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.
Dr. LeWitt added, "The start of this clinical trial provides hope to the
patient population which has had a longstanding need for new treatment
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.
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
- 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
- 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
- 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
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
Joan Kureczka, 415-821-2413
Source: Neurologix, Inc.