- ZTALMY® (ganaxolone) second quarter net product revenue of $4.2
million; 2023 net product revenue guidance increased to between $17
and $18.5 million
- European Commission approved ZTALMY in CDKL5 deficiency
disorder
- Interim analysis for the Phase 3 refractory status epilepticus
trial now expected Q1 2024
- First patient dosed in MAD trial with second generation
ganaxolone formulation
- New emergency IND dosing paradigm for super refractory status
epilepticus showing encouraging initial results
- Granted a new method of use patent by USPTO for ganaxolone in
tuberous sclerosis complex
- Cash runway expected into second half of 2024 with cash, cash
equivalents and short-term investments of $175.3 million as of June
30, 2023
- Marinus to host conference call on August 10, 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 second quarter ended June 30, 2023.
“With continued demand growth and robust payer coverage for
ZTALMY, we are pleased to increase our 2023 net product revenue
guidance to a range of $17 to $18.5 million,” said Scott
Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.
“Additionally, the European Commission's recent approval of ZTALMY
in CDKL5 deficiency disorder offers a novel therapeutic option for
patients and families and creates new opportunities for ganaxolone
in the international market.”
“Our clinical pipeline is progressing at an encouraging pace as
we continue to overcome industry headwinds affecting clinical trial
recruitment. We are confident in our ability to execute against our
updated RAISE timeline and previously stated TrustTSC timeline,
which would result in two pivotal Phase 3 data readouts next year.
We are excited to have initiated the MAD trial of our second
generation ganaxolone formulation and are on track for preliminary
data before the end of the year.”
ZTALMY®
- ZTALMY® (ganaxolone) oral suspension CV net product revenue of
$4.2 million for the second quarter of 2023
- Continued growth in commercial patients with approximately 120
patients active on therapy at the end of the second quarter
- Increased full year 2023 expected ZTALMY net product revenues
to between $17 and $18.5 million from a range of $15 to $17
million
- European Commission granted marketing authorization for ZTALMY
in CDKL5 deficiency disorder in July 2023
- Marinus is eligible to begin receiving royalties and
commercialization and sales milestones under the Collaboration
Agreement with Orion Corporation
- At two years in the open label extension phase of the Marigold
study, patients (n=50) continuing treatment with ZTALMY experienced
a median 48.2% reduction in major motor seizure frequency,
suggesting that patients who remain on treatment long-term may
demonstrate sustained reductions in seizure frequency
Clinical Pipeline
Status Epilepticus
- Topline data from an interim analysis of the Phase 3 RAISE
trial of intravenous (IV) ganaxolone in refractory status
epilepticus (RSE) now expected in the first quarter of 2024, if it
meets the pre-defined stopping criteria
- RAISE sites are being resupplied with the new citrate buffer
formulation of IV ganaxolone
- 18 patients have now been treated for super refractory status
epilepticus (SRSE) under emergency investigational new drug (EIND)
applications
- New EIND dosing paradigm for SRSE showing encouraging initial
results
- 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.
Tuberous Sclerosis Complex
(TSC)
- Continue to enroll patients in the global Phase 3 TrustTSC
trial of oral ganaxolone with topline data anticipated
mid-2024
- New method of use patent granted by USPTO for ganaxolone in
TSC, expiring in 2040
Second Generation Product
Development
- Enrollment in the multiple ascending dose (MAD) trial has
initiated 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; IND application submission targeted for the
fourth quarter of 2024
General Business and Financial
Update
- For the fiscal year 2023, the Company is updating its revenue
and operating expense guidance:
- ZTALMY Revenue: The Company now expects ZTALMY net product
revenues of between $17 and $18.5 million; this represents an
increase from the previous guidance of between $15 and $17
million
- BARDA Revenue: The Company now expects BARDA revenues between
$11 and $12 million; this represents an increase from the previous
guidance of between $8 and $11 million
- GAAP Operating Expense: The Company now expects GAAP operating
expenses, inclusive of G&A and R&D, to be in the range of
$160 to $165 million, of which the Company expects stock-based
compensation to be approximately $15 million; this represents a
decrease from the prior guidance range of $165 to $175 million
- Expect that cash, cash equivalents, and short-term investments
of $175.3 million as of June 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 second half of 2024.
