Funding to support clinical development of treatments for rare cholestatic liver diseases and nonalcoholic steatohepatitis

SAN DIEGO – March 11, 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 that it has secured $45 million in Series B financing. New Enterprise Associates (NEA) led the financing, with Adage Capital Management and RA Capital Management participating and joining existing investors Pappas Ventures, RiverVest Venture Partners and Alta Partners. Founded in 2011 by Pappas Ventures, Lumena will use the funding to advance the clinical development of LUM001, its lead product candidate, for the treatment of rare cholestatic liver disease in pediatric and adult patients, as well as LUM002 for the treatment of nonalcoholic steatohepatitis (NASH).
“Lumena has made significant progress in the three years since the company was founded, advancing our lead candidate through development in multiple Phase 2 clinical trials in patient populations with rare cholestatic liver diseases and significant medical needs,” said Mike Grey, president and CEO of Lumena Pharmaceuticals. “This funding will support the continued development of LUM001 in our ongoing global clinical programs, as well as a planned Phase 2 clinical trial of LUM002 to be initiated later this year in patients with NASH, a significant and growing patient population for which there are currently no approved treatments.”

LUM001 and LUM002 are both inhibitors of the apical sodium-dependent bile acid transporter (ASBT), which is primarily responsible for recycling bile acids from the intestine to the liver. Blocking bile acid transport with ASBT inhibitors reduces bile acid absorption and has the potential to slow disease progression, improve liver function and enhance the quality of life for patients suffering from rare cholestatic liver diseases and NASH. “We see a lot of promise in the therapeutic approach of inhibiting ASBT for the treatment of both rare cholestatic liver diseases as well as the increasingly common NASH,” said Ed Mathers, partner, NEA, who will join Lumena’s board of directors in conjunction with the financing. “The Lumena management team has vast experience in orphan and pediatric clinical development, deep insights into the drug target and its role in disease mechanisms, and has successfully brought multiple drugs to market—in short, we believe the team has the critical experience needed to drive LUM001 and LUM002 efficiently through clinical and commercial development.”

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 four 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 LUM002

Lumena’s second product candidate, LUM002, is a novel, once-daily, orally-administered, highly-potent and selective inhibitor of ASBT being developed for the treatment of NASH, a common, often “silent” liver disease characterized by fat deposits in the liver, leading to inflammation and significant fibrosis. Lumena has completed Phase I safety studies in healthy volunteers and in a cohort of patients with metabolic disease, which showed that LUM002 administration leads to a decrease in serum total cholesterol and LDL-cholesterol. In animal studies, LUM002 treatment resulted in increased plasma glucagon-like peptide 1, which stimulates insulin release in the pancreas, and decreased plasma hemoglobin A1c, a measure of blood sugar levels over time. This suggests that ASBT inhibitors such as LUM002 may provide benefits in patients with NASH. Lumena plans to initiate a Phase 2 clinical trial of LUM002 in patients with NASH in the second half of 2014.

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, including the proceeds from the Series B financing.

Contact:
Heidi Giesing Chokeir
Canale Communications
(619) 849-5377(619) 849-5377
heidi@canalecomm.com