MIRM - Earnings call transcript Q3 2021 | Mirum Pharmaceuticals, Inc.

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Operator

Hello everyone and welcome to the Mirum Pharmaceuticals third quarter business update conference call. My name is Victoria and I will be coordinating your call today. [Operator Instructions]. I will now hand you over to your host, Ian Clements, Chief Financial Officer. Ian, please go ahead.

Ian Clements

afternoon good and Victoria everyone. Thanks to I third to Pharmaceuticals you XXXX would conference like quarter call. Mirum welcome I am and joined Peetz, today by our President head Operating research Chief Pam and of and Radovich, development Peter Officer,. Vig, CEO, Chris this the results announcing XX, for news September company's ended Mirum a quarter Earlier afternoon, XXXX. release issued the news SEC section filings this website. our of be found of Copies can release and Investors in the

differ statements making that current call, based subject begin, the uncertainties the call subsequent and we I September read be forward-looking Before I the the any Chris? programs, over we market and the and and from conference With that are turn by statements. business of in could to this on statements strategies, to Mirum would forth quarter you with would reports about for set opportunities said, development to Mirum's like filed results actual course financial during remind is Mirum's Investors will commercial to These and materially these expectations, certain Peetz. statements program risks XX-Q Chris and and ended forecasts plans, prospects, risk should regarding the like our including anticipated managements' SEC. results XXXX guidance. numerous XX, factors

Chris Peetz

And joining Thank us on good everyone Ian. afternoon to you the call today.

of for impressive quarter. achievement Mirum. launch and in this is year the was first from team a The one the for with of older tremendous approval FDA the by for syndrome, proud I very exceptional this LIVMARLI medicine third pruritus and the LIVMARLI's marked an patients and made quarter FDA product Mirum, ever Alagille cholestatic in period approved strife approved am indication our

Mirum to again The investigators, got again patients, excited results. about the caregivers, all the team parents, to work to and my advocates community humbled means hard are I that it and extend the done. thanks is We appreciation would delivering like eligible and it of what Alagille and employees

preparation efforts, have launch in the strong resulted our many starting our Peter LIVMARLI. a an commercial market. on including multiplying prelaunch the Today, to to world. companies Our update efforts around will speak partner

a also LIVMARLI. the a a for milestones: significant a of in recognition. presented important for Europe Global untreated results clinical the We group. free of Best presentation Liver improvement Overall reduction Alliance and of are data received cholestatic lead was believe the several We seen. syndrome. presentation Meeting, oral in revocation This similar that part was quarter transplant showed that at from patients XX% and survival remarkable. today regulatory compared late-breaking Alagille in Meeting short nothing of patients submitted liver And treatment efforts. submission. the Alagille This witnessed AASLD just studies in today, These with data the landmark risk to Liver of disease highly our these LIVMARLI was

the call over a would couple updates like turning the to Before on team. I leadership of to Peter, share

who Mirum's the next since First, is as of and approval his professional the best leading who thank like I served embarks Dr. LIVMARLI, Tucker, Ed company. now in CMO him would XXXX, has of supporting endeavor. application wish the he We as to luck

our will promoted share is to of Dr. review Vig, that She today. for Chief to Scientific Development Pam our Mirum. ASLD call excited and today's am I Officer, being Research on Second, Head presentation

we the the days, Peter? Now seen. It's confident launch. update have but progress are initial early on an let's turn to in we

Peter Radovich

Thanks Chris.

that LIVMARLI. commercial commercial following off building LIVMARLI busy Today, prepare early we a the am at last month into for these and I a excited commercial Over quite the have of availability. It's our the been world-class conducting to here off Mirum to achievements of months, paid lot of have to insights team a share over XX start. efforts clear work launch as is strong a launch just

that FDA there specific occurred was revenue third revenue not recognized on no LIVMARLI product in or we as providing September associated quarter are in XX, approval this of third the report, the earnings quarter. Given commercial metrics

the syndrome previously We which provide based update on results an of That XXXX of expected medicines. to Alagille observed pathway start faster-than-expected said, that perspective, sometime rare been year. payers. weeks several by has a performance with very next being launch pleased patients have majority are will the was acceptance we disease From on in a an We QX access early the we great the early in reimbursable first have what seen of comparable commercialization. communicated we of half

today, of access our have patients Alagille months. exceeding a reimbursable the the majority syndrome goal pathway States in of As several by United

