Study Rationale:
Although levodopa is an effective treatment for Parkinson’s disease (PD), its ability to activate dopamine D1 receptors also leads to its most vexing side effect: dyskinesia. An optimal PD therapy would selectively activate the therapeutic pathways that lie downstream of D1 receptors while avoiding those that induce dyskinesia. Using machine learning algorithms developed by Sinopia, we have identified a therapeutically effective target — along with a small molecule, SB-0107, that modulates this target. In preclinical models, SB-0107 has been shown to robustly reduce both PD symptoms and dyskinesia when administered with levodopa.
Hypothesis:
This clinical study will assess whether SB-0107 is safe for use in people with PD and whether it shows promise for treating the symptoms of PD as well as levodopa-induced dyskinesia.
Study Design:
This study will consist of three parts. In the first part, we will assess the safety of SB-0107 by administering the drug to individuals with PD. We will begin with a low dose and gradually escalate to higher dosages. In the second part of the study, we will assess the potential efficacy of SB-0107 for alleviating the motor symptoms of PD. In the third part of the study, we will assess whether SB-0107 has a positive effect on individuals experiencing levodopa-induced dyskinesia.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
If successfully developed, SB-0107 may change how PD is treated at all stages of the disease. For individuals with advanced PD, SB-0107 could be used as an add-on therapy to both improve PD symptoms and reduce dyskinesia. For those in the early stages of PD, a combination of SB-0107 and levodopa may provide superior efficacy to the current standard of care and prevent dyskinesia complications.
Next Steps for Development:
The results generated by this study will help determine the doses of SB-0107 that are both safe and effective for the treatment of motor dysfunction in people with PD. This information will be used to design Phase 2 studies to test the utility of SB-0107.
Trial Phase:
Phase Ib/IIa