Marinus Pharmaceuticals, Inc. (Nasdaq:MRNS), a pharmaceutical
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, 2018.
First Half 2019 Clinical
Milestones
- Report top-line IV-to-oral ganaxolone data from Part 2 of the
Phase 2 Magnolia study in women with postpartum depression
(PPD).
- Report top-line oral ganaxolone dose-ranging data from the
Phase 2 Amaryllis study in women with PPD.
- Report top-line data from the Phase 2 dose-ranging,
proof-of-concept study with IV ganaxolone in patients with
refractory status epilepticus (RSE).
“2018 was a pivotal year for Marinus, kicking
off the first-ever single pivotal Phase 3 study in children with
CDKL5 deficiency disorder, and reporting positive results from Part
1 of our Magnolia Study with IV ganaxolone in women with postpartum
depression,” said Christopher M. Cashman, CEO of Marinus. “As my
tenure at Marinus comes to a close, I feel confident that the
company is well positioned to further advance ganaxolone into
additional late-stage studies and prepare for
commercialization.”
Dr. Scott Braunstein, Executive Chairman of
Marinus commented, “Through the multiple clinical studies conducted
to date, we have shown that ganaxolone is safe, well-tolerated and
effective in reducing seizures, anxiety and depression. I look
forward to working with the Marinus team to unlock the value of
ganaxolone, not only in the indications under development, but also
into other refractory epilepsy and neuropsychiatric disorders. I
look forward to keeping shareholders apprised of our developments
in the upcoming months.”
Postpartum Depression (PPD)
Magnolia Study:
- Enrollment is ongoing in Part 2 of the Magnolia Phase 2 Study
to evaluate the safety, pharmacokinetics (PK) and efficacy of a
six-hour intravenous (IV) infusion of ganaxolone followed by
28-days of oral ganaxolone in women with PPD. Patients randomized
to ganaxolone in the current dose cohort receive 20 mg/hr of
ganaxolone IV (> 140 µg/kg/h), for six hours followed by an
evening 900 mg dose of oral ganaxolone on day one. A once daily 900
mg evening dose of oral ganaxolone is then administered for the
duration of treatment. The study is on-track to report top-line
data in the second quarter of 2019.
- In December, Marinus announced results from the first part of
the Magnolia Study, which evaluated a 60-hour infusion of multiple
fixed doses of ganaxolone IV in women with PPD. The best
performing median weight-based dose of ganaxolone IV (140 µg/kg/h)
was safe and well tolerated as were all cohorts. Patients in this
dose group saw a mean HAM-D17 score reduction of 15.1 points at
48-hours (from a mean baseline of 27.1), which separated from
placebo by 5.6 points. These patients maintained a 15.7 point
reduction at day 34, which separated from placebo by 4.1 points. We
believe that this early response and durability of effect is
clinically meaningful and compares favorably to traditional
antidepressants, as well as other drug candidates in
development.
Amaryllis Study:
- Enrollment is ongoing in the open-label, dose-escalation
portion of the Amaryllis study, a Phase 2 clinical study to
evaluate the safety, tolerability and efficacy of oral ganaxolone
in women with PPD. Patients enrolled in the current dose cohort
receive 675 mg of oral ganaxolone at dinner and before bedtime for
the first two days of treatment, and then receive a once-daily
1,125 mg evening dose of oral ganaxolone for the remainder of the
28-day regimen. Top-line data from the additional cohort(s) of
patients, along with top-line data from the full dataset, are
expected in the second quarter of 2019.
- In December, Marinus announced initial results from the
Amaryllis Study, in which a low dose and moderate dose of
ganaxolone were studied. Patients who received a once-daily 675 mg
evening (moderate) dose of oral ganaxolone for four weeks had a
mean HAM-D17 reduction of 15.7 points (from a baseline of 24.7
points) at day 36. These data, along with data from Part 1 of the
Magnolia Study, have informed the oral dosing of ganaxolone that is
currently being evaluated. As with ganaxolone IV, oral ganaxolone
in this study was generally safe and well-tolerated with no serious
adverse events reported and no discontinuations due to
treatment-related adverse events.
