MIRM - Earnings call transcript Q1 2024 | Mirum Pharmaceuticals, Inc.

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Operator

Hello, and welcome to the Mirum Pharmaceuticals First Quarter 2024 Financial Results and Business Update. My name is Terry, and I will be the conference operator today. [Operator Instructions]. There will be an opportunity to ask questions today. [Operator Instructions]. the Relations, to to hand President McKibben, now Investor of begin. will call Andrew over Vice I Please go ahead.

Andrew McKibben

XXXX today release Thanks, Terry, our Chief to Quarter Medical you like President to Call. I'm and good Officer, Quan; afternoon, Mirum everyone. and Chief Officer, Pharmaceuticals Peter Chris Joanne our CEO, Officer. Earlier by for and news I'd release of company's results Operating the welcome Conference Eric Peetz; joined found quarter Investors Radovich; a this First and filings in Bjerkholt, announcing section website. SEC issued news our can be of today, Mirum Chief Copies first Financial XXXX. the the our

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

Christopher Peetz

advanced And progress key the to grow commercial market. of another is XXXX to strategic objectives and tracking our highlight significant volixibat expand our Thanks, be across Andrew, and everyone. pleased to business, of growth medicines us. for very good commercial year to indications the I'm afternoon to

life-changing and continue rare to value medicines on build commercialize while our We create to for delivering commitment disease.

million, were representing growth the from a this LIVMARLI product first our past medicines. XXXX. XXX% quarter performance. $XX.X net commercial sales Now strong first, Total of increase across driving quarter its continues

prevalent the positioned come and meet to revenue internal all demand the PFIC internationally continue of both diagnosed to approval. by well year our international are guidance driven launches and across million, full continued treatment million to $XXX in to medicines, and contribution increases $XXX patients and Newly U.S. we from

towards community. Second, we the significant label our approvals are important culmination and dedication in through announced impact LIVMARLI represents our of an PFIC, In researchers Mirum we also of medicines This March, making potentially approval the patients, progress team. life-changing milestone of expanding U.S. for PFIC years patient expansions. of approval and the and the new from

and the to program we from suffering of We've PBC, apply address we these bile advancing volixibat insights the segment where which submission preparing great also keynote scientific our acid PSC in NDA upcoming highlighting more LIVMARLI post itch, Lancet, We LIVMARLI great community, treatment. this And to improvements a be the those genetic published reception bring with patients of this positive PFIC. The and just off seen bile able week we're and regulatory treatment cholestatic made CTX. And diseases. with start for excited patients in third, in accumulation pivotal are bilirubin LIVMARLI acids, data to rare also was are to to adult particularly are in approval. the common growth for types progress can from the with

on PSC in see will we to Peter? commercial and June. summary, therapies indications advancing upcoming allow important by opportunities. financial program of core the We quarter, approved excellent a analysis our therapies will are and significant no opportunities scheduled further with studies, no strong VANTAGE taking interim VISTAS across our in the objectives, supported strategic be step as pruritus This both PBC in to that underlying which in I'll Peter execute business. to with turn progress cholestatic we the approved over as make call an PBC. In there And volixibat PSC treat position are discuss to continue us that, our to for

Peter Radovich

Thanks, Chris.

and from net to ago. LIVMARLI delivered a year LIVMARLI growth across global $XXX for and revenue grew quarter first million. million up quarter $XX.X product the well to teams of we were sales strong geographies. LIVMARLI with quarter, the of and million, full strong our continued $XX.X geographies, total another were remain our U.S. $XX.X demand $XX.X sales commercial XXXX in dynamics are and achieving Our towards million growth Underlying guidance medicines million $XX.X fourth in tracking across international sales all million. LIVMARLI, $XXX year million

Our business in of mix and total demand seeing in we patients benefit diagnosed a LIVMARLI from prescriptions are durable from to older with launch new our continues also U.S. sustained markets, treatment. Internationally, Alagille to demand we newly in growth most starting Italy. core continue recently and expansion countries,

care a claims impact pharmacy. incident in our we cybersecurity resulted Our which specialty approximately disruption million had during by quarter, $X U.S. health changed QX a our sales to across temporary impacted affecting This the was estimate business on insurance processing the products.

