1. Updated logo and brand identity highlight Marinus' focus on commitment, innovation and community as a leader in rare epilepsies

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today unveiled its new brand identity rooted in the company's guiding principles of commitment, innovation and community which are signified by three interconnected rings. The logo represents a new era as Marinus continues to advance as a leader in rare epilepsies and prepares for commercialization.

    With patients as its focus, Marinus Pharmaceuticals strives to create an awareness for the rare epilepsies it aims to treat. As such, each of the company's patient…

    Updated logo and brand identity highlight Marinus' focus on commitment, innovation and community as a leader in rare epilepsies

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today unveiled its new brand identity rooted in the company's guiding principles of commitment, innovation and community which are signified by three interconnected rings. The logo represents a new era as Marinus continues to advance as a leader in rare epilepsies and prepares for commercialization.

    With patients as its focus, Marinus Pharmaceuticals strives to create an awareness for the rare epilepsies it aims to treat. As such, each of the company's patient communities are embodied in the new logo's interconnected green, purple, and blue rings--the colors that represent CDKL5 deficiency disorder, tuberous sclerosis complex, status epilepticus, and PCDH19-related epilepsy.

    "Our current and future success will be grounded in our commitment to patients, the continued innovation of ganaxolone, and our deep partnerships within the community," said Christy Shafer, Chief Commercial Officer for Marinus Pharmaceuticals. "This new logo not only shows the shared interconnectivity and continued dedication to these foundations but also our evolution as a late-stage pharmaceutical company."

    In accordance with the rebrand, Marinus is unveiling a variety of updated communication channels including its website, Twitter, LinkedIn, Facebook and YouTube.

    To learn more about Marinus Pharmaceuticals and its focus on commitment, innovation, and community, visit: www.marinuspharma.com.

    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, antidepressant and anti-anxiety effects. Ganaxolone is being developed in IV and oral dose formulations 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 initiating a Phase 3 trial in status epilepticus. For more information visit www.marinuspharma.com.

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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 Scott Braunstein, M.D., Chief Executive Officer and Joseph Hulihan, M.D., Chief Medical Officer will present a corporate overview during the 10th Annual SVB Leerink Global Healthcare Conference on February 25 at 1:40 PM Eastern Standard Time. Company management will also participate in the Cowen 41st Annual Health Care Conference on March 1 at 10:20 AM (EST), the H.C. Wainwright Global Life Sciences Conference on March 9at 3:00 PM (EST), and the Oppenheimer 31st Annual Healthcare Conference on March 16 at 8:00 AM (EST).

    Additional details can be found below:

    10th…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that Scott Braunstein, M.D., Chief Executive Officer and Joseph Hulihan, M.D., Chief Medical Officer will present a corporate overview during the 10th Annual SVB Leerink Global Healthcare Conference on February 25 at 1:40 PM Eastern Standard Time. Company management will also participate in the Cowen 41st Annual Health Care Conference on March 1 at 10:20 AM (EST), the H.C. Wainwright Global Life Sciences Conference on March 9 at 3:00 PM (EST), and the Oppenheimer 31st Annual Healthcare Conference on March 16 at 8:00 AM (EST).

    Additional details can be found below:

    10th Annual SVB Leerink Global Healthcare Conference

    • Date and Time: Thursday, February 25 at 1:40 PM (EST)
    • Speakers: Scott Braunstein, M.D., Chief Executive Officer and Joseph Hulihan, M.D., Chief Medical Officer

    Cowen 41st Annual Health Care Conference

    • Date and Time: Monday, March 1 at 10:20 AM (EST)
    • Speakers: Scott Braunstein, M.D., Chief Executive Officer and Joseph Hulihan, M.D., Chief Medical Officer

    H.C. Wainwright Global Life Sciences Conference

    • Date and Time: Accessible Tuesday, March 9 at 3:00 PM (EST)
    • Speaker: Scott Braunstein, M.D., Chief Executive Officer

    Oppenheimer's 31st Annual Healthcare Conference

    • Date and Time: Tuesday, March 16 at 8:00 AM (EST)
    • Speakers: Scott Braunstein, M.D., Chief Executive Officer and Joseph Hulihan, M.D., Chief Medical Officer

    Webcasts may be accessed through the Events & Presentations page of the Investors & Media section of Marinus website, www.marinuspharma.com.

    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 conducting a Phase 3 trial in status epilepticus. For more information visit www.marinuspharma.com.

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  3. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to five new employees. The Compensation Committee of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 72,500 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    Of these stock option grants, 12,500 stock option grants have an exercise price $12.60 per share, which is equal to the closing price of Marinus' common stock on January 15, 2021, the…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to five new employees. The Compensation Committee of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 72,500 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    Of these stock option grants, 12,500 stock option grants have an exercise price $12.60 per share, which is equal to the closing price of Marinus' common stock on January 15, 2021, the date of grant for the stock options; 52,000 stock option grants have an exercise price of $12.67 per share, which is equal to the closing price of Marinus' common stock on January 19, 2021, the date of grant for the stock options; 4,000 stock option grants have an exercise price of $12.65 per share, which is equal to the closing price of Marinus' common stock on February 1, 2021, the date of grant for the stock options; and 4,000 stock option grants will be priced on the closing price of the employee's future start date, which will be considered the date of grant for the stock options. All of the stock options will vest and become exercisable as to 25% of the underlying shares on the one-year anniversary of the applicable employee's start date of employment, and will vest and become exercisable as to the remaining 75% of the underlying shares in 36 equal monthly installments at the end of each month following such anniversary, subject to the applicable employee's continued employment with Marinus on such vesting dates. The stock options were granted as an inducement material to the employee entering into employment with Marinus in accordance with Nasdaq Listing Rule 5635(c)(4), and are subject to the terms and conditions of the applicable award agreement covering such grant.

    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 conducting 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 and the clinical development schedule and milestones. Forward-looking statements in this 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; clinical trial results may not support 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; 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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  4. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it plans to release financial results for the fourth quarter and fiscal year ended December 31, 2020 before the market opens on March 9, 2021. The company will host a conference call at 8:30am Eastern Time on March 9, 2021 to provide a business update and discuss the financial results.

    Tuesday, March 9: 8:30 AM Eastern Time

    Domestic:

     

     

    (833) 979-2765

    International:

     

     

    (343) 761-2590

    Webcast:

     

     

    https://event.on24.com/wcc/r/2948497/012AE4F51A475A8BDC3559C95635FBF2

    Conference ID:

     

    2673136

    About Marinus Pharmaceuticals

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it plans to release financial results for the fourth quarter and fiscal year ended December 31, 2020 before the market opens on March 9, 2021. The company will host a conference call at 8:30am Eastern Time on March 9, 2021 to provide a business update and discuss the financial results.

    Tuesday, March 9: 8:30 AM Eastern Time

    Domestic:

     

     

    (833) 979-2765

    International:

     

     

    (343) 761-2590

    Webcast:

     

     

    https://event.on24.com/wcc/r/2948497/012AE4F51A475A8BDC3559C95635FBF2

    Conference ID:

     

    2673136

    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 conducting a Phase 3 trial in status epilepticus. For more information visit www.marinuspharma.com.

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  5. Marinus and Ceribell, Inc. agree to place handheld Ceribell Rapid Response EEG systems in hospitals in connection with its Phase 3 RSE clinical trial

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the enrollment of the first patient in a Phase 3 clinical trial of IV ganaxolone (RAISE trial) for the treatment of refractory status epilepticus (RSE), a life-threatening occurrence of continued or intermittent seizures lasting more than five minutes in duration without recovery of consciousness. RSE is considered a medical emergency that can cause permanent brain damage or death if not quickly controlled.

    The RAISE trial…

    Marinus and Ceribell, Inc. agree to place handheld Ceribell Rapid Response EEG systems in hospitals in connection with its Phase 3 RSE clinical trial

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the enrollment of the first patient in a Phase 3 clinical trial of IV ganaxolone (RAISE trial) for the treatment of refractory status epilepticus (RSE), a life-threatening occurrence of continued or intermittent seizures lasting more than five minutes in duration without recovery of consciousness. RSE is considered a medical emergency that can cause permanent brain damage or death if not quickly controlled.

    The RAISE trial is a randomized, double-blind, placebo-controlled trial that will evaluate the efficacy and safety of IV ganaxolone in RSE patients who have failed benzodiazepines and two or more second line intravenous antiepileptic drugs (AEDs). The study will examine the proportion of participants with RSE who experience status epilepticus cessation within 30 minutes of initiation of IV ganaxolone as well as the rate of progression to IV anesthesia. Cessation will be measured and determined by clinical and EEG findings.

    Marinus also announced an agreement with Ceribell, Inc. that will place the handheld Ceribell Rapid Response EEG system in up to 80 U.S. hospitals participating in its study. The electroencephalogram (EEG) devices are designed to be compact and easy to use and will facilitate the rapid diagnosis of nonconvulsive SE, which is confirmed by EEG demonstration of ongoing ictal activity.

    The Ceribell Rapid Response EEG system is intended to provide bedside clinicians the ability to quickly triage and continuously monitor patients at risk for seizures. Within the RAISE trial, it will allow clinicians to rapidly establish a diagnosis of nonconvulsive SE in the absence of immediate availability of conventional EEG. The Ceribell 20-minute certification video trains the hospital neurology team members to install the Ceribell Rapid Response EEG headband in less than seven minutes. The headband collects EEG data via a web-based portal, allowing the hospital neurology team to access the data remotely.

    Ganaxolone 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.

    About the RAISE Trial

    The Phase 3 RAISE Trial is a randomized, double-blind, placebo-controlled trial in status epilepticus (SE) patients who have failed benzodiazepines and two or more second line intravenous AEDs. Approximately 80 study sites in hospitals across the U.S. will participate. The trial is designed to enroll approximately 125 patients, who will be randomized to receive ganaxolone or placebo added to standard of care. With this number of participants, the study is designed to provide over 90 percent power to detect a 30 percent efficacy difference between ganaxolone and placebo.

    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 Status Epilepticus (SE)

    SE is a condition characterized by prolonged or repetitive seizures which, if treatment is delayed or ineffective, may result in medical or neurologic complications. SE is a neurologic emergency affecting between 75,000 – 150,00 patients in the U.S. each year. Benzodiazepines are the first-line treatment for SE. If these are not successful in terminating SE, one or more IV AEDs are administered as the second line of treatment. If treatment with the first IV AED following benzodiazepines is ineffective, the patient is considered to have refractory SE (RSE).

    About the Ceribell Rapid Response EEG System

    The Ceribell Rapid Response EEG System consists of a 10-electrode (8-channel) headband, an EEG recorder with Brain Stethoscope feature and a cloud portal for storing and reviewing EEGs. This simplified configuration of EEG enables easy access to EEG within minutes. The Ceribell EEG System received FDA 510(k) clearance in 2017 and is commercially available in the U.S.

    About Ceribell

    Ceribell, Inc. (www.ceribell.com), is headquartered in Mountain View, CA. Ceribell is focused on making EEG widely available, more efficient, and more cost-effective to improve the diagnosis and treatment of patients at risk for seizures.

    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 conducting 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 for clinical trial sites for our Phase 3 trial in refractory status epilepticus; 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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  6. Marinus is moving towards a pre-NDA meeting, targeted for the end of Q1, in support of NDA submission by the end of Q2

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it has received a positive response from the U.S. Food and Drug Administration (FDA) that the efficacy and safety data resulting from the company's pivotal Phase 3 Marigold Study on the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD) appear sufficient to support the filing of a New Drug Application (NDA). Adequacy of these data to support an approval of ganaxolone for the proposed indication will be a matter of…

    Marinus is moving towards a pre-NDA meeting, targeted for the end of Q1, in support of NDA submission by the end of Q2

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it has received a positive response from the U.S. Food and Drug Administration (FDA) that the efficacy and safety data resulting from the company's pivotal Phase 3 Marigold Study on the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD) appear sufficient to support the filing of a New Drug Application (NDA). Adequacy of these data to support an approval of ganaxolone for the proposed indication will be a matter of future FDA review.

    CDD is a rare, genetic epilepsy with refractory seizures. Marinus provided the data to the FDA in a briefing document meant to support a Type C meeting, and the FDA provided written preliminary comments in response. Based on this feedback, Marinus is targeting a pre-NDA meeting by the end of Q1 to support submission.

    "We are pleased with the FDA's assessment of the sufficiency of the efficacy and safety data from one Phase 3 clinical trial to support an NDA filing," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "We remain on track to submit the NDA for use of ganaxolone in CDD for FDA review by mid-2021 and look forward to continued advancement of ganaxolone as the first potential FDA approved treatment specifically indicated in patients diagnosed with CDD."

    In September, Marinus reported positive topline data from the Phase 3 Marigold Study, the first double-blind placebo-controlled trial to provide evidence of efficacy in CDD and the first Phase 3 trial to examine three times a day dosing of ganaxolone in pediatric patients.

    In the Phase 3 Marigold trial, patients treated with ganaxolone showed a significant 32.2% median reduction in 28-day major motor seizure frequency, compared to a 4.0% reduction for those receiving the placebo, achieving the trial's primary endpoint (p=0.002). In this trial, ganaxolone was generally well tolerated with a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence.

    "We see continued progress in our other development programs," continued Dr. Braunstein. "Marinus continues to initiate new sites to participate in the Phase 3 clinical trial of IV ganaxolone for the treatment of Refractory Status Epilepticus (RSE) or RAISE trial. While the COVID-19 pandemic is lengthening the time for new trial sites to open for enrollment, we do not anticipate this having major implications on the ultimate timing of trial results."

    "The Phase 2 Violet Study, evaluating both three times a day dosing of ganaxolone and the role of allopregnanolone sulfate as a biomarker in PCDH19-related epilepsy remains on track. In March, we plan to provide an update on this program," said Dr. Braunstein. "Additionally, we expect to report top line data from our Phase 2 Tuberous Sclerosis Complex trial by late Q2."

    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, antidepressant and anti-anxiety effects. Ganaxolone is being developed in IV and oral dose formulations 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 initiating a Phase 3 trial in status epilepticus.

    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 plans to have a pre-NDA meeting with FDA for submission of an NDA for use of ganaxolone in CDKL5 Deficiency Disorder (CDD) by the end of first quarter 2021; our expectations to file an NDA for ganaxolone in CDD by end of second quarter 2021; our ability to receive FDA approval of ganaxolone in CDD; our plan to provide an update on our Phase 2 Violet Study in March 2021; our expectations that COVID-19 will not have major implications on the ultimate timing of trial results for our RAISE Phase 3 clinical trial in Refractory Status Epilepticus; our expectation to report top line data from our Phase 2 Tuberous Sclerosis Complex trial by late second quarter 2021. Forward-looking statements in this 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; clinical trial results may not support 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; the interpretation of clinical trial results and the ability of clinical trial results to support regulatory approval; our ability to obtain and maintain regulatory approval for our product candidate; 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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  7. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that company management will present a corporate overview during the 39th Annual J.P. Morgan Healthcare Conference. An audio-only presentation will take place January 14, 2021 at 3:40 pm EST. Company management will also participate in the H.C. Wainwright Bioconnect 2021 Conference on January 11.

    Additional details can be found below:

    H.C. Wainwright Bioconnect 2021 Conference

    • Date and Time: On-demand starting Monday, January 11 6:00 am (EST)
    • Speakers: Scott Braunstein, M.D., Chief Executive Officer, Edward Smith, Chief Financial Officer, and Joseph Hulihan, M.D…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that company management will present a corporate overview during the 39th Annual J.P. Morgan Healthcare Conference. An audio-only presentation will take place January 14, 2021 at 3:40 pm EST. Company management will also participate in the H.C. Wainwright Bioconnect 2021 Conference on January 11.

    Additional details can be found below:

    H.C. Wainwright Bioconnect 2021 Conference

    39th Annual J.P. Morgan Healthcare Conference

    Webcast and audio replays will be available following each event under the Investors & Media page of the Company's website at www.marinuspharma.com.

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

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  8. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it has been selected for inclusion in the Nasdaq Biotechnology Index (NBI), which became effective prior to market open today.

    "On the tails of a momentous year, we are pleased to be included in the Nasdaq Biotechnology Index," said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. "Index inclusion recognizes our clinical progress and heightened investor awareness as we remain committed to rapidly advancing our pipeline in rare epilepsies and neuropsychiatric disorders."

    The NBI is designed to track the performance of a set of securities listed…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it has been selected for inclusion in the Nasdaq Biotechnology Index (NBI), which became effective prior to market open today.

    "On the tails of a momentous year, we are pleased to be included in the Nasdaq Biotechnology Index," said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. "Index inclusion recognizes our clinical progress and heightened investor awareness as we remain committed to rapidly advancing our pipeline in rare epilepsies and neuropsychiatric disorders."

    The NBI is designed to track the performance of a set of securities listed on The Nasdaq Stock Market that are classified as either biotechnology or pharmaceutical according to the Industry Classification Benchmark (ICB).

    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 formulations 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 has also initiated 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. Forward-looking statements in this 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; clinical trial results may not support 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; 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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  9. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the pricing of an underwritten public offering of 5 million shares of its common stock for gross proceeds of $70 million, before deducting the underwriting discounts and commissions and other estimated offering expenses payable by Marinus. All of the shares of common stock are being offered by Marinus. The closing of the offering is expected to occur on or about December 10, 2020, subject to the satisfaction of customary closing conditions. In addition, Marinus has granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of common stock…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the pricing of an underwritten public offering of 5 million shares of its common stock for gross proceeds of $70 million, before deducting the underwriting discounts and commissions and other estimated offering expenses payable by Marinus. All of the shares of common stock are being offered by Marinus. The closing of the offering is expected to occur on or about December 10, 2020, subject to the satisfaction of customary closing conditions. In addition, Marinus has granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of common stock.

    Cantor Fitzgerald & Co. is acting as sole bookrunner for the offering. H.C. Wainwright & Co. is acting as lead manager for the offering.

    The underwriters may offer the shares from time to time for sale in one or more transactions on the Nasdaq Global Market, in the over-the-counter market, through negotiated transactions or otherwise at market prices prevailing at the time of sale, at prices related to prevailing market prices or at negotiated prices. On December 7, 2020, the last sale price of the shares as reported on the Nasdaq Global Market was $15.61 per share.

    Marinus intends to use the net proceeds of the offering to fund the development of its product candidates and for general corporate purposes, which may include working capital, capital expenditures, research and development expenditures, clinical trial expenditures, acquisitions of new technologies, products or businesses, and investments.

    The securities described above are being offered by Marinus pursuant to a shelf registration statement on Form S-3 (No. 333-239780) declared effective by the Securities and Exchange Commission (the "SEC") on July 27, 2020.

    A preliminary prospectus supplement relating to the offering was filed with the SEC on December 7, 2020 and is available on the SEC's website at http://www.sec.gov. A final prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and also will be available on the SEC's website. Before investing in the offering, you should read each of the prospectus supplement and the accompanying prospectus relating to the offering in their entirety as well as the other documents that Marinus has filed (or will file) with the SEC that are incorporated by reference in the prospectus supplement and the accompanying prospectus relating to the offering, which provide more information about Marinus and the offering. Copies of the final prospectus supplement, when available, and the accompanying prospectus relating to the offering may be obtained from Cantor Fitzgerald & Co., 499 Park Avenue, 6th Floor, New York, New York 10022, Attn: Capital Markets Department, or by email at .

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    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 formulations 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 initiating a Phase 3 trial in status epilepticus.

    Forward-Looking Statements

    To the extent any statements made in this press release deal with information that is not historical, these are forward-looking statements under the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements regarding the closing of the offering, as well as the anticipated use of proceeds for the offering and other statements identified by words such as "will," "potential," "could," "can," "believe," "intends," "continue," "plans," "expects," "anticipates," "estimates," "may," other words of similar meaning or the use of future dates. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Uncertainties and risks may cause Marinus' actual results to be materially different than those expressed in or implied by Marinus' forward-looking statements. For Marinus, this includes satisfaction of the customary closing conditions to the offering, delays in obtaining required stock exchange or other regulatory approvals, stock price volatility, the impact of general business and economic conditions, the expected gross proceeds from the offering and the intended use of proceeds of the offering. More detailed information on these and additional factors that could affect Marinus' actual results are described in Marinus' filings with the Securities and Exchange Commission, including its most recent quarterly report on Form 10-Q. All forward-looking statements in this news release speak only as of the date of this news release. Marinus undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

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  10. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced an underwritten public offering of its common stock. All of the shares to be sold in the offering will be offered by Marinus. Marinus has granted the underwriters a 30-day option to purchase up to an additional 15 percent of the number of shares offered in the public offering.

    Cantor Fitzgerald & Co. is acting as sole bookrunner for the proposed offering.

    Marinus intends to use the net proceeds of the offering to fund the development of its product candidates and for general corporate purposes, which may include working capital, capital expenditures, research and development…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced an underwritten public offering of its common stock. All of the shares to be sold in the offering will be offered by Marinus. Marinus has granted the underwriters a 30-day option to purchase up to an additional 15 percent of the number of shares offered in the public offering.

    Cantor Fitzgerald & Co. is acting as sole bookrunner for the proposed offering.