the in have commercial three States pathways with reimbursable major claims payers established for example, the For approved largest United payer. each

several Medicaid and agreement the signed On systems. we have their product in CMS front, states as rebate a our LIVMARLI reimbursed added

by receptivity LIVMARLI the by the healthcare pleased professionals. We have very to also been

pleased ranging program as reached minimizing October our all centers. patients XXX team and LIVMARLI MAP and accounts launch. as weeks I community-based been any expectations programs that or has accounts to-date, base prescribers once Our as seen we dispenses of a in Mirum top the and Feedback very from our barriers. say positive. patients the of the in from well in according MAP prescribed smaller prescribing MAP, from we has early as timely well have to seen the physicians include have ensuring of field have is timely of to to top delivering on am financial that Access caregivers to broad Plus The access as goals country

agreement as have international. their we past leveraging we Takeda for in into LIVMARLI disease quarter, hepatology. Japan, as a commercialization entered with announced in to that development expertise rare This GI and Turning well

licensing Korea, the of or each East, South the Europe. Central deals distribution China, Beyond finally, and Eastern Japan, we Baltics Middle LIVMARLI Russia entered for have Greater into Israel, in and and

which capabilities of team in partners bring commercial knowledge rare own geographies a these nicely tremendous disease local the commercialization of in Europe. the our of complements depth Mirum's U.S. and All expertise, and

updated towards We a LIVMARLI. standpoint are and progress tremendous There possible geographies very as there. from pipeline Pam but to partners across on more our for keep will Partnering commercial patients recent we development a ongoing discussions and provide of also from you ensuring update working programs. our our both on data many group such our benefit ahead, as are opportunities progress. grateful are Pam? additional have strong share about will can and an dedicated

Pamela Vig

of excited further presented by a this LIVMARLI significant today To oral at demonstrating disease. presentation data Peter. ongoing are to approval, committed to research analyses end, a we with AASLD we the LIVMARLI's Meeting the so in utility in event-free In survival Thanks validate to in support remain continue Annual this share LIVMARLI's website and demonstrated history improvement drug you. Presentation and late-breaking that This available and transplant-free natural compared Alagille in improved is few with control highlights is landmark first set a survival I the for has data syndrome and a of event-free to devastating cohort. an interventional will time now in the disease. our section Publication

The set of age. is in years and between compared years today syndrome, Alagille history clinical natural interventional survival for reduction and syndrome. groups. demonstrated patients cohort available largest prognosis this that events the transplant-free Alagille liver evaluated than follow-up a statistically pruritus The transplantation the which with is the goal ratio patients of in mind XX.X data and a a Alagille this compare p-value includes pooled leading to six of two first of troubling among was six only the less the significant of clinical quite natural And over X.XXXX history in in a established the results striking with syndrome in hazard setting control time with GALA database, for is event-free this studies survival year a indication XX% X.XXX. the for by XX% X,XXX maralixibat outcomes rare it from this Alagille assessment LIVMARLI patients, largest keeping disease of presentation and overall against of syndrome, treatment For with improvement to

death consistent the in treatment for these in which analyses. included across the submission were evaluates expect test been hazard data of for showed to observed half disease robustness the And a XXXX. transplantation sensitivity and and cholestatic less Agency in decision also In EMA to this were our addition of and Multiple survival, have liver significant of LIVMARLI improvements statistically analyses These Medicines of due p-value a findings to analyses the second and a to and X.XXXX data from the the transplant-free we the X.XXX. syndrome event-free only. European Alagille than conducted ratio survival, subgroup of demonstrated

the predictors were time patients pruritus of work up event-free And survival event-free Now and at long Dr. treated in this years and event-free we bilirubin, Ron with to improvement predictive be of serum with on outcomes. acids critically, management pruritus six may bile XX second analyses analyzes also LIVMARLI. helpful LIVMARLI's XX of these late-breaking this of to term survival transplant-free by and presented impact was This event-free a that demonstrated Sokol. the abstract, analyses data survival. medical inform for remained supports patients treatment. of And of

