Data Presented at the 141st Annual Meeting of the
American Neurological Association
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical
company dedicated to the development of innovative therapeutics to
treat epilepsy and neuropsychiatric disorders, today announced that
in its Phase 1 dose-escalation study, ganaxolone intravenous (IV)
achieved dose levels targeted for efficacy in patients with status
epilepticus (SE) and other indications. Status epilepticus is a
life-threatening medical emergency associated with high mortality
and limited treatments. Typically, single or combination IV
antiepileptic drugs are used in an attempt to break the seizures,
however there are approximately 45,000 patients in the U.S. who do
not respond to first-line treatment.
Albena Patroneva, M.D., chief medical officer at
Marinus, commented, “We are pleased with the results from our Phase
1 study which has provided predictable PK to enable dosing of
ganaxolone IV in clinical studies in SE. There is a significant
need for therapies that can rapidly stop the seizures in patients
with SE. We believe ganaxolone IV could be a promising therapeutic
option in this difficult-to-treat seizure disorder and look forward
to advancing our clinical studies into patients with SE.”
The Phase 1 clinical study enrolled 36 subjects
at Duke University Medical Center and was designed to determine the
pharmacokinetics (PK), pharmacodynamics (PD), and safety of
ganaxolone IV administered as an ascending bolus dose (Stage 1) or
continuous infusion (Stage 2). Four cohorts of subjects were
enrolled in Stage 1 and received escalating doses of ganaxolone,
and one cohort of subjects was enrolled in Stage 2. The
primary study objective was to evaluate the safety and PK of
ganaxolone IV. The secondary study objectives included the PD
effects of ganaxolone IV on electroencephalogram (EEG) parameters,
as well as the effect on clinical sedation scores.
Every dose regimen of ganaxolone IV
administered, either bolus or continuous infusion, was generally
safe and well tolerated and reached targeted dose levels in a short
period of time. Following treatment, six treatment-emergent
adverse events were reported, all of which were mild in severity
and resolved without intervention. Only headache was considered
possibly related to treatment with ganaxolone IV. No subject
discontinued due to an adverse event and no serious adverse events
were reported. Ganaxolone IV plasma concentrations were generally
proportional to the administered dose, with potential
anti-convulsant plasma concentrations achievable with a bolus dose.
Additionally, the continuous infusion of ganaxolone IV achieved the
targeted PK levels.
The data was presented by Dr. Julia Tsai, senior
director of clinical development and project management at Marinus,
in a poster entitled, “Phase 1 study to determine the
pharmacokinetics, pharmacodynamics, and safety of IV ganaxolone in
healthy adults,” at the 141st Annual Meeting of the American
Neurological Association (ANA), October 16-18, 2016.
Marinus is making preparations to commence its
Phase 2 clinical study in patients with SE in 2017. Earlier this
year, the U.S. Food and Drug Administration granted Orphan Drug
Designation to ganaxolone IV for the treatment of SE.
About Ganaxolone
Ganaxolone is a CNS-selective GABAA modulator
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. Ganaxolone acts on a well-characterized synaptic and
extrasynaptic GABAA target known for anti-seizure and
anti-anxiety activity. Ganaxolone has been studied in more
than 1,300 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,
with the most commonly reported adverse events of 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 CNS-selective GABAA modulator that acts on a
well-characterized target in the brain known to have both
anti-seizure and anti-anxiety 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.
Ganaxolone IV is advancing into Phase 2 studies to treat status
epilepticus. Ganaxolone IV is complemented by its oral dose forms,
providing the potential for IV-to-oral continuation therapy for
patients transitioning from acute care to outpatient settings.
Ganaxolone capsule and liquid is being studied in orphan pediatric
indications with comorbidities in seizures and behavior disorders –
PCDH19, CDKL5, Lennox-Gastaut Syndrome and Fragile X Syndrome. 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: Company:
Lisa M. Caperelli
Senior Director, Investor Relations & Corporate Communications
Marinus Pharmaceuticals, Inc.
484-801-4674
lcaperelli@marinuspharma.com
Media Contact:
Tiberend Strategic Advisors, Inc.
Amy S. Wheeler
646-362-5750
awheeler@tiberend.com
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