Publications &

Presentations

In our pursuit of scientific advancement we are committed to sharing our acquired knowledge through actively publishing our data in peer-reviewed journals and academic conferences. The focus of what we have published is on transforming the lives of people living with rare liver disease through a concentration on ileal bile acid transporter (IBAT) inhibition.

IBAT inhibition

An important intestinal transporter that regulates the enterohepatic circulation of bile acids and cholesterol homeostasis: the apical sodium-dependent bile acid transporter (SLC10A2/ASBT)

Xiao L, Pan G. Clin Res Hepatol Gastroenterol. 2017;41:509-515.

Role of the intestinal bile acid transporters in bile acid and drug disposition

Dawson PA. Handb Exp Pharmacol. 2011;201:169-203.

Alagille syndrome

Application of real-world evidence analytics: A 6-year event-free survival analysis in Alagille syndrome of the GALA clinical research database and maralixibat treated patients

Hansen BE, et al. Presentation. AASLD 2021.

Predictors of 6-year event-free survival in patients with Alagille syndrome treated with maralixibat, an IBAT inhibitor

Sokol RJ, et al; AASLD 2021 Late-breaker.

Pediatric cholestasis: itching for an alternative to liver transplantation

Kamath BM, et al. Symposium Presentation, WCPGHAN 2021.

Outcomes of childhood cholestasis in Alagille syndrome: results of a multicenter observational study

Kamath BM, et al. Hepatol Comm. 2020;4:387-398.

Alagille syndrome and other hereditary causes of cholestasis

Hartley JL, et al. Clin Liver Dis. 2013;17:279-300.

Systematic review: the epidemiology, natural history, and burden of Alagille syndrome

Kamath BM, et al. J Pediatr Gastroenterol Nutr. 2018;67(2):148-156.

Alagille syndrome: pathogenesis, diagnosis and management

Turnpenny PD, Ellard S. Eur J Human Genetics. 2012;20:251-257.

Progressive familial intrahepatic cholestasis

Progressive familial intrahepatic cholestasis

Davit-Spraul A, et al. Orphanet J Rare Dis. 2009;4:1.

Normalization of serum bile acids after partial external biliary diversion indicates an excellent long-term outcome in children with progressive familial intrahepatic cholestasis

Schukfeh N, et al. J Pediatr Surg. 2012;47(3):501-505.

ATP8B1 and ABCB11 analysis in 62 children with normal gamma-glutamyl transferase progressive familial intrahepatic cholestasis (PFIC): phenotypic differences between PFIC1 and PFIC2 and natural history

Davit-Spraul A, et al. Hepatology. 2010;51(5):1645-1655.

Systematic review of progressive familial intrahepatic cholestasis

Baker A, et al. Clin Res Hepatol Gastroenterol. 2019;43(1):20-36.

Genotype correlates with the natural history of severe bile salt export pump deficiency

van Wessel D, et al. J Hepatol. 2020;73(1):84-93.

Intrahepatic cholestasis of pregnancy

Association of adverse perinatal outcomes of intrahepatic cholestasis of pregnancy with biochemical markers: results of aggregate and individual patient data meta-analyses

Ovadia C, et al. The Lancet. 2019;393:899-909.

An integrated analysis of long-term clinical safety in maralixibat-treated participants with Alagille syndrome

Raman R, et al. Poster Presentation. EASL. 2021.

Patient-reported outcomes validation

Development of a novel tool to assess the impact of itching in pediatric cholestasis

Kamath BM, et al. Patient. 2018;11:69-82.

Psychometric validation of the itch reported outcome (ItchRO™) observer assessment in pediatric patients with Alagille syndrome or progressive familial intrahepatic cholestasis

Serrano D, et al. Poster Presentation, AASLD 2016.

Psychometric evaluation of the adult itch reported outcome tool, a worst itch numeric rating scale in adults with cholestatic liver disease

Foster E, et al. Poster Presentation, EASL 2020.

