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 first quarter ended March 31, 2019.
“In 2019, Marinus continues to advance its ganaxolone clinical
programs for PPD, orphan pediatric genetic epilepsies and
refractory status epilepticus,” said Dr. Scott Braunstein,
executive chairman of Marinus. “We have seen strong
enrollment trends across our Phase 2 programs and have decided to
take advantage of this interest and increase the size of all three
of these studies with minimal impact to timelines. Both PPD
studies are fully enrolled and we will be expanding our RSE cohorts
to include up to 20 patients. As a result, we expect data
from the PPD studies in the first half of the third quarter and
data from the RSE study by the end of the third quarter. Data from
Magnolia and Amaryllis will be instrumental in shaping next steps
for ganaxolone in PPD and potentially depressive disorders more
broadly. In addition, we are excited by the promise of
ganaxolone as a second line treatment option for treating patients
with RSE.”
Q3 Clinical Data Milestones
- Top-line IV-to-oral ganaxolone data from Part 2 of the Phase 2
Magnolia study in women with severe postpartum depression
- Top-line oral ganaxolone dose-ranging data from the Phase 2
Amaryllis study in women with moderate Postpartum Depression
(PPD)
- Top-line data from the Phase 2 dose-ranging, proof-of-concept
study with IV ganaxolone in patients with refractory status
epilepticus
Postpartum Depression
Magnolia Study:
- Enrollment is complete in Part 2 of the ongoing Magnolia Phase
2 Study. The Magnolia Study is a two-part, Phase 2
double-blind, placebo-controlled study to evaluate the safety,
pharmacokinetics and efficacy of ganaxolone in mothers with severe
PPD. In Part 1 of the Magnolia Study, mothers received a
48-hour infusion followed by a 12-hour taper of ganaxolone
intravenous (IV). Previously reported study results showed
that ganaxolone was generally safe, well-tolerated and provided
reductions in HAM-D17 scores, including an early response at 48
hours and durability of effect through 30 days
post-treatment. Patients enrolled in Part 2 of the Magnolia
Study (n=33) receive a 6-hour infusion of ganaxolone IV (20 mg/hr)
followed by 28-days of oral ganaxolone (900 mg once daily).
Top-line data from the full dataset are expected in the third
quarter of 2019.
Amaryllis Study:
- Enrollment is complete in the Amaryllis Study, an ongoing
open-label, dose-escalation Phase 2 clinical study designed to
evaluate the safety, tolerability and efficacy of oral ganaxolone.
Previously reported interim results from the medium-dose cohort
showed that oral ganaxolone administered at 675 mg for four weeks
was generally safe, well-tolerated and provided a reduction in
HAM-D17 at day 36 consistent with IV administration in Part 1 of
the Magnolia Study. The additional 43 patients enrolled into the
high dose cohort of this study receive 675 mg of oral ganaxolone at
dinner and bedtime for two days, followed by a dinner time dose of
1125 mg once daily for the remainder of the 28-day treatment
regimen. Top-line data from the full dataset are expected in
the third quarter of 2019.
Orphan Pediatric Genetic Epilepsy Programs
CDKL5 Deficiency Disorder (CDD):
- Enrollment continues in the Marigold Study, the Company’s
pivotal Phase 3 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. This global,
double-blind, placebo-controlled, single pivotal Phase 3 clinical
study will enroll between 70 and 100 patients between the ages of 2
and 21 with a confirmed disease-related CDKL5 gene variant. The
Company is on-track for top-line data from this study by
mid-2020.
- Previously reported Phase 2 data from the twelve-month open
label extension demonstrated that four of seven patients who met
criteria for continued ganaxolone treatment beyond the main study
had a 54 percent median reduction in seizure frequency at six
months (primary endpoint), which increased to 66 percent for the
six-month interval that followed the primary endpoint.
PCDH19-Related Epilepsy (PCDH19-RE):
- In March, Marinus announced the initiation of a pivotal Phase 3
study evaluating oral ganaxolone in children with PCDH19-RE, the
Violet Study. This global, double-blind, randomized,
placebo-controlled single pivotal Phase 3 study is expected to
enroll up to 70 patients between the age of 1 and 17 with a
confirmed PCDH19 gene mutation. Patients will be stratified into
biomarker-positive and biomarker-negative groups. The Company
expects to begin enrollment screening in the second quarter of 2019
with top-line data expected in 2021.
