RADNOR, Pa., Oct. 26, 2015 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced key observations from patients enrolled in its ongoing study evaluating ganaxolone, a synthetic analog of the endogenous neurosteroid allopregnanolone, as a treatment for PCDH19 female pediatric epilepsy. PCDH19 is a serious and rare epileptic syndrome characterized by highly variable early-onset cluster seizures, and cognitive and behavioral disturbances. Enrollment is continuing in the study with full data expected in 2016.
This Phase 2 exploratory trial is being conducted at seven sites in the United States and one site in Italy. The study is designed to enroll approximately 10 female pediatric patients between the ages of 2 and 18 years old with a confirmed PCDH19 genetic mutation. Ganaxolone is administered as either oral liquid suspension or capsules, for up to 26 weeks after establishing up to 12 weeks of baseline seizure frequency. The primary efficacy measure in the trial is percent change in seizure frequency per 28 days relative to baseline.
- To-date, 63% (5/8) of patients have experienced a greater than 50% reduction for at least one 28-day treatment period compared to baseline.
- Safety data to-date are consistent with earlier studies where ganaxolone has been shown to be generally safe and well-tolerated and the most commonly reported side-effects were sedation, agitation and dizziness. One seizure-free responder discontinued treatment due to a rash that was deemed to be possibly drug related, and another responder showed large variability in response and later discontinued from the study. Two patients were non-responders.
- Patients enrolled in the study are continuing to receive treatment and have received ganaxolone for durations ranging from one to four months of the planned six month treatment period.
- Improvements in behavior and cognitive skills have been reported to and observed by investigators for some patients.
"This study has been designed to explore rare disease patient populations that we believe are underserved by current approved therapeutic options and may allow for more efficient clinical development pathways," commented Dr. Albena Patroneva, Chief Medical Officer of Marinus Pharmaceuticals. "We continue to learn about the symptom manifestations, complex comorbidities and mutation prevalence of this rare disorder. The results from this study will inform our overarching pediatric plan for ganaxolone."
Marinus has expanded the enrollment in this exploratory trial to also evaluate ganaxolone in other pediatric genetic epilepsies.
These observations were recently presented at the World Conference on PCDH19 in Rome, Italy. Study investigators expressed encouragement with the initial observations and mentioned that in addition to seizures endpoints, parents of patients have reported instances of behavioral improvements allowing for better social interactions and increased involvement in current and new day to day activities.
About PCDH19 Female Pediatric Epilepsy
PCDH19 female pediatric epilepsy is a serious and rare epileptic syndrome that predominantly affects females. The condition, which is caused by a mutation of the protocadherin 19 (PCDH19) gene located on the X chromosome, is characterized by early-onset and highly variable cluster seizures, and cognitive 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. 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 focal onset seizures in adults. Ganaxolone is also being studied in an exploratory 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 an exploratory 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 interpretation of clinical efficacy and safety in our clinical studies, our expectations regarding our 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.