To: Sundanz who wrote (413 ) 2/15/2000 1:25:00 PM From: Skywatcher Read Replies (1) | Respond to of 1137
SciClone Pharmaceuticals Moves Forward With Development of CPX For Cystic Fibrosis SAN MATEO, Calif., Feb. 15 /PRNewswire/ -- SciClone Pharmaceuticals (Nasdaq: SCLN - news) today announced plans to move forward with the next stage of its phase 2 development program for CPX, a novel PRT (protein repair therapy) for cystic fibrosis (CF). SciClone, working closely with scientists and clinical investigators in the Cystic Fibrosis Foundation's Therapeutics Development Network, plans to study a new formulation of CPX, based on results of its initial phase 2 study. In the recently concluded initial phase 2 study of CPX, the unique drug absorption characteristics of CF patients precluded measurement of sustained blood concentrations of CPX necessary to evaluate efficacy in this study. The Company's new oral formulation program is intended to address this issue. In the multi-center, multiple dose trial, CPX was safe and well tolerated. ''Drugs behave differently in CF patients from the way they do in others,'' said Bonnie W. Ramsey, M.D., Director of the Cystic Fibrosis Foundation's Therapeutics Development Network (TDN). ''TDN investigators are excited to continue working with SciClone on phase 2 development of CPX with new protocols and a new formulation that addresses the unique environment of the CF patient.'' ''CPX is one of the most promising therapies for CF patients,'' said Robert J. Beall, Ph.D., President and Chief Executive Officer of the Cystic Fibrosis Foundation (CFF). ''We share SciClone's commitment to the most rapid development of this drug that is possible. We continue to support that development both scientifically and financially.'' ''We know from the preclinical studies conducted at the National Institutes of Health that CPX can repair the protein-associated defect that causes CF. Our phase 2 program is designed to learn how to replicate that effect in patients,''said Donald R. Sellers, SciClone's President and Chief Executive Officer. ''We answered key questions with our initial phase 2 study, and we are continuing our program in close collaboration with the Cystic Fibrosis Foundation.'' Cystic fibrosis affects approximately 70,000 children and young adults worldwide, including 30,000 in the U.S. and 30,000 in Europe. Currently, there is no cure for the disease. Approved therapies for CF treat only the symptoms of the disease, such as lung infections. The average annual cost of care for a CF patient is $50,000. According to the Cystic Fibrosis Foundation's National Patient Registry, only one half of all individuals with CF live to the age of 31. Cystic fibrosis, the most common fatal genetic disease among Caucasians, is caused by a defect in the Cystic Fibrosis Transmembrane Regulator (CFTR) protein. Normally, the CFTR protein transports chloride ions across the membrane of epithelial cells lining several organs in the body such as the lungs and the pancreas. The most debilitating consequence of the defective CFTR protein occurs in the lungs of cystic fibrosis patients, where insufficient chloride transport prevents water from exiting epithelial cells. This causes the lungs to produce abnormally thick, sticky mucus that clogs the airways and leads to fatal lung infections. In preclinical studies sponsored by the National Institutes of Health, CPX repaired the basic CFTR protein-associated defect that causes cystic fibrosis in most patients: (1) it improved the impaired chloride ion transport and (2) it enabled the defective CFTR to reach the epithelial cell membrane (a process called ''trafficking''). SciClone believes that an improved formulation of CPX has the potential to prevent all of the complications of cystic fibrosis, including the production of abnormal mucus. OK...so we're selling off on the basis that the drug didn't absorb well in to the blood...no reactions...so now we start with oral ingestion...hoping something else comes up news wise, but still on track. chris