Refractory Status Epilepticus (RSE)
- Enrollment is ongoing in the proof-of-concept, 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 at least one second-line IV
anti-epileptic drug with the aim of reducing the need for riskier
pharmacological escalations, which require anesthesia, intubation
and extensive cardiovascular monitoring while the patient is in a
medically induced coma. The primary endpoint for the study is the
number of patients who do not require a pharmacological treatment
escalation, including treatment with an IV anesthetic drug.
Improvement in patient EEG readings is a surrogate marker of
efficacy in this study. Top-line study data are expected in the
third quarter of 2019.
Financial UpdateAt March 31, 2019, the Company
had cash, cash equivalents and investments of $60.8 million,
compared to $72.7 million at December 31, 2018. We believe that our
cash and cash equivalents as of March 31, 2019 will enable us
to fund our current scale of operating expenses and capital
expenditure into the second half of 2020.
Research and development expenses increased to $8.9 million for
the three months ended March 31, 2019, as compared to $3.9 million
in the prior year. The increase for the three months ended
March 31, 2019 compared to 2018 was due primarily to increased
patient enrollment of the Magnolia study, expansion of the
Amaryllis Phase 2 study, and the initiation of our Violet Phase 3
study.
General and administrative expenses increased $1.5 million for
the three months ended March 31, 2019 compared to 2018. Of
this increase, $1.0 million was due to severance benefits due to
our former chief executive officer ($0.4 of which was non-cash
equity compensation expense) and the remainder relates to
professional fees and other costs associated with an increased
scale of operations.
The Company reported net losses of $12.5 million and $6.0
million for the three months ended March 31, 2019 and 2018,
respectively. Cash used in operating activities increased to
$11.7 million for the three months ended March 31, 2019
compared to $6.1 million for the same period a year ago.
Readers are referred to, and encouraged to read in its entirety,
the Company’s Quarterly Report on Form 10-Q for the quarter ended
March 31, 2019 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)
|
|
March
31,2019 |
|
|
December
31,2018 |
|
|
|
|
|
|
|
|
|
ASSETS |
|
|
Cash and cash
equivalents |
$ |
60,848 |
|
$ |
67,727 |
Investments |
|
— |
|
|
4,998 |
Other assets |
|
6,771 |
|
|
2,509 |
Total
assets |
$ |
67,619 |
|
$ |
75,234 |
LIABILITIES AND STOCKHOLDERS’ EQUITY |
|
|
Current liabilities |
$ |
6,677 |
|
$ |
6,909 |
Other long term
liabilities |
|
3,197 |
|
|
— |
Total liabilities |
|
9,874 |
|
|
6,909 |
Total stockholders’ equity |
|
57,745 |
|
|
68,325 |
Total
liabilities and stockholders’ equity |
$ |
67,619 |
|
$ |
75,234 |
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
|
2019 |
|
|
2018 |
|
|
|
|
|
|
|
|
|
|
|
|
Expenses: |
|
|
|
|
|
|
|
|
Research
and development |
|
$ |
8,872 |
|
|
$ |
3,927 |
|
|
|
General
and administrative |
|
|
3,667 |
|
|
|
2,187 |
|
|
|
Loss from
operations |
|
|
(12,539 |
) |
|
|
(6,114 |
) |
|
|
Interest income |
|
|
96 |
|
|
|
116 |
|
|
|
Other expense |
|
|
(40 |
) |
|
|
(1 |
) |
|
|
Net loss |
|
$ |
(12,483 |
) |
|
$ |
(5,999 |
) |
|
|
Per share
information: |
|
|
|
|
|
|
|
|
Net loss
per share of common stock—basic and diluted |
|
$ |
(0.24 |
) |
|
$ |
(0.15 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and
diluted weighted average shares outstanding |
|
|
52,465,207 |
|
|
|
40,373,083 |
|
|
|
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 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 has initiated
the first ever pivotal studies in children with CDKL5 deficiency
disorder and PCDH19-related epilepsy and is currently conducting
studies in women with postpartum depression and patients with
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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