- Earnings call transcript Q3 2022

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Operator

Hello, everyone, and welcome to the Mirum Q3 2022 Business Update. My name is Emily, and I'll be coordinating your call today. [Operator Instructions] I'll now turn the call over to our host, Ian Clements, CFO of Mirum. Please go ahead, Ian.

Ian Clements

Thanks, Emily, and good morning, everyone. you Pharmaceuticals welcome Conference Quarter to Call. like to Third Mirum XXXX I'd by CEO, R&D, President Chief joined King's our I'm today and Professor, Thompson Peter our College London. and Operating Radovich, Chris Head Vig, of Pam Richard from Officer, our Peetz, third Mirum the of this results morning, announcing company's a issued Earlier XXXX. release news the for quarter Copies Investors Section the of found be in news this our release website. can and SEC filings of

from to the our Thompson Professor on call the exciting have updates as full be quarter issued discuss earlier updates AASLD, results rest morning. on III we week that Given and can on XX-Q this our found presented Phase today the Full Study keep MARCH-PFIC at to our details in of release the brief. this we the intend news

like With and course results on are Chris? subsequent under development Before making the forth be anticipated and call over risks and to and and our differ said, any regarding opportunities business expectations, with is XX-K Chris. for and forward-looking should like that uncertainties, Mirum's SEC. results to I'd that guidance. remind to December these in they subject from XXXX, materially to financial reports risk ended the by actual XX, conference Investors forecasts Mirum's numerous this statements, we statements. call, programs you to begin, set these turn could read factors current and year update of the market Mirum we'll the the filed strategies, no during prospects, I'd statements Mirum based statements about certain including duty management's programs, the plans,

Chris Peetz

and full last call outside of Setting new and great to commercial LIVMARLI couple Mirum vision today into first of and pruritus CHMP made And we join On strong over we've the joining of a rate to label and treatment with the than everyone of advances growth opinion study. quarter and showing a major pipeline. the we for III approval. higher Phase X a expansion packed bringing hitting potential continue a earlier genetic based participation PFIC remarks the the us Ian, with recently our on today. stage the for you, medicines for life-changing syndrome the our next Alagille countries patients realize Europe age year as milestones key older. morning to for from is for performance III months to note, also build is in the our response for the study. MARCH-PFIC presented U.S., cholestatic share brief for of Peter good in that the commercial for Alagille recently are year outcomes was Thank and with launch further its diving track in in years demand I'll Mirum in an moment well supplemented have sales where received year-to-date, The U.S. clinical with of Israel. launches the business markets the a by recently delighted Thompson around us MARCH-PFIC with track Peter? have rare in syndrome world. patients in dedicated on These net in before data. results product The details And Mirum at about on and call the both for and improved the that, XXXX million LIVMARLI to studies, Professor stride of of from It net suffering million pass to $XX.X possible for from positive diseases only sales excited And like value realizing the estimated the $XX the continues approvals The European patients. we're team announced be of will partner approval the positive on the we're treatment potential researchers. data we experience on Phase type on to broader

Peter Radovich

Today, quarter upcoming commercial plans product and third around net Thanks, Chris. for the share LIVMARLI's sales color I'll additional business.

million the with happy continued are year quarterly million. are growth guidance we quarter, the We with third in net LIVMARLI raising to LIVMARLI in $XX $XX.X full product sales net and for sales estimate

patient population observe the LIVMARLI to and reimbursements. in fall, high the into We we've persistence reaching in continue LIVMARLI to seen continue Coming United States. early acceleration in to and the full maintain Alagille believe syndrome levels new strong patient compliance starts and on an we're of addressable

commercial profitable, markets. international QX us the we turn in to first online of come the XXXX, which is to Our following positions others XXXX strong into country Germany will be in as over with of well and course XXXX. business and launches European

receiving And Alagille Beyond XXX and our markets to access we partner transition patients clinical outside and programs. the States, upon to United from will approvals. that our approvals also to commercial with LIVMARLI will On over expect eligible in I core note, XXXX the patients Pam? Pam. are see turn be currently months. These over U.S. next local XX expanded EU over LIVMARLI markets, to the to from contribution to occurring XX call several

