- ZTALMY® (ganaxolone) Q2 2024 net product revenue of $8.0
million representing strong growth of 87% versus Q2 2023
- On track to achieve full year 2024 ZTALMY net product revenue
guidance of between $33 and $35 million
- Completed enrollment in the Phase 3 TrustTSC trial of oral
ganaxolone in tuberous sclerosis complex (TSC) with topline data
expected in the first half of Q4 2024
- Expanded ZTALMY global footprint with activation of managed
access programs for MENA, Russia and Canada with upcoming
commercial launches anticipated in Europe and China
- Succeeded in post-grant review challenge of Ovid Therapeutics’
U.S. Patent 11,395,817 for IV ganaxolone in the treatment of status
epilepticus (SE); Patent Trial and Appeal Board issued final
written decision determining all remaining claims are
unpatentable
- Reported results from Phase 3 RAISE trial of IV ganaxolone in
refractory SE and plan to request FDA meeting to discuss next
steps
- Company to host investor and analyst event on September 20,
2024 to discuss TSC opportunity
- Conference call today 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 second quarter ended June 30, 2024.
“Since launching ZTALMY in the U.S. two years ago, we have seen
significant growth and adoption with strong revenue for the second
quarter, underscoring the appreciation physicians, patients and
caregivers have for ZTALMY and its role in the treatment of CDKL5
deficiency disorder,” said Scott Braunstein, M.D., Chairman and
Chief Executive Officer of Marinus. “We continue to grow the brand
globally with ZTALMY now approved in the U.S., EU, UK and China. We
look forward to continuing to expand access to this important
treatment option while generating revenue from markets outside the
U.S.”
Dr. Braunstein continued, “Our proven commercial infrastructure
and success in CDD provides us with a solid foundation to expand
into TSC, which represents a significant unmet need in a larger
refractory epilepsy population. With enrollment complete in the
Phase 3 TrustTSC trial and data upcoming, we continue to make the
appropriate investments and plan for a potential launch in the fall
of 2025. We will be hosting an investor and analyst event on
September 20th, where senior management and key opinion leaders
will discuss the treatment landscape, market potential and
commercial opportunity for ZTALMY in TSC.”
ZTALMY® (ganaxolone) Oral
Suspension CV
- Generated net product revenue of $8.0 million for the second
quarter of 2024 representing 87% growth versus the second quarter
of 2023
- On track to achieve full year 2024 ZTALMY net product revenue
guidance of between $33 and $35 million
- Continue to expand global access:
- Activated managed access programs for named patients in the
Middle East and North Africa (MENA), Canada and Russia with ex-U.S.
revenue expected in the third quarter of 2024
- In July, ZTALMY was approved in China for patients with CDKL5
deficiency disorder (CDD); Tenacia Biotechnology is anticipating
commercial launch in early 2025
- Orion Corporation continues to prepare for commercial launches
of ZTALMY in select European countries in the second half of 2024
with regulatory approvals secured in the European Union and United
Kingdom for the CDD indication
Clinical Pipeline
Tuberous Sclerosis Complex
- Enrollment in the global Phase 3 TrustTSC trial of oral
ganaxolone in tuberous sclerosis complex (TSC) is complete with
last patient visit expected in September; topline data is on track
for the first half of the fourth quarter of 2024
- Targeting submission of a supplemental New Drug Application
(NDA) to the U.S. Food and Drug Administration (FDA) in April 2025
with a request for priority review
- TrustTSC maintains a low discontinuation rate of less than 7%
with greater than 85% of patients to date continuing to the
open-label extension
- Launch planning and market access landscape assessment underway
in preparation of anticipated commercial launch in the second half
of 2025
Other Rare Genetic Epilepsies
- Expect to initiate a proof-of-concept study with ZTALMY to
treat a range of developmental and epileptic encephalopathies,
including Lennox-Gastaut syndrome, in the first half of 2025,
pending the TSC topline data
- Targeting submission of an Investigational New Drug application
for a novel oral ganaxolone prodrug in the fourth quarter of
2025
Status Epilepticus
- Announced topline results from the Phase 3 RAISE trial of
intravenous (IV) ganaxolone in refractory status epilepticus
- Planning to submit a request for a Type C meeting with the FDA
to discuss the RAISE trial results and next steps for the IV
ganaxolone program
- Continue to offer IV ganaxolone for patients with super
refractory status epilepticus under emergency investigational new
drug applications with 31 patients treated to date
Ganaxolone development in the RAISE trial has been supported in
part by the Department of Health and Human Services; Administration
for Strategic Preparedness and Response; Biomedical Advanced
Research and Development Authority (BARDA) under contract number
75A50120C00159.