our progressing recent Turning received physician This the and discussions and LIVMARLI PFIC has for of pruritus cholestatic is in an well. patient business, been for I'm important are well to payers say forward patients. happy community, approval to and approval the by with that step the

We materialize paid continue and the dispenses quarters. to over next few anticipate reimbursement to

strong forward recognized the million. coming I'm and with CHENODAL. of Overall, that, $XXX Turning to in CHOLBAM positions net seen first come. the to And to of full this and sales this years the towards quarters million I'll over year-to-date million achieving it turn with Joanne? Joanne. and in $XXX our look $XX.X year. how year we growth product have quarter pleased of demand to to We continued us I guidance

Joanne M. Quan

Thanks, Peter.

continue to to we our forward advance have look a quarter this lot pipeline. We as to

study VISTAS VANTAGE a volixibat would I interim take June talk few study. minutes our PSC First, and upcoming to like for to in analysis PBC our about

conducted PSC those selection. will Starting interim study, support to the VISTAS with a be analysis blinded

efficacy independent will committee based with and of or IBAT a continue continuation, these review and which selected a and is the efficacy experience with safety data X. prespecified if starting points for inhibitors to monitoring have criteria, design threshold cholestatic data the met. The prior for assumed X.XX recommend Using difference whether of pruritus. on standard point the We treatment unblind are not to or thresholds in dose study study deviation the and for pruritus the

allows is a us As numerical assessed a on reminder, accomplish key rating to objectives. This X scale. pruritus approach XX-point

First, meaningful effect. confirm we want a to treatment

to effect. included blinded select pivotal the gets want a same we from time, potentially interim data ourselves results, third, keeping the by set. at This the have to we faster, to Second, and pivotal the meaningful patients dose. best data us will be of treatment in And the reassurance interim

be top analysis the We study sharing interim will the line from VANTAGE of in PBC. data also

included As objective who a data active of dose outposts are take The reminder, set and the select this elevated to ursodiol. is the pivotal study allows with of to patients the X forward both on placebo. X arms study. interim patients the with across interim appropriate XX normal The portion and doses to

goal data a when acids. studies significant and interim With designs, bile is towards for studies seamless supporting addressing off the to to projections provide and XXXX of the studies are a the on adequate our patient the show I sharing over you of PBC, inhibitors a interims accumulation adult data that, is I'll Both trend to enrollment efficacy. the is to top Given expect to treatment adaptive look At we of of cholestasis this The such we portion study turn line call progress to not sized announced interim, these significance. severe pruritus adequately patients IBAT we this to with important now registration. Enrollment lack great acids in both statistical options progressing the and cholestasis to in designed represent show select we well. Eric financial year. improvement, a safety serum for symptomatic broader results. enroll in start, discuss and our burden. We'll for bile continue believe These groups show an with Eric? and in update forward on June. dose is other completion of the where as an on biomarkers with both historical step

Eric Bjerkholt

million and were expenses $XX.X total expenses of of quarter for we a the press revenue XXXX quarter first release $XX.X million $XX.X was million. Total which operating million, to that last operating noncash Total million included sales of of quarter Earlier cost first expenses million quarter briefly results $XX.X for of financial ended $XX.X March I'll million for issued compared today, revenue year. summarize. quarter, Joanne. included approximately total charges. $XX.X includes the in of which Thanks, XX the the in first $XX.X the The of expense R&D

For net million the share. $X.XX per quarter ended March was $XX.X loss XX, or

at million and year, Our investments of capital. end XXXX, up as March $XXX.X a $XXX.X cash, XX, cash million to of and primarily the working due reduction in equivalents last to increased from

will quarter-to-quarter our payments We capital expect that from depending inventory on timing and vary investments. working of balances

position pipeline be of our we continues our the So to support our global well business and business. an in summary, funded, to in are commercial excellent of and advancement expansion

back call over final for Chris turn I'll Now comments. to the

Christopher Peetz

a for grow. to been remain our great full guidance. We start year, It's year Eric. track the continues revenue business to Thanks, on our and

for interims with forward our to that, please much expansion And across label the looking on call opportunity operator, and questions. executing are launches open We are volixibat very the ahead.