    Marinus intends to use the net proceeds of the offering to fund the development of its product candidates and for general corporate purposes, which may include working capital, capital expenditures, research and development expenditures, clinical trial expenditures, acquisitions of new technologies, products or businesses, and investments.

    The securities described above will be offered by Marinus pursuant to a shelf registration statement on Form S-3 (No. 333-239780) declared effective by the Securities and Exchange Commission (the "SEC") on July 27, 2020.

    The securities will be offered only by means of a prospectus supplement and accompanying prospectus relating to the offering that form a part of the registration statement. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC's website at http://www.sec.gov. Copies of the preliminary prospectus supplement, when available, and the accompanying prospectus relating to the offering may be obtained from Cantor Fitzgerald & Co., 499 Park Avenue, 6th Floor, New York, New York 10022, Attn: Capital Markets Department, or by email at .

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    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 formulations 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 initiating a Phase 3 trial in status epilepticus.

    Forward-Looking Statements

    To the extent any statements made in this press release deal with information that is not historical, these are forward-looking statements under the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements regarding the timing and success of the proposed offering, as well as the anticipated use of proceeds for the proposed offering and other statements identified by words such as "will," "potential," "could," "can," "believe," "intends," "continue," "plans," "expects," "anticipates," "estimates," "may," other words of similar meaning or the use of future dates. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Uncertainties and risks may cause Marinus' actual results to be materially different than those expressed in or implied by Marinus' forward-looking statements. For Marinus, this includes satisfaction of the customary closing conditions of the offering, delays in obtaining required stock exchange or other regulatory approvals, stock price volatility and the impact of general business and economic conditions. More detailed information on these and additional factors that could affect Marinus' actual results are described in Marinus' filings with the Securities and Exchange Commission, including its most recent quarterly report on Form 10-Q. All forward-looking statements in this news release speak only as of the date of this news release. Marinus undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

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    • Top-line data from the Marigold Study in CDKL5 deficiency disorder (CDD) presented as late breaking results during AES scientific sessions; An analysis of data from the Marigold Study shows a consistent efficacy signal across multiple subgroups, including a 36.7% estimated difference in major motor seizure frequency relative to placebo in U.S. patients (n=41)
    • Preliminary analysis of the Marigold Study open label extension provides supportive data on the durability of ganaxolone efficacy data, including a 52.7% median major motor seizure frequency reduction in patients who have completed approximately one year of therapy

    • Higher average ganaxolone plasma concentrations correlated with improved seizure frequency reductions in CDD patients.
    • Top-line data from the Marigold Study in CDKL5 deficiency disorder (CDD) presented as late breaking results during AES scientific sessions; An analysis of data from the Marigold Study shows a consistent efficacy signal across multiple subgroups, including a 36.7% estimated difference in major motor seizure frequency relative to placebo in U.S. patients (n=41)
    • Preliminary analysis of the Marigold Study open label extension provides supportive data on the durability of ganaxolone efficacy data, including a 52.7% median major motor seizure frequency reduction in patients who have completed approximately one year of therapy



    • Higher average ganaxolone plasma concentrations correlated with improved seizure frequency reductions in CDD patients. Findings support three times a day dosing as utilized in the Marigold Study and studies in other chronic epilepsy indications, as well as future oral formulation development designed to improve ganaxolone exposure levels
    • Two case studies on pediatric super-refractory status epilepticus (SRSE) are the first published abstracts on the use of IV ganaxolone in treating SRSE
    • Marinus also announces an expanded access program (EAP) for U.S. patients with CDD, making ganaxolone available to patients under a treatment IND

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it will host a virtual Scientific Exhibit at the American Epilepsy Society (AES) Annual Meeting (AES2020) from 8:00 AM to 11:00 AM Eastern Time today. At the Scientific Exhibit, Marinus will have 10 posters highlighting the company's clinical development programs in treating CDKL5 deficiency disorder (CDD) and refractory status epilepticus (RSE).

    "We are excited to share the important insights and data we have compiled for this year's AES meeting, with four scientific posters, two of which were accepted as late breaking results – one in CDD and another in RSE. We will also highlight key data from the Marigold Study during our Scientific Exhibit, with analyses on patient subsets, preliminary durability of effect in the open label portion of the trial, and on the PK/PD profile of ganaxolone," said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. "We also look forward to discussing these data further at our AES Virtual Investor Event later today."

    Highlights from new analyses in the Phase 3 Marigold Study of ganaxolone in CDD presented during the Scientific Exhibit include the following:

    "Effect of Ganaxolone on Seizure Frequency Across Subpopulations of Patients with CDKL5 Deficiency Disorder: Subgroup Analyses of the Marigold Study," by E. M. Pestana-Knight, et al.

    An analysis of data from the Marigold Study showed patients on ganaxolone consistently demonstrated numerical improvements in the change in major motor seizure frequency relative to placebo across the following subgroups: age, gender, baseline seizure frequency, number of concomitant antiepileptic drugs, geography and baseline allopregnanolone sulfate levels. These findings support ganaxolone's effect across the broad CDD population studied. Data collected in patients enrolled in the United States (n=41) showed a stronger effect with a 36.7% estimated difference in major motor seizure frequency relative to placebo.

    "Pharmacokinetic-Pharmacodynamic Analysis in Ganaxolone-Treated Patients with CDKL5 Deficiency Disorder: Results From the Marigold Study," by J. Hulihan, et al.

    Higher plasma ganaxolone concentrations were correlated with greater reductions in major motor seizure frequency. However, no differences in the rates of relevant adverse events were noted across ganaxolone plasma concentration groups. These findings highlight the importance of achieving adequate plasma ganaxolone concentrations and suggest that three times a day (TID) dosing has the potential to increase trough ganaxolone levels and may provide improved seizure control. In addition, this analysis supports efforts to develop new oral ganaxolone formulations that aim to improve pharmacokinetic properties to better achieve target ganaxolone exposure levels.

    "Extended Duration Safety and Efficacy of Ganaxolone for the Treatment of CDKL5 Deficiency Disorder: Preliminary Open-Label Extension Analysis (Marigold Study)," by N. Specchio, et al.

    Preliminary data from patients in the open-label extension phase of the Marigold Study as of September 2020 were summarized. Patients on ganaxolone in the double-blind phase continued to experience a maintained improvement in major motor seizure frequency through eight months in the open-label extension, representing approximately 12 months in total on ganaxolone. This group of patients treated with ganaxolone for 12 months (n=17) experienced a median 52.7% reduction in major motor seizure frequency relative to baseline.

    Patients on placebo in the double-blind phase showed improvements in major motor seizure frequency when transitioning to open label ganaxolone. These preliminary findings suggest that ganaxolone has the potential to provide clinically meaningful, durable seizure improvements in patients with CDD.

    The following posters were presented during the AES Scientific Sessions:

    For full list of posters presented, please visit the Marinus Website Poster Page.

    Corporate Update:

    Status Epilepticus

    Marinus is currently recruiting patients in RSE in its Phase 3 RAISE clinical trial. In addition, the company is planning a clinical trial of ganaxolone in Established Status Epilepticus (ESE) in patients presenting to emergency rooms, with a projected trial initiation in 2H 2021. Marinus also intends to continue making ganaxolone available to clinicians who request the drug for treatment of super refractory status epilepticus on a case-by-case basis under emergency IND treatment.

    Expanded access program (EAP) in CDD makes ganaxolone broadly available to patients

    During AES2020, Marinus announced the launch of an expanded access program (EAP) that will allow the company to offer ganaxolone to U.S. patients with CDD, including children at least two years of age, who were unable to participate in the company's Phase 3 Marigold Study. This will provide patients with CDD access to ganaxolone under a treatment IND.

    CDD Regulatory Plans

    Marinus is preparing to meet with the FDA in Q1 2021 in connection with its planned mid-2021 NDA submission for CDD, with a 1H 2021 pre-marketing authorization application (MAA) meeting with the European Medicines Agency (EMA).

    Investor Meeting Today

    Marinus to Host Virtual Investor Event at AES today from 12:00 PM – 2:00 PM Eastern Time. To register in advance and listen to the live webinar please click on http://wsw.com/webcast/cc/mrns.

    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 formulations 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 initiating 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 plans to initiate a clinical trial of ganaxolone in ESE in the second half of 2021; our expectations to meet with the FDA regarding our NDA for ganaxolone for the treatment of CDD in the first quarter of 2021; our expectation to submit an NDA for ganaxolone for the treatment of CDD in mid-2021; our expectation to have a pre-MAA meeting with the EMA in the first half of 2021; our clinical development plans for ganaxolone; and for the safety and efficacy of ganaxolone. Forward-looking statements in this 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; clinical trial results may not support 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; 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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  11. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the launch of an expanded access program (EAP) that will allow the company to offer ganaxolone to patients with CDKL5 Deficiency Disorder (CDD) who were unable to participate in the company's Marigold Study Phase 3 clinical trial.

    "With the positive results in our clinical trial, we felt it was an important measure to make ganaxolone available to patients with CDD as we prepare to submit a new drug application to the FDA," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "There are currently no approved treatments specifically for patients with CDD, creating…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the launch of an expanded access program (EAP) that will allow the company to offer ganaxolone to patients with CDKL5 Deficiency Disorder (CDD) who were unable to participate in the company's Marigold Study Phase 3 clinical trial.

    "With the positive results in our clinical trial, we felt it was an important measure to make ganaxolone available to patients with CDD as we prepare to submit a new drug application to the FDA," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "There are currently no approved treatments specifically for patients with CDD, creating a strong need to provide early, compassionate use access to investigational ganaxolone therapy for these children."

    Candidates for the EAP must be at least two years of age, with a confirmed diagnosis of CDD, and experiencing uncontrolled seizures. The investigational treatment will initially be available in sites in the United States. It is important to remember that investigational drugs have not yet received regulatory approval; therefore, their potential risks and benefits are not yet established. Doctors and patients should consider all possible benefits and risks when seeking expanded access to an investigational drug.

    In the Marigold Study, patients given oral ganaxolone showed a significant 32.2% median reduction in 28-day major motor seizure frequency, compared to a 4.0% reduction for those receiving placebo. In this trial, ganaxolone was generally well tolerated with a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence. Marinus is preparing to meet with the U.S. Food and Drug Administration (FDA) in Q1 2021 regarding its planned mid-2021 NDA submission, as well as a 1H 2021 pre-marketing authorization application (MAA) meeting with the European Medicines Agency (EMA).

    For more information on the Marinus Expanded Access Treatment Protocol for ganaxolone, please visit https://marinuspharma.com/expanded-access-program/

    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 formulations 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 has also initiated 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 plans to meet with the U.S. Food and Drug Administration (FDA) in Q1 2021; our plans to submit a new drug application to the FDA in mid- 2021; our plans to meet with the European Medicines Agency (EMA) in first half of 2021 regarding a pre-marketing authorization application (MAA). Forward-looking statements in this 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; clinical trial results may not support 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; 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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  12. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to two new employees. The Compensation Committee of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 10,000 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    These stock option grants have an exercise price of $14.54 per share, which is equal to the closing price of Marinus' common stock on December 3, 2020, the date of grant for the stock…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to two new employees. The Compensation Committee of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 10,000 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    These stock option grants have an exercise price of $14.54 per share, which is equal to the closing price of Marinus' common stock on December 3, 2020, the date of grant for the stock options. All of the stock options will vest and become exercisable as to 25% of the underlying shares on the one-year anniversary of the employee's start date of employment, and will vest and become exercisable as to the remaining 75% of the underlying shares in 36 equal monthly installments at the end of each month following such anniversary, subject to the employee's continued employment with Marinus on such vesting dates. The stock options were granted as an inducement material to the employee entering into employment with Marinus in accordance with Nasdaq Listing Rule 5635(c)(4), and are subject to the terms and conditions of the applicable award agreement covering such grant.

    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 initiating 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 and the clinical development schedule and milestones. Forward-looking statements in this 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; clinical trial results may not support 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; 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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  13. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it will host a webinar featuring a clinical update and commercial overview on its pipeline programs in status epilepticus, CDKL5 deficiency disorder, and Tuberous sclerosis complex, on Monday, December 7 from 12:00 PM – 2:00 PM Eastern time.

    Preliminary Agenda:

     

    Marinus Overview

    Dr. Scott Braunstein, M.D., Chief Executive Officer & Director

     

    CDKL5 Deficiency Disorder (CDD) Introduction

    Dr. Elia M. Pestana Knight, Pediatric Epileptologist at the Cleveland Clinic CDKL5 Center of Excellence

     

    Conversations with CDD Caregivers and Community (Video)

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it will host a webinar featuring a clinical update and commercial overview on its pipeline programs in status epilepticus, CDKL5 deficiency disorder, and Tuberous sclerosis complex, on Monday, December 7 from 12:00 PM – 2:00 PM Eastern time.

    Preliminary Agenda:

     

    Marinus Overview

    Dr. Scott Braunstein, M.D., Chief Executive Officer & Director

     

    CDKL5 Deficiency Disorder (CDD) Introduction

    Dr. Elia M. Pestana Knight, Pediatric Epileptologist at the Cleveland Clinic CDKL5 Center of Excellence

     

    Conversations with CDD Caregivers and Community (Video)

    Dr. Orrin Devinsky, a neurologist specializing in epilepsy treatments and Director of the NYU Comprehensive Epilepsy Center and the The Institute of Neurology and Neurosurgery at Saint Barnabas

     

    Heidi Grabenstatter, Science Director at the International Foundation for CDKL5 Research (IFCR)

     

    CDD Posters & CDD Expanded Access Program (EAP) Details

    Alex Aimetti, Head of Scientific Affairs

     

    Status Epilepticus (focus on RSE, ESE & SRSE)

    Joe Hulihan, M.D., Chief Medical Officer & Henrikas Vaitkevicius, M.D., Vice President, Clinical Development

     

    Commercial Strategy/Market Opportunity

    Christy Shafer, Chief Commercial Officer

    To register in advance and listen to the live webinar please click on http://wsw.com/webcast/cc/mrns.

    Dr. Elia M. Pestana Knight is a pediatric epileptologist in the Pediatric Epilepsy Section, Epilepsy Center, Cleveland Clinic Neurological Institute, Cleveland, Ohio. She is also an Associate Professor of Medicine at the Cleveland Clinic Lerner College of Medicine.

    Dr. Pestana Knight completed medical school in Cuba (1991) where she graduated at the top of her class. She trained in general neurology (1995) and pediatric neurology (1996) at the Instituto Nacional de Neurologia y Neurocirugia, Havana, Cuba. After moving to the United States in 1999, Dr. Pestana Knight completed a research fellowship in epilepsy at the Cleveland Clinic (2004), residency training in Pediatrics at Rainbow Babies and Children's Hospital (2007), and Pediatric Neurology residency at the University of Michigan (2010). She is board certified in Pediatrics, Neurology with special qualifications in Child Neurology and Epilepsy.

    Dr. Pestana Knight has more than 40 papers published in peer-reviewed journals related to epilepsy and neurological disorders. Her areas of research interest include epileptic encephalopathy, refractory epilepsies, comorbidities in pediatric epilepsy, epilepsy surgery and pediatric epilepsy outcomes. Dr. Pestana Knight is an enthusiastic teacher. She loves taking care of children with epilepsy and their families, particularly children with refractory seizures. She currently lives in Cleveland, Ohio.

    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 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 initiating a Phase 3 trial in status epilepticus.

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  14. Six Additional Posters to be Presented in the Marinus Scientific Exhibit

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that four abstracts highlighting clinical trial and research data in the use of ganaxolone in treating CDKL5 deficiency disorder (CDD) and refractory status epilepticus (SE) have been selected for poster presentation at the American Epilepsy Society (AES) Annual Meeting (AES2020), being held virtually from December 4 through 8, 2020.

    Six additional Marinus-related posters are to be shown in a virtual Scientific Exhibit to be held Monday, December 7th, from 8:00 to 11:00 AM EST.

    "Throughout 2020…

    Six Additional Posters to be Presented in the Marinus Scientific Exhibit

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that four abstracts highlighting clinical trial and research data in the use of ganaxolone in treating CDKL5 deficiency disorder (CDD) and refractory status epilepticus (SE) have been selected for poster presentation at the American Epilepsy Society (AES) Annual Meeting (AES2020), being held virtually from December 4 through 8, 2020.

    Six additional Marinus-related posters are to be shown in a virtual Scientific Exhibit to be held Monday, December 7th, from 8:00 to 11:00 AM EST.

    "Throughout 2020, Marinus has strived to lead the way in a variety of clinical trials for many rare, difficult-to-treat seizure disorders, and we are excited to share further details on the positive results we saw in our pivotal Marigold Phase 3 study in CDKL5 deficiency disorder, as well as subset analyses and PK data," said Scott Braunstein, M.D., Chief Executive Officer of Marinus Pharmaceuticals. "Furthermore, the potential for ganaxolone, in our intravenous formulation, will also be highlighted at the meeting with the wide range of data and patient case studies in earlier and later lines of status epilepticus."

    AES2020 is an all virtual event for epilepsy professionals in academia, clinical practice, industry, and advocacy. Details for the Marinus poster presentations include:

    "IV Ganaxolone in Pediatric Super-Refractory Status Epilepticus: A Single Patient Case Study"

    R. Singh, et al.

    Poster #209

    Saturday, December 5, 2020

    9:00 to 10:30 AM EST

    "Ganaxolone Significantly Reduces Major Motor Seizures Associated with CDKL5 Deficiency Disorder: A Randomized, Double-blind, Placebo-Controlled Phase 3 Study (Marigold Study)"

    E. M. Pestana-Knight, et al.

    Poster #419

    Sunday, December 6, 2020

    12:30 to 1:30 PM EST

    "Status Epilepticus: Inpatient Burden of Illness and Association with Disease Severity"

    E. L. Guterman, et al.

    Poster #821

    Monday, December 7, 2020

    9:00 to 10:30 AM EST

    "Treatment of Super Refractory Status Epilepticus Using Intravenous Ganaxolone in a Patient with Lennox-Gastaut Syndrome and Angelman Syndrome"

    M. G. Chez, J. Hulihan, and M. Gasior

    Poster #86

    Saturday, December 5, 2020

    9:00 to 10:30 AM EST

    Scientific Exhibit

    Marinus will also participate in a Scientific Exhibit on Monday, December 7, from 8:00 to 11:00 AM EST. Those registered to attend the AES2020 conference can also visit the Scientific Exhibit. Some of the posters being presented by Marinus include:

    • "Intravenous Ganaxolone for the Treatment of Refractory Status Epilepticus: Results From an Open-Label, Dose-Finding, Phase 2 Study," by H. Vaitkevicius, et al.
    • "Effect of Ganaxolone on Seizure Frequency Across Subpopulations of Patients with CDKL5 Deficiency Disorder: Subgroup Analyses of the Marigold Study," by E. M. Pestana-Knight, et al.
    • "Pharmacokinetic-Pharmacodynamic Analysis of Oral Ganaxolone in Patients with CDKL5 Deficiency Disorder: Results From the Marigold Study," by J. Hulihan, et al.
    • "Extended Duration Safety and Efficacy of Ganaxolone for the Treatment of CDKL5 Deficiency Disorder: Preliminary Open-Label Extension Analysis (Marigold Study)," by N. Specchio, et al.
    • "A Double-Blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy and Safety of Intravenous Ganaxolone in Status Epilepticus (RAISE)"
    • "Ganaxolone: Mechanism of Action and Pharmacology"

    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 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 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 our plans for the first commercial launch of ganaxolone; our clinical development plans for ganaxolone; and our expectations regarding the clinical development schedule and milestones for ganaxolone. Forward-looking statements in this 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; clinical trial results may not support 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; 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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  15. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to two (2) new employees. The Compensation Committee of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 134,000 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    These stock option grants have an exercise price of $13.71 per share, which is equal to the closing price of Marinus' common stock on November 9, 2020, the date of grant for the…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to two (2) new employees. The Compensation Committee of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 134,000 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    These stock option grants have an exercise price of $13.71 per share, which is equal to the closing price of Marinus' common stock on November 9, 2020, the date of grant for the stock options. All of the stock options will vest and become exercisable as to 25% of the underlying shares on the one-year anniversary of the employee's start date of employment, and will vest and become exercisable as to the remaining 75% of the underlying shares in 36 equal monthly installments at the end of each month following such anniversary, subject to the employee's continued employment with Marinus on such vesting dates. The stock options were granted as an inducement material to the employee entering into employment with Marinus in accordance with Nasdaq Listing Rule 5635(c)(4), and are subject to the terms and conditions of the applicable award agreement covering such grant.

    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 currently initiating 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 and the clinical development schedule and milestones. Forward-looking statements in this 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; clinical trial results may not support 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; 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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    • Reported positive topline data from pivotal Phase 3 Marigold Study of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD)
    • Awarded five-year cost-sharing contract with 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, for refractory status epilepticus (RSE)
    • Site selection and activation are on-track in RSE Phase 3 IV ganaxolone RAISE clinical trial; targeting vast majority of sites to be open by the end of Q1 2021
    • Enrollment is ongoing in Phase 2 open-label trial to evaluate the safety and tolerability of adjunctive ganaxolone treatment in patients with tuberous sclerosis…
    • Reported positive topline data from pivotal Phase 3 Marigold Study of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD)
    • Awarded five-year cost-sharing contract with 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, for refractory status epilepticus (RSE)
    • Site selection and activation are on-track in RSE Phase 3 IV ganaxolone RAISE clinical trial; targeting vast majority of sites to be open by the end of Q1 2021
    • Enrollment is ongoing in Phase 2 open-label trial to evaluate the safety and tolerability of adjunctive ganaxolone treatment in patients with tuberous sclerosis complex (TSC)
    • Top line data from PCDH19 related epilepsy (PCDH19-RE) Phase 2 Violet Study expected in Q2 2021 and data from Phase 2 TSC clinical trial expected in mid-2021

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided an update on its clinical development activities and reported its financial results for the third quarter ended September 30, 2020.

