication PBC? share the just could So

Charles McWherter

Well, thanks, Jay.

think, do be bit I further sentiment I think is treat we the would we to kind to hold earlier, Whether treat a to frontline, us. little of symptoms. treat actually normal, the away from

to to data population one how additional a Our a getting this trial. to current trial second focus comparator therapy might line upon and going in trying an need specific the was has be It for trial a would very need be based is really it frontline for get UDCA approved indication outcomes. implications would because conduct potentially outcome

So there’s a lot of different implications.

beneficial confirm enormously the RESPONSE. to an be in can Seladelpar think I we patients profile patient if

positioning availability. want terms its that an in of therapy. now least -- has the easy it to to But to think it’s future we’re wouldn’t expectation a I bright move frontline really at I that it’s reach right set

Jay Olson

great. you followup. the much taking Thank so for Okay

Charles McWherter

Thank you.

Operator

we Shah, call further you. Mr. I’ll to the this have And questions no at back time. turn

continue may You your presentation with or closing remarks.

Sujal Shah

a Thank you, close just I’ll today few with thoughts. operator.

thought discussed the ago. our the at work many advocacy study input market today, patient started included months of leaders on IDEAL, on this world. gathering And teams design by and and around merits First, important CymaBay research, we’ve from it groups as

been patients new with excited, we’ve ways this in novel alternatives date. PBC as to many for many who We’re to to treatment discussed study neglected have bring

learn treatment we Seladelpar broader collect from opportunity forward for believe opportunity from may we’re to to reset have the the to that expectations have an patient data we may look potential if population We an to a that successful. benefit IDEAL

Over and weeks, progress proud activities. intensely course, the cleaning across been on focused our the company on about rolling of making and we’re Work the and teams coming as in patients preparatory continuing database, has all clinical RESPONSE we’re are the in ASSURE final and across regulatory accelerating, into eligible functions many well as visits only other conduct many locking fronts.

event in patients are you. before you part a with significant which been and hope being journey the up the not of front soon. open PBC, Finally, this of We Thank talking of have in end RESPONSE. work. to new of our for again chapter results treatment back forward we’re may hope may study we about excited the again from top least this you all that of look the be patients September from to line benefit with that our We can a

Operator

please participation. does conference for that today. you a the lines. all day, you your We great that I And conclude ask Have your everyone. for call thank disconnect kindly

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