Study Rationale:
The goal of this program is to develop a novel drug for Parkinson’s disease with a unique mechanism of action. We have a drug candidate that shows potential as an inhibitor of alpha-synuclein buildup. People with Parkinson's have clumps of the protein alpha-synuclein in brain cells, which researchers believe may lead to disease symptoms and progression.
Hypothesis:
Our drug candidate is designed to inhibit alpha-synuclein protein aggregation and spread of pathology by blocking a receptor known as heparan sulfate glycosaminoglycan.
Study Design:
We will first confirm the therapeutic effect of our leading drug candidate in a pre-clinical model of Parkinson's disease. Then the drug candidate will be synthesized in larger quantities and undergo a series of tests to evaluate its drug-like properties, metabolic stability and toxic liabilities. We also plan to optimize the oral delivery of the drug.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
Following the successful completion of these studies, we will have identified a safe and well-tolerated drug candidate. This candidate will be well-positioned for advancement toward Phase I studies. If successful, this project could yield a unique, disease-modifying approach to treat Parkinson's disease.
Next Steps for Development:
Following these studies, we intend to continue pre-clinical studies. Such activities include optimization of dosing and route of administration, development of a clinical plan, and holding a meeting with the FDA. Completion of the activities in this award should position this project on the development pathway to be successful in moving into Phase I clinical trials.