Phase 2A safety and biomarker study of the drug substance EPI-589 in patients with mitochondrial subtype of Parkinson's disease


Mitochondrial dysfunction contributes to the pathophysiology of PD in both patients with genetic and idiopathic form of Parkinson’s disease. Despite the positive effect of symptomatic treatment, PD remains a highly morbid disease. So far, no disease modifying treatment approach has been verified to slow down disease progression. EPI-589 is a redox active molecule that has demonstrated potency (EC50 < 100nM) and efficacy (>80% rescue from oxidative stress-induced cell death) in cells derived from patients diagnosed with idiopathic and familial PD subtypes including PINK1, Parkin and LRRK2.

Background and aims

Primary Objective of this study is to evaluate the effects of EPI-589 on safety in subjects with idiopathic and genetic PD (e.g. hematology, EEG).

Secondary objectives are the evaluation of the effects of EPI-589 in subjects with PD on:
1. Fasting glutathione cycle biomarkers as measured in blood, cerebral spinal fluid, and urine,
2. Clinical disease state as assessed by movement disorder rating.

In addition the effects of EPI-589 on non-motoric symptoms as well as cognitive disorders and depression are evaluated.


Subjects will participate in a 30-day run-in phase to establish biomarker and clinical baseline measurements. EPI-589 will then be administered for up to 3 months (85 days ± 3 days) unless discontinued for safety or tolerability issues. Post-treatment follow-up will be at least 10 days and up to 30 days after last dose.

Course of the study

The examinations include clinical-neurological assessment, neuropsychological testing as well as assessment of autonomic functions. Subjects with idiopathic PD will have a DatSCAN (scintigraphy of the brain). In addition blood and urine samples as well as spinal fluid will be collected.

Principle investigator: Prof. Dr. Thomas Gasser
Start of the study: 2017
Status: mono centric (DZNE Tübingen), study closed

Study coordination / Project management
Dr. Kathrin Brockmann
Elisabeth van Schalkwyk
+49 7071 298-1465

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