Neurologix, Inc. (OTC Bulletin Board: NRGX), announced today that the results of the Company's Phase 2 clinical trial for its novel, investigational gene therapy NLX-P101 for the treatment of Parkinson's disease (PD) were published in an online-first edition of The Lancet Neurology. The randomized, double-blind, sham surgery-controlled trial of 45 subjects with advanced PD met its primary outcome measurement for efficacy and demonstrated that NLX-P101 gene therapy was safe and well-tolerated over the six month blinded study period.
Study results show that NLX-P101 treatment led to a mean 23.1 percent improvement (8.1 points) in off-medication Unified PD Rating Scale (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 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>
Study results also show that 50% of subjects treated with NLX-P101 achieved previously defined moderate-to-large clinically-meaningful symptom improvements (> / = 9 points in UPDRS), compared to just 14% of subjects who received a sham surgical treatment>
"This is the first Phase 2 study conducted under a rigorous randomized, double-blind, sham-controlled surgical design to conclusively demonstrate that gene therapy can be effective for neurological diseases. This confirms our Phase I results and indicates that NLX-P101 may provide a safe, effective and minimally invasive treatment option for patients with PD," noted senior author and co-principal investigator Andrew Feigin, M.D. of The Feinstein Institute for Medical Research at North Shore – LIJ Health System in Manhasset, New York.
According to lead author and co-principal investigator Peter LeWitt, M.D., Director of Movement Disorders at Henry Ford Health System in West Bloomfield, Michigan, "Subjects in our study who received the NLX-P101 treatment displayed better motor performance and control of Parkinsonism than subjects who received sham surgery. This benefit occurred early and was long-lasting. The NLX-P101 study demonstrates that the promise of gene therapy for neurodegenerative disorders appears more likely to become a reality."
"Based on these data, we are more confident that NLX-P101 has great potential to advance the treatment paradigm for Parkinson's patients, and may offer an important, new therapy for patients with this debilitating disease," said Clark A. Johnson, President and Chief Executive Officer of Neurologix. "The publication of our Phase 2 study results by The Lancet Neurology is important validation for our work in gene therapy, which has been ongoing for over a decade. We are very proud of the high-quality science that has supported our program from the start and that rigorous peer-review has once again supported publication of our data in a top-tier journal. These results strongly support the establishment of a Phase 3 clinical trial of NLX-P101 in subjects with Parkinson's disease. We recently discussed our Phase 2 results with the FDA and plan to submit a Phase 3 protocol under a Special Protocol Assessment later this year."
This novel treatment approach was pioneered by Neurologix, Inc. scientific co-founders Matthew J. During, M.D., D.Sc., Professor of Molecular Virology, Immunology and Medical Genetics, Neuroscience and Neurological Surgery, The Ohio State Medical School, and Professor of Molecular Medicine and Pathology, University of Auckland, New Zealand, and Michael G. Kaplitt, M.D., Ph.D., Associate Professor and Vice-Chairman for Research, Dept. of Neurological Surgery, and Director, Stereotactic and Functional Neurosurgery, Weill Medical College of Cornell University. The two researchers 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 adeno-associated virus (AAV) gene transfer technology. 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. The Company's Phase 2 study findings build upon earlier positive results from the NLX-P101 Phase 1 trial, which was the first ever clinical gene therapy trial for PD or any other adult neurological disorder. Results of that study appeared in 2007 as a cover article in The Lancet and in a second article in the Proceedings of the National Academy of Sciences.