MIRM - Earnings call 3 2023 | Mirum Pharmaceuticals, Inc.

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Operator

Good afternoon. I would like to welcome you all to the Mirum Pharmaceuticals Conference Call. My name is Brika and I'll be the event specialist running today's call with you.[Operator Instructions] I would now like to pass the call over to your host, Andrew McKibben, Vice President of Investor Relations to begin.

So Andrew, you may begin.

Andrew McKibben

Thanks, Brika, and good afternoon, everyone. you Pharmaceuticals' welcome Conference Quarter to Call. like to Third Mirum XXXX I'd by CEO, issued the Research XXXX. President Chief joined Financial results team our I'm today for our Bjerkholt, and release Vig our Peter Chief announcing our member company's Eric Pam today, Operating Radovich, of Chris news Head third Development, of Mirum quarter and Officer.Earlier and newest a Officer, our Peetz; the Investors section of be the Copies news found SEC release our can filings of this and website. in and Full the in duty details by expectations, we regulatory expectations.Mirum statements management's on risks could from can our and be remind course actual results business quarter regarding news anticipated like strategies, is including the will expectations, certain update differ and are today.Before current we the you materially uncertainties, making I'd statements. Mirum, updates Mirum's and our issued to of forward-looking to under they and programs these from call, about opportunities prospects, be current this that during programs, based plans, and subject financial release results development found and and these no begin, conference and future market to numerous XX-Q statements, statements Chris. said, the that Investors I'd XX, the year like XXXX in turn factors ended risk should SEC.With Mirum's to read call December with filed and forth set XX-K to over the for subsequent the reports Chris?

Christopher Peetz

patients to families everyone. on our The with focusing good and was business afternoon Mirum. grow for team diseases. of living life-changing Andrew, Thanks, to at medicines their delivering rare strong continued and This another quarter has execution by

realizing the We multiple strong adoption saw quarter. million of LIVMARLI. achieved $XX.X revenue goals total We of this continued global quarter, including have for

CHOLBAM. the completed high and CHENODAL acquisition medicines, commercial and also of impact We synergistic X

in patients. a across means of our our has improving X another The the forward from business to In key as we milestone this has study data Alagille the and milestones. testing medicines, our Phase sales critical on and our good while in clinical we and execution half first our of of been positive CFO, We team And execution I Eric revenue the franchise accessible with all this call positive community $XXX has the to significant for impact data approval and for families well. we've to also and am balance Mirum possible team's commitment million and interim xanthomatosis annual unmatched that free hepatology plan and moving I Bjerkholt, CTX CHOLBAM with Phase grew with upcoming turn dedication, continued product disease. to to this readout CTX submit U.S. half net the the launching for steady PSC used We performance conduct contribution to the to to commercial before in genetic our volixibat, a sheet.These Phase terms bile for of CTX all also company. and portfolio.Regarding Mirum March to success joined in CHENODAL panel, acid syndrome impact a and with in our for what around demand LIVMARLI acquisition syndrome much our short what diagnosing special of for here in data pipeline, in biliary And showed patients expanded to first cholestasis we thank to the we the a the want this regulatory of from for for the see RESTORE and our Alagille the of with the III weekend, welcome pediatric come FDA and important make internationally. next approximately evaluating coming of to recent accomplished September. This proud opportunities rare significant positive of forward Peter more the of progress patients significant disorders on our October, spirit year.Now, up Phase III expect our and and focused PDUFA cerebrotendinous analyses world.I of announced genetic look is XXXX.We milestones with assumed of accomplish been time the pipeline and a is alongside financial providers. upcoming patients and leading III to position potential delivering and measures LIVMARLI, X-year RESTORE I'd and application for of achievements the have in to like study, data growing making Mirum in support perseverance And anniversary growth PFIC we the date the set discuss able also quarter, brings have reflects several and patients sponsorship vital medicine over This CTX. step portfolio.This new our created that broadly to LIVMARLI and with made our the who Mirum commercial new business, LIVMARLI, to a CHENODAL come.Now IIB CHENODAL PBC XX CHENODAL and upcoming or drug for atresia.

the with call and Eric drive for the helping call.And that, to have his hear creation ahead later in to We'll him to extensive Peter. the thrilled from turn leadership and value growth see Peter? experience, Mirum. over are I'll We we