LIVMARLITM (maralixibat)

Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study

Gonzales E, et al. The Lancet. 2021;398:1581-1592.

Serum bile acid control in long-term maralixibat-treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency

Thompson R, et al. Podium Presentation, WCPGHAN 2021.

Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: an integrated analysis of short- and long-term treatment

Kamath BM, et al. Poster Presentation, WCPGHAN 2021.

Pruritus intensity is associated with cholestasis biomarkers and quality of life measures after maralixibat treatment in children with Alagille syndrome

Gonzales E, et al. Poster Presentation, AASLD 2020.

Natural variability of pruritus in Alagille syndrome; an analysis from the ICONIC study utilizing the itch reported outcome observer (ItchRO[Obs]) tool

Foster B, et al. Poster Presentation, AASLD 2020.

Serum bile acid control in long-term maralixibat-treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency

Thompson R, et al. Podium Presentation, EASL 2020.

Differential expression of bile acid subspecies with maralixibat treatment in pruritus responders with bile salt export pump deficiency

Zhao E, et al. Poster Presentation, EASL 2020.

Genotype and dose-dependent response to maralixibat in patients with bile salt export pump deficiency

Thompson R, et al. Podium Presentation, AASLD 2019.

Durability of treatment effect with long-term maralixibat in children with Alagille syndrome: 4-year safety and efficacy

Gonzales E, et al. Podium Presentation, AASLD 2019.

Safety and efficacy of maralixibat in patients with primary sclerosing cholangitis: an open-label proof-of-concept study

Bowlus C, et al. Poster Presentation, AASLD 2019.

Dose-dependent fecal bile acid excretion with apical sodium-dependent bile acid transporter inhibitors maralixibat and volixibat in a dose-ranging Phase 1 study in overweight and obese adults

Martin P, et al. Poster Presentation, AASLD 2019.

Phase 2 placebo-controlled withdrawal study of the ASBT inhibitor maralixibat in children with Alagille syndrome

Gonzales E, et al. Podium Presentation, EASL 2019.

Growth analysis in children with PFIC treated with the ASBT inhibitor maralixibat

Thompson R, et al. Podium Presentation, EASL 2019.

A randomized, controlled, Phase 2 study of maralixibat in the treatment of itching associated with primary biliary cholangitis

Mayo MJ, et al. Hepatol Commun. 2019;3(3):365-381.

Placebo-controlled randomized trial of an intestinal bile salt transport inhibitor for pruritus in Alagille syndrome

Shneider BL, et al. Hepatol Comm. 2018;2(10):1184-1198.

Phase 2 open-label efficacy and safety study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with progressive familial intrahepatic cholestasis: 48-week interim efficacy analysis

Thompson R. Podium Presentation, AASLD. 2017.

A long-term Phase 2 safety and efficacy study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with Alagille syndrome: preliminary results from the IMAGINE study

Thompson R. Podium Presentation, AASLD. 2017.

Results of ITCH, a multi-center randomized double-blind placebo-controlled trial of maralixibat, an intestinal bile salt transport inhibitor, for pruritus in Alagille syndrome

Shneider BL, et al. Podium Presentation, AASLD. 2017.

Volixibat

Safety, tolerability and efficacy of volixibat, an apical sodium-dependent bile acid transporter inhibitor, in adults with non-alcoholic steatohepatitis: 24-week interim analysis results from a Phase 2 study

Newsom P, et al. Poster Presentation, EASL. 2019.

A Phase 1 dose-ranging study assessing fecal bile acid excretion by volixibat, an apical sodium-dependent bile acid transporter inhibitor, and coadministration with loperamide

Key C, et al. Poster Presentation, AASLD. 2020.

Health economics and outcomes research

Healthcare resource utilization in patients with Alagille syndrome

Ebel NH, et al. Poster Presentation, AASLD 2021.

Cost of pediatric liver transplant among commercial and Medicaid insured patients

Miloh T, et al. Poster Presentation, AASLD 2021.