MRNS Marinus Pharmaceuticals Inc.

12.86
+1.24  (+11%)
Previous Close 11.62
Open 12.25
52 Week Low 4.04
52 Week High 16.28
Market Cap $393,258,388
Shares 30,579,968
Float 30,510,064
Enterprise Value $244,294,642
Volume 1,015,437
Av. Daily Volume 1,544,025
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Upcoming Catalysts

Drug Stage Catalyst Date
Ganaxolone
Tuberous Sclerosis Complex
Phase 2
Phase 2
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Ganaxolone (Violet Study)
PCDH19-related epilepsy
Phase 3
Phase 3
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Ganaxolone
Refractory status epilepticus (RSE)
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
Ganaxolone - Marigold
CDKL5 Deficiency Disorder (CDD)
Phase 3
Phase 3
Phase 3 trial met primary endpoint - September 14, 2020.
Ganaxolone - Magnolia
Postpartum depression
Phase 2
Phase 2
Phase 2 negative data released July 23, 2019.
Ganaxolone
Focal onset seizures
Phase 3
Phase 3
Phase 3 data did not meet endpoints - June 2016
Ganaxolone
Fragile X Syndrome
Phase 2
Phase 2
Phase 2 data released June 2016. Primary endpoint not met but intends to advance development

Latest News

    • 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…

    • 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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  1. - Shares of Common Stock Will Begin Trading on Split-Adjusted Basis on September 23, 2020

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that it will effect a one-for-four reverse stock split of its common stock at 5:00 pm ET today. Beginning with the opening of trading on September 23, 2020, the Company's common stock will trade on The Nasdaq Global Market on a split-adjusted basis under a new CUSIP number 56854Q200.

    The Company's stockholders approved an amendment authorizing the reverse stock split at its Special Meeting of Stockholders on March 31, 2020. The reverse stock split was effected by filing a Certificate…

    - Shares of Common Stock Will Begin Trading on Split-Adjusted Basis on September 23, 2020

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that it will effect a one-for-four reverse stock split of its common stock at 5:00 pm ET today. Beginning with the opening of trading on September 23, 2020, the Company's common stock will trade on The Nasdaq Global Market on a split-adjusted basis under a new CUSIP number 56854Q200.

    The Company's stockholders approved an amendment authorizing the reverse stock split at its Special Meeting of Stockholders on March 31, 2020. The reverse stock split was effected by filing a Certificate of Amendment to the Company's Restated Certificate of Incorporation with the Secretary of State of the State of Delaware.

    The reverse stock split will be effected simultaneously for all outstanding shares of common stock and the ratio will be the same for all outstanding shares of common stock. The reverse stock split will affect all holders of shares of Marinus common stock uniformly and each stockholder will hold the same percentage of Marinus common stock outstanding immediately following the reverse stock split as that stockholder held immediately prior to the reverse stock split, except for adjustments that may result from the treatment of fractional shares as described below.

    No fractional shares will be issued in connection with the reverse stock split. Stockholders who would otherwise be entitled to receive a fractional share will instead receive a cash payment based on the closing sales price of the Company's common stock on September 22, 2020.

    The reverse stock split proportionately reduces the number of shares of common stock available for issuance under the Company's equity incentive plans and proportionately reduces the number of shares of common stock issuable upon the exercise of stock options outstanding immediately prior to the reverse split. The reverse stock split will reduce the number of shares of common stock issued and outstanding from approximately 122.3 million to approximately 30.6 million.

    In connection with the reverse stock split, the number of authorized shares of common stock will be decreased from 300,000,000 to 150,000,000, effective immediately following the effectiveness of the reverse split. There will be no change to the number of authorized shares of preferred stock, or change in the par values of Company's common stock (which will remain at $0.001 per share) or preferred stock (which will remain at $0.001 per share).