ICONIC that Lastly, published Alagille front, our syndrome. patients LIVMARLI with are four The years very on Lancet study excited in we treatment pivotal of the publications with following

of III announce Now the MARCH to expect topline in Mirum's PFIC, progress. to second Alagille continues quarter of syndrome, respect XXXX. from to pipeline With maralixibat outside we the study Phase data

the As a PFIC enrolls MARCH-PFIC study reminder, subtypes. all

maralixibat biliary to enrollment IIb atresia. gain continues momentum in EMBARK Phase study in Additionally, the of

in brief the a adult update on Now volixibat setting.

committed across to We continue of IBAT potential cholestasis. role expanding the inhibition are

the builds underway. are program on Our registration adaptive of volixibat learnings potentially from studies and pediatric our design programs the volixibat currently

the primary And we analyses adults evaluate a am And the sclerosing Phase study in cholangitis of pregnancy. OHANA VISTAS in primary for I cholestasis share As excited intrahepatic IIb both to to expect evaluate the study that to and IIb reminder, said, Phase of studies is we XXXX. in call turn volixibat VANTAGE interim these about very And launched future to with provide volixibat the Ian biliary screening patients. indications. that of and active With financial are cholangitis currently we and to I all Ian? we of our Mirum's of a health. have clear over note, that programs update these an excited vision the on on clinical study will have

Ian Clements

the overview the earlier filed in with the comprehensive release financials this quarterly Thanks SEC. is our available Pam. of in XX-Q press A afternoon and Form distributed

section the mirumpharma.com. what maralixibat the in further all at of pipeline XXXX, our corporate our position third volixibat development to in launch be of will indications. their and both for both us LIVMARLI the website highlights Investors quarter find support we can I overview believe respective of of provide to which You and an of well our current from

in revenue South exclusive has GC Pharma. GC we from a First, million $X agreed this of license maralixibat Pharma Korea. received and Under to develop payment agreement, perspective, from an commercialize upfront licensing

the quarter expenses. supporting and cash, $XX investments financing with And of million. related equivalents third additional to close to at have volixibat for note, other at clinical manufacturing and following, public with voucher review September personnel expenses voucher. and with in of in $XX.X arrangement or financing The Looking compensation-related as million the funded. supply along allowed expenses third relation of increase were to has ended our of approval were activity as XX, $XX.X LIVMARLI LIVMARLI, XXXX upon XXXX the we than of G&A clinical and We ICP the And the of Mirum driven FDA XX, quarter general, increases the primarily PBC quarter XXXX. $XXX the approval for LIVMARLI. of to collaboration XXXX, well as company expenses million. quarter operational operating due and XXXX, program increased we trial years connection In intend as had quarter cash investment expected ended personnel of activities. remains funding more well cash, for G&A of Oberland an well expenses September were priority million PRV. runway. third additional PSC, for by the Of to the a three Capital and R&D this as versus expenses with launch third monetize associated legal received costs of the related increases our the compared million $XX was the increases related

of the of the Given performance strength financial projected structure. of have the business, this decided to our forgo position we financial tranche and

Chris final will for over Chris? So call comments. to turn I with that, any the

Chris Peetz

as everyone joining Great. to devastating has strides the close, Thanks this you of for Mirum quarter a And Ian. great to today. liver advance made thank diseases. as continue To treatment company we

the LIVMARLI. for And Our shows convincing The we launch medicine data stems excitement we a to living efforts is start. our for hard see this the potential to launch. the with presented into the of to off data patients great ahead terrible treating of in Thanks disease. really work a and launch from the great physicians commercialization put new have excitement today

briefly milestones. I to Before I close, wanted recap upcoming our

our next PFIC adaptive interim We cholestasis program plan OHANA and under and expects and volixibat, Europe III for studies are cholangitis year. First, in with from quarter is in application XXXX. a of primary half And the Alagille potentially adult topline Phase the in potential we of MARCH-PFIC data sclerosing second study of of and review the in interohepatic the cholestasis pregnancy registrational analyses in next year. launch preparing VISTAS to a syndrome for maralixibat second share

XXXX. please look for forward Thank Operator, on the our to with continued updating open questions. for us. as into thrilled again the you we achievements are We progress and head launch the LIVMARLI. line joining of recent We

Operator

And Instructions]. Jessica comes go from ahead. question please from the Fye first Jessica, [Operator JPMorgan.

is open. line Your

Jessica Fye

Good my taking questions. afternoon. guys. Thanks for Hi

when First, reference report are trials? XQ? XQ will you remind preannounce look us the you an guys those? for the early typical when to The provide XQ XXXX to line that VISTAS more in expect next we of said results might suggesting for could And communication time was you you And second, each you will that on Street just volixibat, results? OHANA for a interims your update Or when hear to the the what can like maybe year, and

