ALBO - Earnings call transcript Q1 2019

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Operator

Good morning, ladies and gentlemen, and thank you for joining us for Albireo’s Conference Call and Business Update for the First Quarter 2019.

Following management's prepared remarks, we'll open the call for question. I would now like to turn the call over to Paul Arndt from LifeSci Advisors, representing Investor Relations for the Company. Please go ahead sir.

Paul Arndt

Thank you, operator, and good morning, everyone. today's joining for you call. Thank

recent will During press www.albireopharma.com. the performance issued first update noting release highlighting Albireo's quarter at ended XXXX. accessible an the which a financial first This for release Albireo is management website in today, quarter on Earlier via and Company's XX, provide of results accomplishments business March press its XXXX. the

made Reform today the Before of may forward-looking that proceeding, let under Litigation me statements Private comments Securities Act mention XXXX. made include

conduct, cash reimbursement. patients indication, Forward-looking trial will reliance any its revenues, statements expenses. product not approval, including most regarding Albireo's than Phase forward opportunity Partners duration filings looking of including the Including planned SEC. in expectations statement of under for for heading the any or a in Albireo any obligation be by May of or statements in on financial and forward-looking heading plans by operation, and recent in in outlook of in or from made XXXX, of trial, future target program. payment of coverage, that, Albireo's any Also, Any for results differ President those objective, pricing result or sufficient as elobixibat applicable fund that be later EA may program the data, duration, target cautions of implied approval cost, resources for potential period make odevixibat or should is Albireo's or which Thursday, representing pivotal or expressed The as required for or Form today, Pharma today disclaims The with Odevixibat, costs submission any feedback NASH. III indications I'll endpoints, requirements commercial any Albireo's prevalence trial completion date. may speaks for results additional to statement PFIC trial the of looking planned to or statements scope, factors or other upon the over of future results Albireo as any call other any include II any the atresia, not Odevixibat, biliary undue operating candidate or odevixibat any from Royalty candidate materially update in release with Healthcare Ron? law. as forward-looking to to historical timing any With Albireo Albireo. elobixibat, AXXXX except location, X, you risk many initiation its for from only statement views for or reporting Cooper, forward regulatory Albireo's turn Phase or as Ron under clinical fact or earlier or or forward-looking regarding place product Actual to development progress, development, size, plans statements. of including factors XX-K Albireo's statements CEO. described the those press Albireo that population, position, of timing design, relied

Ronald Cooper

with progress benefit conference advancing the focused NASH the in Number Chief our on on me and for Financial in the today our our XXXX pleased a as everybody into first its year. With on fronts. trial one, Dr. first we're moving odevixibat be progressive three, and people Thank clinical Number diseases. to the phase our pediatric step. for our And elobixibat cholestasis liver today's PFIC. cholestatic Medical today to potential and you, program indication launch following: patients with quarter toward to Paul, us multiple approval into Simon Officer. outlook year all thank Albireo's on or you with the Horn, across pleased you with This the here a intrahepatic update odevixibat progress Number We're medicine during two, of joining Harford, and Patrick is are developing Officer; Chief call. cholestasis potential first three we

So odevixibat to PFIC, towards move on beginning continue delivering our commitments potential commercialization. we're to in with

longer You name may candidate information the about to notice, AXXXX. PFIC. the product I'm has WHO When recommended AXXXX. no been in referring published in investigational our talk I Odevixibat now as international drug for

progress to we program. make So the PEDFIC continue in

PEDFIC efforts to timing topline few could expected is liver when the quarter, make additional randomized the and Our double by response. clinical we odevixibat Type with X. designed the pediatric When patients We enrolled. our are label results opening and activities continues approximately odevixibat program Odevixibat an the early extension of We guidance commitment PFIC to data patients enroll designed late build X. expand wide is odevixibat. of we a the will of with we believe the body X first patients by the X, types. approval rare as of PEDFIC XX potential controlled as with anticipate announcement in of completion highly results benefit XXXX. multi-center data enrolled, additional And of PFIC. topline patient of a X, following program clinical durability and in plan to PEDFIC site May is with refine launch the a the Phase or to the our and disease, XX trial PFIC, studying franchise patients trial safety first to last blind to to range PFIC XXXX We ultra X X support trial generating program PEDFIC III an half enroll have fully In XXXX previously consist PEDFIC open study further. or placebo with upon PEDFIC long-term Type to single assess the stated of began to an dependent

