- ZTALMY® (ganaxolone) first quarter U.S. net product revenue of
$3.3 million
- Now planning to conduct an interim analysis for the Phase 3
refractory status epilepticus trial in the second half of 2023
- CHMP opinion for ZTALMY in CDKL5 deficiency disorder expected
by end of May; European Medicines Agency communicated that the
major objection related to the choice of regulatory starting
material has been resolved
- Phase 3 tuberous sclerosis complex (TSC) trial data now
expected mid-2024
- Received a notice of allowance from the USPTO for a new method
of use patent for ganaxolone in TSC; when granted, the term will
run through 2040
- Entered exclusive distribution and supply agreement with
Biologix for ZTALMY in select markets in the Middle East and North
Africa
- Cash runway into second half of 2024 with cash, cash
equivalents and short-term investments of $199.2 million as of
March 31, 2023
- Marinus to host conference call on May 11, 2023, at 8:30 a.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, 2023.
“We are pleased with our 2023 first quarter ZTALMY revenue in
the U.S. and are on track to meet our previously stated full year
revenue guidance of $15-17 million,” said Scott Braunstein, M.D.,
Chairman and Chief Executive Officer of Marinus. “As part of our
commitment to provide ganaxolone to a growing number of patients in
several important global regions, we are collaborating with Orion
Corporation to prepare for a potential launch in Europe, have
entered into a distribution agreement with Biologix for the Middle
East and North Africa, and continue to work closely with Tenacia on
development and commercialization in China.”
“We are now planning to conduct an interim analysis in RAISE,
our Phase 3 refractory status epilepticus trial, and if it meets
the predefined stopping criteria, plan to share these results
before year end,” continued Dr. Braunstein.
ZTALMY®
- ZTALMY® (ganaxolone) oral suspension CV U.S. net product
revenue of $3.3 million for the first quarter of 2023
- Continued growth in commercial patients with approximately 100
patients active on therapy at the end of the first quarter of
2023
- Expanded commercial coverage for ZTALMY with approximately 225
million covered lives, including both commercial and government
programs
Clinical Pipeline
Status Epilepticus
- Phase 3 RAISE trial of intravenous (IV) ganaxolone in
refractory status epilepticus (RSE) continues to advance with
Marinus now planning to conduct an interim analysis, and if it
meets the pre-defined stopping criteria, to report topline data
before year end 2023
- IV formulation of ganaxolone with new citrate buffer planned to
be incorporated into the RAISE trial in the second quarter of
2023
- Registration batches of this formulation have been placed on
stability; shelf life expected to be 24 months
- 16 patients have now been treated for super refractory status
epilepticus under emergency INDs
- Phase 3 RAISE II trial in RSE (for European registration)
enrollment anticipated to begin in the second half of 2023
- Phase 2 RESET trial in established status epilepticus (ESE)
completion of first cohort anticipated before year end 2023
Ganaxolone development in the RAISE trial is being funded in
part by the Biomedical Advanced Research and Development Authority
(BARDA), part of the Administration for Strategic Preparedness and
Response at the U.S. Department of Health and Human Services, under
contract number 75A50120C00159.
CDKL5 Deficiency Disorder Marketing
Authorization Application (MAA)
- The Committee for Medicinal Products for Human Use (CHMP)
opinion on the MAA is expected by the end of May 2023
- The European Medicines Agency communicated that the major
objection related to the choice of regulatory starting material has
been resolved
- Collaborating with Orion Corporation to prepare for a potential
commercial launch in Europe
Tuberous Sclerosis Complex
(TSC)
- Continue to enroll patients in the Phase 3 TrustTSC trial of
oral ganaxolone at sites in the U.S. and Europe with topline data
now anticipated mid-2024, primarily due to administrative and
regulatory delays in certain ex-U.S. countries
- Expect vast majority of clinical sites will be ready to enroll
patients by mid-year 2023, including sites in Canada, Israel,
China, Italy, Belgium and Australia
- Received notice of allowance from the United States Patent and
Trademark Office for a new method of use patent for ganaxolone in
TSC; when granted, the term of this patent will run through
2040
Second Generation Product
Development
- Pending FDA feedback and agreement, multiple ascending dose
(MAD) trial now expected to initiate in the third quarter of 2023
with preliminary data expected by year end 2023
- Planning to finalize clinical program design for Lennox-Gastaut
syndrome in the first quarter of 2024, pending results of the MAD
trial
- Prodrug development continues to advance with lead oral
candidate selected; Phase 1 data targeted for 2024
General Business and Financial
Update
- For the fiscal year 2023, the Company maintains its prior
guidance with expected ZTALMY U.S. net product revenues of between
$15 million and $17 million, BARDA revenues in the range of $8
million to $11 million and total GAAP operating expenses, inclusive
of SG&A and R&D, in the range of $165 to $175 million, of
which the company expects stock-based compensation of approximately
$16 million.
- Entered exclusive distribution and supply agreement with
Biologix for ganaxolone in select markets in the Middle East and
North Africa.
- Initiated U.S. onshoring of the manufacturing capabilities for
ganaxolone API which has the potential to drive a greater than 30%
reduction in API supply cost.
- Expect that cash, cash equivalents, and short-term investments
of $199.2 million as of March 31, 2023, will be sufficient to fund
the Company’s operating expenses, capital expenditure requirements
and maintain the minimum cash balance of $15 million required under
the Company’s debt facility into the second half of 2024.
