Marinus Pharmaceuticals

Marinus Pharmaceuticals, Inc. is a clinical stage biopharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, an extensive safety database, and convenient dosing to improve the lives of patients suffering from drug-resistant seizures and neuropsychiatric disorders. Based on pre-clinical and clinical studies conducted to date, ganaxolone has exhibited anxiolytic, anti-seizure and anesthetic activity by virtue of its GABA_A receptor modulating properties.

Marinus is developing three different dose forms of ganaxolone (IV, capsule, and liquid) to maximize the therapeutic reach to adult and pediatric patients in both acute and chronic care settings where there is a mechanistic rationale for ganaxolone to provide a benefit.

Marinus is conducting a phase 2 study to evaluate the intravenous formulation of ganaxolone in women with postpartum depression, a disease with no approved therapies that affects 15% of women within the first year of childbirth.  Marinus is preparing to initiate a phase 2 study to evaluate the intravenous formulation of ganaxolone in patients with status epilepticus, an orphan indication, which if not treated immediately, could result in high morbidity and mortality.

Marinus is currently evaluating oral ganaxolone in orphan genetic pediatric indications where children suffer from seizures, cognitive impairment and behavioral disorders.  The phase 2 study has enrolled children with CDKL5 disorder and Lennox-Gastaut Syndrome (LGS).

Our goal is to maximize the value of ganaxolone as a first-in-class innovative neuropsychiatric therapy with a portfolio of diversified indications. To achieve this goal, we are focused on the following:

• Targeting underserved and orphan indications where we believe modulating GABA_A would provide a therapeutic benefit to patients
• Developing a range of ganaxolone dose forms for adults and pediatrics for use in acute and chronic care settings
• Establishing proof-of-concept clinical data in support of progressing to advanced pivotal studies
• Leveraging and expanding our extensive intellectual property asset base