Funding to support clinical development of drug candidates to treat rare liver diseases

SAN DIEGO May 8, 2013 – Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the company has secured $23 million in Series A financing. Investors include Pappas Ventures, RiverVest Venture Partners and Alta Partners. Founded in 2011 by Pappas Ventures, Lumena will primarily use the funding to advance the clinical development of LUM001, the company's lead product candidate for the treatment of cholestatic liver disease in pediatric and adult patients. 

LUM001 is being developed as a possible therapy for progressive liver damage and debilitating symptoms associated with a number of liver conditions that result in impaired bile acid flow and retention of bile acids in the liver, leading to progressive liver damage and, ultimately, liver failure.

Cholestatic liver diseases are characterized by elevated bile acids and intractable itch, which is generally the most debilitating symptom afflicting adults and children with these diseases.  Treatment with anti-pruritics typically provides only modest relief, and other available drug treatments are of limited value. Surgical procedures that divert bile from recirculation can lower serum bile acids, reduce itch and improve liver function in some patients.  LUM001 is an inhibitor of the apical sodium-dependent bile acid transporter (ASBT), which recycles intestinal bile acids back into the circulation.  By reducing serum bile acids without surgery, LUM001 may be of value in the treatment of cholestatic liver diseases.

"There is an urgent need for effective pharmacological treatments for patients with cholestatic liver disease who experience itching so extreme that it causes severe sleep disruption and scratching with skin destruction and scarring -- symptoms which significantly impair quality of life," said Mike Grey, president and CEO of Lumena Pharmaceuticals. "Lumena's targeted approach to inhibiting bile acid recirculation to the liver with LUM001 may improve liver function, relieve symptoms and dramatically impact patient health without the need for risky surgeries that reduce bile acids."

In association with the financing, the following were named to Lumena's board of directors: David R. Savello, Ph.D., Scientific Advisory Board, Pappas Ventures; John McKearn, Ph.D., managing director, RiverVest Venture Partners; and Robert Alexander, Ph.D., executive chairman, ZS Pharma and formerly a general partner at Alta Partners.

The company has recruited the following management team:

·         Mike Grey, president, CEO and co-founder, is a venture partner with Pappas Ventures and was previously CEO of SGX Pharmaceuticals, which was acquired by Eli Lilly & Co. in 2008.

·         Alejandro Dorenbaum, M.D., chief medical officer, previously of Genentech and BioMarin, has extensive experience in developing drugs for orphan indications.

·         Ciara Kennedy, Ph.D., vice president, operations, joined Lumena from Cypress Bioscience Inc. where she played a key role in the company's FDA approval of Savella® for fibromyalgia, two corporate acquisitions and the in-licensing of several clinical assets.

·         Bradley T. Keller, Ph.D., vice president, research, is an experienced drug discovery scientist who was previously responsible for the LUM001 program at Searle/Pharmacia.

·         Alana B. McNulty, chief financial officer, joins in a consulting capacity, bringing more than 20 years of experience working with biotechnology companies in various senior finance, business development and operating roles.

·         Slava Gedulin, M.D., Ph.D., vice president, pharmacology, brings drug development experience from Amylin, where her extensive research was instrumental in bringing SYMLIN® and BYETTA® to market.

"Lumena's team has the vital experience necessary to successfully execute the clinical development of LUM001, including leadership in obtaining multiple drug approvals, expertise in pediatric drug development for orphan indications, as well as deep knowledge with this target for drug development," said John McKearn, managing director, RiverVest Venture Partners.

"LUM001 could dramatically impact patient health for a population in desperate need of more effective treatment options, and we are glad to be a part of this potentially game-changing company."

Lumena licensed LUM001 from Pfizer where it was originally evaluated at its Pharmacia legacy company as a cholesterol lowering drug. It has been extensively studied in 12 clinical trials in more than 1,400 subjects and is therefore positioned for rapid progress through late phase clinical development. Clinical studies have demonstrated that LUM001 can reduce serum bile acid levels and may be effective in managing symptoms in many patients with cholestatic liver disease.  Lumena plans to initiate a Phase II study of LUM001 in adults with Primary Biliary Cirrhosis (PBC), a rare chronic disease that presents with inflammation and damage of the bile ducts in the liver. The company also plans to initiate Phase II studies of LUM001 in children with Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC), rare congenital disorders that also present with cholestatic liver disease.

Lumena also licensed the patent rights and a package of clinical and non-clinical data from Sanofi for LUM002, a highly potent, selective inhibitor of ASBT.  LUM002 was previously in phase 1 development by Sanofi as a cholesterol lowering drug.  Lumena plans to complete the Phase I clinical program of LUM002 in healthy volunteers later this year as a first step in launching the development program for this compound. 

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