MIRM - Earnings call 4 2023 | Mirum Pharmaceuticals, Inc.

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Operator

Good afternoon, ladies and gentlemen. Thank you for joining today's Mirum Pharmaceuticals Reports Q4 [ Year-End ] and 2023 Financial Results and provides business update. My name is [ Tia ], and I will be your moderator for today's call. [Operator Instructions] It is my pleasure to pass the call over to Andrew McKibbin, Vice President of Investor Relations and Finance. Please proceed.

Andrew McKibben

and everyone. ], Tia [ good afternoon, Thanks, Chief Peter release I'd quarter Quarter by XXXX welcome Pam our you announcing results the our fourth the full Eric Operating our and Vig, Mirum Scientific year today, Pharmaceuticals Fourth to Mirum Research, Officer, Chief Medical Conference Officer, and Chris company's Officer. Earlier our of like our and Head Peetz, Chief Officer to CEO, President Radovich, issued and Call. I'm XXXX. joined [indiscernible]; Chief Bjerkholt, Financial a news for today be news in website. Copies and of this release Investors SEC our filings of can found section the

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

Christopher Peetz

for achievements I'm the year XXXX, for lies highlights update and off the afternoon, of us many an company. for cover another everyone. for Andrew, what kick in to and ahead good Thanks, significant growth excited Mirum on

lives the an patients reflects XXXX, of world. difference in Mirum and the in opportunity of ahead progress to families their the making continued around a team dedication the Our

for and a $XXX business XXX% portfolio we in net year, the our internationally, sales achieving of patients product driven transformed and expansion in our This by and total million acquisition U.S. and of revenue. CHOLBAM. the to strong and the broadened was integration commercial with LIVMARLI CHENODAL year-over-year growth the reach growth Over in with continued

that have self-sustaining and a drive in medicines, last We of potential the to and unlock value for continue disease commercial XXXX great in year expansion business growing our a of The to positioned expertise our beyond. potential has pipeline. leading our is leverage opportunities, adult rare growth now shown built the the settings upcoming label creation in cholestasis grow strategy

expect strategy. This across growth commercial XXXX. year to from for the Overall, million product net current be driven at available expected execution is business, ahead million medicines. the $XXX our all to in on Looking revenue we to $XXX of commercially by X achieve

to this clinical pipeline. We year advance regulatory catalysts also multiple and have our

conduct an program to interim track these are also Phase of see we as LIVMARLI the expansion. We're a of to CTX with look and enabling ahead For forward opportunity results PDUFA rare our the life-changing programs, PFIC VISTAS XXXX of of proud for to study for to with selection VANTAGE for XX. ] the and date Phase we and of expect have PFIC, we studies bring medicines to dose important our We to potential strong volixibat the the half [ first providing adaptive market great analysis first and enrolled, [indiscernible] of PSC the for our the PBC. been this for March the Mirum. impact year. and ever grow III CHENODAL the opportunity made of studies in half on progressive the the in the excited for in interims Patients the patients preparing label burdensome label with an bring March all on to We III We're therapy year, PSC the continuing patients also for submission of progress to positive potential the And in and first results of disease. opportunity. the disease. I'm progress

turn to I new wanted before the Now over Medical call Mirum our joined Dr. who discuss commercial January. our Quan, Chief Peter welcome to to I business, Officer, Joanne in

and call. And I'll Mirum. her Peter are to We the Joanne to discuss and We'll call turn ahead later from Peter? over we commercial hear to drive this the helping have from thrilled see for leadership extensive now, scientific creation value her to our business. expertise, on

Peter Radovich

Thanks, Chris.

our XXX% tremendous growth by U.S. across that and net to in XXXX. progress have XXXX, Coming are where made compared businesses. of product total We international the off we excited saw we sales

benefits our the $XX.X seeing in enhanced to prescriber achieved quarter driving of Mirum XXXX, of franchise medicines. LIVMARLI, product reflected integration net commercial the the expanded net grew and global the across million amongst X proud this sales are already sales ] of on And offering I'm newly and and product [ million the fourth focus in evident community. hepatology pediatric the Pinedale [indiscernible] which total the seamless We in fourth patients simultaneously all growth team's while total of $XX.X of is we quarter. patient

well, representing quarter-to-quarter are million quarter Our for million uptake saw to seeing but U.S. And we consistent growth. internationally, we business variability anticipate as XX% strong year-over-year international and respectively, $XX.X continue approximately the $XXX in and year, revenue.

