- Marinus has satisfied the FDA’s protocol-specific questions for
the Phase 3 trial in refractory status epilepticus (RSE)
- Company intends to begin enrollment for registrational Phase 3
clinical trial in RSE; over 55 out of projected 80 sites have been
selected for trial participation
- Noted clinical expert in status epilepticus, Henri
Vaitkevicius, M.D., has been appointed Vice President, Clinical
Development
Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical
company dedicated to the development of innovative therapeutics to
treat rare seizure disorders, today announced it has satisfied the
FDA’s protocol-specific questions for the registrational Phase 3
trial (the RAISE trial) in refractory status epilepticus (RSE),
allowing the company to begin enrollment in this clinical
trial.
In July, Marinus submitted a protocol amendment to the FDA for
the RAISE trial for IV ganaxolone in RSE. Following a recent FDA
discussion, Marinus currently anticipates the first patient will be
enrolled in the RAISE trial in October. To date, Marinus has
selected over 55 out of a projected 80 clinical sites to
participate in the trial. The company continues to anticipate
top-line data in 1H 2022.
Marinus is also announcing the appointment of Henri
Vaitkevicius, M.D., to the position of Vice President, Clinical
Development, reporting to Joe Hulihan, M.D., Chief Medical Officer.
During his time as a clinician at Massachusetts General Hospital,
Harvard University Medical School, Dr. Vaitkevicius is credited as
being the first physician to successfully treat a patient in super
refractory status epilepticus with a neurosteroid.
Dr. Vaitkevicius joins Igor Grachev, M.D., Ph.D. and Maciej
Gasior, M.D., Ph.D. in expanding Marinus’ clinical development team
of physicians. Drs. Grachev and Gasior also hold the title of Vice
President, Clinical Development.
“In our recent Phase 2 trial in RSE, status epilepticus was
controlled at a median time of five minutes after starting
ganaxolone, with no patients progressing to IV anesthesia for
control of seizures within 24 hours, the primary study endpoint.
There is a pressing need for better treatments for RSE, and we are
eager to begin our Phase 3 clinical trial in this indication,” said
Joe Hulihan, M.D., Chief Medical Officer. “Having served on our
Scientific Advisory Board and as the principal study investigator
in our Phase 2 RSE study, Henri has a solid understanding of the
strong science behind our research and clearly shares our vision
for developing ganaxolone as a treatment for patients with severe
seizure disorders.”
Ganaxolone (GNX) development for RSE is funded, in part, by the
Biomedical Advanced Research and Development Authority (BARDA) part
of the Office of the Assistant Secretary for Preparedness and
Response at the U.S. Department of Health and Human Services under
contract number 75A50120C00159.
Henri Vaitkevicius, M.D., Vice President, Clinical
Development
Dr. Vaitkevicius was previously an attending neurologist in the
Neurocritical Care, Stroke and Hospitalist Divisions of Brigham and
Women’s Hospital Department of Neurology, and an Assistant
Professor at Harvard Medical School. Dr. Vaitkevicius received his
graduate and medical school training at Wayne State University in
Detroit, Michigan. He completed his neurology residency and
neurocritical care fellowship at Brigham & Women’s and
Massachusetts General Hospital in Boston, Massachusetts.
“I have seen firsthand how devastating status epilepticus can be
for patients and their families,” commented Dr. Vaitkevicius.
“Despite the availability of many antiepileptic medications,
patients often continue to have disabling seizures and suffer from
countless life threatening side effects of currently available
therapies. With the positive results from the RSE Phase 2 data, and
the potential for the Phase 3 trial, I made a personal decision to
leave 12 years of a successful clinical practice behind me in order
to have an opportunity to contribute to the further development of
ganaxolone. I am honored to be part of the Marinus team.”
Dr. Vaitkevicius has appeared in over 40 peer-reviewed
publications, and multiple other scientific and medical materials
and chapters. He has served as the director of Brain Hub: Studio
for Research and Innovation in Critical Care Neurology, where he
focused on fostering collaborations among academic departments and
pharmaceutical industries to bring novel treatments to the
bedside.
In connection with his appointment, Marinus granted Dr.
Vaitkevicius a non-statutory stock option outside of the company’s
2014 Equity Incentive Plan as an inducement material to Dr.
Vaitkevicius entering into employment with Marinus in accordance
with Nasdaq Stock Market Listing Rule 5635(c)(4). The stock option
to purchase 25,000 shares of the Marinus’ common stock was approved
by the compensation committee of the company’s board of directors
and has an exercise price of $8.44 per share, which is equal to the
closing price on a split adjusted basis of the company’s common
stock on September 14, 2020, the date of the grant. The stock
option will vest and become exercisable as to 25 percent of the
underlying shares on the one-year anniversary of the Dr.
Vaitkevicius’ start date, and will vest and become exercisable as
to the remaining 75 percent of the underlying shares in 36 equal
monthly installments at the end of each month following such
anniversary, subject to the Dr. Vaitkevicius’ continued employment
with Marinus on such vesting dates.
