Earnings call transcript Q1 2023

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Operator

Ladies and gentlemen, welcome to the Mirum Pharmaceuticals Reports First Quarter 2023 Financial Results and Profile Business Update. My name is Glenn, and I will be the operator for today's call. [Operator Instructions].

I'll now hand over to your host, Andrew McKibben to begin. ahead. go please Andrew,

Andrew McKibben

our good Vig. Pharmaceuticals' I'd call. first our Research Chris today and and Peter you Head our I'm to to by of Peetz; CEO, and Mirum quarter Development, President Operating afternoon, Glenn, Officer, like Radovich, Pam everyone. and welcome joined Chief Thanks, XXXX conference

Investors release the of today. XXXX. the Copies Earlier today, release of news found quarter a section filings announcing the be this in XX-Q Full first updates news and news website. Mirum issued company's SEC can issued results can release and details for the from our of our be in and found quarter

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

to Chris? I'd With to over like that Chris. turn call the said,

Chris Peetz

to focused life-changing been Mirum, disease It's everyone high Andrew, position on and call us as advance another on today. company as leadership quarter you, rare afternoon a exceptional to for growth our medicines. joining Thank good we continue the

needs business are by unmet Our achievements revenue in patients $XX.X quarter team's of the with million total addressing disease. our passion driven urgent expertise for rare in clinical, first and with and regulatory

we're place, disease progress already global become pleased made leader we've in five in in our a part strategy to XXXX. with With rare the

in driven Alagille on of starts. by we've on First, growth to the well build XX% continued in guidance product syndrome launch of year-over-year sales net new patient U.S., LIVMARLI tracking the successful our

Second, early XXXX. our markets. France in forward in programs other international building launch this the launches into well with later also recent We and access international around Germany business look additional and year the globe to and is partner

accomplished our strategy older. we've to the step Third, expansion announced first the we include and three to important in LIVMARLI. for label quarter, expand months patients an In label

equivalent our Phase In addition, we European with and PFIC X positive data. LIVMARLI submitted a NDA supplemental

PDUFA for preparing Our the excited patients. December treatment team option new bringing is as PFIC advancing XX for about care date LIVMARLI and is a

We indication data in from also Atresia LIVMARLI. year, anticipate second a top-line this for Biliary EMBARK of study half third the the potential providing in

opportunities pivotal advancing strategy, indications, the Biliary in look fourth, Primary providing our leverage industry-leading capabilities. rare the of And And Mirum's potentially part in conducting we year. half forward studies for evaluate into are and diseases Cholangitis Volixibat Sclerosing continue fifth across studies interim in Primary adult from we these this and to updates of second to Cholangitis for

interest the also of enabled $XXX approximately in strategy recently excited with We significant our enabling aggregate principal step amount. repurchase capital level took to This of strategy convertible the note million the a see in prior our towards supporting of this to committed the financing XX% our growth financing royalty nearly high balance We and investor oversubscribed transaction. well sheet. added additional to royalty were

balance our three that project plus beyond runway years to sheet continue provides cash flow of We breakeven.

team this with about is provide beyond We life-changing what poised to the off great achieve have patients and medicines. as momentum, excited XXXX with and started year Mirum I'm we

before detail to that, discuss call in the Pam gives Peter? pass update. I'll Peter to over commercial R&D with an And more our business

Peter Radovich

Thanks, Chris.

We million the patients $XX.X growth and in the which first are quarter included $X.X of U.S. the in with XXXX, sales U.S. $XX.X And substantially of markets. all million The was a we're the addition strong well the from report product as net accounts key million that cadence. U.S. LIVMARLI prescribed to pleased from retail LIVMARLI. new pleased as international of liver pediatric have by driven in the

Alagille of experience Access they the their of health to on across increase syndrome access intend professionals care positive Based clinical report LIVMARLI and Mirum Plus, that ease their use patients. with

U.S. XX% in the together, against well product performance is XXXX tracking growth business for our Taken guidance net sales.

