Marinus Awarded BARDA Contract to Develop IV Ganaxolone for Treatment of Refractory Status Epilepticus Caused by Nerve Agent ...
September 14 2020 - 8:00AM
Business Wire
- BARDA to fund up to $51 million of $84 million contract for
programs related to refractory status epilepticus (RSE)
development
- Five-year contract supports clinical development of IV
ganaxolone for treatment of RSE, including treatment of individuals
exposed to nerve gas
- BARDA will fund pre-clinical studies of IV ganaxolone treatment
following nerve agent exposure in established animal models
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat rare seizure disorders, today announced it has entered into a
five-year development contract with the Biomedical Advanced
Research and Development Authority (BARDA), part of the Office of
the Assistant Secretary for Preparedness and Response within the
U.S. Department of Health and Human Services, to support the
development of IV ganaxolone for the treatment of refractory status
epilepticus (RSE), a life-threatening condition in which a
significant number of patients do not respond to first- and
second-line anticonvulsant drugs.
RSE can occur as a result of a variety of serious, acute medical
conditions or after exposure to nerve agents. The agreement covers
a base period during which BARDA will provide subject matter
expertise and $21 million to fund, on a cost share basis, the
company’s planned Phase 3 clinical trial of ganaxolone for the
treatment of RSE (as a result of an underlying medical condition)
and will fund preclinical studies of ganaxolone in nerve agent
exposure animal models. Contingent on favorable clinical and
preclinical outcomes in the base period, the contract includes up
to approximately $30 million of additional BARDA funding spanning
three options in support of manufacturing, supply chain, clinical,
regulatory and toxicology activities. Under the contract, Marinus
will be responsible for cost-sharing in the amount of $33 million
if all development options are completed.
“On behalf of the entire Marinus team, we are grateful to BARDA
for their collaborative approach throughout this process and for
the opportunity to continue to innovate in the field of seizure
disorders, while supporting the government’s efforts to be prepared
to protect U.S. lives in the event of a chemical attack,” said
Scott Braunstein, M.D., Chief Executive Officer of Marinus.
“Through this contract, we are demonstrating our commitment to
develop innovative anticonvulsant agents, and we believe this
funding will help to strengthen our ganaxolone franchise.”
Organophosphate nerve agents (chemical warfare agents and
organophosphate-based pesticides) are highly toxic compounds, which
may cause prolonged seizures that become more difficult to treat as
they progress. Ganaxolone has a complementary and potentially
synergistic mechanism to benzodiazepines, which are currently used
to treat nerve agent exposure-induced seizures and may help to stop
unremitting seizures when other drugs fail.
On a successful development, BARDA and Marinus may negotiate a
procurement agreement for a supply of ganaxolone for potential
response to nerve gas exposure threats.
“Having medical products at-the-ready to save lives in
emergencies requires strong public-private partnerships,” said
BARDA Acting Director Gary Disbrow, Ph.D. “To help our country
respond effectively to public health threats and be cost-efficient,
we look for product candidates that can fill a need on the
commercial market as well as meet public health emergency
needs.”
Marinus plans to pursue further discussions with BARDA to
evaluate additional routes of administration for ganaxolone that
would support field-based rapid response treatment in the event of
a nerve gas attack.
About Marinus Pharmaceuticals
Marinus Pharmaceuticals, Inc. is a pharmaceutical company
dedicated to the development of innovative therapeutics to treat
rare seizure disorders. Ganaxolone is a positive allosteric
modulator of GABAA receptors that acts on a well-characterized
target in the brain known to have antiseizure, antidepressant, and
anti-anxiety effects. Ganaxolone is being developed in IV and oral
dose forms intended to maximize therapeutic reach to adult and
pediatric patient populations in both acute and chronic care
settings. Marinus is conducting the first ever Phase 3 pivotal
trial in children with CDKL5 deficiency disorder, along with a
Phase 2 trial in tuberous sclerosis complex, and a Phase 2
biomarker driven proof of concept trial in PCDH19-related epilepsy.
The company is planning to initiate a Phase 3 trial in refractory
status epilepticus. 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 clinical
development plans for ganaxolone; the cost of our development
program for ganaxolone for the treatment of RSE), including nerve
gas exposure countermeasure; the potential for additional routes of
administration for ganaxolone; expected payments under our
agreement with BARDA; the potential safety and efficacy of
ganaxolone and the therapeutic potential of ganaxolone.
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, uncertainties and delays relating to the design,
enrollment, completion, and results of clinical trials;
unanticipated costs and expenses; 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 U.S.
Food and Drug Administration 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 obtain and maintain patent protection for our product
candidates; delays, interruptions or failures in the manufacture
and supply of our product candidate; our ability to raise
additional capital; the effect of the COVID-19 pandemic on our
business, the medical community 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.
Marinus undertakes no obligation to update or revise any
forward-looking statements. For a further description of the risks
and uncertainties that could cause actual results to differ from
those expressed in these forward-looking statements, as well as
risks relating to the business of the company in general, see
filings Marinus has made with the Securities and Exchange
Commission.
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version on businesswire.com: https://www.businesswire.com/news/home/20200914005181/en/
COMPANY: Sasha Damouni Ellis Vice President, Investor
Relations & Corporate Communications Marinus Pharmaceuticals,
Inc. 484-253-6792 sdamouni@marinuspharma.com MEDIA: Katie
Kennedy Vice President Gregory FCA 610-228-2128; 610-731-1045
katiek@gregoryfca.com
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