MIRM - Earnings call transcript Q4 2024 | Mirum Pharmaceuticals, Inc.

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Operator

Hello, and welcome to Mirum Pharmaceuticals Reports Fourth Quarter and Year End 2024 Financial Results and Provides Business Update. My name is Elliot, and I'll be your coordinator today. [Operator Instructions] I would now like to hand over to Andrew McKibben, Senior Vice President of Strategic Finance and Investor Relations. Please go ahead.

Andrew McKibben

Thanks, Elliot, and good afternoon, everyone. you Pharmaceuticals welcome Conference Quarter to Call. like to Fourth Mirum XXXX I'd by Peetz; Financial CEO, Officer, joined our I'm today Chris and Bjerkholt, Joanne Eric our our Chief Officer. Quan; Medical Chief and Chief as this today an week. Peter Radovich, at our Operating could with he's in not event launch Officer, international us be Europe President Earlier issued release news the a today, for year and quarter full announcing fourth Mirum XXXX. the company's results our the website. release and SEC can Investors Copies section in of of news this be filings found

and Before like conference during actual product that we current forward-looking approved I'd regarding for and we inherently These results course medicines will the on statements risks based today opportunities certain that start, Mirum's candidates. may you materially remind to and market programs discussed. to making uncertainties of results represent management's from the our call, differ cause statements its this including statements involve judgment of and as expectations, be

Please update said, I'd and We subsequent Chris. that for information. With are turn XX-K under more risk statements. SEC Form to no Chris? filings these like in refer latest the duty call our to over the factors to to

Christopher Peetz

cash ultra-rare broader important that's on in since in to patients, positive medicines attractive start and share our Mirum's time disease everyone. afternoon, Andrew, have excited focus we a founding, call for XXXX. bringing year through I'm populations. goals of strategic Thanks, a business with an our created strong with short recap achievements patient to our last good the pipeline on over flow today's and update the In an

yet believe in we to early and come. still firmly are that the We journey, best is our

the next cholestatic pivotal in end accomplish guidance just approximately the the step-up our on all million, delivering in discipline. XXXX and Alagille product $XXX.X including syndrome $XXX with with four, range. our the our initiating EXPAND continued program We of continue. revised opportunities years for scientific above the -- net million next upper are to PFIC the In of uptake cerebrotendinous creation, product significant were $XX since potentially the in growth cholestasis driving year, and we the one, Total in high-impact launch strategic finished and of success continued recent expansion, early were by product year, PFIC Mirum. are Alagille U.S. of of adult LIVMARLI's international PFIC our completing Three, and approval for strong million Our a net of drivers and the with and second rare Two, LIVMARLI business of new the U.S. for And highlighted CTX, pipeline, xanthomatosis, in sales our big of we global medicines, CTEXLI acquisition year for demand license goals. volixibat patient was to, strategic million, half $XXX and starts or X priorities year will from financial internationally. the potential commercial believe seeing further syndrome X pruritus. over pursue of MRM-XXXX data value impact in patient LIVMARLI the as patient and enrollment have exceeding selectively for disease for last growth. LIVMARLI Fragile study sales and total advance

and million XXXX we with cholestatic in the CTX growth Joanne? begin $XXX name that of beyond to well LIVMARLI, promotional Europe. beyond exclusivity With chenodiol approval the our comes capabilities medicine. for significant Phase the with positions bend and and total medicines breakthrough CTEXLI. Pulling need resulting growth saw orphan to of rare of with designation execution opportunity the robust We is PSC with in this year. XXXX finally, brand. launch years neurology aligns line strong patients that and our its in with for new pipeline achieved was Fragile net to broader of LIVMARLI, PFIC team in high-impact setting sales another III for between the week, our product of in $XXX PBC X million VISTAS to have And field XXXX that, all of approval million. syndrome. acid initiated I'm brand FDA reach XXXX I'll exciting built to us pipeline our addition happy which continued X the to we sales also we business for approved on for last to also positive well where of it say update of is results brief for commercial efforts and unmet EXPAND adults. for in dynamics million the an label the for $XXX And we our potentially in expanded curve in and this on U.S. this the study both underdiagnosed in CTEXLI, the we for current bile condition. Ultimately, across a pipeline. work our MRM-XXXX are We here turn also product set add volixibat for our genetic to expect PBC. pruritus, a are LIVMARLI We in Beyond both anticipated all with up our we VANTAGE net pipeline. think settings we together, have the growth medicines. And high Joanne LIVMARLI's both to top over healthy a starting expanded $XXX opportunity significant year close studies, with This the interim give with

