and Officer Commercial Financial today Albireo's Chief Chief is and you Paul who Dr. morning's me With Horn; Simon have Stephenson thanks our Officer of you, to all our this Medical Thank call. joined us Harford. conference on Pamela Pat Chief Officer
proprietary compelling for liver the potential opportunity disease. candidate homeostatic believe IBAT in we've to development the this or range pediatric the product odevixibat you a progress our be inhibitor. made bile acid We're of the months to on option a update ileal We pleased treatment wide in meaningful holds of recent to
Our odevixibat Cholestasis other we is study Familial Progressive to in or diseases. lead Intrahepatic And plan PFIC. cholestatic indication
by life can all types across genetic result in or are Patients PFIC their about progress that the for XX,XXX the of to [ph] one early for bile an cirrhosis, quickly and new insatiable symptoms Now, affect families. mutations ages there in which the experience and in to in of most be PFICs they that is acid the untreated. death troublesome in those intense to They can are of to and liver two at be estimate to the Albireo buildup tend caused X,XXX multiple of the often patients failure you disease US with We Europe. that spectrum liver. diagnosed a variety and itch, tends pruritus also both if patients
medications stoma permanent are procedures bile limited or PFIC. approved There bag diversion surgical such are invasive biliary transplant. liver a Treatment to surgery no as acids that's to options or used treat to drain a
ultimate Our well-tolerated hopefully or provide goal can liver option that ease to postpone reduce treatment a patients bile pruritus acid, transplant. and prevent with is a their
responder odevixibat for of assessment primary planned XX the The endpoint the for acid change evaluation pruritus. or the formulation of for rate. is EMA the or commercial odevixibat PEDFIC-X XX is months XXX double-blind X X. XX microgram six receiving are is trial patients, ages Patients that PFIC clinical kilogram a of or type in evaluating to years placebo-controlled XX dose per oral either The placebo endpoint FDA an weeks. bile with randomized serum is in primary once-daily the
our in has well progressed summer Enrollment update. PEDFIC-X since
been the actively patients number by screening encouraged XX who've the world. the entering of be to over sites continue around We recruiting at
patients to there this are more prescreening adequate of randomized we three than trial. the in screening of quarters complete the that to patients or note to October, we'd needed in the It's today also end complete additional trial. As of important randomization believe
the position determined in we disease and on patients, exact randomization track assuming end to results launch an be in announce we'll for plan results is to and the line potential this a report from last is remain ultra-rare firmly few top-line will finished in trial we the confident positive when will top mid-XXXX. be Now trial in of that timing study the that XXXX. us the put by outcome for PFIC
PEDFIC-X dose portion trial. rolling as over who patient have extension third We the are XX-week cohort to in patients X trial we have the odevixibat, or daily. odevixibat patients or screened now expanded per Meanwhile the PEDFIC-X. refer our our treatment. cohort we patients X approaching All the cohort higher year the been of a which PEDFIC-X rollover cohort on first micrograms XXX of receive in In this study extension voluntary the of quarter kilogram to of also
XX PFIC composed for the cohort years types of and expanded and older of is have eligibility who bile patients meet or than acid includes do serum still PEDFIC-X, age. than six levels This elevated not but months younger The of PFIC who patients all pruritus. with patients criteria
a data evidenced separately will commitment different with providing for new reflects potential affecting cohort The the cohort set and new patients possible of of second to many body odevixibat. be if PEDFIC-X. without option of X maximizing The our analyzed while for types PFIC treatment patients of
also our great are we engagement critical odevixibat. our provide plans key to with opinion on for program, feedback as Now, to our continue robust deepening Phase leaders make headway III with we
this patient meeting engage engagement US, world. the liver the taking here pre-commercial from access to our which and Europe we fronts week liver and important at as work market clinicians events cholestatic place For preeminent regulatory Boston from We're advancing advocacy. in AASLD around multiple multiple the well have on disease instance that manufacturing is
batches our all a for pleased CMC that report work or well to and Controls Manufacturing our access the and regulatory is refine the economics under filings. health we the as preclinical From way under continue value a we've US way. produce perspective odevixibat. is work validation studies payer toxicology completed perspective planning to also EU market for well for required We're Chemistry, From and to story manufacturing and
of pleased success campaign. really early advocacy Voices And we've our terms in PFIC of with the been
up That profiles. one disease of Piecyk audiences. diagnosed a just by expert family we're powerful PFICvoices.com and are were aim our when she initiative and who side, community the such was and this with Eleanor, is PC vehicles PFIC We became story unaware teaming on with key after months awareness patients and are was that number mother discovering One understanding disease. hospitals the who employing. there growing across website of growing with the is this of old, On the three local doctors about her Claire, the of necessity,
that as during and comfort marriage, to diagnosis, disease inability describes daughter pain, the with in her life the well-being. put struggle strain Clara her chronic mental work the as a well
about entire resulting You also campaign patients the The so hear that months. affects media the in the experience debilitating includes also recent and outreach deprivation Voices traction that PFIC often families. element has it significant the provides, generated sleep
for be to stories We patients bringing Media commend by They unmet face patients. challenges been in Kentucky thank been find lacked have an to want their the outlet. involved, PFIC. and but they Despite them the outlets world. profiling all PFIC Delaware and Colorado the parents support the to has community from And in the sharing to sharing they needs with and focus experiences reception very positive.
