- ZTALMY® (ganaxolone) Q3 net product revenue of $5.4 million;
2023 net product revenue guidance increased to between $18.5 and
$19 million
- Over 75% of patients required for the interim analysis are now
enrolled in the Phase 3 RAISE trial in refractory status
epilepticus; if the trial meets pre-defined stopping criteria at
the interim analysis, topline data now anticipated Q2 2024
- Initiated the Marinus Access Program to expand global
availability of ZTALMY
- Cash runway projected into Q4 2024 with cash, cash equivalents
and short-term investments of $176.4 million as of September 30,
2023
- Marinus to host conference call on November 7, 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 third quarter ended September 30,
2023.
“With strong quarter over quarter growth and robust payer
coverage one year into the launch of ZTALMY, we continue to
demonstrate our unique commercial capabilities in the orphan
epilepsy space and are enthused by the opportunity ZTALMY and the
ganaxolone franchise represent as an important long-term value
driver for Marinus,” said Scott Braunstein, M.D., Chairman and
Chief Executive Officer of Marinus.
Dr. Braunstein continued, “We remain acutely focused on
advancing our Phase 3 clinical trials in refractory status
epilepticus and tuberous sclerosis complex. While we’re
disappointed that we now project RAISE enrollment to conclude by
the end of the first quarter, we remain confident in the benefit
that IV ganaxolone could bring to critically ill RSE patients and
the significant commercial opportunity. We are committed to
successfully completing both the RAISE and TrustTSC trials in 2024
and continue to make the investments to prepare for these
commercial launches.”
ZTALMY®
- ZTALMY® (ganaxolone) oral suspension CV net product revenue of
$5.4 million for the third quarter of 2023
- Continued growth in commercial patients with approximately 140
patients active on therapy at the end of the third quarter
- Increased full year 2023 expected ZTALMY net product revenues
to between $18.5 and $19 million from a range of $17 to $18.5
million
CDKL5 Deficiency Disorder
- Initiated the Marinus Access Program expanding global
availability of ZTALMY for eligible patients with seizures
associated with CDKL5 deficiency disorder (CDD) in geographies
where the product is not commercially available and as supported by
local regulatory requirements
- The Center for Drug Evaluation (CDE) of the China National
Medical Products Administration (NMPA) has accepted and granted
priority review of a New Drug Application (NDA) for ZTALMY in CDD;
the NDA was submitted in China by Tenacia Biotechnology under the
terms of a collaboration agreement with Marinus
- Orion Corporation continues to prepare for commercial launches
of ZTALMY in select European countries in 2024
Clinical Pipeline
Status Epilepticus
- Over 75% of patients required for an interim analysis are now
enrolled in the Phase 3 RAISE trial of intravenous (IV) ganaxolone
in refractory status epilepticus (RSE)
- Enrollment for the interim analysis expected to conclude in the
first quarter of 2024 with topline data now anticipated in the
second quarter of 2024, assuming pre-defined stopping criteria for
an interim analysis are met
- 21 patients have now been treated for super refractory status
epilepticus (SRSE) under emergency investigational new drug (EIND)
applications
- Phase 3 RAISE II trial in RSE (for European registration)
enrollment anticipated to begin before year end 2023
- To focus additional resources on the expansion of RSE clinical
programs, including further investigation of potential development
opportunities in SRSE, Marinus voluntarily discontinued the Phase 2
RESET trial in established status epilepticus
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.
Tuberous Sclerosis Complex
- Continue to enroll patients in the global Phase 3 TrustTSC
trial of oral ganaxolone in tuberous sclerosis complex with topline
data anticipated mid-2024
Second Generation Product
Development
- Enrollment in the multiple ascending dose (MAD) trial is well
underway with preliminary data expected by year end 2023
- Planning to finalize clinical program design for Lennox-Gastaut
syndrome in the first half of 2024, pending results of the MAD
trial
General Business and Financial
Update
- For the fiscal year 2023, the Company is updating its revenue
and operating expense guidance:
- The Company now expects ZTALMY net product revenues of between
$18.5 and $19 million; this represents an increase from the
previous guidance of between $17 and $18.5 million
- The Company now expects GAAP operating expenses, inclusive of
G&A and R&D, to be in the range of $158 to $162 million, of
which the Company expects stock-based compensation to be
approximately $16 million; this represents a decrease from the
prior guidance range of $160 to $165 million
- Expect that cash, cash equivalents, and short-term investments
of $176.4 million as of September 30, 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 fourth quarter
of 2024
- During the quarter, a total of 3.7 million shares were sold
through the Company’s at-the-market (ATM) facility contributing net
proceeds of $25.9 million
Financial Results
- Recognized $5.4 million and $13.0 million in net product
revenues for the three and nine months ended September 30, 2023,
respectively, as compared to $0.6 million in each of the same
periods in the prior year. Net product revenue consists of ZTALMY
product sales, which was launched in the U.S. in the third quarter
of 2022.