    "This was a milestone quarter, highlighted by the positive results from our Marigold Study, which demonstrated ganaxolone's potential as the first treatment specifically indicated in CDD patients, and the significant progress achieved in our status epilepticus program, including the execution of a cost-sharing contract with BARDA," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "The next few months will be equally exciting, including our continued work as we plan for our NDA submission (for CDD), site activation and education on our Phase 3 refractory status epilepticus program, and the opportunity to share further details of our Marigold Study results with the medical community at the American Epilepsy Society Annual Meeting in December."

    Dr. Braunstein added, "We continue to strengthen the organization to support our ongoing clinical trial programs, commercial planning, and portfolio development. Most recently, we brought on Henri Vaitkevicius, M.D., as Vice President of Clinical Development to complement our growing team of clinicians, and Christy Shafer as our Chief Commercial Officer, who brings a unique background of leading teams in both the hospital and rare neurological diseases settings. The entire company is working tirelessly to advance our many programs and realize ganaxolone's potential to help those suffering from rare seizure disorders."

    Pipeline Update:

    CDKL5 Deficiency Disorder (CDD)

    • Marinus reported positive topline data from the Phase 3 Marigold Study, the first double-blind placebo controlled trial to provide evidence of efficacy in CDD and the first Phase 3 trial to examine three times a day dosing of ganaxolone in pediatric patients.
    • Patients given oral ganaxolone showed a significant 32.2% median reduction in 28-day major motor seizure frequency, compared to a 4.0% reduction for those receiving the placebo, achieving the trial's primary endpoint (p=0.002). In this study, ganaxolone was generally well tolerated with a safety profile consistent with previous clinical studies, with the most frequent adverse event being somnolence.
    • In early December, Marinus expects to open an expanded access program (EAP) that will allow the company to offer ganaxolone to patients with CDD who were unable to participate in the Phase 3 trial.
    • Marinus is preparing to meet with the FDA in Q1 2021 in connection with its planned mid-2021 NDA submission.
    • The company is also planning for the European Medicines Agency (EMA) pre-marketing authorization application (MAA) meeting to occur in first half 2021 to support a MAA submission.
    • Marinus anticipates having a major presence at the American Epilepsy Society (AES) meeting in December, where the company intends to present additional details of the Marigold Study, including several subset analyses and pharmacokinetic data, which will be a critically important roadmap to further development of oral ganaxolone.

    Refractory Status Epilepticus (RSE)

    • Greater than 75 percent of sites have been selected for the Phase 3 clinical trial of IV ganaxolone (RAISE trial) for the treatment of RSE. Site identification and activation is focused on neurocritical care and neurointensivists, who are on the front lines of treating patients experiencing RSE.
    • Site selection expected to be completed by year end, in-line with expectations prior to COVID-19.
    • Marinus targets opening the vast majority of sites by end of Q1 2021 with topline data in 1H 2022.
    • The RAISE trial is a randomized, double-blind, placebo-controlled trial that will evaluate the efficacy and safety of IV ganaxolone in patients with RSE who have failed benzodiazepines and two or more second line intravenous antiepileptic drugs (AEDs). The trial will examine the proportion of participants with RSE who experience seizure cessation within 30 minutes of initiation of IV ganaxolone as well as the rate of progression to IV anesthesia within 36 hours. Cessation of RSE will be determined by both clinical and EEG findings.
    • Ganaxolone development for RSE is being 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.
    • Marinus has submitted a request for scientific advice to the EMA Committee for Medicinal Products for Human Use (CHMP) for feedback on the design of its second RSE trial to be conducted in Europe.
    • Several patients with super refractory status epilepticus (SRSE) have been treated under Emergency Investigational New Drug (eIND) with IV ganaxolone. Further details will be discussed during AES.
    • The company is also planning to investigate earlier intervention for treatment in SE in established status epilepticus (ESE). This future trial is expected to target convulsive patients who have been treated with and failed benzodiazepines. Further details to be discussed at the company's analyst meeting being held during AES.

    Tuberous Sclerosis Complex (TSC)

    • Marinus continues enrolling patients in its open label Phase 2 trial in TSC (the CALM Study).
    • The trial protocol is designed with a four-week baseline period, followed by a 12-week treatment period during which patients will receive up to 600mg of ganaxolone (oral liquid suspension) three times a day. Patients who meet eligibility criteria may continue ganaxolone treatment during a planned 24-week extension. The trial is expected to enroll approximately 30 patients ages 2 to 65.
    • The primary endpoint for the trial is percent change in median 28-day primary seizure frequency for the treatment period relative to baseline. The company plans to analyze allopregnanolone sulfate levels as part of the trial efficacy analysis.
    • Marinus expects to report topline data in the 1H of 2021.

    PCDH19 Related Epilepsy (PCDH19-RE)

    • Recruitment is complete in the ongoing Phase 2 Violet Study evaluating allopregnanolone sulfate as a biomarker and ganaxolone as a treatment in PCDH19-RE patients.
    • The company believes the trial has the potential to detect a meaningful signal as proof-of-concept for the allopregnanolone sulfate biomarker hypothesis as well as support the value proposition supporting a three-times-a-day dosing regimen.
    • Marinus remains on-track to report topline data from this proof-of-concept trial in early Q2, 2021.

    Corporate Update:

    • The company anticipates having a major presence at the AES Meeting in December, where it has submitted multiple abstracts, including the presentation of multiple data subset analyses from the Marigold Study.
    • Marinus is also planning to host a meeting with the investment community and analysts during AES on December 7, 12PM – 2PM Eastern Time.
    • Christy Shafer has joined as Chief Commercial Officer to lead U.S. commercial strategy for Marinus' oral and IV-ganaxolone franchise.
    • In September, the company was awarded a BARDA cost-sharing contract to develop IV ganaxolone for treatment of RSE.
    • In October, Marinus received The LouLou Foundation's Company Making a Difference Award – Clinical, in recognition of the first successful recruitment of a pivotal and international clinical trial and recent positive data from the Marigold Study. The LouLou Foundation is dedicated to helping those with CDD.

    Financial Update:

    At September 30, 2020, the company had cash, cash equivalents and investments of $91.3 million, compared to $91.7 million at December 31, 2019. Marinus believes its cash and cash equivalents as of September 30, 2020 will enable it to fund its operating expenses and capital expenditures into 2022.

    Federal contract revenue was $0.2 million for the three and nine months ended September 30, 2020, compared to $0 for the three and nine months ended September 30, 2019 as a result of the execution of the BARDA contract in September, 2020.

    Research and development expenses were $11.3 million and $38.1 million for the three and nine months ended September 30, 2020, respectively, as compared to $11.6 million and $30.5 million for the same periods in the prior year. The year to date increase was due primarily to Marinus' ongoing enrollment in its CDD Phase 3 Marigold Study, which top-line data were announced during Q3 2020, and increased drug development activity, including preclinical studies and manufacturing activities in preparation for a potential NDA submission for CDD and our recently initiated Phase 3 clinical trial in SE.

    General and administrative expenses were $4.6 million and $12.5 million for the three and nine months ended September 30, 2020, respectively, as compared to $2.3 million and $8.5 million for the same periods in the prior year, respectively. The increases were due primarily to increased legal and consulting fees as Marinus scales up operations and prepares for potential commercialization, and non-cash stock-based compensation.

    The company reported net losses of $15.7 million and $58.9 million for the three and nine months ended September 30, 2020, respectively, compared to $13.8 million and $38.7 million in the same period a year ago. Cash used in operating activities increased to $44.5 million for the nine months ended September 30, 2020 compared to $32.4 million for the same period a year ago.

    Readers are referred to, and encouraged to read in its entirety, the company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, to be filed with the Securities and Exchange Commission, which includes further detail on the above-referenced transactions and the company's business plans, operations, financial condition and results of operations.

    Marinus Pharmaceuticals, Inc.

    Selected Financial Data (in thousands, except share and per share amounts)

    (unaudited)

     
     

     

    September 30,

    2020

    December 31,

    2019

     

     

     

     

    ASSETS

     

     

    Cash and cash equivalents

    $87,337

     

    $90,943

    Investments

    3,966

     

    739

    Other assets

    8,223

     

    7,160

    Total assets

    $99,526

     

    $98,842

    LIABILITIES AND STOCKHOLDERS' EQUITY

     

     

     

    Current liabilities

    $9,439

     

    $8,031

    Other long term liabilities

    2,664

     

    3,042

    Total liabilities

    12,103

     

    11,073

    Total stockholders' equity

    87,423

     

    87,769

    Total liabilities and stockholders' equity

    $99,526

     

    $98,842

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Three Months Ended September 30,

     

    Nine Months Ended September 30,

     

     

    2020

     

    2019

     

    2020

     

    2019

     

     

     

     

     

     

     

     

     

     

     

     

     

    Federal contract revenue

     

    $

    171

     

     

    $

     

     

    $

    171

     

     

    $

     

    Expenses:

     

     

     

     

     

     

     

     

     

     

     

     

    Research and development

     

    $

    11,306

     

     

    $

    11,572

     

     

    $

    38,062

     

     

    $

    30,454

     

    General and administrative

     

     

    4,564

     

     

     

    2,327

     

     

     

    12,543

     

     

     

    8,496

     

    Loss from operations

     

     

    (15,699

    )

     

     

    (13,899

    )

     

     

    (50,434

    )

     

     

    (38,950

    )

    Interest income

     

     

    79

     

     

     

    93

     

     

     

    459

     

     

     

    280

     

    Other expense, net

     

     

    (39

    )

     

     

     

     

     

    (31

    )

     

     

    (42

    )

    Net loss

     

    $

    (15,659

    )

     

    $

    (13,806

    )

     

    $

    (50,006

    )

     

    $

    (38,712

    )

    Deemed dividends on convertible preferred stock

     

     

     

     

     

     

     

     

    (8,880

    )

     

     

     

    Net loss applicable to common shareholders

     

    $

    (15,659

    )

     

    $

    (13,806

    )

     

    $

    (58,886

    )

     

    $

    (38,712

    )

    Per share information:

     

     

     

     

     

     

     

     

     

     

     

     

    Net loss per share of common stock—basic and diluted

     

    $

    (0.51

    )

     

    $

    (1.05

    )

     

    $

    (2.29

    )

     

    $

    (2.95

    )

    Basic and diluted weighted average shares outstanding

     

     

    30,552,947

     

     

     

    13,135,885

     

     

     

    25,737,981

     

     

     

    13,127,653

     

    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 anti-seizure and anti-anxiety activity via its effects on both synaptic and extrasynaptic GABAA receptors. Ganaxolone has been studied in more than 1,600 patients, both pediatric and adult, 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 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 currently initiating 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; our goal to have the vast majority of sites for our Phase 3 IV ganaxolone RAISE clinical trial open by the end of the first quarter of 2021; our expectation to release top line PCDH19-RE in Q2 2021 and TSC data in mid-2021; our plans to meet with the FDA in Q1 2021 for our planned mid-2021 NDA submission for CDD; our plans for the EMA pre-marketing authorization application meeting to occur in the first half of 2021 to support a MAA submission for CDD; our expectation to complete site selection for the Raise trial by year end; our target to open the vast majority of sites for the Raise trial by end of Q1 with topline data in 1H 2022; the expected trial design of the Raise trial; our expectations to release topline data from our Phase 3 trial in status epilepticus in the first half of 2022; our expectations regarding the enrollment of our Phase 2 open-label trial for patients with TSC; our expectations to release topline data from our Phase 2 open-label trial for patients with TSC in the first half of 2021; our expectations regarding enrollment in our Phase 2 Violet Study; our expectations to release topline data from our Phase 2 Violet Study in the first half of 2021; our expectations that our cash, cash equivalents and investments will be sufficient to fund our operating expenses and capital expenditures into 2022; 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, 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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  16. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that Christy Shafer has been appointed Chief Commercial Officer, reporting to Scott Braunstein, M.D., Chief Executive Officer of Marinus.

    "As we continue to build our senior team with formidable leaders, we believe Christy is the right person to drive our commercial strategy, and to prepare the company for its potential first launch of ganaxolone in the area of CDKL5 deficiency disorder," said Scott Braunstein M.D., Chief Executive Officer of Marinus. "With her combined rare disease and hospital directed expertise, Christy will provide pivotal leadership in steering our…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that Christy Shafer has been appointed Chief Commercial Officer, reporting to Scott Braunstein, M.D., Chief Executive Officer of Marinus.

    "As we continue to build our senior team with formidable leaders, we believe Christy is the right person to drive our commercial strategy, and to prepare the company for its potential first launch of ganaxolone in the area of CDKL5 deficiency disorder," said Scott Braunstein M.D., Chief Executive Officer of Marinus. "With her combined rare disease and hospital directed expertise, Christy will provide pivotal leadership in steering our commercial strategy and execution."

    Ms. Shafer has spent almost 20 years in the biotech, pharma, and medical device spaces, with her most recent position being Business Director of Neurology at Alexion Pharmaceuticals. There, she led teams through transformational growth in first-in-class neurology product launches, which catalyzed significant growth for the organization.

    "Marinus' thoughtful focus on scientifically driven development for ganaxolone in key indications of patient need, along with strong positive results in its clinical trial in CDD, are truly exciting opportunities. This foundation, along with the pipeline of upcoming clinical trial readouts in PCDH19-related epilepsy, tuberous sclerosis complex, and status epilepticus, positions Marinus in the rare and orphan seizure disorder market," said Ms. Shafer. "I look forward to working closely with the rest of the Marinus team to maximize ganaxolone's commercial potential with the goal of improving the lives of patients and families who may benefit from this important drug candidate."

    Prior to her tenure at Alexion, Ms. Shafer held positions of increasing responsibility at Pacira BioSciences, where she was responsible for multimillion-dollar sales forecasts, operational budgets, and leading highly functioning regional business leaders and their teams. Ms. Shafer has also held the roles of Regional Sales Director for Sanofi Biosurgery and Regional Vice President at I-Flow Corporation. She earned her Post-Baccalaureate degree in Immunology and Pharmacology and her Bachelor of Life Science in Cell/Molecular Biology and Genetics from the University of Maryland, College Park.

    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 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 our plans for the first commercial launch of ganaxolone; our clinical development plans for ganaxolone; and our expectations regarding the clinical development schedule and milestones for ganaxolone. Forward-looking statements in this 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; clinical trial results may not support 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; 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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  17. Details the Incumbent Board's History of Presiding Over Dismal Governance Practices, Numerous Commercial and Strategic Missteps, and Hundreds of Millions of Dollars in Value Destruction

    Highlights That Chairman Scott Tarriff has Fostered an Anti-Shareholder Culture Defined by Dilutive Actions, Poor Investor Engagement, and a Misaligned Management Team

    Urges Shareholders to Vote on the WHITE Consent Card to Reconstitute the Board with WaterMill's Three Highly-Qualified and Independent Director Candidates

    Warns Shareholders to Avoid Being Misled by Mr. Tarriff and his Allies, Especially Given That Their Last Self-Directed Board Refresh was Followed by a ~40% Share Price Decline

    WaterMill Asset Management Corp. (together with its affiliates…

    Details the Incumbent Board's History of Presiding Over Dismal Governance Practices, Numerous Commercial and Strategic Missteps, and Hundreds of Millions of Dollars in Value Destruction

    Highlights That Chairman Scott Tarriff has Fostered an Anti-Shareholder Culture Defined by Dilutive Actions, Poor Investor Engagement, and a Misaligned Management Team

    Urges Shareholders to Vote on the WHITE Consent Card to Reconstitute the Board with WaterMill's Three Highly-Qualified and Independent Director Candidates

    Warns Shareholders to Avoid Being Misled by Mr. Tarriff and his Allies, Especially Given That Their Last Self-Directed Board Refresh was Followed by a ~40% Share Price Decline

    WaterMill Asset Management Corp. (together with its affiliates, "WaterMill" or "we"), which collectively with the other participants in its consent solicitation beneficially owns approximately 3.3% of the outstanding shares of Ziopharm Oncology, Inc. (NASDAQ:ZIOP) ("Ziopharm" or the "Company"), today issued the below letter to shareholders in support of the proposals included in its definitive consent statement filed with the U.S. Securities and Exchange Commission on October 30, 2020. Notably, WaterMill has put forth proposals to reconstitute Ziopharm's Board of Directors, including a proposal to remove four incumbent directors and a proposal to elect three highly-qualified and independent nominees: Robert Postma, Jaime Vieser, and Holger Weis.

    We are asking shareholders to consent to all of our proposals by voting on the WHITE consent card. We urge shareholders to sign, date, and return their WHITE consent card today. Please return each and every WHITE consent card received. Do not return any green revocation card (even as a protest vote).

    Please note that important information pertaining to WaterMill's campaign, including the below letter, is available at www.FixZiopharm.com.

    ***

    November 5, 2020

    Fellow Shareholders:

    WaterMill Asset Management Corp. (together with its affiliates, "WaterMill" or "we") invested in Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") because we believe in the innovation and science behind the Company's immuno-oncology platform. Like many of you, we have spent years investing in the biotechnology sector and understand that considerable patience is required on the road to value creation. This is why we have never before engaged in an "activist campaign" and strive to maintain cordial, productive relationships with corporate leadership teams. Unfortunately, we have been forced to deviate from our long-preferred posture due to the extraordinarily anti-shareholder actions and value-destructive reign of Ziopharm's Board of Directors (the "Board").

    After assessing the various ways in which we could potentially catalyze change at Ziopharm, we determined that it was in the best interest of all shareholders for WaterMill to launch a consent solicitation to reconstitute the Board in a pragmatic and targeted manner. This is why we are soliciting shareholder support to remove four incumbent directors – Scott Braunstein, J. Kevin Buchi, Elan Z. Ezickson, and Scott Tarriff – and elect three highly-qualified and independent individuals – Robert Postma, Jaime Vieser, and Holger Weis – who we believe possess the type of business acumen and strategic vision that has been lacking in the boardroom for far too long.

    In our view, a seven-member Board that includes four of the incumbents and all three of our director candidates will have the ideal mix of institutional knowledge, industry expertise, commercial intensity, and meaningful ownership perspectives. We believe a Board that is truly focused on running Ziopharm like a high-potential business can finally establish value-enhancing partnerships, line up appropriate financing sources, incentivize the right management team, and reverse the Company's five-year tailspin. These are the types of outcomes that will benefit all of Ziopharm's stakeholders, ranging from shareholders and employees to patients and caregivers.

    THE CASE FOR MEANINGFUL, SHAREHOLDER-DRIVEN CHANGE ATOP ZIOPHARM IS CRYSTAL CLEAR

    Since Chairman Scott Tarriff and Chief Executive Officer Laurence Cooper joined Ziopharm in 2015, we contend that shareholders have been systemically disregarded and deprioritized. This is evidenced by the Company's negative total shareholder returns ("TSR") and staggering underperformance over several time horizons:

     

    1-Year TSR*

    3-Year TSR*

    5-Year TSR*

    Ziopharm

    -39.31%

    -52.43%

    -76.28%

    S&P 500

    18.50%

    44.63%

    90.33%

    Russell 3000

    19.18%

    43.19%

    87.84%

    SPDR S&P Biotech ETF

    52.53%

    39.17%

    83.19%

    *Source: Bloomberg (TSR figures reflect share price and performance up until October 15, 2020, which is the day before WaterMill filed its preliminary consent statement).

    We believe it is critical for shareholders to see through Ziopharm's brazenly misrepresentative investor communications and realize that value has eroded at an even faster pace following the Company's self-directed Board refreshment in 2018. In fact, Ziopharm's share price is down nearly 40% over the past twelve months alone.1 Mr. Tarriff and Dr. Cooper apparently chose to ignore this fact in their November 2nd letter that claims "Ziopharm continues to drive value with fresh Board perspectives."2 We question how shareholders can have any faith in Ziopharm's current leadership when it is so willing to disseminate consultant-manufactured spin and disregard shareholder suffering.

