Amgen's Phase 2 Study of GDNF for Advanced Parkinson's Disease Fails to Meet Primary Endpoint; Six Months of Treatment Showed Biological Effect But No Clinical Improvement
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--June 28, 2004--Amgen
(Nasdaq:AMGN), the world's largest biotechnology company, today
announced that the Phase 2 study of its novel glial cell line-derived
neurotrophic factor, or GDNF, for the treatment of advanced
Parkinson's disease did not meet the primary study endpoint upon
completion of six months of the double-blind treatment phase of the
study. In the study, GDNF was safe and well-tolerated.
The Phase 2 randomized, double-blind placebo-controlled study
involved 34 patients with advanced Parkinson's disease who received
direct, continuous infusion of GDNF into the putamen, a region of the
brain known to be affected by Parkinson's disease. The primary
endpoint of the study was improvement of symptoms as defined by the
Unified Parkinson's Disease Rating Scale, a measurement tool that
assesses the status of patients suffering from Parkinson's disease.
Initial analysis of the preliminary data showed no clinical
improvement compared to placebo following six months of treatment,
despite evidence of alteration in brain function. All patients in the
trial are receiving GDNF in an open label extension study.
"We are currently analyzing the data to understand why this study
differs from the long-term improvement of the patients, who have been
treated with GDNF for close to three years in an ongoing open-label
study being conducted in the United Kingdom," said Beth Seidenberg,
M.D., chief medical officer and senior vice president, Amgen. "We are
committed to understanding if a different approach, including
evaluating a higher dose, may yield an outcome that is consistent with
the open label study."
Further details about the trial will be available when all data
analyses are complete. Data from the study are being submitted for
presentation at the annual meeting of the American Neurological
Association in October.
Amgen's recombinant GDNF protein is a duplicate of a naturally
occurring GDNF found in the central nervous system that promotes the
growth, regeneration and protection of specific nervous tissue.
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