Marinus Pharmaceuticals Initiates Pivotal Phase 3 Trial in Children with CDKL5 Deficiency Disorder
June 19 2018 - 7:30AM
Marigold Study targets rare form of pediatric
epilepsy with no approved therapies
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS) (“Marinus” or
“Company”), a biopharmaceutical company dedicated to the
development of innovative therapeutics to treat epilepsy and
neuropsychiatric disorders, today announced the initiation of a
pivotal Phase 3 clinical trial (Marigold Study) evaluating the use
of oral ganaxolone in children and young adults with CDKL5
Deficiency Disorder (CDD).
“We are excited to offer the CDKL5 patient community, for the
first time, participation in a pivotal study of ganaxolone that may
have the potential to reduce the seizure burden of the disorder,”
said Christopher M. Cashman, Chief Executive Officer of Marinus.
“After seeing the long-term results from our Phase 2 trial showing
that ganaxolone provides a meaningful reduction in the number of
seizures for children with CDD, as well as an impressive increase
in seizure-free days, we focused our efforts to expedite the
initiation of this registration study. CDKL5 patients are in
need of new treatment options and we look forward to working with
the entire community in the conduct of this study.”
The Marigold Study is a global, double-blind,
placebo-controlled, Phase 3 clinical trial that will enroll
approximately 70 patients between the ages of 2 and 21 with a
confirmed disease-related CDKL5 gene variant. Patients will
undergo a baseline period before being randomized to receive either
ganaxolone (up to 1,800 mg/day) or placebo for 17 weeks, in
addition to their existing anti-seizure treatment. Following the
treatment period, all patients that meet certain eligibility
requirements will have the opportunity to receive ganaxolone in the
open label phase of the study. The study’s primary efficacy
endpoint is percent reduction in seizures. Secondary outcome
measures will include non-seizure-related endpoints to capture
certain changes in behavioral and sleep disturbances that were seen
as improvements in previous clinical studies with ganaxolone.
CDD is caused by a mutation of the cyclin-dependent kinase-like
5 (CDKL5) gene, located on the X chromosome. The CDKL5 gene encodes
proteins essential for normal brain function. CDD predominantly
affects girls and is characterized by early-onset,
difficult-to-control seizures and severe neurodevelopmental
impairment. Most children affected by CDD cannot walk, talk, or
feed themselves, and many are confined to wheelchairs, dependent on
others for their care.
Currently, there are no approved therapies for CDD worldwide.
Existing anti-epilepsy medications do not work well in CDD, and at
best, any efficacy is short-lived.
Ganaxolone is a neurosteroid designed to provide anti-seizure
activity by calming the brain and restoring its electrical balance.
Ganaxolone’s method of action is different from existing epilepsy
medications, binding to unique GABAA receptors and has been shown
in animal models to quickly inhibit seizure spread and prevent new
seizures from occurring.
Last September, Marinus reported top-line data from a Phase 2
open-label study in patients with CDD. Oral ganaxolone, in addition
to baseline treatment, showed a sizable and durable
seizure-frequency reduction with a large increase in the number of
seizure-free days in the majority of patients and with some
reporting behavioral benefits. Ganaxolone has been administered in
more than 200 children, as young as four months old and dosed for
more than four years. Ganaxolone is generally safe and
well-tolerated with no related serious adverse events reported to
date in this population. Marinus has received Orphan Drug
Designation from the FDA for ganaxolone in CDD.
More information on the Marigold Study will be available at
www.themarigoldstudy.com or www.clinicaltrials.gov.
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 anti-seizure, anti-depressant 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. Marinus has initiated
the first ever pivotal study in children with CDKL5 deficiency
disorder, a rare form of epilepsy, and is currently conducting
studies in patients with postpartum depression and refractory
status epilepticus. For more information visit
www.marinuspharma.com. Please follow us on Twitter:
@MarinusPharma.
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, the attainment of clinical trial results that will be
supportive of regulatory approvals, and other matters, including
the development of formulations of ganaxolone, and the availability
or potential availability of alternative products or treatments for
conditions targeted by the Company 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. CaperelliExecutive Director,
Investor & Strategic RelationsMarinus Pharmaceuticals,
Inc.484-801-4674lcaperelli@marinuspharma.com
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