Data Published in Neuropharmacology
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical
company dedicated to the development of innovative therapeutics to
treat epilepsy and neuropsychiatric disorders, today announced the
publication of preclinical data in the January issue of
Neuropharmacology, showing that its positive allosteric modulator
of GABAA, ganaxolone ameliorates many of the behavioral
abnormalities in a mouse model of adult Angelman syndrome
(AS). Evidence from experimental models suggests that AS may be
associated with deficient extrasynaptic GABAA receptor function. AS
is a rare neurogenetic disorder characterized by severe
developmental delay, motor impairments and epilepsy.
The published article, co-authored by, among
others, Michael A. Rogawski, M.D., Ph.D, professor of neurology and
pharmacology at the School of Medicine, University of California,
Davis, and titled “Effects of the synthetic neurosteroid ganaxolone
on seizure activity and behavioral deficits in an Angelman syndrome
mouse model,” provided the outcomes of a study in an AS mouse model
which evaluated the potential therapeutic benefit that could be
achieved by restoring extrasynaptic GABAA receptor function. The
results showed that ganaxolone-treated mice exhibited improved
anxiety measures, positive motor benefits, and reduced seizures
with both short- and long-term treatment. Importantly, there was no
tolerance to the antiseizure efficacy of ganaxolone or to its other
therapeutic actions.
As stated in the published article, “Our present
results indicate that ganaxolone might be particularly well suited
as a symptomatic treatment for AS, with the potential to not only
treat the seizures but also to provide long-lasting improvement in
the diverse neurobehavioral and motor symptoms.”1
Dr. Jaakko Lappalainen, vice president of
clinical development at Marinus Pharmaceuticals commented, “I am
impressed with these data showing that ganaxolone was anxiolytic,
anticonvulsant, and improved motor deficits in a mouse model of AS.
One of the most notable effects of ganaxolone is the reversal of
the motor and learning deficits in the AS mice, which if
translatable to humans, could significantly improve the quality of
life in AS patients. These data support the rationale to
investigate ganaxolone in the future as a symptomatic treatment for
AS.”
1 Ciarlone, S.L., Wang, X., Rogawski, M.A.,
Weeber, E.J., Effects of the synthetic neurosteroid ganaxolone on
seizure activity and behavioral deficits in an Angelman syndrome
mouse model, Neuropharmacology (2017), doi:
10.1016/j.neuropharm.2016.12.009.
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
1,400 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.
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
lcaperelli@marinuspharma.com
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