CBAY - Earnings call transcript Q2 2021 | Cymabay Therapeutics Inc

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Operator

Good day ladies and gentlemen and welcome to CymaBay's Second Quarter 2021 Financial Results and Business Update Conference Call. At this time, all participants are in a listen-only mode.

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

turn over to Mr. like I'd the to call Now, CymaBay. of Dan President at Menold Vice Finance

You begin. may

Dan Menold

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

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

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

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

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

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

Sujal Shah

Dan. seladelpar. Good year today. staff approximately you, this midst of gained ago, restart development We you this the joining XX us Thank employees to for the continues regulatory did that the and day. clearance we clinical of of with global in One afternoon to successfully even thank pandemic

Association across to for innovative innovative saw achieve are the positioned needed a us regulatory secured presentation us our The also need latest patients rare us our On will or advanced non-dilutive a Congress seladelpar life funding the milestones in vital development to with primary life-threatening financing global potential with of are to for Liver our International then and ask attain and includes International PBC. of development and biliary with a through Congress while studies recent in we provide summary In getting patients in a cirrhosis, of months of successful the Dan's our summarizing quarter have in sciences results Abingworth. the history for I long partner who many presentations value with study agreement, Study to providing update this RESPONSE progress call, second the patients risk-sharing manufacturing submissions global to launch. this all our of second Chuck key funding stage and begin agreement and complete has Abingworth financial those and Disease. we quarter of early-stage in with our the the Liver delivering by III RESPONSE, shareholders. We will once highlights areas the funding have today's who that the preparing by those functional disease. experienced key companies in at commercial the most on treatments necessary seladelpar's our including development significant European June to followed core objective filings, now Liver programs featured the potential at Dennis Paramount the also plan product, the Underscoring the six this again regulatory talent more shares patients first to cholangitis with added agreement brief aspects our discuss Phase PBC. This treatment meaningfully sponsored seladelpar and to by of the to improve have executing pipeline importantly a that belief of year.

We our presentations. will Q&A have following

to to are regulatory of expected sheet, for studies deliver NDA-enabling to our and filings us capital patients and RESPONSE various III we study to over a patients the improved completion to ongoing and with Proceeds value Abingworth PBC three as and funding of with the the support chronic terms balance By up PBC. program deliver to we ahead securing will six on be development open-label to our months. means for installments including well-positioned next well. agreement receive the like transaction, Our of of the to find of in non-dilutive development which our approximately study announcement are strengthening diseases this ASSURE strategic shareholders. funding PBC million will million liver support treatment transaction inflammatory development to alternatives of this up from commitment to the of $XXX seladelpar Under Phase global risk-sharing the innovative $XX of highlights our time from in promising received seladelpar the opportunity long-term Abingworth the mission fund

the the capped payments receive regulatory spread US payments of funded In we will of million the sales. within the this in with of week. payment agreement are exchange, found sales the kind. repayment SEC of back-end precedent weighted months product And including Specific $XX are an recent to sales US on following most equal make year have schedules in this in X-K either enrollment additional for period based completion the or outlined times filed and two the two regulatory last six the addition, fixed amount funded RESPONSE. some terms transactions We over repayment first in favorable the the X.X of and approval approximately schedule the a at the option believe the to EU. caps We amount in milestone milestones

and We US CymaBay the agreements competitive come are that payment was legal selection. Commonly, in We significant believe commercial to data upon reflects PBC. closely for a a approval. of the in at interest have background to advisers also retain process the with least biotech worldwide ability from for consummating regulatory variety final a or deal. commercial are for programs seladelpar the Abingworth worked at the upside regulatory accelerate of funding vehicles for parties risk-sharing long-term significant We efficacy full and multiple the in along financial to reduced funding seladelpar in shared conduct Finding to-date of generated making rights. financial view diligence to potential wide safety and amount with a a key and partner examined retains approval. seladelpar outside a level with in the potential before

experience support as in and they have are operating excited Abingworth's deep to We financial biotech. have scientific,

Moving on to updates. our business key

with key turning development over for to Completing by PBC Dennis to-date Let the the me to RESPONSE forward. before top seladelpar discuss reviewing call remains patients of priority. achieved focus and milestones begin our going our

is bilirubin, same normalization the ursodeoxycholic placebo-controlled III for only change patients phosphatase of key RESPONSE global include rate PBC. endpoint evaluating a seladelpar is a value from study baseline Phase As XX XX-week used after safety in XXX phosphatase as second-line or pruritus alkaline milligrams intended the alkaline with reminder, a of to oral four biochemical endpoints to patients and the XX to responder in greater. in of first-line approved in patients seladelpar alternative or Ocaliva, placebo to study randomized response registration to weeks for patients the outcome efficacy reflected the months the The enroll secondary of once-daily rating a PBC. and composite randomization an rate inadequate primary pruritus, numerical pruritus XX to six scale with X:X with measure weeks therapy. acid and The moderate-to-severe an add-on treatment baseline as or at intolerance Two in register is therapy

of based our off ENHANCE milligrams symptom III design of statistically meaningful, demonstrated many improvements the burden in markers three disease in is you study only after for months significant know, seladelpar with in biochemical PBC. XX which in RESPONSE Phase As dosing, reductions patients and of

