The objective of this application is to determine whether harkoseride (ADD 2340037) is potentially a safe and effective treatment for epilepsy. Specifically, two clinical studies are proposed to achieve this objective. The first is an open-label ascending dose proof of principle study in a limited number of patients to assess pharmacokinetics, potential drug interactions, and safety in this population and to give an indication of efficacy and a rough estimate of dose. From the information gained in this first study, a double blind placebo controlled second study will be designed to establish an optimal, effective dose and to statistically determine whether harkoseride is a clinically effective and safe agent. Based on nonclinical efficacy and mechanism of action studies and clinical pharmacokinetic and tolerability studies to date, harkoseride has demonstrated a unique mechanism, an ideal pharmacokinetic profile, and acceptable safety margin to be a major contribution to the treatment of epilepsy,
Harkoseride (ADD 234037) has demonstrated an excellent safety, efficacy, and pharmacokinetic profile in nonclinical and clinical studies. Its broad ramp of activity, unique mechanism(s) of action, and availability of both an oral and parenteral formulation make a most promising compound for further development to treat epilepsy, a disease which is not completely controlled with existing medication.
