CBAY - Earnings call transcript Q1 2022 | Cymabay Therapeutics Inc

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Operator

Good day, ladies and gentlemen and welcome to the CymaBay’s First Quarter 2022 Financial Results and Business Update Conference Call. [Operator Instructions] Please be advised that the call will be recorded at the company’s request. It is also being webcast live on the Investors section at the CymaBay website at www.cymabay.com.

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

Paul Quinlan

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

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

Dan on and call Executive the Finance. me are Sujal VP, Joining Chief Shah, today Officer; Menold,

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

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

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

Sujal Shah

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

X attention PBC. than call, will key less patients or bring year biliary our As months of today it since the unwavering in milestones brief this to to our journey in half end is cholangitis, primary updates with turn XXXX seladelpar to be second we our our as year

investigational for for for patients were data ongoing we with by studies; Our long-term potential diseases a seladelpar open-label with the PBC shared Journal our preeminent comprehensive have differentiated development from visibility extensive drug Hepatology. marketing of efficacy Phase Results continued address and for NDA-enabling to XX-week with clinical We elevates world’s in the study believe this in needs these PBC dosed to from extensive our in safety drug RESPONSE, Phase support gained is seladelpar PK to approval. for These includes program program safety ASSURE, any experience our Phase X in program Phase we patient PBC. a X database; prior the the from study and safety draw treatment X development where development, most and for PBC. second PBC. currently of the seladelpar’s studies complete as seladelpar patients unmet have patients in candidate to faced X journals the medical the Phase study of to featured many XXX of data support previously The opportunity published liver one a last month over X

with to We X needs PBC. believe address important potential the as unmet continue patients seladelpar to for

PBC. We and believe tests aspiration be First, an normalization of phosphatase, that include therapy opportunity that liver responses patients of incomplete should improved poor in have disease, the their been as many activity for in and response patients biochemical levels and transaminases, cholestasis commonly and liver the against injury. exhibited need the biomarkers These alkaline therapies tests biochemical observed to associated for improved with bilirubin outcomes histological the in PBC. patients especially progression achieve offering with lab with

Second, many new patients’ symptom this associated only therapies to addressed therapy that and acceptability including symptoms, relief can have patients of exacerbation approved lives, Currently would the provide approved of onset PBC and or therapy. a from not been burden acid potentially adherence need with has pruritus. pruritus, significant second-line suffer with improving treatment obeticholic improve

patients data portal of of spectrum medical PBC spectrum ENHANCE call, treatment from option to who including have those PBC have past compensated an presented inadequate motivate data leading acid. that RESPONSE, study we first-line our their excite registration X and stage Lastly, our clinical our across and this non-cirrhotic compensated be X our are Phase stages last patients These we for without Central disease. in XXX a to or and development ongoing steady investigators our prior at meetings to with patients encompass Since had study with is treatment ranging seladelpar response second cirrhotic PBC enroll of Phase along to to studies. have targeted from cirrhosis, to ursodeoxycholic should in progress towards continue those global disease enrollment. intolerant completing clinical cirrhosis safe program to made participate A with hypertension.

However, resource of of impact the have for our as and on A emerged been and activities subsided easing clinical Timelines COVID-XX by at globally. our line then clinical pandemic also enrollment Ukraine Russia. sites and of affecting RESPONSE. have cycle continued suspension enrollment been projected issues enrollment variants for halting at sites restrictions further in in primary time lingering factors screening affected to and implementing have an

of initiatives months, increased despite has investigators, countries. activated to additional global ongoing in sites. XX the at included progress consistent now of patient efforts This and sites over in-person the have few We past U.S., investigator activation investigators challenges. have sites, Our resulted visits with their over additional delay and European XXX for meetings referral American mitigate across implementation Latin

in our development and to monthly experience ability globally. enrollment in have be confident we clinical forecast into and our remain with third final the execute visibility we the seladelpar quarter. We As given what occurring PBC now of completion we operational months approach greater project metrics to screening, of in extensive

continue Importantly, ASSURE seladelpar clinical the we expand to study. experience the open-label in with long-term

into ASSURE. with XXX with seladelpar patients taking study patients in Together that now This are to prior patients this approximately RESPONSE there have completing We entered ASSURE seladelpar, daily. begun level roll from and underscores into studies in of and approval long-term in seladelpar. durability of commitment on to marketing this of investment seeking success collect We safety fundamental efficacy data our the believe seladelpar. our is to eventually over

efforts, we that meetings prior of remainder data began XXXX. addition our PBC in been development with datasets has to analyses provided Through at rich of medical to fueling and GI feature the us be the clinical abstracts are seladelpar upcoming since Digestive In featured will in our XX for at the from In three have X hepatology featured major at patients congresses. Diego seladelpar Disease May ongoing excited experience opportunities and San held to this year, presented XXth. about Week annual weeks, multiple we in

be Two predicts an prognosis promise associated transplant-free The plenary score earlier Toronto. seladelpar in the with in entitled The an for first the the presentation also Bettina Primary University of additional was treatment disease. poster oral view is supports emerging will Biliary oral with the and Seladelpar that long-term Professor made. of presentations session Cholangitis Patients of survival, regarding presentation and by with Treatment X intervention improved Presidential GLOBE PBC improves Hansen improved supports from years

