Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) (“Marinus” or
“Company”), a pharmaceutical company dedicated to the development
of innovative therapeutics to treat epilepsy and other
neuropsychiatric disorders, today provided a corporate update for
investors, including its roadmap for advancing ganaxolone in 2020.
Key 2020 Milestones:
- Initiate Phase 2 clinical trial in patients with Tuberous
Sclerosis Complex (TSC) – 1H 2020
- Initiate U.S. pivotal Phase 3 clinical trial in patients with
Status Epilepticus (SE) – mid-2020
- Report top-line data from global, pivotal Phase 3 clinical
trial in children with CDKL5 Deficiency Disorder (CDD) – Q3
2020
“Our 2019 clinical progress, along with the bolstering of our
executive leadership and balance sheet, now positions us to begin
to unlock the value of our ganaxolone franchise,” said Scott
Braunstein, M.D., Chief Executive Officer of Marinus. “Our strategy
is focused on mechanistically relevant disease states for
ganaxolone that have the potential to significantly improve
outcomes for patients. With our Phase 3 study reading out in CDKL5
deficiency disorder later this year, we are making preparations for
our first NDA filing and commercial launch with oral
ganaxolone. In addition, we are advancing our
hospital-directed intravenous dose form into a Phase 3, potentially
NDA enabling, study in status epilepticus later this year.”
Clinical Development
OverviewMarinus is developing an oral and intravenous (IV)
formulation of ganaxolone to treat adults and children suffering
from acute and chronic rare neuropsychiatric conditions where there
is a mechanistic rationale for ganaxolone to provide a benefit.
Unlike benzodiazepines, ganaxolone exhibits anti-seizure,
anti-anxiety, and antidepressant activity via its effects on
synaptic and extrasynaptic GABAA receptors.
Status Epilepticus (SE)Following the successfully completed
phase 2 study in patients with refractory SE, Marinus is preparing
for an End-of-Phase 2 meeting with the FDA, which is expected
in the first quarter of 2020. Status epilepticus is a rare
condition consisting of a prolonged state of continuous or
near-continuous seizure activity that can cause permanent damage to
the brain and even death if not quickly brought under
control. The Phase 2 dose-finding trial enrolled 17 medically
heterogeneous patients who received an infusion of IV ganaxolone,
added to second line standard of care antiepileptic drugs for the
treatment of SE. In the study, 100% of patients achieved the
primary endpoint of preventing progression to IV anesthetics within
24 hours of treatment initiation. The median time to SE cessation
was five minutes across all doses evaluated and a numerical dose
response trend was observed in evaluable patients at clinically
relevant early timepoints and the four-week long-term follow-up
visit. Every patient receiving the identified target dose (713
mg/day) met the primary endpoint and did not have recurrence of SE
up to the long-term follow-up visit. This target dose will be
further evaluated in a pivotal study that is planned to commence in
mid-2020. Additionally, the Company plans to request
scientific advice later this year to discuss the approval pathway
for ganaxolone in Europe.
CDKL5 Deficiency Disorder (CDD)Marinus is
currently in the final stages of recruitment in the Marigold Study,
its pivotal Phase 3 study evaluating oral ganaxolone in children
and young adults with CDD, a rare refractory form of pediatric
epilepsy with no currently approved treatments. The global,
double-blind, placebo-controlled, pivotal study will enroll
approximately 100 patients between the ages of 2 and 21 with a
confirmed disease-related CDKL5 gene variant. The Company remains
on-track to report top-line data in Q3 2020. If the Phase 3 CDD
study is successful, this could be the first approved indication
for ganaxolone and the first approved treatment for CDD. Marinus
has received orphan drug designation from both the US FDA and The
European Medicines Agency (EMA) for ganaxolone for the treatment of
CDD.
PCDH19-related Epilepsy (PCDH19-RE)
International site initiation and enrollment is continuing in
the Violet Study, a biomarker stratified pivotal Phase 3 study
evaluating oral ganaxolone in children with PCDH19-RE.
PCDH19-RE is a serious and rare epileptic disease characterized by
highly variable early-onset cluster seizures with comorbid
cognitive and behavioral disturbances with or without intellectual
disability. Currently, there are no approved therapies for
PCDH19-RE. The Violet Study will enroll up to 70 patients between
the ages of 1 and 17 with a confirmed PCDH19 mutation.
Patients are stratified into biomarker positive and negative groups
for Allo-S, which could potentially provide the epilepsy community
with the first diagnostic blood test that predicts the likelihood
of a treatment response. Top-line data from the Violet study are
expected in 2021.
Tuberous Sclerosis Complex (TSC)Marinus is planning to initiate
a Phase 2, open label study to evaluate the safety and tolerability
of adjunctive ganaxolone treatment in patients with seizures
associated with TSC. TSC, a leading cause of genetic epilepsy, is a
rare genetic disorder that affects many organs and causes
non-malignant tumors in the brain, skin, kidney, heart, eyes, and
lungs. The decision to expand the ganaxolone epilepsy program in
TSC was strategically informed by the discovery of a potential new
epilepsy biomarker, Allo-S, in the Phase 2 study in PCDH19-RE. This
led to additional analyses that identified TSC as another rare
genetic disorder that may be similarly impacted by Allo-S
levels.
The planned Phase 2 study will be conducted at approximately 4-6
sites in the United States and enroll 20-40 patients ages 2 to
65. The primary endpoint for the study is percent change in
28-day primary seizure frequency through the end of the 12-week
treatment period relative to the 4-week baseline period.
Dr. Joe Hulihan, Chief Medical Officer stated, “Our clinical
momentum in 2019 has further increased our confidence that
ganaxolone has the potential to transform the treatment paradigm
for a number of severe and life-threatening epilepsies with no
currently approved treatments. We are proud of our progress and
remain committed to the rapid advancement of ganaxolone for
patients in need.”
Presentation at 38th Annual J.P. Morgan Global
Healthcare ConferenceDr. Scott Braunstein will present a
corporate overview at the 38th Annual J.P. Morgan Global Healthcare
Conference on Thursday, January 16, 2020 at 11:00am Pacific Time.
The conference will take place at the Westin St. Francis Hotel in
San Francisco, California. A live webcast of J.P. Morgan 2020
Global Healthcare presentation may be accessed on the “Investors”
tab of the company’s website, www.marinuspharma.com. An
archived version of the presentation will be available for 30
days.
About Marinus Pharmaceuticals Marinus
Pharmaceuticals, Inc. is a pharmaceutical 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 depression.
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 IV and oral dose forms intended to maximize
therapeutic reach to adult and pediatric patient populations in
both acute and chronic care settings. Marinus is conducting
the first ever pivotal studies in children with CDKL5 deficiency
disorder and PCDH19-related epilepsy. Based on results from a
recent Phase 2 study in refractory SE and from biomarker analysis
research, the Company intends to initiate later this year a Phase 3
study in SE and a Phase 2 study in Tubular Sclerosis Complex (TSC),
respectively. 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 study testing
schedule and milestones, the ability to complete enrollment in our
clinical studies, 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 studies, the timing of the clinical studies, enrollment in
clinical studies, availability of data from ongoing clinical
studies, expectations for regulatory approvals, the attainment of
clinical study 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. Caperelli Executive Director,
Investor & Strategic Relations Marinus Pharmaceuticals, Inc.
484-801-4674 lcaperelli@marinuspharma.com
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