secondary we patient first In XX in in RESPONSE ENHANCE, to same over key into to same our around population, randomized in we taking as prior and countries enrolled patients studying XXX dose the our leverage of sites did plan optimal same patient evaluating endpoints by world. RESPONSE ENHANCE as where XXX enroll set success as work pandemic. seladelpar is that months. and in were We XXX from primary this and over XXXX, enrolling we In to have mentioned focus to aggressive at always and experience patients for we on quarter the we our remains of RESPONSE. first And priority are our first aware year-end, in would global end the April. with as with complete initiated randomization through enrollment the presented intending although an year to was occurring by patient quickly challenges in possible continued last addition, Screening RESPONSE, approximately the patient to the goal the a XX XX-milligram

study global have PBC, and available. size Study this the Lately, have been affected as added ethics rare vaccines and begin of and variant and to research. activations clinical complexity as accelerate. including IRB enrollment to in any a approvals and screenings related site such have the site slowly As begins become activities activations with risks pandemic accelerates as uncertainty and the disease recently committee Delta even effective spread by to evolving start-up

the to the half active into of to patients impact developments, these first be assess we of and prepared we keep have enrolling XXXX. As sites

will to around clarity progress of I'll time basis, as to to pandemic as greater we Phase lines and III initiatives effectiveness complete enrollment Dennis? additional quarterly over to the and strategies into address progress Dennis We our to and on date impacts the a provide the the our of gain now challenges. turn we these program. our call we implementing are review guidance

Dennis Kim

Thank you, the should also many be of to what come. calls opportunity Sujal to in the earnings thanks first and participate CymaBay for

Sujal in Phase did Before XXX we a detailing remind as that in enrolled you I'll patients study the ENHANCE just of year. pivotal our under status RESPONSE, III

in and as see and because anticipated expect increases it to at this of activated. Our of are part company sites site And year. in we meaningful is of as quickly, screened from conducted studies enrollment. the you in We're in well screening the study potential in a experience have a having ENHANCE. these fewer targeting study get clinical are typically patient been summer returned RESPONSE study targeting greater We as RESPONSE PBC the that least deal of challenges coming great XX begin Due at – in number XX headwinds, patient especially growth to on XX to months, greater patients the though and volunteers earlier sites goal to personnel greater intention XXX there are still patients put this has longer to taken by we clinicaltrials.gov, Even into enrolled stages for sites as as enroll end the patients breaks. more in is our countries mitigate their look effort enrollment activated patient activated, RESPONSE, having recruitment process patients. than to has for even to approximately competition COVID-XX study having if

coming sites As To competition compounded activations overcome the patient enrollment we pandemic, and/or are we patient see expect months, and engaged in as and number of the the we participating patients, challenges activated. study countries, by as potential study across to sites in to numbers commensurate identified related increasing actively enrollment to activities maximize for in increase well intensified accelerate of follow. programs a additional site the screening efforts to the already

to inflection project these better recruitment near we be our grasp to completion patient and a future, in patient site in numbers the come of better study we points date. have enrollment screening rate the will As able activation, and

XXXX to Before over the for discuss hand European Increasingly, liver our updates, pipeline Association seladelpar recent to of portal recent disease, been to the I abstracts for Chuck remind may diseases. the important compensated you and study cirrhosis clinical like quickly patients patients at the presentations things aware substage of I'd because made PBC you of is Ocaliva of now as which an are of and have cirrhosis contraindicated. with in label changes with hypertension made

These drug PBC increases tolerability with reported severity a in titled and second liver be of in improvement of in spectrum only several with parameters from compensated find cholestasis. exposure stages PBC the results and more previously presented showed biochemistry with three-month of II in compensated PBC, A cirrhosis of seladelpar consistent analysis tolerability option which the safety placebo-controlled markers importantly compensated safety compensated due efficacy, safety, those cirrhosis. III We clinical an that and with patients presentation and biochemical years seladelpar. XX as website. in have non-cirrhotic been treatment cirrhosis cirrhotic meaningful accompanied for with patients of seladelpar no cirrhosis, of including pooled potential reported with in and can tolerated, a response that seladelpar Phase a to studying are After at had patients and pattern We a in and poster nominal liver prior and of analysis was pooled X by patients efficacy dose-dependent with than patients and analysis and across significant treatment fibrates, well of profile. disease a milligrams our data with dose. in you Phase biliary presentation patients obeticholic three XX cholangitis, PBC ENHANCE more And of importantly, demonstrated months, on patients accumulation study open showed seladelpar appear similar cirrhosis, or the of safe, efficacy study primary patients, This II to consistent to of acid PK Phase label safety or XX-milligram biochemical this our a on studies. and recently and magnitude

with these prior therapies. patients those treatment have fibrates as PBC or addition, OCA obeticholic acid, exposure prior In as to responsive did with or seladelpar were not to who

efficacy these We benefits gleaned toward our the potential an educate leveraging overall to profile of with seladelpar, and study the highlighting study exciting to the sites, safety are study eye Chuck? and by results RESPONSE and communicate patients. be with

