A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy

An Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With Primary Mitochondrial Myopathy (PMM), With an Optional Extension of Treatment

The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy

Inclusion Criteria:

• Subjects must give written, signed and dated informed consent
• Confirmed diagnosis of PMM according to the 2016 Rome Consensus recommendations
• Confirmed mitochondrial mutation with evidence of myopathy
• Able to remain on stable medication throughout the study

Exclusion Criteria:

• Documented evidence of ongoing rhabdomyolysis
• Subjects with motor abnormalities other than related to mitochondrial disease
• Treatment with an investigational drug within 3 months prior to Day 1
• Hospitalised within 3 months prior to screening for any major medical condition
• Pregnant or nursing females