Pamela Vig

Thanks, Peter.

in where team Liver pediatric data. presented our from returned Meeting, to Over for diseases. continued we has last just LIVMARLI late-breaking exciting take cholestatic strides the advancing few We've AASLD the months, major

year on X those in infants children to reported These results safety from of in similar than and X to we observed greater months tolerability in X-year age. infants observed were PK, of Alagille age age. of First, with

with was no demonstrated this analysis. including discontinuations diarrhea from showed a and also that well tolerability disorders, data, We GI-related real-world safety GI-related which tolerated only X% and profile

these use along has really in impact observed LIVMARLI aligns lives. tolerability product, a of X-year been patients' the the where feedback very profile, received. efficacy Now including that of previously this with well highlights Alagille, been This make the in the outcomes the profile has the data presented commercial from to meaningful ability with

that hand by PFIC MARCH not study late-breaking The a are studied. gone presentation Professor? Now thrilled we're enrolled now doses until have played MARCH-PFIC [sleep] has III magnitude that response by secondary of the through study, patients, types higher met PFIC Thompson. rates across to Professor expectations. PFIC the digging postulation PFIC in and set primary study deeper. the greater that magnitude XX about we're to our excited to been endpoints patients largest will for and study it effects exceeded Phase is to higher questions have its translate And said, PFIC this types so means observed the and of we looking most forward pleasure and the that even unanswered, which would is over Thompson. a Professor response the And our endpoint conducted, help included data previously with answer and The what of

Richard Thompson

Thank indeed. very you much,

with pleased circulation pruritus these following of liver for consequences historically -- patients characterized these a quality a I the cholestasis, particular number genetic day, interruption disorder pandeficiency pediatrician, which acids, underlying study really is every bile go XB PFIC primary the are off diseases, Pam's consequence who will drugs, patients the threshold straight most which they there, export many and I'm is I through they on important been have mainly see are the do in of a of we major treated what But that of research with but in been The cohort, to described other atrial but basalt group have deficiency a not Slide because in those not progressive talks also life thing will than we FIC-X of flow. introduction, this impact which the as surgery that progressive for have the actually data surgery have And familial a but this particular, an XX% got as is stoma is just bile be requires and surgery, all through variety has of improvement. approximately and chance frequent looks into and the disease. have patients high they clinically, present benefit there's fairly drainage which of and and GJPX jump conduct enteropathic about genetic causes treated form The is important. So intense we've external from deficiency, used after disorders subtypes which deficiencies, of some have which all the family. is this XX% think to of these way, then most bile myosin and liver by them, are because composition this X on abnormalities of abnormality other intrahepatic is in But that helped significant in consequence focus PFIC are you today. of quite PFIC it whole think surgical the you X formation other a to who able believe of thereof rather pruritus children the can as I is, particular offer which believe, with the of which a of types and of of cohort have disease. slide children Previously, the liver to is a that disease patients I of these we off-label I'll have

form majority of transplants up repertoire which in Historically, risk. ended of consequence, an the effective obviously, considerable limited childhood, liver major a of treatment is but undertaking a As is our of treatment. patients with have associated

available now for IBAT delighted alternative inhibitors are I'm an becoming as the So and that conditions. treatment pharmacological

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study, the of seen those and liver survival used native this study and less transplantation. study. in with you we dose acids. in in who And children initial this have X see prolonged pool is do II respond the certainly which in into was from bile the capable of -- a serum got And marker Phase peripheral results you Phase The drug elevated moderate in receive the Phase patients. of we XX further they study the the on those pruritus, right-hand I and [Indiscernible] open-label patients maralixibat top to bile cohort, this pruritus the previous residual in The acid considerably the drug have acid but Phase reducing previously the XX% months over And been referred study right-hand the XX, full potential fall a be is PFIC was deficiency. majority vast III [Indiscernible]. X study study study. BSEP marks II which cohorts for a myosin on either dose than is we shows age, studies, they those II BSEP quite FICX other that's [Indiscernible] so as this randomized the have function. MDRX, or had shown that to into where patients the to the with with our placebo. shows bile vast size schema III are important X side BSEP cohort a difference patients of Slide will studies. of seen of of to measured be deficiency, by liver as the system on randomized this was [Indiscernible] is other associated for higher an box category, have TJP maralixibat persistent think with that the below of the to PFIC with Slide Phase with majority, maralixibat Combined than serum the that The -- distribution XB went had has As entry the we've had improving deficiency or I -- primary of non-truncating severe corner probably XX-XX on some on

