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Lumena Pharmaceuticals Receives Orphan Drug Designation From FDA and Launches Global Clinical Program


11/1/2013 9:22:26 AM

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November 01, 2013 -- Lumena Pharmaceuticals develops oral therapeutics for rare liver diseases and has been in the news for two recent company announcements. First, the U.S. Food & Drug Administration (FDA) Office of Orphan Product Development granted Lumena’s lead drug candidate, LUM001, Orphan Drug 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). In addition, the company has announced it has launched a Phase II global study to evaluate the safety and efficacy of LUM001 in children with ALGS.

Cholestatic liver diseases, such as ALGS, PFIC, PBC and PSC, result in impaired bile acid flow and retention of bile acids in the liver, leading to progressive liver damage that can cause liver failure. By reducing serum bile acids with a once-daily oral drug, LUM001 may offer a novel therapeutic approach to reducing elevated bile-acid levels, preventing liver damage and alleviating severe itching, which is generally the most debilitating symptom afflicting patients with these diseases. “Orphan Drug Designation from the FDA allows us to pursue an accelerated pathway for development of LUM001 so we can deliver new drug treatments for the patients in desperate need,” said Alejandro Dorenbaum, M.D., chief medical officer of Lumena. “Current therapeutic options for cholestatic liver diseases are of limited efficacy, and surgical approaches to reducing bile acid levels to manage disease symptoms can be disfiguring and prone to complications. The only remaining option for many patients is liver transplant.”

The company currently has two clinical trials underway and additional studies planned. The company has initiated an international Phase II study of LUM001 in children with ALGS, and the first patient has been dosed in the IMAGO Phase II study being conducted in the U.K. and plans to enroll pediatric patients later this year in the U.S. The company is also currently enrolling adult patients with primary biliary cirrhosis experiencing moderate to severe pruritus (itching); further details on that study can be found here. Later this year, Lumena plans to initiate a Phase II study of LUM001 in pediatric patients with PFIC, and a Phase II study in adults with PSC. LUM001 has already been studied in more than 1,400 patients in 12 different clinical studies.

Lumena Pharmaceuticals is a San Diego-based company developing oral therapeutics for patients with rare and debilitating liver diseases. The company is privately held and has raised $23 million in Series A financing from Pappas Ventures, RiverVest Venture Partners and Alta Partners.

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