Lead Optimization for a Parkinson's Disease Therapeutic
Therapeutics Development Initiative, 2012
CuII(atsm) has the potential to delay disease progression in Parkinsonís disease, based on extensive pre-clinical model data. CuII(atsm) has been shown to significantly improve motor function in standard models of Parkinsonís disease. The observed motor improvement correlates with preservation of dopaminergic neurons in the brain and biomarkers of neuronal health and function.
CuII(atsm) is sparingly soluble and requires formulation for oral administration prior to entering human clinical development. Procypra will pursue two parallel approaches to develop a proprietary oral formulation: (1) Procypra will work with the Warner Babcock Institute for Green Chemistry to develop a proprietary formulation of CuII(atsm) incorporating GRAS (Generally Regarded As Safe) excipients; and (2) Procypra will evaluate the solubility of proprietary CuII(atsm) analogues. The utility of these formulations will be evaluated using standard solubility and bioavailability assays and efficacy will be compared to the parent formulation in the MPTP toxic lesion pre-clinical model of Parkinsonís disease.
Relevance to Diagnosis/Treatment of Parkinsonís Disease:
CuII(atsm) has the potential to delay disease progression in Parkinsonís disease. Successful clinical development of an optimized formulation of CuII(atsm) would provide Parkinsonís disease patients, on diagnosis, the opportunity to delay the progression of their disease and maintain their quality of life for a much extended period of time. In patients with more advanced Parkinsonís disease, co-administration of CuII(atsm) with symptomatic therapeutics would delay further disease progression and halt the debilitating motor and cognitive deterioration.
On successful completion of The Michael J. Fox Foundation project, Procypra will have developed an improved formulation of CuII(atsm) suitable for human clinical development. The optimized formulation will be advanced into formal IND-directed toxicology studies in preparation for the commencement of clinical trials.
INTERIM PROGRESS REPORT
At this interim point in the program, a number of variant candidates of the parent drug candidate have†been prepared and tested in a variety of diagnostic tests to show an improvement over the parent drug†candidate. A number of these variants show promise as improved drug candidates. The remainder of the†program will be spent demonstrating which of these variants shows the most promise as a therapeutic†candidate, using accepted models of Parkinsonís disease. This work will hopefully culminate in the selection†of a lead candidate with improved drug characteristics, which Procypra Therapeutics will further develop†as a pre-clinical drug candidate for Parkinsonís disease.
Location: Melbourne, Australia