- ZTALMY® (ganaxolone) U.S. commercial launch on track for July
2022
- Phase 3 RAISE trial in refractory status epilepticus continues
to advance with resumption of screening and recruitment; data
readout expected second half 2023
- Site activations advance in Phase 3 TrustTSC trial in tuberous
sclerosis complex; topline data anticipated first quarter 2024
- Patient dosing underway for Phase 1 trial of ganaxolone second
generation formulation; topline data on track for mid-2022
readout
- Continue to strengthen cash position with Oaktree Capital
drawdown and expectation to monetize priority review voucher
- Marinus to host conference call today at 4:05 p.m. ET
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat seizure disorders, today reported business highlights and
financial results for the first quarter ended March 31, 2022.
“It’s been a productive year thus far as we prepare for our
first commercial launch and make important advancements across our
clinical programs. We believe the diligent efforts of our internal
teams to achieve the resumption of our Phase 3 RAISE trial in
refractory status epilepticus this month keeps us on track for this
key trial, and our next generation formulation development will
provide meaningful data this year for our next steps in new
indications, such as Lennox-Gastaut syndrome,” said Scott
Braunstein, M.D., Chief Executive Officer of Marinus
Pharmaceuticals. “I look forward to building on the strong
foundation we’ve established as we continue to grow the ganaxolone
franchise to help patients suffering from rare epilepsies.”
Commercial Preparedness
- ZTALMY® (ganaxolone) oral suspension on track to launch
commercially in the U.S. in July through a designated specialty
pharmacy following scheduling by the U.S. Drug Enforcement
Administration
- Market Access team strengthened, and field force fully
onboarded with payer and customer engagement underway
- “Now Approved” marketing campaign live and comprehensive
marketing campaign on track for July launch
- The Marigold pivotal trial results recently published in The
Lancet Neurology will be available for healthcare providers and
payers
- Preparations continue to advance for a simultaneous launch of
The ZTALMY One™ Program, a comprehensive patient services program
to provide assistance to healthcare providers, patients and
caregivers with product access and ongoing product support, and
offers programs for eligible patients who need financial support
for their ZTALMY prescription
Clinical Pipeline Update
CDKL5 Deficiency Disorder (CDD)
- Targeting submission of responses to the European Medicine
Agency (EMA) Day 120 List of Questions for the Marketing
Authorization Application (MAA) by mid-year which would result in
the EMA Committee for Medicinal Products for Human Use opinion on
the MAA by year end 2022
- Targeting business collaborations to expand commercial
footprint to additional ex-U.S. geographies by year end 2022
- Committed to identifying opportunities throughout the world to
improve the lives of more patients, including growing the CDD
Expanded Access Program to Europe
Tuberous Sclerosis Complex
(TSC)
- Actively recruiting patients at U.S. sites for the Phase 3
TrustTSC trial
- Site activations advance with 58 sites selected and additional
sites being identified globally
- Expect to expand trial to include a target of 65-75 sites,
including several TSC centers of excellence, predominantly in the
U.S., Western Europe, Canada and Israel
- Topline data targeted for first quarter of 2024
Status Epilepticus
- Resumed Phase 3 RAISE trial in refractory status epilepticus
(RSE) with screening, recruitment and U.S. site activations
continuing to advance
- Target sites expanded to include U.S., Canada, Israel and
Australia
- Topline Phase 3 trial results continue to be expected in the
second half of 2023
- Consistent with the projected topline data readout, the BARDA
research contract extended through 2023*
- Phase 2 RESET trial in established status epilepticus is on
track to begin U.S. enrollment in the second half of 2022
- Topline data from the first dose-finding cohorts anticipated by
year end 2023
- Phase 3 RAISE II trial in RSE (for European registration)
expected to begin enrollment in the first half of 2023
*Ganaxolone development in the RAISE trial is being funded in
part by the Biomedical Advanced Research and Development Authority
(BARDA), part of the Office of the Assistant Secretary for
Preparedness and Response at the U.S. Department of Health and
Human Services, under contract number 75A50120C00159.
