Marinus Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development of innovative neuropsychiatric therapeutics. The Company's clinical stage drug candidate, ganaxolone, is a novel synthetic analog of the endogenous neurosteroid, allopregnanolone. Ganaxolone is known for its anticonvulsive and antianxiety effects, and was designed to avoid hormonal side-effects associated with endogenous neurosteroids. Ganaxolone is presently being studied in a multinational, randomized, placebo-controlled, Phase 2B/3 clinical trial in adult subjects for adjunctive treatment of partial-onset seizures. The Company currently has a Phase 2 proof-of-concept pediatric clinical trial in progress for ganaxolone as a treatment for behaviors in Fragile X Syndrome and is initiating a Phase 2 proof-of-concept clinical study later this year for the treatment of PCDH19 female pediatric epilepsy. Both Fragile X Syndrome and PCDH19 female pediatric epilepsy are potential orphan disorders that have been related to mutations affecting neurosteroid signaling at extrasynaptic GABA A receptors.
Marinus Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development of innovative neuropsychiatric therapeutics. The Company's clinical stage drug candidate, ganaxolone, is a novel synthetic analog of the endogenous neurosteroid, allopregnanolone. Ganaxolone is known for its anticonvulsive and antianxiety effects, and was designed to avoid hormonal side-effects associated with endogenous neurosteroids. Ganaxolone is presently being studied in a multinational, randomized, placebo-controlled, Phase 2B/3 clinical trial in adult subjects for adjunctive treatment of partial-onset seizures. The Company currently has a Phase 2 proof-of-concept pediatric clinical trial in progress for ganaxolone as a treatment for behaviors in Fragile X Syndrome and is initiating a Phase 2 proof-of-concept clinical study later this year for the treatment of PCDH19 female pediatric epilepsy. Both Fragile X Syndrome and PCDH19 female pediatric epilepsy are potential orphan disorders that have been related to mutations affecting neurosteroid signaling at extrasynaptic GABA A receptors.