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We our remains address cohort highest patients. front, clinical expect been our need a expanded program continue unmet range building will potential foundation. The across PEDFIC on strengthen odevixibat and of as to the inside priority wide our deliver this it to we we've commercial

buildup that data. been rate approach have advance and activities early limiting pivotal be Our has our we accelerate then to on when could

hold biliary Gap] from atresia. We the which the and the this regulatory trial year. half U.S. pivotal feedback FDA drug odevixibat orphan in designation strong Grants We EU in have in second have for Encouraging a our agencies in approach the plan to designation initiate [Audio atresia. biliary odevixibat now of orphan both informed the we in structuring the

more provide on trial will We design the when details finalized. it's

patients conferences. Association We Phase April major held for in Liver of bile the the from that shows Vienna, the continue with serum these II pediatric of potential cholestatic patients our to wide Well atresia in in treat to in the honored best in European syndrome cholestasis the biliary of trial further in study, Liver acids, odevixibat presentation odevixibat present reinforce We odevixibat. based Study Annual a liver for Phase we treated to small selected important scientific Conference. with number At inclusion were at odevixibat our in reductions range International alagille diseases. in II data on belief patients Conference alagille syndromes data in of results a presented in of on have our

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liver drugs X serious, estimate the of Bile fatal Finally, a our bile occurs but disease, people in will markers European alcohol. X.X% We treatment chronic patients. has people drink a States that to Elevated to rationale we of strong that no X% in modulation sometimes effect and NASH. XX NASH who adult is no we levels resembles of currently the create NASH also in one major NASH disease momentum, in for are which believe acids representing little alcoholic continues over build in approved opportunities. NASH United NASH. program million Union. the acid are and There partnering patients common and or million

is insulin with that GLP-X, markers in is and Additionally, to we safety fibrotic approve constipation, to has sensitivity increased levels constipation patients inhibitor, profiles treat a elobixibat markers. preclinical demonstrating reduction data clinical chronic a safety standpoint, chronic which in From in of improved and X,XXX with dyslipidemia a of Japan it have exposures. pro-inflammatory IBAT more database or and improve going it and elobixibat cholesterol than data which in approved showing

clearance national landscape believe mechanism, to begin Syndrome a we Phase on NASH are definitions. our of elobixibat IND. in patients or biopsy-confirmed has We've receive or NASH multi-center that to study expected for in a diagnosis as preclinical to potentially the FDA We monotherapy this report NASH based NAFLD combination place controlled novel that emerging the and to XX this to Metabolic the placebo pleased enroll plan or with elobixibat potential and we or find II compound quarter trial with therapy. significant designed the Given

per makes on with XXX or XXX once We than deciliter, compare lipid to liver or if medication. LDLC. greater lowering than per The from five-milligram weeks deciliter study they're The a on will than with equal greater already placebo. fat enroll elobixibat will daily patients in primary endpoint versus LDL XX will over greater be makes And XX% baseline MRI. changed a dosage

liver IBAT on Secondary we study our will is Exploratory with provide the fat serum will elobixibat as NASH. designed endpoints homeostasis, consistent bile guidance studies the levels. in measures inflammation glucose insulin acid ALT, include include the AST NASH on of and recent The by biomarkers FDA endpoints impact on for insights of valuable from really inhibitor imaging and fibrosis.

cumulative versus is our parameters continue believe disease one a significant impact showing metabolic We a approach fundamentally to across multiple and based a is impact NASH on measure. on

data We topline expect mid-XXXX.

disease launch & was rare last and and she for led roles to of financial an at Sales Value, at products. at Pfizer through operations around Pamela significant successful outstanding commercial and Vertex, I her welcome update, spent roles senior CCO. marketed period turning held was Pamela opportunity Global drove and as having strategy commercial she therapy. Vertex She the want to Patient pedigree a another leadership at Vertex Access where Before Services to role take and decade pipeline of Simon to the new our decade a brings growth. for In in over has Marketing, in of Stephenson, responsible a Albireo Head a was

delighted given driver odevixibat building successfully. to and access, Now on company be and drug launch launch Albireo, to attracted a increasing preparing Pamela's experience she the should Pamela will recent key scrutiny to us be the invaluable. We're in orphan have as pricing absolutely