PBC Wayne and in of Division efficacy Professor The Gordon, Mr. Health of of safety State Hepatology at of Ford patients of first Phase liver compensated Henry at a Research and University and Director seladelpar the System, data pooled Medicine by X the and GI describes cirrhosis. X analysis Stuart the on with

patients the We forward engagement obeticholic with at ongoing presentation for seladelpar Center PBC. by Assistant to PBC Liver these the treatment of second or of acid with prior safety The Dr. had seladelpar many key further Guam presentations Professor experts look of in describes analysis work University efficacy in supporting fibrates. an have Hussain, Disease, Elia with and of the who our and Toronto

of Before a review has Last patient Research studies, and by including diabetes. the an glucagon on and The second me X X diabetes. clinical current rights is study the in let with to retains first evaluate to blood subjects release technique and concept to AdventHealth agonist type conditions clinical The Florida, Harry the in Type Charitable MBX-XXXX is Trust. Leona study by It in clamp pre-diabetes being funded GPRXXX ask by X a CymaBay our of prevent is Orlando, with financials, Dan year, completed as under for fully in a or MBX-XXXX product hypoglycemic M. potentially discovered a I in being B. agent AdventHealth previous MBX-XXXX X-period low of conducted and program. Helmsley patients all investigated is Translational levels hypoglycemia using MBX-XXXX. update Institute developed minimize CymaBay. crossover to sugar. dosing counter-regulatory our initiated pharmacology study brief provide quarter

area In for This to addition is up tolerability, glucagon XX-day guide endpoints the primary placebo to to under with diabetes. whether and will and for associated safety on to the These XX our release study maximal pursue results period. curve MBX-XXXX treatment development further decision hypoglycemia are targeted enroll participants. versus glucagon

in have projecting months. these end enrollment are the We if prior to coming data to progress in recent continues year

we year activities industry as of financial well opportunities line We environment our biotech as value. several market years. gives over RESPONSE managing with the current seen believe and weather results our last today. allows expenses market The clinical the for to balance us in report industry that to we quarter, where near-term in our significant us pressures for Turning attention as those to data have we create we first the raised facing been focusing positioned our our diligent from advance have have seladelpar global the a remained to top challenging and activities XXXX. well capital are The sheet global we as has

Dan Before first to our financials. taking I’ll questions, ask Dan? quarter review

Dan Menold

you, Thank Sujal.

and our objectives. our has clinical additional and necessary of RESPONSE, the PBC. activities we our enroll made we conduct patients in studies in PBC our Sujal development and late-stage XXXX, development, of to complete to continue in development are on PBC quarter plan NDA-enabling progress first clinical clinical ongoing that As required efforts advances other seladelpar other In to highlighted, ASSURE in treat to make

$XX.X PBC. and as quarter additional well to plan we received also funding million Abingworth for the was medical I of potential first equivalents first our on efforts in quarter financing launch XXXX, in investments is of XXXX. and this review with $XX.X per PBC late-stage additional development our driven quarter and where began our interest associated from XX, seladelpar trial with the operating million of Net operating XX, a of by clinical of cash, compared our ASSURE, XXXX clinical available increase agreement, expansion seladelpar to in loss under activation, $XXX.X or personnel ended hand, progress as RESPONSE together ended now site operating higher to compared related cash made We plan We of of and in turn or in hiring of to early-stage as the in $XX $X.XX through our to manufacturing March loss XXXX. the believe Abingworth sufficient associated capital increase ended other the for were loss third commercial, in activities as March committed an results. global well current associated included in million million $X.XX affairs corresponding XXXX, Abingworth cash net totaled will per XXXX. largely March share PBC development in us future of cost development Overall, tranche fund brief support in financing the expense a to of a with in additional enrollment, primarily was with trials the due accretion particular, arrangement. XX, employee trial as higher XX, increases the the to patient ongoing March quarter compensation development the activities to share quarter an two to XXXX PBC In and clinical active period Net late-stage plan. an

expect development, plans We continue commercial manufacturing in increase clinical execute readiness our operating to on and we PBC. our the future as for expenses to

hand now me back the Sujal. call to Let

Sujal Shah

road long we as ever committed to a PBC, process are been to bringing as patients seladelpar has back and through completion. we It to seeing towards Dan. you, Thank with the been have

pandemic global pressure teams environment Our a has that industry. our a entire put tirelessly have of market worked against backdrop the on and

seen exist significant control, differentiated believe months, create believe relative facing this on potential and one today. patients our a which to remains we options in positioned Seladelpar care of area PBC Our have PBC well stakeholders. has unencumbered. today. underscores that fully strategic significant We things has believe can only we managing to patients In is that to few seladelpar patients been the focus is has way us we value interest the for recent that unmet opportunities very in for advance needs we the the in late-stage that now

in out look to highlight opportunities we close expect important to medical meetings As to multiple upcoming enrollment this year. at have datasets QX, in RESPONSE throughout we

an host world. of to to highlight our positioning advance the plan also the Day Investor seladelpar year We before vision to around PBC patients care for end

are Operator? We now to happy questions. take

Operator

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

Unidentified Analyst

This is for our taking Hi. you Yas. Emma Thank on for questions.