    American Stock Transfer & Trust Company, LLC (AST) is acting as the exchange agent and transfer agent for the reverse stock split. AST will provide instructions to stockholders with physical certificates regarding the process for exchanging their pre-split stock certificates for post-split shares in book-entry form and receiving payment for any fractional shares.

    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 has conducted 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 intends 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 the timing and effectiveness of the reverse stock split and Marinus' expectations to initiate a Phase 3 trial in status epilepticus. 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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    • Trial met primary endpoint, median 28-day major motor seizure frequency reduction of 32.2 percent compared to 4.0 percent for placebo (p=0.002)
    • Ganaxolone was generally well tolerated and the discontinuation rate in the active treatment arm was less than 5 percent
    • New Drug Application (NDA) submission planned for mid-2021; commercial launch targeted for 1H 2022
    • Conference call scheduled for September 14 at 4:30pm EDT

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced positive top-line results from its registrational Phase 3 clinical trial (Marigold Study) evaluating the use of oral ganaxolone in children and young…

    • Trial met primary endpoint, median 28-day major motor seizure frequency reduction of 32.2 percent compared to 4.0 percent for placebo (p=0.002)
    • Ganaxolone was generally well tolerated and the discontinuation rate in the active treatment arm was less than 5 percent
    • New Drug Application (NDA) submission planned for mid-2021; commercial launch targeted for 1H 2022
    • Conference call scheduled for September 14 at 4:30pm EDT

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced positive top-line results from its registrational Phase 3 clinical trial (Marigold Study) evaluating the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy with refractory seizures.

    In the trial, patients given ganaxolone showed a significant 32.2 percent median reduction in 28-day major motor seizure frequency, compared to a 4.0 percent reduction for those receiving the placebo, achieving the primary endpoint (p=0.002). The trial's primary efficacy endpoint was the percentage change in 28-day frequency of major motor seizures during the double-blind phase relative to the 6-week prospective baseline period. Ganaxolone was generally well tolerated with a safety profile consistent with previous clinical studies. The most frequent adverse event was somnolence.

    Based on these results, Marinus plans to submit an NDA for ganaxolone in the treatment of CDD to the U.S. Food and Drug Administration (FDA) in mid-2021 and a Marketing Authorization Application (MAA) for ganaxolone for the treatment of CDD to the European Medicines Agency (EMA) by the end of Q3 2021.

    "The Marigold Study has two important firsts. It's the first double-blind placebo controlled study providing evidence of efficacy specific to CDD and the first Phase 3 trial to examine three times a day dosing of ganaxolone in pediatric patients," said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. "We believe we are one step closer to providing the first treatment indicated for CDD, and plan to continue our investments in the oral ganaxolone franchise."

    The trial showed numerical trends favoring ganaxolone across several predefined secondary endpoints, however, ganaxolone did not meet statistical significance. Ganaxolone did meet statistical significance in exploratory secondary endpoints.

    "Today's success with ganaxolone in CDD will pave the way for us to accelerate our clinical studies in tuberous sclerosis complex and possibly other rare pediatric epilepsies as well," said Joe Hulihan, M.D., Chief Medical Officer of Marinus. "We will continue to explore the potential for ganaxolone's unique mechanism of action to address other areas of unmet medical need."

    The global, double-blind, placebo-controlled, Phase 3 trial enrolled 101 patients. Children and young adults ages 2 to 21 with a confirmed, disease-related CDKL5 gene variant were eligible to enroll. Following a 6-week baseline period, trial participants were randomized to receive either oral ganaxolone (up to 1,800 mg/day) or placebo for 17 weeks, in addition to their existing anti-seizure treatment. Following the double-blind phase, patients were eligible to continue receiving ganaxolone in an open-label extension.

    Marinus is planning to present the top-line results at an upcoming scientific meeting.