    Although the incumbent Board has signaled its intent to distort and ignore reality, we feel shareholders deserve to be reacquainted with data and facts that underscore the need for expedited boardroom change:

    • The incumbent Board has repeatedly shown that it is unwilling to accept and address shareholder feedback.
      • A majority of voting shareholders withheld support for three directors – Mr. Braunstein, Mr. Ezickson, and Douglas Pagán – at this year's annual meeting. Rather than engage with shareholders to finally commence a credible Board overhaul, Ziopharm allowed two of the aforementioned directors to retain their positions and replaced Mr. Pagán (Chief Financial Officer of Dicerna Pharmaceuticals, Inc.) with Mr. Buchi (Chairman of Dicerna Pharmaceuticals, Inc.). We question how it improves the Board's damaged credibility and serves shareholders' interests to replace Mr. Pagán – who clearly lost investors' confidence – with one of his current bosses.
      • Long-term shareholders have suffered through sustained losses in recent years. In an apparent attempt to add insult to injury, Ziopharm asked shareholders to increase the Company's authorized share count by 195 million shares at this year's annual meeting. This dilutive, poorly-conceived proposal was voted down.
      • Instead of assuming accountability for the hundreds of millions of dollars destroyed during their tenures, Mr. Tarriff and Dr. Cooper boasted about purported accomplishments and hurled unsubstantiated smears at WaterMill in its November 2nd letter. We encourage shareholders to examine their insulting communication, which we believe reads like it was ghost written by individuals with no pulse on Ziopharm's attentive and engaged investor base.
    • The incumbent Board lacks alignment with shareholders and important ownership perspectives.
      • According to Ziopharm's consent revocation statement, the Board paid out $1.84 million in total compensation to seven directors in the year ended on December 31, 2019. It underscores just how out of touch Ziopharm's Board is that directors received an average compensation of more than $260,000. In our view, this represents egregious compensation for a small market capitalization company that has been going in the wrong direction for years.
      • WaterMill's three-member slate, which collectively owns more than 3.3% of the Company's outstanding shares, has a significantly larger ownership position than all eight of the incumbent directors combined. We question how shareholders can trust the Board to urgently correct years of underperformance when it is so misaligned and maintains such meager shareholdings.
    • The incumbent Board is plagued by what appears to be a troubling web of performance issues and interlocking relationships among directors.
      • Among Ziopharm's incumbent directors, we have uncovered numerous instances of possible conflicts and overlap in Board service at other companies, including:
        • Mr. Ezickson serves on the Board of Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), where fellow Ziopharm director Dr. Braunstein also serves as Chief Executive Officer and President.
        • Since 2007, Mr. Tarriff has served as a director and the Chief Executive Officer at Eagle Pharmaceuticals, Inc. ("Eagle") (NASDAQ:EGRX). The brother of Mr. Tarriff's fellow Ziopharm director, Scott Braunstein, served on Eagle's Board of Directors from July 2016 to November 2019.
        • Since February 2012, Mr. Tarriff has also been a director on the Board of Synthetic Biologics, Inc. (NYSE:SYN), where shareholders have seen Synthetic Biologic's stock drop from over $70 to less than $1. Ziopharm did not disclose this mark on Mr. Tarriff's record in its November 2nd letter to shareholders.
        • As noted, Mr. Pagán's replacement on the Board – Mr. Buchi – is the Chairman of Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA), where Mr. Pagán also serves as Chief Financial Officer.
    • The incumbent Board has rewarded management with excessive and unjustifiable compensation as shareholders have endured unrelenting pain.
      • Ziopharm's consent revocation statement notes that all of the Company's eligible named executive officers received base compensation raises from 2018 to 2019. Dr. Cooper was rewarded with a 14.6% raise in his base compensation. All of this occurred despite the fact that the Company's share price declined by more than 50% over the course of the year ended December 31, 2018.
      • In January 2019, the Board awarded restricted shares and options with a total equity value of more than $3 million to Dr. Cooper and three other executives. Once again, this was provided after the Company's share price declined by more than 50% in 2018.
      • For performance in the year 2019, the Board determined that each member of the executive team met 100% of his individual objectives and paid out more than $1.5 million in bonuses. Dr. Cooper was showered with a more than $1 million bonus despite the fact that Ziopharm's share price has dramatically declined over his tenure. We believe this excessively generous and misaligned compensation system reflects the low-bar objectives set by the Board and is symptomatic of the disease of poor boardroom stewardship.
    • The incumbent Board has championed dilutive actions and tolerated dismal business execution.
      • Since 2017, the Board has overseen multiple public offerings and private placements that have diluted existing shareholders by more than 50%. These share issuances were at prices significantly below the market and led to only $255 million raised.
      • Ziopharm has failed to accelerate the monetization of its attractive immuno-oncology assets, going on for years without a partnership that further validates the Company's therapies and its commercial prospects. We feel the evidence of this failure is embedded within the Company's beaten down shares – and the Board's attempts to point to qualitative accomplishments does not change reality.
      • The Board and management have rewarded themselves with lofty compensation but have not invested in the type of transformative business development and commercialization talent that Ziopharm needs to completely or partially monetize assets.
    • The incumbent Board, under Mr. Tarriff's leadership, has amassed an industry-worst governance profile.
      • One of the leading independent proxy advisory firms, Institutional Shareholder Services, Inc. ("ISS"), shares our view that the Board fosters anti-shareholder practices, as evidenced by its issuance of a score "9" (on a scale of 1-10, with "10" representing the highest governance risk) in its QualityScore Governance Profile Report. It is very unfortunate that this ISS assessment did not serve as a wakeup call to Mr. Tarriff, Dr. Cooper, and other incumbent directors that we believe are focused on their self-interests.
      • ISS recommended shareholders vote "WITHHOLD" with respect to the election of Mr. Braunstein to the Board at the 2020 Annual Meeting due to his service on more than three public company boards while also serving as Chief Executive Officer of a separate public company, noting that "[a] CEO cannot reasonably be expected to balance the responsibilities of serving on more than three public boards while also fulfilling full-time executive duties."
      • In 2019, Mr. Ezickson attended less than 75% of Board and committee meetings. How can directors address investor concerns and effectively evaluate management with 75% participation? Effective boards of directors have strong, robust engagement from all members.

    CHANGE ATOP ZIOPHARM IS NEEDED NOW – SHAREHOLDERS CANNOT AFFORD TO CONTINUE ROLLING THE DICE ON MR. TARRIFF AND THE INCUMBENTS

    Ziopharm's shares are currently trading near a ten-year low because leadership has failed to provide a credible path to unlocking value. To the contrary, it seems to us that Mr. Tarriff and Dr. Cooper only have a roadmap for enriching insiders at the expense of shareholders. We fear that another year under the incumbent Board's leadership may lead Ziopharm toward a de-listing and joining the long list of biotechnology firms that have squandered their potential on route to becoming a penny stock. This is why we have taken the extraordinary action of running a consent solicitation and are seeking to facilitate targeted boardroom changes as quickly as possible.

    Now that WaterMill has exposed Ziopharm's contempt for shareholders and prioritization of insiders, we expect the Company and its growing army of high-priced external consultants – who are paid for with our capital – to continue attacking our slate and disseminating misrepresentations. We urge shareholders to see through the incumbent Board's empty spin and reject any attempts to pass off cosmetic changes as substantive improvements. In our view, any reactionary moves announced by Ziopharm in the weeks to come will represent nothing more than self-preservation tactics and entrenchment maneuvers.

    There is no need for shareholders to continue to roll the dice on Mr. Tarriff and the other three underperforming incumbent directors we are targeting. It is clear to us – and hopefully now other shareholders – that these individuals appear content to run Ziopharm like a rudderless non-profit organization. By removing these individuals and installing our slate of proven business and financial experts, we believe shareholders will finally have a Board that is capable of running the Company like a publicly-traded entity that is supposed to produce value for its shareholders.

    OUR THREE-MEMBER SLATE OF DIRECTOR CANDIDATES IS ALIGNED, EXPERIENCED AND FULLY FOCUSED ON UNLOCKING VALUE

    Although Ziopharm boasts it has "directors with diverse skills and experiences relevant to our industry and operations," recent evidence suggests its Board is most skilled and experienced in presiding over rapid and significant value destruction. We also find it odd that Mr. Tarriff and Dr. Cooper are trying to score points with shareholders by saying they are "executing on the Company's long-term strategy," particularly in light of the fact that this so-called "strategy" has been a recipe for years of losses. Fortunately, our slate will not allow Ziopharm to continue bleeding cash, overpaying underperforming leadership, and wagering all of its remaining equity value on three partnerships that are yet to yield even a hint of value for shareholders.

    We believe a properly reconstituted Board that includes our three director candidates will be able to oversee the implementation of a superior corporate strategy – one that leads to enhanced governance, improved financing decisions that serve shareholders' best interests, and value-enhancing business development opportunities. Our slate's business and financial acumen, commercial vision, and ownership perspectives are the ideal complement to the four Board members that we are not seeking to remove. Our slate includes:

    Director Candidate

    Relevant Experience

    Notable Qualifications and Skills

    Robert Postma

    • Principal and founder of WaterMill Asset Management Corp.
    • Four decades of experience investing across equity and fixed income markets
    • Frequently analyzes, invests in, and engages with healthcare and biotechnology companies
    • Sizable and long-term shareholder of Ziopharm
    • Extensive knowledge of Ziopharm's assets, governance, and financials
    • Strong capital allocation acumen
    • Valuable relationships with prospective industry and financial partners

    Jaime Vieser

    • Manager of Brushwood LLC, a private investment firm
    • Previously Co-Founder and Chief Investment Officer of Castle Hill Asset Management LLC, a multi-billion dollar asset manager
    • Former banker at Deutsche Bank AG and Bankers Trust Company
    • Sizable and long-term shareholder of Ziopharm
    • Deep understanding of Ziopharm's assets, governance, and financials
    • Strong and useful relationships with banks and prospective sources of capital
    • MBA

    Holger Weis

    • Two decades of various c-level business and strategy roles at life science companies
    • Currently Chief Financial Officer of PhenoTarget Biosciences, Inc.
    • Co-author of several scientific papers and presentations
    • Former Ernst & Young executive
    • Deep knowledge of the biotech industry and operational, financial, and strategic planning practices
    • Valuable expertise in financial management, planning, audits, and accounting
    • Extensive knowledge of Ziopharm's assets, governance, and financials
    • Certified Public Accountant

    In the weeks to come, we will release additional information pertaining to our slate and its strategic vision. We are committed to communicating with shareholders in a frequent and transparent manner in order to demonstrate how our slate will act and think upon entering the boardroom.

    Please visit www.FixZiopharm.com and sign up for e-mail alerts in order to receive our updates.

    Thank you for your support.

    Robert Postma

    Principal and Founder

    WaterMill Asset Management Corp.

    ***

    We urge Ziopharm shareholders to consent to all five proposals on the WHITE consent card today and return it in your postage-paid envelope provided. December 11, 2020 is our goal for the submission of written consents. Effectively, this means that you have until December 11, 2020 to consent to the proposals.

    Should you have any questions or need assistance with voting, please contact Saratoga Proxy Consulting LLC at (888) 368-0379 or (212) 257-1311 or by email at .

    PROTECT YOUR INVESTMENT. SIGN, DATE AND RETURN YOUR FILLED OUT WHITE CONSENT CARD TODAY.

    ____________________________

    1 Ziopharm's share price was $4.35 as of market close on October 15, 2019 and the Company's share price was $2.64 as of market close on October 15, 2020 (the day prior to WaterMill filing a preliminary consent statement).

    2 Press release entitled "Ziopharm Files Definitive Consent Revocation Statement and Sends Letter to Shareholders" issued on November 2, 2020.

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  18. 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 release financial results for the third quarter ended September 30, 2020 after the market closes on November 9, 2020. The company will host a conference call at 4:30pm Eastern Time on November 9, 2020 to provide a business update and discuss the financial results.

    Monday, November 9th @ 4:30pm Eastern Time

    Domestic:

     

     

    833-979-2765

    International:

     

     

    343-761-2590

    Webcast:

     

     

    https://event.on24.com/wcc/r/2782252/98CE860F96E5E61156B02BB7CC5A934B

    Conference ID:

     

     

    3686467

    About Marinus Pharmaceuticals

    Marinus…

    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 release financial results for the third quarter ended September 30, 2020 after the market closes on November 9, 2020. The company will host a conference call at 4:30pm Eastern Time on November 9, 2020 to provide a business update and discuss the financial results.

    Monday, November 9th @ 4:30pm Eastern Time

    Domestic:

     

     

    833-979-2765

    International:

     

     

    343-761-2590

    Webcast:

     

     

    https://event.on24.com/wcc/r/2782252/98CE860F96E5E61156B02BB7CC5A934B

    Conference ID:

     

     

    3686467

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

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  19. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that company management will participate in a Truist Virtual Fireside Chat, the Guggenheim Virtual Conference: 2nd Annual Neuro/Immunology Day and the Jefferies Global Healthcare Conference over the next few weeks.

    Additional details can be found below:

    Truist Virtual Fireside Chat
    Friday, October 23, 2020
    Time: 12:30 PM Eastern Time
    Fireside chat with Joon Lee, M.D., Ph.D., Equity Analyst and Dr. Scott Braunstein, Chief Executive Officer, Ed Smith, Chief Financial Officer, and Dr. Joseph Hulihan, Chief Medical Officer
    For registration and webcast details, see: https://www.netroadshow.com/events/login?show=6debe4f7&confId=25029

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that company management will participate in a Truist Virtual Fireside Chat, the Guggenheim Virtual Conference: 2nd Annual Neuro/Immunology Day and the Jefferies Global Healthcare Conference over the next few weeks.

    Additional details can be found below:

    Truist Virtual Fireside Chat

    Friday, October 23, 2020

    Time: 12:30 PM Eastern Time

    Fireside chat with Joon Lee, M.D., Ph.D., Equity Analyst and Dr. Scott Braunstein, Chief Executive Officer, Ed Smith, Chief Financial Officer, and Dr. Joseph Hulihan, Chief Medical Officer

    For registration and webcast details, see: https://www.netroadshow.com/events/login?show=6debe4f7&confId=25029

    Guggenheim Healthcare Talks - Idea Forum | 2nd Annual Neuro/Immunology Day

    Monday, November 16, 2020

    One-on-one meetings with Marinus executives

    Jefferies London Healthcare Conference

    Wednesday, November 18, 2020

    Fireside chat with Andrew Tsai, Equity Analyst and Dr. Scott Braunstein, Chief Executive Officer, Ed Smith, Chief Financial Officer, and Dr. Joseph Hulihan, Chief Medical Officer

    One-on-one meetings with Marinus executives; for registration and webcast details, see: https://wsw.com/webcast/jeff141/mrns/1869600

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

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  20. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to 11 new employees. The Compensation Committee of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 144,050 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    Of these stock option grants, 138,250 have an exercise price of $14.53 per share, which is equal to the closing price of Marinus' common stock on October 9, 2020, the date of grant for…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to 11 new employees. The Compensation Committee of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 144,050 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    Of these stock option grants, 138,250 have an exercise price of $14.53 per share, which is equal to the closing price of Marinus' common stock on October 9, 2020, the date of grant for the stock options. The remaining 5,800 stock options have an exercise price of $14.86 per share, which is equal to the closing price of Marinus' common stock on October 12, 2020 corresponding with the applicable employees' start date. All of the stock options will vest and become exercisable as to 25% of the underlying shares on the one-year anniversary of the employee's start date of employment, and will vest and become exercisable as to the remaining 75% of the underlying shares in 36 equal monthly installments at the end of each month following such anniversary, subject to the employee's continued employment with Marinus on such vesting dates. The stock options were granted as an inducement material to the employee entering into employment with Marinus in accordance with Nasdaq Listing Rule 5635(c)(4), and are subject to the terms and conditions of the applicable award agreement covering such grant.

    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 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 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 our clinical development plans for ganaxolone and the clinical development schedule and milestones. Forward-looking statements in this 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; clinical trial results may not support 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; 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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    • 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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  21. - 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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  22. 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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  23. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to seven new employees. The independent members of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 453,500 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    Of these stock option grants, 293,500 have an exercise price of $1.77 per share, which is equal to the closing price of Marinus' common stock on August 5, 2020, the date of grant for…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to seven new employees. The independent members of the Board of Directors of Marinus approved the grant of non-qualified stock options to purchase an aggregate of 453,500 shares of its common stock as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    Of these stock option grants, 293,500 have an exercise price of $1.77 per share, which is equal to the closing price of Marinus' common stock on August 5, 2020, the date of grant for the stock options. The remaining 165,000 stock options will be priced on the applicable employee's start date. All of the stock options will vest and become exercisable as to 25% of the underlying shares on the one-year anniversary of the employee's start date of employment, and will vest and become exercisable as to the remaining 75% of the underlying shares in 36 equal monthly installments at the end of each month following such anniversary, subject to the employee's continued employment with Marinus on such vesting dates. The stock options were granted as an inducement material to the employee entering into employment with Marinus in accordance with Nasdaq Listing Rule 5635(c)(4), and are subject to the terms and conditions of the applicable award agreement covering such grant.

    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 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 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 our clinical development plans for ganaxolone and the clinical development schedule and milestones. Forward-looking statements in this 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; clinical trial results may not support 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; 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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    • Clinical trials remain on track, with Refractory Status Epilepticus (SE) trial expected to begin enrollment shortly
    • CDKL5 deficiency disorder (CDD) pivotal topline results on track for Q3; Conference call to be scheduled to discuss CDD data release and business update
    • Organization further strengthened with new additions to both the Board of Directors and management team

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided an update on its clinical development activities and reported its financial results for the second quarter ended June 30, 2020.

    "We are very pleased to report that the company's clinical programs…

    • Clinical trials remain on track, with Refractory Status Epilepticus (SE) trial expected to begin enrollment shortly
    • CDKL5 deficiency disorder (CDD) pivotal topline results on track for Q3; Conference call to be scheduled to discuss CDD data release and business update
    • Organization further strengthened with new additions to both the Board of Directors and management team

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided an update on its clinical development activities and reported its financial results for the second quarter ended June 30, 2020.

    "We are very pleased to report that the company's clinical programs continue to progress in line with the current timelines most recently discussed at our June 30 R&D Day, with limited disruptions attributable to COVID-19," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "We continue to expect topline data from our pivotal Phase 3 clinical trial in CDKL5 deficiency disorder in the third quarter, and in anticipation of this, we are planning for both our first NDA submission and the potential commercial launch of ganaxolone. Furthermore, we recently reported that the FDA granted Rare Pediatric Disease Designation for ganaxolone in CDD, an important milestone for both Marinus and patients suffering from this devastating condition."

    Dr. Braunstein added, "In addition to progressing our clinical trials, we are also strengthening the organization and preparing Marinus for the next phase of growth. The company continues to execute on the multiple initiatives and clinical trials we believe will drive long term value for shareholders, including efforts in developing next generation formulations. To bolster this strategy, we were pleased to appoint Chuck Austin to our Board of Directors, as well as adding new leadership in our research, clinical development, legal and commercialization teams to ensure we have strong and experienced talent around the table to guide the execution of Marinus' mission."

    Pipeline Update:

    Status Epilepticus (SE)

    • Marinus is on track to start enrolling the Phase 3 study in Q3, with 50 of the targeted 100 U.S. sites already pre-selected.
    • The Phase 3 study is a randomized, double-blind, placebo-controlled trial in SE patients who have failed benzodiazepines and 2 or more second line intravenous anti-epileptic drugs (AEDs). 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% power to detect a 30% efficacy difference between ganaxolone and placebo.
    • Patients will receive a 36-hour infusion followed by a 12-hour taper for a total 48-hour treatment period, which, for patients randomized to receive ganaxolone, targets a plasma concentration of greater or equal to 500ng/mL for 12-hours (same target concentration, 50% longer duration as the target dose evaluated in Phase 2).
    • The co-primary endpoints for the trial are (i) proportion of patients with SE cessation within 30 minutes of treatment initiation without medications for the acute treatment of SE and (ii) proportion of patients with no progression to IV anesthesia for 36 hours following treatment initiation.
    • Topline data is expected in the first half of 2022.

    CDKL5 Deficiency Disorder (CDD)

    • Marinus remains on-track to report topline data from the pivotal Phase 3 Marigold Study in Q3, evaluating the use of oral ganaxolone in children and young adults with CDD. The global, double‑blind, placebo‑controlled, clinical trial enrolled 101 patients between the ages of 2 and 21 with a confirmed disease‑related CDKL5 gene variant.
    • On July 30, the company announced the FDA has granted Rare Pediatric Disease Designation for ganaxolone for the treatment of CDD.
    • In advance of topline data, preparations have begun for an expanded access program (EAP) in CDD that would allow the company, on positive data, to offer ganaxolone to patients who were unable to participate in the Phase 3 trial.
    • The company continues to enhance its plans for a potential NDA submission and the development of its commercial strategy.

    Tuberous Sclerosis Complex (TSC)

    • The first patient has been enrolled in the Phase 2 open‑label trial to evaluate the safety and tolerability of adjunctive ganaxolone treatment in patients with TSC. The trial is expected to enroll approximately 30 patients ages 2 to 65.
    • The protocol is designed for patients to undergo a four-week baseline period, followed by a 12-week treatment period where they will receive up to 600 mg of ganaxolone (oral liquid suspension) three times a day. Patients who meet eligibility criteria may continue ganaxolone treatment during a 24-week extension planned for the trial.
    • The primary endpoint for the trial is percent change in 28-day primary seizure frequency for the treatment period relative to baseline. The company plans to analyze allopregnanolone sulfate levels as part of the trial efficacy analysis.
    • The company expects to report topline data in the first half of 2021, subject to any delays that may be caused by COVID-19.