We have three you. for

trigger? interest what you total partnering is the DDW us Could kindly and us junction? some you percent are including think will What XQ ‘XX? please we confident new presenting you seladelpar finishing on you you. certain Plenary how are walk of M&A enrolled in if us congrats on provide presentations, pull not there this it Second, aspects this at are we that X RESPONSE yet on are securing third, seen dataset? junction the new Thank most session, at First, enrollment through PBC. commentary appropriate that could you be kindly you the of seladelpar of have Presidential are about And the getting data could by any you to patients significance fully tell

Sujal Shah

questions, the ask presentations Emma. the will first Chuck on DDW. additional at provide to some answer context and I Appreciate will the I two

advancing mentioned patients and pandemic for been PBC to with in we folks face competition deal know, everything So, last patients. year, on have over and focused I XXX% the in the pressures I our additional think we seladelpar as done have the for remarks, control the most effectively as relative that to with

different As and the geographies mentioned, challenges in one time. been periods waning the of at in of biggest restrictions waxing various has

is, now over the very last seeing the consistent and started we’ve progress. months, Now positive few steady

we in predict April So over last it completion you the a occurred than I’ll patient had enrollment in the gives us last of randomized greater to remind ability year. first now that RESPONSE of previously year.

we’re study. broad not So of into crescendo the as number in little a have activated a reach in RESPONSE first program typical as the months a across as XX from we It’s coming we over the of a patient XX sites and to not meaningful get the by increase atypical some in around that see to a And countries mass challenges start to the of of seeing world acceleration you of enrollment. critical to the start an absent presented what’s sites. as pandemic is number

the the towards the acceleration, I that’s backdrop think things the Harder enrollment. pandemic. consistent started of been that’s steady I of different to one enrollment. months project gives of several ability consistent, see as very see the to That us completion we’ve but to of with mentioned, now

We’ve since prior again, which this experiencing indication in sites these study. with Now before. projections, but relationships been is X are enrolled globally. of we development seladelpar an have a fully Phase done strong XXXX, We’ve this

need this guide and towards again, as need we complete visibility towards the how around that the and the ago. we had percentage completing and are execution months Emma, study in course third comes greater in I in the we even to and much enrollment. provide enrollment We visibility With given in to line to necessary don’t we comments than a tend, the the confident today take third think I view coming have what’s to do several just of over a really where the really confidence the vision greater time view quarter, executing enrollment would enrollment this quite completing months. in we’re do quarter. So But any around to what we

and in months, last M&A the Obviously, the unencumbered PBC. talked interest remarks, partnering prepared well. believe, X seen you the bit space as assets in in late-stage a this mentioned transactions in Now seladelpar, one again in of really I is separate as we’ve only two we PBC And little the about space. fully

and not been provide Europe. and both it we also seladelpar, opportunity as extensive but will U.S. the as program just the development our in to us believe with program in Now inclusive of has RESPONSE the ASSURE register

PBC, the a as interest of ultimately have and greatest can. patients alternative ultimately always And differentiated we patients the as that to it’s meaningful I of creates seladelpar profile PBC we’ve putting position, ultimately hands to the possibly certainly we of sheet the as very possible. principle as the space, in balance But to the the globally ours as of seen in so And hands globally in an on on of seladelpar through execute strong And the seladelpar to program. amount and are in value. I strategic the wherewithal completion strategic think we a many highly capitalize the in of execute as mentioned, interest to many evaluate put de-risked plan sitting

And that to So opportunities frankly, But of globally. really to patients have excited, I certainly, those seladelpar continue our discussions types partner us, would an say again, this discussions remain patients simply U.S. parties. ongoing. vision we’re the bring it with seladelpar very potentially global various is to We about to for ongoing to strategy bring throughout we will evaluate to within so the important

Unidentified Analyst

may about you much. that last very DDW you’ll the of presentation, of new not data the And presenting aspects new could kindly dataset? be tell if you the there one yet seladelpar Thank and any is the us have significance that the seen we

Charles McWherter

the here respect very, to very do DDW. Yes, for this are excited plenary question. to what I I’d for get And happy to have be presidential section. embargo presenting selected take – the that is Chuck. Yes, we we’re

see of are related are just the take but not going to your to can continuous and interesting. identify risk alkaline I using that platelet there to I comprised be is what at that GLOBE bilirubin and five now score interest hope score, which quite those factors: tell age, to progression I in a known we’re risk being levels, limitations – be And can to validated basically albumin, to for the is but progression. used it’s of prognostic – phosphatase This tool count this that go treatment levels, risk really would I factors system something do all is So of think you, look response.

patients event-free in so improvement seladelpar. that the data is is story score with GLOBE in survival the those that suggest, changes in X that been for suggests, And years there the an presentation treated