Chuck McWherter

release sugar. in pharmacology today Dennis. and diabetes, for being one has low initiation evaluate and study clamps study conditions healthy the of a currently providing I announced stimulation be dose of of GPRXXX single ongoing studied updates in multiple with II and internally MBX-XXXX Last PK dosing patient patients crossover Phase study using two I'll pharmacology Phase in just and X two-period you, which safety to agonist completed volunteers. Thank CB-XXXX, of a quarter, for in type an discovered other proof of insulin we glucagon under hypoglycemic blood MBX-XXXX

with developed reminder, brief The and MBX-XXXX. type for Charitable is planned a clinical CymaBay. prevent goal and the funded previous study patients in completed Orlando and to at MBX-XXXX hypoglycemia Institute in rights and study. by approximately for forward all a with subjects it's quarter, in being Research is conducted retains M. the prediabetic moving the subjects. agent fully including As concept recruitment for is has to XX B. MBX-XXXX Translational investigated GPRXXX agonist Harry It the AdventHealth Helmsley potentially Florida diabetic Leona completing continued studies, and This is an recruitment The being by discovered CymaBay five Xdiabetes. Trust. patient last has product of

hypoglycemia. In the for guide on and addition, versus curve These pursue safety maximal treatment glucagon are whether development to results endpoints area periods. diabetic under primary decision, will further for glucagon and release to MBX-XXXX tolerability, our placebo the

CB-XXXX and greater the possibility safety prodrug, monkeys, XXX of a exposure was dose for now The involving this to was and dosing of had to we and through the in the NLRPX been block days CB-XXXX and the tolerated doses CB-XXXX. and milligrams does it and arhalofenate. that greater the need CB-XXXX the explore wide healthy ligand previously a effective were daily It for NF-Kappa than XXX immune B than gout. raising had This genes. It safe XX metabolite increases unmet attenuates X,XXX high innate milligrams of The doses approximately PK its pattern drug were and good tolerability. inflammasome to In a portion to found for even disorders that give XXX study study, of of to is single nonagonist now more with With safety. pathways. in exposure ascending studied that Moving a be the it confirm have up exposure confirmed dose could to of form PPAR inflammatory the prodrug CB-XXXX shown dosing greater the milligrams. able and from I gave in PK well subjects, studies mind, is that diabetes of completed gamma multiple active We multiple sponsors to expression arhalofenate, XXX exposure Phase for the of XXX up has NLRPX. historically that we in prodrug ascending to cohorts from single milligrams. it in at of dose those achieved its too single examining proportional variety

of unexpectedly and of in events, be capital-efficient cannot one that respectively. we have a of nature were after safety severe easily that without challenge overcome. the exposure and sequelae. given thrombocytopenia, based with findings prodrug on noting a moderate stop case program have achieved the subjects of each was its is development than dosing of understanding the conceived worth Accordingly, and and CB-XXXX. we additional to decided arhalofenate. that cohorts well We this further concluded fully the X,XXX development goal consultation with believe to manner. XXX serious the subjects Both and unexpected is led of dosing XXX-milligram and However, Nonetheless, these experts recovered more It

our be to able we held are research. we to the believe publish promise of because disappointed we course, of hope Dan? some Of preclinical and compound the

Dan Menold

Thank you, Chuck.

accelerate program development PBC XXXX, in half the management late-stage to execute of its highlighted, of programs company continued have for the first early-stage the members and to other other in seladelpar to advance efforts As development. team

our Our ASSURE patients RESPONSE and Phase most I continued as were resources and enroll primary well we directed in studies. PBC where to focus other NDA-enabling of towards as

As recruited regulatory of employees successfully June the clinical grew key, XX, company's new as we talent. to XX and commercial headcount

enable the our continue which second in cash knowledge our look will both investments appropriately current the our now our efforts half recruiting and million operating as $XXX.X with into strategic and Our at milestones to $XX believe of us We extend us provide for XXXX together and operational to and growing enrollment XXXX, Overall, development to regulatory of short-term completed will the available of augment from commercial capabilities of, and the plan seladelpar the investments existing we prepare the further to runway. to June financing million Abingworth, clinical, totaling our upon agreement cash PBC. capital million. additional we future XX, RESPONSE ended initial quarter development will plan in equivalents and critical for us proceeds sufficient $XX fund available in with would option XXXX. Furthermore, further development completion financing with in

the Net for diluted to and were minus period XXXX and incurred and XXXX. with $XX.X clinical $X.XX following months $X.XX PBC. our seladelpar active XXXX enrollment our minus our $XX.X a respectively. Turning million and XXXX the ASSURE, cost XXXX XX, during operating June late-stage ended driven and to in primarily diluted half clinical share months XX, two respectively. trials loss of development due Net expenses, were higher $X.XX global million PBC minus six brief particular second of per review operating three XXXX increases in $X.XX or by increases resumption RESPONSE minus which activities the or XX, was June comparable in June $XX.X $XX.X million than largely loss associated ended and loss in the months for was and six the and Net during now of the three share results. of ended XXXX, was million clinical In per

and as of we clinical We clinical we plans expect on further exploration as development opportunities. our operating our other XXXX in expenses execute to in development to PBC increase continue

all we update I'd global of to the activity to a to seamlessly employees, business operations coronavirus provide conduct continue to for remotely pandemic, Due our operating Finally, which has on as ongoing brief current continue with like the as allowed environment. you possible. impact

we to will and While monitor Therefore, COVID future closely where will including second continue operating and to developments the on vaccination take have recent it risks to risks their progressed available mitigate our surge possible. encouraging, these has of cases delta milestones. through effects that is and to of still worldwide quarter will the our clinical development was led business, variant in due seladelpar duration to uncertainty regarding we associated the and in to ongoing actions the the ongoing pandemic continue pandemic XXXX PBC

studies. ensuring by As guided enrolled will employees, the actions Sujal? be always our commitment in and well safety of as health our a our continue to to as patients clinical