Chris Peetz

for question. Thanks the Yes.

year. we totally for So about line. To the transparent into versus on decided yet with update we as it, I quarter am haven't be typical one fourth next the on preannouncement get starting filing first the time the

And analyses studies that and something that could get balance season quarter sense conference something the that's a it closed a fourth year. we in year. here. for early interim And So studies, through the we Certainly, the volixibat next of the VISTAS-PSC VISTAS that's will preannounce difference the towards as through worth work on about OHANA study, as potential the makes have is the get be of as those interim. talk towards to analyses highlighting year for then key we the for the this we

interim and that analyses rules on a work all interim a the predefined with on conduct be a FDA towards conduct eye share potentially set that be selecting It of it will little for data OHANA-ICP this once from data, adaptive So an with the that closed and of how open we we all bit study study moving will registrational into an the will towards have alone at with interim with that study, continuing headline dose, The registrational lead interim study PSC. and will a an this least will of and and will discussed aligned study conduct portion how plan differently. announce to its data, analyses.

Jessica Fye

Great. Thank you.

Operator

SVB Leerink. our And Jessica. Foroohar you ahead. from Mani Thank comes Great. Please next question from go

Mani Foroohar

in I in street guys. York the at middle the Hi moment. City I noise. the for question. standing New am for Thanks the apologize background taking the of

its on patients majority discounting got think that Should to be story of and how Is flow specifically? it of of Medicaid is Or comments to to the asset rate like it where we more reimbursement sort centers discounting directionally discounting. for path similar some the to think likely an part other given Is the to looking this falls? small around minimum? about the about made look of assets? really You on scale commercial a transplant these is which

Chris Peetz

our some for color expectations All Mani. there. will to give Peter look Thanks question, I to right. the on

Peter Radovich

that discounting with the statutory question, the there. Yes. really There's the your the is The in requirements vast participate to of no The expect majority That's of will Mani. you Medicaid program. line latter is here beyond that net. what the comments in to should answer more Thanks for really gross drive internally. strategy

Mani Foroohar

Thanks. you Great. a follow-up, as talked to a reimbursement. And path about

first gap little diagnosis think should and that hub we or of sort the a how should little complete to from commercialize about receive And drug?. So a anticipate three further of how to should to drug? as patient delay think And we be months about some bit about will there kind of looking think a as that that a through like process reimbursable you around do we presumably world, the as, bit listing one longer? enrollment

Peter Radovich

for the that. Yes. question on Thanks

pleased how really are far. the has the with We MAP performed program so correct the did give steps And in you process.

the far already Alagille Of syndrome, And medicine goals. their patients lines a lines, we a diagnosed out different to a is, anecdote have throw early after have are start of occur basis course, But And kind MAP a there's key of the of especially days complete on and we form variable, have get access a an to with it's so then the seen of time different between I dispenses support program, case prescription and of point to one payers. with in providers goals this timely a paperwork. are we say days, really of fill. a seen, pleased enrollment and patients and prescription the time the terms also an diagnosis is receiving And fill. there's what to quickly the seen there and kind just handful then of the in as can of as we that at lot of both

Mani Foroohar

all Congrats Okay. progress. on Thanks the guys.

Chris Peetz

Mani. Thanks

Operator

ahead. our Great. from Thank Evercore next question go Josh you Schimmer from And comes Mani. Please ISI.

Josh Schimmer

of outcomes? terms trial. on that OHANA FDA that be need may Good. registration-enabling guess, also, since taking ultimately to much I that the could the registration deliver study, I guess, package the consider very a it to pregnancy a Maybe in you indicated for questions. this first is Thanks What have you

Chris Peetz

our pruritus. other Overall, the with focused of question. FDA has, has for indications interaction on Josh, some the Thanks,

expect we this endpoint for approval FDA much the at to do setting, with study improvement in be more. pruritus. The So for looks

talk I implications other actually the potential. endpoints we and it pass So will see some to Pam to of over outcomes the through across

Pamela Vig

for the Josh. question, Thanks Yes.