Why score used is While available, the And in which variable, continuous online has it the so it’s risk, in and more it’s different sense what’s that’s the type basically a that important? the this GLOBE regulatory approval, for scientific numbers been here? sense. it’s is an different tool also physician and patient’s than get makes prediction a for can validated,

If it the level is of much that you patient from sense to regulatory the X.XX alkaline of whose amount, change doesn’t very and phosphatase change been have a risk. close really is there that a small make a they threshold in

On hand, would variable. the continuous the a other GLOBE be score

change, in that improvement have If a are their outlook that had you’ve they an large but outcome. they below establish you X.XX, get really not and their

of will score, continuous And regulatory it so future be into for endpoint while the research. likely we believe clinical medical part this GLOBE may will on score system, we think we to anchor that the practice, incorporated and become continue approval,

going attend the gets some some session population in analysis think I might where that hopefully to paving for think patient it us of that see be at insights give if seladelpar you’re And to way So aspects to market. we’re there. looked able and those real different once audience directed the into the I I think the you’re really the can,

Unidentified Analyst

much. very Thank you

Operator

Thank you.

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

Steven Seedhouse

what the basically for $XXX Sujal, you Ocaliva on interest Good about speaking how with depending being I’ve about sold through. sort saw Thank strategic and in million milestones of coming more somewhere past ex are taking you just given between and $XXX you’re the you interesting U.S. million, that it’s rights than thinking afternoon. question. and heard pretty we

thinking think and you wanted about ground, how with CymaBay? I just that opportunity way what ex and that do on in ask for to value best peg price? the of U.S. to And So the capitalize are that you

Sujal Shah

Yes, I appreciate the question, Steve.

and as development, progress anchored carry our not seladelpar, U.S. think ability development certainly the by to and for our own just we’ve we prepare research forward we’re opportunities will, the with to continued program needs see, together in putting in more patient you that excited a, potential but complete seladelpar unmet I to number clearly, this for deliver a look in as really registration, will as in and we’re to population. the to today consistent the I upon program. year, of if a expand ourselves we half we well work, market for development to But as really more commercial success of But do about the patients mentioned, significant opportunity bit solution, even we’re second the an strategy forward share as Europe. meet mentioned, that a as of addressable to

to seladelpar believe we opportunity an again little This think I other something sale patient that that a to population believe is expand U.S. really to over specifically U.S. the to mentioned transactions, you Advent on. outside with transaction million rights we’ve think we what an obeticholic here, is underscores capitalize outside I for absolutely for acid of addressable $XXX of the really what Pharma, some opportunity the be the seen in

priority as to an we’ve alternatives. progressed, a evaluate different for important it’s so And us host of strategic been always

us more the I’d in for us fact capitalizing I think say a And as that the as on simply right that position a we it forward. positions so, about, talked about really bit move to we’ve said, alternatives

Steven Seedhouse

expanded It to little like more adding what already Alright. of it I was questions. wanted noticed entry on just XX, but say, of to it more was I criteria I inclusion/exclusion couple on I March there. for bit sort A clarify housekeeping a would the clintrials.gov. details ask RESPONSE was because appreciate that. looked

data data you criteria partner to in to to that ask wanted first Thanks. MBX-XXXX for reporting of sort that half of I So I second, And if changes in study? expect if then there is inclusion/exclusion Advent? in this know if in hands year? want material or your your is of been actually And any

Sujal Shah

Yes. questions. Happy to answer those two

protocol atypical to like think see of study. the RESPONSE a conduct global I throughout amendments some not the in

think some patients work in threshold in GFR of I some recently which This effect renally enroll patients of patients. we’ve of as exclusion that from the seladelpar. of for obviously the study study significant out for that makes it we has the criteria done easier most the didn’t impaired because come into lowered for an see much to

a some obeticholic that So washout across positive, on treatment, was specifically study. have patients and again, opens to acid that shorter into that historically. period the for enroll and/or up is threshold fibrates, patients a for the There come also been opportunity prior for that those

And helps enroll. study the so if least, into up again, for at again, more that patients are they quickly, to an opportunity offer to just eligible us, come

here RESPONSE. I the in With those to we’ve were study. enrollment So we us respect in most to seen recently think trends completing MBX-XXXX, enrollment continue have that some in two Phase and significant help will Xa probably helped believe positive again, certainly us that

You the involved in run actually outside those data. is study We mentioned with and certainly publicizing we correctly of full rights retain funded but the fully will by sharing be CymaBay, and AdventHealth being groups to program. the the

so we’re year-end. share before in that, be inclusive parties data CymaBay. to we expect study of can to from a fact, see about all And position should you that that So the excited involved, coming for the months potential come to complete in of enrollment in really the that

Steven Seedhouse

detail. the all Appreciate Sujal. Thanks,

Sujal Shah

Thank you, Steve.