- Recognized $1.9 million and $10.8 million in Biomedical
Advanced Research and Development Authority (BARDA) federal
contract revenue for the three and nine months ended September 30,
2023, respectively, as compared to $1.8 million and $5.1 million
for the same periods in the prior year, respectively. The increase
on a year-to-date basis was primarily driven by activities
associated with the startup of the API onshoring initiative.
- Research and development (R&D) expenses were $23.7 million
and $73.0 million for the three and nine months ended September 30,
2023, respectively, as compared to $19.0 million and $58.5 million,
respectively, for the same periods in the prior year; the increase
on a year-to-date basis was due primarily to increased investment
associated with our API onshoring effort, increased TSC and RSE
clinical trial activity, and increased headcount.
- Selling, general and administrative (SG&A) expenses were
$14.9 million and $45.8 million for the three and nine months ended
September 30, 2023, respectively, as compared to $13.4 million and
$42.2 million, respectively, for the same periods in the prior
year; the increase on a year-to-date basis was due primarily to
increased headcount associated with the U.S. launch of ZTALMY.
- The Company had net losses of $33.0 million and $99.6 million
for the three and nine months ended September 30, 2023,
respectively; cash used in operating activities was $91.0 million
for each of the nine months ended September 30, 2023 and 2022
- At September 30, 2023, the Company had cash, cash equivalents,
and short-term investments of $176.4 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 nine months
ended September 30, 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.
Financial Results
Selected Financial Data (in thousands, except share and per
share amounts)
September 30, 2023
(unaudited)
December 31, 2022
ASSETS
Cash and cash equivalents
$
140,437
$
240,551
Short-term investments
35,919
-
Other assets
24,450
18,967
Total assets
$
200,806
$
259,518
LIABILITIES AND STOCKHOLDERS’
EQUITY
Current liabilities
$
30,555
$
25,017
Long term debt, net
64,783
71,018
Revenue interest financing payable,
net
32,855
29,857
Other long-term liabilities
18,076
17,626
Total liabilities
146,269
143,518
Total stockholders’ equity
54,537
116,000
Total liabilities and stockholders’
equity
$
200,806
$
259,518
Three Months Ended September
30,
(unaudited)
Nine Months Ended September
30,
(unaudited)
2023
2022
2023
2022
Revenue:
Product revenue, net
$
5,429
$
555
$
13,010
$
555
Federal contract revenue
1,891
1,785
10,753
5,088
Collaboration revenue
18
—
36
12,673
Total revenue
7,338
2,340
23,799
18,316
Expenses:
Research and development
23,661
19,002
73,006
58,488
Selling, general and administrative
14,868
13,389
45,794
42,187
Cost of product revenue
455
48
1,047
48
Cost of IP license fee
—
—
—
1,169
Total expenses:
38,984
32,439
119,847
101,892
Loss from operations
(31,646)
(30,099
)
(96,048)
(83,576
)
Interest income
1,895
514
6,366
610
Interest expense
(4,242)
(2,634
)
(12,597)
(6,982
)
Gain from sale of priority review voucher,
net
—
107,375
—
107,375
Other income (expense), net
1,021
(114
)
1,105
(1,179
)
(Loss) income before income taxes
(32,972)
75,042
(101,174)
16,248
(Provision) benefit for income taxes
—
(1,752
)
1,538
(1,752
)
Net (loss) income
$
(32,972)
$
73,290
$
(99,636)
$
14,496
Net income allocated to preferred
shareholders
—
1,656
—
336
Net (loss) income applicable to common
shareholders
(32,972)
71,634
(99,636)
14,160
Per share information:
Net (loss) income per share of common
stock—basic
$
(0.61)
$
1.93
$
(1.89)
$
0.38
Net (loss) income per share of common
stock—diluted
$
(0.61)
$
1.89
$
(1.89)
$
0.37
Basic weighted average shares
outstanding
53,920,109
37,202,269
52,755,114
37,084,060
Diluted weighted average shares
outstanding
53,920,109
37,910,511
52,755,114
38,393,754
Other comprehensive income (loss)
Unrealized gain (loss) on
available-for-sale securities
43
—
(71)
—
Total comprehensive (loss) income
$
(32,929)
73,290
(99,707)
14,496
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 first introduced FDA-approved prescription
medication ZTALMY® (ganaxolone) oral suspension CV in the U.S. in
2022 and continues to invest in the potential of ganaxolone 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; our net product
revenue guidance; 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;
statements regarding our expected clinical development plans,
enrollment in our clinical trials, regulatory communications and
submissions for ganaxolone, and the timing thereof; our expected
data readouts; our expected cash runway; 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 our
strategic partners; 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; 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/20231107561480/en/
Investors Jim DeNike Senior
Director, Investor Relations Marinus Pharmaceuticals, Inc.
jdenike@marinuspharma.com
Media Molly Cameron Director,
Corporate Communications & Investor Relations Marinus
Pharmaceuticals, Inc. mcameron@marinuspharma.com
Marinus Pharmaceuticals (NASDAQ:MRNS)
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