BioMarin Pharmaceutical Inc. has licensed from the University of California, San Francisco (UCSF) intellectual property covering compounds demonstrated to improve cystic fibrosis transmembrane conductance regulator (CFTR) protein functionality. BioMarin expects the lead compounds to undergo additional animal testing and optimization, with the goal of filing an IND in 2009.

Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin commented. "Dr. Verkman's laboratory has performed pioneering work in the field of CFTR modulation. The compounds licensed have been developed through a rigorous high-throughput screening process and have demonstrated a high degree of potency and specificity for the folding and activation of the abnormal CFTR protein. We look forward to accelerating the lead optimization activities in collaboration with Dr. Verkman and his colleagues so that we can take the very best of these molecules into clinical development."

"Over the last decade, considerable efforts and funding from the Cystic Fibrosis Foundation and the biotechnology industry have resulted in the development of therapeutics that have significantly extended and improved the lives of cystic fibrosis patients," said Dr. Alan Verkman, Professor of Medicine and Physiology and Director of the Cystic Fibrosis Research Development Program at the University of California, San Francisco. "Despite this progress, there is no therapeutic approved that addresses the underlying molecular defect in cystic fibrosis, and most patients succumb to respiratory failure as young adults. BioMarin has a strong track record of aggressively moving disease modifying therapeutics through clinical development, and I am very pleased that they have made a commitment to further this important work."

"We are pleased to add this program to our growing product development pipeline. The cystic fibrosis indication aligns well with our existing product portfolio and core competencies as it is a well-defined and relatively large orphan disease," said Jean-Jacques Bienaime, chief executive officer of BioMarin. "It has a clear clinical and regulatory path and will allow us to leverage our commercial infrastructure targeting specialists."

Cystic fibrosis is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. With an incidence of approximately 1/3,500 births, cystic fibrosis is the most common, lethal genetic disease in the US. CF affects an estimated 30,000 patients in the U.S. and approximately 40,000 patients outside the U.S. CFTR is a transmembrane protein that functions primarily as a chloride channel in the plasma membrane of epithelial cells. The most common mutation in cystic fibrosis, the deltaF508 mutation, causes the protein to be misfolded and through a cascade of events, leads to mucus buildup and ultimately, organ dysfunction and severe infections in the lungs. The median age of survival of a CF patient is 36.5 years.