marker improvements biologically all cohorts, but of look right BSEP on So into change in I'll which a included I responder analysis right-hand the serum patient published. right-hand which some study, at growth, in cohort. in on groups, a are the got I secondary number other important which full discuss. in details particular, but the endpoints times, distribution more shows by cholestasis. hetrogeneous. PFIC end combined the on to And the primary are other and And showed of and preliminary of for of at pruritus look you as the Range biologically the they you all acid data, are the at shown bilirubin are X was the is which endpoints than important then bile the on the in enrollment data particular, which cohort, safety different of patients today in the by on has and PFIC pruritus, basis, then form PFIC patient a BSEP the well. PFIC in point as clinically the on the clinically BSEP of those that changes the along show data on improvement terms cohort the changes tool, that's with bottom we'll measured rest important, the old haven't data I'll of The as Slide then then cohort show presented you of We in to side, in the previously the secondary today, [Indiscernible] those biologically the the cohort endpoints side, [Indiscernible]. and ItchRO cohort. endpoint improvement an on primary with been time group cohort A an and side. I similar left-hand clinically is which of I'll the such in

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on bottom, at you the measured this both to cohort think baseline. all using the variables the analysis XX model to repeated primary all and as these XX assessed endpoints to measurement which mixed overall, the the see Slide you XX can height score I baseline. failing shows compared and details weeks interrogates by in thrive were technique weight data were at from of

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continued persisted longitudinally the side, the week fact, exposure. steady to pruritus and and very treatment On be with to the over other with few and the to of XX these you diseases was XX response of for these months on represented of you other see the separation similar and left. Slide And duration can of right-hand anything else, patients. weeks the if the greater listed a placebo data The for will clear state placebo the cohort approximately in even data appears anything, group of then of if X treatment, improve PFIC about treatment months the obviously, same group very with from have early separation study. top first in additional other shows again, patients significant and maralixibat highly X

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associated XX% PFIC when reduction based we analysis, transplantation and we [Indiscernible] surgical hand baseline right XX-year circulation, in on is a terms we looked or the the was to per side, reduction -- a reduction derive bile our This in surgical use at interruption strongly statistically On liter, of below the certain with which the in cutoff in all micromoles the a used XXX from in follow-up. cohort. avoidance retrospective are of of acids. And cutoff, an here of response the

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side ended variety hope maralixibat III PFIC in we the told patients those. see, [Indiscernible] what PFIC. you [Indiscernible] As placebo of disease. were all where that patients that's from most was just to expect the you of subgroups. learned that X now up side would consequence you think use gastrointestinal significantly responder frequency bottom -- in of significant previous of the the but treating and those is highlighted demonstrated acids met. severe with events in in . stool I treatment And which The clinically colon. patients the capable is group and study. an Phase deemed summarized it There and at these does included only just into comfortably, None increased greater majority like to I've other across a unhappy study there undertaken was studies some in is secondary dose mild on was very much endpoints It in we've vast and optimized say in this this study, we've and maralixibat a lead which the resolved acid dosing were with producing the II as bile the of a Slide of improvements, I early is years have did both look It the study, discontinuation. to most mild. some was a this bile diverting serum And the and first primary was really pruritus, significant good drug And result a One manageable. of can particularly in adverse effects, this, teenager of improvements are deemed are difference adverse after tested. was expected and and who one ago Phase that seen XX rate they was Mainly between the I really There those the think in that maralixibat, mild usually with compared to diarrhea between the I effects treatment-emergent events groups the much that discontinuation that of studies. of difference patients side do observed was summarizes is effects I've not with have slide deficiency particular, think diarrhea, and greater where I the study probably liver types that fairly BSEP group in the studies. severe, the less biologically

