SciClone Expands Protein-Repair Program for Cystic Fibrosis
SAN MATEO, Calif., Oct. 13 /PRNewswire/ -- SciClone Pharmaceuticals (Nasdaq: SCLN - news) today announced that it has acquired DAX, a potential second-generation ''protein-repair'' therapy for cystic fibrosis discovered at the National Institutes of Health (NIH). DAX is a small molecule and an analog of the Company's lead cystic fibrosis drug, CPX, which SciClone also acquired from the NIH. SciClone is currently conducting CPX Phase 2 clinical trials in the U.S.,
results from which are expected to be reported in the first quarter of next year. Like CPX, DAX targets the protein-associated defect that causes cystic fibrosis in over 70 percent of patients.
In preclinical studies conducted at the NIH, both CPX and DAX repaired the basic CFTR protein-associated defect that causes cystic fibrosis in over 70 percent of patients: (1) improving the impaired chloride ion transport and (2) enabling the defective CFTR to reach the epithelial cell membrane (a process called ''trafficking''). SciClone believes that both CPX and DAX have the potential to prevent all of the life-threatening complications of cystic fibrosis, including the production of abnormal mucus.
''We have a long-standing commitment to cystic fibrosis patients, and we believe protein-repair therapy, as discovered by the NIH, is the most promising new approach,'' said Donald R. Sellers, SciClone's President and Chief Executive Officer. ''CPX could be the first breakthrough for patients and that justifies building a pipeline behind it. As we have throughout our CPX development program, we will work closely with the FDA, the Cystic Fibrosis Foundation and the Boomer Esiason Foundation to develop DAX.''
Preclinical development of DAX is underway. SciClone plans to request fast-track designation from the FDA for its DAX program. The Company also intends to seek orphan drug designation and development grants for DAX. A recently issued U.S. patent covers the composition of matter of DAX.
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 cystic fibrosis. Approved therapies
for cystic fibrosis treat only the symptoms of the disease, such as lung infections. The average annual cost of care for a cystic fibrosis patient is $50,000. According to the Cystic Fibrosis Foundation's
National Patient Registry, only one half of all individuals with cystic fibrosis live to the age of 31.
Cystic fibrosis, the most common fatal genetic disease in the U.S., 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.
chris |
