Funded Studies
Objective/Rationale:
CuII(atsm) has the potential to delay disease progression in Parkinson’s disease, based on extensive pre-clinical model data. CuII(atsm) has been shown to significantly improve motor function in standard models of Parkinson’s disease. The observed motor improvement correlates with preservation of dopaminergic neurons in the brain and biomarkers of neuronal health and function.
Project Description:
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 Parkinson’s disease.
Relevance to Diagnosis/Treatment of Parkinson’s Disease:
CuII(atsm) has the potential to delay disease progression in Parkinson’s disease. Successful clinical development of an optimized formulation of CuII(atsm) would provide Parkinson’s 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 Parkinson’s disease, co-administration of CuII(atsm) with symptomatic therapeutics would delay further disease progression and halt the debilitating motor and cognitive deterioration.
Anticipated Outcome:
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.
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 Parkinson’s 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 Parkinson’s disease.
January 2014