reimbursement to already line Now with LIVMARLI XXXX, European throughout commercial off in have expect and to in and early in and Germany countries anticipate markets European quarter-to-quarter beyond major turning and full distributor novo in what patient discussions We've a variability U.S. we In drug, program patterns. Germany, which it's international XXXX. markets, XXXX, access continue in de start. we saw are unfold Pricing we to we seen launch. although Also, throughout we converted in the launched ongoing named to expect demand, order European great in launches impressive sales to with we in QX, markets. patients starting

exciting is cholestatic familiarity on is this a syndrome and horizon. globally support. as profile and and access indication position, long-term well position outcomes. to opportunities patient XXX and in propelling providing in rapid exceptional confidence LIVMARLI over standard LIVMARLI's pediatric a in clinical of a as just strong strong with believe growth LIVMARLI date. after cholestasis the first Clinicians in patients we its pruritus have Alagille the competitive robust continued And provides Alagille becoming expansion a in with with only treatment treating Alagille gained year in story as treated for market syndrome improvement syndrome symptomatic care summary, half In in is that relief

Pam? And over on call that the note, I'll turn to Pam.

Pamela Vig

milestones and Thanks, growth the programs more to first in Peter. our we important our impact that In patients. pipeline achieved the of quarter, ability support

been accumulation reduce toxic of As bile versus acids. and shown is showed LIVMARLI in has standard pipeline a diseases of care. accumulation, problem a the setting, reminder, cholestatic directly outcomes that acid classic burden symptomatic our of studied In to these improved such associated Alagille bile long-term debilitating a IBAT inhibition an all in

was of profound PFIC the two benefits age end PFIC PDUFA to supplemental the of the impressive of of for accepted our Phase in our for And submission last shown patients April, date at the we're in December was by share of submitted XX X in also data that March EMA year. older. The months we and happy NDA inhibition of study And this from LIVMARLI year. approval announced to IBAT

statistically both pruritus bile drove greater our genetic Now types the PFIC acids the data broadest and reduction proved study IBAT range using ratio studied a higher significant PFIC across in serum highly the with from dosing hypothesis inhibition date. for March of to LIVMARLI

growth they as for in as versus treated in early and bilirubin, find levels group. with in where bilirubin of XX% bilirubin patients addition, improvements seen zero statistically the we've normalized In abnormal in that patients LIVMARLI, well significant placebo with

Now physician an population. beyond forward to heard and the these have pruritus community from if expand strong and pivot feedback improvement approved, data overall suggest look patients, a the to clinical positive for these broad we to LIVMARLI opportunity

track study placebo-controlled an to is when year. in really the that program for for of readout happy share them We're Atresia our data we This second remain this to IBAT first data announce study, excited the this the inhibitor completed. half on Biliary all for will and top-line also study for provide we're

is forward and assessment Atresia the at the bilirubin marker a progression And of the our primary bilirubin to study Atresia Biliary As and reminder, the BA study dosing the readout. is XX this as the same the improvements in encouraging study as is most endpoint in PFIC observed March setting. in bilirubin regimen predictive weeks disease we extremely March look transplant are the as we Biliary

pipeline programs cholestatic Now to adult turning our indications. in

of analysis acids inhibition the of second marked pruritus serum We've and setting. from studies our can be in potential this half previously Volixibat with in shown in Volixibat underscoring both PSC achieved PBC and for interim in bile reductions expect this PSC, the that year. IBAT We in

for bile these being with off in BID acids. learnings for are XX% therapies, which are a pediatric roughly upcoming actively our higher confidence at adds our Building off-label dosing none patients to dosing ineffective level interim currently, regimen, of of we PSC of And approved there are therapies lower which program, managed our and analysis. pruritus largely from no and

we real continue world. the I'm academic team's collaborative our And these of to the deep and leadership. leveraging as committed advance disease we as to benefit studies to proud continue efforts We're our cholestatic finally, from our scientific all clinical settings patients of across for experience

back to over that, Chris? the Chris. And I'll call with turn

Chris Peetz

studies. PFIC dataset XXXX is poised for transformative anticipate We growth, throughout Pam. year rare Volixibat an commercial Biliary strong in data and new that of work our Mirum will analysis label potential be to about and Mirum the We growth excited continued we interim ahead. disease. pivotal expansion, top-line Atresia prospects of the the of to medicines randomized inhibitor remain potentially years with Thanks, patients another with as from provide potential IBAT first growth

with please open And questions. for call the operator, that,

Operator

you. Instructions]. Thank [Operator

line We have Jessica now comes Jessica your from is our Fye JPMorgan. question open. first from