$XX.X to quarter XXXX Mirum's for all quickly net bile as from with CHOLBAM. trends. Taking growth ahead, million we to-date sales And we X with products. we first portfolio these leadership, to CHENODAL increased was million full very for The quarter be been represents together, these looking product commercially quarter, quarter acid fourth year contribution with how our summary, over with all have the product the X the happy for strong worldwide. expect line the into $XXX expect steady to medicines available growth X medicines net million, dynamics able in business. transition This our the in business sales to of Moving historical team expanded fourth was XX% recognized and $XXX and under XXXX. In our products commercial these the and I'm to in both XXXX our and from medicines highest the presence on medicines. In for grew we a fantastic U.S. we

continue patients in Joanne. to of I'll to see medicines look mission the stay X We turn we committed communities, tremendous all as across their it with impact patient expansion over of we life-changing that, Joanne? our to delivering to worldwide. And and further to products forward XXXX

Unknown Executive

Thanks, Peter.

short and a continue XXXX, I'm in join the the regulatory I'm treatment PFIC Before rare of half and the regulatory we I we progress like excited of to thrilled Mirum. in accomplished diseases. In of well such decision team experienced period momentum EMA begin, are towards development our time. the first to a milestones. to that and perspective, and has say This multiple so date looking I'd the From are tracking year. in a of for March PDUFA forward to expect much XX

CTX, NDA of in we're progress FDA XXXX. and positive good We've making our CHENODAL are is here the clinical to which had well. as no support first planned interactions required studies submission, half Regarding and additional with the

upcoming interim in VANTAGE volixibat. to to our analysis patients. in study PSC PBC forward study looking Turning and We're our our VISTAS also

this the to the supporting are that enroll now half We track year. of read the Note are registration. goal continuing out for analysis in patients with to on interim studies of the first

be As a and the part a analysis in PSC dose selected interim arms patients reminder, blinded just the selection. included study placebo upon VISTAS will this data the dose will allow have to Based set. pivotal to the data, from

without discuss we a unblind in safety. For where will for include the of to cholestasis from now the treatment look PBC, results. sharing team bile of is represent that, and top ] to [indiscernible]. analysis, portion options adult the acids important Both its [ these significant financial change designed improvement, studies part and and turn data bile pruritus our adequate [indiscernible]. With be are the acids interim show progress Eric.to with that and indication of accumulation serum an to addressing groups forward step this larger I'll the patient I'm the over our symptomatic towards call of cholestasis line so severe on excited results Eric? with the study which in of patients broader

Eric Bjerkholt

XX, fourth quarter the compared XXXX, ended total revenue million and year sales total and revenues million, December today, was for the audit and next This the respectively. is planned that $XX.X full year of results I'll $XXX.X million $XXX.X net financial included was million of the our which quarter Joanne. filing for as XX-K procedures issued Earlier in year, and finalize release is we and our week Mirum. Total $XX.X for a million. to the other press product including revenue quarter briefly total XXXX Thanks, fourth XXX(b) full auditors $XX.X summarize. Note, first for for

SG&A $XXX ended and for For million, of and expenses which includes $XXX.X of were operating year research expenses sales million, $XXX.X cost development expenses $XX.X of million. the million, XX December of

For $X a reflected the or in December XXXX included is of sold. In amortization million in of the amortization acid stock-based intangible for noncash compensation primarily net million This acquisition. Net loss COGS per the and was the of bile million our $XX.X also excess year quarter, inventory expense year $X.XX to largely $XXX.X reserve intangible XX, related fourth reflected goods ended loss for share. cost million. $XX of

the activities year XX, was business. used $XX.X December We before. well had million XX, equivalents December ended investments cash, a and with For million XXXX. remain million, strong in down and the year We of cash $XXX.X of as 'XX, $XXX.X funded cash from self-sustaining operating our

business development over and years. places us the of coming revenue increasing commercial base an Our clinical global exceptional to continued through the position grow expansion in presence our