    PCDH19 Related Epilepsy (PCDH19-RE)

    • Marinus remains on-track to enroll approximately 25 patients in the ongoing Phase 2 Violet Study evaluating allopregnanolone sulfate as a biomarker and ganaxolone as a treatment in PCDH19-RE patients.
    • The company believes the increased patient enrollment has the potential to strengthen the ability to detect a meaningful signal as proof-of-concept for the allopregnanolone sulfate biomarker hypothesis.
    • On-track to report topline data from this proof-of-concept trial in the first half of 2021.
    • Marinus will continue to evaluate if the allopregnanolone sulfate biomarker hypothesis could have broader utility in other targeted indications.

    Corporate Update:

    • Marinus raised $46 million in gross proceeds from an underwritten public offering of its common stock.
    • Appointed Martha Manning, Esq., who has over 20 years of life sciences experience, as Vice President, General Counsel and Secretary.
    • Appointed Charles Austin, a recognized leader in the pharmaceutical and medical device industries, to its Board of Directors.
    • Hosted aclinical update and commercial overview on its pipeline programs in Status Epilepticus (SE), CDKL5 Deficiency Disorder (CDD) and Tuberous Sclerosis Complex (TSC), with four key opinion leaders participating in the event.

    CDD Phase 3 Topline Clinical Data Conference Call:

    • As mentioned previously, Marinus plans to host a conference call later in the third quarter coincident with the release of topline data from its Phase 3 CDD clinical trial.

    Financial Update:

    At June 30, 2020, the company had cash, cash equivalents and investments of $105.9 million, compared to $91.7 million at December 31, 2019. We believe that our cash and cash equivalents as of June 30, 2020 will enable us to fund our current scale of operating expenses and capital expenditures into 2022.

    Research and development expenses increased to $11.8 million and $26.8 million for the three and six months ended June 30, 2020, respectively, as compared to $10.0 million and $18.9 million for the same periods in the prior year. The increases were due primarily to our ongoing enrollment in our CDD trial, which completed enrollment during the first quarter of 2020, and enhanced drug development activity, including preclinical studies and manufacturing activities in preparation for a potential NDA filing for CDD and a Phase 3 clinical trial in SE expected to commence in the third quarter of 2020.

    General and administrative expenses were $4.1 million and $8.0 million for the three and six months ended June 30, 2020 as compared to $2.5 million and $6.2 million for the same periods in the prior year. The increases were due primarily to increased legal and consulting fees as we scale up our operations and prepare for potential commercialization, and noncash stock-based compensation.

    The company reported net losses of $15.7 million and $34.3 million for the three and six months ended June 30, 2020, respectively, compared to $12.4 million and $24.9 million in the same period a year ago. Cash used in operating activities increased to $30.0 million for the six months ended June 30, 2020 compared to $20.4 million for the same period a year ago.

    Readers are referred to, and encouraged to read in its entirety, the company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, to be filed with the Securities and Exchange Commission, which includes further detail on the above-referenced transactions and the company's business plans, operations, financial condition and results of operations.

    Marinus Pharmaceuticals, Inc.

    Selected Financial Data (in thousands, except share and per share amounts)

    (unaudited)

     

     

     

     

    June 30,

    2020

    December 31,

    2019

     

     

    ASSETS

     

     

    Cash and cash equivalents

    $

    98,930

    $

    90,943

    Investments

     

    6,966

     

    739

    Other assets

     

    7,547

     

    7,160

    Total assets

    $

    113,443

    $

    98,842

    LIABILITIES AND STOCKHOLDERS' EQUITY

     

     

    Current liabilities

    $

    9,503

    $

    8,031

    Other long term liabilities

     

    2,795

     

    3,042

    Total liabilities

     

    12,298

     

    11,073

    Total stockholders' equity

     

    101,145

     

    87,769

    Total liabilities and stockholders' equity

    $

    113,443

    $

    98,842

    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 anti-seizure and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors. Ganaxolone has been studied in more than 1,600 subjects, both pediatric and adult, at therapeutically relevant dose levels and treatment regimens for up to four years.

     

    Three Months Ended

    June 30,

     

    Six Months Ended

    June 30,

     

     

    2020

     

    2019

     

    2020

     

    2019

     

     

     

     

     

     

     

     

     

    Expenses:

     

     

     

     

     

     

     

     

    Research and development

     

    $

    11,752

     

     

    $

    10,010

     

     

    $

    26,756

     

     

    $

    18,882

     

    General and administrative

     

     

    4,130

     

     

     

    2,502

     

     

     

    7,980

     

     

     

    6,169

     

    Loss from operations

     

     

    (15,882

    )

     

     

    (12,512

    )

     

     

    (34,736

    )

     

     

    (25,051

    )

    Interest income

     

     

    212

     

     

     

    90

     

     

     

    398

     

     

     

    186

     

    Other expense, net

     

     

    (5

    )

     

     

    (1

    )

     

     

    (9

    )

     

     

    (41

    )

    Net loss

     

    $

    (15,675

    )

     

    $

    (12,423

    )

     

    $

    (34,347

    )

     

    $

    (24,906

    )

    Deemed dividends on convertible preferred stock

     

     

     

     

     

     

     

     

    (8,880

    )

     

     

     

    Net loss applicable to common shareholders

     

    $

    (15,675

    )

     

    $

    (12,423

    )

     

    $

    (43,227

    )

     

    $

    (24,906

    )

    Per share information:

     

     

     

     

     

     

     

     

    Net loss per share of common stock—basic and diluted

     

    $

    (0.16

    )

     

    $

    (0.24

    )

     

    $

    (0.46

    )

     

    $

    (0.47

    )

    Basic and diluted weighted average shares outstanding

     

     

    99,770,498

     

     

     

    52,522,225

     

     

     

    93,180,795

     

     

     

    52,493,874

     

    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 our clinical development plans for ganaxolone; our expectations to release topline data from the pivotal Phase 3 Marigold Study in the third quarter of 2020; our expectations to initiate a Phase 3 trial in status epilepticus in the third quarter of 2020; our expectations to release topline data from our Phase 3 trial in status epilepticus in the first half of 2022; our expectations regarding the enrollment of our Phase 2 open-label trial for patients with TSC; our expectations to release topline data from our Phase 2 open-label trial for patients with TSC in the first half of 2021; our expectations regarding enrollment in our Phase 2 Violet Study; our expectations to release topline data from our Phase 2 Violet Study in the first half of 2021; our expectations that our cash, cash equivalents and investments will be sufficient to fund our operations into 2022; 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, 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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  24. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company's product candidate ganaxolone for the treatment of CDKL5 deficiency disorder (CDD), a rare refractory form of pediatric epilepsy.

    "We are pleased that ganaxolone has received Rare Pediatric Disease Designation from the FDA and are enthusiastic that the timing of this news aligns with our continued expectation to report top line data this quarter from our pivotal Phase 3 Marigold Study evaluating oral ganaxolone in patients with CDD," said Scott Braunstein…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company's product candidate ganaxolone for the treatment of CDKL5 deficiency disorder (CDD), a rare refractory form of pediatric epilepsy.

    "We are pleased that ganaxolone has received Rare Pediatric Disease Designation from the FDA and are enthusiastic that the timing of this news aligns with our continued expectation to report top line data this quarter from our pivotal Phase 3 Marigold Study evaluating oral ganaxolone in patients with CDD," said Scott Braunstein, M.D., Chief Executive Officer at Marinus. "This designation for ganaxolone for CDD underscores the significant unmet medical need for children and young adults with this serious and rare genetic disease, which causes early-onset, difficult-to-control seizures and severe neuro-developmental impairment. Our goal is to advance a pipeline of programs that can treat rare seizure disorders, including CDD, tuberous sclerosis complex and PCDH19-related epilepsy, and bring much-needed medicines to patients as soon as possible."

    FDA grants RPD Designation for diseases that affect fewer than 200,000 people in the U.S. in which the serious or life-threatening manifestations are primarily in individuals 18 years of age and younger. If a new drug application (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. The program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

    "The data that were used to support the Rare Pediatric Disease Designation comprised an analysis of existing literature as well as unpublished data specific to the CDD population, documenting that the serious or life-threatening manifestations of CDD primarily affect children," said Joseph Hulihan, M.D., Chief Medical Officer at Marinus. "We plan to publish these new data that will describe important clinical and health economic outcomes related to how seizure burden in children with CDD may affect future developmental milestones."

    The current program for RPD Designation will expire after September 30, 2020 unless Congress renews the program; however, a drug designated for a rare pediatric disease on that date can still receive a voucher if the drug is submitted and approved by the September 30, 2022 deadline.

    In advance of topline data from that study, Marinus has begun preparations for an Expanded Access Program (EAP) in CDD that will allow the company, on positive data, to offer ganaxolone to patients who were unable to participate in the Phase 3 study. The global, double blind, placebo-controlled Phase 3 Marigold Study evaluating the use of oral ganaxolone in children and young adults has enrolled 101 patients between the ages of 2 and 21 with a confirmed disease related CDKL5 gene variant.

    About 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. It predominantly affects females and is characterized by early‑onset, difficult‑to‑control seizures and severe neuro‑developmental impairment. The CDKL5 gene encodes proteins essential for normal brain function. Most children affected by CDD cannot walk normally, talk, or care for themselves. Many also suffer from scoliosis, visual impairment, gastrointestinal difficulties and sleep disorders. There are no treatments approved specifically for CDD. Genetic testing is available to determine if a patient has a mutation in the CDKL5 gene. To our knowledge, no previous late-stage clinical trials have been conducted in this patient population.

    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 anti-seizure and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors. Ganaxolone has been studied in more than 1,600 subjects, both pediatric and adult, 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 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 our clinical development plans for ganaxolone; our expectations to release top line data from the pivotal Phase 3 Marigold Study in the third quarter of 2020; our expectations to initiate a Phase 3 trial in status epilepticus; 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, 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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  25. Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the appointment of Charles ("Chuck") Austin to its Board of Directors.

    "Chuck is a recognized leader in the pharmaceutical and medical device industries," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "As a board member, we are excited to draw from his tremendous manufacturing and biopharma expertise as Marinus continues to grow and develop as an organization."

    With more than 25 years of experience in the life sciences sector, Mr. Austin has held numerous roles in supply chain operations, research and development, and engineering throughout his career…

    Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the appointment of Charles ("Chuck") Austin to its Board of Directors.

    "Chuck is a recognized leader in the pharmaceutical and medical device industries," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "As a board member, we are excited to draw from his tremendous manufacturing and biopharma expertise as Marinus continues to grow and develop as an organization."

    With more than 25 years of experience in the life sciences sector, Mr. Austin has held numerous roles in supply chain operations, research and development, and engineering throughout his career with Johnson & Johnson. Prior to his appointment to Marinus' Board of Directors, Mr. Austin served as Corporate Vice President, Global Supply Chain at Johnson & Johnson, where he was a member of the Johnson & Johnson Management Committee and was responsible for all manufacturing, logistics, quality, compliance, direct procurement, environmental, health and safety, and engineering and real estate for the corporation.

    As Company Group Chairman of Ethicon Surgical Care – a Johnson & Johnson Company – Mr. Austin helped combine four separate operating units, re-engineer a global commercial model, and return the overall franchise to growth.

    "We are delighted that Chuck will be joining the board, and we feel confident that his successful commercial, as well as operational background will bring a complementary skill set to our current board," said Nicole Vitullo, Director, Chairman of the Board at Marinus. "We believe that he will be instrumental in advancing the company's strategy as we approach key milestones in our epilepsy programs."

    Mr. Austin obtained a Bachelor of Science in Engineering from the United States Military Academy, West Point, New York, and served in the United States Army for over nine years. He currently serves on multiple boards in the medical and consumer spaces and is a principal in JK Advisors, a San Diego based firm focused on the medical space.

    About Marinus Pharmaceuticals

    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of treatments in rare epilepsy and neuropsychiatric 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. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical development schedule and milestones, the ability to complete enrollment in our clinical trials, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, and potential efficacy and therapeutic potential of our product candidate. Forward-looking statements in this 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, the uncertainties inherent in the conduct of clinical trials, the timing of clinical trials, enrollment in clinical trials, availability of data from clinical trials, expectations for regulatory approvals, the attainment of clinical trial results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, unanticipated costs and expenses, our ability to raise additional capital, the effect of the COVID-19 pandemic on our business and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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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    • Status Epilepticus pivotal Phase 3 trial on-track to begin in Q3 2020
    • CDKL5 Deficiency Disorder Phase 3 trial results in Q3 2020
    • First patient enrolled in Phase 2 Tuberous Sclerosis Complex trial
    • Enrollment in PCDH19 proof-of-concept trial increases to 25-30 patients
    • Pipeline update webinar featuring four KOLs today at 8:00am ET

    RADNOR, Pa., June 30, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided a pipeline update in advance of it clinical and commercial overview webinar to be held today, Tuesday, June 30, 2020 from 8am – 10:30am Eastern Time.

    "With a final protocol in place, we are on-track…

    • Status Epilepticus pivotal Phase 3 trial on-track to begin in Q3 2020

    • CDKL5 Deficiency Disorder Phase 3 trial results in Q3 2020
    • First patient enrolled in Phase 2 Tuberous Sclerosis Complex trial
    • Enrollment in PCDH19 proof-of-concept trial increases to 25-30 patients
    • Pipeline update webinar featuring four KOLs today at 8:00am ET

    RADNOR, Pa., June 30, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided a pipeline update in advance of it clinical and commercial overview webinar to be held today, Tuesday, June 30, 2020 from 8am – 10:30am Eastern Time.

    "With a final protocol in place, we are on-track to begin our Phase 3 pivotal trial in status epilepticus next quarter," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "Our Phase 3 trial is designed to demonstrate both rapid onset of antiepileptic activity along with sustained status cessation, allowing physicians to focus their attention on the patients' underlying disease state and avoid the often devastating consequences of uncontrolled SE and anesthesia induced coma."

    Dr. Braunstein added, "Our oral ganaxolone program has focused on rare genetic epilepsies with a biology-driven scientific foundation underserved by current available treatment options. To that end, we have initiated a Phase 2 study in tuberous sclerosis complex-related epilepsy and are preparing for our Phase 3 data readout in CDD next quarter. We look forward to sharing further details on our clinical and commercial strategies during our pipeline update event today and are thrilled to have KOL engagement and insight as we discuss our programs." 

    Pipeline Update:

    Status Epilepticus (SE)

    • Marinus has finalized the protocol for its planned Phase 3 pivotal clinical trial in SE following its End of Phase 2 Meeting held in March 2020 and based on the successful results from its completed Phase 2 trial.
    • The Phase 3 study will be a randomized, double-blind, placebo-controlled trial in SE patients who have failed benzodiazepines and 2 or more intravenous anti-epileptic drugs (AEDs).
    • The trial will enroll 124 patients randomized to receive ganaxolone or placebo adjunctive to standard of care at approximately 80-100 U.S. sites.
    • Patients will receive a 36-hour infusion followed by a 12-hour taper for a total 48-hour treatment period, which, for patients randomized to receive ganaxolone, targets a plasma concentration of greater or equal to 500ng/mL for 12-hours (same target concentration, 50% longer duration as the Target Dose evaluated in Phase 2).
    • The co-primary endpoints for the study are (i) proportion of patients with SE cessation within 30 minutes of treatment initiation without medications for the acute treatment of SE and (ii) proportion of patients with no progression to IV anesthesia for 36 hours following treatment initiation.
    • Trial initiation on-track for Q3 2020 with topline data expected first half of 2022

    CDKL5 Deficiency Disorder (CDD)

    • Marinus remains on-track to report top‑line data from the pivotal Phase 3 Marigold Study, which is evaluating the use of oral ganaxolone in children and young adults with CDD. The global, double‑blind, placebo‑controlled, clinical trial has enrolled 101 patients between the ages of 2 and 21 with a confirmed disease‑related CDKL5 gene variant.
    • In advance of topline data, Marinus has begun preparations for an expanded access program (EAP) in CDD that will allow the Company, on positive data, to offer ganaxolone to patients  who were unable to participate in the Phase 3 study.

    Tuberous Sclerosis Complex (TSC)

    • The first patient has been enrolled in the Company's Phase 2 open‑label trial to evaluate the safety and tolerability of adjunctive ganaxolone treatment in patients with TSC. The trial is expected to enroll approximately 30 patients ages 2 to 65.
    • Patients will undergo a four‑week baseline period followed by a 12‑week treatment period where they will receive up to 600 mg of ganaxolone (oral liquid suspension) three times a day. Patients who meet eligibility criteria may continue ganaxolone treatment during a 24‑week extension to the trial.
    • The primary endpoint for the trial is percent change in 28‑day primary seizure frequency for the treatment period relative to baseline. The Company plans to analyze allopregnanolone sulfate levels as part of the trial efficacy analysis.

    PCDH19 Related Epilepsy (PCDH19-RE)

    • Marinus remains on-track to enroll 25-30 patients (an increase from its previous guidance of 15-20 patients) in the ongoing Phase 2 Violet Study evaluating allopregnanolone sulfate as a biomarker and ganaxolone as a treatment in PCDH19-RE patients.
    • The Company believes the increased patient enrollment has the potential to strengthen the ability to detect a meaningful signal as proof-of-concept for the allopregnanolone sulfate biomarker hypothesis.
    • Marinus remains on-track to report topline data from this POC trial in the first half of 2021.             

    Webinar Details:



    To listen to the live webinar at 8am ET today, please click here.

    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 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 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 our clinical development plans for ganaxolone; expected dosing in our clinical trials; the clinical development schedule and milestones; our expected timing to begin and complete enrollment in our clinical trials, including our expectation to dose the first patient in our Phase 3 clinical trial for SE in third quarter 2020; the expected trial design, target patient population and endpoints for our clinical trials; interpretation of scientific basis for ganaxolone use; timing for availability and release of data, including the expected release of data from the Marigold Study in the third quarter of 2020 and from the proof of concept study in PCDH19 in the first half of 2021; the potential safety and efficacy of ganaxolone; the therapeutic potential of ganaxolone; and our expectations regarding the effect of the COVID-19 pandemic on our business and clinical development plans; 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, 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.       

    CONTACT:   

    Sasha Damouni Ellis

    Vice President, Investor Relations & Corporate Communications

    Marinus Pharmaceuticals, Inc.

    484-253-6792

     

    Primary Logo

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  26. RADNOR, Pa., June 24, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it will host a webinar featuring a clinical update and commercial overview on its pipeline programs in Status Epilepticus, CDKL5 Deficiency Disorder and Tuberous Sclerosis Complex. In addition, there will be four key opinion leaders participating in the event, which will take place on Tuesday, June 30, 2020 from 8am – 10.30am Eastern Time.

    Preliminary Agenda:

    Marinus Overview  Dr. Scott Braunstein, Chief Executive Officer
        
    Status Epilepticus  Dr. Joe Hulihan, Chief Medical Officer
    Dr. Larry Hirsch, Yale Comprehensive Epilepsy

    RADNOR, Pa., June 24, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced it will host a webinar featuring a clinical update and commercial overview on its pipeline programs in Status Epilepticus, CDKL5 Deficiency Disorder and Tuberous Sclerosis Complex. In addition, there will be four key opinion leaders participating in the event, which will take place on Tuesday, June 30, 2020 from 8am – 10.30am Eastern Time.

    Preliminary Agenda:

    Marinus Overview  Dr. Scott Braunstein, Chief Executive Officer
        
    Status Epilepticus  Dr. Joe Hulihan, Chief Medical Officer

    Dr. Larry Hirsch, Yale Comprehensive Epilepsy Center

    Dr. Henrikas Vaitkevicius, Brigham and Women's Hospital

    Department of Neurology

    Dr. Alex Aimetti, Head of Scientific Affairs

        
    CDD Overview  Dr. Scott Demarest, Children's Hospital Colorado
       Dr. Joe Hulihan, Chief Medical Officer
        
    Tuberous Sclerosis  Dr. Darcy Krueger, Cincinnati Children's Hospital Medical Center
    Complex Overview  Dr. Alex Aimetti, Head of Scientific Affairs
        
    Commercial Strategy   Thomas Lyons, Vice President of Business Development & Licensing

    To register in advance and listen to the live webinar please click here.

    Lawrence Hirsch, M.D., Professor of Neurology, Chief of Division of Epilepsy and Electroencephalography (EEG), and Co-Director of the Yale Comprehensive Epilepsy Center. He has held leadership positions at the American Clinical Neurophysiology Society (ACNS), American Epilepsy Society, American Academy of Neurology and the Epilepsy Foundation, and is founder and former chair of the Critical Care EEG Monitoring Research Consortium, which now includes more than 50 centers across North America. He is lead author of the ACNS guideline on critical care EEG terminology, and has been an active researcher throughout his career, having published more than 150 original research manuscripts and more than 100 reviews, editorials or book chapters on topics including status epilepticus, EEG, and antiepileptic drugs. Dr. Hirsch is co-author of the first-ever atlas on EEG in critical care.

    Henrikas Vaitkevicius, M.D. is an Attending Neurologist in the Neurocritical Care, Hospital Neurology, Neurological Infections, Inflammatory Diseases, and Stroke Divisions of Brigham and Women's Hospital Department of Neurology. Dr. Vaitkevicius, who is also an Assistant Professor at Harvard Medical School, received his graduate and medical school training at Wayne State University in Detroit, MI. He completed his neurology residency and neurocritical care fellowship at Brigham & Women's and Mass General Hospital in Boston, MA. Dr. Vaitkevicius has appeared in 38 peer-reviewed publications, five non-peer reviewed scientific or medical materials, and in 2015 was an ad hoc reviewer for the New England Journal of Medicine. He serves as the Director of Brain Hub: Studio for Research and Innovation in Critical Care Neurology where he focuses on fostering collaborations among academic departments and pharmaceutical industries to bring novel treatments to the bedside.