Unidentified Analyst

that shift? shift questions. Nick from the little resulted is timing Thanks second quick that that mid-XXXX on for a this a for Hey, me. factors just trial narrowing could 'XX? the half of in ones One, more about for Is Phase VISTAS Jess. or bit Two a Xb the our taking for you to talk

Pamela Vig

question. Yes, Thanks hi. the I'm happy question. to answer that for

a really for and then reason scoring. in One why compliant, delay that study. sales are delay lab the make their So they minimum out. to the because screening the kicked mostly slight not during meeting the compliance want pruritus if for in to they on patients some the And also remain PSC number some mix enter study, of study. there's put the values out, have screen it's pruritus and not where screening score the for of threshold reasons that also can a based in is to we get And is they're sure itch they

plans just good see movement What starting those on continuing to this some of and with we're advancing of is got we're and drive interpretation and scores how and we we've forward, activity. on education training itch tools standardize doing to more and

Unidentified Analyst

Can help mitigate as know trial discuss there that quick a how the some on the just to past in of into VANTAGE itch. risks? And run have PBC have those I kind you designed been with follow-up. of placebo kind Great. is issues responses high trials

Pamela Vig

question. the for Thanks Yes.

-- So you we rate. clarity in know, -- placebo a PBC ourselves study, we was a had our response there as

for that. that and that about in taken give specifics way but applied this that We design. accommodate we assumptions for study failures study we into the of to in placebo baked for I don't we're trial, also took into very I So that about some the learnings the doing deeply thought those designed that into our our we analysis, and account think them can adjusted have account.

Unidentified Analyst

Great. much. Thanks so

Chris Peetz

the for questions. Thanks

Operator

Thank you.

Mani now Our next line open. from Mani is your SVB question comes Securities. Foroohar from

Mani Foroohar

quick patients, would Atresia. pursue expect the on year, feedback two your the of life? A expectation treated? how on well here drug terms some we years, scale Or successful should period that look patients leave defined, of you'd case position would of they number is the both question a treated a study? it would long that Is withdraw from be than Biliary them time, I one you which also study How of what patients clinical rather of commercialization in but to for at about fairly the opportunity, point be think in and the think lifelong would after

Chris Peetz

is of I'll first I want for transplant kind most just Mani, common transplant the -- indication and liver the Thanks, Atresia pediatrics. mean, question. comment, for Biliary I in to

bit indication terms to build over scale Peter there some ask have, of duration need to this you nuances maybe time. important over speak a how of and impact because think of treatment, time, little are about think I how of this we indication. can to unmet in how really evolves this really on a shall So is the can

Peter Radovich

is incident think Yes. and think piece it Chris about threefold bit newborn more generally think because an more that common on pretty the is because of you when it's the But and Atresia than as Mani, probably from perspective, PFIC on about I less by you in Alagille. progressive there's there, transplant. of And a much towards describing, there liver pool that fair prevalent heads common Alagille incidents Biliary dynamics rapidly a

the therapy -- the that eligible of the it and if other we again, does on than from in drug does But indications. are being within hope of born approval, population of So maybe you two think who I the could order we'd liver XX after to and, hopefully would XX patients over what early last envision the the keep kind kind better time them on kids months. as build years and be transplant get patient

Mani Foroohar

[indiscernible]. And certainty the hopes the regulatory product it's target our when is how in think that about by, little talk about show regulatory because to the you less more up need ask dynamic helpful. it little certainty drug give we a clinical can one profile? effects you here, And there what of Obviously, sense questions? a doesn't Can very what do. for That's target around internally your uptake? as a on also is air bit Or view us in

Chris Peetz

Thanks for thinking there's are is this I we'll mind. do mean question have data-driven. that a about have aspects And conversations patient after to they the data and the of there. regulatory unblinded, couple in set consider interest regulators

which of bilirubin, the be on could a is in case show really work for approval convincing and and a potentially data we parallel. with the we're That EMBARK some study set importance doing understanding also of some the advancing paired conversation good really Biliary if of the in disease strong basis will the support Atresia. think I why so And

We generate do need into that data, design. kind of that's the baked all and study to

I addition three the on for There's level in Pam to are really change to think just looking that at profile the talked PFIC transplant months. I'd study, established mean in well that from point data bilirubin. of that at about terms thing the in categories of bilirubin also risk one bilirubin

Each X% is close a a patients outcome. X% X% on bilirubin below to difference step that have score. above down It's for we and bilirubin. in of on X%, So meaningful risk the categorize able being eye to

of So we'll kind a shift look those at or table responder in format.