Operator

you. Thank

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

Patrick Dolezal

or on taking as And pre-commercialization, would you what there community a just whether pipeline merger on such really Or for in response kind engaged at of the second company I’m all? is questions. guess of transformative space consider then payers if so? and ever the how question with development, you’ve thus business the of a focus on the been so, expansion, topic be if far, transaction, preparations, And thanks Hi, thinking if curious perhaps? has the kind a later-stage assets? about would more I a similar earlier-stage a you’re physician

Sujal Shah

of as think value the appreciate we’re shareholders well. patients, and opportunity Patrick, for our grounded to create Yes, I near-term I question. by first for foremost, Look, course,

so that the center of And seladelpar. execution is on

program And we’re our a really level development to prior our most to of global near-term will in of because clinical differentiated in as the that program being know well of history on we of seladelpar unwavering grounded and as inclusive completing profile greatest and program. value in executing study de-risked position date. focus today think in enrollment created and is extensive the development Phase that X the really as development in PBC we’re that to seladelpar so we RESPONSE, that’s for of the That’s amount really program where our the ASSURE, adds off us be executing on

expertise development, the we’re we and seladelpar have believe we additional here clinical successful at from certainly with opportunities and value, as we’ve infrastructure certainly with So commercial, on around that’s teams final to CymaBay question opportunities. first and about future extensive experience clinical add foremost our CMC. priority. There we is capitalize to built operations research no And quality, that if that regulatory, as to think we leg, internally create

could little here spaces, experience inside in and about a company a talked the other course, and certainly near-term opening to similar GI, of You envision bit depth opportunities we in today, amount there fibrosis. of obviously, differentiated expanding up around but is of ourselves here opportunities. and A focus, inflammation liver tremendous

right and and the they right expertise to I it internal of the our opportunity. timing to with be and have focus have fit think areas well would

advisory see importantly anchored our a we’ve as to do the longer-term. that inherent to market continues inputs from we’ve started much that in anti-cholestatic the these think, we’ve of continue data that of we’ve of years. will to think center and we’ve With thought beyond feed Steve. meetings, symptoms of seladelpar improving seen disease, is key dosed collected I’ve on on we pruritus. mentioned, I’d to research. a ultimately, at seladelpar, far, of the north data that as well out that well, dataset, with date, the of X fatigue as patients clinical seladelpar process of again say So market benefits we the which of in years is anti-inflammatory support we’ve of have many work. again, in response as seeing to medical with of the we many of globally to thus even XXX I internal community, importantly, to the the And We’ve date, I demonstrating continue with to the that value. of data, continued near-term But from actually PBC amount continue should including community an as the significant the response. wouldn’t payer final absolutely pipeline positive that, believe to That data effects certainly, benefits away That’s as profile we’ve involved it a profile, we as And it seladelpar to really we’re focus gone take drug well treatment. perspective, we Obviously, opportunity talked been from just for But to community gather ours we’ve research progress. fair physicians. create growth respect had pre-commercial bit bit positioned at a fair PBC today, to been experts tremendous gathered closely we’ve opportunities do

opportunity the see later complete get in we this you’ll about of third lot perspective. insight year. that excited the share think quarter again, We’re of more commercial part enrollment as And this to we from a and a I us

Patrick Dolezal

Sujal. Appreciate it.

Sujal Shah

Thank Patrick. you,

Operator

you. Thank

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

Ed Arce

here, may. for taking And for my on the Hi, enrollment everyone. multiple in face the challenges questions me, progress lately. I of with questions. congrats Four if Thanks

degree of RESPONSE. is been study, to many something over enrolled? see we Firstly, patients get you Now could I from be study, in? with than how tell XXX the rolling to you long-term now open-label acceleration the RESPONSE, on were have looking said less to that end Secondly, that full ASSURE the XX there to enrollment? to additional enrollment, regards enrollment patients at some over And us actually think interested you’re you’d of

questions. ask XXX of and think, from looking that broadly, I And of that? under and or at confirm the to looking mentioned. the curve either lastly, we then in about Is should see it looking year, as you’re do maximum question, And release sort filing? total R&D increases the just and at I looking the that this glucagon patients and the to available would wanted you’re we you’re to the area part the throughout specifically, initial the for reasonable NDA flat? Dan, the that’s steady of Just Thirdly, answering of the the financials that while relatively submit in you Any be qualitatively time you data thank for, fourth response efficacy wanted that a benchmarks that is my through year. more MBX-XXXX, expect G&A filing And quantitatively? some for at, study, it’s a

Sujal Shah

Ed. Absolutely,

written down. got I’ve all me you here If anything, that think I I miss up here. can catch

with First of specifically indicated all, number not the that randomized respect the patients of enrollment we’ve to study. are into RESPONSE, in

enrollment ex the in of be our face-to-face accelerate Clearly, to to face-to-face If enrollment, talked we over objective sites correctly X countries in the last consistent the that than various over this, pandemic, we prior if as internally, got as less around XXX think a is sites. or across XX% think almost been calls or tell investigators. What at U.S. study we I project that the maintain we about time that We’ve And always simply little XX months. mentioned patients Where world. but then enrolled members not activated, sites we’ve we for read carry specifically our did number is I now the a enrollment. a XX risk. the X so try on quarterly a I mention been We’ve we’ve sit that you European bit level XXX today. months you with no do always so that randomized, QX. about one data some today, our of well heard is projecting correctly. all tricky activities more of to time of level that is I’d given least is has a be backdrop point key of X where than team of at the bit and as investigator have last fact at today is we consistent of to challenge lines actually talked the