    Scott Demarest, M.D. is an Assistant Professor, Pediatrics-Neurology at the University of Colorado. Dr. Demarest's research focuses on the characterization of neurogenetic conditions through phenotyping and natural history studies, development of better outcome measures as tools for both natural history studies and clinical trials and the design of disease modifying clinical trial for rare diseases. Additionally, Dr. Demarest is the PI of the International CDKL5 Clinical Research Network (ICCRN). The ICCRN includes eight US institutions and one international site in Australia all working collaboratively to develop clinical trial readiness for disease modifying trials for CDKL5 Deficiency Disorder through improved natural history data for CDKL5 and the development and validation of appropriate outcome measures for CDKL5 Deficiency Disorder.  Dr. Demarest attended medical school at the University of Texas Health Science Center in San Antonio. He completed his residency in pediatrics and child neurology at Children's National Health System in Washington, DC and his Epilepsy fellowship at Children's Hospital Colorado.

    Darcy Krueger M.D., Ph.D. is Director of the Tuberous Sclerosis Clinic, Associate Professor of Clinical Pediatrics and Neurology, and Associate Director of Research in Neurology at Cincinnati Children's Hospital Medical Center. He is a founding member of the Tuberous Sclerosis Complex Clinical Research Consortium and served as its first Director from 2011 to 2013. Dr. Krueger received PhD and MD degrees from Saint Louis University in 2000 and 2002, respectively, and completed a combined residency in pediatrics, neurology, and child neurology at Cincinnati Children's Hospital Medical Center and the University of Cincinnati College of Medicine in 2007. Dr. Krueger was a leading investigator for the landmark clinical trial using mTOR inhibitors to treat subependymal giant cell astrocytomas that led to the first ever FDA-approved treatment for TSC in 2010. Dr. Krueger leads multiple clinical research studies in TSC, including the TSC Autism Center of Excellence Network funded by the National Institutes of Health and the TS Alliance. Additional projects are aimed at better understanding the underlying mechanisms of TSC disease pathogenesis and treatment response, with the ultimate objective of developing treatment strategies that reverse or prevent disease progression and secondary complications.

    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 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 initiating a Phase 3 trial in status epilepticus.

               

    CONTACT: 

    Sasha Damouni Ellis

    Vice President, Investor Relations & Corporate Communications

    Marinus Pharmaceuticals, Inc.

    484-253-6792

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  27. RADNOR, Pa., June 22, 2020 (GLOBE NEWSWIRE) -- 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 SVB Leerink's CybeRx Series: 2nd Annual CNS Forum.

    Additional details can be found below:

    Title: A Pipeline Update with Scott Braunstein (CEO) and Joe Hulihan (CMO)
    Date: Tuesday, June 23, 2020
    Time: 3:20 PM Eastern Time
    Format:  Fireside chat

    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…

    RADNOR, Pa., June 22, 2020 (GLOBE NEWSWIRE) -- 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 SVB Leerink's CybeRx Series: 2nd Annual CNS Forum.

    Additional details can be found below:

    Title: A Pipeline Update with Scott Braunstein (CEO) and Joe Hulihan (CMO)
    Date: Tuesday, June 23, 2020
    Time: 3:20 PM Eastern Time
    Format:  Fireside chat

    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 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 intends to initiate a Phase 3 trial in status epilepticus.

               

    CONTACT: 

    Sasha Damouni Ellis

    Vice President, Investor Relations & Corporate Communications

    Marinus Pharmaceuticals, Inc.

    484-253-6792

    Primary Logo

    View Full Article Hide Full Article
  28. RADNOR, Pa., June 16, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of an inducement award to a new employee. The Compensation Committee of the Board of Directors of Marinus granted Martha Manning, Esq., the Company's new Vice President, General Counsel and Secretary, a non-qualified stock option to purchase an aggregate of 300,000 shares of its common stock as an inducement material to Ms. Manning entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    The stock option grant has an exercise price of $2.52 per share…

    RADNOR, Pa., June 16, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of an inducement award to a new employee. The Compensation Committee of the Board of Directors of Marinus granted Martha Manning, Esq., the Company's new Vice President, General Counsel and Secretary, a non-qualified stock option to purchase an aggregate of 300,000 shares of its common stock as an inducement material to Ms. Manning entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    The stock option grant has an exercise price of $2.52 per share, which is equal to the closing price of Marinus' common stock on June 15, 2020, the date of grant for the stock option. The stock option will vest and become exercisable as to 25% of the underlying shares on the one-year anniversary of the date of grant, and will vest and become exercisable as to the remaining 75% of the underlying shares in 36 equal monthly installments at the end of each month following such anniversary, subject to Ms. Manning's continued employment with Marinus on such vesting dates. The stock option was granted as an inducement material to Ms. Manning entering into employment with Marinus in accordance with Nasdaq Listing Rule 5635(c)(4), and is subject to the terms and conditions of the applicable award agreement covering such grant.

    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 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 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 our clinical development plans for ganaxolone and the clinical development schedule and milestones. Forward-looking statements in this 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; clinical trial results may not support 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; 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.          

               

    CONTACT:   

    Sasha Damouni Ellis

    Vice President, Investor Relations & Corporate Communications

    Marinus Pharmaceuticals, Inc.

    484-253-6792

    Primary Logo

    View Full Article Hide Full Article
  29. RADNOR, Pa., June 15, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat orphan seizure disorders, today announced that Martha Manning, Esq., has been appointed Vice President, General Counsel and Secretary.

    "We are thrilled to welcome Martha to our growing team at Marinus, and believe her legal experience supporting biopharma companies across all development stages will be instrumental as we navigate through our upcoming milestones for ganaxolone in orphan seizure disorders," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "Martha will bring invaluable industry leadership to our organization as we begin to plan for…

    RADNOR, Pa., June 15, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat orphan seizure disorders, today announced that Martha Manning, Esq., has been appointed Vice President, General Counsel and Secretary.

    "We are thrilled to welcome Martha to our growing team at Marinus, and believe her legal experience supporting biopharma companies across all development stages will be instrumental as we navigate through our upcoming milestones for ganaxolone in orphan seizure disorders," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "Martha will bring invaluable industry leadership to our organization as we begin to plan for and execute potential commercialization, strategic alliances and value enhancing portfolio expansion opportunities."

    Ms. Manning has spent over 20 years in the life sciences sector, providing legal advice for development stage and commercial biopharma companies. Immediately prior to joining Marinus, Ms. Manning was Executive Vice President, General Counsel and Secretary for Achillion Pharmaceuticals, Inc., where she helped to develop and execute corporate strategy, managed corporate governance for Board of Directors, and handled all legal and compliance matters. Before her tenure at Achillion, she was General Counsel for ICeutica Inc. where she was also responsible for all legal matters, including supporting corporate growth through R&D, product and clinical development and out licensing, and corporate governance. Ms. Manning has held roles that have included Chief Legal Officer for OraPharma, Inc., Vice President and General Counsel for Sandoz, Inc. (Novartis), Senior Vice President, General Counsel and Secretary for Adolor Corporation, and Business and Finance Associate for the law firm Morgan Lewis & Bockius. Ms. Manning earned her Juris Doctor degree from the University of Pennsylvania Law School and her Bachelor of Business Administration from the University of Massachusetts. She is a member of the Pennsylvania Bar.

    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 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 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 our clinical development plans for ganaxolone and the clinical development schedule and milestones. Forward-looking statements in this 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; clinical trial results may not support 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; 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.

    CONTACT:

    Sasha Damouni Ellis

    Vice President, Investor Relations & Corporate Communications

    Marinus Pharmaceuticals, Inc.

    484-253-6792

    Primary Logo

    View Full Article Hide Full Article
  30. RADNOR, Pa., June 02, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), ("Marinus" or the "Company") today announced the closing of an underwritten public offering of 18,400,000 shares of common stock at a public offering price of $2.50 per share, including the exercise in full by the underwriters of their option to purchase up to 2,400,000 additional shares of common stock. The offering raised gross proceeds of approximately $46 million, before deducting underwriting discounts and commissions and other offering expenses payable by the Company.

    Cowen and Cantor Fitzgerald & Co. acted as lead book-running managers for the offering. SunTrust Robinson Humphrey acted as a bookrunner for the offering. Oppenheimer & Co. Inc…

    RADNOR, Pa., June 02, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), ("Marinus" or the "Company") today announced the closing of an underwritten public offering of 18,400,000 shares of common stock at a public offering price of $2.50 per share, including the exercise in full by the underwriters of their option to purchase up to 2,400,000 additional shares of common stock. The offering raised gross proceeds of approximately $46 million, before deducting underwriting discounts and commissions and other offering expenses payable by the Company.

    Cowen and Cantor Fitzgerald & Co. acted as lead book-running managers for the offering. SunTrust Robinson Humphrey acted as a bookrunner for the offering. Oppenheimer & Co. Inc. acted as lead manager for the offering. H.C. Wainwright & Co. acted as co-manager for the offering.

    Marinus intends to use the net proceeds of the offering to fund the development of its product candidates and for general corporate purposes, which may include working capital, capital expenditures, research and development expenditures, clinical trial expenditures, acquisitions of new technologies, products or businesses, and investments.

    The shares of common stock described above were offered by the Company pursuant to a registration statement on Form S-3 (No. 333-221243) previously filed with the U.S. Securities and Exchange Commission (the "SEC") and declared effective by the SEC on December 1, 2017. A preliminary prospectus supplement related to the public offering was filed with the SEC on May 28, 2020. A final prospectus supplement relating to the public offering was filed with the SEC and is available on the SEC's website located at http://www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying prospectus may be obtained from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Attn: Prospectus Department, by telephone at (833) 297-2926, or by email at , or Cantor Fitzgerald & Co., 499 Park Avenue, 6th Floor, New York, New York 10022, Attn: Capital Markets Department, or by email at .

    This press release does not constitute an offer to sell or the solicitation of offers to buy any securities of Marinus being offered, and shall not constitute an offer, solicitation or sale of any security in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    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 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 intends to initiate a Phase 3 trial in status epilepticus.

    Forward-Looking Statements

    To the extent any statements made in this press release deal with information that is not historical, these are forward-looking statements under the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements regarding the anticipated use of proceeds for the proposed offering and other statements identified by words such as "will," "potential," "could," "can," "believe," "intends," "continue," "plans," "expects," "anticipates," "estimates," "may," other words of similar meaning or the use of future dates. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Uncertainties and risks may cause the Company's actual results to be materially different than those expressed in or implied by the Company's forward-looking statements. For the Company, this includes obtaining regulatory approvals, stock price volatility and the impact of general business and economic conditions. More detailed information on these and additional factors that could affect the Company's actual results are described in the Company's filings with the Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2019 and subsequent quarterly reports on Form 10-Q. All forward-looking statements in this news release speak only as of the date of this news release. The Company undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

    Contact

    Sasha Damouni Ellis
    Vice President, Investor Relations & Corporate Communications
    Marinus Pharmaceuticals, Inc.
    484-253-6792

    Primary Logo

    View Full Article Hide Full Article
  31. RADNOR, Pa., May 28, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) ("Marinus" or the "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the pricing of an underwritten public offering of 16,000,000 shares of its common stock at a public offering price of $2.50 per share. The gross proceeds of the offering to the Company are expected to be approximately $40,000,000, before deducting the underwriting discounts and commissions and other estimated offering expenses. In addition, Marinus has granted the underwriters a 30-day option to purchase up to an additional 2,400,000 shares of common stock at the public offering price, less underwriting…

    RADNOR, Pa., May 28, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) ("Marinus" or the "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the pricing of an underwritten public offering of 16,000,000 shares of its common stock at a public offering price of $2.50 per share. The gross proceeds of the offering to the Company are expected to be approximately $40,000,000, before deducting the underwriting discounts and commissions and other estimated offering expenses. In addition, Marinus has granted the underwriters a 30-day option to purchase up to an additional 2,400,000 shares of common stock at the public offering price, less underwriting discounts and commissions.

    The closing of the offering is expected to occur on or about June 2, 2020, subject to the satisfaction of customary closing conditions.

    Cowen and Cantor Fitzgerald & Co. are acting as lead book-running managers for the offering. SunTrust Robinson Humphrey is acting as a bookrunner for the offering. Oppenheimer & Co. Inc. is acting as lead manager for the offering. H.C. Wainwright & Co. is acting as co-manager for the offering.

    Marinus intends to use the net proceeds of the offering to fund the development of its product candidates and for general corporate purposes, which may include working capital, capital expenditures, research and development expenditures, clinical trial expenditures, acquisitions of new technologies, products or businesses, and investments.

    The securities described above are being offered by the Company pursuant to a shelf registration statement on Form S-3 (No. 333-221243) previously filed with and declared effective by the Securities and Exchange Commission (the "SEC") on December 1, 2017.

    A preliminary prospectus supplement relating to the offering was filed with the SEC on May 28, 2020 and is available on the SEC's website at http://www.sec.gov. The final prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and also will be available on the SEC's website. Before investing in the offering, you should read each of the prospectus supplement and the accompanying prospectus relating to the offering in their entirety as well as the other documents that the Company has filed with the SEC that are incorporated by reference in the prospectus supplement and the accompanying prospectus relating to the offering, which provide more information about the Company and the offering. Copies of the final prospectus supplement, when available, and the accompanying prospectus relating to the offering may be obtained from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Attn: Prospectus Department, by telephone at (833) 297-2926, or by email at , or Cantor Fitzgerald & Co., 499 Park Avenue, 6th Floor, New York, New York 10022, Attn: Capital Markets Department, or by email at

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    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 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 intends to initiate a Phase 3 trial in status epilepticus.

    Forward-Looking Statements

    To the extent any statements made in this press release deal with information that is not historical, these are forward-looking statements under the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements regarding the closing of the proposed offering, as well as the anticipated use of proceeds for the proposed offering and other statements identified by words such as "will," "potential," "could," "can," "believe," "intends," "continue," "plans," "expects," "anticipates," "estimates," "may," other words of similar meaning or the use of future dates. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Uncertainties and risks may cause the Company's actual results to be materially different than those expressed in or implied by the Company's forward-looking statements. For the Company, this includes satisfaction of the customary closing conditions to the offering, delays in obtaining required stock exchange or other regulatory approvals, stock price volatility, the impact of general business and economic conditions, the expected gross proceeds from the offering and the intended use of proceeds of the offering. More detailed information on these and additional factors that could affect the Company's actual results are described in the Company's filings with the Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2019 and subsequent quarterly reports on Form 10-Q. All forward-looking statements in this news release speak only as of the date of this news release. The Company undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

    Contact
    Sasha Damouni Ellis
    Vice President, Investor Relations & Corporate Communications
    Marinus Pharmaceuticals, Inc.
    484-253-6792
     

    Primary Logo

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  32. RADNOR, Pa., May 28, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) ("Marinus" or the "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that it intends to offer and sell shares of its common stock in an underwritten public offering. All of the shares to be sold in the offering will be offered by Marinus. The offering is subject to market conditions and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. In addition, Marinus intends to grant the underwriters a 30-day option to purchase up to an additional 15 percent of the number of shares offered in the…

    RADNOR, Pa., May 28, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) ("Marinus" or the "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced that it intends to offer and sell shares of its common stock in an underwritten public offering. All of the shares to be sold in the offering will be offered by Marinus. The offering is subject to market conditions and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. In addition, Marinus intends to grant the underwriters a 30-day option to purchase up to an additional 15 percent of the number of shares offered in the public offering.

    Cowen and Cantor Fitzgerald & Co. are acting as lead book-running managers for the offering. SunTrust Robinson Humphrey is acting as a bookrunner for the offering. Oppenheimer & Co. Inc. is acting as lead manager for the offering. H.C. Wainwright & Co. is acting as co-manager for the offering.

    Marinus intends to use the net proceeds of the offering to fund the development of its product candidates and for general corporate purposes, which may include working capital, capital expenditures, research and development expenditures, clinical trial expenditures, acquisitions of new technologies, products or businesses, and investments.

    The securities described above are being offered by the Company pursuant to a shelf registration statement on Form S-3 (No. 333-221243) previously filed with and declared effective by the Securities and Exchange Commission (the "SEC") on December 1, 2017.

    The securities will be offered by means of a prospectus supplement and accompanying prospectus relating to the offering that form a part of the registration statement. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC's website at http://www.sec.gov. Copies of the final prospectus supplement, when available, and the accompanying prospectus relating to the offering may be obtained from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Attn: Prospectus Department, by telephone at (833) 297-2926, or by email at , or Cantor Fitzgerald & Co., 499 Park Avenue, 6th Floor, New York, New York 10022, Attn: Capital Markets Department, or by email at .

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    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 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 intends to initiate a Phase 3 trial in status epilepticus.

    Forward-Looking Statements

    To the extent any statements made in this press release deal with information that is not historical, these are forward-looking statements under the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements regarding the timing and success of the proposed offering, as well as the anticipated use of proceeds for the proposed offering and other statements identified by words such as "will," "potential," "could," "can," "believe," "intends," "continue," "plans," "expects," "anticipates," "estimates," "may," other words of similar meaning or the use of future dates. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Uncertainties and risks may cause the Company's actual results to be materially different than those expressed in or implied by the Company's forward-looking statements. For the Company, this includes satisfaction of the customary closing conditions of the offering, delays in obtaining required stock exchange or other regulatory approvals, stock price volatility and the impact of general business and economic conditions. More detailed information on these and additional factors that could affect the Company's actual results are described in the Company's filings with the Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2019 and subsequent quarterly reports on Form 10-Q. All forward-looking statements in this news release speak only as of the date of this news release. The Company undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

    Contact
    Sasha Damouni Ellis
    Vice President, Investor Relations & Corporate Communications
    Marinus Pharmaceuticals, Inc.
    484-253-6792

    Primary Logo

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  33. Constructive End of Phase 2 Meeting with FDA for Status Epilepticus Completed in March; Pivotal Phase 3 Clinical Trial Expected to Begin Next Quarter

    Topline Data on Track for Q3 2020 from Pivotal Phase 3 Marigold Study in CDKL5 Deficiency Disorder and Preparations Continue for Potential NDA Filing

    First Patients to be Screened this Quarter for Phase 2 Tuberous Sclerosis Complex Clinical Trial

    RADNOR, Pa., May 04, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided an update on its clinical development activities and reported its financial results for the first quarter ended March 31, 2020.

    "We…

    Constructive End of Phase 2 Meeting with FDA for Status Epilepticus Completed in March; Pivotal Phase 3 Clinical Trial Expected to Begin Next Quarter

    Topline Data on Track for Q3 2020 from Pivotal Phase 3 Marigold Study in CDKL5 Deficiency Disorder and Preparations Continue for Potential NDA Filing

    First Patients to be Screened this Quarter for Phase 2 Tuberous Sclerosis Complex Clinical Trial

    RADNOR, Pa., May 04, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided an update on its clinical development activities and reported its financial results for the first quarter ended March 31, 2020.

    "We are proud to have made real progress across all of our clinical programs prior to and during the unprecedented and challenging global impact of the COVID-19 pandemic," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "We remain confident that our key milestones are still on track, including the topline data from our pivotal Phase 3 clinical trial in CDKL5 deficiency disorder in Q3 2020. With positive topline data, we intend to continue preparations for both our first NDA submission and the commercial launch of ganaxolone."

    Dr. Braunstein added, "We had a highly constructive end-of-Phase 2 meeting with the FDA for our status epilepticus program and anticipate enrolling the first patient in the Phase 3 trial later this year. In addition, we have made significant headway in expanding our biomarker-informed trial for tuberous sclerosis complex, as our first site has been activated for patient enrollment. This will be a year of noteworthy milestones for the organization, and we are confident that we are just beginning to unlock ganaxolone's potential to improve the lives of patients and families affected by rare and severe epilepsies."

    Pipeline Update:

    Status Epilepticus (SE)

    • Marinus is moving forward with plans for its Phase 3 pivotal clinical trial in SE after a constructive end-of-Phase 2 meeting with the FDA. Based upon feedback from the FDA, the Company anticipates the following trial design and timeline:
      • Co-primary endpoints that focus on status cessation within 30-minutes and suppression of status for at least 24 hours.
      • Ganaxolone administered intravenously for 48 hours, the first 12 hours of which is expected to target a 500 ng/ml serum concentration.
      • Enrollment of approximately 125 patients; providing greater than 90 percent power to detect a 30 percent efficacy difference between ganaxolone and placebo.
      • Patients enrolled will have previously failed a benzodiazepine and at least two second-line antiepileptic drugs.
      • Patient enrollment to begin in Q3 2020 and trial sites have already identified and are in process of being readying.
      • Topline data expected in the first half of 2022.