Operator

[Operator Instructions].

question first on of the JPMorgan. line Your Fye Jessica from comes

Jessica Fye

the results, are medications into how to volixibat allowed be at remind the For on of pruritus? if that us also all? patients And that kind you expectations what for factors X studies, background your address can may

Christopher Peetz

to bit Thanks, a and background about Joanne Jess, setting. talk for the the question. in little jump

Joanne M. Quan

actually for of way Yes. for designed that the populations. the Thanks, Chris, make these and applicable we that think it Jess both We really the question. actually studies thanks broadly

we're with -- we're or of for and ursodiol level in So [indiscernible]. allowing are PBC, who patients any not either with allowing patients instance, on on varying

some the bit that little this other trials area. than So for other of been the kind a agents in of have different

first to So this line we in for translates ultimately PBC. really think use

patients as therapies diseases, pruritus. PBC majority do PSC we no have know, there's you and other both For these that and a PSC, know of with

So we've issue inclusion world kind studies very into this with a we the a real -- our very allow and broad the think way give of therefore, do information. studies to [indiscernible] us designed a translate criteria

Jessica Fye

us remind standpoint I tolerability And you what if volixibat? Can to Great. could. just one for a from expect maybe more,

Joanne M. Quan

Yes. Well, Jessica. follow-up, thanks for the

know inhibitors of we volixibat we these and and will we I what so look And how to dose to what exactly tracks for. know know exactly quite in IBAT say, far, kind know So well. we

any really surprises expect in this don't way. we So

Operator

next Your from from Partners. Leerink question Foroohar comes Mani

Mani Foroohar

one. quick A

sort when in between a that is sort of is a that you products million, opposed acquired changed LIVMARLI some about next onetime it we in to about think this $X and as million that kind of try You underlying terms your revenue metrics, might mentioned quarter, To the this that quarter health extent function of about sounds separated separately, what back And as recognition issue. think like, and out the pop $X versus a quarter? of impacy revenue assets? split care between up TBTX how loss demand

Christopher Peetz

question, details. let Thanks I Mani. a Peter answer mean of short a for give onetime the I'll into kind dive color background it's here. can the on Peter the effect, this, little but to

Peter Radovich

concluded Yes, quarter. the onetime that by is the of effect end

lingering So Total And quarters. all products prescriptions overall to our of the from grew. effects future grew the any I in over for don't see demand expect quarter. this

$XXX confident we're million. in So -- $XXX the not to very million really why that's

I and you the think $X asked -- also about million.

of precision impact $X across the $X we here. a kind think by million I of I U.S. kind product probably million business. mean about as is individual think assigning an false

Mani Foroohar

great products? of Okay. But is it by of LIVMARLI geographics those the driven given was assume majority it the reasonable to

Peter Radovich

I generally think of -- it's about to probably think you the the could products. balanced. in proportion It's size it

Mani Foroohar

end a in about Okay. the the between obviously, the commercial $XXX on we sort think staying tempo questions, year, of between And Should into think you is and think weighted we're like consistent of into growth maintained the that XQ, back we from perspective we reaching how $XXX deeper million fairly That's year? now helpful. as that modeling guidance. and about somewhere about your more as should XQ end now to the now million of until

Peter Radovich

Yes.

strong, I demand cadence the we think about mean, it the is quarter-to-quarter. it see I we consistent. mean generally think way -- of growing I and is

I build be XXXX. that on, although in the PFIC XXXX, contributing more most commented, million. dispensers expect That we be have dynamic play to many later to coming will consistent they're generally, $XXX of might towards million as pretty probably a comes of the You But and think it's into we've the in the PFIC predrug year. one more $XXX