General Business and Financial
Update
- On August 1, 2024, the Patent Trial and Appeal Board issued its
final written decision in the post-grant review of Ovid
Therapeutics’ U.S. Patent No. 11,395,817 for IV ganaxolone in the
treatment of status epilepticus, determining that all remaining
claims—claims 22 and 25-31—were unpatentable as obvious. The
decision is publicly available on the Patent Office’s website
here.
- In June 2024, Marinus restructured its agreements with Oaktree
Capital Management, L.P. and Sagard Healthcare Partners. Under the
restructuring, the $15 million minimum liquidity requirement was
removed from both the Oaktree and Sagard agreements, and
amortization payments due to Oaktree in 2024 have been reduced by
50%. In return, Marinus made a one-time principal payment of $15
million to Oaktree in the second quarter of 2024.
- Cost reduction plans initiated in the second quarter of 2024
are expected to reduce combined selling, general and administrative
(SG&A) and R&D expenses by approximately 30% from $80.3
million in the first half of 2024 to between $55 and $60 million in
the second half of 2024. The full on-going impact of cost savings
is expected to be achieved in the third quarter of 2024.
- Full year 2024 guidance remains unchanged with projected ZTALMY
net product revenue between $33 and $35 million and combined
SG&A and R&D expenses in the range of approximately $135 to
$140 million, including stock-based compensation expense of
approximately $20 million.
- Through the execution of the cost reduction plans and
restructured financing agreements, the Company expects that cash
and cash equivalents of $64.7 million as of June 30, 2024, will be
sufficient to fund the Company’s operating expenses and capital
expenditure requirements into the second quarter of 2025.
- The Company continues to make investments to expand ZTALMY
manufacturing capacity necessary for the global launch of the CDD
indication and potential TSC expansion.
Financial Results
- Recognized $8.0 million and $15.5 million in net product
revenue for the three and six months ended June 30, 2024,
respectively, as compared to $4.2 million and $7.6 million for the
same periods in the prior year, respectively.
- Recognized $0.1 million and $0.2 million in Biomedical Advanced
Research and Development Authority (BARDA) federal contract revenue
for the three and six months ended June 30, 2024, respectively, as
compared to $1.8 million and $8.9 million for the same periods in
the prior year, respectively. The decrease was primarily driven by
activity associated with the start-up of the API onshoring
initiative in the first quarter of 2023 and completion of the base
period funding in the fourth quarter of 2023.
- Research and development (R&D) expenses were $20.9 million
and $45.0 million for the three and six months ended June 30, 2024,
respectively, as compared to $21.4 million and $49.3 million for
the same periods in the prior year, respectively. The three month
decrease was due primarily to reduced personnel costs in 2024,
partially offset by increased costs associated with the RAISE
Trial. The six month decrease was also driven by costs associated
with start-up of the API onshoring effort in the first quarter of
2023.
- Selling, general and administrative (SG&A) expenses were
$16.7 million and $35.3 million for the three and six months ended
June 30, 2024, respectively, as compared to $15.7 million and $30.9
million for the same periods in the prior year, respectively; the
primary drivers of the increases were increased commercial spending
and headcount costs in 2024.
- Restructuring costs were $2.0 million for the three months
ended June 30, 2024, resulting from cost saving initiatives
implemented in the second quarter of 2024.
- The Company had net losses of $35.8 million and $74.5 million
for the three and six months ended June 30, 2024, respectively;
cash used in operating activities increased to $68.3 million for
the six months ended June 30, 2024, compared to $65.8 million for
the same period a year ago.
- At June 30, 2024, the Company had cash and cash equivalents of
$64.7 million, compared to cash, cash equivalents and short-term
investments of $150.3 million at December 31, 2023.