Joanne M. Quan

confidence [indiscernible]. Starting versus highly multiple the the PSC clinical on call we inhibition say currently following of in continue on mechanism across pleased with XX interim PFIC, to potentially a including now PSC. X in into We analysis VISTAS study, the that builds pruritus We've conducted momentum year our program, IBAT was this enrolling year, the seen Based for of our Thanks, milligram with single clinical for evidence progress placebo. the volixibat June dose, registrational Chris. last active the is XXXX. in and Alagille, potential of the in agent, biliary impact excited are positive our safety volixibat. made inhibitor, this further we've the both and with and cholestatic XXXX studies engagement settings continued this we with investigators atresia, I'm we PBC. Starting I'm pleased passed where productive and progress XXXX of IBAT a committee, had great efficacy and interim study. thresholds analysis patient with BID the to

There on treatments we for a medicine health ], globally. in work patient study global community an look with to [ complete another We no PSC, second this half to opportunity is high-impact to given in expect are the forward that approved are to bring and a this XX-patient line results track underserved XXXX. And enrollment year. authorities of to top

Turning enrollment to to Enrollment in track XXXX. on complete in PBC. VANTAGE we and study the full remain also is progressing well,

our and strength importance do interim good where received response of from treatment results --. of patient a statistically of placebo, PBC second-line designation the significant, potential in the proportion UDCA option first-line patients large community. seen improvement the acknowledges clinical investigator Given Majority see. VANTAGE have as last a the to analysis to in from but showed of they and of positive address The interim FDA rapid, not adequate response sustained versus year the for patients we treatment a their maintain with able options we we've first-line pruritic volixibat impactful have patients, a potentially Breakthrough a symptoms. physicians will results therapy are

we there's track expand up opportunity III to to and LIVMARLI. by benefits. to The settings treatment, MRM-XXXX, are complete This We the the label are new inhibitor clear a an an is on Fragile study the also and on EXPAND are seeing pruritus X excellent enrollment enthusiasm for in excited where include approved, additional cholestatic to encouraged is we our PDEXD need about for study ramping for syndrome. LIVMARLI for well, Phase effective in we're XXXX. Shifting

We are study FDA. with initiate II a preparing to discussions year the this following Phase

of Fragile proof profile believe mechanism pathway As look all momentum PDEXD, year. penetrant we're the MRM-XXXX's on has potentially And I'll to Eric? the forward demonstrated with We now high the clinical the through all to and it very importance turn progress expression we engagement given communities. investigators of updates our of [indiscernible], active ]. In our this continued the sorts of is further summary, emerging Eric. providing with patient concept this brain and in happy X. reminder, products [ of a of over

Eric Bjerkholt

million the growth sales of in in performance in you, Thank fourth net product continued excellent year product I net strong to $XX.X position, XX% the will summarize. XXXX by was year-over-year XXXX, a commercial increase. Joanne. million that before, I'm X quarter strong another financial medicines. compared representing Total our our which now we say in XXXX supported year was $XX.X sales to briefly of pleased are an

the XXXX. $XXX.X year product in sales million to total million XXXX, full For net were $XXX.X compared

the net total XXXX. LIVMARLI $XX.X product quarter compared million. Total million, was LIVMARLI, XX% to net sales representing $XXX.X XXXX for For a was product in sales XXXX increase fourth

