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CAMEO Phase 2 Clinical Trial of LUM001 Now Enrolling Adults with Primary Sclerosing Cholangitis

SAN DIEGO – May 9, 2014 – Lumena Pharmaceuticals (Lumena), a biopharmaceutical company focused on the development and commercialization of novel products for rare cholestatic liver diseases and serious metabolic disorders, today announced the dosing of the first patient in the INDIGO Phase 2 clinical trial of its lead drug candidate, LUM001, in children with progressive familial intrahepatic cholestasis (PFIC). Additionally, the CAMEO Phase 2 clinical trial of LUM001 in adults with primary sclerosing cholangitis (PSC) is open for enrollment.

Company receives first positive opinion in the European Union for Primary Sclerosing Cholangitis

SAN DIEGO – November 18, 2013 – Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced that it has received positive opinions for four Orphan Drug Designations by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) for LUM001. The company’s lead drug candidate, LUM001, received the first positive opinion granted by the EMA for primary sclerosing cholangitis (PSC). In addition, the company received positive opinions for LUM001 in three other rare cholestatic liver diseases including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and primary biliary cirrhosis (PBC). This follows the Orphan Drug Designation in September by the U.S. Food & Drug Administration (FDA) Office of Orphan Product Development for LUM001 in the same indications.

Data Presented Today at Annual Meeting of the American Association for the Study of Liver Diseases in Washington DC Provides Additional Preclinical Validation for LUM001 Currently Being Evaluated in Phase II Studies in Multiple Cholestatic Liver Disease Populations

SAN DIEGO – November 5, 2013 – Lumena Pharmaceuticals, a company developing oral therapeutics for cholestatic liver diseases, today announced that inhibition of the apical sodium-dependent bile acid transporter (ASBT) improves liver function in animal models of cholestasis. The data will be presented at 11:15 a.m. EST today in an oral presentation titled “SC-435, an Oral Inhibitor of ASBT, Improves Liver Function in a Rat Partial Bile Duct Ligation Model of Cholestasis,” at the 64th Annual Meeting of the American Association for the Study of Liver Diseases in Washington DC.

Novel therapeutic approach to be evaluated for its ability to reduce elevated bile acid levels and associated severe itching

SAN DIEGO – October 29, 2013 – Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the dosing of the first patient in the CLARITY Phase II study of its drug candidate LUM001 in patients with primary biliary cirrhosis (PBC). LUM001 is being developed as a possible therapy for a number of cholestatic liver diseases including, PBC, Alagille syndrome, progressive familial intrahepatic cholestasis and primary sclerosing cholangitis, that result in impaired bile acid flow and retention of bile acids in the liver, leading to progressive liver damage and, ultimately, liver failure. In the CLARITY Phase II study, LUM001 is being evaluated for its ability to alleviate the symptom of severe itching associated with PBC.

Company Pursuing Orphan Path to Development in Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis, Primary Biliary Cirrhosis and Primary Sclerosing Cholangitis

SAN DIEGO – September 26, 2013 – Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) Office of Orphan Product Development for LUM001, the company’s lead drug candidate. LUM001 received Orphan Designation in four rare cholestatic liver diseases including Alagille syndrome (ALGS); progressive familial intrahepatic cholestasis (PFIC); primary biliary cirrhosis (PBC); and primary sclerosing cholangitis (PSC).

First patient dosed in U.K. in Phase II IMAGO Study; Phase II ITCH Study to start in U.S.

Cooperative Research and Development Agreement established with the National Institutes of Health and the Childhood Liver Disease Research and Education Network (ChiLDREN)

SAN DIEGO – September 26, 2013 – Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the initiation of a global clinical program to evaluate its drug candidate LUM001 in children with Alagille syndrome (ALGS). The first patient has been dosed in the IMAGO Phase II study being conducted in the U.K., and enrollment of pediatric patients is expected to begin later this year in the ITCH Phase II study in the U.S.