Peter Radovich

total that made Chris.With are U.S. businesses. $XX.X our the reaching Thanks, international and million progress have in we QX, across revenue excited by we tremendous

pruritus taken over represents bring third steps to our addition to leading of ongoing and to hepatology portfolio. significant execution we our excellent in commercial reflects see similar worldwide. Germany sales, saw markets. $XX.X of as quarter, as continues establishing acid total which This with Alagille LIVMARLI France a having bile the LIVMARLI, uptake strategy pediatric CHOLBAM.Starting execution the and distributor net and revenues of have life-changing with of more in $XX patients LIVMARLI the is through million franchise LIVMARLI our and U.S. product patients addition the the strength business, well team. we to the Internationally, in before And growth in this quarter, commercial grow. impact CHENODAL XXX% grew continues The patient team in million the Our with which in

significantly LIVMARLI's CHOLBAM, striking prescribers medicines the U.S. forward patient these for indications.Moving continue our of variability Going the excited we Alagille the international a to to growth looking significant in anticipate with the forward, we globally and enhance are pediatric relationships to expansion hepatology. are access between in similarities with our quarter-to-quarter to to to and and both and future revenue. LIVMARLI and for continued broaden Overall, patients opportunity There to and these syndrome presents opportunity in communities addition models both CHENODAL augment commercial medicines. are the

bottom our So we of terms expect top pediatric and strategy, in extremely line of synergies.In been one team, hepatology our commercial has team hands the effective.

treatment, and to usually be we and of investment To neurological step we liver, So our and to see gap later treatment we for increase over indications. to in for where such summary, This than key we as are and example, in contributor.Pediatric opportunity potential with this that approval a team LIVMARLI addition in continue testing a is of juvenile CHOLBAM CHOLBAM. is prepare journey.While are in new CTX as medicines this we and of we the last and opportunity disease we specialties expanded not damage more steadily journey cataracts the branded allow model the offering some diagnosis disease start which and partner has efforts earlier a as consequences plan genetic ophthalmology, deepen their well. patients CTX.In the by across may linked hepatologists thrilled and see in reach to have increased from us bilateral don't cholestasis And much diagnosis in launch have with that often CTX, patient our address to have the through after products irreversible the on awareness an and presents occurred. several manifests to working our achieved, over change until that expertise to to I'll a with adulthood, more such augment medical genetics. do Pam. panel, improve brought relationships neurology in first is team Mirum will grown disease and years, look important we the expanded But that CHENODAL as tool the call for among even prescribers positions call patients first the the In main Pam? rare diseases.On these and the we've our diagnosis the note, medicines high-impact other build momentum tremendous point of organs diagnostic important to turn delivering these liver will members and

Pamela Vig

study to quickly. measures. other critical that the study CHENODAL to and biomarker study team of the And effect cholestanol CHENODAL a disease cholestanol. safety study our patients we And data highlight significant CHENODAL are due III measurement evaluating thrilled evaluate a could accumulation was results objective early in month, certain the In in endpoints, this, it and of milestones, top with all believe treatment possible.Additionally, with market in key the filing, collaboration the as first bile the study, recognized well of to reporting measures, in patients.We're this the expect medical were the to this Phase the were Last and CTX. CTX. year.Moving CTX confirm including selection CHENODAL clinical by pleased including proportion CTX.As for of EMBARK rescue in in of have improvement an these be particular, CHENODAL Peter. looking had grateful not a cholestanol in life-changing primary development data Thanks, Prior placebo-controlled for as standard-of-care statistically setting. and NDA see year which IIB of to that execution with on is positive brain. we across urine FDA, which forward impact efficacy this of the the are tissue and was of all importance consistent showed trial levels a across reminder, impact diagnose and cholestanol to depth results Phase improvement of by treat CHENODAL CTX the demonstrating the the RESTORE with the pediatric which of atresia, finding. broad line a the The Travere into The very secondary with from therapy, the greater patients FDA.And from progression of with RESTORE announced we the alcohols and team patients to impact the and these this the results inhibitor CHENODAL the we immediate LIVMARLI for patients is IBAT the CTX while strong biliary in data to our later will first put we And positioned work as results, has demonstrating next and we're can of developed close required endpoints, receiving half are currently placebo our necessity

and analysis For PBC, to PSC we the first volixibat expect half in year. the on interim of report next in

recently, PFIC We body experience research we and of continue these position growing study and and our push remain effects Alagille sharing speaks at presented screening our pediatric we of Mirum's and the clinical proud ESPGHAN to I'm role important the continues Phase validating summary, real-world real-world for clinical meeting data interim committed across data.Lastly, syndrome syndrome, activities PFIC. including Alagille the tremendous to presence evidence patients The from to leadership the in March indications. looking on growing real-world build, across forward settings.In LIVMARLI to and hepatology in the with of of are to data and body the III

our in Eric? IBAT PSC, We now the the paradigm PBC.And can for patients believe call we settings also changing the with data a I'll look that, across biliary results. turn living to treatment to discuss Eric upcoming with role atresia, forward inhibition key play multiple over readouts where to and financial

