There is currently no reliable therapy and no cure for tinnitus. Scientists at INSERM in Montpellier, France demonstrated the effectiveness of gacyclidine in an animal model of tinnitus. Later, clinical scientists the Medical University of Hannover (MHH) confirmed that gacyclidine suppressed human tinnitus in compassionate use individual patient therapy. However, for effective and practical tinnitus control, NST-001 (gacyclidine) must be formulated for chronic and long-term administration. In this regard, NST-001 is not sufficiently stable;not sufficiently soluble in its stable free base form;and suffers from problems of loss to catheters and other components of delivery systems during administration. In Phase I and follow-up research, NeuroSystec has successfully developed a squalene-based nanoparticle formulation that addresses the problems with NST-001 formulation. In this Phase II proposal NeuroSystec will continue with this formulation;the ultimate goal is to produce a commercially viable product that can be safely used for long term administration via an osmotic pump in humans. To accomplish this goal the Company has the following Specific Aims 1) Finalize a squalene (or other pharmaceutically acceptable lipid)-based nanoparticle formulation with and without NST- 001 suitable for animal studies, a pilot sterile manufacturing procedure for drug product and a sterile filtration methodology with <5% drug loss. 2) Demonstrate that this formulation meets criteria for drug delivery and long term stability. Develop manufacturing and storage procedures for loading osmotic pumps with the sterile nanoparticles containing NST-001 formulation. 3) Establish the safety and efficacy of the NST-001 nanoparticle formulation in comparison to solution formulation in an animal model of tinnitus;compare the effectiveness of middle and inner ear delivery of the formulation. At the completion of Phase II NeuroSystec will be able to commence clinical testing of NST-001 to treat severe tinnitus.
There is currently no cure and no effective therapy for severe tinnitus, a disease that afflicts approximately 2 million Americans. NeuroSystec will develop and evaluate a novel lipid based nanoparticle formulation to deliver a promising new drug to the inner ear for the treatment of severe tinnitus.
