Marinus Pharmaceuticals' Ganaxolone Receives FDA Orphan Drug Designation in PCDH19 Female Epilepsy

Print 25 March 2015

RADNOR, Pa., March 25, 2015 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc.(Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative neuropsychiatric therapeutics, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation for ganaxolone, a synthetic analog of the endogenous neurosteroid allopregnanolone, for the treatment of protocadherin 19 gene (PCDH19) female epilepsy. Marinus is currently conducting a Phase 2 clinical trial evaluating the safety and efficacy of ganaxolone as adjunctive therapy for uncontrolled seizures in PCDH19 female pediatric epilepsy, and expects initial data in 2015.  

"We are pleased to receive the Orphan Drug Designation for ganaxolone in PCDH19 female epilepsy," commented Christopher M. Cashman, Chief Executive Officer of Marinus Pharmaceuticals. "This designation underscores the significant unmet medical need for girls suffering from this severe epileptic syndrome, associated with clusters and other types of seizures. We believe that the novel mechanism of ganaxolone, along with the established safety profile seen in pediatric epilepsy trials, supports the potential for ganaxolone to control seizures in these young girls who currently have no approved treatment options." 

Orphan Drug Designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees. The OOPD also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.

About Ganaxolone

Ganaxolone is a small molecule that is a synthetic analog of allopregnanolone, an endogenous neurosteroid produced in the central nervous system that modulates the brain neurotransmitter GABA. Ganaxolone was designed to have the same GABA modulation effects as allopregnanolone without steroidal effects. Ganaxolone and allopregnanolone differ from other GABAA agents by interacting with unique binding sites on the GABAA receptor that are located both within and outside the GABA synapse (synaptic or extrasynaptic binding).  Ganaxolone's activation of the extrasynaptic receptor is a unique mechanism that provides stabilizing effects that we believe differentiates it from other drugs that increase GABA signaling. Preclinical studies provide evidence that the GABA modulatory activity of ganaxolone is responsible for its anticonvulsive activity in epileptic seizures and its antianxiety effects in animal models of other neuropsychiatric disorders.

About PCDH19 Female Pediatric Epilepsy

PCDH19 female pediatric epilepsy is a serious and rare epileptic syndrome that affects approximately 15,000-30,000 females in the United States. The condition, which is caused by an inherited mutation of the protocadherin 19 (PCDH19) gene, located on the X chromosome, is characterized by early-onset cluster seizures, cognitive and sensory impairment of varying degrees, and behavioral disturbances. The PCDH19 gene encodes a protein, protocadherin 19, which is part of a family of molecules supporting the communication between cells in the central nervous system. In case of mutation, protocadherin 19 may be malformed, reduced in its functions or not produced at all. The abnormal expression of protocadherin 19 is associated with occurrence of seizures beginning in the early years of life, mostly focal clusters of seizures that last from one day to weeks. Often, but not always, the syndrome is also associated with a cognitive impairment of varying nature, and behavioral or social disorders with autistic traits. Currently, there are no approved therapies for PCDH19 female pediatric epilepsy.

About Marinus Pharmaceuticals

Marinus Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the development of innovative neuropsychiatric therapeutics. The Company's clinical stage drug candidate for the treatment of seizure disorders in adults and children with epilepsy is ganaxolone. Ganaxolone is a novel synthetic analog of the endogenous neurosteroid, allopregnanolone (known for its anticonvulsive and antianxiety effects) and was designed to avoid hormonal side-effects associated with endogenous neurosteroids. The Company is currently conducting a multi-national, randomized, placebo-controlled, Phase 3 clinical trial to evaluate ganaxolone as adjunctive treatment of partial-onset seizures in adults. Ganaxolone is also being studied in a Phase 2 proof-of-concept clinical study for the treatment of the rare, genetic disorder, PCDH19 female pediatric epilepsy. To complement the existing formulations and to provide continuity of care, the Company is developing an IV formulation of ganaxolone for use in the hospital setting to control epileptic seizures.   In addition, ganaxolone is being evaluated in a Phase 2 proof-of-concept investigator-sponsored clinical trial as a treatment for behaviors in Fragile X Syndrome. For additional information, please visit the Company's website at www.marinuspharma.com.

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", 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 expectations regarding our development plans for our product candidate, including, the clinical trial testing schedule, timing for availability and release of data, and the safety, potential efficacy and therapeutic potential of our product candidate. 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.

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