Neurologix, Inc. (OTCBB: NRGX) announced today several new results from the Company's successful Phase 2 clinical trial for its novel, investigational gene therapy NLX-P101 for the treatment of Parkinson's disease (PD) as part of a comprehensive presentation of study findings to the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC). Recently completed analysis of new data showed patients treated with NLX-P101 had significantly increased "ON" times, or periods in which the symptoms of PD are best controlled, compared to sham subjects over the course of 12 months following treatment (p<0.05; ANOVA). Significant reductions in complications due to medical therapy were also seen following treatment with NLX-P101. These new findings, which demonstrate patients treated with NLX-P101 maintained statistically significant, positive, long-term improvements at 12 months, were included in today's presentation of results from the landmark Phase 2 trial by Matthew J. During, M.D., D.Sc., one of the scientific co-founders of Neurologix.
Patients with moderate to advanced PD often suffer from reduced effectiveness of medication, including less time spent each day in the better or "ON" state. The new findings presented today of the NLX-P101 Phase 2 study demonstrated that treatment with the novel gene therapy resulted in an average daily increase of 2.5 hours of "ON" time at 3 months (p<0.01 relative to baseline; t-test) and this increase was sustained at 2.1 hours of greater "ON" time at 12 months (p<0.01 relative to baseline; t-test). There was no significant increase in "ON" time in sham patients at any time point.
There was also a significant reduction in complications of medication in the NLX-P101 group at 6 and 12 months following treatment (p<0.01 and p<0.05, respectively; t-test) as measured by Part 4 of the Unified Parkinson's Disease Rating Scale (UPDRS), with no such reduction in the sham group at any time point. Furthermore, 35% of sham subjects were reported to have worsening PD symptoms as an adverse event during the one year follow-up, consistent with the progressive nature of the disease, whereas such an adverse event was not recorded in a single NLX-P101 subject (p<0.005; Fisher's exact test).
"We were very pleased to be able to summarize our results to date for the RAC, which is an important step in our progress toward a definitive Phase 3 study," said Dr. During, 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. "These new results provide strong support for our earlier observation that NLX-P101 can substantially improve effectiveness of medications in patients who are no longer adequately responding to treatment while reducing complications of these same therapies. These findings support the results of our 2002 preclinical study reported in the journal Science in which we showed that GAD gene transfer is neuroprotective and provides encouragement that NLX-P101 may also have a disease-modifying effect. The strong results observed in both our Phase 1 and now Phase 2 studies increase our confidence in the potential of NLX-P101, and, combined with the outstanding safety profile to date, further justifies our plans to move toward 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 improvement (4.7 points) 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 periods 10th World Congress meeting by Neurologix scientific co-founder Michael G. Kaplitt, M.D., Ph.D., Associate Professor of Neurological Surgery at Weill Cornell Medical College. He reported sustained improvement of an average of 8.2 points in the off-medication UPDRS motor score at one year. Furthermore, while 50% of patients at six months achieved previously defined moderate-to-large clinically meaningful symptom improvements (> 9 points in off-medication UPDRS motor score), 63% of patients achieved such results at one year, with an average 14 point, or 37% improvement in off-medication UPDRS motor scores in this group.
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.
"Neurologix is committed to providing patients a novel, sustained, long-term option for managing and controlling the debilitating conditions of Parkinson's disease," said Clark A. Johnson, President and Chief Executive Officer of Neurologix. "The statistically significant extension of "ON" time, lessening of medication side effects and favorable safety profile in the treated patient group at 12 months are encouraging results that further support NLX-P101 as a viable treatment option against Parkinson's. We look forward to the opportunity to conduct Phase 3 testing, with the goal to bring NLX-P101 to the market for patients in urgent need of a new treatment option."