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Lumena Pharmaceuticals Launches Global Clinical Program to Evaluate Safety and Efficacy of LUM001 in Children with Alagille Syndrome

SAN DIEGO, Sept. 26, 2013 /PRNewswire/ -- 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.

ALGS is a rare form of cholestatic liver disease that results in impaired bile acid flow and retention of bile acids in the liver, leading to progressive liver damage that can cause liver failure. LUM001 is being evaluated for its ability to lower serum bile acids and alleviate severe itching, which is generally the most debilitating symptom afflicting patients with the disease. By reducing serum bile acids with a once-daily oral solution, LUM001 may offer a novel therapeutic approach to prevent bile acid mediated liver damage and alleviate severe itching.

The IMAGO study is a 13 week placebo-controlled study to evaluate the safety and efficacy of LUM001 in children with ALGS. The U.K. based study will enroll children, two years of age and older, with ALGS.  

"Symptoms of Alagille syndrome include intense itching and sleep disruption on an ongoing basis, which is extremely problematic for the whole family when a child is suffering," said Alastair Baker, M.D., Consultant Paediatric Hepatologist, Kings College Hospital, London, and IMAGO study principal investigator. "Pediatric patients have very little relief from the aggravating symptoms, and the need for treatment options is pressing."

"LUM001 represents a potential new approach to treating the progressive liver damage and symptoms associated with Alagille syndrome that severely affect a patient's quality of life," explained Benjamin Shneider, M.D., Pediatric Hepatologist, Children's Hospital of Pittsburgh of UPMC, the ITCH study chair. "Currently, there are no approved drugs to treat the cholestasis and pruritus associated with ALGS, and frequent options for children suffering with this disease are off-label treatments with limited efficacy and potential liver toxicity or surgical diversion of bile, which comes with risk of complications and quality of life issues."

The ITCH study is a multi-center U.S.-based study which is expected to begin later in 2013 and will be seeking pediatric patients, two years of age and older, with ALGS. The ITCH study is being conducted in collaboration with the Childhood Liver Disease Research and Education Network (ChiLDREN), a collaborative research network supported by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), a part of the National Institutes of Health (NIH),which has a Cooperative Research and Development Agreement (CRADA) with Lumena. Lumena and ChiLDREN are also collaborating with European investigators to evaluate LUM001 in patients with progressive familial intrahepatic cholestasis (PFIC), another rare cholestatic liver disease.

"This public/private partnership is an example of a fruitful collaboration to find treatments for rare diseases," said Edward Doo, M.D., director of the Liver Diseases Research Program at NIDDK. "These cholestatic liver diseases have devastating symptoms and there is a significant need for additional treatment options, especially for children."

Patients that complete either the IMAGO or ITCH study will be eligible for participation in a long-term extension study.  For more information about the IMAGO study, visit the study page at, a registry of funded research studies in the United States and around the world. Information about the ITCH study will also be available at once the study starts in the U.S.

"I think that I can speak on behalf of the Alagille syndrome community when I say that we enthusiastically support Lumena's work to develop safe, oral therapies to treat Alagille syndrome, and I am especially glad that the company is planning an extension study that allows for patients to have long-term access to this treatment while it is in development," said Cindy Hahn, founder and CEO of the Alagille Syndrome Alliance.

About LUM001

LUM001 is an inhibitor of the apical sodium-dependent bile acid transporter (ASBT), which recycles intestinal bile acids back into the circulation. LUM001 has been studied in 12 clinical trials in more than 1,400 subjects. In previous trials, LUM001 was shown to be generally safe, and the most common side effect, gastrointestinal disturbance, was usually mild and transient in nature. Clinical studies have demonstrated that LUM001 can reduce serum bile acid levels. Reductions in bile acids may alleviate symptoms and improve liver function in many patients with cholestatic liver disease.

Lumena plans to initiate Phase II studies of LUM001 in adults with primary biliary cirrhosis (PBC), children with progressive familial intrahepatic cholestasis (PFIC), and in patients with primary sclerosing cholangitis (PSC) in late 2013. Lumena has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) Office of Orphan Product Development for LUM001.

About Alagille Syndrome

ALGS is a rare genetic disorder that can affect the liver, heart, and other parts of the body. Approximately one in every 70,000 children is born with ALGS. In patients with ALGS the bile ducts are abnormally narrow, malformed and reduced in number. This prevents normal bile flow from the liver into the small intestine. As a result, bile builds up in the liver, causing damage that prevents the liver from working properly. ALGS is characterized by elevated bile acids and severe itching, which is generally the most debilitating symptom afflicting children with this liver disease. Treatment with anti-pruritics (anti-itching drugs) typically provides only modest relief. Procedures that remove bile from the circulation can lower serum bile acids, reducing itch and improving liver function in some patients, but tend to be invasive in nature.

About Lumena Pharmaceuticals
Lumena Pharmaceuticals is a San Diego-based company developing oral therapeutics for patients with rare and debilitating liver diseases. Lumena's lead candidate, LUM001, has been studied in more than 1,400 patients in 12 different clinical studies and is currently being evaluated in a Phase II study in patients with Alagille syndrome. The company is privately held and has raised $23 million in Series A financing from Pappas Ventures, RiverVest Venture Partners and Alta Partners.

SOURCE Lumena Pharmaceuticals
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