sites a visited but that, will and majority been is not have over of think engagement question We representatives. results. largely CymaBay, America, across America, no CRO, APAC, There we’ve our takes well we Latin at that Europe just ourselves generate of North level CymaBay that but commitment, clearly,

just enrolled sites we needs of involved every are seen study quarter. have level quite I positive as last as of anchored trying get seen of forward, consistent the So that quickly And to But as we this of the what in we level we maintain enrollment that around all third certainly, study three with again, initiatives in we possible. do have our to through that try And enrollment. few around the site, to going we’re accelerate are mentioned, working to this over months recently the actively – actively if months. enrollment expect complete last

bit XXX the Now about a just do on again, data, safety of seladelpar. efficacy patients ASSURE we amount both you tremendous where now gives as also ASSURE a as that, long-term asked a us of about study have long-term well study,

of patients study sets first the was last first the Importantly, of into the mentioned patient year. I RESPONSE, April completing

the patients clinical That’s ENHANCE study So, been much Phase clearly, already are open-label The started X that our our that prior prior to however, have treatment those XXX, of that that ASSURE. from. study. or period. I Phase studies, majority either vast comes came over X population sets in where we into happy say are have into RESPONSE rolling first And that beyond patients am we of XX-week of

don’t north patients that number ASSURE. started sure even mentioned of had million. We am what dosed XXX also the have we from many see to. $XXX seladelpar Again, patients RESPONSE. of that not to quite expect I refers with that we You as been

excited to half are the study. almost ASSURE into those already see come of We

in commit in are RESPONSE subpart in historically what I from to a novel have relative approval study pathway H an and regulators accelerated ASSURE, around we we seen think we to approach be to PBC. study. stages do very we of outcome Again, have that what occurs final with because the agreement PBC think a approval beyond commitment ultimately, to a outcome long-term will

we, And process that that that but the RESPONSE will eventually we are have finalized. study, as Phase clinical dialogue we so we to agency again, that’s completing with as complete on X and more speak. as complete And the yet enrollment share and is to come,

question, around was And and in response the study. Your to again MBX-XXXX really think efficacy pharmacology I – study. here, proof Chuck in initial you around of will expectations this and ask around some third give I with had jump context

Charles McWherter

a used the Yes. so that in subjects. I are good including healthy sense, also Hi design Ed, that was one, think the we basic we

the And we that good of subjects for restore ability the terms then us we mechanism of that low clamp. healthy levels related a So, its with will the from glucose the to in elaborate ability to is what benchmark course, give from levels its expect to normal hyperinsulinemic glucagon. induced

Sujal Shah

year. And then financials around to this will of through answer ask Dan questions rest the I the

Dan Menold

noted, the we a you the development, will Sure. doubt, Without question. focused for on Yes. executing Thanks R&D. as the be

unwavering. Our investments there are

be studies. throughout With that it towards really our thoughtful trajectory and through the year. the want be NDA-enabling be spending data. this will G&A, through time to really largely and ASSURE enrollment of completion and to respect thinking flat We will of We and completing other the RESPONSE ultimately for towards

forward. be costs as to move going are very our we watching So, we

Ed Arce

it. my Thanks for I taking questions appreciate guys.

Dan Menold

Thanks Ed.

Operator

Thank you.

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

Jay Olson

I and other, on on the study the to one would the probably M&A like RESPONSE front. follow-up

to on. need seladelpar. or M&A, prelaunch the package? more you also, on complete just Thank see and that rate open what’s it’s for also market like your if a So, infrastructure the projection whether enrollment XXX as for the projections building preparation? in on you where we XQ one want wondering just as in the the run comment there months? on the size currently commercial that to active U.S. opposed And RESPONSE on like PBC or include and few run view ex-U.S. sites investment to see cash past separate you confirm have to study, the I color Do in more Is And can – also additional based your need you you. opportunities

Sujal Shah

Yes. Thank you for the question.

do, So, RESPONSE, respect complete with in QX. we as mentioned, to expect I enrollment to

on the you project have today we that today disposal we pandemic. allow where of believe activated that asked the that’s sites. into future. whether sites the at to to one Certainly, challenges the accomplish completion enrollment I can additional predicated mitigate the or as us of our actually think what some well of again we things and the That’s we on are not based that are of

timelines. CymaBay to includes It is With with are to at very globally it on the clearly again may near-term. what well sites you those includes U.S. goal us than of will Latin Europe. versus think fundamentally, represented The independently. That geographies to ex-U.S., that can benefit. ultimately so we confident enrollment additional But the we Asia, outside where allow respect includes to somewhat to within M&A, I to U.S., are jurisdictions, in geographies patients I And that think complete look hit ensure execute get seladelpar America. currently plan even here we clinicaltrials.gov are that once our see plan, we these the

