Marinus Pharmaceuticals Announces Positive End-of-Phase 2 Meeting With FDA for Ganaxolone in Focal Onset Seizures
RADNOR, Pa., Sept. 9, 2015 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, announced today the successful completion of an End-of-Phase 2 meeting with the Food and Drug Administration (FDA). The objective of the meeting was to obtain FDA feedback on the nonclinical and clinical programs to support registration of ganaxolone for the adjunctive treatment of focal onset seizures in adults.
"We are very pleased with our recent positive interaction with the FDA," commented Christopher M. Cashman, Chief Executive Officer of Marinus Pharmaceuticals. "Based on the valuable guidance received from our discussions with the FDA, we believe our clinical development and registration strategy for ganaxolone will support an NDA submission. We look forward to completing our on-going Phase 3 clinical trial in adults with focal onset seizures and initiating a second Phase 3 clinical trial in 2016."
At the meeting, the FDA was in agreement with Marinus on its planned path to support registration of ganaxolone for adjunctive treatment of focal onset seizures, which, among other anticipated preclinical and clinical studies, includes a single additional Phase 3 registration study. Marinus and FDA are in agreement on the design, population and primary endpoint for both the ongoing and planned second Phase 3 clinical study. Marinus intends to submit the protocol of the second Phase 3 study to the FDA for a Special Protocol Assessment.
The second Phase 3 clinical trial is expected to be a global, randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of ganaxolone in adults, age 17 years or older, diagnosed with focal onset seizures. The planned primary endpoint for the study will be percent change in seizure frequency per 28 days relative to baseline.
Marinus is currently conducting a multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial of ganaxolone in approximately 300 adults with drug-resistant focal onset seizures. The primary endpoint of the study is percent change in seizure frequency per 28 days relative to baseline. Top-line data from the study are anticipated in the first quarter of 2016. Further information about the Phase 3 study can be found on ClinicalTrials.gov, using Identifier NCT01963208.
Ganaxolone is a small molecule that is a synthetic analog of allopregnanolone, an endogenous neurosteroid produced in the central nervous system that modulates GABA, the major brain inhibitory neurotransmitter. Ganaxolone was designed to have similar GABA modulation effects to allopregnanolone without unwanted steroidal side effects. Ganaxolone and allopregnanolone differ from other GABAA agents by interacting at both synaptic and extrasynaptic GABAA receptor binding sites that are located both within and outside the GABA synapse. Ganaxolone's activation of the extrasynaptic receptor is a unique mechanism that, in addition to other effects, increases tonic GABA inhibition that we believe differentiates it from other available drugs that increase GABA levels in the brain. 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. Ganaxolone is being developed in three dose forms; oral capsule, oral liquid suspension, and intravenous formulation in order to bring convenient dosing to a variety of clinical situations.
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 intravenous 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.
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 expectations regarding our interpretation of discussions and correspondence with the United States Food and Drug Administration, 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.
Lisa M. Caperelli
Senior Director, Investor Relations & Corporate Communications
Marinus Pharmaceuticals, Inc.
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Amy S. Wheeler