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

Christopher Peetz

biggest for a has the portfolio. for [ Mirum, Eric. with quarter Thanks, the strong Biloxi revenue ] It been finishing and LIVMARLI year ever

another catalysts look disease we We clinical continued that, XXXX updated products in make several please are operator, advances rare and our with for X with around exciting throughout questions. commercial the year. And forward regulatory We to for to world. the great on for call open strive and patients pleased growing with you year progress keeping the as

Operator

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

Unknown Analyst

an more update CHENODAL, if for Maybe any? the growth This versus to Jess. looking in seeing is guidance what net bit there a LIVMARLI baked a is competitive of much Nick on bit for sales can $XXX million you're maybe CHOLBAM. on the how at about little Alagille [indiscernible], provide XXXX dynamics product And maybe on million, you contributions discuss LIVMARLI also on from comment $XXX

Christopher Peetz

question. we're little across product color the point, give really dynamics on Peter business. let for this great point, And of just a across kind I'll Thanks starting at the from and seeing level Yes. we're the products, guidance but a first, what not giving

Peter Radovich

kind then product see dynamics portfolio, think trajectory LIVMARLI, of think can impacted continue expect impact. the grow And that. question of do variability the other growth order the now side, you Yes. And about competitive last commentary been LIVMARLI of in have growth all the the over it quarter has a has as looking to LIVMARLI team. measurable seen spirits, those as just forward you its move acid to I then we into for numbers which sales. And on from that really [indiscernible] some trends, then think as to the the component U.S. bile Expected at there of growth historical I color been on see by your there, on case international strong overall afford it the with to marketing. quarters we the year-on-year international expect mid-single-digit from rates. And been in about

Unknown Analyst

maybe a these differences observed one settings, Great. you itch populations? Alagille the X little and/or study severity patients second-line patient VANTAGE maybe post the And it across how more. do is talk setting that rate X first-line more [indiscernible], observed Thinking about the setting and these [indiscernible] beyond how in the can about presents of just [indiscernible] the about patients of bit maybe in

Christopher Peetz

Joanne therapy. between can and difference line I to the speak the

Unknown Executive

Yes.

Pruritus So is of big issue diseases. as you a these know, in both

the positioned of this remind agreement [indiscernible] population this looking have the registration that studies. have than, you actually, analysis. to registrational we entrants terms instance, us space be with both interim broader [indiscernible]. accessing that that end FDA point actually I'll in broad we're the there's provide the at of biochemical We patient -- after other are for And is and who these are in from some studies a

at in terms selection pretty the [indiscernible] the moving itself, drug feel forward our confident we design of dose So point. both and this

Operator

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

Unknown Analyst

This is for on [indiscernible] Gavin.

two Just us. for questions

For the first one.

higher start grow? so especially on just LIVMARLI, the wondering doses For patients reweighted and have being the with if of year, they as you seen

Peter Radovich

of some with weight-based's And seen product. published to is that LIVMARLI published adjustments. dose information have growth year, rate-based We we that and see a last not we've surprisingly, the

Unknown Analyst

you commentary access a impact name, the also on would one [indiscernible] business. Right. the to of [indiscernible]? or there potential have Do for EU? more implications different then under [indiscernible] I EU And confirm with brand on that then And be in reimbursement LIVMARLI EU want refiling any no not

Christopher Peetz

other Good [indiscernible] international grow markets. making across care a we question. we're Alagille getting to really much progress all point, and and Can't see international rolled demand across LIVMARLI, all Europe broadly focused speak great the than markets. with out LIVMARLI other At we're standard procedure continuing progress great too really getting access with and as markets [indiscernible] this of on that to and having [indiscernible] across