Operator

Gavin The Evercore of from ISI. the next on question comes Clark-Gartner line

Gavin Clark-Gartner

in I expected expectations And believe or deviation placebo progress. X.XX your pruritus that the us there informing arm X.XX for for that placebo assumptions. was your a absolute standard just on benefit was trial? Was you noted powering PSC, Congrats adjusted. remind maybe the X this

Joanne M. Quan

Yes.

question, thanks Gavin. for the yes, So

X.XX, to we of to compared you where study some putting point by active placebo. mean kind of at design. a So difference, And starting And But fairly the conservative least by looking as are always the our point for for took together. we those that the we put did the study. treatment so starting you kind assumptions at want at share know,

Gavin Clark-Gartner

for either volixibat? to And trial helpful. for are that's Yes, you able the pruritus baseline share scores

Joanne M. Quan

we when Not show interims at happy point June be the you. in information share when we this with will to

that with would be think folks. look we that -- forward you to along So I

Gavin Clark-Gartner

good. sounds That

on for PFIC drug Just the for one. the Any last EU? status updates orphan in potential the

Christopher Peetz

for are to the the LIVMARLI Thanks one have hope is that strong in patients. conviction in data real Yes. an really our still dialogue follow-up continuing providing to advantages regulators for program in But European there. This and the come PFIC, update back some for we soon. with

hoping have update an soon. that to on one So

Operator

comes The next from from Gon the line question on Stifel. Ha Dae

Dae Gon Ha

will question the And the with can if comment wondering between done? And to ] PFIC about the you of sort you in mind June when are glaxo need towards come [ expansion label dialogue, in of be over end the your both label side the dialogues story. reimbursement and filled have the was interim And the ] both Maybe a guys this out think about strategy it impact I once back that to be VISTAS of there get volixibat, you on X-part dialogue the year? being. the had might your switching subsequent given you VANTAGE, time to physicians bearing drug update disparity on [ this for how positive? thinking PBC how the and

Christopher Peetz

all just for a about Peter Yes. pass and what make I'll briefly we're to I on then quick and to how the think kind work Thanks potential and for we've the comment talk Maybe It's advantage at the we're volixibat dose all response in terms on in programs, curve. provides we've question. of PFIC. of where seeing approached competition dosing done understanding a level. we've across IBAT volixibat, for volixibat activity of And LIVMARLI something the over the particular, the real learned the with on

activity think have I strong to provides the really potential here.

course, questions. study mean is we'll to the play PFIC but for forward excited about you can with Of we're regimens sets out Maybe what evaluating see the actual and speak that the data the that come they in VANTAGE can dosing this patients. something as

Peter Radovich

Sure. PFIC Yes, very thanks and question. Feedback for the LIVMARLI positive. the on profile has been

as favorable make depending labeling, of -- reflected can of profile the March notable genetic which in PFIC market I as a that in the well feedback efficacy in the lot is on observed study a broader was policies. included sometimes types difference are on access, that payers' think we the the

age you then patients favorable emerged. clinicians have happy in So from we LIVMARLI And far policies days conversations older age. label conversations when those Those really in kind updated age. early really, We heterogeneous, and policies. occur with younger quickly. have year label the of are those as -- to X happy than mean, of going lowered And saw then payer occur feedback initial payers quickly would mentioned, that X available up with really after, a but the generally U.S., potential I well. the years that and that still, for patients. of the is It's of policies subsequent Alagille but to expect of follow-up have had pretty I'd [indiscernible] generally, so there's yes, and those terms lot pretty a update

Operator

James. Raymond Steven from line next on Seedhouse the The comes question of

Steven Seedhouse

Two as straightforward. separate questions. they're I both just now pretty asked

call second mentioned be pipeline synthesis would a primarily? expanding parameters? And also sense or priorities if [indiscernible], there's function there be you'll any over XX acid the I XX bile next, focus that standpoint, for what's safety First, is just safety the just even bile on are focusing release. development. novo ] view you months, improvement, -- de from get on impact liver overall I'm on on of of then your if one, sharing bilirubin, of those PBC thinking volixibat the comments And and serum happening on [ Alpass the to to of or business or that Mirum just about be prices tests, any readout, acids ALT, curious curious thoughts a it, the you would data just the of