    CDKL5 Deficiency Disorder (CDD)

    • Enrollment has been completed in the pivotal Phase 3 Marigold Study, which is evaluating the use of oral ganaxolone in children and young adults with CDD. The global, double‑blind, placebo‑controlled, clinical trial has enrolled 101 patients between the ages of 2 and 21 with a confirmed disease‑related CDKL5 gene variant.
    • Patients randomized to ganaxolone receive up to 600 mg (oral liquid suspension) three times a day.
    • The primary endpoint of the trial is percent change in 28‑day seizure frequency. The discontinuation rate in this trial is in-line with expectations (less than 10 percent), and enrollment in the open label extension part of the trial continues to be high.
    • Marinus remains on-track to report top‑line data from the trial in Q3 2020 with no expected material delays due to COVID-19. The Company has begun preparations for a potential NDA filing and the development of its commercial strategy.
    • Phase 1 supportive clinical trials have experienced delays in enrollment due to COVID-19, which, as of today, are not expected to impact timing for a potential NDA filing.

    Tuberous Sclerosis Complex (TSC)

    • Marinus is conducting a Phase 2 open‑label trial to evaluate the safety and tolerability of adjunctive ganaxolone treatment in patients with TSC. The trial is expected to enroll approximately 30 patients ages 2 to 65.
    • Patients will undergo a four‑week baseline period followed by a 12‑week treatment period where they will receive up to 600 mg of ganaxolone (oral liquid suspension) three times a day. Patients who meet eligibility criteria may continue ganaxolone treatment in a 24‑week extension to the trial.
    • The primary endpoint for the trial is percent change in 28‑day primary seizure frequency for the treatment period relative to baseline. The Company plans to analyze allopregnanolone sulfate levels as part of the trial efficacy analysis.
    • Marinus intends to start screening patients this quarter. As of today, the Company does not anticipate COVID-19 will have a material impact on enrollment or trial progress and expects to report top line data in Q1 2021.

    PCDH19 Related Epilepsy (PCDH19-RE)

    • After careful consideration, Marinus will transition the ongoing Phase 3 Violet Study to a proof-of-concept (POC) trial evaluating allopregnanolone sulfate as a biomarker in the patients currently enrolled with a confirmed PCDH19 mutation. Marinus has decided to limit trial enrollment due to several factors, including the significant requirements needed to enroll a global trial (including COVID-19 impact), the episodic nature of seizures in PCDH19 patients, and potential commercial challenges. The Company now expects to complete the double-blind portion of the trial with approximately 15-20 patients and announce results of this POC trial in the first half of 2021.
    • The scale down to a POC trial is part of the Company's strategic plan to focus capital resources on indications with high unmet needs and a meaningful percentage of patients being underserved by available treatment options.
    • Marinus plans to continue to evaluate if the allopregnanolone sulfate biomarker hypothesis could have broader utility in other targeted indications.
    • The POC biomarker trial will stratify patients into one of two biomarker groups based on baseline allopregnanolone sulfate levels and randomized (ganaxolone or placebo) within each stratum. The trial will consist of a 12‑week prospective baseline period to collect seizure data, followed by a 17‑week double‑blind treatment phase. Patients randomized to ganaxolone will titrate over four weeks to a dose of up to 600 mg of ganaxolone (oral liquid suspension) three times a day and maintain that dose for the following 13‑weeks.
    • Marinus intends to provide access to ganaxolone for PCDH19-RE patients who saw benefits in the Violet Study.

    Corporate Update:

    • In April, the Company announced the formation of Scientific Advisory Board with six globally recognized seizure disorder experts.
    • In March, Sasha Damouni Ellis was appointed Vice President, Investor Relations and Corporate Communications.

    Financial Update:

    On March 31, 2020, the Company had cash and cash equivalents and investments totaling $77.8 million compared to $91.7 million on December 31, 2019. Marinus believes that its cash, cash equivalents and investments as of March 31, 2020 will enable the Company to fund operating expenses and capital expenditure requirements into the third quarter of 2021.

    Research and development expenses increased to $15.0 million for the three months ended March 31, 2020, as compared to $8.9 million for the three months ended March 31, 2019. The primary drivers for the increase to our research and development expenditures are clinical and manufacturing activities in support of our Phase 3 trial in CDD and preparations for a Phase 3 trial in SE, partially offset by decreased costs for non-seizure disorder indications.

    General and administrative expenses were $3.9 million for the three months ended March 31, 2020 as compared to $3.7 million in the prior year. The primary drivers of the increase were professional fees and other costs associated with an increased scale of operations.

    The Company reported a net loss of $18.7 million for the three months ended March 31, 2020, compared to $12.5 million for the three months ended March 31, 2019. Cash used in operating activities increased to $14.0 million for the three months ended March 31, 2020 compared to $11.7 million for the three months ended March 31, 2019.

    Readers are referred to, and encouraged to read in its entirety, the Company's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 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.

    Marinus Pharmaceuticals, Inc.
    Selected Financial Data (in thousands, except share and per share amounts)
    (unaudited)

         
        March 31,   December 31,  
        2020   2019  
                   
    Assets:              
    Cash and cash equivalents   $ 70,326   $ 90,943  
    Investments     7,460     739  
    Other assets     6,772     7,160  
    Total assets   $ 84,558   $ 98,842  
    Liabilities and stockholders' equity:              
    Accounts payable and accrued expenses   $ 10,325   $ 8,031  
    Other liabilities     2,921     3,042  
    Total liabilities     13,245     11,073  
    Series A preferred stock     28,200     28,200  
    Total stockholders' equity     43,113     59,569  
    Total liabilities and stockholders' equity   $ 84,558   $ 98,842  


                   
        Three Months Ended March 31,  
        2020
      2019
     
                   
    Expenses:              
    Research and development   $ 15,004     $ 8,872    
    General and administrative     3,850       3,667    
    Loss from operations     (18,854 )     (12,539 )  
    Interest income     222       96    
    Other expense     (40 )     (40 )  
    Net loss     (18,672 )     (12,483 )  
    Deemed dividends on convertible preferred stock     (8,880 )      —    
    Net loss applicable to common shareholders   $ (27,552 )   $ (12,483 )  
    Per share information:              
    Net loss per share of common stock—basic and diluted   $ (0.32 )   $ (0.24 )  
    Basic and diluted weighted average shares outstanding     86,661,845       52,465,207    

    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 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 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 our clinical development plans for ganaxolone, expected dosing in our clinical trials, the clinical development schedule and milestones, our expected timing to begin and complete enrollment in our clinical trials, the expected protocols for our clinical trials, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the potential safety and efficacy of ganaxolone, the therapeutic potential of ganaxolone, our expectations regarding the effect of the COVID-19 pandemic on our business and clinical development plans, and our expectations regarding the sufficiency of our working capital. Forward-looking statements in this 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; clinical trial results may not support 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; 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.

    CONTACT:
    Sasha Damouni Ellis
    Vice President, Investor Relations & Corporate Communications
    Marinus Pharmaceuticals, Inc.
    484-253-6792

    Primary Logo

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  34. RADNOR, Pa., April 30, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the formation of a Scientific Advisory Board (SAB) with the appointment of six leading experts in clinical neurology and seizure disorders: Jacqueline French, M.D., Lawrence Hirsch, M.D., Aatif Husain, M.D., Michael Rogawski M.D., Ph.D., Eugen Trinka, M.D. and Henrikas Vaitkevicius, M.D.

    "We believe the strength and enthusiasm of our newly-formed scientific advisory board, with an expertise in clinical development and global registration strategies for novel anti-epileptic drugs, is a testament to our commitment to ganaxolone…

    RADNOR, Pa., April 30, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the formation of a Scientific Advisory Board (SAB) with the appointment of six leading experts in clinical neurology and seizure disorders: Jacqueline French, M.D., Lawrence Hirsch, M.D., Aatif Husain, M.D., Michael Rogawski M.D., Ph.D., Eugen Trinka, M.D. and Henrikas Vaitkevicius, M.D.

    "We believe the strength and enthusiasm of our newly-formed scientific advisory board, with an expertise in clinical development and global registration strategies for novel anti-epileptic drugs, is a testament to our commitment to ganaxolone in patients with severe seizure disorders," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "The insights from these scientific thought-leaders will be invaluable as we approach key milestones in our epilepsy programs."

    Marinus SAB members will include:

    Jacqueline French, M.D., Professor of Neurology in the Comprehensive Epilepsy Center at NYU Langone School of Medicine and Founder/Director of the Epilepsy Study Consortium, an academic group that has performed several early phase clinical trials in epilepsy. Dr. French trained in Neurology at Mount Sinai Hospital in New York and did her fellowship training in EEG and epilepsy at Mount Sinai hospital and Yale University. Over the past 20 years Dr. French has served as the Principle investigator on several trials for new epilepsy drugs and has focused her research efforts developing new therapeutics for epilepsy and novel methodologies for clinical trials. Dr. French, who serves as Chief Medical/Innovation Officer for the Epilepsy Foundation, has been active in creating guidelines for the American Academy of Neurology and the International League Against Epilepsy. She is the editor of three books and has authored over 300 articles and chapters.

    Lawrence Hirsch, M.D., Professor of Neurology, Chief of Division of Epilepsy and Electroencephalography (EEG), and Co-Director of the Yale Comprehensive Epilepsy Center. He has held leadership positions at the American Clinical Neurophysiology Society (ACNS), American Epilepsy Society, American Academy of Neurology and the Epilepsy Foundation, and is founder and former chair of the Critical Care EEG Monitoring Research Consortium, which now includes more than 50 centers across North America. He is lead author of the ACNS guideline on critical care EEG terminology, and has been an active researcher throughout his career, having published more than 150 original research manuscripts and more than 100 reviews, editorials or book chapters on topics including status epilepticus, EEG, and antiepileptic drugs. Dr. Hirsch is co-author of the first-ever atlas on EEG in critical care.  

    Aatif Husain, M.D., Professor of Neurology and Division Chief of Epilepsy, Sleep, and Clinical Neurophysiology at the Duke University School of Medicine, is a world-renowned expert in clinical neurophysiology, with experience serving as president of the American Clinical Neurophysiology Society, treasurer of the International Federation of Clinical Neurophysiology. Through his research Dr. Husain has been highly influential in the developing treatments for seizures, with landmark studies on the treatment of refractory seizures and non-convulsive status epilepticus. Amongst the more than 100 publications and nine books he has authored, he is also the editor-in-chief of the Journal of Clinical Neurophysiology.

    Michael Rogawski M.D., Ph.D., Professor of Neurology and Pharmacology at the University of California, Davis School of Medicine is an internationally recognized expert on drug discovery and development for epilepsy, with a research focus on neuroactive steroids. He serves as lead of the University of California Drug Discovery Consortium. Dr. Rogawski was formerly chief of the Epilepsy Research Section at the National Institute of Neurological Disorders and Stroke. He received his M.D. and Ph.D. (pharmacology) from Yale and trained in neurology at Johns Hopkins. He has served on the board of directors of the American Epilepsy Society and has been a member of advisory panels to the National Institutes of Health. He has authored more than 275 research papers, reviews, and book chapters, and edited five books.

    Eugen Trinka, M.D., Professor and Chairman of the Department of Neurology, Christian Doppler University Hospital at the Paracelsus Medical University in Salzburg, Austria. Dr. Trinka's research interests include clinical epileptology with a focus on epidemiology, prognosis, epilepsy surgery, and status epilepticus. He is the founding member of the European Epilepsy Consortium, dedicated to clinical drug trials in the field of epilepsy and has authored more than 480 scientific publications and book chapters, including the seminal paper describing the diagnostic criteria for non-convulsive status epilepticus called the Salzburg criteria. Dr. Trinka is the current President of the Austrian Society of Neurology, past president of the Austrian Society for Epilepsy, Austrian Society for Clinical Neurophysiology and the Society of Epilepsy Surgery Centres in Germany, Austria. He has been elected as Chair of ILAE-EUROPE in 2017. He also serves on the ILAE Task force of Classification of Status Epilepticus, the Commission on Medical Treatment, the ILAE Working group on pre-clinical drug discovery, and the ILAE Task Force on Regulatory affairs.

    Henrikas Vaitkevicius, M.D., is an Attending Neurologist in the Neurocritical Care, Hospital Neurology, Neurological Infections, Inflammatory Diseases, and Stroke Divisions of Brigham and Women's Hospital Department of Neurology. Dr. Vaitkevicius, who is also an Assistant Professor at Harvard Medical School, received his graduate and medical school training at Wayne State University in Detroit, MI. He completed his neurology residency and neurocritical care fellowship at Brigham & Women's and Mass General Hospital in Boston, MA. Dr. Vaitkevicius has appeared in 38 peer-reviewed publications, five non-peer reviewed scientific or medical materials, and in 2015 was an ad hoc reviewer for the New England Journal of Medicine. He serves as the Director of Brain Hub: Studio for Research and Innovation in Critical Care Neurology where he focuses on fostering collaborations among academic departments and pharmaceutical industries to bring novel treatments to the bedside.

    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. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical development schedule and milestones, the ability to complete enrollment in our clinical trials, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, and potential efficacy and therapeutic potential of our product candidate. Forward-looking statements in this 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, the uncertainties inherent in the conduct of clinical trials, the timing of clinical trials, enrollment in clinical trials, availability of data from clinical trials, expectations for regulatory approvals, the attainment of clinical trial results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, unanticipated costs and expenses, our ability to raise additional capital, the effect of the COVID-19 pandemic on our business and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.

    CONTACT:   
    Sasha Damouni Ellis
    Vice President, Investor Relations & Corporate Communications
    Marinus Pharmaceuticals, Inc.
    484-253-6792

    Primary Logo

    View Full Article Hide Full Article
  35. RADNOR, Pa., April 27, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to two new employees. A majority of the independent members of the Board of Directors of Marinus granted the new employees non-qualified stock options to purchase an aggregate of 45,500 shares of its common stock as inducement awards material to each employee entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    One of the stock option grants has an exercise price of $1.79 per share, which is equal to the closing price of Marinus…

    RADNOR, Pa., April 27, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company" or "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to two new employees. A majority of the independent members of the Board of Directors of Marinus granted the new employees non-qualified stock options to purchase an aggregate of 45,500 shares of its common stock as inducement awards material to each employee entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    One of the stock option grants has an exercise price of $1.79 per share, which is equal to the closing price of Marinus' common stock on April 15, 2020, the date of grant for that stock option. The other stock option grant will have an exercise price equal to the closing price of Marinus' common stock on its date of grant, which is April 29, 2020. The stock options will vest and become exercisable as to 25% of the underlying shares on the one-year anniversary of each recipient's start date, and will vest and become exercisable as to the remaining 75% of the underlying shares in 36 equal monthly installments at the end of each month following such anniversary, subject to the employee's continued employment with Marinus on such vesting dates. The stock options were granted as inducements material to the new employees entering into employment with Marinus in accordance with Nasdaq Listing Rule 5635(c)(4), and are subject to the terms and conditions of the applicable award agreements covering the grants.

    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 is conducting the first ever pivotal trials in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Later this year, the Company intends to initiate a Phase 3 trial in SE and a Phase 2 trial in Tuberous Sclerosis Complex (TSC), respectively. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical development schedule and milestones, the ability to complete enrollment in our clinical trials, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, and potential efficacy and therapeutic potential of our product candidate. Forward-looking statements in this 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, the uncertainties inherent in the conduct of clinical trials, the timing of clinical trials, enrollment in clinical trials, availability of data from clinical trials, expectations for regulatory approvals, the attainment of clinical trial results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, unanticipated costs and expenses, 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 the Company that could affect the availability or commercial potential of our drug candidates. 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.

    CONTACT:
            Sasha Damouni Ellis
            Vice President, Investor Relations & Corporate Communications
            Marinus Pharmaceuticals, Inc.
            484-253-6792
            

    Primary Logo

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  36. RADNOR, Pa., April 23, 2020 (GLOBE NEWSWIRE) -- 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 release financial results for the first quarter ended March 31, 2020 before the market open on Monday, May 4, 2020. The Company will also host a conference call at 8:30am Eastern Time on Monday, May 4, 2020 to discuss the financial results with investors.

    Monday, May 4th @ 8:30am Eastern Time
    Domestic: 877-705-6003
    International:    201-493-6725
    Webcast:  http://public.viavid.com/index.php?id=139413
    Conference ID:  13702686

    About Marinus Pharmaceuticals
    Marinus Pharmaceuticals, Inc. is a pharmaceutical company…

    RADNOR, Pa., April 23, 2020 (GLOBE NEWSWIRE) -- 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 release financial results for the first quarter ended March 31, 2020 before the market open on Monday, May 4, 2020. The Company will also host a conference call at 8:30am Eastern Time on Monday, May 4, 2020 to discuss the financial results with investors.

    Monday, May 4th @ 8:30am Eastern Time
    Domestic: 877-705-6003
    International:    201-493-6725
    Webcast:  http://public.viavid.com/index.php?id=139413
    Conference ID:  13702686

    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. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    CONTACT: 

    Sasha Damouni Ellis
    Vice President, Investor Relations & Corporate Communications
    Marinus Pharmaceuticals, Inc.
    484-253-6792

    Primary Logo

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  37. HAYWARD, Calif., April 14, 2020 /PRNewswire/ -- Benitec Biopharma, Inc., a successor entity to Benitec Biopharma Limited (NASDAQ:BNTC), a development-stage biotechnology company focused on the advancement of novel genetic medicines, today announced the appointment of Edward Smith to the Board of Directors.

    Edward brings more than 20 years of experience in executive finance and operations leadership in the biotechnology industry to Benitec's Board of Directors. Mr. Smith currently serves as the Chief Financial Officer of Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a clinical-stage pharmaceutical company focused on developing and commercializing innovative therapeutics to treat patients suffering from rare seizure disorders. Prior to the start…

    HAYWARD, Calif., April 14, 2020 /PRNewswire/ -- Benitec Biopharma, Inc., a successor entity to Benitec Biopharma Limited (NASDAQ:BNTC), a development-stage biotechnology company focused on the advancement of novel genetic medicines, today announced the appointment of Edward Smith to the Board of Directors.

    Edward brings more than 20 years of experience in executive finance and operations leadership in the biotechnology industry to Benitec's Board of Directors. Mr. Smith currently serves as the Chief Financial Officer of Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a clinical-stage pharmaceutical company focused on developing and commercializing innovative therapeutics to treat patients suffering from rare seizure disorders. Prior to the start of his tenure at Marinus Pharmaceuticals in 2013, Mr. Smith served as the CFO of PolyMedix, Inc., and before his time at PolyMedix, Inc. he served as the executive director of finance at InKine Pharmaceutical Company, Inc. 

    Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma, Inc. commented on Mr. Smith's appointment, "Edward's extensive background in finance and operations in the biotherapeutics space will make him an exceptional contributor to the Board.  We are excited to welcome him as a director."

    About Benitec Biopharma, Inc.

    Benitec Biopharma, Inc. ("Benitec" or the "Company") is a development-stage biotechnology company focused on the advancement of novel genetic medicines, with headquarters in Hayward, California. The Company's proprietary "Silence and Replace" approach combines DNA-directed RNA interference, or ddRNAi, with gene therapy to create medicines that facilitate sustained silencing of disease-causing genes following a single administration. The Company is developing ddRNAi-based therapeutics for chronic and life-threatening human conditions including oculopharyngeal muscular dystrophy (OPMD), and chronic hepatitis B. A comprehensive overview of the Company can be found on Benitec's website at www.benitec.com.

    Safe Harbor Statement:

    This press release contains "forward-looking statements" within the meaning of section 27A of the US Securities Act of 1933 and section 21E of the US Securities Exchange Act of 1934. Any forward-looking statements that may be in this announcement are subject to risks and uncertainties relating to the difficulties in Benitec's plans to develop and commercialize its product candidates, the timing of the initiation and completion of preclinical and clinical trials, the timing of patient enrolment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec's product candidates, potential future out-licenses and collaborations, the intellectual property position and the ability to procure additional sources of financing. Accordingly, you should not rely on those forward-looking statements as a prediction of actual future results.

    Investor Relations
    M Group Strategic Communications
    Jay Morakis
    Managing Director
    Tel: +1 646 859 5951
    Email:

    Cision View original content:http://www.prnewswire.com/news-releases/benitec-biopharma-appoints-edward-smith-to-the-board-of-directors-301040679.html

    SOURCE Benitec Biopharma Inc.

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  38. RADNOR, Pa., March 23, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company", "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to three new employees. The Compensation Committee of the Board of Directors of Marinus granted the new employees non-qualified stock options to purchase an aggregate of 228,500 shares of its common stock and an aggregate of 21,000 shares of restricted common stock, as inducement awards material to each employee entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    The stock options have an exercise price of $1.44 per share…

    RADNOR, Pa., March 23, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company", "Marinus"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the grant of inducement awards to three new employees. The Compensation Committee of the Board of Directors of Marinus granted the new employees non-qualified stock options to purchase an aggregate of 228,500 shares of its common stock and an aggregate of 21,000 shares of restricted common stock, as inducement awards material to each employee entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

    The stock options have an exercise price of $1.44 per share, which is equal to the closing price of Marinus's common stock on March 17, 2020, the date of grant. The stock options will vest and become exercisable as to 25% of the underlying shares on the one-year anniversary of each recipient's start date, and will vest and become exercisable as to the remaining 75% of the underlying shares in 36 equal monthly installments at the end of each month following such anniversary, subject to the employee's continued employment with Marinus on such vesting dates. The restricted common stock vests 50% on March 17, 2021 and the remaining 50% on March 17, 2022. The stock options and restricted common stock were granted as inducements material to the new employees entering into employment with Marinus in accordance with Nasdaq Listing Rule 5635(c)(4), and are subject to the terms and conditions of the applicable award agreements covering the grants.