So we There's late significant aware And and second spontaneous women are trimester when for women bile you a already the these ICP, a that delivery these very or length acid. above serum trimetric of of XXX of having just of little stays, elevated know that above that we considered, bile pregnancy are probably with the background that associated acids pruritus, serum XX present are iatrogenic in outcomes, also often or third so bit that and fetal at poor also stillbirth. including at extreme look birth are all in cetera. endpoints of risk cases preterm et will hospitalization with

secondary So we of and our that will those filing. all of of part are the in part key the are analyses conducting be

Josh Schimmer

a maybe elevated there the because have follow-up longer that. fetus the outcome as guess, delivery? concern indications normalized, haven't some Just acids prolonged And is exposure to the adverse think in time that gestational not you bile I could bile acids? extending been as on a the but kind normal potentially if still early of gestation What for serum of time, about, are to

Pamela Vig

Yes.

with it different. volixibat cholestatic that that with bile expect in as you is And acids anticipate to our serum this be reduction we no would So a liver experience disease. in significant with maralixibat there see

and are early for premature having lower bile that of that maybe in helps born helpful associated itself. medical So and a you there are also a when with that acids in that lot see decisions in serum on hopefully of spontaneous bile birth. leaving inform children is know not preterm longer kids the risk acids that We the delivery for and in baby reductions iatrogenic and serum

and as goal serum that. bile in So keeping lower the help baby hopefully long is acids a as will possible

Josh Schimmer

on if there approved merged couple into Got And of trial a risk the in a for or that approved filings? Does numerically advance or it. data comparable the Phase III that you so, then may drug to from therapy? to say there are be therapy, advancing? patients an competitor And in you what now Helpful. questions looking is Is there better of an why? MARCH-PFIC a worth set kind continue it a approved concern results And some that given number markets. lose to any program, quick that is maybe need to of

Chris Peetz

at the so may number we nearly knowing some no study the for there. actually just are study, in overall of to this meaningful not the the And it's question what worth international From so that countries, standpoint, seeing, real point. on impact conduct approved, a far there study which have it's agent of is access Thanks conduct. an approved

having well. actually are over of label as So many with open the them onto these enrolled time extension already the some have periods of prior patients

is of also less and terms an And of as in this PFIC syndrome the competitive LIVMARLI So for well. with in issue. profile Alagille and

our today some that's One in present data we PFIC big PFIC and indications. data have we of impact advantages of like ability event-free we the findings of in the in data on X. program LIVMARLI to longevity did six-year been these have in similar is And the the survival showing presented earlier previously

we with are of paired increased next MARCH-PFIC optimistic gets because also improved ongoing see will that from that year study. in that's dose involved that the rate So the results response the study an we

Josh Schimmer

Thanks much. Okay. very

Operator

Arce Thanks Great. will question. your now H.C. please Josh,, move Ed And from ahead. go on you, Sir, Wainwright. we for

Ed Arce

the had and launch. have it's wondering that? you it. any very discuss are a But today's sense now just long shown the payers? on my thought, just I maybe recognize them you presentation. payers in I place the effects engagement lot LIVMARLI, start of discussions of with, Are going recent than been less does progress, wanted declinations overall And have Thank the taking questions. seen how given through Great. including perhaps hurdles put so far that you with still congrats with you the yet? that still that inform to payer And in really early all for with seeing

Chris Peetz

that let I question. for thanks Ed, Peter will one. into jump the

Peter Radovich

many team invested an then as engaging is Ed. first Thanks early And the Alagille out I it. think question, even this sales heard there's U.S. to the I this ring. With about our they back point the of syndrome and for Yes. important regards kind have time with they of very payers We pivot or will well. knew Europe, is and for little there to payers, in

reimbursable today regards of European So late-breaking of their natural to as it's really pathways. European kind really Best high really these Alagille, LIVMARLI interactions with the of to childhood profile with children of is And been actually natural and HTA the comparison just had number abstract to history what package they and medical and compared of have a has I think ASLD health our robbed are safety unmet efficacy with outcomes the anticipated data we the able we part recent that that to a part them. Lancet long-term of in publication the persuasive to stages package of we long-term you really, the on earlier in a be all payers. And quite follow-up, initial that's exciting terms to assessment presented today really appreciate than is have to history think come as very need have and seen what cohort. of technology And of and part updated have we and

played well year. second Mirum those of have European So half launches about and those conversations quite frankly, in have, in the data us excited the next at very

