Study Rationale: Although levodopa is an effective treatment for Parkinson’s disease (PD), it has a debilitating on-target side effect of dyskinesia. An optimal PD therapy would selectively activate therapeutic pathways that lie downstream of dopamine receptors while avoiding those that induce dyskinesia. Using machine learning algorithms developed by Sinopia, we identified a therapeutically effective target — along with a small molecule, SB-0110, that modulates this target. In preclinical models, SB-0110 has been shown to robustly reduce both PD symptoms and dyskinesia when administered with levodopa.
Hypothesis: This clinical study will assess whether SB-0110 is safe for use in humans and determine the pharmacokinetics of the compound.
Study Design: This study will consist of two parts. In the first part, we will assess the safety and tolerability of single doses of SB-0110 in healthy volunteers in a dose escalating fashion. We will begin with a low dose and gradually escalate to higher dosages. In the second part of the study, we will further assess safety and tolerability in healthy volunteers receiving SB-0110 over a ten day period. In both parts, we will also collect blood samples for assessing concentrations of SB-0110 in plasma.
Impact on Diagnosis/Treatment of Parkinson’s Disease: If successfully developed, SB-0110 may change how PD is treated at all stages of the disease. For individuals with advanced PD, SB-0110 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-0110 and levodopa may provide superior efficacy to the current standard of care and prevent or delay dyskinesia complications.
Next Steps for Development: The results generated by this study will help determine the doses of SB-0110 that are safe in healthy volunteers. This information will be used to design Phase 1b/2a studies to test the safety and potential efficacy of SB-0110 in PD patients.
Trial Phase: Phase 1a