    "CDD is a severe genetic epilepsy that can cause hundreds of seizures for patients each day," commented Scott Demarest, M.D., Principal Investigator (PI) at Children's Hospital Colorado; PI of the International CDKL5 Clinical Research Network (ICCRN); Assistant Professor of Pediatrics-Neurology at the University of Colorado. "Existing antiepileptic medications fail to produce an adequate and durable response in the majority of patients. The positive results from Marinus' trial demonstrate that ganaxolone can provide significant seizure reduction in patients with CDD, an important advance for the CDD community."

    The company plans to launch an Expanded Access Program (EAP) in the fourth quarter, which will allow patients who were not able to participate in the clinical trial to begin receiving treatment with ganaxolone under a treatment protocol.

    "CDD impacts each patient differently and is an incredibly challenging form of epilepsy to treat," commented Heidi Grabenstatter, Science Director, International Foundation for CDKL5 Research. "Despite these challenges, as a patient community, we have come a long way in helping to drive innovation. We are grateful to Marinus for enabling researchers from across the globe to establish a clinical trial network, connecting with patients in need of care, and collaborating in a model for clinical research that will benefit the CDD community and the greater rare disease field in the future."

    Marinus will continue its pre-commercial development plans, while simultaneously exploring commercialization opportunities for ganaxolone in CDD with third parties to maximize access for CDD patients.

    Marinus has received a Rare Pediatric Disease (RPD) Designation from the FDA for ganaxolone for the treatment of CDD. The FDA grants an RPD Designation for diseases that affect fewer than 200,000 people in the U.S. and in which the serious or life-threatening manifestations occur primarily in individuals 18 years of age and younger. If an NDA for ganaxolone in CDD is approved, Marinus may be eligible to receive a priority review voucher from the FDA, which can be redeemed for priority review in a subsequent marketing application by Marinus or monetized by being transferred to a third party. The program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

    Corporate Update

    In July, Marinus submitted a protocol amendment to the FDA for its planned Phase 3 trial for IV ganaxolone in refractory status epilepticus (RSE), and recently received FDA feedback on the protocol. Currently, the company is engaging with the FDA to respond to their feedback prior to enrolling patients in the trial. To date, Marinus has selected 55 out of a projected 80 clinical sites to participate in the trial. The company continues to target top-line data in 1H 2022.

    Marinus will also continue its Phase 2, placebo-controlled trial of ganaxolone in PCDH-19-related epilepsy (Violet Study), with data expected in the first half of 2021. As a result of COVID-19 related delays in outpatient visits, Marinus plans on reporting data from the Phase 2 trial in tuberous sclerosis complex (TSC) in mid-2021. The company is planning to begin a Phase 3 registrational trial in mid-2021 should the Phase 2 trial support moving forward to Phase 3.

    Marinus earlier today announced a five-year cost-sharing contract with the Chemical Medical Countermeasures division of the Biomedical Advanced Research and Development Authority (BARDA), part of the U.S. Department of Health and Human Services. This agreement includes $21 million in non-dilutive funding to support the Phase 3 clinical trial Marinus is planning in RSE and preclinical studies of ganaxolone in nerve agent exposure animal models, with up to approximately $30 million in additional optional funding contingent on favorable clinical and preclinical outcomes. The BARDA contract enables Marinus to expand development of ganaxolone for treatment of RSE caused by nerve agent toxicity and supports manufacturing, supply chain, clinical, regulatory, and toxicology activities. Marinus will be responsible for cost-sharing in the amount of $33 million if all development options are completed.

    Conference Call and Webcast

    Marinus Pharmaceuticals' management will host a conference call with a live webcast today, September 14 at 4:30 pm Eastern time to discuss details of the trial findings and provide an overall business update. To listen to the conference call, interested parties within the U.S. should call +1-833-979-2765. International callers should call +1-343-761-2590. All callers should ask for the Marinus conference call. The conference call will also be available through a live webcast, which can be accessed via the company's website at www.marinuspharma.com/investors. Please note that the company will be using slides for this call, which are available on the company's website.

    A replay of the call will be available approximately one hour after the end of the call through September 21, 2020. The replay can be accessed via the Company's website or by dialing +1-800-585-8367 or +1-416-621-4642. The replay conference playback code is 7984226.