Orphan Pediatric Genetic Epilepsy
Programs
CDKL5 Deficiency Disorder (CDD):
- Enrollment is on-going in the Company’s pivotal Phase 3
clinical study (Marigold Study) evaluating the use of oral
ganaxolone in children and young adults with CDD, a refractory form
of pediatric epilepsy with no currently approved treatments. The
Marigold Study is a global, double-blind, placebo-controlled,
single pivotal Phase 3 clinical study that will enroll
approximately 70 patients between the ages of 2 and 21 with a
confirmed disease-related CDKL5 gene variant. The study will
consist of a six-week prospective baseline period to collect
seizure data, followed by a 17-week double-blind treatment
phase. Patients randomized to ganaxolone will titrate over
four weeks to a dose of up to 600 mg of oral liquid suspension
three times a day, and maintain that dose for the following
13-weeks. The Company is on-track to complete enrollment in this
study by the end of 2019 with top-line data expected by
mid-2020.
- In December the Company presented long-term data from the
patients that entered the six-month open label extension of the
Phase 2 clinical study in CDD. The four of seven patients who
continued on ganaxolone beyond the main study experienced a 54
percent average reduction in seizure frequency at the extension
study primary endpoint at six months (12 months total treatment
with ganaxolone).
PCDH19-Related Epilepsy (PCDH19-RE):
- Earlier this month Marinus announced the initiation of the
Violet Study, a global, double-blind, randomized,
placebo-controlled single pivotal Phase 3 study evaluating
ganaxolone in children with PCDH19-RE. The study will enroll up to
70 patients between the age of 1 and 17 with a confirmed PCDH19
mutation. Patients enrolled in the study will be stratified into
one of two biomarker groups and randomized (ganaxolone or placebo)
within each stratum. The study will consist of an eight-week
prospective baseline period to collect seizure data, followed by a
17-week double-blind treatment phase. Patients randomized to
ganaxolone will titrate over four weeks to a dose of up to 600 mg
of oral liquid suspension three times a day and maintain that dose
for the following 13-weeks. After the double-blind period, all
patients who meet certain eligibility criteria will have the
opportunity to receive ganaxolone in an open-label phase of the
study. The Company expects to begin screening patients for
enrollment into the study in the second quarter of 2019 with
top-line data from the study expected in 2021.
- In December, the Company presented additional data at the
American Epilepsy Society annual meeting from its Phase 2 study in
patients with PCDH19-RE, which identified evidence of a plasma
neurosteroid biomarker. The post-treatment review of baseline
plasma neurosteroid levels in patients with PCDH19-RE revealed what
we believe to be a significant association between these levels and
response to ganaxolone treatment. Patients with a very low level of
a specific neurosteroid showed a medically notable reduction in
seizure frequency when treated with ganaxolone as compared to
patients with a very high level. The difference in the levels of
this neurosteroid between responders and non-responders was
approximately 1.5 orders of magnitude.
Refractory Status Epilepticus
(RSE)
- Enrollment is continuing in the open-label portion of the Phase
2 study evaluating the tolerability, efficacy and PK of ganaxolone
IV in patients with RSE. Ganaxolone IV is being administered as
second line treatment after a patient has failed benzodiazepines
and two IV anti-epileptic drugs. The primary endpoint for the study
is the number of patients who do not require a pharmacological
treatment escalation, including IV anesthetic drug. Improvement in
patient EEG readings will be used as a surrogate marker of efficacy
in this study. Top-line data from this proof-of-concept,
dose-ranging study are expected in the second quarter of 2019.
