Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a biopharmaceutical
company dedicated to the development of innovative therapeutics to
treat epilepsy and neuropsychiatric disorders, today provided a
business update on its clinical development activities and reported
its financial results for the year ended December 31, 2017.
Near-term Clinical Value
Catalysts
- Initiate Phase 3 pivotal study with oral ganaxolone in children
with CDD (CDKL5 Deficiency Disorder) mid-2018
- Report top-line intravenous (IV) ganaxolone data from Phase 2
Magnolia study in women with severe postpartum depression (PPD)
third quarter 2018.
- Report top-line oral ganaxolone data from Amaryllis study in
women with moderate PPD fourth quarter 2018.
“2017
was a significant year for Marinus, and one that has positioned us
for important data readouts and advancing ganaxolone into late
stage development in 2018,” said Christopher M. Cashman, chief
executive officer of Marinus Pharmaceuticals. “The data obtained
last September from our study in children with CDKL5 deficiency
disorder were impressive and drove our decision to advance this
program into a global, pivotal study which we will initiate this
year. We, along with the CDKL5 community of caregivers, physicians
and investigators, are excited to participate in this first ever
late-stage clinical trial for these children suffering from this
rare and debilitating epilepsy with no approved treatments or even
any reasonable standard of care. Similarly, our two phase 2 studies
in women suffering from severe and moderate PPD are expected to
generate data with IV and oral regimens of ganaxolone this year to
support the design and initiation of pivotal Phase 3 studies next
year. We remain focused on our goal of being able to treat
underserved patient populations with patient-convenient, setting
appropriate, effective and safe treatment regimens.”
CDKL5 Deficiency Disorder (CDD)
- In November, the Company presented the successful results from
its Phase 2 study evaluating the safety and efficacy of ganaxolone
in children with CDD at the annual CDKL5 Forum in Boston. Marinus
was recognized as the leader in clinical research in CDD. The data
presented showed that ganaxolone provided substantial and durable
anti-seizure efficacy in children with CDD and was generally safe
and well-tolerated with no serious adverse events.
- The last patient enrolled into the Phase 2 study, recently
completed the six-month treatment period, and based on good seizure
control, entered the one-year extension to the study. The majority
of CDD patients from the Phase 2 study entered the one-year study
extension and continue to receive ganaxolone.
- The Company is engaging in successful, collaborative
discussions with regulatory agencies and expects to initiate a
global, pivotal study in mid-2018.
Postpartum
Depression (PPD)
- Marinus is enrolling patients into the Magnolia Study, a Phase
2 double-blind, placebo-controlled, dose-optimization clinical
trial to evaluate the safety, efficacy and pharmacokinetics of
ganaxolone in women diagnosed with severe PPD (Hamilton Depression
Rating Scale (HAMD17) score ≥26). Patients randomized into the
first part of the study will undergo an infusion of either
ganaxolone or placebo and will be followed for 30 days. The goal of
the first part of the study is to evaluate multiple regimens of
intravenous (IV) ganaxolone, which will inform dosing for the
second part of the study. Patients enrolled into the second
part of the study could receive IV ganaxolone of various infusion
lengths followed by administration of oral ganaxolone. The goal of
the second part of the study is to identify an optimized dose or
doses for further testing in phase 3. Based upon the effect size
shown in a recent study for a compound with similar mechanism of
action, the Company has increased targeted enrollment in this
study. This increase in study scope and the corresponding
forecast for patient recruitment have extended our expected timing
for completion of the first part to the third quarter of 2018.
- The Company is enrolling patients into its Amaryllis Study, a
Phase 2 double-blind, placebo-controlled clinical trial to evaluate
the safety, tolerability and efficacy of oral ganaxolone in women
with moderate PPD (HAMD17 score > 20 and < 26). The study is
designed to enroll approximately 50 women at 20 sites within the
US. Data from this study are expected fourth quarter of
2018.