Mani Foroohar

guys. That's All Thanks helpful. very right.

Chris Peetz

for Thanks the question.

Operator

Thank you.

We Seedhouse now next comes line from from Steve open. is have question our Steve Raymond James. your

Ryan Deschner

This Hi, two Ryan questions for Deschner on Seedhouse. is me. Steve from

The reasons? often weight? for step-up have growth in year than more patient appreciable first And the -- treating you seen how increases launch, average into an sales now LIVMARLI other physicians due to that a in are dose in adjusting we're

Chris Peetz

Thanks for We considered increases. do just year, the prescribing real new there a consistent the world setting novo the Really, dose think they're patients in patient and the with I information once growth question. majority is is about the dose de for What are LIVMARLI. weighed demand we're is and we of see starts. see for increases seeing

Ryan Deschner

odevixibat able then Excellent. And in from Atresia? differentiate what in anticipate Biliary being also, to areas you do competitor potentially

Peter Radovich

-- Ryan. In thanks terms question, of for the

to big setting. this Atresia, to EMBARK a LIVMARLI part Biliary data of terms terms on In sizable this time have data thinking and in really randomized our will a really data I our a of half on data set think set here. having was of the give us having strategy of The a year in track study robust LIVMARLI, second lead on

open-label the also at patients a bill of where the We on not that study, have in base study six feature is months, to roll therapy.

to So look prevalent really the there. important later out starting effect is at are that patients the of treatment from

here. at that that's looking we're So really part key a

similar bigger is other to our then think, we the expect clinical what to And settings, have of dose LIVMARLI, higher in a I lastly, we've impact. seen

we've I to that's like IBAT of feel to how show top-line data. really in think inhibitors dose our and understanding our So that up really going advanced

Ryan Deschner

you very Thank much.

Peter Radovich

questions. Thanks for the

Operator

Thank you.

have our Debanjana now from coming question open. is from Chatterjee We line next Debanjana Citi. [ph] your

Unidentified Analyst

I'm Hi, from thanks David for taking Lebowitz on Citi. call. for our

And build? expect U.S. what could ex terms So U.S. the comment forward? in sale of strategy ex should you the we inventory if were on wondering we going

Chris Peetz

in where we and true we a very little we're get of -- sales markets in there markets the Central programs are Germany The sales effectively distributor some those have started of Western countries. Yes. or was selling patient zero lot in, really other We the our inventory, our directly Middle Eastern $X.X named Europe, QX, of in demand as that in -- not the million in terms international balance do but in that sales there's Europe, from partners saw East.

cases, seen patient country-specific mechanism. and our a and the supply local identified reimbursement partner Oftentimes, And get mechanism through generally, there's one a is of those in works patient. XX what six happens individual ordered physician some and patients those we've the dynamics for even distributor months three, in access --

-- So days of not those XX to with of cadence see kind the kind obviously, refill where like U.S. so you're every going you see that's consistent orders. in a

of we the that's So kind dynamic see.

think it's of from excited And gone early just has we get of given that feedback to started the the visibility XX% international have excited sort we on international kind patient the guidance a getting, But setting, provide in and is lot U.S. really I for terms position well. too that on there. the we're And based firm we're sales like really in perspective, and the just we're growth the Germany U.S. of guidance about looks

Unidentified Analyst

That's very helpful. Thank you.

Chris Peetz

the for Thanks question.

Operator

Thank you.

