Lumena Pharmaceuticals Now Dosing Patients in the INDIGO Phase 2 Clinical Trial of LUM001 in Pediatric Patients with Progressive Familial Intrahepatic Cholestasis

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

SAN DIEGO, May 9, 2014 /PRNewswire/ -- 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.

LUM001 is being developed as a therapy for cholestatic liver diseases including PFIC, PSC, Alagille syndrome and primary biliary cirrhosis. These diseases result in impaired bile acid flow and retention of bile acids in the liver, leading to progressive liver damage that may ultimately result in liver failure. Severe itching is generally the most debilitating symptom afflicting children and adults with these diseases. LUM001 is an inhibitor of the apical sodium-dependent bile acid transporter (ASBT), which recycles bile acids from the intestine to the liver. Blocking bile acid transport in the intestine with ASBT inhibitors, such as LUM001, has the potential to lower bile acid levels in the body, slow disease progression, improve liver function and enhance the quality of life for patients suffering from cholestatic liver diseases.

"LUM001 may reproduce the effects of more invasive surgical procedures for children suffering from PFIC," said Dr. Richard Thompson, King's College London, one of the investigators enrolling patients into the INDIGO trial. "The potential to alleviate debilitating itching whilst improving overall liver function with a once-daily drug represents a much-needed pharmacological treatment option for this rare, but serious, disease."

INDIGO is an open-label, Phase 2 clinical trial designed to evaluate the efficacy and safety of LUM001 for the treatment of cholestatic liver disease in patients with PFIC. This international trial will enroll approximately 12 patients under the age of 18 who have been diagnosed with PFIC. The primary endpoint of the trial is change from baseline in fasting serum bile acids. Secondary endpoints include changes from baseline in liver enzymes and pruritus (itching).

"The children affected by PFIC suffer from constant itching, and this affects every aspect of their lives. We are encouraged by the start of this clinical trial since it represents a much needed step forward in the development of a treatment for PFIC. These patients and their families have had no solution to speak of, and Lumena's trial and dedication offers them hope," said Robin Marceca, founder of PFIC.org.

CAMEO is a 14 week open-label clinical trial designed to evaluate the safety, tolerability and efficacy of LUM001 in approximately 20 adult patients with PSC. The primary endpoint of the trial is change from baseline in fasting serum bile acids. Secondary endpoints include changes from baseline in pruritus and other biochemical markers of cholestasis and liver disease.

"Currently, there are no effective medical therapies which prevent the progression of primary sclerosing cholangitis, which on average leads to liver failure and the need for liver transplantation within 15 years of diagnosis," said Christopher Bowlus, M.D., University of California, Davis, principal investigator of the CAMEO trial. "There is a significant need for drug treatments for this serious condition, and this trial is an important step in that direction."

About LUM001
LUM001 is a novel, once-daily, orally-administered, potent and selective ASBT inhibitor, which reduces bile acid intestinal absorption leading to an increase in bile acid excretion and lower levels of bile acids in the liver and systemic circulation. LUM001 is being developed as a treatment for rare cholestatic liver diseases characterized by elevated bile acids in the liver, leading to progressive liver damage, as well as debilitating symptoms such as pruritus, or severe itching. Lumena is currently evaluating LUM001 in five ongoing trials including: a Phase 2 clinical trial in children with Alagille syndrome (ALGS); a Phase 2 clinical trial in children with progressive familial intrahepatic cholestasis (PFIC); a Phase 2 clinical trial in adults with primary biliary cirrhosis (PBC); and a Phase 2 clinical trial in adults with primary sclerosing cholangitis (PSC). LUM001 has been studied in more than 1,400 human subjects in 12 different clinical studies evaluating the product candidate as a treatment for elevated cholesterol levels. LUM001 has received Orphan Drug Designation for ALGS, PFIC, PBC and PSC from both the U.S. Food & Drug Administration Office of Orphan Product Development and the European Medicines Agency.

About Progressive Familial Intrahepatic Cholestasis
PFIC is a rare genetic disorder estimated to affect one in every 100,000 people in the United States and two in every 100,000 people in the European Union, and it is generally diagnosed in children. Patients with PFIC have mutations in certain proteins that prevent their liver cells from properly secreting bile. In the absence of treatment, the resulting bile buildup in the liver cells causes progressive liver disease and typically leads to liver failure.

About Primary Sclerosing Cholangitis
PSC is a disease that causes chronic inflammation and subsequent scarring of the bile ducts both inside and outside the liver. The blockage of the bile ducts prevents bile from being transported to the small intestines and gall bladder. The accumulation of bile in the liver leads to progressive liver damage, and eventually, liver failure. Primary sclerosing cholangitis is estimated to affect approximately 14 in every 100,000 people in the United States and 3 in every 100,000 people in the European Union, and for reasons that are unknown, it impacts men approximately twice as often as women.

About Lumena Pharmaceuticals
Lumena Pharmaceuticals is a San Diego-based biopharmaceutical company focused on the development and commercialization of novel products for rare cholestatic liver diseases and serious metabolic disorders, such as NASH, where there is a high unmet medical need. Lumena's lead product candidate, LUM001, has been studied in more than 1,400 human subjects in 12 different clinical trials evaluating the product candidate as a treatment for elevated cholesterol levels, and is being evaluated in Phase 2 clinical trials in patients with rare cholestatic liver diseases, including ALGS, PFIC, PBC and PSC. Lumena's second product candidate, LUM002, is being developed for the treatment of NASH. Lumena plans to initiate a Phase 2 clinical trial of LUM002 in NASH patients in the second half of 2014. Lumena is privately held and has raised a total of $78 million in private financings.