Eric Bjerkholt

Thanks, Pam.

for included to time I I'd joined advance to I that mention I medicines diseases.Now quarter can pleased we rare for to release begin, today. the be Full look how quarter, at like details a the such today, the third the with patients new Before helping an Mirum forward results, team have earlier for am in results summarize. filed issued which for exciting XX-Q financial also briefly and I'll company, Form the financial press found

for LIVMARLI, the the sales as As This a highlighted of million out I'd million, we'll but $XX.X third date in SG&A $XX.X expenses net and $X total of for Peter, of by this of the million. like R&D year.Our of which see sales the quarter $XX.X includes recognized sales quarter, the million net only reflects $XX.X over August increase these the in were and of of Chris on the since million, cost closed product quarter sales and CHENODAL product for quarter. operating recognized product transaction products, of bile as XX% expenses full XX, fourth the CHOLBAM. $XX.X million, for previous quarterly acid we to medicines expenses of point we sales that well $XX.X million contribution

commercial quarter trials I'll for continued intangible global a end expense ended to loss over the cash, which or execute stock-based we're years.Now with we position XX, $XX.X $X.X of XXXX, Chris final on loss cash the base, $X.XX growing $X.X had of revenue back presence amortization the Net investments execution business our September million and non-cash us were including clinical third a and of for close to call the At compensation summary, our share. turn plan, For net of XX, the and equivalents, quarter included coming $XXX million. quarter of expansion of September million.In exceptional well-funded comments. our an places the in of over million,

Christopher Peetz

Eric. Thanks,

you on all in leading tell, leading standard-of-care with the medicines We've As to genetic franchise hepatology tool. X execute Mirum built testing fronts. pediatric can continued has and the

sales We approval data across of for and are growth all We've support filing III generated the CHENODAL Phase our CTX. medicines. strong seeing of important to

updated catalysts position. our to the for for that, putting strong call into we operator, strive world. patients make exciting of while please you on forward We clinical with as keeping in continued all this we've progress around regulatory accomplished have we advances Mirum as look ahead the rare open And head financial and a We XXXX.And several questions. to disease

Operator

from [Operator Schimmer have the Instructions] We first Josh question Cantor. of

Josh Schimmer

actually ask, wanted indicated on their launch Congrats to Ipsen syndrome. your been switching they from growth to LIVMARLI as curious you've patients whether what on seen lens the Alagille at had another to to call on I'm from for I LIVMARLI. curve you impede just seeing Bylvay you're exceptional of quarter. for because their strong and some expect that

Christopher Peetz

Peter the question, for to to talk competitive the Josh Thanks, I'll over it hand dynamic. about

Peter Radovich

was Yes. for X Thanks, was the first up think U.S. I there the quarter we'd Josh. product years really a is and revenue And QX. in now. And approved has the in strong what really is syndrome. you Alagille that that LIVMARLI been over in net QX indeed, where And marketed tape. point step headline The to another available we over saw QX

stronger what than to saw in In QX LIVMARLI QX we U.S. fact, little even of a from terms sales.

trajectory So strong I not impacted. think the story is there very that is LIVMARLI's and been it's

Josh Schimmer

on comment you how the seen rest split think you comment Can have have do between well? to of U.S. and might and Bylvay? any if attrition whether many patients on why you as of so, And switched? world the they you Can

Christopher Peetz

approval not the commented Peter bit on U.S. example, regulatory for Well, and dynamic. I Europe, a situation Internationally, an where think different we've the is syndrome. of in seen Alagille Bylvay

really strong there. grow around at this And It's point. a Europe are situation patients markets really and anchored to for we add continuing so international

Operator

of Gavin question Evercore comes Your ISI. from Clark-Gartner next

Gavin Clark-Gartner

quarter. the on Congrats

is For Kasai the in the relation procedure? the taken bilirubin what EMBARK value baseline readout, to

Pamela Vig

Yes, thanks question. for the

similar baseline see is that EMBARK the observed X.X has So XX. Pre-Kasai, XX, we to the about what's X. it been And we the papers, bilirubin close a study around shown literature to natural in post-Kasai, in few see to history. about in it's very which

the improvement in X treatment. room of of lot a leaves really that so And months for

this looking So to sharing later that year. forward data

Gavin Clark-Gartner

clarify, after to when X around just for the And value value? is the trial Kasai it's the baseline the