Ed from H.C. line Wainwright. have open. next is We now question Ed Arce from our comes your

Unidentified Analyst

is This here couple Ed. a for asking Thomas questions of

First, congratulations on the very impressive quarter.

with the tell appreciated? you to out on would to So factors percentage if be of stay patients trying LIVMARLI LIVMARLI you chronic tease LIVMARLI stay us Can what can on XXX or just segment? the Alagille well major roughly that's being with -- first globally. -- as treated by some syndrome patients that for off stay provide And

Chris Peetz

all the protocol. part -- familiarity the Hi, strong in prescribing see study the been with statement and history prescribed patients as of number of really with been the Alagille number question. who have on for interest a sources thanks That LIVMARLI. for program over really comes XXX from syndrome the clinical we We or

that really by makes sense by feed -- out or source. parse a to So really that into to all number country the sources kind it's of Not that.

that to products, ways compassionate in their different what expanded But access had products all way we that formats. use clinical study, across is commercial different LIVMARLI patients say of program, the can found

really the same of see persistence. high high You compliance, profile do

staying of plus impact it that six years have. patients can these the of program, with the Some because profound

Unidentified Analyst

for Thanks Alagille Perhaps sticking the with finishing syndrome. [ph]. proper

the age tell Can months are from both you standpoint us just U.S. down With above. what and as well? three for label to expansion your a top-line and expectations strategic of were also

Chris Peetz

expansion the for of are where on really Yes, important patients down age, months diagnosed. label because three to that's step Alagille most

And these allows we for get view, consistent So look smaller mind guidance really in top-line XX% long-term as a impacts. it fold Keep option international, we'll on. younger, treatment we into guidance with our at growth that important but well. of in the staying patients, early that U.S. the as as how LIVMARLI Overall, in, further year-over-year are

Unidentified Analyst

Got And to the application it. PFIC gears next we should expect milestone, in switching with then submitted your When perhaps to Europe. the opinion including perhaps? CHMP EMA

Chris Peetz

Yes.

So QX date we the QX. in or -- late PDUFA CHMP and about opinion potential that, would shared an December, for be would timing be excited the from in

Unidentified Analyst

it. question. financial. one final one Got This Perhaps is

convertible With estimated April, can the you in growth tell runway to? us offering what's your out cash

Chris Peetz

Yes.

fully are to to current similar the and So really flow prepared positive. in with the breakeven funded described business remarks, cash

of really plus be So just operate strength to there to the three years and the that the to grow runway continue business see we and here long-term meant an have products. for the indicator

Unidentified Analyst

Thank Understood. much. it. you our so Got for questions. Thank you again taking

the Looking forward data. to Biliary Atresia

Chris Peetz

the Thanks question. Yes. for

Operator

Thank you.

comes Baird. our have Brian is question open. your line from from next now We Skorney Brian

Unidentified Analyst

Brian. Luke this Hi, for is on

you maybe of enrolled of And any an particular or versus the generally Do Thanks. Just able the with territories? patients have terms the geographic of top-line update a readout to then dispersion include couple in outcomes beyond are you on data? might bilirubin on other in the just to expect detail U.S. EMBARK. you characterize scope

Chris Peetz

can't for other to getting the top-line, be the updates in other thanks The is overall, outcomes at time And would there disclosed. than speak specifically that not point. So else on what primary, for study powered than material really you question.

then sites nothing geographies, we're to point. have to would anything around and on point of globe, across the that we if unclear to activity seeing share So really geographic presence And all really there.

Unidentified Analyst

Or be on Thanks. to you Do invest lean you team? see need just the think any this Great. one launch. the do add-on? And then PFIC a further commercial into will

Chris Peetz

some describe maybe of launch to Peter the ask plans. I'll

Peter Radovich

Yes. straightforward. incremental It's a see need for We for don't OpEx PFIC.

prescribed probably care of are fact, a taking PFIC the subset LIVMARLI syndrome. by today prescribers Alagille who've patients for In the

Unidentified Analyst

in queue. back you. Thank I'll Great.

Chris Peetz

Thanks for the questions.

Operator

[Operator you. Instructions]. Thank

line. questions We further have no on the

Chris Peetz

day. to Great. Thank Hope you you, operator. everyone And have you a thank joining for today's Bye-bye. great call.

Operator

and Ladies this call. you. for today's concludes Thank joining. gentlemen, you Thank

disconnect You your may lines. now

Mirum Pharmaceuticals (MIRM) 6 May 232023 Q1 Earnings call transcript