Operator

Foroohar Partners. Mani with comes next The of line question Leerink from the

Lili Nsongo

way couldn't and hear a but if sorry, Nsongo as we increases This was then that, to the This answer. is existing about the earlier, or we look I'm get was expected new for and -- I to patients the dosage think it for growth [indiscernible] Mani. I the and asked -- should if ones? based older hear heard we could apologize couldn't versus patients regarding how in the of on add of terms Lili should I comment for [indiscernible] question, you

Christopher Peetz

Yes.

it and label. question color -- I -- here per do see are make the over patients around that And recap over time adjustments Basically, question do was we as original ask on we question see time. the any just for So dose from therapy the that sure to to Peter. the and

poster we mentioned a recent think it. been I on there's presentation that

So a with physicians. see we that out that is dynamic the treating

the we're pick able so And to microphone -- up us time. this

Lili Nsongo

opportunities hear in clearly. the maybe could assets. a growth of you those assets? follow-up we are the comment as international what terms for regarding very on Could Yes, And Yes. valuation you

Christopher Peetz

Yes.

also I'll Peter about question have bit label bit and a expectations about the CHENODAL opportunity today CHENODAL, the speak a a opportunities. And So for CHOLBAM little to then dynamic we let what growth of expansion of little CTX.

Peter Radovich

Yes.

to in CTS III we next continue approval CTS. I line products, recently year all certainly see think data trends, for Phase CHENODAL for historical with and growth with potential announced with

CHENODAL been never for I opportunity think there's an promoted CTS before.

CTS XX% under and an there's are and disease maybe is rate. opportunity know the more only find patients awareness So patients I think to One diagnosis increasing hopefully about through the state of thing we diagnosed about management.

is to more So patients and the to opportunity therapy. there identify on try

Operator

from the with of comes Ulz line question Morgan Stanley. next The Mike

Unknown Analyst

for is any that And This off-label? And just [indiscernible] Can LIVMARLI it using the Mike. PFIC? on think you do patients upcoming for currently in preface associated? talk about are your you launch expectations PDUFA any

Christopher Peetz

]. Thanks question, the for Rohit [

for to we're the opportunities And On preparing I'll see up. excited some expect there. Peter to the let how we're regulatory so a front, about coming first date we the be little bit where that speak of and PDUFA we

Peter Radovich

potential the PFIC. for really Yes. and excited about We're approval LIVMARLI

to The and to I our is universe that that help certainly strong offers us. LIVMARLI will I physician data the stakeholder identical essentially proposition prescribing various audience. think think reinforced the the [indiscernible] March ] [ value groups,

no really patients receiving need it. expense increase on to or clinical expanded to a commercial be to in it's And have the number access operating we roll access mid-XXs So are meaningful LIVMARLI who approval. do eligible would who of the for rollover

Operator

comes question from next with the Seedhouse Raymond Steve James. line of The

Unknown Analyst

Steve for [indiscernible] Seedhouse. is This

is margin. to related question first So our the gross

to mentioned the think acquisition inventory in there I was charge you an related XQ.

the Just XXXX? trajectory for in margin And expectations in what perhaps your are XXXX? gross also OpEx

Unknown Executive

I related acquisition for XX acid amortizing amortization which margin, do the the to mean gross over will us, intangible largely we're intangibles, that it's quarters. we continue. As expect bio

of actual the as sort of it product goods, As of cost cost traditional and royalties. consists far of

So of same inventory did with have -- at of the the a approximately or larger amortization we then forward sort that continue will we sales. And going that percentage than acquisition. the expect came for reserve

quarter. So some the we but the extent fourth had in not some have reserve might quarters, in to we

and of R&D SG&A. overall expect we of pretty quarter operating probably next few is quarters. the terms In expenses, what Fourth representative

Unknown Analyst

Okay.