There, going is we today. most particularly develop And where the opportunity geographies are an bring outside so that exists from but a our to regions that We patients challenge in alone. there do the we on to U.S., outside have on think, build Clearly, partners, I the to strategy to execute and continue likely based commercialize execute can ultimately that is our involve in is in a and seladelpar certainly infrastructure U.S. to and perspective. we to recognized plan our the own. opportunity ultimately, significant are plan of

about all strategic still today can continue cash include potential value, of provided our for actively will so you create fact, in us cash to-date, that that even in a Then little thinking to asked The doesn’t finally, allow a runway obviously, stakeholders. cost-effective we commercial again, have launch. significant manner in runway. bit a about involved and we partnerships are and effectively be once And to do

for look end last of next of to and different end to that us the as successful development non-dilutive Again, of as sources the of through value well. long-term vehicles in capital past as our upon of Clearly, expecting data, well the last other stage we we we and there are execute is year. inclusive around So, alternatives. line a various fund believe partnership cash create host ultimately, next financing strategy we at to strategy top continue of a variety deployed as have being potential of to opportunities –

as post continue post-NDA though year line through file is to We possible we certainly guidance end So, we we approval – cash launch. to have as the feel levers to of few next filing top predicated upon not NDA ultimately But running NDA as move forward. and current quite look around the as data. quickly a pull would

All of projections. that, of our in already embedded course, is

Jay Olson

the for color. Thanks Great.

Operator

you. Thank

Our comes Cantor Fitzgerald. of next Kluska with line question Please with Kristen the from your question. proceed

Unidentified Analyst

question. is on for you Kristen. This our everyone. Rick Hi for Thank taking

production in respectively. T-cells role greater potential, Frontiers here. immunology, and model subsets CDX that proliferation published identifying we distinct in Paper two recently PBC the of one with high of at looked got just hepatic have of We saw mouse researchers in a T-cells cytokine

with for potential its role be and are potential thinking associated in about immune how the with Given role fibrosis, cells in you. PBC resident pathogenesis? how you could and the and interacting inflammation Thank seladelpar liver that

Sujal Shah

you comments some that ask here, give will for question. I Chuck to Thank Chuck?

Charles McWherter

I just want of make to was off mute. Yes, I sure

do publication it, for liver or your at the that looked the we indicates we in did are presented seladelpar really data have saw immune-mediated T-cells or where liver would an that that of that And we short fibrosis. in a cells one that like way cells, question. study but increasing The yet thank renal looked any we whether developed where or decrease is both a established to I is its autoimmune So, by is, fibrosis question we and intrigued that various immune a of that you role. answer us one at think with whether you We cited is a that have model, at to meeting haven’t be interest roles and diseases. an abstract we recent did NK they number another, affecting not

it’s one that is indication going may question just something one currently will – area be have forward, to to I we an continuing So, there there. that understand of that something that’s advance, it that and be liver but But immunology is that’s the interesting. think may is

Unidentified Analyst

Okay. Thank very much. you

Operator

Thank you.

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

Unidentified Analyst

do Thank biopsy is you Hi. questions. now you This Yuan patients taking completion, have sense close on our Since taken? our have portion for Mayank. near you of a enrollment for of a what the

Sujal Shah

that Yes. question. Thank for you

course, many fact, fashion, of a for are in So blinded volunteered we have, in patients how see biopsy. able to

I and to biopsy. think fair that XX-week biopsy study have required the not a fact patients. then little are in a They past bit we in a have still around for liver talked the this baseline is voluntary

a to of We the encouraging required those so, patients share course, to are, do of agency. subset with

And again, to with confident is in so agreement our on the to are ability agency. track. a we able has deliver been once we what And our are it number

context that that for the We baseline little is that about we in accomplish is actually the study, ENHANCE agreement about past of RESPONSE patients as can And volunteered the our agency the fact have XX% in a of biopsy. something in Phase talked bit think certainly X with our something well. we

Unidentified Analyst

And remind from you now? be right than question that in you us. color. BD the evaluating more those inform territories? Got regulatory in your it. U.S.? additional how And EU be about for one us what might transactions other you might Thank And path how different your might Can

Sujal Shah

Yes, certainly both to what as We historically in guidelines setting believe, have us in and RESPONSE, seen well U.S. question. precedents we in allow in well seladelpar, of the PBC as the believe, as Europe on it’s based we Europe. good will a that U.S. as the and register

that So, the in allow RESPONSE us to geographies. endpoint will from regulatory in a believe register both we perspective, primary again,

we are you sets in an think geographies, and territories, some again, The potential a to population. set demonstrate where I think we to that when bit is of Clearly, some about there supportive. PK think example, supportive. have other data are endpoints data have least at you think the little Japan, as

Japan So, there like could well. China be in and clearly additional as work geographies

host of there outside support the and data just in are Europe. some from this a geographies So, of RESPONSE really we outside will specifics U.S. fundamentally, U.S. But set registration think

Unidentified Analyst

for that and Thank you color. data

Sujal Shah

Thank you.

