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NIH Begins Phase II Clinical Testing of GPI 1485 for Parkinson's Disease

BALTIMORE, Feb. 23 /PRNewswire-FirstCall/ -- Guilford Pharmaceuticals Inc. (Nasdaq: GLFD) announced today that the National Institutes of Health (NIH) has initiated a Phase II clinical trial to test the Company's neuroimmunophilin ligand, GPI 1485, as part of a comprehensive program investigating novel neuroprotective therapies for Parkinson's disease. The NIH program is currently studying four promising investigational therapies, including GPI 1485. The study is being funded by the National Institute of Neurological Disorders and Stroke (NINDS), a division of the NIH. It will be carried out in cooperation with the Clinical Trials Coordinating Center at the University of Rochester and the Statistical Coordinating Center at Medical University of South Carolina and over 50 participating clinical sites in the United States and Canada. Bernard Ravina, M.D., is the Program Director for the trial at the Clinical Trials group of NINDS.

The NINDS selected GPI 1485 based upon strong, consistent preclinical data in animals and encouraging preliminary data from early human clinical testing. The research team has begun patient enrollment, and results of the trial are expected in the second half of 2005. Compounds that demonstrate neuroprotection in the Phase II program may be entered into Phase III studies.

Craig R. Smith, M.D., Chairman, President and Chief Executive Officer of Guilford, remarked, "Including the NIH study, there are currently three ongoing Phase II clinical trials of GPI 1485, including two protocols in Parkinson's disease and a separate study evaluating GPI 1485 for the treatment of post-prostatectomy erectile dysfunction. The scope of these trials will give us the opportunity to more comprehensively assess the therapeutic effects of GPI 1485, in which earlier testing has suggested its ability to retard the loss of dopamine transporters, a marker of disease progression, in patients with Parkinson's disease."

Dr. Ravina commented, "Parkinson's disease is a chronic, progressive, debilitating disorder which presently affects approximately one million Americans. Existing therapies focus only on controlling or minimizing the symptoms of the disease. NINDS intends to pursue novel treatment options focusing on neuroprotective therapies that may be able to slow disease progression. We believe that this important collaborative effort will help elucidate new and better potential treatments for the benefit of patients with Parkinson's disease."

About the NIH Clinical Trial Program in Parkinson's Disease
The Phase II trial is currently recruiting and will enroll 195 patients who are newly diagnosed with Parkinson's disease and are not taking anti- symptomatic medications, such as levodopa or dopamine agonists, for their disease. Patients in the trial will be randomized into one of three groups and receive either treatment with GPI 1485, treatment with another agent, or placebo. In all, over 50 sites are planned and enrollment is expected to take up to six months to complete. The treatment duration will be 12 months. For more information about the trial visit www.clinicaltrials.gov, or call 1-800-352-9424.

The primary outcome measure will be change in the total Unified Parkinson Disease Rating Scale (UPDRS) between either (1) baseline and 12 months, or (2) baseline and the time of sufficient disability to require symptomatic therapy. That is, if a patient completes the 12-month treatment period without requiring the addition of symptomatic medication such as levodopa or a dopamine agonist, the endpoint for that patient will be the change from baseline to 12 months. Alternatively, if a patient's condition worsens enough to require symptomatic therapy, the outcome measure for that patient will be the change from baseline to the last visit before starting medication, thus avoiding the confounding effect of symptomatic therapy on UPDRS.

Guilford will be responsible for supplying active drug and placebo for the study. If the results of the initial pilot study of GPI 1485 appear promising, NINDS may consider conducting Phase III clinical testing of GPI 1485. All financial and other obligations related to the conduct of the studies will be supported by NINDS. Further, NINDS and its investigators will provide Guilford access to data from the study, and will cooperate with Guilford in meeting Food and Drug Administration (FDA) requirements for submission of the results of the trial as part of a broader filing should this occur.

About GPI 1485 and Neuroimmunophilin Ligands
GPI 1485 is an investigational new drug that belongs to a class of small molecule compounds called neuroimmunophilin ligands. In preclinical experiments, neuroimmunophilin ligands have been shown to repair and regenerate damaged nerves without affecting normal, healthy nerves. Neuroimmunophilin ligands may have application in the treatment of a broad range of diseases, including: Parkinson's disease, spinal cord injury, brain trauma, and peripheral nerve injuries including post-prostatectomy erectile dysfunction.

About Parkinson's Disease
Parkinson's disease is a chronic, progressive degenerative disorder that involves a specialized region of the brain that controls muscle tone and coordination and affects over one million people in the United States. Most patients are affected in mid-life and usually develop hand tremors, muscle rigidity, and postural instability, among the many manifestations of the disease. The disease is caused by the degeneration of nerve cells that use dopamine as a chemical messenger. Treatment currently consists of administering drugs that increase the amount of dopamine in the affected regions of the brain or substitute for the lost dopamine. Unfortunately, there are no current treatments that can reverse, or even slow down, the progressive degeneration of the affected dopamine nerve cells.

Source: Guilford Pharmaceuticals Inc.

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