Date: 2016-10-19
Type of information: Presentation of results at a congress
phase: 1
Announcement: presentation of results at the 141st Annual Meeting of the American Neurological Association
Company: Marinus Pharmaceuticals (USA - PA)
Product: ganaxolone i
Action
mechanism: neurosteroid. Ganaxolone is a synthetic analog of allopregnanolone, an endogenous neurosteroid produced in the central nervous system that modulates GABA through activation of GABAA receptors. Neurosteroids have the ability to enact brain changes rapidly in response to the brain environment, unlike many molecules whose action depends on gene expression or protein synthesis and can take days or weeks.
Disease: status epilepticus
Therapeutic area: Rare diseases - CNS diseases - Neurological diseases
Country:
Trial details:
Latest
news: * On October 19, 2016, Marinus Pharmaceuticals announced that in its Phase 1 dose-escalation study, ganaxolone intravenous (IV) achieved dose levels targeted for efficacy in patients with status epilepticus (SE) and other indications. The Phase 1 clinical study enrolled 36 subjects at Duke University Medical Center and was designed to determine the pharmacokinetics (PK), pharmacodynamics (PD), and safety of ganaxolone IV administered as an ascending bolus dose (Stage 1) or continuous infusion (Stage 2). Four cohorts of subjects were enrolled in Stage 1 and received escalating doses of ganaxolone, and one cohort of subjects was enrolled in Stage 2. The primary study objective was to evaluate the safety and PK of ganaxolone IV. The secondary study objectives included the PD effects of ganaxolone IV on electroencephalogram (EEG) parameters, as well as the effect on clinical sedation scores. Every dose regimen of ganaxolone IV administered, either bolus or continuous infusion, was generally safe and well tolerated and reached targeted dose levels in a short period of time. Following treatment, six treatment-emergent adverse events were reported, all of which were mild in severity and resolved without intervention. Only headache was considered possibly related to treatment with ganaxolone IV. No subject discontinued due to an adverse event and no serious adverse events were reported. Ganaxolone IV plasma concentrations were generally proportional to the administered dose, with potential anti-convulsant plasma concentrations achievable with a bolus dose. Additionally, the continuous infusion of ganaxolone IV achieved the targeted PK levels. The data was presented by Dr. Julia Tsai, senior director of clinical development and project management at Marinus, in a poster entitled, "Phase 1 study to determine the pharmacokinetics, pharmacodynamics, and safety of IV ganaxolone in healthy adults," at the 141st Annual Meeting of the American Neurological Association (ANA), October 16-18, 2016. Marinus is making preparations to commence its Phase 2 clinical study in patients with SE in 2017.