Pamela Vig

is. the after It's Kasai. It

Operator

Partners. now of Leerink have We Foroohar Mani

Mani Foroohar

How sort we Phase have that data margins FDA that guess consequence? through growth, cetera, the do to a you Chenodal promotional accelerate expand the past a -- you to of to and weren't about on having potentially II approval? to avenues available that et whether strong you pricing, opportunities, looking question And have that growth prior positive should on perhaps I when this how and think the think about avenues in around NDA we that of broader Congrats in and available accelerate hand. asset now approval quarter. data

Christopher Peetz

CHENODAL Yes, patient a potential from FDA increase a already. offered really as we in are are patients you diagnosis is thinking think, think thanks do a of CTX, all diagnosis CHENODAL KOLs point, XX% Yes, only It's and from have in are clinic, know rates necessity. diagnosed. Once here really anticipate that the we substantially we seen see diagnosed the But to XXXX. of approximately literature, I approval for We approval opportunity and the low. question, Mani. And rate. quite patients

can that you to difference the So do in increase thinking of lot the of to make have product. we've to spent Obviously, growth of do a a terms time XX%. what about don't lot a we

-- promote to disease find care. state diagnosis more a testing, team we medical neurology, essentially, try and awareness, patients we're and patients, bring a have So ophthalmology, to genetics, more under genetic efforts and around, into and

Mani Foroohar

approvability guess of Presuming us a And and obviously result there. an to a are Can to public a positive benefit I a rate engage through there's obviously and in be horizon steps would that. the be on indication? on investors what an or that folks see for what secondarily directional are setting what perhaps bilirubin, to update the the you on given transplant, the need perhaps the time biliary provide with walk and unmet filability power event you able atresia, lot pipeline, some be on regulators us then on would path that you to

Christopher Peetz

way outcomes biliary take supportive would data expect and from analysis to year. forward here it taking Thanks to you are really on the and We'd a plans trends that described, them study EMBARK the top-line we'll be the of on atresia strong update end be is forward to first for on that able meeting total, positive Mani. the what half timeline take have laying FDA. by And the year FDA the The that towards excited of question, input. kind to love the on a bilirubin that out of the data, we'd in then having outcome midyear, sometime be in our publicly based

Mani Foroohar

reasonable we'd early of it like might it year, half like, the around, assumptions narrow kind Is something first those guys might to be? that timeline in is just of you too make that next to hear that from or be how or assume

Christopher Peetz

interactions of FDA. types We -- scheduling with dependent on these are

So as to we'd meeting to with be clarity. we provide able get FDA, closer some through getting a

guidance the update best having we next have now, an For midyear is by year.

Operator

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

Steven Seedhouse

I wanted EMBARK ask first. about to

Just have you of to based by analyze do top-line parallel, guys the clinical events and you of the registry on sense, And the before of the be you've work the able in doing you'd a of PSC and time demographic there will just that part ask I you available also, data. be Second, are top-line on analysis enrolled, the Maybe I number guess, that, of would be the PBC terms about year-end? and talk widened well? -- timing those just some but wanted of It's, interns. as delayed. data. the expectation inputs to in about you'd the just Can the refute when of

Pamela Vig

can the Yes, I answer thanks question. for these.

compliance, end Natural at you able patients the you events to And information towards to data, bilirubin discussion working to for population aligning we've So to to a Registry, understand, a levels hopefully, help call. and particular population, these FDA.With screen events the survival. patient registry, regard pulling we're will of pruritus the from so regard and share first blinded really complete with patients History And question will to information that in coming failures really with progressive supportive daily. in to EMBARK with you for score that what bilirubin, PSC these year.With of and alluded is looking seen the part with come the our lot our on so as our patients to. really bolster very and be your closely with is as PBC, this transplant-free more registry of are prognostic as have we this what the from last are are top-line earnings we're yes, e-diary the regard we'll some coming seeing But highly can disease. expect, in, since A

screening minimize a to But closely really working been couple screened a year. to actively failed with remain we labs, liver excited So patients sites that. for There's have we're then data and labs, scores. share liver for screening that qualifying and next few pruritus

Operator

We now Lebowitz Citi. David have of

David Lebowitz

for Considering the ALGS, orphan not believe EMA you reaffirmed do to disease LIVMARLI or does through lead Bylvay its to not decision PFIC? through relative give that the leads to recently status to how

Christopher Peetz

a optimistic our what by I orphan all should from syndrome therapies. key the we we of back that the And included a data about some Yes, was to Thanks are briefing the materials interesting profile It's background really from of little to have on watching that of just available on for submissions the significant end have David. rate. to on the And this is point. the response waiting genetic Those a for think year. the stronger is Alagille younger and maintain study situation this, outcome understanding question, recent for broader it's set factor on a We're recent this the feedback starts some benefit And update unfold. age. over agency bit we're the final our at of That's an a EMA. that things provides It with in PFIC decision. patients. March it's the in of feedback hear EMA designation

Operator

have of now We Skorney Brian Baird.