Financial Results
- Recognized $3.3 million in net product revenues for the three
months ended March 31, 2023. Net product revenue consists of ZTALMY
product sales, which was launched in the U.S. in the third quarter
of 2022.
- Recognized $7.0 million in Biomedical Advanced Research and
Development Authority (BARDA) federal contract revenue for the
three months ended March 31, 2023, as compared to $1.5 million for
the same period in the prior year. The increase was primarily
driven by activity associated with start-up of the API onshoring
initiative.
- Research and development expenses were $27.9 million for the
three months ended March 31, 2023, compared to $18.0 million for
the same period in the prior year; the increase was due primarily
to increased costs associated with our API onshoring effort,
increased TSC and RSE clinical trial activity, and increased
headcount.
- Selling, general and administrative (SG&A) expenses were
$15.2 million for the three months ended March 31, 2023, as
compared to $11.7 million for the same period in the prior year;
the primary drivers of the change were increased headcount and
commercial support for the U.S. launch of ZTALMY.
- The Company reports a net loss of $34.7 million for the three
months ended March 31, 2023; cash used in operating activities
increased to $41.5 million for the three months ended March 31,
2023, compared to $27.7 million for the same period a year
ago.
- At March 31, 2023, the Company had cash, cash equivalents, and
short-term investments of $199.2 million, compared to $240.6
million at December 31, 2022.
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, 2023, 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,
2023
(Unaudited)
December 31,
2022
ASSETS
Cash and cash equivalents
$
146,904
$
240,551
Short-term investments
52,297
-
Other assets
27,616
18,967
Total assets
$
226,817
$
259,518
LIABILITIES AND STOCKHOLDERS’
EQUITY
Current liabilities
$
21,514
$
25,017
Long term debt, net
71,498
71,018
Revenue interest financing payable,
net
30,893
29,857
Other long-term liabilities
17,827
17,626
Total liabilities
141,732
143,518
Total stockholders’ equity
85,085
116,000
Total liabilities and stockholders’
equity
$
226,817
$
259,518
Three Months Ended March 31,
2023
(unaudited)
2022
(unaudited)
Revenue:
Product revenue, net
$
3,332
$
-
Federal contract revenue
7,048
1,513
Collaboration revenue
-
12,673
Total revenue
10,380
14,186
Expenses:
Research and development
27,933
17,991
Selling, general and administrative
15,204
11,737
Cost of product revenue
206
-
Cost of IP license fee
-
1,169
Total expenses:
43,343
30,897
Loss from operations
(32,963)
(16,711)
Interest income
2,343
12
Interest expense
(4,147)
(1,692)
Other income (expense), net
37
(970)
Net loss
$
(34,730)
$
(19,361)
Per share information:
Net loss per share of common stock- basic
and diluted
$
(0.67)
$
(0.52)
Basic and diluted weighted average shares
outstanding
51,769,685
36,890,568
Other comprehensive income
Unrealized gain on available-for-sale
securities
74
-
Total comprehensive loss
$
(34,656)
$
(19,361)
Conference Call Information Participants may access the
conference call via webcast on the Investor page of Marinus’
website at ir.marinuspharma.com/events-and-presentations. An
archived version of the call will be available approximately two
hours after the completion of the event on the website.
About Marinus Pharmaceuticals Marinus is a
commercial-stage pharmaceutical company dedicated to the
development of innovative therapeutics for seizure disorders. The
Company’s commercial product, ZTALMY® (ganaxolone) oral suspension
CV, has been approved by the U.S. FDA for the treatment of seizures
associated with CDKL5 deficiency disorder in patients two years of
age and older. The potential of ganaxolone is also being studied in
other seizure disorders, including in Phase 3 trials in tuberous
sclerosis complex and refractory status epilepticus. 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 formulations to
maximize therapeutic reach for adult and pediatric patients in
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 commercialization and
marketing plans for ZTALMY; the potential benefits ZTALMY will
provide for physicians and patients; the potential benefits from
the U.S. onshoring of the manufacturing capabilities for ganaxolone
API; our expectations regarding the ZTALMY One program; statements
regarding our expected clinical development plans, enrollment in
our clinical trials, regulatory communications and submissions for
ganaxolone, and the timing thereof; our expected cash runway; 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 expectation regarding the impact of the
COVID-19 pandemic on our business and clinical development plans;
our financial projections; 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, unexpected results or delays in the commercialization
of ZTALMY; unexpected market acceptance, payor coverage or future
prescriptions and revenue generated by ZTALMY; unexpected actions
by the FDA or other regulatory agencies with respect to our
products; competitive conditions and unexpected adverse events or
patient outcomes from being treated with ZTALMY, uncertainties and
delays relating to the design, enrollment, completion, and results
of clinical trials; unanticipated costs and expenses; the varying
interpretation of clinical data; 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 including with respect to the
CDD MAA; 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 our
ex-US partners to breach their obligations under their respective
agreements with us or terminate such agreements in accordance with
their respective 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/20230511005063/en/
Sasha Damouni Ellis Senior Vice President, Corporate Affairs
& Investor Relations Marinus Pharmaceuticals, Inc.
sdamouni@marinuspharma.com
Marinus Pharmaceuticals (NASDAQ:MRNS)
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