So turn the Simon a to over Simon? financial to call now pleasure it's update. my for

Simon Harford

Ron. you, Thank

XX, compared results financial at our million we today. balance to ended highlight quickly XXXX equivalents me XX, XXXX. first million Let $XXX.X quarter quarter cash closed the which We earlier first in released with cash for the of December March and a $XXX.X

cash Our PEDFIC as reduced expenses. Phase our III the related related a well as balance costs result by X $XX.X quarter primarily people trial, as during to million of

are the ended year. revenue of ongoing QX March was XXX,XXX year first to in same Pharma to to sales the compared EA elobixibat period a which the royalty last partner HCR. constipation in related revenue period from $XX.X for from XX, our of for partner we Our Japan chronic Pharma quarter XXXX passed ultimately was the to EA approval the elobixibat by royalties on during Last same related payment million one-off milestone this for Revenue received Japanese MHLW. year in our

odevixibat, NASH to well development related preclinical first driven first XXXX the XXXX. were as headcount quarter of to million in our quarter to programs million expenses were primarily elobixibat, by and for activities programs. compared the research and spend to QX Our as expenses additions in related R&D support $X.X $X.X

expenses to $X.X let attributable G&A loss XXXX of was $X.XX loss million expenses support Based were G&A in what $XX anticipate current $X.XX additions prior to XXXX. in back to the the to $X.X be increase to a G&A headcount period. to for compared per of Our of net for million quarter sufficient million quarter our on R&D the cash Company's that, The Net March compared closing with million first of plans, range share over a operating strategy. first the we turn to XX, the $X.X first was including to million XXXX. or needs year share for operating operating of to XXXX expenses or $XX.X in previously. balance per XXXX in our for principally loss consistent current ended quarter loss the $XX our have a be of we million into our with meet We total me anticipate the And remarks. said expenses and call Ron

Ronald Cooper

Great. Thank Simon. you,

odevixibat Albireo So completion to we're as summarize, to Phase closer and this is topline getting an exciting time data. for III

and accelerating greater multiple We're accelerating programs always develop to plan diseases pediatric II for realize in of potential Our in PFIC preclinical our bile beginning multiple has excited begin the types atresia. with liver and acid into cholestatic our help Nash product starting a to we're one diseases and largest biliary also compounds. platform our even Phase to modulation trial liver been study of leading the elobixibat, a odevixibat generating data additional pediatric

as organization to we prepare grow commercialization. continues Our for hires key with

So with Operator? open call we'll up the that, questions. to

Operator

Instructions] of Cowen. Thank be line you. first We’ll now conducting the a question-and-answer from your question Baral with Please with [Operator Our Ritu session. proceed comes question.

Ritu Baral

Good question very you getting strategy thanks wanted queue. help for I about briefly commercialization Thanks for the taking the ask to the morning, and guys. touched Ron the that on. into

Forgive if misheard. me I

a pediatric you Are number then of certain excellence? population? herpetologists are that you that prescribing I targeting are the that How like bit think how about said NASH little gotten the mapping may centers in I XXX find trial. a physician have those wrong. on you number follow-up Can were have And them. you you they herpetologist a elaborate do and of

Ronald Cooper

much morning, question. Good the Ritu for Great. and very thanks

prepared good the have I that note as Stephenson think right part in a sure Commercial Pamela Chief pretty noted limiting is our as strategy. sure delighted comments the of We're us is I that thing trying about to we're have is making first Officer. team, being is and we my taken we're launch to anything of our that care and joined commercialization to serious that make rate to

in think targets, when them about herpetologists actual the are of XXX in about perspective from NFL geographic right. and number. cities, sort we think an really is to about And Europe there there Now way equivalent a pediatric estimate we U.S. the the

medical So they are well. tertiary the in Europe in centers as in U.S. and

a So that could we're with XX relatively field we we believe XX force, deployed individuals to access between estimating small somewhere field these individuals.