the called of [Indiscernible] surgical study. As community, NAPPED circulation study pulled survival bile of I the in the in enteropathic And across was included previously in bile enteropathic the showed that NAPPED also all acids study, improvements study, acids producing interruption together academic correlated similar study native bile as looking said, clearly, cutoffs a basis is at which serum the the that we of of changes on it like PFIC circulation liver looks identified different in of transplantation. interruption academic an we the And of of through in types surgical audience acids. the of with

am to of I collective the patients course, the complex I also my who data. weight are well recruited trend think this in effects. who with using we've a looks height. the bilirubin scores, if early there's They're colleagues in As and all grateful study collected dose. are to patients no you, tolerated. this Usually large side monitoring signals, grateful up and shown on families nature. improvements Overall, in in put perform extremely safety all increased to like of on the new and significant manageable. cohort disease, it There maralixibat got transient a of I direction the is in data higher is right necessary But most and study and

study. So looks And after the on beginning, this patients. from who these as data as I said the far pediatrician so are these a at clearly,

important will got the reflection we involved still the in in now has got We certainly, probably now sense sister for my view follow-up. from pruritus. patients are who have in I've quality intense keep sticks her because out most of life my which is patients got point Stage years the ago she [Indiscernible]. And are study, delighted several the tools, that transplanted that help a and of II us control an that was years mind patient X the internal And we've

gone and the back moving studied that's if her vitamin II at amount we all. long-term acid. the which here so Phase be transplanted. growth. you on of levels you on pleased to about normal in And expect takes biochemistry, bile with that looked a of he's direction. cetera, X able very that post nothing patient study and She you and her that are we're if obviously of liver involved exactly [Indiscernible], is end normal, has now et years, got -- My after at parameters up D. but achieve vitamin were she have biochemistry. I'm normal do well has in is really, normal would the spleens her, are transplant. point was transplant, serum we've who for And wrong there in her that studies takes a sort to been to She normal. say meet point, necessary, course, has sister but And her the has say to maralixibat you clearly all done safety All small the was to

a it's think I years few ago. on So huge a improvement we were where

to will take very Chris. I'm hand but any happy So I questions, Thank you. over to

Chris Peetz

Professor Okay. Thank you, Thompson.

both PFIC a for to pediatric year As was get to line suffering patients you And and submissions the Mirum the study for the for we'll III Thompson team. open for cholestasis. promise the MARCH-PFIC quarter and line that, Professor can tell, the for with possible. next Phase questions. LIVMARLI. as for It Mirum convincing begin questions early open Operator, LIVMARLI family quickly please plan show great results We to with as regulatory from

Operator

Jessica first line comes with Our question Instructions] from today [Operator JPMorgan. the of Fye

Jessica Fye

the and Two, physician one for one for investigator. management team

missed for in talk about LIVMARLI prepared think to but how fast just you And that approved when acceleration forward mentioned think agents a management, new if with LIVMARLI I to for First Bylvay? remarks, and for patients? of deceleration And the wondering And see reported? for between acceleration, in clear how differences looks is an time PFIC, you between what revenue I you you physician, time LIVMARLI. reconcile the on a as growth I may the X PFIC when that starts frame select of you we saw like what can was then a those would have

Chris Peetz

on ahead line your it the Professor to Thanks, hand and then Jess, for top to second the comment question. and Peter straight part. Thompson for I'll go to

Peter Radovich

Yes. Thanks, Jeff.

acceleration $XX that continuing XX% raising acceleration to new see starts in So implies million to to confidence really QX quarter-over-quarter of in in gives growth QX. LIVMARLI. guidance we us the seeing the are in that in the terms of That's an comments, going what and from with fall, patient

summer, children QX U.S. XX every full once in picture the months. to in every at many did of the X mind we Looking the question, that these even your are keep softness see in some seeing or

months. of the what kind on appointment in vacation, typically position choose So going phenomenon where starts patient occur we as do observed families those not to new summer their