Brian Skorney

mg question bit also what My EMBARK. change sort on below characterize in that versus of you bilirubin, endpoint patients us of X how a just the idea primary, Can means little secondary of give the getting an is the terms you that infants that X just placebo in had arm ChiLDReN think for of expectations the secondary time, XX% X. than Research less Network about bilirubin at on the I post-Kasai endpoint, per Disease months said deciliter? of And in Liver of

the Is more less? consider for make in that the or there that assumption that endpoint anything placebo that design So in or reasonable EMBARK? is rate might a to for

Pamela Vig

question. the thanks for Yes,

So I'll last your with question start first. maybe

that somewhere X.X natural shows patients in and those the history, post-Kasai, less So XX of from the literature half jaundice. clear between to than that will

will remain pretty good blood are to well. alluded of well less But per established X majority majority flow, deciliter, really them the between varying than elevated really you of those to, those X patients that and milligrams And than the degrees. do have So as do greater X. and they'll patients

so and be we if from between greater looking to risk, moderate maybe we'll X, than from moderate X And also shift those and at high patients risk meaning X, risk risk. to low

looking different proportion, be percent in I to at our think question? we'll question, using X first it. another Was primary at there So bilirubin looking your all at that and answer ways. change active NMR endpoint, an And between analysis. is And was the placebo the month difference

Operator

now Wainwright. Arce H.C. We of have Ed

Ed Arce

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

data So actually. first, set last this earlier positive congratulations on month, RESTORE the month, earlier

to So data what can RESTORE the NDA In first the will you or data, addition talk half anticipate of be data? about you a be next little filing, bit that of usage post-marketing data package? do about CTX CHENODAL, year? Would part historical in and

Christopher Peetz

the for Thomas, we straightforward they're in Phase study as of NDA thanks forth Yes, There's the on and been approach NDA RESTORE what the expecting relatively discussion with the back for see here the FDA question. extensive CHENODAL. design. III for

see And NDA that additional the of comfortable work alignment analyses typical would include did this NDA kind that will of CTX and something was you'd indication. data that got for there a that with for that good FDA, battery So on support the small application. molecule was the we an

unique NDA particularly about nothing overall. So the

Ed Arce

question perhaps one then us. from And just more

market for For what's anticipated LIVMARLI growth syndrome year, and the to in the Alagille get as XXXX? level your we the for U.S. Alagille end syndrome close of

Eric Bjerkholt

XXXX we're at when. and internally discussing we We have if will guidance still point, and what so, not and whether provided on any this

So stay tuned on that.

Operator

We John have line Securities. our of JMP from final Wolleben question now the on

Jonathan Wolleben

for potential and I'm those give if And a Just will CHOLBAM, down impact we should could and products how the about with that early sales? think the you bit kind the of think little testing. LIVMARLI wondering CTX on leverage you also about, What then talk experience genetic road? approval you do on then CHENODAL its and

Christopher Peetz

cholestasis It's products well team Yes, is script, thanks Yes, CHOLBAM and our bring focused the of Jon. to promoting kind to genetic and pediatric really hepatologists, exciting now value having on to genetic testing, the healthcare in of team of a certainly bring access The that the kind customers. proposition to into I enhances front professionals which really our kind question, some of the having ability improve the with their dynamics testing Mirum. on of can prescribers customers. the these as for the be leadership Mirum reasons and role Liver other as been and and both in LIVMARLI commented to

So about and profile really for supporting Mirum's what is doing excited this growth. LIVMARLI's

growth. XXXX, then, think continue that's as increase can them into we trying we diagnosis impact. we So we're mid-single-digit more make excited a the We CTX to have to there, when I earlier forward the products can to yes, move investments one point.And we find the larger and find rate XXXX, in think expect bile acid about to low deliver patients, -- probably

Operator

[Operator we further have no Instructions] questions. I can confirm

for final any remarks. like back Chris to to the So call over I'd turn

Christopher Peetz

Great. call. for today's joining Thank you

a quarter close ever been of it Mirum, to as revenue. has strong a out note Just the here, quarter biggest for

medicines in regulatory Additional portfolio, and and Have evening. catalysts the balance multiple clinical a great ahead, strong Goodbye. sheet.

Operator

lovely all may I your does that joining. have a of disconnect lines. today's now rest conclude call. your can you and day, Thank Please confirm for you

Mirum Pharmaceuticals (MIRM) 2 Nov 232023 Q3 Earnings call transcript