SciClone Awarded $200,000 FDA Research Grant For Potential Cystic Fibrosis Therapy
09:22 a.m. Oct 07, 1998 Eastern

SAN MATEO, Calif., Oct. 7 /PRNewswire/ -- SciClone Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has awarded the company a $200,000 Orphan Drug Grant for Phase 2 trials of CPX, its proprietary drug for cystic fibrosis (CF). This is the second consecutive year the FDA has awarded SciClone the maximum amount available for CPX clinical development. Last year, the FDA awarded SciClone a $100,000 grant for Phase 1 development of CPX, and designated CPX as an Orphan Drug.

CPX is an orally administered drug that targets the underlying protein dysfunction in CF. SciClone's ongoing CPX Phase 2 trial is designed to evaluate the safety, initial efficacy and optimal dosage of CPX. Fifty adult CF patients will receive an oral dose of CPX or placebo up to four times daily for one week at four leading CF centers in the U.S. The primary endpoints of the trial are changes in sweat chloride content and changes in nasal epithelial transmembrane potential difference (NEPD), two surrogate markers for efficacy in CF. The measurement of sweat chloride content is currently the standard diagnostic test for CF.

"We are extremely pleased to receive this second FDA grant for CPX," said Donald Sellers, SciClone's President and Chief Executive Officer. "It is a continuing indication of how seriously the FDA regards CF and how firmly they believe CF patients need new treatment options."

CF is the most common fatal genetic disorder in the U.S. It 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 CF. 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. The median age of survival for a person with CF is 31 years.

CF is caused by a defect in the cystic fibrosis transmembrane regulator (CFTR) protein, which transports chloride ions across the membranes of epithelial cells lining several organs in the body. The most debilitating consequence of the defective CFTR protein occurs in the lungs of CF 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.

CPX is an orally available, non-toxic drug which can bind to defective CFTR protein. In preclinical studies sponsored by the National Institutes of Health, CPX repaired the two key defects seen with CFTR: (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 CPX has the potential to prevent the production of abnormal mucus and other symptoms of CF in patients with the most common genetic defect, called the delta F508 mutation.

SciClone Pharmaceuticals is a global biopharmaceutical company that acquires, develops and commercializes specialist-oriented drugs for treating chronic and life-threatening diseases, such as hepatitis B, hepatitis C, cystic fibrosis, cancer and immune system disorders. Press releases and corporate information from SciClone Pharmaceuticals are available on the Internet at www.sciclone.com and by calling the Company's Investor Relations Department at 800-724-2566. SOURCE SciClone Pharmaceuticals

Copyright 1998, PR Newswire 

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