Sujal Shah

Dan. you, Thank

agreement CymaBay we future As the our all enables us for tasks on bring seladelpar to on us today, PBC. patients development execute us complete position discussed have Abingworth company opportunity and the for with to The success. necessary centrally to with of required Phase remain focused III development funding at to

patients Our and for rich We're generated we highlight global challenges confident that have questions. a team's today. opportunity to facing upon today. limited are the meet to and are PBC commitment patients be prior remain Operator? alternatives data our believe treatment significant the ability now in we date improve very what exist in to to to we the to sets take our We steadfast experience we happy

Operator

from Instructions] this Yasmeen At question. with Sandler. Rahimi you. time, session. be proceed conducting we’ll with Thank comes first Please a Our question [Operator Piper question-and-answer your

Yasmeen Rahimi

the all I you Maybe sites. team Dennis for additional that question you Thank on had much commented you, for first so the updates.

ongoing is the on sites of What for have share And initiate? preparedness is on additional that and opening you of you directed the those type all maybe this question to on extent in Can interested to findings. and you you us can are commercial some XX us with done Can maybe activities clinicaltrial.gov. market Sujal. hope color maybe and a then -- some provide discussions? the beyond give and are sites work totality second the you new payer listed that the You're being perspective on of to research I that's

questions. So thank for that would my you be very taking helpful. And

Sujal Shah

Thank you, study a Phase we appreciate in I so questions. over countries as ENHANCE, opportunity XX in we've XXX Yas. Generally and Yeah. as patients, a study randomized did previously with over an to we've had initiate XXX speaking, III full the discussed, sites. with

so the And in fewer recognized that global we RESPONSE, although, challenges we're pandemic patients, competition targeting today. we present. at the for greater do outset, XX have patients would the And

So well the with dedicated has approach what our company always same through at this ENHANCE. country we scale had with capital sites into on par strategy Abingworth had been RESPONSE. to the as has allows to effort. mix have funding for size, on we execute agreement a a come study with of the to number as as We're us that The

fully So in sites in activated RESPONSE expect of this we have XXX and to north ultimately patients trial. screening

us, think insights some the the market through opportunity to future share of for for had seladelpar. also thinking some questions I the just around around opportunity you

our focused continue been sets additional about second-line patient expanding addressable broadening choice. of thinking But the data support, of significant that, doing seladelpar from patient would the very been that in a Clearly, addressable an work many fundamentally that to patients to be think we benefit clinical can we've has the believe studies think drug. I we seladelpar of a opportunity population, get with population. as amount to that alternative treatment approach we've around, preferred to the

have EASL, population promising to cirrhosis to early a significance that, more prior at and albeit in speak having an had about XX with the patient smaller in will to the in to That data, PBC the view towards albeit of the expand clinical of expect that data Street the opportunity view set, how with market, PBC, experience, patients how present fact, patient Day. opportunity Analyst overall relatively future. relatively we of in give is continue to we our us seladelpar Some we a now we with we meaningful long-term. and in we had actually PBC, an Yasmeen, a populations would an around this hold in November, about share to with look year AASLD how all constituents end and positioning and think of and opportunity

having look that forward share more to to So opportunity second this with year. of you, towards we'll half the much

Yasmeen Rahimi

much great. Thank very for we you. That’s much And you look that. Thank so to sharing forward it.

Operator

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

Steven Seedhouse

a that the hoping you. executive CymaBay financing. But how any Abingworth new or seladelpar from structured? was role and the extends have development review Phase also that's Good PBC related comprising commercialization if committee afternoon. there's What committee could Thank I future in you just like III. It recent PBC? current that on looks or in comment to will decisions beyond

Sujal Shah

It's great a question Yeah. Steve.

III of same the dose same seladelpar, two and We're So of our endpoints. the built struck agreement was we patient and ENHANCE. as optimal primary, is population, really development to RESPONSE secondary same the studying fund the importantly, Phase the And the in key remaining know, study seladelpar. experience really off same of prior clinical

really So the to changes existing to clinical to any the executive committee there meaningful have continue discussions is strategy. around development

well approved. we set, get need on we're I we think to seladelpar execute to what believe

for committee any continue to the And of make study. from really towards have us around So an filing the progress discussions decisions joint fundamentally, the here of would we the NDA. a path to it's different

I think is Abingworth success. as we've risk-sharing and risk of disclosed, clinical the such agreement carries that regulatory this

with are really that's focused both committee. where groups executive so this And

and organization also in I'd as folks want the Steve technical even through of industry have this a the think something to space. and that valuable A group diligenced out number experience of within also is various there in the scientific And this committee. is experience that across an I opportunity leverage fact investor. to Abingworth that, we space our broad think has set targets that that experience, is rich point

