SciClone Initiates Phase 2 Trial of CPX in Cystic Fibrosis
02:41 p.m Sep 17, 1998 Eastern

SAN MATEO, Calif., Sept. 17 /PRNewswire/ -- SciClone Pharmaceuticals (Nasdaq: SCLN) today announced the start of patient dosing in a Phase 2 clinical trial of the Company's oral drug candidate, CPX, for the treatment of cystic fibrosis (CF). The trial will evaluate CPX's efficacy in repairing the underlying protein dysfunction in CF. In addition, the trial is designed to identify the optimal therapeutic dose of CPX.

This randomized, double-blind, placebo-controlled, multiple ascending dose Phase 2 trial is designed to treat 50 CF patients with mild to moderately severe CF at four leading CF centers. Patients will receive an oral dose of CPX or placebo up to four times daily for one week. 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.

In June 1998, the Company announced results from its single oral ascending dose Phase 1 trial of CPX in 37 patients with CF. This trial demonstrated that oral dosing of CPX produced levels of CPX in the blood that are expected to be required for therapeutic effect. The trial also demonstrated CPX to be safe at all doses tested.

"It is exciting to work with leading CF clinicians who share our belief that CPX is the most promising drug therapy for CF," said David A. Karlin, M.D., Vice President and Medical Director of SciClone. "Because CPX targets the underlying causes of CF, it was possible to design a trial to measure changes in physiological processes believed responsible for CF, such as the transport of chloride ions across epithelial cell membranes. In the scientific community, the trial endpoints of sweat chloride and NEPD are considered to be valid surrogate measures for efficacy.

"Much as diabetic patients need insulin," continued Dr. Karlin, "CF patients need a drug which can return the underlying defective protein to normal function, not just address the symptoms, and which can be administered safely for the ongoing maintenance of normalcy."

Cystic fibrosis (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. In the lungs, insufficient chloride transport prevents water from exiting epithelial cells, causing the body to produce abnormally thick, sticky mucus that clogs the airways and leads to fatal infections. Approximately 70% of CF patients have the delta F508 mutation of the gene coding for the CFTR protein. The delta F508 mutation causes the CFTR protein to be defective. All patients enrolled in SciClone's Phase 2 trial will have the delta F508 mutation.

In preclinical studies sponsored by the National Institutes of Health, CPX repaired the two key defects in the CFTR protein: (1) abnormal trafficking and (2) impaired chloride ion transport. CPX is an orally available, non-toxic compound. The Company believes that CPX has the potential to prevent the production of abnormal mucus in CF patients with the delta F508 mutation. CPX appears to bind with the defective CFTR protein and enable it to reach epithelial cell membranes and permit the CFTR to transport chloride ions.

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, Inc.

Copyright 1998, PR Newswire 

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