    About CDKL5 Deficiency Disorder

    CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. CDD is characterized by early-onset, difficult-to-control seizures and severe neuro-developmental impairment. Most children affected by CDD cannot walk, talk, or feed themselves. Currently, there are no therapies approved specifically for CDD.

    About Ganaxolone

    Ganaxolone, a positive allosteric modulator of GABAA receptors, is being developed in intravenous and oral formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. Unlike benzodiazepines, ganaxolone exhibits antiseizure, antidepressant and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors. More than 1,600 study participants, both adults and children, have received ganaxolone at therapeutically relevant dose levels and treatment regimens for up to four years.

    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 has conducted 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 file an NDA for ganaxolone for the treatment of CDD with the FDA by mid-2021; our expectations to file an MAA for ganaxolone for the treatment of CDD with the EMA by end Q3 2021; our expectations to begin commercial launch of ganaxolone for the treatment of CDD in the first half of 2022; our expectations to continue our investments in the oral ganaxolone franchise; our expectations to present the top-line CDD results at an upcoming scientific meeting; our expectations regarding possible commercialization opportunities for ganaxolone in CDD with third parties; our expectations to open clinical trial sites for our Phase 3 trial in status epilepticus; our expectations to release data from our Phase 3 trial in status epilepticus in 1H 2022; our expectations to release top line data from our Phase 2 open-label trial for patients with TSC in mid-2021; our expectations to begin a Phase 3 registrational trial in mid-2021; our expectations to release top line data from our Phase 2 Violet Study in the first half of 2021; our expectations regarding our agreement with BARDA; the potential safety and efficacy of ganaxolone; expectations regarding our ability to receive and utilize a priority review voucher; the therapeutic potential of ganaxolone; and our plans for an expanded access program for 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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    • 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…

    • 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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  2. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that management will present at the Morgan Stanley Virtual Global Healthcare Conference, H.C Wainwright Virtual Global Investment Conference and Cantor Virtual Global Healthcare Conference during the week of September 14.

    Additional details can be found below:

    H.C. Wainwright Virtual Global Investment Conference
    Date: Tuesday, September 15
    Time: 12:00 noon Eastern Time
    Presenter: Ed Smith, Chief Financial Officer, and Dr. Joseph Hulihan, Chief Medical Officer
    Link to webcast: https://wsw.com/webcast/hcw7/register.aspx?conf=hcw7&page=mrns

    Morgan Stanley Virtual Global

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that management will present at the Morgan Stanley Virtual Global Healthcare Conference, H.C Wainwright Virtual Global Investment Conference and Cantor Virtual Global Healthcare Conference during the week of September 14.

    Additional details can be found below:

    H.C. Wainwright Virtual Global Investment Conference

    Date: Tuesday, September 15

    Time: 12:00 noon Eastern Time

    Presenter: Ed Smith, Chief Financial Officer, and Dr. Joseph Hulihan, Chief Medical Officer

    Link to webcast: https://wsw.com/webcast/hcw7/register.aspx?conf=hcw7&page=mrns

    Morgan Stanley Virtual Global Healthcare Conference

    Date: Tuesday, September 15

    Time: 1:15 pm Eastern Time

    Presenter: Dr. Scott Braunstein, Chief Executive Officer, and Ed Smith, Chief Financial Officer

    Link to webcast: https://morganstanley.webcasts.com/starthere.jsp?ei=1362519&tp_key=5ef6204e02

    Cantor Virtual Global Healthcare Conference

    Date: Thursday, September 17

    Time: 2:00 pm Eastern Time

    Presenter: Dr. Scott Braunstein, Chief Executive Officer, and Ed Smith, Chief Financial Officer

    Link to webcast: https://www.webcaster4.com/Webcast/Page/2495/37404

    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 has conducted 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 intends to initiate a Phase 3 trial in status epilepticus. For more information visit www.marinuspharma.com.

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