atresia. of our as provide patients on of an with treatment group therapy biliary work for namely touch option desperate odevixibat approved a children another to let's need Now, in
untreated indication PFIC, XX,XXX bile cause a we US be to liver a generally While to biliary in is rare Europe. still left the and XX,XXX disease, of impacting blocked which estimate if in larger caused ducts non-existent is atresia within months. by patients about failure BA or
of Children flow. Virtually intestine to all connects Kasai receive BA within procedure quickly the part life. bile the restore undergo the to of a months with children their which first liver surgically diagnosis small
XX. is by of still the for transplant liver two a XX% about the liver While require infant age patients and require the years transplant lifesaving this a approximately XX% within of procedure
work orphan Both atresia the a recent been in granted biliary continue and for that very the report have further the work FDA And in XXXX. FDA regarding for plan constructive in to bit odevixibat. sharing to the undertaken through there's this and forward details been work odevixibat believe to the complete. with look pleased months never clinical we in has to a of initiation we're pivotal we've atresia is path about the of agency. EMA the trial trial interactions bulk quite Since biliary with had and We designation
So odevixibat advantages. holds distinct product we summary believe that in a has is and candidate tremendous several potential
it's First, most in humans. the potent IBAT inhibitor
gastrointestinal the need is population. once-daily food with perspective dosed it to be our capsules medication. demonstrated a sprinkled From believe minimal to the allow remains advantage odevixibat and the we've into when PK not be dealing systemic do exposure. They medication big that refrigerated. a with is which for Also Second, we in onto oral pediatric a oral as tract
diarrhea very that we extensions, opportunity. PFIC including were doses day. III per odevixibat in physician odevixibat expected no With In receiving kilogram treatment a potential into the believe patients micrograms planned small study for audience, Phase program. us II XXX first orphan the of in the approved ongoing believe Phase up attractive in world. We're target and finally patent strong there multiple And be the of odevixibat commercial using an takes will a odevixibat formulation of we represents our to that reports pricing estate rights, unencumbered XXXX per of global
NAFLD randomized IBAT NASH patients first our other the and II third we Turning in during to our in inhibitor quarter. trial elobixibat, Phase
parameters that our therapeutic disease. most on stage As is NASH the upside as real including impact It's disease approach Albireo our worthy preclinical potential fibrosis of and view our metabolic program GLP-X. apparent small multiple at as based And treatment becoming a for belief we across investment. increasingly to cumulative and this serum know, liver holds liver fundamentally based inflammation a a that well work show bile NASH investors of on for a is relatively NASH IBAT pure cholesterol, promise acids, of inhibition
we or patients our to Phase The multi-center all trial this outstanding We're when diagnosis Albireo. enroll study. syndrome on metabolic by II progress completed. testing The XX definition. possible theory based placebo-controlled an enroll is to with NAFLD continues and is suspected culture trial and this in NASH an at biopsy-confirmed update plan a team provide Naturally in made or a NASH special will enrollment
play development TESARO, Chief Human Bristol-Meyers XXXX Bausch look Integer, organizations. biopharma Graham As organization senior will She that Parexel, develops retains build as growth our Squibb. Michelle leadership really held and Resources accelerates, has experience Officer. world-class attracts, + team we and extensive our to and as human resources company welcome Lomb organizational and successful excited a at developing to Michelle a to Michelle has key I'm role roles global top talent. to forward need we
pleasure it's a that the call to turn over update. with Simon? to for financial my So Simon