QX, we now. for in acceleration months obviously, fall most coming the the but and serving of summer that's into continuing the So what

Richard Thompson

neither positive are of drugs the studied as studied. of you a effectively are Jessica, delighted that all being and in we've IBAT disappointing, that X patients obviously, from course, groups I'm have So that got inhibitors And drug treats the there conditions. know, been response both

So, moment, that there the And They drugs. we at significant head-to-head a for the difference excellent the I do excellent rates done a that if an they're XX% think distinction an statistically the of PFIC, truth response response yes, similar rate, a to is this honest studies, right rate is in you would -- find the have between haven't between mean significant response pruritus. this and not terms do X fact X is I that comparison. you drugs. try despite XX%

greater table I that sitting odevixibat, statistically think that, the than is the but people will sure shown tell not around in I'm that me that significant.

if and bile to conditions view, transport. basis, residual picture determinants amount whether rates, not any think major on differences of have can is see individual see or the response into of between patient I this point in my believe from the of acid I dig these patient an a the responds we'll we

meaningful that is that's factors down between have other only sure Obviously, drugs. the we've territories, there's I biochemical but kind the genetic of if we way whether of do looking mutations we is look the important and these [Indiscernible] drug in dig be best factor, price the into clinically any something done certainly, carry. individual of any drug, formulation see an in is really of want drug the availability used. response, those of there predictors predictors to of deciding any that, at to patients really of difference will certainly, drug, can these see is I'm when some the at that and response, which the to come And to the So there's those into

that So to we've but impact which learn, got I pharmacological financial outside limit. it think there's there's also and factors they're which but on will my are other

Operator

Raymond Our from Seedhouse with Steve next question comes James.

Steven Seedhouse

extent being if comment. can there patient acceleration mostly that's driven wondering again, the through on you by prescribers you at patients prescribers? still any Is of repeat Or Just starts, that, point? I'm this new gaining are

Peter Radovich

more these we there's I defined. think pretty we well that see take both certain pediatric come terms that medium or the in I much board, smaller as it comes hepatologists U.S. of the I Quarter-over-quarter, see prescriber base of number the particularly volume. on But across of only is from mean not a centers the kind patients. from think in of the board. might do have care acceleration,

Steven Seedhouse

that interim at enrollment picked next comment then in in on first like, with some mention? you wanted that study pregnancies? with there's was noteworthy least after talked ask just an mostly you're Just seems earlier or of about hoping third -- need year. has trending it would And data you making this protocol demographic respect ICP changes about that anything for be the learning anything of Okay. you in the some study patients had is I and implementing to patients up year. that second you're seeking to half treatment could to like because trimester also unmet analysis just that first of I up number study the just ICP, enrolling It coming in

Chris Peetz

this terms we Nothing on in Thanks of that to for comment point, looks the question analysis it'd premature what until call the on be get out that. to really like to cohort. at specific the demographics

are protocol. for the next updated this and track We of seen have year on in patients come with half first

So the direction. I'll right kind of point it in

like Steve got have off. Sorry, you sounds follow-up? another you cut Did

Steven Seedhouse

Yes. the the about I MARCH-PFIC just with and patient asking was transplant. liver

of for share details any they could color you were ultimately that there? If the why nonresponders, elected

Chris Peetz

that, I obviously was mean transplant the that perceived my to and for understanding they family's reason nonresponders. be the is choice, were and

transplant figures making pretty to for obviously The the haven't all placebo families acids bile -- X on evident was X everything and the sure does the of were I than group, I the hand, group. hard were ones the them obviously, the not decisions deterioration actual X. [Indiscernible] that I'm physicians are of the of in the the but -- slightly were drug, the was placebo got discontinuations were grounds nonresponders them the authority went There not were and responders on clinical the decisions were on in was group. drug there think -- or greater majority in rather function the data. in of placebo than and making storing pruritus that But and are that

Operator

with from Our Mani Foroohar SVB next question comes Securities.