with other XXXX, million, fourth of the million, the full cash Total $XXX.X growth happy included This $X.X includes operating under million say fourth $XX.X payment We of R&D product reflected cash the million, $XX.X million. Total over million year. goods Expenses continue non-cash start for MRM-XXXX stock-based and also this the million. the XXXX, I'm was portfolio of $XX and expect non-cash Total compensation our of paid for expense and million approximately $XXX.X of items, sales an bile of and net the was which the adjusting year, of for expense the year full sold. quarter this operation. was Turning $XXX.X our was million, business expense and of acid positive for cost for XXXX R&D first largely intangible items investments was non-cash ended an cost the $XXX $X we in product medicines. increase year. XXXX the In license. When of from XXXX $XXX.X items XXXX other sales is amortization upfront portfolio in representing in for to quarter non-cash to of million $XX amortization expense million. equivalents we the cash, of the flow SG&A fourth expense Enthorin quarter and to and approximately to XX% net of reflected sales quarter the intangible all expense

million net guiding expect $XXX product of flow again are and $XXX this year. to towards we positive million sales cash XXXX, ahead, in Looking we

the our we We toward are well resources funded, our plans and on catalysts. to have progress multiple pipeline upcoming execute and

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

Christopher Peetz

the another execution company, that one Thanks, to is have for and excited come. and I'm continue And for year ahead questions. to medicines advancing opportunities what we XXXX call With built years have in growth model, set and and with for rare the open for of operating the their be life-changing positioned a please operator, pipeline Mirum. patients deliver disease big we is as disciplined proud industry-leading families. we accomplished for leading to Eric. commercial a sustained that,

Operator

First with Clark-Gartner Instructions] Evercore comes [Operator from question ISI. Gavin

Gavin Clark-Gartner

Congrats on the great progress.

to had in think of able So notes of I does bigger know one advancement, I But call question. change above as more general? next conversion about this performance, aren't has for back earnings that year. well and just redemption how just allocation traded picture, the your really capital you strong BD strategic you pipeline until the all stock price, convertible

Christopher Peetz

speak the comment Eric of thing dynamic anything the to pointing really a balance Thanks out Mirum make to what of some add backdrop value The up navigate advantage is able and else take the for opportunities for disease I'll we there the plans. you're financial question. disease financing I'd position, been particularly to where acquire we basically I'll that unique of our sheet. can this and specifically first to the company. products behind Eric as rare a to ask great to and that out, And point ask roll on performance these position kind we've see rare creation there, market in for more

Eric Bjerkholt

doesn't The our really in meaningfully. that years, factor you. X into Yes. plans matures so convert Thank

business us alternatives the that significant a find think is consider as BD attractive. and continue we lot flexibility financing of to I and we progress might our flexibility financial gives to

Operator

We now Morgan. with J.P. turn Fye to Jessica

Jessica Fye

X. or that that share, about pursue? I talk penetration there any potential $X for in indications billion et talking Fragile you we're sense that And the guess rationale are was MRM-XXXX I competition, underlying market are make mechanistic hoping about while could the revenue behind with asset, other potential cetera, like assumptions could the to

Christopher Peetz

the simply, Jessica. And question, male X, I'll think look market quite kind have XX,XXX-patient the the for potential, it's sizing the at of the and market. settings. other with to a speak Thanks about comment mechanism when in applicability just to Joanne market about way of patients Fragile then we

a can $X at the see easily. the quite of total market look or So to different ways assumptions that more you is that billion lot addressable

looking watermark to Joanne, about when you opportunity. beyond substantial talk really, U.S. -- that So comment a pretty we upside see It's that kind of even. Do number at number, put to we're want there. And about simple the

Joanne M. Quan

Yes.

asked kind So what actually I terms learning of is think, sort in PDEXD in kind the We and opportunities. Jessica, of of other are memory of you know areas rightly brain in the very important. where question highly expressed that

we kind wide for. of could be The proof it Fragile we're once way could have the for this use indications number possibility envision associated there, our is a a a conducting entering approaching that are of into then be up there of And opens used -- conditions with disability. then dose. of as concept so could you first X some this think other really intellectual to that, entry I And kind identifying this point,

lot So we're potential a access. certainly of there excited to that

Operator

now Partners. to Foroohar Leerink with We turn Mani

Unknown Analyst

Ryan have on Mani. for You

do really IBAT And kind of diagnoses more that are So look growth penetration when new Alagille I'm going be curious, forward, in population? think greater yet? into you at prevalent penetration driven of of terms the is those we on market, by into kind or the where are not you going to U.S.

