Neurologix, Inc. (OTC Bulletin Board: NRGX), announced that new details of the company's landmark, randomized, double-blind Phase 2 clinical trial of NLX-P101, its investigational gene therapy for advanced Parkinson's disease (PD), were presented during a symposium at the 2nd World Parkinson Congress in Glasgow, Scotland. Co-principal investigator of the trial, Dr. Peter Lewitt, Director of the Parkinson's Disease and Movement Disorders program at Henry Ford Hospital, reported that, in addition to statistically significant improvements over the entire blinded study period in the off-medication motor scores between the treated and sham groups on the Unified Parkinson's Disease Rating Scale (UPDRS) Part 3 (Motor section), 75 percent of patients in the treatment group were considered to have had a meaningful response to NLX-P101 (i.e., a five point or greater reduction in the off-medication UPDRS motor scores), which is comparable to the response rate of 71 percent in a recent study of Deep Brain Stimulation (Weaver FM, Follett K, Stern M, et al. Bilateral deep brain stimulation vs. best medical therapy for patients with advanced Parkinson's disease: a randomized controlled trial. JAMA 2009; 301:63-73) (see also Central Nervous System Diseases).
Dr. Lewitt also emphasized that results showed a positive safety profile for NLX-P101, with no serious adverse events related to the gene therapy or surgical procedure reported. Although details regarding the study methodology were also provided, such as catheter placement in the subthalamic nucleus (STN), specific quantitative outcomes were not discussed during the presentation, as these will be presented in a peer-reviewed publication, currently under review.
Earlier this year, Neurologix disclosed top-line results for this Phase 2 trial, announcing that study participants who received NLX-P101 experienced statistically significant and clinically meaningful improvements in off-medication motor scores compared to control subjects who received sham surgery. Patients enrolled in the trial had moderate to advanced PD and were not adequately responsive to current therapies.
"We are very pleased that Dr. Lewitt had the opportunity to provide more information about our study methodology and some further details as to the nature of the improvements seen in patients treated in our Phase 2 study. We remain confident in our study results, which we feel confirm that NLX-P101 has great potential to offer an important new therapy for patients with Parkinson's disease," said Clark A. Johnson, President and Chief Executive Officer of the Company.
Mr. Johnson added, "We continue to believe that the best venue for the full disclosure of our Phase 2 results would be in a respected peer-reviewed medical or scientific journal. All of the major details of our previous pre-clinical and clinical studies have been published in leading journals, including Science, The Lancet and the Proceedings of the National Academy of Sciences. We believe that the credibility provided by these publications enhance the value of our product and of our company."
Neurologix, Inc. is a clinical-stage biotechnology company dedicated to the discovery, development and commercialization of gene transfer therapies for serious disorders of the brain and central nervous system (CNS).Neurologix's investigational AAV (adeno-associated virus) vector gene therapy, NLX-P101, is a novel, non-dopaminergic approach that uses an inhibitory gene (glutamic acid decarboxylase or "GAD") to selectively alter the neural circuitry affected in PD and, thereby, normalize brain physiology. Neurologix's technology is the only gene therapy strategy currently in development which bypasses the dopamine system.
Neurologix and Gene Therapy - A Novel 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), the major inhibitory neurotransmitter in the brain. This contributes to an abnormal increase in activity of the STN of the brain, a key regulatory center for movement, and 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 therapy approach to PD aims to reset the overactive brain cells to inhibit electrical activity and return brain network activity to more normal levels. The strategy involves restoring GABA and thus improving the patient's motor control by using an AAV vector (a disabled, non-pathogenic virus) to deliver the GAD gene back into the STN. Increasing GAD causes more GABA to be synthesized, thus helping to calm the STN over-activity.
NLX-P101 is delivered to the brain through a standard, minimally-invasive surgical procedure that uses similar techniques to those currently employed in traditional surgery for PD. The Neurologix gene therapy procedure, however, does not require general anesthesia nor implantation of a permanent medical device in the brain.