Mani Foroohar

fall can I want cetera, on us age, in weight, more any there's on if little sort bit mix of year, start detail And monetizing And as to patients any from you seen additional you're you [Indiscernible]. in you bit partnered exactly we initial for as mean that for the places how as patient be next geographies aware give revenue going on and a come to shift we where body geographies account to recent to Have of drill need opposed down we yourself? forward. of growth see this little look in dynamics et nuances coming coming a patients versus on forward.

Chris Peetz

I'll international a about then markets. it think international the the to the step comment pass back, of U.S. specifics on take broader when date. to piece Peter And the for kind in and we

a world, the for Western maybe in around licensing And quarter more and is time in these a in geographies kind what for at of building we markets a expect monthly other launching for that Asia. distributor at ourselves they partners Europe and that patients orders of are using the will in We time. distributors have multiple some

pharmacy and programs And markets smaller approach some an into market those in dive So they in you pass it with we'll patients. year. pretty orders when some come with that approvals for it we're where of countries little start doing a online order to but to it rare in, deeper. of in and see the for more to recognize -- could based I'll expected see we'll -- could through ourselves, start we for next to where to even, standard we And disease, we're of Peter this come sell that, as year in when come to revenue to early how the access then

Peter Radovich

Sure.

mix consistent that patients terms also in the were consistent really the patient On months, types approval, recent and of changes study is put with on in clinical program. pivotal the no which early in the body of weight, FDA with after there iconic age the

you more of go severe broaden product with be to for only ones we be about that average impact to life -- ballpark that physicians their you of the of are quality refer comfort patients are the Early then moderate. and will we that and comment the the prescribing first similar see Maybe they and of maybe phenotype. more most with get experience perceived of pruritus the perceived we've to perception kind see. and to gain to back we other to disease what to the days or the before, other prescriptions the that see age those as the weight if in is saw and So papers, talked kind patients the

seeing we're play that So in out quarters. the phenomenon recent

Mani Foroohar

Great. That's really helpful.

order quarter-over-quarter introduce On structure bit kind of your some that, markets, monthly choppiness I the a to the for have assume little and of distribution that? more it chunkiness is into am that kind numbers? reasonable dynamic we around of quarterly will Or overreading

Chris Peetz

point, just come that thing different several say countries. distributors, how in. see to there at this The and don't one the know, are I'd note several different of have of cadence we is I'd we the kind orders

be where our be So sources, smooth expect and we I a could this coming that's at of would chunky, thinking think lot different kind which from point. out to it of that anything do is

Operator

Our next with David question comes from Lebowitz Citi.

David Lebowitz

in morning with and another. comparability given such calculated And as trials the one study in how measuring on differences comment in versus morning Bylvay efficacy to in night. able overall, be you was the Would versus measuring just the

Chris Peetz

we from I can our how our speak a Thanks really what to high were know points mean data, That's for we question. end level, the calculated. in detail.

morning comparisons. say that is in on is that I not a make effectively position our kind same study of what across cross-study we any would comparisons can definitive see versus evening really just results. So in we the measurements, drug the

all it's evening, something and whether we've worst, of the morning, you we've across looked highly et had, at average, cetera. at readouts So it's consistent look

measurement So up. for We be expect that a holds wouldn't standpoint. that the difference to

David Lebowitz

in it. you you extrapolating at Got on and when data the look any how And ALGS? compare on PFIC, thoughts compare what they might in see

Chris Peetz

the Well, what through I in -- preferred that walked and arm we've activity. what results of of maralixibat can from have great here call, in look or if say over XX% program higher scores FDA the forward response the step one we of at at look forward scores treatment in here. taken a history, is a the what the is of on PFIC proportion the you at the patients. we see see are these When terms step for kind back what and proportion is impact X, huge step we X And analysis. a the of rates you days are of for much

of the coming out results study. So and great rate response

Operator

question from Our with the Yasmeen line is Sandler. of Piper Rahimi next

Yasmeen Rahimi

directed Thompson, us. much actually are and for so questions My to with being Dr. thank you

are higher you think belong when data the I The absolutely to you first for you to MARCH-PFIC, appreciate want questions a you mechanistic MARCH-PFIC, drive the this -- products is thoughts we rationale dolobaxabat, then adoption? be on about your you there you inhibitors number Any these And comments call on from your would at on to compare success, comparing stronger result comments would really to share if Mirum from the the another one. of having of is, X this so ICP and question then of where question is PBC. have a one know, And That's the look label but to contrast Alagille think you part with could is is indications asking morning. in many stronger, for of you Alagille a as X question could you probability IBAT that and into PSC recently I X data that the but and love hear other really question bile saw may is for there to helpful. third sorry take working acid. advantage critical

