Data from Studies in Rare Pediatric Epilepsies
and Severe Postpartum Depression Expected in 2nd Half
Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS) (the “Company”), a
biopharmaceutical company dedicated to the development of
innovative therapeutics to treat epilepsy and neuropsychiatric
disorders, today provided a business update and reported its
financial results for the quarter ended June 30, 2017.
“This quarter Marinus significantly advanced the
development of ganaxolone, which positions us to achieve several
value-creating milestones in the second half of this year,” said
Christopher M. Cashman, Chief Executive Officer of Marinus
Pharmaceuticals. “With intravenous dosing occurring in our Magnolia
study and plans to initiate the Amaryllis study with ganaxolone
capsules, we are well positioned to evaluate inpatient and
outpatient treatments with ganaxolone in women diagnosed with both
severe and moderate PPD.
We expect to announce top-line data from our
Phase 2 trial evaluating children with CDKL5 disorder this quarter.
We are interested in ganaxolone’s anti-seizure and anti-anxiety
activity in improving the quality of life for these children and
their families who suffer from seizure and behavioral
co-morbidities.”
Second Quarter Clinical Highlights and
Updates
- Initiated the Magnolia Study, a phase 2 double-blind,
placebo-controlled clinical trial to evaluate the safety, efficacy
and pharmacokinetics of intravenous (IV) ganaxolone in women
diagnosed with severe PPD. The study consists of multiple cohorts
of women with a Hamilton Depression Rating Scale (HAMD17) score
≥26. Patients randomized to the first study cohort will undergo an
infusion of either ganaxolone or placebo and will be followed for
at least 30 days. Subsequent cohorts could include shorter- or
higher-dose IV regimens alone, or in sequential administration with
ganaxolone capsules.
- Received Orphan Drug Designation from the U.S. Food and Drug
Administration (FDA) for ganaxolone for the treatment of CDKL5
disorder. CDKL5 disorder is a severe, rare genetic disorder
with no approved therapies that affects children at an early age
and causes difficult-to-control seizures and neuro-developmental
impairment.
- Preparations are underway to initiate the Amaryllis Study later
this year. This Phase 2 study will evaluate ganaxolone capsules in
women with moderate PPD. Ganaxolone capsules have the potential to
provide the largest segment of the PPD patient population with
access to convenient, oral outpatient therapy. The Company plans to
leverage the extensive safety profile associated with ganaxolone
capsules to advance the regulatory path of this program. The
specific details of the trial design and estimated completion
timing will be announced at the time of study initiation.
- Preparations are underway to initiate a Phase 2 study in
patients with SE later this year. If SE is not treated
immediately, permanent neuronal damage may occur, which contributes
to high rates of morbidity and mortality. The Company’s approach to
address SE is differentiated from other drugs in development.
The ganaxolone treatment regimen will target refractory
patients with the goal of stopping the seizures earlier and
preventing the need for medically-induced coma, which should result
in better outcomes for these patients. The specific details of the
trial design and estimated completion timing will be announced at
the time of study initiation.
Financial Update
At June 30, 2017, the Company had cash, cash
equivalents and investment balances of $20.3 million. The
Company believes that its cash, cash equivalents and investments,
as of June 30, 2017, inclusive of subsequent financing activities,
are adequate to fund operations into the fourth quarter of
2018.
Research and development expenses were $2.8
million and $6.4 million for the three and six months ended June
30, 2017, respectively, a decrease of $4.4 million and $6.4 million
for the three and six month periods ended June 30,
respectively. The decrease was primarily associated with our
drug-resistant focal onset seizures program, which discontinued in
June 2016, and partially offset by an increase of $0.3 million and
$1.0 million for the three and six month periods ended June 30,
respectively, associated with our IV programs in PPD and
SE.
General and administrative expenses were $1.7
million and $3.5 million for the three and six months ended June
30, 2017, respectively, as compared to similar expense amounts of
$1.6 million and $3.2 million for the same periods in the
prior year.
Cash used in operating activities
$11.2 million for the six months ended June 30, 2017, a
decrease of $1.0 million from the same period a year ago. The
change in operating activities was driven primarily by a decrease
in net loss of $6.2 million, partially offset by a decrease in the
change in operating assets and liabilities of $5.2
million.
Readers are referred to, and encouraged to read
in its entirety, the Company’s Quarterly Report on Form 10-Q for
the quarter ended June 30, 2017 to be filed with the Securities and
Exchange Commission, which includes further detail on the Company’s
business plans and operations, financial condition and results of
operations.
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
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 women with PPD and 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. 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”,
“should”,“anticipate”, “estimate”, “intend”, “believe”, “on-track”,
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.
Marinus Pharmaceuticals, Inc. |
Selected Financial Data (in thousands, except
share and per share amounts) |
(unaudited) |
|
|
|
|
|
June 30,2017 |
|
December
31,2016 |
|
|
|
|
|
|
|
|
ASSETS |
|
|
|
Cash and cash
equivalents |
$ |
19,531 |
|
$ |
26,178 |
Investments |
744 |
|
3,922 |
Other assets |
1,820 |
|
1,347 |
Total
assets |
$ |
22,095 |
|
$ |
31,447 |
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
Total current
liabilities |
$ |
5,674 |
|
$ |
8,084 |
Notes payable,
long-term portion |
— |
|
1,743 |
Other long term
liabilities |
132 |
|
141 |
Total
liabilities |
5,806 |
|
9,968 |
Total
stockholders’ equity |
16,289 |
|
21,479 |
Total
liabilities and stockholders’ equity |
$ |
22,095 |
|
$ |
31,447 |
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
|
2017 |
|
|
2016 |
|
|
2017 |
|
|
2016 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
$ |
2,817 |
|
|
$ |
7,258 |
|
|
$ |
6,390 |
|
|
$ |
12,752 |
|
|
General
and administrative |
|
|
1,691 |
|
|
|
1,586 |
|
|
|
3,503 |
|
|
|
3,190 |
|
|
Loss from
operations |
|
|
(4,508 |
) |
|
|
(8,844 |
) |
|
|
(9,893 |
) |
|
|
(15,942 |
) |
|
Interest income |
|
|
31 |
|
|
|
34 |
|
|
|
71 |
|
|
|
56 |
|
|
Interest expense |
|
|
(72 |
) |
|
|
(124 |
) |
|
|
(156 |
) |
|
|
(248 |
) |
|
Other expense |
|
|
(3 |
) |
|
|
(15 |
) |
|
|
(12 |
) |
|
|
(31 |
) |
|
Net loss |
|
$ |
(4,552 |
) |
|
$ |
(8,949 |
) |
|
$ |
(9,990 |
) |
|
$ |
(16,165 |
) |
|
Per share
information: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
per share of common stock—basic and diluted |
|
$ |
(0.21 |
) |
|
$ |
(0.46 |
) |
|
$ |
(0.47 |
) |
|
$ |
(0.83 |
) |
|
Basic and
diluted weighted average shares outstanding |
|
|
21,985,213 |
|
|
|
19,509,220 |
|
|
|
21,288,545 |
|
|
|
19,486,944 |
|
|
CONTACT:
Lisa M. Caperelli
Executive Director, Investor & Strategic Relations
Marinus Pharmaceuticals, Inc.
484-801-4674
lcaperelli@marinuspharma.com
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