Next Generation Ganaxolone; Lennox-Gastaut
Syndrome (LGS)
- Phase 1 patient dosing underway for second generation
ganaxolone formulation
- Topline data on track for mid-2022 readout
- Following evaluation of Phase 1 data, Phase 2 LGS trial is
expected to begin in the second half of 2022 utilizing second
generation formulation
- Anticipate initiating modified release candidate development in
2022
- Second formulation candidate selected with the potential for
improved pharmacokinetic profile and reduced dosing frequency;
expected to begin Phase 1 development in the first quarter of
2023
- Two prodrug candidates have been identified and are targeted to
advance into Investigational New Drug (IND) enabling studies in the
second half of 2022
Financial Updates
- In March 2022, Marinus received an additional $30 million in
funding under the existing Oaktree Capital Management, L.P.
(Oaktree) credit agreement. This additional funding became
available as a result of the U.S. Food and Drug Administration’s
(FDA) recent approval of ZTALMY.
- Marinus was awarded a rare pediatric disease priority review
voucher (PRV) by the FDA in March 2022 as a result of the ZTALMY
approval; Marinus remains committed to monetizing this PRV with
plans actively underway.
Financial Results
- At March 31, 2022, the company had cash and cash equivalents of
$126.3 million, compared to $122.9 million at December 31,
2021.
- Marinus recognized $14.2 million in revenue for the three
months ended March 31, 2022, as compared to $1.8 million for the
three months ended March 31, 2021. The increase in 2022 revenue was
driven by a one-time revenue recognition of $12.7 million related
to the previously received upfront payment, associated with our
European collaboration with Orion Corporation, which is no longer
subject to a clawback provision as a result of successful
completion of required M2 metabolite testing. Excluding the Orion
collaboration revenue, the company recognized $1.5 million in BARDA
federal contract revenue for the three months ended March 31, 2022,
as compared to $1.8 million in BARDA federal contract revenue for
the three months ended March 31, 2021.
- Research and development expenses were $18.0 million for the
three months ended March 31, 2022, compared to $18.6 million for
the three months ended March 31, 2021; the decrease was due
primarily to reduced CDD development and safety study activities
partially offset by increased headcount costs and TSC clinical
development costs.
- General and administrative expenses increased to $11.7 million
for the three months ended March 31, 2022, compared to $10.4
million for the three months ended March 31, 2021; the primary
drivers of the increase were increased support for scale up of the
company’s operations as well as preparation for
commercialization.
- A one-time cost of IP license fee of $1.2 million was
recognized as expense during the three months ended March 31, 2022,
associated with the recently signed agreement with Ovid
Therapeutics to license patents and patent applications for the use
of ganaxolone in the treatment of CDD.
- The company reported net losses of $19.4 million and $27.1
million for the three months ended March 31, 2022 and 2021,
respectively; cash used in operating activities increased to $27.7
million for the three months ended March 31, 2022, compared to
$16.2 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 three months
ended March 31, 2022, to be filed with the Securities and Exchange
Commission, which includes further detail on the company’s business
plans, operations, financial condition, and results of
operations.
Selected Financial Data (in thousands, except share and per
share amounts)
March 31, 2022
December 31,
2021
ASSETS
Cash and cash equivalents
$ 126,319
$ 122,927
Other assets
14,491
13,913
Total assets
$ 140,810
$ 136,840
LIABILITIES AND STOCKHOLDERS’
EQUITY
Current liabilities
$ 19,536
$ 40,566
Long Term Debt, Net
69,927
40,809
Other long-term liabilities
10,942
1,979
Total liabilities
100,405
83,354
Total stockholders’ equity
40,405
53,486
Total liabilities and stockholders’
equity
$ 140,810
$ 136,840
Three Months Ended March
31,
2022
2021
Revenue:
Federal contract revenue
$ 1,513
$ 1,806
Collaboration revenue
12,673
—
Total revenue
14,186
1,806
Expenses:
Research and development
17,991
18,591
General and administrative
11,737
10,376
Cost of IP license fee
1,169
—
Total expenses
30,897
28,967
Loss from operations
(16,711)
(27,161)
Interest income
12
24
Interest expense
(1,692)
—
Other expense, net
(970)
(4)
Net loss and comprehensive loss
$ (19,361)
$ (27,141)
Per share information:
Net loss per share of common stock—basic
and diluted
$ (0.52)
$ (0.74)
Basic and diluted weighted average shares
outstanding
36,890,568
36,599,701
Conference Call Information
Thursday, May 12, at 4:05 p.m. ET
Domestic: (888) 550-5280 International: (646) 960-0813 Webcast
Link: https://events.q4inc.com/attendee/993378901 Conference ID:
2696394
About Marinus Pharmaceuticals
Marinus is a pharmaceutical company dedicated to the development
of innovative therapeutics to treat seizure disorders. Ganaxolone
is a neuroactive steroid GABAA receptor modulator that acts on a
well-characterized target in the brain known to have anti-seizure
effects. It is being developed in IV and oral dose formulations
intended to maximize therapeutic reach to adult and pediatric
patient populations in both acute and chronic care settings. For
more information visit www.marinuspharma.com.