Richard Thompson

I are -- key the contributing the Alagille PFIC, believe So [Indiscernible] in the in questions indirectly not absolutely PBC, and those are as all Clearly, pruritus important causing know which a symptom part the very we I you're phenotypes, there and where production I sort conditions damage -- other disease just about merge talking of liver and the the initiation acids et from mean of of the an last of that damage but both think PSC, to clearly, bile to X are because do well. the different that are way cetera.

in the features. connection So to clinical as of draw disease offloading changing therefore, easier acids a nature to of the that between it's much the and disease the bile signals the opposed I natural natural and we're think history improving earlier something

Clearly, patient I seeing And And in reflection much disappointing. more component more I important patients in think the can studies the was got that once way But we're higher I think their of to view, in coming now predict if to on find the life life. clearly, of already, perfect need Alagille to we all the shown start and where disappointing was responses the a drugs initial is I logically I'm And a were to as which greater achieved And do with original transition mean, using the reduction that into or that years, it is not And should studies diseases, with point want improving maralixibat to because are studies still think dose I initially much other acids That's these PFIC, work in the these used. something when XX% that if as we getting. maralixibat and studies response. Obviously, acids I ICONIC that was here, are to view, patients think believe said all a where driver out next of the we've in our response. of giving higher such transplant of extremely and we're I which the they're of clear the likely that's bile we're the which know. to And that's importantly, some in my important early liver interventions PFIC, I and can actually heart of of [Indiscernible] clearly view, phenotypes point can to the response came a and we the avoidance in we're the real pruritus. that study I us say clearly, change phenotype. involved few I what terms sense because whether happen those retention from patient's is than damage patients. experience. any about previous and learn acids equally bile interested of with, From that in of you these in the about pruritus respond. we in we bile thing asking I is shown the in that's what dominates meaningful acid of an it's randomized do distinctions believe absolutely an of ICONIC driver is has the -- because from the getting bile it previous PFIC easier thinking as a that that liver that's that are as pruritus study studies I long-term involved dose they the get a in them, damage you think point in what the I've to pruritus expecting the we're compared of So these to the we've show been

meaningful we of good I how bits cohort less was interested the those dose And in I and we're And who as obviously, aspects nonresponders is original is patients is -- maintain also your respond, now of current important that and dose, as say, and I've did the increased got in So they're then point study, the combines that the what's responders years response, with in most PFIC What think dichotomists. a the my first to dose important response particularly long-term avoid the question was lower I'm are analysis the in that said, view. study. to one than can and whether responders transplantation. is when going X in we've the in who out. now responders the increased long-term the dose Actually, the on using still dose even because

a am questions. I significant studies number of just to So I quite the respond drug, do believe weren't there in to to original but made of because who a not in patients attempt failed of was some thinking exposed right they sort -- your to respond

Operator

next Our is Brian question Baird. Skorney from with

Luke Herrmann

that X/X curative, to Thompson, have also you couple other had that who get heterogen biliary the isn't you you accurate patients we've on so the physicians in characterize noted some doesn't I an the among this could atresia any estimate? have This Professor for think [Indiscernible] path ITCH transplant. one Do us with just other of And is a markers around who and and clinical FDA on successful X/X Brian. require an AD partially to Have Luke but recent And have subset. Embark procedure, immediate then that help this for is discussions management. a approval and had talked for maybe indication.