Steven Seedhouse

on great. study. That's question a III And Thanks. PBC multipart Okay. Phase just

or on has baseline And patients if Ocaliva patients. number are maybe of sort patients monitoring to an cirrhotic of expectations change that label from accrue accrue in, that to for for enrollment study you're influence your the of met biopsies question similar type at volunteering just I'm alternatives? the is of in looking want if electing As coming curious for And you'd patients cirrhotic in data on biopsies. funneling the having

Sujal Shah

Yes. questions Good Steve.

the let some about how provide where with to are respect a with Chuck we of have I'll to and Ocaliva. Dennis the that and cirrhotic progress. think some study around little I can commentary talk the recent data we updates bit And we perhaps continue

Dennis Kim

of predict to patients monitor category. to to With come see. to little expect we too able can RESPONSE the closely Yes. FDA that the what that biopsy to that just have would in we're many I going say to we would think study the that how number close early it's in Suffice respect number would to that a so have be we

So in a you able more date. it we'll to tell be later about

Chuck McWherter

question as as Steve, for of out information cirrhotic to hi according patients. estimates the on are PBC our on thanks Patients our compensated coming which UK cirrhotics it's clinical based global well access the well studies PBC patients. group XX% And study with of the as as to cohort about

disease patient treatments bile to importantly patients do more and a homeostasis that's the to affect symptoms. improve need which stage agents so for that acid of which and is respond And advanced could anti-inflammatory

a seladelpar many XX two fortunate with been and experience. some of this years. than patients XXX dosed year more have So out that XX who having cirrhotic as an good PBC we've those even with body dosed area over -- now a is to been of past have

well the continue look We safety. side efficacy at both as as to

hard far to now we'll I that in hard think that committed sets something patient data the this seen - that that's be really -- that's continue your I really seladelpar collect believe for of interest is able to gauge. And do confirm of area treatment understanding question that that advanced is if think an study? to what to In the we've terms patients And on. alternative stage of I something those thus terms to in stages. disease? will interesting we're we're across more with influencing

Steven Seedhouse

you. Thank

Operator

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

Patrick Dolezal

for thanks taking Hi, question. the

terms respect of data that in attained. that financing a financing with perhaps the what look it were relates initial surrounding transaction nondilutive this you readout you then other selecting process type timing background then it us of a Phase and process whether was the as program and And So would the decision-making provide successful speak initial IIa? remind the to some us to characterize like could this enabled just the versus can options? of with MBX-XXXX And competitive to just

Sujal Shah

Yes. Patrick. Thanks questions for the

company So we've at on long-term We're to the we've and that focused it. the the the quite investors. shareholders we and been been continuing today. years story proud had of successful over have oriented seladelpar surround always And I we've fundamentally think quite with

Phase that the the we RESPONSE and III As as really beginning an we objective part position this of we new and the entirety company year corner always the to had into our study success. file to make our initiated to sure complete it study of NDA had turn capital upon the

structured past. the as many to the could in sources year cost capital do that throughout we followed of We to did of explored we we've we evaluated raising capital triggered. on analysis we traditional believe different as So, had various as the fundamentally alternatives even say I'm the have pads we happy financing of that, capital to an alternatives relative multiple

up valuation based a we long-term opportunity interest the on believe think our for between and value disconnect valuation agreements. the process had risk-sharing what running given around we multiple our these is own seladelpar, we ended funding inbound I and potential

So competitive. in process the quite fact was

while success. willing the we in crossing And That's for have transactions to our a to total agreement capital is recent the company that us advisers milestone provides multiple of some financial capital hired the our that agreements of X.X, press products one the have in inclusive week, is that this release process. full Abingworth return agreements, comparable done for run think to typically repayments. approximately funding at or repayments that already backdrop terms cost an mentioned we look at mentioned in to fundamentally, to and think in as are we this of the already in remarks and remarks, approval turns again, capital had a to at last our fact cap have the prepared these evaluate companies recent put many of regulatory course, have in in in done. lower even a seen of actually, launch. milestone And you lowest some risk-sharing as also finalize available best of are in shareholders the case, we've market. clinical agreement, In I As perhaps the position significant in least of I commercial those expertise. been funding here that provided today take risk struck been talked sales of well than X.X about drug terms When regulatory and as bringing about than We hurdles

the So company relative going we felt that our best this transactions in really shareholders of other forward. many been of own interest have this the the to that was terms style to and transaction was done that best provided a

Chuck McWherter

Yes.

question MBX-XXXX. the answer about I'll maybe So

think I to points bring up. three there's

Just give of background you the agreement. of the little on to a nature bit

bit to we to just like. timing success look a of to asked could about; and Secondly, talk you about which the speak what factors think third, little would

that. by a interest So, Type view. X of Helmsley to and space patient in trial are we're philanthropic Trust, agreement, concluded to knowledge And needs diabetes really clinical we the compensated care. as And an for from expertise the together, point as regulatory we and material approached in advance advance expertise were potentially and an their who has where provide well only we

the from So and capital study. that which on metabolism conduct expertise us that. And engaged clinical there's deep TRI, to diabetes a CRO they no has is in

we're is, the means that we're running what but not actually collaborator So a study.