And on question your in enrollment Okay. dynamics disadvantage. just a overall then this

in your on only interim think patients. VISTAS, XX is I

enrollment about the And you date? comment overall target enrollment you of the differences in do the see can dynamics? any terms studies between So

Christopher Peetz

mean in guidance is enroll patients we give Thanks at able for I for have we're up. now. for be interim Part it comes the for the the X continuing better question. when the we'll And answer to here to interim, full enrollment study simple that

Operator

The next question comes David of from the Labels Citi. with line

Unknown Analyst

wanted We PSC This the blinded on ask interim is to for analysis. [indiscernible] David. about

would selection, share any other data blended dose points the besides So at you a basis?

Unknown Executive

So and blinded predictive pivotal and if the this be [indiscernible] in that it designed be interim is It significant analysis so will of to threshold. we the positive end. design the passes in statistically a

I really know on about ]. that's and the point. share really can the what this it's class at PSC special So drug, of general this all based And with drugs, pruritus developed we is you and [

feel being a So of to for us. we the well confident productive robust study pretty and in registration this of up being set kind design terms

Christopher Peetz

of of VISTA the communication would extent study, would team the of occurred at positive that be study assuming has here communicate terms the that the interim for received and continues on as we the point planned, we In well. as the interim information interim, a which

Unknown Analyst

Okay.

you like mean, a not more is the is [indiscernible]. you So bit like about I see that basis that, on out us tell somehow other as while follow-up efficacy the a like -- blinded to scenario a was would there that crossing unlikely, that if could play threshold any

Christopher Peetz

of that next the scenario. signings where for design are top look an study open look that share to we'd it the in and the at analysis, basically interim the study met, to threshold be in steps converts at able event the data, line the what So not is

Operator

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

Brian Skorney

you and other And impact and pruritus, the thinking outcomes to in about beyond on physicians markers this help PBC, how patients? you make are how can the On case VANTAGE?

Christopher Peetz

Joanne little registrational kind Thanks of plan the speak Yes. for to for bit let question. the I'll a PBC.

Unknown Executive

Yes.

So in going to just looking terms interim, the line. at of we're be top

So And in big issue as therapies. no particular for know, as this really [ there's pruritus a line. and top safety patient you ] is it [indiscernible] population particular which

this actually it's important pretty medical we're addresses at point. this confident an the need So designed, way that unmet study,

Christopher Peetz

just criteria. to criteria points VANTAGE the of not alkaline one the the And for eligibility an recap, made study does Yes. have earlier, phosphatase

are that and still line on patients So severity UDCA it of rates the same experience label treatment. as only first [indiscernible] thought of has as later includes of traditionally really and that as lines

across lot a need patient of unmet So the PBC population.

Operator

next from comes of with H.C. the The line Arce Ed question Wainwright.

Antonio Arce

Congrats on another quarter. may. strong if I have I X,

Firstly, VANTAGE on the in PBC. study

sort LIVMARLI, U.S. was was Thanks and Peter, think hear in the through sorry, for international competitive a much. remaining [indiscernible]. it's that you're as also to reviewing year. And as -- I Obviously, you'd also specific fourth thresholds opportunity the so looking this the so and But of are proceed, didn't bile [indiscernible] if quarter. landscape speak at, Peter not trajectory it's it about quarters you then products the to as sales pruritus acid when the placebo blinded but wondering the said, ramp, is, what you data where the lastly, the of activity of come on both think and speed numbers as well you of you're the the sales, As I'm in? Secondly, be this terms if there PFIC I whether expecting basically for pruritus you readout. to just bit mentioned, about versus of kind like see that I'm of could much a

Christopher Peetz

of recap down comes and of Peter various on on I'll to patients the pass think this and majority and patients, PBC, here therapy expected labeled we pruritus. sales the agents first-line PFIC some way in to recap points numbers. about hit the really one The Maybe to then are lines highlight them And are talk and have of be it the of about labeled. -- where those over the and VANTAGE it VANTAGE majority just and to volixibat of on

after. the So that that's going unmet need we're really primary

meaningful. And be going we're impact towards to look significant that's will that the next at pruritus, pivotal that, and when a interim to continuing that be that on we steps. decision think, we lens showing very So into it on X I'll to data of the clinically make study. With portion a roll and the Peter over pass it points. the taking on next

Peter Radovich

try a Sorry, that here. to I'll bit little speak

XXXX we'll positioned closely approval LIVMARLI PFIC revenue potential I think upon payers spending their the piece, be on get working we'll new-to-market to here be So bit fair a in policies. a time in expectation March of with

So with in more we'd PFIC I path think or free of think expect XXXX, really lot XXXX. a about you coming drug in

of similar And million, high totaled the [indiscernible] $XX.X Somewhat to for early pop we where from the million QX, what million worldwide, were acid then in saw terms quarters QX. then launch $XX.X LIVMARLI were in $XX drug. And bile products numbers, $XX.X initial the million international.