Readers are referred to, and encouraged to read in its entirety,
the Company’s Quarterly Report on Form 10-Q for the quarterly
period ended June 30, 2024, 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)
June 30, 2024
(unaudited)
December 31,
2023
ASSETS
Cash and cash equivalents
$
64,676
$
120,572
Short-term investments
-
29,716
Other assets
22,407
20,620
Total assets
$
87,083
$
170,908
LIABILITIES AND STOCKHOLDERS’
(DEFICIT) EQUITY
Current liabilities
$
35,867
$
40,624
Long term debt, net
45,075
61,423
Revenue interest financing payable,
net
35,431
33,766
Other long-term liabilities
18,055
18,330
Total liabilities
134,428
154,143
Total stockholders’ (deficit) equity
(47,345)
16,765
Total liabilities and stockholders’
(deficit) equity
$
87,083
$
170,908
Three Months Ended June
30,
Six Months Ended June
30,
2024 (Unaudited)
2023 (Unaudited)
2024 (Unaudited)
2023 (Unaudited)
Revenue:
Product revenue, net
$
7,951
$
4,249
$
15,460
$
7,581
Federal contract revenue
87
1,814
239
8,862
Collaboration revenue
18
18
36
18
Total revenue
8,056
6,081
15,735
16,461
Expenses:
Research and development
20,897
21,412
45,015
49,345
Selling, general and administrative
16,710
15,722
35,336
30,926
Restructuring Costs
1,950
-
1,950
-
Cost of product revenue
735
386
1,491
592
Total expenses:
40,292
37,520
83,792
80,863
Loss from operations
(32,236
)
(31,439
)
(68,057
)
(64,402
)
Interest income
1,109
2,128
2,571
4,471
Interest expense
(4,617
)
(4,208
)
(8,963
)
(8,355
)
Other (expense) income, net
(84
)
47
(48
)
84
Loss before income taxes
(35,828
)
(33,472
)
(74,497
)
(68,202
)
Benefit for income taxes
-
1,538
-
1,538
Net loss applicable to common
shareholders
$
(35,828
)
$
(31,934
)
$
(74,497
)
$
(66,664
)
Per share information:
Net loss per share of common stock—basic
and diluted
$
(0.63
)
$
(0.61
)
$
(1.31
)
$
(1.28
)
Basic and diluted weighted average shares
outstanding
57,064,095
52,551,918
56,957,953
52,162,962
Other comprehensive loss
Unrealized (loss) gain on
available-for-sale securities
-
(188
)
20
(114
)
Total comprehensive loss
$
(35,828
)
$
(32,122
)
$
(74,477
)
$
(66,778
)
Conference Call Information
Tuesday, August 13, 8:30 a.m.
ET
Domestic: (877) 405-1242 International: (201) 389-0852 Webcast
Registration: Click Here
An archived version of the call will be available approximately
two hours after the completion of the event on the Marinus website
at ir.marinuspharma.com/events-and-presentations.
About Marinus Pharmaceuticals Marinus is a
commercial-stage pharmaceutical company dedicated to the
development of innovative therapeutics for seizure disorders. The
Company’s product, ZTALMY® (ganaxolone) oral suspension CV, is an
FDA-approved prescription medication introduced in the U.S. in
2022. For more information, please visit www.marinuspharma.com and
follow us on LinkedIn, X and Facebook.
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 and other
financial guidance and projections; the potential benefits ZTALMY
will provide for physicians and patients; 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 expectations
regarding our cost reduction plans; our plans to continue to expand
global access; 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, the Company’s ability to continue as a going concern;
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 company’s
cash and cash equivalents may not be sufficient to support our
operating plan for as long as anticipated; 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; 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; the size and growth potential of the markets for the
company’s product candidates, and the company’s ability to service
those markets; 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; uncertainties related to 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 trials and
product approvals; 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 candidates. 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/20240813656110/en/
Company
Investors Sonya Weigle Chief People
and Investor Relations Officer Marinus Pharmaceuticals, Inc.
sweigle@marinuspharma.com
Media Molly Cameron Director,
Corporate Communications & Investor Relations Marinus
Pharmaceuticals, Inc. mcameron@marinuspharma.com
Marinus Pharmaceuticals (NASDAQ:MRNS)
過去 株価チャート
から 8 2024 まで 9 2024
Marinus Pharmaceuticals (NASDAQ:MRNS)
過去 株価チャート
から 9 2023 まで 9 2024