Operator

Thank you.

question Please of the comes Smith line your proceed with Securities. final Thomas question. Our from with SVB

Michael Kratky

expanding bit talked Hi for is Thanks about for the our Tom. Sujal, population This a everyone. you PBC. addressable in question. taking Mike on little

pretty until have it Just market? a time seems down like looking think you at meaningful that U.S. should see is slow you that Do growth from the to meaningfully. sales that pretty the market get this now market, to started

Sujal Shah

acid. question. And about little there talked are to Yes. When you we It’s plateau, presume think starting and we think a about a a are opportunities. referring of sales obeticholic bit seladelpar, key number great to we I

in with of who patients response in reducing treatment clearly those First all, for ursodeoxycholic cholestasis there starting efficacy, normalize in to is are patients. but really inflammation, and biochemical first-line in complete acid, just responders a to need greater not

or have obeticholic you cause think worsen is to pruritus us we adherence just So, look that one about When acid. that thing opportunity differentiated not We alternatives, alone data to can at second-line data, for course, think opportunity separate significant potentially PBC. treatment with think head-to-head tolerability growth we sets, we think in – the that’s of When seladelpar that. is key patients we demonstrates persistency quite an another treatment. element of and separate know even but in obeticholic from acid

when their level acid, tolerability, impacts we actually that have a with about seladelpar having And benefit itch. a obeticholic bit and look clearly, some opportunity market, a on be reducing think we a we data sets greater adherence. potential in pruritus just of talk can the at so differentiated, clearly, And and the but of quite

would outcome think is I efficacy an argue actually patients for them.

those are along of potentially safety. some of the the core seladelpar with so profile, elements And

more obeticholic seen We in a couple of particularly advanced have label acid, populations. for revisions

We compensated and hypertension. have and portal cirrhotics even shared some data sets with in portal with hypertension without

potential to treat disease the second do think and in spectrum the seladelpar is here. more the And to opportunity work for support line an a a more just of so of here, is setting. would say, again, third yet for growing much population, But we spectrum once clearly, differentiator there that a treatment much us opportunity,

confidence excitement market that being generated patients expanding tremendous tremendous think amount benefit with of earlier fully have opportunity. a amount normalization. certainly, of a we treating around them to with There I potentially that data the around get of is treatments profile, and now biochemical

treat wait overall expansion treatment, So, again, patients, things lead second-line otherwise can these patients for to some that to them earlier opportunities population. improve might the patient potential of of we that addressable and for outcomes the think are

So, and to continue to our that that potentially see support to explore we sets are would where to continue data growth. excited

Michael Kratky

Got Thanks. it. helpful. very That’s

Sujal Shah

Thank you.

Operator

Thank you.

question proceed is a next follow-up with Securities. Mamtani your Riley Please B. question. Our from with Mayank

Unidentified Analyst

seems us. Phase from Thank you. data of that more One you one guys. of X It slightly peers question like have your are ahead

do for be expectation we if we are they shift RESPONSE? to should that for anticipating the And narrative patients you know So, how included? you. are wondering severe more Thank will the what have

Sujal Shah

the question. appreciate I

month. the referring where elafibranor hear in last sponsor So, the I with screening ongoing you PBC, presume referred development of did are to completing patients to we

coming study usually is with of screening the period this month. the projection potentially could end weeks, Our that enroll that, goes in that beyond not if run-in here the towards

every tremendous we efficacy, set, in not program as potential that focused development the this I I in RESPONSE RESPONSE, data just gives safety us the also think quickly ASSURE as setting think yet just data will Phase enrolled in be importantly to think fundamentally, think, some to still of getting effort X. amount we what we putting quite Obviously, the these the but than even much for of possible. population. But extensive will be that from as I are addition We in will we of ultimately PBC. These on programs can important are are sets we a elafibranor control. say program this data more things two the see supporting, currently

RESPONSE a targeting pruritus normalization we for in of given around of we end that We key biochemical sets. powering something response, and secondary and are advantaged also number point primary around the biochemical have but around patients not that’s response also prior our benefit treating data think of effectively just the had

also most something set, at day, against But to the certainly can. seladelpar push think extensive, potential end is quite the it’s position of to latter is have well the think as enrolled treatment I study the data and expansive get quality will as So, our quickly the we alternatives. we other that

Unidentified Analyst

Thank taking our for you Great. questions.

Sujal Shah

you. it. Appreciate Thank

Operator

any turn further no for are like at questions conference time. There the you. back closing Thank to Mr. I over this remarks. to would Shah

Sujal Shah

I it. appreciate

are and enrollment capitalize once completing again, look on positioned de-risked we We we approach to multiple second highly once differentiated a So, a grounded ongoing by of market, believe provide highlight in the value. we and of with seladelpar soon. the months are Against to creating program of challenging of again the well through you investor the and believe for to ahead speaking balance significant development conferences data major half meetings we to as all in late-stage very opportunity significant backdrop a that this coming our updates sheet year, the biotech what We PBC. strength and forward year. lead has medical the a expect been at strong we existing remainder

Operator

Thank concludes you. today’s This teleconference.

your your wonderful Thank participation, for a at and disconnect time. may you day. have this You lines

Cymabay Therapeutics (CBAY) 13 May 222022 Q1 Earnings call transcript