Christopher Peetz

came Steve, And remains for the business looking. anything I'll really efforts development rare much and Thanks, questions. course on its number come to that's value add of about across for what disease back disciplined the that and in and of with on a that development approach and look we strategy and making company that consistent Mirum. very kind how helps our terms grow on our settings be we've a let started Joanne of comment company interims. done business to approach the different at to being the over and of can It's something to It's is speak is DNA. to something sure where PBC our we we're bring the and

looking lucky PBC maybe that expansion opportunities, then speak And commercial do of grow ways at developments in volixibat we're so the urgency there's we're no a Joanne a label the there's in to lot can think can that to something the quite I remain the in business, where question. company. position in disciplined we to growth

Joanne M. Quan

the question. and Yes. thanks for Thanks, Chris,

is interim As analysis. an this I mentioned,

scope. ensure mainly at in of We're to the select limited pretty terms it looking safety it's So moving and dose to forward.

pruritis, with going The set is look safety, to look we'll we'll particular. that, be and at data bile acids in So limited. at serum pretty

quite So we will parameters. in it limited other terms certainly be of any any think about conclusions, making

look data the we'd for to final think that. I set

Operator

comes on from line the next Brian The Baird. question of Skorney

Luke Herrmann

This is Brian. Luke for on

the reported reasonable particular As itch same are then in aware than the you a scale you using? with the comp of the NRS scale we to study is seladelpar subgroup benefit? do for set that expectations used pruritus in response for that XX-point think a about the NRS greater they baseline thinking X study as you're is VANTAGE And that in study, outcome comp

Christopher Peetz

for it mean -- registrational scale there's Thanks the XX uses what study. -- guidance. But differences. in line Yes. using this for I FDA used questions. mean That's very the some of that studies similar. is I measurements adult in scale. pruritus is all our minor we're kind It with in

is off similarity there. -- at, some treatment placebo. effect subset from the at there that far from that with not placebo and we assumptions we looked some So And looked difference it's X.XX definitely how the too right, at powering, change our look they in where

potentially far effect too looking little in -- if off an really at not and general, it's about more So But from a assumptions. study design IBAT. bit you're thinking

this to baseline, is what the is particular what doing patient and Of like. of address that change seeing which from course, really what we're getting quite you're the and excited burden data disease. about looks experience

Operator

The on Citi. next comes David Lebowitz the line of from question

David Lebowitz

to we a pivotal pruritus. portion at you the point view you're how through use what of thresholds type through in portion to and consider point time billion perhaps needed, talking the you whether that actual subsequent could expect? us if will is the that as elaborate Could point about blinded difference on On it upsizing give just X.XX of what some or

Joanne M. Quan

the the design, deviation we're The some going not kind for I sense that design of Thanks at. details X.XX a what the could standard study. general the and We're of overall a of treatment is the that effect difference X study you just the into we're but provided Yes. get looking of treatment so looking to terms in get number number just of for the of question.

we where we'll results be any Obviously, of won't study. we of the with hope So of since continuing be share the the that in certainly terms the be interim blinded. kind are interim. sharing we'd specifics we'll We

So be see to that's June. what we in in hope to

Christopher Peetz

month David, sorry, That of to final want the X, over measurement add that, effect and and time, analysis. looking add treatment the the It's into analysis. And adult try what actually PFIC for design. to to deal is X at that at I we -- roll study Applying with study allow settings. power the used X, actually does factor X, to right? from for the looking the and potential X to That's placebo response because any is how and kind points you're of they time multiple another the X the

Operator

The JMP. from Citizens next question Wolleben on the John line comes of

Jonathan Wolleben

PSC. Just rolled on you to logistic When you have on the select in enrollment could either dose, one those dose you comment how the for higher long get placebo do at the me and patients reenroll take back new can studies? dose, both PBC or on to And complete in or patients?