Ritu Baral

there there and are thoughts do And those after it. ROI identified, targeting like tertiary centers tier XXX? how in second understand. you many that to a there is Initial Got have is those

Ronald Cooper

think targets. here think you have call you that we those our When for have sites we then really almost would tertiary about X B our A Of course and PEDFIC the study. of targets to have, we the centers

we that that, the hematology as similar group of sites well. PEDFIC sites in And much We X for gastro, Europe our that estimate to X,XXX covers the think study, somewhere XX level right. announced estimate are between as there's we've a I probably mix of But be pretty we've activated that So something targets X,XXX that well. we achieved pediatric, them. of will target and targets, there majority some B because then B in would they're a individuals

Ritu Baral

And versus defining your NASH a desired helpful. And the get diagnosis patients any you in of order confirmed between – you what you patients biopsy then clinical is NASH give are in by to a enough III? study, NAFLD need robust design to enrollment NAFLD. those your there Very you how Phase signal breakdown of team to

Patrick Horn

Yes.

is have of we're Pat. FDA our in Again, on and early NASH at guidance this so looking the the prove-of-concept. latest we So design based the study

in So and primary we metabolic an amount the endpoint NASH. that to just of elevated patients NASH. will are an biopsy and and criteria. characteristics also find than to metabolic we that and themselves said is criteria its be and glucose that have treat proof-of-concept based expected that enhance hypoglycemia we traditional We required the NAFLD rather and/or at should to our enrollment. NASH. hepatic In there anticipate meeting this meeting a biopsy confirmed a cholesterol defined looking NASH a I long having specified that addition is or of fat distribution say is NASH, would don't as could set study, NAFLD is on tolerance diagnosis efficacy for or glucose decreased they requiring have NAFLD disease NAFLD LDL not with have the majority the

Ritu Baral

drugs the move just other study? around class. in mechanisticly on expectations What Last in based question the LDL in are your this

Patrick Horn

seen we previously chronic in the looked a and consistently see lowering that's here. some with what cholesterol, in in constipation LDL We we volunteers. and even odevixibat So at we even patients have and hyperlipidemia have in and anticipate healthy

Ritu Baral

Thanks for all questions. it. taking Got the

Ronald Cooper

welcome. are you So

at about NASH, LDL, significant liver effect I to think including multiple parameters excited think and liver we're elobixibat efficacy, we an but an that parameter on way like have including in IBAT looking things. GLP-X, effect, inhibitor on one effect the the about we're not potential other inflammation the for fibrosis,

for you thank So your We appreciate it. questions. do

Operator

you. Thank

comes next Our Liana from with the with proceed of Moussatos question. Securities. question your line Wedbush Please

Liana Moussatos

with you for of do taking to perhaps my X. PEDFIC Thank the questions. It's design

followed to going PEDFIC second X to and You're be and X – and exclude from is types cohort are treated X? ages like in have [PX] how overall X X has going certain PEDFIC patients in rollover PEDFIC terms profile type going work. PEDFIC to targeting the have mix a of you're that in And like patients PEDFIC and PEDFIC of you or it that in of the to going then PFIC additional for you then they X how in are do

Ronald Cooper

much Liana. Yes. very for Good Thanks morning, question. the

I'll kind with over me of comments, few a question. through of some Let Pat details to hand the your start go to it then

we'll types speak feedback received data. Pat, variety a a overall we're excited question. patients from disease cohort franchise. across second and Liana's build continue can more the potential generate generate investigators pretty belief to multiple that our to data I patient trying to And fits of key of that specifically and little made this cohort enroll about about commitments pediatric with liver before are our and excited to the pretty really leaders opinion We start starting think and second you based of maybe we've the on

Patrick Horn

Yes.

is So X PEDFIC the six if X. two at PEDFIC X PEDFIC PEDFIC studies, you and exactly of X like six and X look months month the PEDFIC But X, XX months. are PEDFIC is initial

that enroll will that X enrolled in they PEDFIC in directly with patients X. the they who all procedures have have in So fact had PEDFIC would of

is answer that. that So

make collect we be information placebo be will some to we and similar be X. will even something that comparisons studies draw across although PEDFIC to in separate – able a the is to able So to able group

to to and – design study a the the lot us separated people actually be they'll going are So rich allows really analysis gather from the up cohort. We be include. who by data will opened of still

not PEDFIC even a different predominately disease randomization will that maybe available. PEDFIC in of desire is include Patients who odevixibat into analysis on it is X X some are PEDFIC And patients causes we X So PEDFIC? to eligible, to in X from for original directly to PEDFIC even X. do good and of types of in the lot based into of enroll rarer the be range the to is the still going occasion information. X but the that we are eligible X closes. kind X PEDFIC PEDFIC on additional will is a going and really us hope So the the then in – but what after the we're of will roll same into more PEDFIC understand, for the investigators the of go who Cohort was while X to are Cohort the PEDFIC, can lot is They'll open, on use include potential depend our who in PEDFIC really enroll

be want and enroll those of the set cap Some going see have on who patients. range into of Cohort go number is don't as wait and going we're We to the is. enroll eligible X. to kind might to to We – we a patients see of what

