Trial did not meet pre-defined stopping
criteria at the interim analysis; Marinus has completed RAISE
enrollment at approximately 100 patients with topline results
expected summer 2024
Future development of IV ganaxolone in
refractory status epilepticus to be assessed following review of
the final RAISE results
Enrollment in the TrustTSC trial expected to
complete mid-May with topline data on track for the first half of
Q4 2024
Cost reduction activities to extend cash runway
are under review and expected to be implemented Q2 2024
ZTALMY® (ganaxolone) Q1 2024 preliminary net
product revenue of between $7.4 and $7.6 million; preliminary
unaudited cash, cash equivalents and short-term investments of
$113.3 million as of March 31, 2024
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat seizure disorders, today announced that an independent Data
Monitoring Committee (DMC) has recommended continuing the pivotal
Phase 3 RAISE trial evaluating intravenous (IV) ganaxolone for the
treatment of refractory status epilepticus (RSE) following an
interim analysis.
Marinus has decided to complete enrollment in the RAISE trial at
approximately 100 patients with topline results expected in the
summer of 2024. Those results will be used to determine whether to
continue development of IV ganaxolone. Marinus remains blinded to
the RAISE trial data.
“While we are disappointed that RAISE did not meet the early
stopping criteria, we will only be able to determine the trial’s
outcome once we unblind and analyze the full data set,” said Scott
Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.
“We will also be evaluating potential cost-saving strategies to
provide the strongest capital position as we approach enrollment
completion in the global Phase 3 TrustTSC trial in tuberous
sclerosis complex.”
Ganaxolone development in the RAISE trial is being 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
Marinus expects to complete enrollment in the Phase 3 TrustTSC
trial of ZTALMY® (ganaxolone) oral suspension CV with approximately
130 patients in mid-May 2024. The Company expects topline results
early in the fourth quarter of 2024 and anticipates filing a
supplemental New Drug Application to the U.S. Food and Drug
Administration in the first half of 2025 with a request for
priority review.
Marinus also continues to develop a second-generation ganaxolone
formulation intended to provide improved pharmacodynamic and
pharmacokinetic profiles that could improve safety, efficacy and
tolerability and enable less frequent dosing.
The Company continues the successful U.S. commercial launch of
ZTALMY resulting in preliminary unaudited net product revenue of
between $7.4 and $7.6 million for the first quarter of 2024.
Marinus estimates preliminary unaudited cash, cash equivalents, and
short-term investments of $113.3 million as of March 31, 2024. Cost
reduction activities to extend the cash runway beyond the fourth
quarter of 2024 are under review and are expected to be implemented
in the current quarter.
The preliminary first quarter 2024 net product revenue results
and cash, cash equivalents, and short-term investments included in
this release were calculated prior to the completion of a review by
the Company’s independent registered public accounting firm and are
therefore subject to adjustment.
About Status Epilepticus
Status epilepticus (SE) is a life-threatening condition
resulting from either the failure of the mechanisms responsible for
seizure termination or from the initiation of mechanisms which lead
to abnormally prolonged seizures.1 SE is the one of the most common
neurological emergencies in the U.S., affecting up to 150,000
patients each year, and is associated with substantial morbidity,
mortality, and healthcare costs.2,3,4 Patients who do not respond
to 1st- and 2nd-line treatments (benzodiazepines and intravenous
antiseizure medications) are considered to have refractory SE
(RSE).4,5
About Intravenous (IV) Ganaxolone
Ganaxolone is a neuroactive steroid that works by modulating
both synaptic and extrasynaptic GABAA receptors via a unique
binding site to potentiate two types of inhibitory signaling.6 IV
ganaxolone has pharmacokinetic and pharmacodynamic properties
well-suited for the treatment of status epilepticus, with rapid and
sustained SE cessation observed in pre-clinical and clinical
studies.7,8,9 IV ganaxolone has received orphan drug designation
from the U.S. Food and Drug Administration for the potential
treatment of status epilepticus.
About the RAISE Trial
The RAISE (Randomized Therapy in Status Epilepticus) trial
(NCT04391569) is a Phase 3 double-blind, randomized,
placebo-controlled clinical trial to evaluate the safety and
efficacy of IV ganaxolone in patients with refractory status
epilepticus. The RAISE protocol provides for an independent data
monitoring committee to conduct an unblinded interim analysis when
two-thirds of participants, or approximately 82 patients, have
completed the trial.
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 about Marinus visit www.marinuspharma.com.
1 Trinka E, et al. Epilepsia. 2015;56(10):1515-1523. 2 Betjemann
JP, Lowenstein DH. Lancet Neurol. 2015;14(6):615-24. 3 Guterman EL
et al. JAMA Neurol. 2021;78(5):588-95. 4 Glauser T et al. Epilepsy
Curr. 2016;16:48-61. 5 Brophy GM et al. Neurocrit Care.
2012;17:3-23. 6 Reddy, D. S. Front Cell Neurosci 7, 115 (2013). 7
Zolkowska D et al. Epilepsia. 59(suppl 2):220-227. 8 Gasior M et
al. Clin Pharmacol Drug Dev 13: 248-25. 9 Vaitkevicius H et al.
Epilepsia 2022; 63(9): 2381-2391.
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 the expectation
that the results from the RAISE trial will be used to determine
whether to continue development of IV ganaxolone; our plans to
evaluate potential cost-saving strategies to provide the strongest
capital position and extend cash runway, and the implementation and
related timing thereof; our belief that a second-generation
ganaxolone formulation may provide improved pharmacodynamic and
pharmacokinetic profiles that could improve safety, efficacy and
tolerability and enable less frequent dosing; the intent to explore
new clinical programs for ZTALMY in other refractory epilepsies,
and the timing thereof; the potential benefits ZTALMY will provide
for physicians and patients; statements regarding our expected
clinical development plans, enrollment in our clinical trials,
results from our clinical trials, regulatory communications and
submissions for ganaxolone, and the timing thereof; the safety and
efficacy of ganaxolone, as well as its therapeutic potential in a
number of indications; and other statements regarding our future
operations, financial performance, financial position, prospects,
objectives and other future events.
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 our product candidates, and our ability to
service those markets; our ability to continue as a going concern;
our cash and cash equivalents may not be sufficient to support its
operating plan for as long as anticipated; our expectations,
projections and estimates regarding expenses, future revenue,
capital requirements, and the availability of and the need for
additional financing; our ability to obtain additional funding to
support its clinical development and commercial programs; the
potential for our ex-U.S. 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; 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/20240415026329/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
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