set. and is meeting study CRO responsible patients, quality this get the a so recruiting all needs for to And conduct the data executing to and

about or TRI committed believe as kind goal but and be subjects of we'll the and question the two-period next little our the to mean towards this to that quarter. fourth continue study bit which doing plays from timing, that perspective, but be able that the year, end a would recruit this we we into still to study confidence year. outside we around into is that there's hope And crossover do in That's that would is proceed update available really to early control, the of XX have year this give so, we you an your that data this the

understand regimen to see volunteer. in is is So, in well study, replicate blood look the would of with the which their seen I compared placebo, can in that as elaborate really designed human does as healthy studies, to to agonist design we the that effects of like? think conditions what success what say islets of rodent glucose XXX the isolated low levels a are of glucagon you

don't glucagon when of the the number. We and the a of ability conditions would that's so increase you're to decision. matters of of blood the inform release We data think specific hypoglycemia. And I a levels really have Ultimately, kind benchmark. really quality glucose the the let under data is what

I being -- a success levels area the diabetic explore that's think and look about this the studies key further I in experts response with factors hypoglycemia. think make you So glucagon glucose sense to direct whether about make decision together that we would to for of the as in would to would

Patrick Dolezal

Great. Thanks the questions. for taking

Operator

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

Matt Hershenhorn

opportunity I appreciate it. current as presented thinking patients Jay gotten Olson. I were follow-up curious And well the well. market Hey. recent Thanks seladelpar. have had And what that This might at the for inform cirrhotic for your as potential you Matt Hershenhorn a We in might physician our is EASL? from we feedback that. really about then how on mentioned taking you analyses questions. just for And think to

Chuck McWherter

thanks that question, Well, for Matt.

to answer I've I earlier to think, something. you're think, tried my on I in highlight as

of obvious those high there's for with cirrhosis. need PBC, course think, an patients unmet I with especially

course, the patients recent are do for is include to alterations And there no able a around interest also population. Ocaliva, Given with off-label agent adjustments that in the as the heightened in to the be to concerns used well of that might mention as disease. in advanced an -- liver bezafibrate label labels around doubt having be that, with cirrhosis. I'd language fenofibrate patients used

trials that systematically their de-risked randomized So particular been those use in in -- have not patients. that remains controlled an agents in been cirrhotic area terms has of

So of the that's feedback. kind

profile discussions encouraging experts. confirm and that I been us expand able I the our had think And studies to with boards. we've continue We've had so to to they're we've advisory far. see think our

focus where sure forward seladelpar spectrum make our So of I understand to think across the is disease. going that's really is, we

Sujal Shah

community, PBC Matt, XXXX, consistent. And the only add from think it I'd we've the developing medical since Yes. to in is as the thing been those I has in sentiment here, continued be seladelpar

of run continue patients. multiple we of generate PBC, medical at the I meetings, to think, multiple meet in the transparency, trying sets they share appreciate full in needs studies we've the data interest to that really with fact that

medical is presentations as year, rich their continues that needs turn rest can sets as addressing patient input, we population. the well. from the those something meetings as panel This through So of the of as have many have we to We very of of think among experts the data frequently. is Chuck mentioned, about to the around, discussion to more have committee close. look the how we And of at advisory seek the and of that with continue in these relationship this much broader we interest significant to continue we very sets the mine, community will

can that sets opportunity them, fact data going work for to to we're that continue seladelpar. we in that closely to So generate so with continue broaden

Matt Hershenhorn

your it. you're Thank thinking also Makes And perfect partnership and Okay. had is great. I that you. would other I And sense. the NASH potentially Got ex-US? was PSC, appreciate in on thinking current for what question there, opportunities, be just, it. current PBC

Sujal Shah

Yes.

fact significant work. a with now think respect I of to PBC, we entrenched are in in amount

in broadly outside believe we'll just the US as evaluating US not but and in year Fundamentally, the RESPONSE PBC in for both more look to US us the Europe, this and mentioned, well. just outside doing Day the Analyst a later approval I for positions market seladelpar we regulatory opportunities Europe. As

to patients we're interest in ourselves, as least as and forward to continue march strategies at the level US And the going in continue so, many of the basis the possible. to US to strategy taking the of seladelpar, that, patients assess outside on the seladelpar hands of with and to get of also

from partnerships. will come of very some potential likely And that

as to this to that recognize well. upcoming explored. We at that about be as So we'll we more something work you to and do is continue those calls speak here continue

Matt Hershenhorn

it. Thanks, the Got for Congrats on progress. questions. the Okay. again, taking

Operator

Our proceed next Please Young Fitzgerald. your question question. is Cantor from Alethia with with

Emily Bodnar

taking the NDA? the for focus study? for Thanks safety what in you on This it's curious, to since ASSURE mentioned that file Thank And you for on specifically long-term Hi. Alethia. needed the looking are with FDA questions. you our I'm asked Emily is to there, has you. anything a data

