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CPX - der Durchbruch in der CF-Behandlung?!
Eine Pressemeldung betreffend CPX lässt die Hoffnung aufkeimen,
dass für DeltaF508 CF'ler in einer nicht zu fernen Zukunft
eine lebenslange Behandlung verfügbar sein wird, die zwei wesentliche
Funktionsstörungen des CFTR beheben kann (keine Gentherapie!).
Die Ergebnisse der vorklinischen Studien wurden auf der im Oktober
1997 stattfindenen CF-Konferenz in Nashville/ Tennessee (USA) verkündet. Danke an Ray Goldstein, der
die Meldung auf Cystic-L geposted hat. Hier folgt der englische Originaltext. Subj: SciClone Reports Breakthrough Preclinical Discovery involving
CPX at Annual Cyst SAN MATEO, Calif., Oct. 25 /PRNewswire/ -- For the first time,
preclinical data demonstrate that a new drug, CPX, is the only cystic
fibrosis (CF) drug in clinical development that can correct the
two fundamental problems caused by the most common genetic defect
in CF patients -- abnormal trafficking and impaired chloride ion
transport. The new preclinical findings were presented by Harvey B. Pollard,
MD, PhD at the Eleventh Annual North American Cystic Fibrosis Conference
in Nashville. SciClone Pharmaceuticals, Inc. (Nasdaq: SCLN) is conducting
a multi-center Phase 1 clinical study in CF patients receiving CPX,
an orally administered compound that targets the biochemical abnormality
in CF, the malfunctioning cystic fibrosis transmembrane conductance
regulator (CFTR) protein. "These significant new preclinical data should generate considerable
excitement within the CF research community," said Bonnie W.
Ramsey, MD, Professor of Pediatrics at Seattle's Children's Hospital
Medical Center, in her keynote presentation, "New Clinical
Developments in CF: From the Test Tube to the Bedside." "Together
with recent amendments which relax the entry criteria for SciClone's
Phase 1 trial, Dr. Pollard's new data are likely to accelerate the
company's CPX clinical program." The new data show that CPX promotes trafficking of the delta F508
mutant CFTR protein, the most common genetic mutation in CF patients.
Trafficking refers to the ability of the CFTR protein to traverse
the cell cytoplasm and reach the proper location on the cell membrane.
Previously published preclinical data from the National Institutes
of Health (NIH) have established that CPX also interacts directly
with the delta F508 mutant CFTR and stimulates chloride ion transport.
The new preclinical data demonstrate that CPX is the only CF drug
in clinical development that can address the two functional cellular
defects in CF patients caused by the delta F508 mutant CFTR. "The Cystic Fibrosis Foundation provided substantial support
for Dr. Pollard's research and is very excited by his new data,"
said Robert J. Beall, PhD, President and CEO of the Cystic Fibrosis
Foundation. "These new data strengthen our belief that CPX
could lead to a whole new strategy for fighting CF and extending
the lives of patients. There is now further preclinical evidence
that CPX targets the biochemical abnormality at the root cause of
CF, unlike currently available therapies that only address symptoms
of the disease," he added. In its ongoing multi-center Phase 1 clinical study in the U.S.,
SciClone hopes to confirm in CF patients Dr. Pollard's preclinical
findings. This Phase 1 study for CPX is the first to attempt to
measure both sweat chloride and nasal epithelial transmembrane potential
difference (NEPD) in a multi- center trial. These two surrogate
markers for efficacy might provide an early indication of CPX efficacy.
The Cystic Fibrosis Foundation supported SciClone in its Investigational
New Drug Application (IND) filing with the Food and Drug Administration
(FDA) to gain approval to begin testing of CPX directly in CF patients. "Dr. Pollard's preclinical findings reinforce our belief
that CPX can become a leading life-long treatment for CF patients,"
said David A. Karlin, MD, SciClone's Vice President and Medical
Director. "CPX could well be the long-awaited breakthrough
in our fight against this terrible disease." SciClone licensed exclusive rights to CPX from the NIH. In April
1997, the Company obtained Orphan Drug status for CPX from the FDA.
Earlier this month, the FDA awarded SciClone a $100,000 Orphan Drug
Grant for Phase 1 development of CPX as a treatment for CF, the
Company's first federal funding for its drug development activities. CF is a fatal genetic disorder that occurs in approximately one
of every 3,300 live births in the U.S. Today, approximately 55,000
patients in the U.S. and Europe are afflicted with the disease.
The basic genetic defect in CF cells is the faulty transport of
chloride ions across epithelial cell membranes. This faulty transport
causes the body to produce abnormally thick, sticky mucus which
clogs the lungs and leads to fatal infections. In various in vitro
studies performed at the NIH and published by Dr. Pollard (formerly
with the NIH) and Kenneth Jacobson, PhD (currently with the NIH)
involving explanted epithelial tissue from the respiratory tracts
of CF patients and cells in culture, CPX was effective in stimulating
the transport of chloride ions. Dr. Pollard is currently Professor
and Chair of the Departments of Anatomy, Cell Biology and Physiology
at the Uniformed Services University of the Health Sciences in Bethesda. The statements made in this press release contain certain forward-looking
statements that involve a number of risks and uncertainties, including,
without limitation, those associated with the success of clinical
trials of CPX as a life-long treatment for CF. Actual events or
results may differ from the Company's expectations and beliefs.
Factors which may affect the actual results achieved by the Company
include future actions by the FDA, or equivalent regulatory authorities
in foreign countries, results of pending or future CPX clinical
trials, as well as risk factors listed from time to time in SciClone's
SEC reports, including, but not limited to, its Annual Report on
Form 10-K. SciClone Pharmaceuticals, Inc. is an international biopharmaceutical
company that acquires, develops and commercializes specialist-oriented
drugs for treating chronic and life-threatening diseases for which
there are no adequate treatment modalities, including hepatitis
B, hepatitis C, cancer, immune system disorders and cystic fibrosis. Press releases and corporate information from SciClone Pharmaceuticals,
Inc. are available on the Internet at www.sciclone.com and by fax
at 800-996-7256. Additional background information regarding cystic
fibrosis, CPX and the centers participating in the study is available
on the Internet at the Cystic Fibrosis Foundation's website (www.cff.org)
and from SciClone at 800-INFO-CPX (800-463-6279). SOURCE SciClone Pharmaceuticals Inc. |