Ronald Cooper

a also our that, patients qualify wouldn't other pretty that PFIC, patients, PFIC range we're studies. they circumstance original patients, of recall but other you studies think So, PEDFIC types and ages and excited that PEDFIC other X of other wide one for two they X, there's Liana those

generate to excited we're in data patients. So these some

Liana Moussatos

much. Very very you Thank helpful.

Operator

Thank you.

Please ROTH. next Rahimi Our comes question your Yasmeen from proceed question. of with with the line

Yasmeen Rahimi

on the us three congrats trial to insurers start on team. your remind in what off progress. questions. elements you minimize again we Maybe can Can provided? PFIC placebo team design have of continued reported I Hi,

Patrick Horn

Yes. This is Pat.

of subjective measurements is provide you of So all most there know placebo response. in with possibility us the the

are couple that of I we a things six-month here have think there study. in

typically realize you to a in And the maybe it at longer and goes. it's reported the early or in private you a their up. placebo actually see they tends patient And go that. if the score better. when studies you So see response course They work look previously

So terms that's of private. in one the things big of

think we consideration, I the study. is took in large we a - when placebo took really we response thing the other did that calculations large into for placebo response power the

even assumed we relatively we our power active So and relatively expected large we calculation. a than response did in actually placebo a response when smaller drug the

Ronald Cooper

these elements. I two key that with think are I and Pat think patients. has variability has we indicated, that been We as understand there's those

is a that rates. these time we've are tested powering in parent well, in assumptions have length patients, as of over XX%. these we have stated has You and And which a tool been

us with help hopeful as will patients. to well that differentiate we're so tool And

Yasmeen Rahimi

you Thank team.

of noticed filing give For in I moving color or on barring against for this million? and complaint the little to you Can second maybe damages an had on bit compensatory litigation a that a the you us international update? €XX more question. filed

Ronald Cooper

the Yasmeen. for question, Thanks Yes.

think, we've and within I expect comment said this just I enrolled what queue.

a complaint but would license the had we So the a refer queue, have of ask under claims €XX with to I arising filed million. with agreement barring number you to we

a information. with in potential update XX reward. And for at contingency any on more are fees third we'll legal you Our

Yasmeen Rahimi

and least question, regards you a tell on the more this recent inclination Ron. extent program FDA you come and versus us tell a to to Thank way the to or it's guidance last EMA you update could what can different between the maybe where the maybe a and then can? tiny little it's that that FDA the in in discussions is what had with And EMA be is this at regards you, the the biliary up on us atresia, headed bit different and similar

Simon Harford

Yes.

the So this thing. is interesting

been guidance. are rare in no they haven't registration diseases there really studies where So these

So of own agencies on They're with parallel through having each working their but are is and the agencies. this we both separate talks. talks

the we registration that the agencies to consider as the so key agencies would trying is really And endpoints. and to discussion and place different what expect same are come endpoints hinging get acceptable on the to you

having will with we're consider the and the addition discussions acceptable endpoint. KOLs ongoing In what clinicians

able place So down, them we're same narrowing same actually its the get be Whether towards an was to getting kind of the it we'll to place. unknown. we’re focusing

PFIC There the ongoing For have has And can't FDA, entirely final similar endpoint up we could the just we example situation. program, until the a in on really of EMA comment and different a in end unknown. possible is the it's in we kind discussions some decision.

Ronald Cooper

the is require say liver high. within life. decades cause for transplant unmet of first to atresia data regulatory in for their transplants pediatric a of them those need the significant, from patients couple a the XX% chrome enthusiasm suggest pretty in biliary perspective, Surprise population. process is The is number of one of the

able agents. [indiscernible] make having these to with So patients and we hope be discussions our that to difference we're with a

Yasmeen Rahimi

taking for you my Thank question.

Ronald Cooper

Yes. Yasmeen.