Sujal Shah

jump safety large that large, field that's possible. an and We in us special the NDA actually is study standard to open-label as NDA a database timing, ASSURE. if the safety to even think to and course, filing, others we're data data, as as with we to study. need we nothing Phase and even But here robust patients of well alignment as key have relative to can advantaged amount out in I of have three as to patient This we're continue answer NDA in our that assembled years. that and with commensurate at as as the one the filing. to efficacy required, that is longer has just database well. patient study of this been Chuck monitor safety miss to we patient ask to longer-term likely robust year I registration agency collect database larger open-label prepared RESPONSE in safety if experience of to as to the X a We've about full And with There's Dennis opportunity may Fundamentally, in of Happy with our with is file in two so not long-term, asked X Yes. time study. and as think of the ASSURE collected question that We think taking safety ever seladelpar a than significant we're the collect gives data ASSURE. us Phase as fact timing collect anything.

nothing we advantage in but fundamentally, or in the So, us, it's think called an for ASSURE. agency has just special out particular

Operator

your comes with Leerink. from SVB Smith Thomas with question. question next proceed Please Our

Mike Kratky

Hi Tom. everyone. on for This Mike is

outweighing patients? drag is point, enrollment COVID? the you PBC study. competing against at for these on Phase are from of confirmatory these subject And RESPONSE competing and the do studies more seeing for the extent patients ongoing impact studies that impact you the X from feel To enrollment actually on Just specifically, what of this ongoing other for second-line other the

Sujal Shah

That's a great question.

IRB I as we fact we more headwinds. really at we've facing the is the think in outset very recognize And And first global and competition initiate execute RESPONSE, is that the that would see fundamentally say clear for patients committee ethics second impacts really two-key to been that we're the sites pandemic. previously. global course than is I the slowdowns pandemic. and We had of ability approvals in we get ultimately really our activated. one the number headwind that

challenges I impacting in the of clinical that staffing at in have think Some terms many as sites various lines. others well the of time space experiencing been

think in to I here have competition, robust facing. respect an we're most are advantage. we With challenges these think I fact the

patients. ongoing. think, there and that's seladelpar and CymaBay a is said I X all study recognize, this Phase our other study. Having we is of disease execution again, advantage Of fewer the course, for to And set that, we're seladelpar significant relative that of the There's very through. significant things, think, seladelpar. is in advantaged is patients. the using patient again, medical with the something But also that great significant working data community I in competing There's one PBC we're around, for experience rare particularly for for and are a patients experience

had. relationships leverage specifically And leverage so for it's those experience us the we've to to route important

see initiated enrolling. fact where I to some actually not think are or go other studies us of you'll in countries these

of some experience well. as our prior actually comes that from And

So, a your it's think significant more of quantitative But I broad say, are the the global and challenges more perhaps. to fundamentally, answer question. can't I pandemic

Mike Kratky

and very Thanks taking it. questions. Got much it you appreciate our

Operator

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

William Wood

make first-line study, Mayank group for think going the Hi. Mamtani. Yes. RESPONSE you're an can thinking for This intolerance you is UDCA. William therapy? Wood you going broadly after just case in Do after on -- I to you're a your was

Sujal Shah

question. That's good a

that The as estimated me are relative in small majority to proportions that fact UDCA I think X% out of patients vast to take as second-line patients that RESPONSE. are patients may point of or have first important the for intolerant inadequate an XX%. to that treatment proportions be and UDCA require

actually not majority of of label progressing be how nearly is of population so set a to pathway nevertheless, it's here the In how The seladelpar To So would responders. the intolerant on That subpart addition the is setting of UDCA. patients been I'll in patient that ahead allow are for is UDCA treatment label for on in inadequate – those and in population. would what envision UDCA, for use. we approval the top really approved has UDCA, to you this ultimately use and remind H actually outcomes. and approval. very study of to accelerated seladelpar is order disease first-line course slowly the the in

patients. decreases thinking And seladelpar. that markers so really expanding not above when the the disease outcomes case patients we the times for or know X.XX it's Those classically think we that from as alkaline we translate that of that benefit in X.X defined not line biochemical even limit of into for use can and are about additional in can patient long-term potentially are still about second think population it's normal, where patients seladelpar, upper have elevated phosphatase of segments if improvements

potential generating of case. in that use areas to support about And we sets the so those the of think future are some data

William Wood

or are Excellent. differentiation possibly an it study? you label? NRS Thank specific you make the your very how much. in itching how to then might evaluating And RESPONSE specification And

Sujal Shah

Yes.

electronic I XX-hour XX. it an scale months. patients a we're in we're the data actually That's and their a having think using itch ultimately numerical rating collecting worst to here we're period scale collecting So the diary record and using six by zero over how of

compare the in period from six-month is the key to endpoint so study the that change secondary baseline in itch in to patients. And these

So had as same similar in endpoint well. ENHANCE of we course study our the as similar – experience

we're scale, patients XX the the compliance importance good PBC. setting point out collecting questionnaire, these also itch additional itch of of I experience, we've So from good endpoint validated in PBC got the are in key XD although – we've will scales collecting had that PROs, NRS. is that to regulators, the

I approved There in of really setting all and differentiator to this as ENHANCE, once the have again us key hitting PBC. UDCA itch. potentially on has treatment or know placebo second-line of itch. we with first-line versus I the shown drugs think upon pruritus successfully for necessarily no can here a patients. are positions benefit not well did benefit, that not worsen treatment a also in XX pruritus And shown we is milligrams endpoint a think at cause PBC Ocaliva

months three to So patients on the did of again, we study life they that significant once changer impact and statistically benefit that in RESPONSE, to meaningfully today. if think really alternatives it really the have quality a beyond could we treatment and be a any we're as for their game and show itch able in ENHANCE improve access

