Die Phase 2 Studien von CPX haben gezeigt, dass mit der bisherigen Zusammensetzung von CPX bei CF-Patienten nicht genügend hohe und damit wirksame Blutkonzentrationen erreicht werden können. Die Zusammensetzung muss daher vor einer neuen Studie erst überarbeitet werden.

SciClone Initiates Phase 2 Trial of CPX in Cystic Fibrosis

SciClone Pharmaceuticals Moves Forward With Development of CPX For Cystic Fibrosis

SAN MATEO, Calif., Feb. 15 /PRNewswire/ -- SciClone Pharmaceuticals (Nasdaq: SCLN) 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.

The Cystic Fibrosis Foundation was established in 1955 to assure the development of the means to cure and control cystic fibrosis and improve the quality of life for those with the disease. Press releases and additional information from the Cystic Fibrosis Foundation are available on the Internet at www.cff.org.

The mission of the Cystic Fibrosis Foundation is to assure the development of the means to cure and control cystic fibrosis and to improve the quality of life for those with the disease. The Therapeutics Development Network was created in 1998 to capitalize on the increasing number of discoveries being made about the basic defect in CF. By establishing specialized clinical care centers, researchers can seize these opportunities to intervene in the disease process through new CF treatments. Clinical research underway through the network includes early-phase evaluations of several novel CF therapies.

SciClone Pharmaceuticals is a global biopharmaceutical company that acquires, develops and commercializes specialist-oriented drugs for treating chronic and life-threatening diseases, such as cancer, hepatitis B, hepatitis C and cystic fibrosis. Press releases and corporate information from SciClone Pharmaceuticals are available on the Internet at www.sciclone.com or by calling the Company's Investor Relations Department at 800-724-2566. SciClone's common stock is listed on The Nasdaq National Market(R) under the symbol SCLN.

The information in this press release includes certain forward-looking statements concerning the Company's current expectations regarding future events, including the ongoing and prospective development and commercialization of an improved formulation of CPX as a potential protein-repair therapy for cystic fibrosis and future benefits to the Company, its shareholders, and patients from development and commercialization of CPX. Due to the nature of product development and the regulatory approval process, the forward-looking statements contained in this press release are subject to risks and uncertainties, including those reflected in the Company's filings with the Securities and Exchange Commission.