Plans to Apply for Orphan Drug Designation
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), today announced
positive preliminary data from the initial CDKL5 patients enrolled
in its ongoing Phase 2 open-label study evaluating its
CNS-selective GABAA modulator, ganaxolone, as a treatment for
orphan, genetic disorders. CDKL5 is a severe, rare genetic disorder
that results in early-onset, difficult-to-control seizures, and
neuro-developmental impairment. Enrollment is continuing in the
study with top-line data expected in mid-2017.
Four patients have been enrolled in this cohort
of the study and received up to 1800 mg/kg of ganaxolone per day
for an average treatment duration of five-months. Three of the four
patients experienced a notable reduction in seizure frequency
compared to baseline ranging from 52% to 88%. All responders
continue to receive treatment, two of whom have completed
six-months of treatment and have elected to participate in the
study extension. One patient discontinued the study after
four-months of treatment due to lack of efficacy. Safety data to
date are consistent with earlier studies where ganaxolone has shown
to be generally safe and well-tolerated.
“We are encouraged by the results in these
difficult-to-treat pediatric patients,” commented Dr. Jaakko
Lappalainen, Vice President of Clinical Development of Marinus
Pharmaceuticals. “Concurrent with completing this study, we will be
evaluating the potential for breakthrough therapy and applying for
orphan drug designation with the United States Food and Drug
Administration. CDKL5 pediatric epilepsy may prove to be an
attractive and efficient path for ganaxolone and we look forward to
evaluating results from the final patients enrolled in this cohort
of the study.”
Michael G. Chez, MD, Director of Pediatric
Neurology Research and Pediatric Epilepsy, Sutter Neuroscience
Institute in Sacramento, CA commented, “I am impressed with the
responder rate and magnitude of seizure control seen with
ganaxolone in the initial CDKL5 patients. The CGIs (clinical global
impression scales) are consistent with seizure control, with
responders showing ‘much improved’ under this scale. I look forward
to further evaluating these children and seeing the final
results.”
At the annual meeting of the American Epilepsy
Society, Dr. Chez presented EEG data from one CDKL5 and two PCDH19
patients that he treated with ganaxolone in the on-going Phase 2
open-label study. The patients received up to 1800 mg/day of
ganaxolone. EEG measurements were taken at baseline and followed-up
at 8-12 weeks of treatment.
The CDKL5 patient showed a >67% seizure
reduction and EEG changes consistent with clinical improvement (50%
reduction in awake slow-spike wave discharges). The two patients
with PCDH19 showed an 80% and 75% reduction in seizure frequency,
respectively, and EEG improvement in slow-spike and wave frequency
of >90% and 80% on awake and asleep EEG.
This Phase 2 open-label trial is currently
accepting patients at five sites in the United States and one in
Italy. The multi-cohort study is designed to enroll up to 10
patients with each of CDKL5 disorder, Lennox Gastaut Syndrome (LGS)
and PCDH19 pediatric epilepsy. The study is actively recruiting
CDKL5 and LGS patients. The PCDH19 cohort of the study is currently
closed for enrollment, however, there are still children receiving
ganaxolone in the study extension. For more information about
the study visit clinicaltrials.gov.
About CDKL5 Disorder
CDKL5 is a serious and rare genetic disorder
that is caused by a mutation of the cyclin-dependent kinase-like 5
(CDKL5) gene, located on the X chromosome. It predominantly affects
girls and is characterized by early-onset, difficult-to-control
seizures and severe neuro‑developmental impairment. The CDKL5 gene
encodes proteins essential for normal brain function. Most
children affected by CDKL5 cannot walk, talk, or care for
themselves. Many also suffer from scoliosis, visual impairment,
gastrointestinal difficulties, and sleeping disorders. Currently,
there are no approved therapies for CDKL5 disorder. No previous
formal clinical trials have been conducted in this population.
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 its anti-seizure and
anti-anxiety activity. 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,
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.
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 clinical and preclinical studies, assessment
of positive nature and notability of preliminary data,
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
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