Ed Arce

then And Great. just Okay. follow-up. one

early, to in I patients but overall other that I about thoughts. couple you a is acceptance wanted VANTAGE, impression screening there landscape competitive known, more the commercial here, not but your programs terms get wondering competitive your Regarding it's is what here, of just which started development know Recognize and bar of in approval, but just are late-stage of well there? necessarily given

Chris Peetz

kind an of as setting Thanks activity surrogate highlighting the Yes. alluding line alkaline in competitive there volixibat Ed. PBC of some worth studies our third PBC that in a primary for is strategy of approval. as for or Actually, that are marker PBC. And use a endpoint a approach to, competitive of for important you focused quite for second different. phosphatase set with in is on agents element is for in there's question, VANTAGE And the that for are

endpoint are second the the being expect we but we using portion as for possibility study phosphatase of the not the using which and the as for the So endpoint, pruritus, of in has and alkaline primary approval primary again, the registrational study. outcome

In treatment is or terms and means of competitive of from in can alkaline paradigm, agnostic. line what it volixibat dynamic phosphatase lower can really occur in the Pruritus a fit where elevated PBC that therapy levels. with

as first of kind or a third can the line of in competitive second think some second the that track agent that on kind to line of be or parallel a of it other trials setting. So are

Ed Arce

Okay. helpful. Thank That's you.

Operator

you, Ed, your Great. Thank question. for

We Yasmeen Yasmeen, Rahimi Piper go please Sandler. ahead. to will now move from

open. is line Your

Dave Amsellem

for for It's Yasmeen. question. Hi Dave, everyone. on the line Thanks taking my

started So after a So on shed awareness the And a lives? of of percentage XXX color and follow-up XX% to percent I percent what if basis, have that I the scripts? what was some questions of Alagille few have that on write hoping that. you accounts could key cover have an with LIVMARLI

Chris Peetz

question Yes. the Thanks for there.

very provide out the we But to mentioned, it's specific As to Peter we there. some are days seeing color of of metrics. early what speak can

Peter Radovich

all different gain of with to Yes. have in and And Thanks Chris. access October. been kind XXX awareness. raise interacted And of we since ways, able have the

are say, other those am smaller we in country, accounts. we specific have a a little the also encouraged broad large by into really six not the given universe to that to weeks regional the uptake get there. out launch, of early it's centers So than but top And there. from both I pleased days we it's of are seen the quantification out where sort done I in have coming think the from, terms here prescriptions

as updating So through I think that's a good sign for where move the we you launch things forward to process. more stand and look

Dave Amsellem

data what are the will compared this use road? efforts to gears then the made we do how to characteristics to guys switch could baseline Great. think patients being Thanks to at you late-breaking regard if treating were educate abstract With GALA between you on And the color. physicians? for the LIVMARLI similar how cohort? additional AASLD, And And the to abstract down the that here. presented adoption the impact

Pamela Vig

regard with to question. Yes. I and I for over Thanks Peter that. for adoption, to will hand will the off then start

characteristics So yes, to and It's physician can to analyses. with including to natural you that of this and if ensure we we And Group. lot similarity process this of we baseline Study this a GLAL the LIVMARLI these was about involvement robustness, the compared a robust try that the way efficacy. sensitivity of very of, statistical to clinical rigorous labor to And robustness ensure get in a just six community were database Natural which through methodologies, a there history imagine, History of been really and outcomes cohort. patients means all and the years excited selection blinded are effort included actually by were GALA pre-specified, between subgroups was from

once based that on control the This assessment So phosphatase. to alkaline representative from at entry of and the the were a studies, two try is and the cohort, selected comparability looking appropriate there this disease cohort using maralixibat across key get showed the criteria was groups. an to And between groups. selection, cohort there two this is is similar severity

were potential variables. GGT, ALP, we bilirubin, And cetera. were these both et found well aligned. There that ALT, AST, So of on sensitivities

analyses. cetera, sex, this bilirubin, we adjustments using year everything included where So analyzed of of ALT, from et this adjustment all AHT, these looked and baseline were different birth,, is GGT, at at

I maralixibat think treatment of in real higher that bile note we were at it's where to world. two collected groups think important with baseline acid, important is between commonly SBA it statistical to And no found group. serum that the the the not I exception there note the also in was that differences

as times. So bias. the clock subset for well subgroup components for we analyses intermodal the survival GALA you do at pruning using includes start data. ran that also other cohort But looking analyses on and several index of adjust these to transplant-free that different When a as time

So the taking I analyses maybe survival the demonstrate this improvements and event-free all have just the question the together, And Peter. two to sensitivities statistically another in analyses, subgroup I will me? between with you with of really it Did these pre-specified and of that significant for robustness of mentioned all highly transplant-free hand is data cohorts.