Chris Peetz

see discussed and do that to talk real the the what next on for potential can if in think the of pass primary patients I'll FDA atresia about go comment impact excited possible Thompson back what plans. the we the the to are those it Thanks strong potential portion first the for I'll agency second about over is at initiating the when to program Professor prior quite study, registration about we question. study. we I'm endpoint with the the to the in are have And year. setting it. drug biliary we'll excited X-month for the question, of And results, what

Richard Thompson

these the acid based think levels on [Indiscernible] I response the of response, to those observations responder long-term original terms we achieve in mean you have those that the peripheral not empirical appear the nature in bile studies, does we to don't are interesting the bile arrival So of largely showed based studies. I serum have surgical on to I based appear data for on although the the have of got progressive But in think obviously, is the do have normal, itself the studies. to pruritus don't we its us -- and achieve analysis acids analysis it's not by liver normal enough, data. And was down serum made now low but that is I liver to the fact the but only improve, disease. protecting liver and compare about that telling we we something

we'll wide need? the I And responders between question And is, sort at quite the data, you and actually level partial those Actually, at for of changed what means the significant are response responders So it there surgical a quite actually can study, the put think we result. off, exactly widely And transplantation, without avoid nonresponders. of away and really go is going do that was have if difference to to put it you it's look separation making instance? to what be similar the look you go to cut that detail. whether a back in

And separation. of distinction, responders actually, lower to responses the a which nonresponders, so reduction difference gave us although the below or really made off the that lesser are improvement are cuts micromole best of XXX XX% a or little level

day, as And a the of expected marker was end the whether can at data those but time acid make And remains whether results So be believe those obviously, But reaction same. we are bile it to we of using serum being believe clinical is seen the But we drug, for biological decisions. are that of that be to those correlate. see valid. the improvement the with we the have. the reflection have that we those be equally sorts the will the obviously, will data to

Operator

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

Thomas Yip

a Ed. the [Indiscernible] margin patients? study Among most in consider couple LIVMARLI be which asking the data of in for of Thompson. Thomas we've for seen one to important is Dr. do that This and first to Perhaps questions PFIC, attributes you unique

Richard Thompson

I avoid endpoints. linked going from I to be I able important to that say, I mean, meaningful to are patients. going are are it's attributes we're what patient, to The most a about clinically that -- to improvement. the what say And chances that, to as that chances think the these And are want they're get a transplantation. also

I think seen as results them. so And the you've are,

things long a transplantation -- discussing long-term them. on term are said going and X which described there's X/X chance on I'm but I'm with to basis, be clinical that improvement And the at from So those avoid to telling that it but they to I've be pruritus. so looks I'm patients have these results the that based going get our be like doubt, will extrapolation to meaningful going the a far approximately we in that XX% data, be the the to going from they're a delay a in moment, No previous able clinically chance studies. there's

Thomas Yip

some PFIC Understood. differences Can interim one perhaps potential the of [Indiscernible] your launch, strategy population? and the overlaps between outline are components between and U.S. PFIC And Dr. question what Thompson. LIVMARLI's you for team. key

Peter Radovich

take the to and Alagille. that to of terms on largely care comment identical Alagille I that. strategy, And -- think patients health to Happy take of the care patients. in are physicians care Sure. commercialization professions the -- Physicians

the going already to as it's products same upon and heard now centers years, right, for X-plus would PFIC and groups years. an LIVMARLI So obviously, we're both patient promotion the as other working in, back that approval, for demand promoting with available. the today, Alagille's include stakeholders We've syndrome, Alagille the you've been already FDA

if patients addition access with are world. have the other from receiving about we expanded comment only to who LIVMARLI around straightforward make thinking perspective. pretty an it programs you're is and clinical So currently SG&A from Maybe the about our XXX PFIC

the converting part early will good clinical of upon a So approval, drug. to effort be our receiving patients commercial drug the

Operator

turn we Those remarks. questions today. for are call the Chris to all concluding back now have I'll the for

Chris Peetz

joining today, Thompson and joining these thank to steady has you, great us a hope and I call. all for for made again, families like today's investigators of results Professor the Goodbye. operator, thanks, everyone day. I'd that possible. Thank And everyone,

Operator

us may Thank call, for our everyone, lines. today. you joining and you, your now This disconnect concludes

Mirum Pharmaceuticals (MIRM) 9 Nov 222022 Q3 Earnings call transcript