SciClone, der Hersteller von CPX gab bekannt, dass die Zusammensetzung von CPX überarbeitet wurde und man nun plane die Phase 2 Studien in erweiterter Form durchzuführen. Nach dem herben Rückschlag könnte es ja nun doch noch klappen...

SciClone Pharmaceuticals Announces Successful Reformulation of CPX For Expanded Phase 2 Studies in Cystic Fibrosis Patients

Cystic Fibrosis Foundation Pledges Up to an Additional $1 Million in CPX Clinical Development Funding Pending Phase 2 Results

SAN MATEO, Calif., Nov. 10 /PRNewswire/ -- SciClone Pharmaceuticals (Nasdaq: SCLN) today announced at the Cystic Fibrosis Foundation's (CFF) 14th Annual North American Cystic Fibrosis Conference in Baltimore that CPX, the Company's lead protein-repair therapy for cystic fibrosis (CF), has been successfully reformulated as an oral administration to address the unique pharmacokinetic characteristics of CPX in the environment of the CF patient. SciClone said it plans to initiate expanded U.S. Phase 2 CPX studies, working closely with clinical investigators in the CFF's Therapeutics Development Network.

The CFF, which already has granted SciClone over $500,000 for the development of CPX, has pledged up to an additional $1 million in clinical funding to SciClone, pending the outcome of results in CF patients in the Company's upcoming Phase 2 studies.

Attempting to replicate in patients the protein-repair results demonstrated by the U.S. National Institutes of Health in preclinical studies, an initial Phase 2 study completed earlier this year showed CPX to be safe and well tolerated. However, the absorption characteristics of CPX in CF patients precluded attainment of the sustained CPX plasma concentrations that are necessary to evaluate efficacy.

The new formulation, which is an oral suspension, takes advantage of the solubility characteristics of CPX and has shown promising absorption characteristics and much higher blood levels than achieved previously in multiple animal model systems, and will soon be tested in healthy volunteers and CF patients.

"Our initial Phase 2 study yielded valuable information about the unique drug absorption characteristics of CPX in the CF patient, not the least of which pointed us toward the new formulation," said Donald R. Sellers, SciClone's President and Chief Executive Officer. "CPX is one of the first drugs studied in CF patients that attempts to address the underlying defective protein-related causes of the disease rather than just address the symptoms, and one of the few CF drugs in a phase 2 program. We hope this new formulation of CPX will allow us to validate in CF patients the repair of the defective CFTR protein demonstrated by the National Institutes of Health in preclinical studies. We remain absolutely committed to these patients and would like to acknowledge the CFF for its critical support in almost every area of CPX development."

"We continue to regard CPX as one of the most promising therapies for CF patients and we are gratified that SciClone has reformulated CPX on such an aggressive schedule," said Robert J. Beall, Ph.D., President and Chief Executive Officer of the Cystic Fibrosis Foundation. "SciClone has proven again to be a valuable ally in our mission to help CF patients. We offer our continuing support in the hope of mutual success."

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 29.

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 U.S. 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 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 specialty pharmaceutical company that develops and commercializes novel medicines for the treatment of a broad range of the world's most serious diseases. The Company is currently targeting malignant melanoma, liver cancer, hepatitis B, hepatitis C, HIV, drug-resistant tuberculosis 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 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, particularly its Annual Report on Form 10-K for the year ended December 31, 1999.