Christopher Peetz

question. the for Ryan, Thanks,

of In so U.S., in with XX% for eligible are terms the penetrated. range treatment we're of the or that Alagille population probably syndrome overall patients in the pre-transplant, so pruritus penetration

So way, that behind lower new that, new infants, the are of entering. are this several so actually driving alone, into patients, they're but there is just growth continued the patients. the and And that just patients growth dosing, is weight-based then dose think on in year. One generally further dose along prevalent of diagnoses about see prevalent you time, usually that that. over start each that from adjustments They're diagnosed we population. patients lot a do some see penetration come potential what's new so statistic over There's the there so And those are tailwinds time.

that So show that growth syndrome there's of kind Alagille for U.S. combines continued long U.S. all the for of in to a of the runway

Operator

We now Citi. Lebowitz turn David to with

David Lebowitz

how use point you, sales trajectory in of population? In terms recent elaborate this guess, approval you already affect on there's expect Could the I for that given could congratulations CTX, forward, the on particular this going that. on

Christopher Peetz

Thanks for question, the David. Yes.

So we understanding diagnosed getting goal your question, kind your here patients have over coming -- of number in to is those to substantial The patients CTX treatment. there's have think to already months. the in where the the have chenodiol. that dynamic is to more and we plan then CTEXLI impact convert and diagnosed of on here diagnosis the And been

of the preventing and curve of buildup some it So earlier to the accumulation bending we new diagnosis irreversible of effects to of patients disease. of and expect gradual do of be the a kind bringing these

Operator

Our with Stanley. comes next from Morgan Ulz Mike question

Rohit Bhasin

about and on Rohit talk Alagille fourth out is for expanded syndrome on between Mike. program drug? you additionally, many Can you expect play patient are quarter in the how for PFIC this the then now how of LIVMARLI And in paid on This of PFIC patients XXXX? the mix and access to XXXX just

Christopher Peetz

there one first. question, the the on Rohit. I'll last for Thanks touch

expanded the During commercial to patients were year we to products. the last convert access of able U.S. all year, over

the throughout are was LIVMARLI we specific by patient quarter last it mix, patients. today, kind numbers. most complete, Alagille stand terms don't we post syndrome year. of far of that in of And patients So But most really into where third the

the contribution today. of that's vast So the majority

starts, have patient year, Alagille new of we of seen with really seen terms starts, patient -- since patient we have QX starts. PFIC steady in next a the step-up In nice

new they top and patient in So going the you see to are performance. that contributing substantially quite reflected forward, line starts

Operator

We with now Raymond Deschner turn to James. Ryan

Ryan Deschner

would much Phase have in the team expanded result of you cholestatic III And commercial pending is, Congrats on question My quick patients, how I expansion doing for follow-up. study? the EXPAND anticipate a ultra-rare then quarter. the pruritus

Christopher Peetz

Ryan. question, the for Thanks

EXPAND, LIVMARLI's Alagille universe potential at for largely same syndrome label the expansion looking prescribers. anticipate planning that's prescribing and For as the PFIC with we

already it the that's field So team expansion wouldn't of in be an place.

lot you you a question? we And commercially. a there said had So that leverage follow-up can

Ryan Deschner

some forward? now feel at big you point component feel a -- of to other this point you in growing to many is sales actually in Do moving growth grow here? How are the Yes. this tailwinds weights compared have expect of do growth Alagille? And impacting you at do how patient it an for -- how impact this how much the specifically, average

Christopher Peetz

way that have at think new look And we and is come this you kind I to the part time, quite describe changes continue in it modestly because mean have dispense. is in. we it about -- average would over I of the diagnoses

So way. there years. overall of have we increase effect. tempers the seen the along kind But starts gradually are new infant over of That the it patient kind

Operator

Skorney We with turn to Baird. Brian now

Luke Herrmann

This pruritus just is study an Brian. OCA bit not on hoping account prior you little for about as recently Luke OCA on discontinuing VANTAGE could And patient discontinuing? think you measurements a into compared patients to the impact do takes how a were We see and could use talk enrollment.

Christopher Peetz

the into I'll jump let question. that for Joanne Thanks one.

