Omeicos Therapeutics has announced the completion of enrollment in its phase 2a PMD-OPTION study, a multi-center, open-label trial evaluating OMT-28 in patients with primary mitochondrial disease (PMD) suffering from myopathy and cardiomyopathy. The company anticipates top-line data from the study will be available by mid-2025.
The PMD-OPTION study enrolled a total of 28 PMD patients. The trial design includes a 12-week untreated run-in phase to capture the patients’ natural disease history and baseline parameters. Subsequently, all patients receive a 24 mg once-daily dose of OMT-28 for a treatment period of up to 24 weeks.
Study Objectives and Endpoints
The primary objectives of the PMD-OPTION study are to assess the safety and tolerability of OMT-28 and to determine the response rate of patients showing a reduction of Growth differentiation factor 15 (GDF-15) levels by at least 20% compared to baseline. GDF-15, a marker produced in response to mitochondrial stress, inflammation, or hypoxia, is increasingly recognized as a risk predictor in various diseases, including cardiometabolic disorders and PMD.
The study also evaluates a range of secondary and exploratory endpoints. These endpoints are designed to determine the effect of OMT-28 on relevant clinical symptoms, standard functional parameters of physical strength, heart function, quality of life, and key metabolic biomarkers.
OMT-28: A Novel Therapeutic Approach
OMT-28 is described as a first-in-class small molecule with demonstrated cell-protective, anti-inflammatory, and anti-atherosclerotic properties. Preclinical and clinical data have indicated a strong safety profile and tolerability, as well as therapeutic potential in cardiovascular diseases targeting inflammation in atherosclerosis and cardiomyopathy, and other age-related diseases like AMD.
Clinical Significance
PMD patients often experience debilitating and life-threatening health consequences, including severely limited physical stamina, disease-related changes in the heart and skeletal muscles, and associated neurological disorders. Omeicos' therapeutic strategy with OMT-28 aims to improve cell metabolism and mitochondrial function, potentially leading to significant improvements in the quality of life for PMD patients and their families.
"Meeting our PMD-OPTION enrollment target about a year after including the first patient is a significant milestone for the OMT-28 development program. Initial data analysis confirms the strong safety profile and very good tolerability of OMT-28 in the target population. Furthermore, the clinical data obtained so far looks promising for relevant endpoints," said Dr. Robert Fischer, CEO/CSO of Omeicos Therapeutics.