Igor Grachev, M.D., Ph.D., Vice President, Clinical
Development
Igor Grachev, M.D., Ph.D., joined Marinus as vice president,
clinical development. He brings nearly 20 years of pharmaceutical
industry experience to Marinus, having led clinical development and
medical affairs programs at both multinational pharmaceutical and
biotech organizations. Prior to joining Marinus, he served as chief
medical officer at Cellectar Biosciences, and previously had
progressive leadership roles at TEVA Branded Specialty
Pharmaceuticals, Novartis, GSK, GE Healthcare, BioClinica, Merck,
Sanofi-Aventis, Schering Plough, and Guide Pharmaceutical
Consulting. Over the course of his career, Dr. Grachev has been
responsible for clinical development, clinical operation,
regulatory affairs, medical affairs and drug safety, execution and
management of clinical programs and trials Phases 1 through four
worldwide in neurology/neurodegenerative disorders and psychiatry,
neuro-oncology and other adjacent therapeutic areas, achieving
regulatory approvals in multiple countries.
Dr. Grachev is a former assistant professor at SUNY Upstate
Medical University and served as a fellow/instructor/assistant
professor at Massachusetts General Hospital, Harvard University
Medical School. Dr. Grachev is well-published in the field of
neurology, neurodegeneration and psychiatry with over 150
peer-reviewed publications. He is also the editor-in-chief of the
Journal of Neurology and Brain Disorders and serves as associate
editor of several neurology and medical journals. Dr. Grachev
earned his M.D. with highest honor in general medicine from
Bogomolets National Medical University; his Ph.D. in neuroscience,
and neurology and psychiatry residency trainings from the Institute
of Gerontology at the Shupyk National Medical Academy of
Postgraduate Education; and completed a fellowship training at
Massachusetts General Hospital, Harvard University Medical
School.
Maciej Gasior, M.D., Ph.D., Vice President, Clinical
Development
Maciej Gasior, M.D., Ph.D., joined Marinus as vice president,
clinical development in 2018. Dr. Gasior has nearly 15 years of
pharmaceutical experience. Prior to joining Marinus, he served as a
senior medical director at TEVA Pharmaceuticals, where he led
several R&D projects in the company’s pain portfolio that led
to a regulatory approval. Other prior positions were with
Bristol-Myers Squibb (Exploratory Clinical and Translational
Research in Neuroscience and Pain) and Cephalon Inc. (Discovery and
Clinical Research in CNS and Pain). Within these positions, Dr.
Gasior was responsible for the clinical development and execution
of global clinical programs and trials from Phases 1 through 4. Dr.
Gasior is a former staff scientist in the Epilepsy Research Section
(NIH-NINDS), junior faculty fellow at the Alcohol and Drug Abuse
Research Center (Harvard Medical School) and post-doctoral fellow
at the Drug Development Group (NIH-NIDA).
Dr. Gasior’s main research interests and expertise are in
epilepsy, pain, and drug development for neuropsychiatric
disorders. He has published over 90 papers in peer-reviewed
journals, serves as a member of editorial boards for the Journal of
Translational Neuroscience and Journal of CNS & Neurological
Disorders – Drug Targets. He is also an adjunct professor of the
Department of Pharmacology and Physiology (Drexel University
College of Medicine). Dr. Gasior earned his M.D. with honors and
Ph.D. in neuropharmacology from the Medical University of Lublin,
Poland, where he is a full professor.
About the RAISE Trial
The Phase 3 RAISE Trial is a randomized, double-blind,
placebo-controlled trial in SE patients who have failed
benzodiazepines and two or more second line intravenous
anti-epileptic drugs (AEDs). Approximately 80 study sites in
hospitals across the U.S. will participate. The trial is designed
to enroll approximately 125 patients, randomized to receive
ganaxolone or placebo adjunctive to standard of care and to provide
greater than 90 percent power to detect a 30 percent efficacy
difference between ganaxolone and placebo.
Patients on ganaxolone will receive an IV bolus, then a 36-hour
infusion followed by a 12-hour taper for a total 48-hour treatment
period. This regimen targets a plasma concentration of greater than
or equal to 500ng/mL for 12 hours (the same target concentration,
for a 50 percent longer duration at this level compared to the
Phase 2 trial).
The co-primary endpoints for the RAISE trial are (1) proportion
of patients with SE cessation within 30 minutes of treatment
initiation without other medications for the treatment of SE, and
(2) proportion of patients with no progression to IV anesthesia for
36 hours following treatment initiation.
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 anti-seizure, anti-depressant 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 recently completed the first ever Phase 3 pivotal
trial in children with CDKL5 deficiency disorder and is conducting
a Phase 2 trial in tuberous sclerosis complex, as well as a Phase 2
biomarker-driven proof-of-concept trial in PCDH19-related epilepsy.
The company is planning to initiate a Phase 3 trial in 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; our expectations to open clinical
trial sites for our Phase 3 trial in status epilepticus in October;
our expectations to release data from our Phase 3 RAISE trial in
status epilepticus in 1H 2022; our expectations regarding 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, results of clinical trials, and
interpretation of clinical trial results; 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/20200924005877/en/
Sasha Damouni Ellis Vice President, Investor Relations &
Corporate Communications Marinus Pharmaceuticals, Inc. 484-253-6792
sdamouni@marinuspharma.com
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