Joanne M. Quan

the for question. Thanks Yes.

at thought patients It who So there when terms would this not are point. started, the really in study out it's was issue bigger OCA. be on contribution turns actually we a of an

And kind this actually not reflects picked really seeing that of I otherwise. candidates lot We're with OCA think OCA interim much overall forward contender issue with that usage patterns quite an are patients general, space. terms patients, study so up of since a in of nicely really the that the that's there's in in who of a for just And enrollment out. our not -- has came coming results

Christopher Peetz

of In that, addition about patients recall first-line. PBC, to for that study, VANTAGE the those X/X are

kind been be before PPARs -- are used So be would or that would have used. would of the new OCA before

Operator

Our next question from with comes Jonathan Citizens JMP. Wolleben

Jonathan Wolleben

piggy What And you it's U.S. your like EU if X,XXX prior collection small envision have do wondering, wrap looks prevalence, patient a question. EXPAND Phase how backing around bunch a that heads you of in you Just trial? the then do label given a on and that of success indications? III

Christopher Peetz

nail as basket Jonathan, a Alagille several It's thanks is, a down, -- causes something to It exclusion. point of PFIC. somewhat indication of the question. definition the PSC, for ICP, out, frankly. of cholestasis, The is it's label different PBC, statement excluding syndrome you and

all it's many of less the of causes cholestasis. common So

So exact to get wording label will as need to ironed that be out expansion. we

think numbers requests for and it, that the at In landed some we coming PFIC-sized demand as we we counting of where it's use and how looked And physicians simply compassionate or about think of patient patient way of X,XXX the number, opportunity about kind Europe. that terms and we at kind from to though, interest how U.S. those patient was from, larger. up that's think -- quite of it's the a real estimate

feel as it's study, very to opposed example. estimate tangible market a I So maybe for an epidemiology

So that I for confident opportunity feel LIVMARLI. sizable quite a it's

Operator

question Arce comes H.C. final our with And Wainwright. from Ed

Unknown Analyst

questions is This asking for Thomas Ed.

target in second good the half for to on study this the first, enrollment year. it's in volixibat So that PSC, to complete hear

complete on details you Just process. outline data announced, many can see enrollment should top or is wonder months after if the expect when line how to some we

Christopher Peetz

is think Yes, thanks close it's an the X-month endpoint clean it approximately database. time of The to Thomas. way and to for simple and the question,

do announce project like get enrollment there. that kind of amount we after to enrollment So when time completion completion. of plan We some

Unknown Analyst

LIVMARLI on with well. perhaps it. Got as then And question EXPAND study one the

experience are date rare if that share which have of can common you type wonder Just to and conditions most received far. cholestatic patient so you some

Christopher Peetz

question. speak AASLD for at thanks maybe of point. on Yes, last ask I'll year to to that Joanne some the data the

Joanne M. Quan

study. are that treatment this earlier strikingly their Yes. biliary was were studied a and We use group EMBARK positive for of quite pruritus. patient note, is And do than actually for of patients those have compassionate in the a of atresia, through results different number treated

disease, often they that are atresia that were do they disease these biliary progress and young, their know Kasai likely time, pruritus. patients that as on. liver had continue will part of have when of patients transplant, progression of and quite then they will and So the develop require over a in We terms

we using there's inhibition. cases And where an treatment been so a quite outcomes have of do with good abstract IBAT handful

a also what So us confidence have that. that's partly do an ultimately like conducting that that study study that in the we is a address product there can and gives really EXPAND need unmet

Unknown Analyst

Understood. from conferences medical we this Perhaps data one interim kind some follow-up expect Should study see posters? to that. on perhaps of in

Joanne M. Quan

we an interim Actually, do plan the study. not to for do analysis

in XXXX plan is year. next to Our enrollment complete

interim, So -- did we if things actually if an any it slow would down.

think in to and the XXXX they're do for full results study. the we're available to forward we look enrollment So sharing on when track complete

Operator

for remarks. our concludes final This back Chris any to hand now CEO, I'll Q&A. Peetz,

Christopher Peetz

you Thank for a all Great. Thanks. joining and us great have today, afternoon.

Operator

now Ladies We'd participation. like today's has you thank gentlemen, to for concluded. and call your

You disconnect lines. may your now

Mirum Pharmaceuticals (MIRM) 26 Feb 252024 Q4 Earnings call transcript