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Neurologix Commences its Phase 2 Clinical Trial of Novel Gene Transfer Approach for Treatment of Parkinson's Disease

FORT LEE, N.J.--(BUSINESS WIRE)--Jan. 21, 2009--Neurologix, Inc. (OTCBB:NRGX), a biotechnology company engaged in the development of innovative gene therapies for the brain and central nervous system, announced today that it has initiated its Phase 2 clinical trial for the treatment of advanced Parkinson's disease. The first trial participants have undergone surgery at multiple institutions and additional subjects are currently being enrolled.

The purpose of the trial is to validate the safety and efficacy of Neurologix's gene transfer therapy, a novel non-dopaminergic approach to restore motor function in Parkinson's patients who are sub-optimally responsive to available drug therapy. Neurologix's approach is to reestablish the production of GABA (gamma-aminobutyric acid), the major brain inhibitory neurotransmitter that helps "quiet" excessive neuronal firing and has been determined to be deficient in patients in the advanced stages of Parkinson's disease.

John E. Mordock, President and Chief Executive Officer of Neurologix, stated, "Initiating this Phase 2 clinical trial represents a significant milestone. We expect to enroll 40 subjects across six to eight leading U.S. academic research centers, with completion of enrollment expected during the second half of 2009."

In Parkinson's disease there is degeneration of many cells in the central nervous system including those that produce dopamine, which leads to a downstream deficiency in GABA signaling in areas of the brain that regulate movement. Most current therapies and research approaches target dopamine. Mr. Mordock commented, "In contrast, our preclinical and clinical research suggests that directly targeting GABA production rather than dopamine replacement may be a more effective way of improving brain function in late-stage Parkinson's disease while also avoiding the known therapeutic limitations and complications associated with the over-production of dopamine."

The Co-Chairmen of the trial Steering Committee are Dr. Andrew Feigin, Director of the Neuroscience Experimental Therapeutics Research Program at the Feinstein Institute of Medical Research of the North Shore-Long Island Jewish Health System, and Dr. Peter LeWitt, a neurologist who directs the Parkinson's Disease and Movement Disorders Program at Henry Ford Hospital in Southfield, Michigan.

"Based on the encouraging functional and imaging results seen in the Phase 1 study of this innovative approach to improving Parkinson's disease, we are extremely excited to be part of this study," said Dr. Feigin.

Dr. LeWitt added, "The start of this clinical trial provides hope to the patient population which has had a longstanding need for new treatment options."

The scientific underpinnings of Neurologix's approach have undergone rigorous peer review resulting in highly cited articles in Nature Genetics and Science by the company's co-founders, Drs. Matthew During and Michael Kaplitt. Moreover, the current trial follows a successful Phase 1 study, as published in the Lancet and Proceedings of the National Academy of Sciences, USA, in which 12 subjects completed the study showing no related serious adverse events and significant functional benefit with supportive imaging data.

Phase 2 Clinical Trial Design

As previously announced, 20 participants will receive an infusion of the gene-based treatment bilaterally via a catheter temporarily placed by stereotactic surgery in each participant's subthalamic nucleus (STN), a deep brain structure that is the main target of surgery to treat Parkinson's disease. The other 20 participants will receive sterile saline solution into a partial thickness burr hole made into the skull, with no brain infusion. Trial participants will be assessed for treatment effects by standardized Parkinson's disease ratings at multiple time points post-procedure. The primary endpoint for the trial will be a clinical assessment of motor function at 6 months using the Unified Parkinson's Disease Rating Scale (UPDRS). All participants in the trial will also be monitored for safety for 12 months following the gene transfer procedure. If the primary endpoint is met following the analysis of 6 month data, then the sham-control participants will be offered the opportunity to crossover into an open label study of the Neurologix gene transfer therapy if they continue to meet all entry, medical and surgical criteria.

For details about participating in the clinical trial, please visit the following link: http://clinicaltrials.gov/ct2/show/NCT00643890?term=neurologix&rank=1. Details about trial participation are also available at http://www.pdtrials.org/en/browse/all/view/241.