Financial Update
At December 31, 2018, the Company had cash, cash
equivalents and investments of $72.7 million, compared to
$58.4 million at December 31, 2017. We believe that our cash, cash
equivalents and investments as of December 31, 2018 will
enable us to fund our current scale of operating expenses and
capital expenditure requirements into 2021.
Research and development expenses increased to
$28.4 million for the year ended December 31, 2018, as compared to
$12.4 million in the prior year. The increase for the year
ended December 31, 2018 compared to 2017 was due primarily to
increased patient enrollment of the Magnolia study, expansion of
the Amaryllis Phase 2 study, and additional preclinical studies and
manufacturing activities, in support of all formulations and
indications.
General and administrative expenses increased
$2.1 million for the year ended December 31, 2018 compared to
2017, primarily due to an increase in noncash, stock-based
compensation expense.
The Company reported net losses of $36.7 million
and $18.9 million for the years ended December 31, 2018 and 2017,
respectively. Cash used in operating activities increased to
$27.8 million for the year ended December 31, 2018 compared to
$18.8 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, 2018 to be filed with the Securities and
Exchange Commission, which includes further detail on the
above-referenced transactions and the Company’s business plans,
operations, financial condition and results of operations.
Marinus Pharmaceuticals,
Inc.Selected Financial Data (in thousands, except
share and per share amounts)
(unaudited)
|
|
December 31, |
|
|
|
|
2018 |
|
|
2017 |
|
|
|
|
|
|
|
|
|
|
|
ASSETS |
|
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
|
Cash and
cash equivalents |
|
$ |
67,727 |
|
|
$ |
33,531 |
|
|
Investments |
|
|
4,998 |
|
|
|
24,825 |
|
|
Other
assets |
|
|
2,509 |
|
|
|
2,316 |
|
|
Total
assets |
|
$ |
75,234 |
|
|
$ |
60,672 |
|
|
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
|
|
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
|
|
|
|
Accounts
payable |
|
$ |
2,472 |
|
|
$ |
927 |
|
|
Accrued
expenses |
|
|
4,437 |
|
|
|
1,617 |
|
|
Total
current liabilities |
|
|
6,909 |
|
|
|
2,544 |
|
|
Other long-term
liabilities |
|
|
— |
|
|
|
120 |
|
|
Total
liabilities |
|
|
6,909 |
|
|
|
2,664 |
|
|
Total stockholders’ equity |
|
|
68,325 |
|
|
|
58,008 |
|
|
Total
liabilities and stockholders’ equity |
|
$ |
75,234 |
|
|
$ |
60,672 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Year Ended December 31, |
|
|
|
2018 |
|
|
2017 |
|
|
|
|
|
|
|
|
|
|
Expenses: |
|
|
|
|
|
|
|
Research and
development |
|
$ |
28,394 |
|
|
$ |
12,376 |
|
|
General
and administrative |
|
|
8,785 |
|
|
|
6,667 |
|
|
Loss from
operations |
|
|
(37,179 |
) |
|
|
(19,043 |
) |
|
Interest income |
|
|
454 |
|
|
|
324 |
|
|
Interest expense |
|
|
— |
|
|
|
(159 |
) |
|
Other expense |
|
|
(1 |
) |
|
|
(20 |
) |
|
Net loss |
|
$ |
(36,726 |
) |
|
$ |
(18,898 |
) |
|
Per share
information: |
|
|
|
|
|
|
|
Net loss
per share of common stock—basic and diluted |
|
$ |
(0.90 |
) |
|
$ |
(0.80 |
) |
|
Basic and
diluted weighted average shares outstanding |
|
|
40,895,406 |
|
|
|
23,540,738 |
|
|
|
|
|
|
|
|
|
|
|
|
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 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 studies in children
with CDKL5 deficiency disorder and PCDH19-related 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 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. CaperelliExecutive Director,
Investor & Strategic RelationsMarinus Pharmaceuticals,
Inc.484-801-4674lcaperelli@marinuspharma.com
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