Status Epilepticus (SE)
- The Company has initiated its Phase 2 study with ganaxolone IV
in patients with refractory status epilepticus (RSE). Data
from this proof-of-concept study are expected fourth quarter of
2018.
Financial Update
At December 31, 2017, the Company had cash, cash
equivalents and investments of $58.4 million, compared to
$30.1 million at December 31, 2016. We believe that our cash,
cash equivalents and investments as of December 31, 2017 will
enable us to fund our current scale of operating expenses and
capital expenditure requirements into 2020.
Research and development expenses decreased to
$12.4 million for the year ended December 31, 2017, as compared to
$22.0 million in the prior year. The decrease was
primarily due to a decrease of $11.0 million associated with our
drug-resistant focal onset seizures program, which we discontinued
in June 2016. Additionally, we sold $0.4 million in
state research and development tax credits which we used to offset
research and development expenses. The decrease was
partially offset by an increase of $2.3 million associated with our
IV programs in PPD, for which a Phase 2 clinical trial was
initiated in June 2017.
General and administrative expenses increased
$0.4 million, to $6.7 million, for the year ended
December 31, 2017, compared to 2016. The increase in general
and administrative expenses was primarily due to an increase in
noncash stock-based compensation expense.
The Company reported net losses of $18.9 million
and $28.6 million for the years ended December 31, 2017 and 2016,
respectively. Cash used in operating activities was
$18.8 million for the year ended December 31, 2017 compared to
$24.8 million for the same period a year ago.
Readers are referred to, and encouraged to read
in its entirety the Company’s Annual Report on Form 10-K for the
year ended December 31, 2017 to be filed with the Securities and
Exchange Commission, which includes further detail on the
above-referenced transactions and the Company’s business plans and
operations, financial condition and results of operations.
|
Marinus Pharmaceuticals,
Inc.Selected Financial Data (in thousands, except
share and per share amounts)
(unaudited) |
|
|
|
December 31,2017 |
|
December 31,2016 |
|
|
|
|
|
|
|
|
|
|
ASSETS |
|
|
Cash and cash
equivalents |
$33,531 |
$26,178 |
Investments |
|
24,825 |
|
3,922 |
Other assets |
|
2,316 |
|
1,347 |
Total
assets |
|
$60,672 |
|
$31,447 |
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
Total current
liabilities |
$2,544 |
$8,084 |
Notes payable,
long-term portion |
|
— |
|
1,743 |
Other long term
liabilities |
|
120 |
|
141 |
Total
liabilities |
|
2,664 |
|
9,968 |
Total
stockholders’ equity |
|
58,008 |
|
21,479 |
Total
liabilities and stockholders’ equity |
|
$60,672 |
|
$31,447 |
|
|
|
|
|
|
|
|
|
|
Year Ended December 31, |
|
|
|
2017 |
|
|
2016 |
|
|
|
|
|
|
|
|
|
|
Expenses: |
|
|
|
|
|
|
|
Research
and development |
|
$ |
12,376 |
|
|
$ |
22,005 |
|
|
General
and administrative |
|
|
6,667 |
|
|
|
6,237 |
|
|
Loss from
operations |
|
|
(19,043 |
) |
|
|
(28,242 |
) |
|
Interest income |
|
|
324 |
|
|
|
128 |
|
|
Interest expense |
|
|
(159 |
) |
|
|
(464 |
) |
|
Other expense |
|
|
(20 |
) |
|
|
(65 |
) |
|
Net loss |
|
$ |
(18,898 |
) |
|
$ |
(28,643 |
) |
|
Per share
information: |
|
|
|
|
|
|
|
Net loss
per share of common stock—basic and diluted |
|
$ |
(0.80 |
) |
|
$ |
(1.47 |
) |
|
Basic and
diluted weighted average shares outstanding |
|
|
23,540,738 |
|
|
|
19,498,143 |
|
|
|
|
|
|
|
|
|
|
|
|
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 preparing to initiate a pivotal study in children
with CDKL5 deficiency disorder, a rare form of epilepsy, and
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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