About the Neurologix Gene Transfer Approach to Parkinson's Disease

In Parkinson's disease, patients lose dopamine-producing brain cells, resulting in substantial reductions in the activity and amount of GABA (gamma-aminobutyric acid). This reduction in GABA causes a dysfunction in brain circuitry responsible for coordinating movement. GABA is made by a gene called glutamic acid decarboxylase, or GAD.

Neurologix's gene transfer approach to Parkinson's disease seeks to restore GABA -- and thus improve the patient's motor control -- by inserting the GAD gene back into an area of the brain called the subthalamic nucleus, a key regulatory center for movement.

About Neurologix

Neurologix, Inc. (NRGX.OB) is a clinical-stage biotechnology company dedicated to the discovery, development, and commercialization of life-altering gene transfer therapies for serious disorders of the brain and central nervous system (CNS). Neurologix's therapeutic approach is built upon the groundbreaking research of its scientific founders and advisors, whose accomplishments have formed the foundation of gene therapy for neurological illnesses. Current programs of the company address such conditions as Parkinson's disease, epilepsy and Huntington's chorea, all of which are large markets not adequately served by current therapeutic options. For more information, please visit the Neurologix website at http://www.neurologix.net.

Cautionary Statement Regarding Forward-looking Statements

This news release includes certain statements of the Company that may constitute "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and which are made pursuant to the Private Securities Litigation Reform Act of 1995. These forward-looking statements and other information relating to the Company are based upon the beliefs of management and assumptions made by and information currently available to the Company. Forward-looking statements include statements concerning plans, objectives, goals, strategies, future events, or performance, as well as underlying assumptions and statements that are other than statements of historical fact. When used in this document, the words "expects," "promises," "anticipates," "estimates," "plans," "intends," "projects," "predicts," "believes," "may" or "should," and similar expressions, are intended to identify forward-looking statements. These statements reflect the current view of the Company's management with respect to future events. Many factors could cause the actual results, performance or achievements of the Company to be materially different from any future results, performance or achievements that may be expressed or implied by such forward-looking statements, including, but not limited to, the following:

  • The Company is still in the development stage and has not generated any revenues. From inception through September 30, 2008, it incurred net losses and negative cash flows from operating activities of approximately $32.9 million and $26.5 million, respectively. Management believes that the Company will continue to incur net losses and cash flow deficiencies from operating activities for the foreseeable future. Because it may take years to develop, test and obtain regulatory approval for a gene-based therapy product before it can be sold, the Company likely will continue to incur significant losses for the foreseeable future. Accordingly, the Company may never be profitable and, if it does become profitable, it may be unable to sustain profitability.
  • At September 30, 2008, the Company had cash and cash equivalents of approximately $20.1 million, which management believes will be sufficient to fund the Company's operations through at least March 31, 2010. The Company does not know whether additional financing will be available when needed, or if available, will be on acceptable or favorable terms to it or its stockholders.
  • The Company will need to conduct future clinical trials for treatment of Parkinson's disease using the Company's NLX technology. If the trials prove unsuccessful, future operations and the potential for profitability will be materially adversely affected and the business may not succeed.
  • There is no assurance as to when, or if, the Company will be able to successfully receive approval from the Food and Drug Administration (FDA) on its Investigational New Drug Application to commence a Phase 1 clinical trial for the treatment of epilepsy
  • There is no assurance as to when, of if, the Company will be able to successfully complete the required preclinical testing of its gene therapy for the treatment of Huntington's disease to enable it to file an Investigational New Drug Application with the FDA for permission to begin a Phase 1 clinical trial or that, if filed, such permission will be granted.

Other factors and assumptions not identified above could also cause the actual results to differ materially from those set forth in the forward-looking statements. Additional information regarding factors that could cause results to differ materially from management's expectations is found in the section entitled "Risk Factors" in the Company's 2007 Annual Report on Form 10-KSB. Although the Company believes these assumptions are reasonable, no assurance can be given that they will prove correct. Accordingly, you should not rely upon forward-looking statements as a prediction of actual results. Further, the Company undertakes no obligation to update forward-looking statements after the date they are made or to conform the statements to actual results or changes in the Company's expectations.

CONTACT: Neurologix, Inc.
Marc Panoff, 201-592-6451
Chief Financial Officer, Treasurer and Secretary
Kureczka/Martin Associates
Joan Kureczka, 415-821-2413

Source: Neurologix, Inc.

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