Dave Amsellem

will go forever. contain I to Well, am have it I I a but follow-up did going or question,

Pamela Vig

I appreciate it. Okay.

Dave Amsellem

Thanks. Yes.

greater here that. the analyses the representative, fact good So database is subset in setting? your final probably liver you patients with segue those of that the diversion biliary decompensation have surgery there to a How perhaps were where my GALA the transplantation discuss could over that benefit is? real-world question and And or either view, are LIVMARLI to can a others events, any data it's or

Pamela Vig

Yes.

I late-breaker nicely Sokol. was and which GALA the hand-in-hand presentation and natural there's goes we another with a first So This predictors author Ron think history shows eventual. poster of had Dr. comparison really really, a

identify predictors the to So we data survival entree are what and-free try of and looked survival. at our

specifically, discussion in identify we pruritus in at this had remained and and what a improvement of we our survival event-free improvement that point for looked improvement XX% data I think, And event-free found, the are to So that and tried event-free predictors where of was importantly, survival predictive patients one survival. transplant-free transplant of pruritus, who transplant-free free.

Dave Amsellem

thoughts. my all of and I appreciate the I appreciate questions you Great. and answering time

Chris Peetz

question. the for Thanks

Operator

very Brian you Skorney. comes from Thank much. Great. next our And question

next from go Our James. Steve ahead. comes Seedhosue from Raymond question Please

Ryan Deschner

everybody. be on ask many question. This Seedhosue. is just have march Hi on how a genetic I will reported And want follow-up Steve topline subtypes for Deschner, in to I readout? Ryan the then

Chris Peetz

the there. subtypes study Thanks do in The and representation of for really subtypes have MARCH-PFIC the seen PFIC. all across study. genetic question the we And see broad includes Yes. multiple

PFIC for we study in placebo-controlled be included So subtypes randomized, on. all that the data report to able will

Ryan Deschner

your length Okay. interesting. ASLD what III expectations little the for were if Terrific. that z-score And you was data some in competitor give also, height more Phase at us wondering could then There on study? and I a was detail MARCH are

Chris Peetz

I But share of in have just of seen months expect time we six I worth periods in, longer that other noting to happens see the and to at six pass some Pam think wouldn't on will what first studies. that, that of some over sort effect it over to full follow-up than this months. we

of have study that So continue gather some on. extension that as an we we do of open-label data part to the will

Pamela Vig

Thanks. Yes.

yes, speak to second. about happy So for this a

for patients have know XXX% significant by was of our we shown to And patients acid when patients also serum statistically acid have growth have acid those transplant-free was and PFIC. bile of predictive Phase threshold in of which registry applied And data and acid ALT, And response patients, bile the history II we as who saw survival course. course quality serum had serum is in bile our study, bile transplant-free was on a in response. pruritus, study, a serum that those peak and survival. response. we AST is And life. the So of presented NAPPED, this we INDIGO who This data those natural a have who

broad-based bile clinical have growth patients across, including are in So this sitting see serum as improvement you control. who you acid in parameters,

And five on years this. data presented we so of

early, open-label will know, said, continue we at continuing as patients Chris extension. into we but as study, an III MARCH-PFIC As look to six months Phase are the

So come across certainly all subtypes PFIC more on to this.

Ryan Deschner

you Thank very much. Okay.

Chris Peetz

question. for the Thanks

Operator

[Operator Instructions].

Chris Peetz

queue I there Operator, close if can questions. think we no the further are

Operator

it remarks. I No Peetz problem. to will final over Sure. Chris now pass for

Chris Peetz

Mirum. exciting everyone showing potential thanks data further We presentation of outcomes. And forward as and goodbye. and LIVMARLI sharing term you and time progresses. call to updates groundbreaking the Great. joining today's your in the an It's look long for again launch to for of Thank to interest the launch improve with today's

Operator

for you joining Thank everybody call. today's

disconnect may lines. You your now

Mirum Pharmaceuticals (MIRM) 16 Nov 212021 Q3 Earnings call transcript