Platform Presentation of Phase 2 Data in Fragile X Syndrome
RADNOR, Pa., April 18, 2017 -- Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, announced today that data on ganaxolone, Marinus’ positive allosteric modulator of GABAA, will be presented at the 69th Annual Meeting of the American Academy of Neurology in Boston, MA on April 22-28, 2017.
The presentation details are as follows:
Title: A Randomized Double-blind, Placebo-controlled, Cross-over Trial of Ganaxolone in Children and Adolescents with Fragile X Syndrome
Abstract #: 1232
Platform Presentation #: 005
Platform Presentation Session: S46: Child Neurology: Molecular Biology of Clinical Trials
Date and Time: Thursday, April 27, 2017 @ 4:18 pm ET
Presenting Author: Julia Tsai, Ph.D.
Title: A Multi-center, Open-label Trial of Ganaxolone in Children with PCDH19 Epilepsy
Abstract #: 3477
Session #: Poster Session 5
Poster #: 236
Date and Time: Thursday, April 27, 2017 8:30 am – 7:00 pm ET
Presenting Author: Jaakko Lappalainen, MD
Title: A Multi-center, Double-blind, Randomized, Placebo-controlled Phase 3 Trial to Determine the Efficacy and Safety of Ganaxolone as Adjunctive Therapy for Adults with Drug-Resistant Focal Onset Seizures
Abstract #: 3476
Session #: Poster Session 5
Poster #: 237
Date and Time: Thursday, April 27, 2017 8:30 am – 7:00 pm ET
Presenting Author: Jaakko Lappalainen, MD
About Ganaxolone
Ganaxolone, a positive allosteric modulator of GABAA, is being developed in three different dose forms (intravenous, capsule, and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Ganaxolone exhibits antiseizure and antianxiety actions via its effects on synaptic and extrasynaptic GABAA receptors. Ganaxolone has been studied in more than 1,500 subjects, both pediatric and adult, at therapeutically relevant dose levels and treatment regimens for up to two years. In these studies, ganaxolone was generally safe and well tolerated. The most commonly reported adverse events were somnolence, dizziness and fatigue.
About Marinus Pharmaceuticals
Marinus Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and neuropsychiatric disorders. Ganaxolone is a positive allosteric modulator of GABAA that acts on a well-characterized target in the brain known to have both antiseizure and antianxiety effects. Ganaxolone is being developed in three different dose forms (IV, capsule and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Marinus is currently evaluating ganaxolone in orphan pediatric indications for the treatment of genetic seizure and behavior disorders, and preparing to initiate Phase 2 studies in status epilepticus, an orphan indication, and postpartum depression. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.
Forward-Looking Statements
To the extent that statements contained in this press release are not descriptions of historical facts regarding Marinus, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as “may”, “will”, “expect”, “anticipate”, “estimate”, “intend”, “believe”, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Examples of forward-looking statements contained in this press release include, among others, statements regarding our interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical trial testing schedule and milestones, the ability to complete enrollment in our clinical trials, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this release involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the conduct of future clinical trials, the timing of the clinical trials, enrollment in clinical trials, availability of data from ongoing clinical trials, expectations for regulatory approvals, and other matters, including the development of formulations of ganaxolone, that could affect the availability or commercial potential of our drug candidates. Marinus undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the Company in general, see filings Marinus has made with the Securities and Exchange Commission.
CONTACT: Lisa M. Caperelli Executive Director, Investor & Strategic Relations Marinus Pharmaceuticals, Inc. 484-801-4674 [email protected]


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