Christopher Peetz

the John, Thanks, question. for

study. we'll give patients the to see let Joanne lines, the get through of more of formal expect full we bit June -- on update how what to the flow the a mechanics data to for when little speak have in set on we I'll to interim. time Just a

Joanne M. Quan

Yes.

in going with active to we'll then enrollment and continuing we one study we're -- are and placebo. be So this the dose be continuing point. continuing at

we to set. ultimately all ultimately in patients we analysis the the So expect data whole when include the unblind

Operator

Ulz Next the Stanley. Morgan of line on Michael from comes question

Unknown Analyst

the ] is for any prep patients on you access ongoing when This PFIC? to get paid [ drug? just program Robin on And Mike. you expanded the do Can color for on provide on launch expect

Peter Radovich

prescriptions seeing LIVMARLI, patients in ]. question, the launch now. PFIC U.S., for for obviously, underway, Robin Thanks Yes. come [ In is

have them in roll drug. coming commercial those throughout and Most about clinical drug are XX expect the year. over are to the patients We this we talked in this who at to -- eligible of U.S. we'd about quarters come have to on time, over

forward. see we how that's the cadence playing So

Operator

H.C. the comes from from line Ed on The Arce Wainright. question next

Wing Yip

couple Ed. is questions asking Thomas for a This of

following first, performance LIVMARLI U.S. on for PFIC. up the So

million was some PFIC also investors net then in the wonder Just to? that from $XX.X sales metrics And the first quarter, how launch up it approximately? early of much came what are

Christopher Peetz

Thanks, approval for patients. we're of kind there came March yet. And PFIC -- QX, really in no over In rolling question. there just and clinical Thomas, now those the there's contribution -- in the just

So to expect where we revenue PFIC minimal X contribution of year as quarter reimbursement. half pretty into the be that from next expect over the we more back get or the

Wing Yip

the any ] And year? it. European gears PFIC? this then in given with Got recently any interaction switching front. ongoing Have the first to positive, you market for your half EMA a preparations And CHMP of commercial recommendation [ on expectation for European if of

Christopher Peetz

there. the for Thanks follow-up

we the have -- EMA with discussing EMA. have active the discussion, On in been you over

As that an have our soon. I on feel and update to confident arguments mentioned, hope in

So maybe no little prep in And formal to can a the yet. determination Europe speak launch bit Peter PFIC. for

Peter Radovich

of essentially Europe. prescribers upon would Alagille for PFIC for to are launch ready certainly, in a Yes, potential to LIVMARLI the be Prescribers identical approval, LIVMARLI syndrome. it

submissions with help as well. awards go technology you'll reimbursement on buy pricing agencies, and level [indiscernible] to So a be ready to that dossier to and cetera, et

Wing Yip

question Okay. sales that's And one bile million major for acid main last by $X of quarter-to-quarter, some practice? slightly then the us. product from the Discuss [indiscernible] drivers

Peter Radovich

the entire we for kind Healthcare play Change of Yes. portfolio. I mean mentioned our was in cyberattack

to the in to patients FDA disease is products excited chance patients to look you time. opportunity the do acid there. build number year, forward, find see time mid-single-digit growth, with this back CHENODAL and year of going and the by -- small go nature on if really I continue of think expectation. there given And are there's year-on-year product our in next approval quarter-to-quarter historically about But an and out sales, potential more CTX the bile ultra-rare over volatility consistent those over for

Wing Yip

Understood. and look questions. in again, -- Thank to presentation efficiently We kind you'll soon also forward recommendation the for for hear the you, of volixibat.

Operator

Therefore, further CEO, back I no will Chris final have hand remarks. the questions. for Peetz, to call We

Christopher Peetz

Have our in today. all appreciate interest programs. the and Great. Thank Goodbye. us joining you a Really for evening. good Mirum

Operator

This all you concludes joining. today's conference Thank for call.

You disconnect your may lines. now

Mirum Pharmaceuticals (MIRM) 8 May 242024 Q1 Earnings call transcript