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 expected
clinical development plans, enrollment in our clinical trials,
regulatory communications and submissions and product launches for
ganaxolone, and the timing thereof; our commercialization and
marketing plans; our expectations regarding scheduling by the U.S.
Drug Enforcement Administration and the expected timing thereof;
our plans to launch a patient assistance program that provides
ongoing financial support to eligible patients and caregivers; our
expectations regarding BARDA funding; our expectations and beliefs
regarding the FDA and EMA with respect to our product candidates;
our expectations regarding the development of new formulations and
prodrug candidates; our expectations regarding the Orion
Corporation collaboration; our expectations regarding our license
agreement with Ovid Therapeutics; our expectations to monetize the
PRV; our expectation regarding the impact of the COVID-19 pandemic
on our business and clinical development plans; our financial
projections; and the potential safety and efficacy of ganaxolone,
as well as its therapeutic potential in a number of indications;
and other statements regarding the company's future operations,
financial performance, financial position, prospects, objectives
and other future event.
Forward-looking statements in this press 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, our ability to establish commercial infrastructure
and capabilities to launch ZTALMY; physician and patient acceptance
of ZTALMY; our ability to obtain adequate market access for ZTALMY;
the varying interpretation of clinical data; the scheduling of
ZTALMY by the U.S. Drug Enforcement Administration; our ability to
comply with the FDA’s requirement for additional post-marketing
studies in the required time frames; the timing of regulatory
filings for our other product candidates; the potential that
regulatory authorities, including the FDA and EMA, may not grant or
may delay approval for our product candidates; uncertainties and
delays relating to the design, enrollment, completion, and results
of clinical trials; unanticipated costs and expenses; early
clinical trials may not be indicative of the results in later
clinical trials; clinical trial results may not support regulatory
approval or further development in a specified indication or at
all; actions or advice of the FDA or EMA may affect the design,
initiation, timing, continuation and/or progress of clinical trials
or result in the need for additional clinical trials; our ability
to obtain and maintain regulatory approval for our product
candidate; our ability to develop new formulations of ganaxolone or
prodrugs; our ability to obtain, maintain, protect and defend
intellectual property for our product candidates; the potential
negative impact of third party patents on our or our collaborators’
ability to commercialize ganaxolone; delays, interruptions or
failures in the manufacture and supply of our product candidate;
the size and growth potential of the markets for the company’s
product candidates, and the company’s ability to service those
markets; the company’s cash and cash equivalents may not be
sufficient to support its operating plan for as long as
anticipated; the company’s expectations, projections and estimates
regarding expenses, future revenue, capital requirements, and the
availability of and the need for additional financing; the
company’s ability to obtain additional funding to support its
clinical development and commercial programs; the potential for
Orion to breach the collaboration or terminate the agreement in
accordance with its terms; the risk that drug product quality
requirements may not support continued clinical investigation of
our product candidates and result in delays or termination of such
clinical studies and product approvals; the effect of the COVID-19
pandemic on our business, the medical community, regulators and the
global economy; and the availability or potential availability of
alternative products or treatments for conditions targeted by us
that could affect the availability or commercial potential of our
product candidate. This list is not exhaustive and these and other
risks are described in our periodic reports, including the annual
report on Form 10-K, quarterly reports on Form 10-Q and current
reports on Form 8-K, filed with or furnished to the Securities and
Exchange Commission and available at www.sec.gov. Any
forward-looking statements that we make in this press release speak
only as of the date of this press release. We assume no obligation
to update forward-looking statements whether as a result of new
information, future events or otherwise, after the date of this
press release.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220512005992/en/
Company Sasha Damouni Ellis Vice President, Corporate
Affairs & Investor Relations Marinus Pharmaceuticals, Inc.
484-253-6792 sdamouni@marinuspharma.com
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