Dennis Kim

of to need I outcomes for in we powering study this gone outcome the if terms composite efficacy as us for especially, powered because bit add, might a for as response. such And we've the the patients we're important as little an what well, and adequately is pruritus primary well the beyond of for that endpoint

at change So benefit hopeful we'll on the encouraged RESPONSE. we're a and of see real end this that endpoint

William Wood

And of vision. and question, make last then And then sort more you you that. external mission think if to allow large that programs appreciate sense Really a creative company's received just of the can yes, Do strategic a given funding. amount Excellent. pipeline? obviously what be non-dilutive building in might you

Sujal Shah

question. Good Yes.

funding So seladelpar specifically to agreement this in be is clear, for very PBC.

with fact us sources of get PBC. continue to our the being dedicated future and finish to in across all does centered to sources non-dilutive expand So in to the transaction your however other to proceeds the are capital pipeline. Abingworth of It around seladelpar open up this of point capital capital line

in I foremost, success. this believe, should foremost. we From say that, believe we want firmly continue be first and and on PBC that to we First to at here focused can centrally build we seladelpar upon all company

the Helmsley creatively the us being study funded to MBX-XXXX exactly example by programs, IIa an Charitable is seen that. Trust ways Phase fully find other of move You've

capital, a But history of to focus all broadens fibrosis, look other potential other future and that disease. long and in to we really continues and We've think even beyond this of us sources success for assets. focusing built allow particularly that. liver I expertise as on inflammation here

in we with rare we'd need, to on fundamentally, disease high unmet but to settings these things will think that like the to I continue explore continue stay future. focused be

William Wood

thank my questions it. Thank you, so much. Appreciate today. And you for taking

Sujal Shah

Thank you.

Operator

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

Thomas Yip

COVID, Yip afternoon calling it with good XXXX, in impact for PBC everyone. Good points enrollment RESPONSE take year-end pressure Hi, over where Thomas some is studies. like by still restrictions This time enrollment by With full see progress possible, to the Ed. sounds renew is lines? but COVID impeded it could can

Sujal Shah

start you of the not discussed for Look, give kind. the approximately of that about in activated to We took you months Thank to the patients. first a sites as patients randomize course Yes. because to in a begin. four it's idea to enrollment global only I in of you clinical this growth you months. exponential to in a gives question. past, idea when that experience clinical atypical see lower study, some have we've XX% ENHANCE XXX the X% is It months of atypical This enrolled had in you not some first it's we total studies four important think here the XX few

encouraged. we've sites couple last number that months, we're meaningful the over added very of closely a continue area of and looked you've an today. be That's If focusing actually to

slow talk some getting you times in to those fully when and alleviate about So, sites activated. turnaround points, contracts trying we're pressure of are

our our screening further of the and really site some a increase in at can and successful concerted are which we seeing those experience, prior to activations, ultimately areas effort then in an which in So be randomization. leads with that know we acceleration

pointed quickly you the as focused out, enrollment on as this as completing study So, possible. in we're very

But progress I there think keep as to eyes importantly, towards because through are to our the the Delta continues of continue particularly variant the spread. end We unknowns, year.

message make it's also sites patients sure for year. Given enrolling in us next to sites we have we first our unknowns, half may to of know, keep and those fact to the into we important that active

mindset. So enrolled We study quickly as want get that's to possible. our as this

additional towards restrictions with focused in respect we're of now, pandemic, but uncertainty year-end, countries. that recognizing For the the various to some

Of as we're likely some have to to further. very we progress impact, course, continue

So, as well. clarity updates we gain to more to it future quarterly we'll look as provide

Thomas Yip

That info. makes a lot that of sense. Thanks for and

Japan? sites Speaking to China of any open today. and XX in mentioned are clinical over Are open sites, there there plans you

Dennis Kim

are of can countries the in those on not you see As activated the two list countries. clinicaltrials.gov,

we see as So, updates will more give you them. we

Thomas Yip

Understood. Thank our so look again to with taking you, forward much. Thank and you you. questions talking for

Operator

reached over I'd the of comments. end to like turn Mr. have the We Shah question-and-answer closing now call session. to back the for

Sujal Shah

of you, all We joining operator. Thank us you appreciate today.

relationships year, we and confident we experience the focus PBC. our team's groups and in half into As in have and the built over I'm to move our second of patient years ability with in leverage investigators advocacy

medical this look updates year. III meaningful and have at AASLD, As prior share I PBC look also where later Analyst We sized evaluated mentioned, you. through updates sets providing to we will will for year. any Phase and approach also you to forward opportunity we we be as II mining the the market will hold gathered Phase further we these rich highlight, future meetings studies we have data Day, And seladelpar. the we to potential and look progress Thank how to a our as we through

Operator

conference. today's concludes This

for You we lines and at may your you time thank your this disconnect participation.

Cymabay Therapeutics (CBAY) 13 Aug 212021 Q2 Earnings call transcript