Operator

Thank you.

line Jefferies. the of question. Please Our next question comes Yang from proceed your with Kyung with

Kyung Yang

Thank you.

study the patients for rolling PEDFIC there? for are extension label are doses being from over what the randomized X in So phase, open who utilized the

Ronald Cooper

from that open dose, PEDFIC enter X the patients X with open kilogram who label to higher per label the micrograms all who So treated XXX and extension it's PEDFIC in per be X patients will extension PEDFIC roll PEDFIC the fact day. X the

Kyung Yang

now? to you transition label many the been I by on how are extension has open X patients PEDFIC see. Can from comment

Ronald Cooper

our we're about status we So or of ongoing at. the where talked haven't studies

Kyung Yang

Okay, all right.

Patrick Horn

[indiscernible] do so have working and that We and the trial is running up more.

Kyung Yang

talk study, nature expect the of the assumptions the also powering about you can placebo history to on be? X, what are? PEDFIC rate do then And Okay. based you And what

Patrick Horn

Okay.

are those So questions. of separate kind

intervention had no natural have these the are therapeutic no So really pretty patients from PEDFIC it's data. history just data Because history. there all who really the placebo with natural is much

therapeutic the cholestatic that intervention we diseases other have at a if be placebo information would of in similar studies. response placebo had So comes that look many you haven't sense you on one. some been PEDFIC, liver from really what have studies Again of the but pediatric give the can there

that's assumptions we of Like response. our we on our placebo the powering our of in the kind of response. size information So the where larger power assumed did I got said size the placebo we calculation sample when magnitude our

of terms In powering the assumptions.

endpoints recall you in different regions. primary the are there two As

So U.S. it pruritus is and acids. the in bile the EU its

power higher. serum for calculations the that Then pruritus that of power power a So the much bile and have we we said when as we acid endpoint means above did calculations for actually XX%. is the

Ronald Cooper

what add just Pat an said interesting That have our associated or Meeting, XXX if and data reach bile below Kyung, more Liver XX% recall only of have to and you reduce that where they the greater. of you X had reduction XX%. positive a a and/or acid to level outcomes to the natural presented favorable PEDFIC acids to XX% reduction and might the in a you responder for of is or endpoint was said history. the with that demonstrated consortium definition were outcomes bile share that study data at able from data So We and to European acid they your from recent NAPPED bile the –

Kyung Yang

much. very Thanks

Ronald Cooper

Kyung. you, Thank

Operator

you. Thank

Please proceed Lugo question. comes question the next William from line of with Blair. Our with your Tim

Timothy Lugo

cohort the leaves the question. the data. Kyung even PEDFIC X patients happy additional you're at touched upon blinded neonate. safety tea the you're dose those far are X question, with in taking that you a but bit And so in And a for high for Thanks PEDFIC maybe seeing suggests the the reading all

Patrick Horn

really we in and and will enroll – it. get that know who patients blinded about talked dose. And PEDFIC the all the is X other we really study and don't much So ongoing about higher

Ronald Cooper

right. per for kilogram a this I is remember dose neonates, the think

is XXX the weight adjusted whatever So is. for the

Timothy Lugo

Understood stand disease, sites? you in X there obviously you or about it's today, could the and talk one or sites XX PEDFIC XX two kind rare fairly conduct kind open. seeing you out site Are a generally enrollment of XX of general site around and in conduct that's have sites those each of are broadly those

Ronald Cooper

PFIC thing The consanguinity on. you some of of diseases cholestatic diseases aren't one the about versus other right, other

consanguinity. are are the So result there's geographies as patients, of where there a clusters there more some practice where

are lower there bit expect others of across is a would so amount, uneven sites. sites, the the group larger you of as a patients some whereas it's a it So

Timothy Lugo

Okay expanded you a that talk broad decision a be rare in about the general relatively for broad can a for on elobixibat and more think you your and little support things that of breadth diseases could will like labels label disease tend PEDFIC-X just is the for focused subtypes. in cohort they label be the liver a to whereas

Ronald Cooper

that data is say the much in to have once X we'll different the that and I then this we agency as PEDFIC possible to formats what at our results of generate think with have Hard share data discussions job them. stage, as multiple and with we'll

Timothy Lugo

you Thank the Understood. for question.

