Study Rationale: A growing body of research suggests that low doses of carbon monoxide (CO) protect against neuronal cell loss in Parkinson’s disease (PD). Preclinical efficacy data in two rodent models of PD demonstrate that low doses of CO are a promising approach to neuroprotection. In addition, epidemiologic studies have associated cigarette smoking with a lower risk of PD, and nicotine does not appear to underlie the protective effect of tobacco, as a clinical study of nicotine in PD did not show significant improvement. Together, these observations suggest that low doses of CO may be neuroprotective in PD.
Hypothesis: We hypothesize that HBI-002, a novel oral formulation of carbon monoxide (CO), will show neuroprotective benefits in a Phase 2a clinical study in people with PD.
Study Design: This study will evaluate the clinical safety, tolerability, pharmacokinetics and target-engagement biomarker profile of HBI-002, an oral low dose carbon monoxide (CO) drug product, in a Phase 2a clinical study in PD.
Impact on Diagnosis/Treatment of Parkinson’s Disease: Current treatment strategies for people with PD minimize symptomatic but do not slow the course of the disease. Thus, there is a significant need to identify neuroprotective therapies that can impact the course of PD and prolong survival of dopamine neurons.
Next Steps for Development: If successful, this research will provide critical safety, dosing, and biomarker information for advancing HBI-002 into later stage clinical trials in PD, with the ultimate objective being to provide a therapeutic to prevent or slow the course of PD.
Trial Phase: 2a