FORT LEE, N.J., May 26, 2011 -- Neurologix, Inc. (OTCBB: NRGX) announced the presentation today of efficacy results through one year of follow-up in patients treated as part of the Company's successful Phase 2 clinical trial for its novel, investigational gene therapy NLX-P101 for the treatment of Parkinson's disease (PD). Improvements in the Unified Parkinson's Disease Rating Scale (UPDRS) at 12 months for the randomized, double-blind, sham surgery-controlled trial of 45 subjects with advanced PD confirmed that patients treated with NLX-P101 experienced sustained, positive, long-term benefits. The one-year follow-up data were presented by one of the scientific co-founders of Neurologix, Michael G. Kaplitt, M.D., Ph.D., at the International Neuromodulation Society's (INS) 10th World Congress in London.
In the one-year follow-up analysis, patients treated with NLX-P101 who achieved previously defined moderate-to-large clinically-meaningful symptom improvements (≥ 9 points in off-medication UPDRS motor score), increased from 50 percent at six months to 63 percent at one year. This major subgroup of NLX-P101-treated patients experienced a mean 37 percent improvement in their symptoms after one-year, with an average improvement of 14 points in off-medication UPDRS motor score. Among all patients included in the NLX-P101 treatment group, the clinical improvements demonstrated at six months were maintained, with an average 8.2 point improvement in off-medication UPDRS motor score at 12 months compared to an 8.1 point improvement at six months. No serious adverse events (SAEs) related to the gene therapy or surgical procedure were reported in either the blinded six-month study period or the completed 12 month study phase.
"These results demonstrate that treatment with NLX-P101 improved the primary motor symptoms of Parkinson's disease in patients overall, with a majority obtaining a highly clinically meaningful improvement," said Neurologix scientific co-founder Matthew J. During, M.D., D.Sc., Professor of Molecular Virology, Immunology and Medical Genetics, Neuroscience and Neurological Surgery, The Ohio State University Medical School, and Professor of Molecular Medicine and Pathology, University of Auckland, New Zealand. "Further, the positive, durable responses we saw one-year after treatment with NLX-P101 in the Phase 2 study are consistent with the long-term efficacy demonstrated in our Phase 1 trial, the first ever to demonstrate the viability of gene therapy for the treatment of Parkinson's disease. The results from the first two phases of NLX-P101 clinical research give us great confidence in the potential of NLX-P101 as we move forward with our plans for the first Phase 3 trial for gene therapy in the treatment of this debilitating disease."
The six month results of the Company's landmark Phase 2 study were published in the peer-reviewed journal The Lancet Neurology in March of this year. Study results through six months of follow-up showed that NLX-P101 treatment led to a mean 23.1 percent improvement (8.1 points) in off-medication UPDRS motor score at the six-month study end-point, compared to a mean 12.7 percent (4.7 points) improvement with sham treatment. Improved motor control in the NLX-P101 group was seen at one month and continued virtually unchanged throughout the blinded six month study period. The improvement in UPDRS motor scores from baseline in the NLX-P101 group was significantly greater than sham subjects over the six month study period (p=0.04).
The Company's Phase 2 study findings build upon earlier positive results from the NLX-P101 Phase 1 trial, which appeared in 2007 as a cover article in The Lancet and in a second article in the Proceedings of the National Academy of Sciences. Neurologix plans to submit a Phase 3 protocol to the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment later in 2011.
"The results presented today at the INS World Congress add to a growing body of clinical evidence demonstrating that our novel gene therapy approach may provide significant clinical benefits to Parkinson's disease patients," During added. "In the treatment arm of the Phase 2 study, improved motor control was seen at one month, continued virtually unchanged throughout the six-month blinded study period and persisted through 12 months of follow-up. This suggests that the treatment effect from NLX-P101 is meaningful and durable over the long-term."
Drs. During and Kaplitt have been at the forefront of gene therapy research since 1989 and the Phase 2 trial was the culmination of nearly 20 years of progress with their work in gene transfer technology using an adeno-associated virus (AAV) vector (a disabled, non-pathogenic virus). They were the first to demonstrate that AAV could be an effective gene therapy agent in the brain, which they reported in their landmark Nature Genetics paper in 1994. Drs. During, Kaplitt and colleagues subsequently published additional research demonstrating the beneficial effects of AAV-GAD gene therapy for PD in the journal Science in 2002.
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