Ronald Cooper

Tim Thanks,

Operator

Thank you.

of Matt Thalmann. proceed your with question from Ladenburg Our Kaplan the next Please line comes question. with

Matthew Kaplan

the with bit long-term taking to zero are wanted for guess to Hi. a Good for where I respect manufacturing NDA to the necessary tox I and in then on you requirements questions. for respect filing. guess Thanks are package on guys. that a and the then morning I for preparations data with NDA safety little question your I preclinical PEDFIC-X guess elobixibat and

Ronald Cooper

second don’t why and the first you take Pat. with start one, question, then So I'll the

Patrick Horn

Yes. Okay.

we an FDA. have said results again studies ongoing be PEDFIC They in positive that itself have So X study with by sufficient would to from the application. our discussion the two support

to data that, but it's the a really requirement chance long-term and application. not open So do a initial the is get for label extension

Ronald Cooper

filing. terms in our the be of you the X to then study leading should PEDFIC as And is readiness it factor

news. good that's So

on already some means talks schedule And study that going. from dialogue – that of last plan forward. to it's The the drugs odevixibat on going agreed elobixibat that long and the of used late needed able a scale. beauty elobixibat are with perspective So and commercial we've a chain a the supply CMC go are been doc commercial same are that we they had and and we've to year all quality long-term FDA up for for the for

get So to our ability we're pretty confident there.

looking good the X the end forward that data should at be I thing it that is early like this to beginning, we're year having So way XXXX. the at and and its data PEDFIC gating factor said the of very

Matthew Kaplan

study terms Can a in you primary in criteria for Great. NAFLD the you're That's II what chance NASH terms give targeting, odevixibat sense of endpointing for then Phase LDL. for And and in us in entry of helpful. LDL?

Ronald Cooper

– can't on clinical trials tell I to go haven't off it's we into made, should my you the I I that on think remember be I but criteria there. its think So truth, head, don't And the top XXX? of believe

Patrick Horn

XXX if believe they XXX. have its IBAT then – with and lowering I a XXX, Yes. agent [indiscernible]

Matthew Kaplan

the questions. taking for Thanks Okay.

Patrick Horn

Matt. Thank you,

Operator

Thank you.

Alan proceed Needham of question. your question from Carr with Company. the line and Please Our with comes next

Joseph Stringer

the target Alan. What's is our Hi, for for thanks on X? Joe PEDFIC This for the sites on guys. progress questions. of and number taking Congrats

Ronald Cooper

think We've guided was – I to from Joe guidance our XX. XX

I we've site think finalize. we're that to that sites looking So and there's reached a or achieved still two having XX

Joseph Stringer

Okay. will PEDFIC symptomatic serum or patients based patients that? those the it clarify inclusion just levels into of acid they like X, to sort for be And terms brick just patients would any genotypes have be the be is you protocol amendment of – Thanks. to or bile PFIC on anything for to in And including to that.

Ronald Cooper

So study in nature and do demonstrate the PEDFIC, includes patients be study. type won't of have this makes have And their study that PFIC to because difficult their that it it and genotypes the all we be have in but to design. episodic the disease some of of types of symptoms that's BRIC to included current patients and they very

Joseph Stringer

Thanks. the atresia trial. see the overlap X could terms Is docs PEDFIC do of side some you PFIC and of see. in types a the biliary atresia potentially in question large biliary terms and of in Last both hepatologists and of there overlap X patients and Okay. and pediatric patients see that – they

Ronald Cooper

franchise, predominantly in Thanks of and as atresia, by I liver Joe. odevixibat by build are beauties Alagille diseases the position a treated to think treated is diseases hepatologist. one PFIC pediatric a other same and the biliary same desire pediatric Yes and the position that syndrome, the that's such that's as PSC

as question across tremendous your engage indications for believe them synergy we the Joe. have we different So our we call and on company. Thanks with physicians these

Operator

Thank you.

line of comes next Please from H.C. Our your question. Arce Ed Wainwright. with with proceed question the

Ed Arce

Hi, Ron. taking questions. Thanks my for

ask this to that in Could added around this the and is wanted be it thinking recently this you announcement just slightly additional and other other neonatal obviously this your wondering than question beyond PFIC was adults beyond PFIC this second second with new X agency types around morning even mentioned X. and PFIC cohort, could add of additional different behind X cohort the a include I perhaps a the So ultimately decision what as label? discussions I'm to was what PFIC, drove the impacts how so as and

