Alnara Pharmaceuticals’ announces results of its international Phase 3 study of liprotamase

Oct 19 2009

Alnara Pharmaceuticals, a pharmaceutical company developing novel, non-systemic orally delivered protein therapeutics for the treatment of metabolic diseases, today announced results from an international, Phase 3 open-label, long-term safety study demonstrating the safety and nutritional benefits of liprotamase, a novel pancreatic enzyme replacement therapy (PERT) for patients with cystic fibrosis (CF). The nutritional parameters measured during the study showed nutritional maintenance relative to the U.S. population, which is a major finding for this historically challenged patient group.

This is the first time a long-term prospective study has been completed that shows that nutritional status is maintained in CF patients with respect to nutritional measures such as height, weight and vitamin levels over 12 months relative to the healthy U.S. population. The 12-month study is the largest and longest prospective nutritional and safety study ever completed for a PERT. This trial completes the liprotamase new drug application (NDA) clinical development program which has included approximately 600 subjects in various efficacy and safety studies.

Liprotamase is a novel, oral, non-porcine PERT designed to treat maldigestion, malabsorption and malnutrition as a result of exocrine pancreatic insufficiency associated with CF, chronic pancreatitis (CP), pancreatic cancer, pancreatectomy and other pancreatic conditions. Approximately 90 percent of CF patients receive PERT to improve nutritional status and bowel-related symptoms related to pancreatic insufficiency. Overall health in people living with pancreatic insufficiency is directly related to their nutritional status. For those living with CF, nutritional status is important in terms of respiratory health and has been directly tied to better lung function and survival.

These results were presented at the 23^rd Annual North American Cystic Fibrosis Conference in Minneapolis, Minnesota by Drucy Borowitz, M.D., principal investigator and professor of clinical pediatrics at State University of New York at Buffalo.

“There is a clear need for new and improved treatments that address the serious issues related to pancreatic insufficiency and these positive and clinically relevant results demonstrate that liprotamase has the potential to make a meaningful difference in the management of patients with cystic fibrosis,” said Alexey Margolin, Ph.D., president and CEO of Alnara Pharmaceuticals. “We are delighted with the data from this study and appreciate the support we have received from the Cystic Fibrosis Foundation Therapeutics, Inc., the CF community and everyone who worked to complete the liprotamase clinical program. This marks another important milestone for Alnara and we are now focused on filing a new drug application with the FDA for liprotamase as soon as possible, as well as preparing to launch this important therapy.”