Financial Results
- Recognized $4.2 million and $7.6 million in net product
revenues for the three and six months ended June 30, 2023,
respectively. Net product revenue consists of ZTALMY product sales,
which was launched in the U.S. in the third quarter of 2022.
- Recognized $1.8 million and $8.9 million in Biomedical Advanced
Research and Development Authority (BARDA) federal contract revenue
for the three and six months ended June 30, 2023, respectively, as
compared to $1.8 million and $3.3 million for the same periods in
the prior year, respectively. The increase on a year-to-date basis
was primarily driven by activity associated with start-up of the
API onshoring initiative.
- Research and development (R&D) expenses were $21.4 million
and $49.3 million for the three and six months ended June 30, 2023,
respectively, as compared to $21.5 million and $39.5 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
$15.7 million and $30.9 million for the three and six months ended
June 30, 2023, respectively, as compared to $17.1 million and $28.8
million, respectively, for the same periods in the prior year; the
increase on a year-to-date basis was due primarily to increased
commercial headcount associated with the US launch of ZTALMY.
- The Company had net losses of $31.9 million and $66.7 million
for the three and six months ended June 30, 2023, respectively;
cash used in operating activities increased to $65.8 million for
the six months ended June 30, 2023, compared to $61.3 million for
the same period a year ago.
- At June 30, 2023, the Company had cash, cash equivalents, and
short-term investments of $175.3 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 six months
ended June 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.
Selected Financial Data (in
thousands, except share and per share amounts)
June 30,
2023
(Unaudited)
December 31,
2022
ASSETS
Cash and cash equivalents
$
127,787
$
240,551
Short-term investments
47,549
-
Other assets
25,864
18,967
Total assets
$
201,200
$
259,518
LIABILITIES AND STOCKHOLDERS’
EQUITY
Current liabilities
$
25,603
$
25,017
Long term debt, net
68,136
71,018
Revenue interest financing payable,
net
31,950
29,857
Other long-term liabilities
18,172
17,626
Total liabilities
143,861
143,518
Total stockholders’ equity
57,339
116,000
Total liabilities and stockholders’
equity
$
201,200
$
259,518
Three Months Ended
June 30,
Six Months Ended
June 30,
2023
2022
2023
2022
Revenue:
Product revenue, net
$
4,249
$
—
$
7,581
$
—
Federal contract revenue
1,814
1,790
8,862
3,303
Collaboration revenue
18
—
18
12,673
Total revenue
6,081
1,790
16,461
15,976
Expenses:
Research and development
21,412
21,495
49,345
39,486
Selling, general and administrative
15,722
17,061
30,926
28,798
Cost of product revenue
386
—
592
—
Cost of IP license fee
—
—
—
1,169
Total expenses:
37,520
38,556
80,863
69,453
Loss from operations
(31,439
)
(36,766
)
(64,402
)
(53,477
)
Interest income
2,128
84
4,471
96
Interest expense
(4,208
)
(2,656
)
(8,355
)
(4,348
)
Other income (expense), net
47
(95
)
84
(1,065
)
Loss before income taxes
(33,472
)
(39,433
)
(68,202
)
(58,794
)
Benefit for income taxes
1,538
—
1,538
—
Net loss applicable to common
shareholders
$
(31,934
)
$
(39,433
)
$
(66,664
)
$
(58,794
)
Per share information:
Net loss per share of common stock—basic
and diluted
$
(0.61
)
$
(1.06
)
$
(1.28
)
$
(1.59
)
Basic and diluted weighted average shares
outstanding
52,551,918
37,155,917
52,162,962
37,023,976
Other comprehensive loss
Unrealized loss on available-for-sale
securities
(188
)
—
(114
)
—
Total comprehensive loss
$
(32,122
)
$
(39,433
)
$
(66,778
)
$
(58,794
)
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
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 expectations
regarding our strategic partners; 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; 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/20230810667944/en/
Molly Cameron Director, Corporate Communications & Investor
Relations Marinus Pharmaceuticals, Inc.
mcameron@marinuspharma.com
Marinus Pharmaceuticals (NASDAQ:MRNS)
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