Ronald Cooper

morning. agency Thanks good opinion prompting liver not from I with Ed, our the about they're the as working pediatric investigators key from the prompting in very question. a the much cholestatic leaders, actually the potential think pretty excited and much as for of diseases. It's odevixibat

I a to think PFIC that up help think to they as and X pleased I going patients, but have are patients trial wanted they for other well.

cohort up not the working And to where patients to do because open on our decision dialogue therapeutic and so becomes help children that our very option second these we're and investigators made it a a with that based franchise leading them closely and we feedback, to also player through But a to process our help insights space liver build into in getting and commitment generate a being the data odevixibat. now. us right to to option pediatric odevixibat on to this approval fulfill an have additional with

Ed Arce

about then or curious opposed primary accumulate two really this disease NASH. endpoint how across in elobixibat for NASH put of ultimately in is times program trials for that quarter. expect reductions or you is measures you’ve and you responses out Thanks. upon the Obviously, perhaps guidance multiple you deep FDA to is And start that helpful. impact effects measures, consistent turning comment your to your expand differentiating this elobixibat this a with up LDL stage see the or partnering if That's key combination mentioned earlier could in here to that Okay. couple earlier do I'm which these robust either as and/or to in one studies? recently

Ronald Cooper

Thanks, Ed.

efficacy excited a medical pronged why need standpoint from that's an this approach think think that between here. we approach our that from in we're disease”. standpoint in understanding two unmet I is medical elobixibat is about unmet convenience have NASH “asymptomatic [indiscernible] a there need and an really both We

on that data IBAT think problem bile fibrosis. this, have have liver So NASH clinical and part acids our with problem who that with patients with the generated have transport, with efficacy have problem information those all and we some we a know inhibitors, cholesterol, preclinical is the a elevated And parameters. and liver beauty we've on of glucose

offerings impact on that parameters an that now. So can of some we there other the believe unlike we out have are multiple

is So that's of one systemic product, day once aspect aspect, but convenience elobixibat that's it, it very the right. has effects – little a

and these we other believe may most some being effects So we potentially I pharmacy of has within little side cardiovascular be think because aspect that nicely it very systemic there. believe are NASH there or drugs GI with other quite poly that the it itself combines exposure drugs

a offerings other elobixibat of of think So the in space. that once compounds of and our of on I day multiple is it could with our co-travel exciting interesting makes in convenience parameters kind multiple potentially preclinical the the combination others, non-systemic NASH some in NASH effects a that product of

Ed Arce

a couple commercial prepared the multiple in the much focused the stated of And progress forward be sort remarks, years again. your on plan. moving and based And ultimately one study expanding expect question Thanks as beginning great. pivotal of in your – and last then first very just That's brand on to you and here ultimately approach PFIC strategy now next you've had at and FDA would as mentioned disease perhaps looking odevixibat. you're I franchise you over times picture big excited at

So inform as these strategy does how does how and plan? building multiple think indications, that you about with that work potentially and out your your

Ronald Cooper

question. the for Ed, Thanks

liver multiple uses the across data franchise indications pediatric across beauty having pediatric with strong brand of liver I to that hepatologists earlier so think PFIC stated provide elobixibat. right be with we're also as of relationships I diseases, very cholestatic the and multiple sort I physician stronger going think strong that initial diseases same product and other developing a by some in managed right that's has are excited and and to and a build we're then a but data to additional

Ed Arce

Appreciate again. it. Thanks Great.

Operator

Thank you.

question-and-answer closing any We Cooper like our I the have remarks. the call back over session. turn end of would for reached to Mr. to

Ronald Cooper

you, operator. Thank Great.

like thank into today. First of our all, to I'd for everybody tuning call

our changed note feedback. earlier till time might in o’clock the of day based from some on we've XX the call You that

So a it's we'd love to special to just better And works saying not. hear for time or at from Albireo. by you that whether close you that

looking company And We plan Thanks over day. continue great our to forward very our we're on commitments. and on much. time. deliver to to data execute a Have the additional grow generating

Operator

Thank teleconference. you. concludes today's This

day. at this and disconnect your a wonderful your time. for lines Thank participation you may have You

Albireo Pharma (ALBO) 9 May 192019 Q1 Earnings call transcript