Antonio Arce

PFIC. the Great. comments. And just on appreciate then a maybe further I clarification

to given would wondering, expanded over Just those soon there's that able roll How about XX-ish patents you access. be on mentioned on now drug? right to you patients to commercial earlier, expect

Peter Radovich

we in What occurred two, quarter launches we what in the generally, saw. saw that or Yes. is a [indiscernible]

that's so I with for those And 'XX probably so. our or expectation consistent think

Operator

the Citizens The line next from comes of Jonathan JMP. Wolleben question with

Unknown Analyst

what there's also of as a coming. kind earlier out just launch, that I pruritus, might PBC of kind it is SG&A quick And going whether ] know about setting PFIC in that of of questions. of get know said follow-up how [indiscernible] that else proof be of too into be you sort John. potentially agents on as effective? much for of the and to being of [indiscernible] see I of sort a to done word is an the it's Catherine the growth, patients? monotherapy the fitting two kind kind [ not volixibat investment combo other far This like kind guys paradigm preparing on therapy just for as then see on had Also as for the you there you far

Christopher Peetz

study U.S., kind where one look to upstream front positioning right? example, The for in And pruritus, that [indiscernible] you recap are play that patients -- a VANTAGE can of [indiscernible] when is just a more criteria used questions. programs, being first-line therapies, includes at unique biochemically setting time eligibility gets outside have the can the and the the really how for role the where So here. for PPAR in of other PBC Thanks OCA, are the for the they that's substantial we still patient second-line of when controlled, PPARs area very and example.

we add using pretty where see positioning bringing So endpoint, really in and unique a an an on for PBC. positioned that here for as only patients. I'd therapy by is to PSC, for really the these therapy exciting be new outcome first use volixibat pruritus opportunity gives to to PSC

So positioning here. quite going X pass to I'm excited about Peter for it over to the there. part

Peter Radovich

been mean, obviously, in on country U.S., the take data there's talking have these about I care approval. II of on But the the emphasize patients. medical positions until of of mean, data going back probably take been in as at to of care that Phase that regards In PFIC, just order for years. promotion conferences physicians -- the number data I There there's the presentations major XXX I entire a no well III that as patients. the you're kind Phase PFIC of really Yes. an it's small

support with clinical our Plus familiarity LIVMARLI's kind of using with prescribing a kind it. there's So of a from profile, of high awareness patient comfort relatively and commercialization, hub, certainly, the [indiscernible] [indiscernible], the I approval it, working data cetera. program, et a think lot [ ] Miramax with and of

that's think stand a summary where today. of things I So probably fair

Operator

back additional are this Peetz There closing no Chris any questions remarks. for time. at it will to I pass

Christopher Peetz

of call, research us to long-term joining to plug-in than years. Tomorrow, for in part I today around the everyone today. to hepatology Alagille data some tomorrow, for again Thanks build published Day in excited in that featuring patients to with Rare put X,XXX world. able rare LIVMARLI. a we're Day, on the in impacting diseases a want for Disease treated manuscript Great. share the recent as with and the for meaningful do for [indiscernible] celebrate advances of syndrome more Disease Rare be patients seen we've awareness

patients many advocacy Goodbye. efforts supporting research please, a support and and tomorrow, have [indiscernible], So to evening. the in passionately lead rare great that disease groups

Operator

today's conference Thank concludes you. That call.

now disconnect may lines. You your

Mirum Pharmaceuticals (MIRM) 28 Feb 242023 Q4 Earnings call transcript