Can ganaxolone offer safety and efficacy advantages when compared to other marketed antiepileptic medications?




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Video title: Can ganaxolone offer safety and efficacy advantages when compared to other marketed antiepileptic medications?
Released on: December 01, 2015. © PharmaVentures Ltd
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In This Episode:
    Marinus Pharmaceuticals, Inc.
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Marinus Pharmaceuticals, Inc. is a clinical stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for the treatment of epilepsy and other targeted neurological and behavioral disorders. During BIO 2015, Chris Cashman, CEO of Marinus explained to Fintan Walton CEO of PharmaVentures, how their clinical stage product candidate, ganaxolone, a synthetic small molecule analog of a naturally produced neurosteroid (allopregnanolone) may offer safety and efficacy advantages compared to other marketed antiepileptic medications by targeting the same natural mechanism as allopregnanolone.
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Christopher M. Cashman is President and Chief Executive Officer of Marinus and also serves as Chairman of the Board of Directors. Chris is a recognized leader in the biopharmaceutical industry and has decades of experience leading life sciences companies. Prior to joining Marinus, Chris was co-founder, President and CEO of Protez Pharmaceuticals, Inc., a company specializing in the development of antibiotics, which was acquired by Novartis. Prior to his time with Protez, Chris was President and CEO of Message Pharmaceuticals Inc., and held various leadership roles at both Pfizer Inc. and SmithKline Beecham plc. Chris currently serves on the board of directors of Rapid Micro Biosystems, Inc., Noble Biomaterials, Inc. and MBF Therapeutics Inc. Chris holds an M.S. in Economics from Purdue University and B.S. in Business Management from the University of Minnesota.
Marinus Pharmaceuticals, Inc. is a clinical stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for the treatment of epilepsy and other targeted neurological and behavioral disorders. Our clinical stage product candidate, ganaxolone, is a synthetic small molecule analog of a naturally produced neurosteroid (allopregnanolone) which modulates overexcited neurons in the brain. By targeting the same natural mechanism as allopregnanolone, ganaxolone may offer safety and efficacy advantages compared to other marketed antiepileptic medications. We are focused on developing ganaxolone for both the large epilepsy market and targeted pediatric orphan opportunities that we believe offer both a mechanistic rationale for clinical success and the potential for an efficient path to market. Data from a completed Phase 2 clinical trial in 147 patients with focal onset seizures showed that patients who added ganaxolone to their medication regimen experienced a statistically significant reduction in seizures as compared to patients who added placebo. We are currently conducting a multinational Phase 3 clinical trial evaluating the safety and efficacy of ganaxolone as adjunctive therapy for the treatment of focal onset seizures in adults with epilepsy. We are also conducting a proof-of-concept Phase 2 clinical trial with ganaxolone in patients with PCDH19 Female Pediatric Epilepsy and as a treatment for behaviors in Fragile X Syndrome (FXS). Both PCDH19 Female Pediatric Epilepsy and FXS are potential orphan disorders that have been related to genetic mutations affecting neurosteroid signaling.