    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 is conducting the first ever pivotal trials in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Later this year, the Company intends to initiate a Phase 3 trial in SE and a Phase 2 trial in Tuberous Sclerosis Complex (TSC), respectively. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical development schedule and milestones, the ability to complete enrollment in our clinical trials, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, and potential efficacy and therapeutic potential of our product candidate. Forward-looking statements in this 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, the uncertainties inherent in the conduct of clinical trials, the timing of clinical trials, enrollment in clinical trials, availability of data from clinical trials, expectations for regulatory approvals, the attainment of clinical trial results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, unanticipated costs and expenses, our ability to raise additional capital, the effect of the COVID-19 pandemic on our business and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.

    CONTACT:
      Sasha Damouni Ellis
    Vice President, Investor Relations & Corporate Communications
    Marinus Pharmaceuticals, Inc.
    484-253-6792

    Primary Logo

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  39. RADNOR, Pa., March 19, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat orphan seizure disorders, today announced the appointment of Sasha Damouni Ellis as Vice President, Investor Relations and Corporate Communications, reporting to Scott Braunstein, M.D., Chief Executive Officer of Marinus. In this role, Ms. Damouni Ellis will lead strategic investor relations and communications programs, and will work closely with investors, employees, advocacy groups, patient organizations and the media.

    "As we build on our recent clinical success to strategically develop ganaxolone in orphan seizure disorders, the addition of Sasha to the Marinus…

    RADNOR, Pa., March 19, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat orphan seizure disorders, today announced the appointment of Sasha Damouni Ellis as Vice President, Investor Relations and Corporate Communications, reporting to Scott Braunstein, M.D., Chief Executive Officer of Marinus. In this role, Ms. Damouni Ellis will lead strategic investor relations and communications programs, and will work closely with investors, employees, advocacy groups, patient organizations and the media.

    "As we build on our recent clinical success to strategically develop ganaxolone in orphan seizure disorders, the addition of Sasha to the Marinus team significantly strengthens our capabilities to enhance relationships with key stakeholders," said Dr. Braunstein. "Sasha's diverse communications expertise will be a critical asset as we advance through a number of near-term milestones that include data from our pivotal Phase 3 trial in children with CDKL5 deficiency disorder, our first potential new drug application and additional trial initiations; including a pivotal Phase 3 trial for status epilepticus. We are thrilled to have her on the team and look forward to her leadership in communicating our exciting mission."

    Ms. Damouni Ellis said, "I am excited to be joining the Marinus team, who have shown a profound commitment to providing desperately needed treatments for patients with serious orphan seizure disorders. The data across the company's clinical programs are compelling, and I look forward to playing a key role in communicating our vision and unique approach in advancing ganaxolone across targeted and mechanistically relevant disease states."

    Ms. Damouni Ellis brings close to 20 years of healthcare experience, including financial communications, issues management, and executive visibility to Marinus. Immediately prior to joining the company, Ms. Damouni Ellis was Director of External Communications and Media Relations for Bayer U.S., where she drove external brand-building strategies in therapeutic areas of oncology, hemophilia, cardio-pulmonary, and women's health. Prior to Bayer, she was a Senior Healthcare Journalist at Bloomberg News where she focused on articles related to high profile FDA advisory committee meetings, drugs in development, intellectual property and mergers and acquisitions. Ms. Damouni Ellis launched and was Global Editor of BioPharm Insight, formerly a division of the Financial Times Group, where she managed a group of three analysts and nine journalists in London and New York. She has also held roles at Mergermarket, and a number of leading international newspapers. Ms. Damouni Ellis earned her Master of Arts from City, University of London's Graduate School of Journalism, in the United Kingdom, and her Bachelor of Arts Degree from Richmond, the American International University in London, United Kingdom.

    About Marinus Pharmaceuticals
    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the dedicated to the development of innovative therapeutics to treat orphan seizure disorders. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Based on results from a recent Phase 2 study in refractory SE and from biomarker analysis research, the Company intends to initiate later this year a Phase 3 study in SE and a Phase 2 study in Tuberous Sclerosis Complex (TSC), respectively. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical study testing schedule and milestones, the ability to complete enrollment in our clinical studies, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this 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, the uncertainties inherent in the conduct of future clinical studies, the timing of the clinical studies, enrollment in clinical studies, availability of data from ongoing clinical studies, expectations for regulatory approvals, the attainment of clinical study results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.

    CONTACT: 

    Sasha Damouni Ellis
    Vice President, Investor Relations & Corporate Communications
    Marinus Pharmaceuticals, Inc.
    484-253-6792

    Primary Logo

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  40. RADNOR, Pa., March 16, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided a business update on its clinical development activities and reported its financial results for the year ended December 31, 2019.

    Key 2020 Planned Milestones:

    • Initiate a pivotal Phase 3 clinical trial in patients with status epilepticus (SE) – mid-2020
    • Initiate a Phase 2 clinical trial in patients with tuberous sclerosis complex (TSC) – Q2 2020
    • Report top-line data from a global, pivotal Phase 3 clinical trial in children with CDKL5 deficiency disorder (CDD) – Q3 2020

    "We have started 2020 in a strong position, with recent…

    RADNOR, Pa., March 16, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today provided a business update on its clinical development activities and reported its financial results for the year ended December 31, 2019.

    Key 2020 Planned Milestones:

    • Initiate a pivotal Phase 3 clinical trial in patients with status epilepticus (SE) – mid-2020
    • Initiate a Phase 2 clinical trial in patients with tuberous sclerosis complex (TSC) – Q2 2020
    • Report top-line data from a global, pivotal Phase 3 clinical trial in children with CDKL5 deficiency disorder (CDD) – Q3 2020

    "We have started 2020 in a strong position, with recent accomplishments in the clinic that support our strategy to develop ganaxolone in mechanistically relevant disease states where we have the potential to significantly improve patient outcomes," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "We have recently announced enrollment completion for our Phase 3 trial in CDD and remain on track for topline data later this year. Preparations are underway for our first potential NDA filing and commercial launch with oral ganaxolone. Our team continues to build momentum in the status epilepticus program and we are on track for the initiation later this year of our pivotal Phase 3 trial. Taken together, we believe we have developed thoughtful, data-driven clinical programs designed to provide new treatments to patients suffering from severe and rare seizure disorders."

    Clinical Development Overview and Highlights
    Marinus is developing oral and intravenous (IV) ganaxolone formulations to treat adults and children suffering from acute and chronic rare seizure disorders where there is a mechanistic rationale for ganaxolone to provide a therapeutic benefit to patients with unmet or underserved medical needs. 

    Status Epilepticus (SE)

    • Marinus is making preparations for an End of Phase 2 meeting with the U.S. FDA in Q1 2020, with the goal of commencing a Phase 3 registration study by the middle of 2020.
    • In September 2019, the Company announced positive top‑line results in its open‑label, dose‑finding Phase 2 trial evaluating IV ganaxolone in patients with refractory SE. Ganaxolone met the primary endpoint in the trial with 100% of  patients (n=17) not progressing to IV anesthetics within 24 hours of treatment initiation. Additionally, in the target dose group (713 mg/day), no patients (n=8) progressed to additional IV anti-epileptic drugs (AEDs) or IV anesthetics for status relapse at any time through the end of the 24-hour post treatment follow‑up period. Ganaxolone had an acceptable safety and tolerability profile for the refractory SE patient population in all dose groups.
    • Additional long‑term data presented at the American Epilepsy Society (AES) Annual Meeting demonstrated that all patients in the target dose cohort did not experience status relapse during the four‑week follow up period (n=6). An independent retrospective central review of seizure EEG data using objective criteria demonstrated a clear dose effect with the target dose level providing sustained reductions in seizure burden (greater than 80%) throughout the entire analysis window.

    CDKL5 Deficiency Disorder (CDD)

    • Enrollment has been completed in the pivotal Phase 3 clinical trial (Marigold Study) evaluating the use of oral ganaxolone in children and young adults with CDD. The Marigold Study is a global, double‑blind, placebo‑controlled, clinical trial that has enrolled approximately 100 patients between the ages of 2 and 21 with a confirmed disease‑related CDKL5 gene variant and allopregnanolone sulfate levels below a pre‑specified limit.
    • The trial's primary efficacy endpoint is percent change in 28‑day seizure frequency. Secondary outcome measures will include non‑seizure‑related endpoints to capture certain changes in behavior and sleep.
    • The Company plans to announce top‑line data from the trial in Q3 2020. If the Phase 3 Marigold Study is successful, this could be the first approved indication for ganaxolone and the first approved treatment for CDD.

    PCDH19-related Epilepsy (PCDH19-RE)

    • Enrollment is ongoing in a pivotal Phase 3 clinical trial (Violet Study) evaluating the use of oral ganaxolone in children and young adults with PCDH19‑RE. The Violet Study will enroll up to 70 patients between the ages of 1 and 17 with a confirmed PCDH19 mutation. Patients enrolled in the trial are stratified into one of two biomarker groups based on baseline allopregnanolone sulfate levels and randomized (ganaxolone or placebo) within each stratum. The trial consists of a 12‑week prospective baseline period to collect seizure data, followed by a 17‑week double‑blind treatment phase. Patients randomized to ganaxolone are titrated over four weeks to a dose of up to 600 mg of oral liquid suspension three times a day and maintain that dose for the following 13‑weeks. After the double‑blind period, all patients who meet certain eligibility criteria will have the opportunity to receive ganaxolone in an open label phase of the trial.
    • The Company plans to announce top‑line data from the Violet Study in 2H 2021.

    Tuberous Sclerosis Complex (TSC)

    • Marinus is planning to initiate a Phase 2 open‑label trial to evaluate the safety and tolerability of adjunctive ganaxolone treatment in patients with TSC in 2Q 2020. 
    • The trial will be conducted at approximately 6 sites in the U.S. and enroll approximately 30 patients ages 2 to 65. Patients will undergo a four‑week baseline period followed by a 12‑week treatment period. All patients who meet certain eligibility criteria will have the opportunity to receive ganaxolone in a 24-week extension of the trial. The primary endpoint for the trial is percent change in 28‑day primary seizure frequency through the end of the 12‑week treatment period relative to the 4‑week baseline period.
    • Efficacy results will be evaluated for correlation to baseline endogenous allopregnanolone sulfate levels.

    Financial Update
    At December 31, 2019, we had cash and cash equivalents of $90.9 million compared to $67.7 million at December 31, 2018. We believe that our cash, cash equivalents and investments as of December 31, 2019 will enable us to fund our operating expenses and capital expenditure requirements into the third quarter of 2021.

    Research and development expenses increased to $43.0 million for the year ended December 31, 2019, as compared to $28.4 million in the year ended December 31, 2018.  The primary drivers for the increase to our research and development expenditures were clinical and manufacturing activities in support of our Phase 3 trials in CDD and PCDH19-RE, partially offset by decreased costs for non-seizure disorder indications.

    General and administrative expenses were $11.5 million for the year ended December 31, 2019 as compared to $8.8 million in the prior year.  The primary drivers of this increase were $1.3 million in severance expenses related to the departure of former executive officers ($0.4 million of which was non-cash equity compensation expense), and approximately $1.2 million in professional fees and other costs associated with an increased scale of operations.

    The Company reported a net loss of $54.1 million for the year ended December 31, 2019, compared to $36.7 million for the year ended December 31, 2018. Cash used in operating activities increased to $48.6 million for the year ended December 31, 2019 compared to $27.8 million for the year ended December 31, 2018.

    Readers are referred to, and encouraged to read in its entirety, the Company's Annual Report on Form 10-K for the quarter ended December 31, 2019 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.

    Marinus Pharmaceuticals, Inc.
    Selected Financial Data (in thousands, except share and per share amounts)
    (unaudited)

                   
        December 31,  
        2019   2018  
                   
    Assets:              
    Cash and cash equivalents   $ 90,943   $ 67,727  
    Investments     739     4,998  
    Other assets     7,160     2,509  
    Total assets   $ 98,842   $ 75,234  
    Liabilities and stockholders' equity:              
    Accounts payable   $ 2,763   $ 2,472  
    Accrued expenses     5,268     4,437  
    Other liabilities     3,042      
    Total liabilities     11,073     6,909  
    Series A convertible preferred stock     28,200      
    Total stockholders' equity     59,569     68,325  
    Total liabilities and stockholders' equity   $ 98,842   $ 75,234  


                   
                   
        Year Ended December 31,  
        2019   2018  
                   
    Operations:              
    Research and development   $ 42,966     $ 28,394    
    General and administrative     11,456       8,785    
    Loss from operations     (54,422 )     (37,179 )  
    Interest income     354       454    
    Other expense     (53 )     (1 )  
    Net loss   $ (54,121 )   $ (36,726 )  
    Per share information:              
    Net loss per share of common stock—basic and diluted   $ (0.99 )   $ (0.90 )  
    Basic and diluted weighted average shares outstanding     54,512,778       40,895,406    





















    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 is conducting the first ever pivotal trials in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Later this year, the company intends to initiate a Phase 3 trial in SE and a Phase 2 trial in Tuberous Sclerosis Complex (TSC).For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical development schedule and milestones, the ability to complete enrollment in our clinical studies, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this 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, the uncertainties inherent in the conduct of clinical trials, the timing of clinical trials, enrollment in clinical trials, availability of data from clinical trials, expectations for regulatory approvals, the attainment of clinical trial results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, unanticipated costs and expenses, our ability to raise additional capital and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.

    CONTACT: 
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

    Primary Logo

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  41. RADNOR, Pa., Feb. 25, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced it has reached the 100 patient enrollment target for the Marigold Study.  The Marigold Study is a pivotal Phase 3 study evaluating oral ganaxolone in children and young adults with CDKL5 Deficiency Disorder (CDD), a rare refractory form of pediatric epilepsy with no currently approved treatments.  The Company will continue enrollment through the end of the month to capture the remaining few patients in the screening phase.  Marinus is on-track to report top-line results in the third quarter of 2020.

    "Enrolling…

    RADNOR, Pa., Feb. 25, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced it has reached the 100 patient enrollment target for the Marigold Study.  The Marigold Study is a pivotal Phase 3 study evaluating oral ganaxolone in children and young adults with CDKL5 Deficiency Disorder (CDD), a rare refractory form of pediatric epilepsy with no currently approved treatments.  The Company will continue enrollment through the end of the month to capture the remaining few patients in the screening phase.  Marinus is on-track to report top-line results in the third quarter of 2020.

    "Enrolling 100 patients in our registrational, pivotal Phase 3 trial evaluating ganaxolone in children with CDD is a significant milestone for both Marinus and the CDD community," said Joe Hulihan, M.D., Chief Medical Officer of Marinus. "Our ability to identify and rapidly enroll qualified patients into this study is indicative of the significant need for a new therapy to reduce seizure burden and improve patient outcomes. The study has been well conducted and we are encouraged by the limited adverse events, low dropout rates and the vast majority of patients entering the open-label extension. We are appreciative of the patients and broader CDD community for their participation in the Marigold study and we remain committed to addressing the unmet need of these patients who currently have no approved treatment options."

    Karen Utley, President of the International Foundation for CDKL5 Research, commented, "We are grateful for Marinus' recognition of the tremendous need of children with CDD and its dedication to advancing the development of ganaxolone as a potential new treatment for these children through the Marigold study. We are proud of our collaborative partnership to lead the way in finding a cure and treatments for children with CDD and are hopeful that ganaxolone may be a novel treatment option that improves outcomes for our children."

    The Marigold Study is a global, double-blind, placebo-controlled, Phase 3 clinical trial designed to enroll approximately 100 patients between the ages of 2 and 21 with a confirmed disease-related CDKL5 gene variant.  Patients undergo a baseline period before being randomized to receive either ganaxolone (up to 1,800 mg/day) or placebo for 17 weeks in addition to their existing anti-seizure treatment. Following the treatment period, all patients that meet certain eligibility requirements will have the opportunity to receive ganaxolone in the open label phase of the study.  The study's primary efficacy endpoint is percent reduction in seizures.  Secondary outcome measures include non-seizure-related endpoints to capture certain changes in behavioral and sleep disturbances that were seen as improvements in previous clinical studies with ganaxolone.

    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, and many are confined to wheelchairs, dependent on others for everything. Currently, there are no approved therapies 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 anti-seizure and anti-anxiety activity via its effects on synaptic and extrasynaptic GABAA receptors.  Ganaxolone has been studied in more than 1,600 subjects, both pediatric and adult, at therapeutically relevant dose levels and treatment regimens for up to four years. In these studies, ganaxolone was generally safe and well-tolerated. The most commonly reported adverse events were somnolence, dizziness and fatigue.

    About Marinus Pharmaceuticals
    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Based on results from a recent Phase 2 study in refractory SE and from biomarker analysis research, the Company intends to initiate later this year a Phase 3 study in SE and a Phase 2 study in Tuberous Sclerosis Complex (TSC), respectively. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical study testing schedule and milestones, the ability to complete enrollment in our clinical studies, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this 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, the uncertainties inherent in the conduct of future clinical studies, the timing of the clinical studies, enrollment in clinical studies, availability of data from ongoing clinical studies, expectations for regulatory approvals, the attainment of clinical study results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.

    CONTACT: 
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

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  42. RADNOR, Pa., Feb. 24, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced that management will present a corporate overview at the SVB Leerink 9th Annual Global Healthcare Conference, the Cowen and Company 40th Annual Healthcare Conference, and the Oppenheimer 30th Annual Healthcare Conference.

    SVB Leerink 9th Annual Global Healthcare Conference
    Date: Tuesday, February 25
    Time:  11:00am Eastern Time
    Presenter: Dr. Scott Braunstein, Chief Executive Officer
    Format: Fireside Chat
       
    Cowen & Company 40th Annual Healthcare Conference
    Date:  Monday, March 2
    Time:  4:10pm Eastern Time

    RADNOR, Pa., Feb. 24, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced that management will present a corporate overview at the SVB Leerink 9th Annual Global Healthcare Conference, the Cowen and Company 40th Annual Healthcare Conference, and the Oppenheimer 30th Annual Healthcare Conference.

    SVB Leerink 9th Annual Global Healthcare Conference
    Date: Tuesday, February 25
    Time:  11:00am Eastern Time
    Presenter: Dr. Scott Braunstein, Chief Executive Officer
    Format: Fireside Chat
       
    Cowen & Company 40th Annual Healthcare Conference
    Date:  Monday, March 2
    Time:  4:10pm Eastern Time
    Location: Boston Marriott Copley Place
    Presenter: Ed Smith, Chief Financial Officer
       
    Oppenheimer 30th Annual Healthcare Conference
    Date: Tuesday, March 17
    Time: 8:35am Eastern Time
    Location: InterContinental Barclay Hotel in New York City
    Presenter: Dr. Scott Braunstein, Chief Executive Officer

    A live webcast of the Cowen and Oppenheimer presentations may be accessed on the "Investors" tab of the company's website, www.marinuspharma.com.  An archived version of the presentations will be available for 30 days.

    About Marinus Pharmaceuticals
    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Based on results from a recent Phase 2 study in refractory SE and from biomarker analysis research, the Company intends to initiate later this year a Phase 3 study in SE and a Phase 2 study in Tuberous Sclerosis Complex (TSC), respectively. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

               
    CONTACT:   
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

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  43. RADNOR, Pa., Feb. 14, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company", "Marinus"), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced that the Board of Directors of Marinus granted non-qualified stock options to purchase an aggregate of 60,000 shares of its common stock to new employees, with a grant date of February 5, 2020.

    The options have an exercise price of $2.13 per share, which is equal to the closing price of Marinus's common stock on February 5, 2020, the date of grant. The options will vest and become exercisable as to 25 percent of the shares on the one-year anniversary of the recipient's start…

    RADNOR, Pa., Feb. 14, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company", "Marinus"), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced that the Board of Directors of Marinus granted non-qualified stock options to purchase an aggregate of 60,000 shares of its common stock to new employees, with a grant date of February 5, 2020.

    The options have an exercise price of $2.13 per share, which is equal to the closing price of Marinus's common stock on February 5, 2020, the date of grant. The options will vest and become exercisable as to 25 percent of the shares on the one-year anniversary of the recipient's start date, and will vest and become exercisable as to the remaining 75 percent of the shares in 36 equal monthly installments at the end of each month following the anniversary, subject to the employee's continued employment with Marinus on such vesting dates.  The stock options were granted as inducements material to the new employees entering into employment with Marinus in accordance with NASDAQ Listing Rule 5635(c)(4), and are subject to the terms and conditions of a stock option agreement covering the grant.

    About Marinus Pharmaceuticals

    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Based on results from a recent Phase 2 study in refractory SE and from biomarker analysis research, the Company intends to initiate later this year a Phase 3 study in SE and a Phase 2 study in Tuberous Sclerosis Complex (TSC), respectively. 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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical study testing schedule and milestones, the ability to complete enrollment in our clinical studies, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this 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, the uncertainties inherent in the conduct of future clinical studies, the timing of the clinical studies, enrollment in clinical studies, availability of data from ongoing clinical studies, expectations for regulatory approvals, the attainment of clinical study results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.          
               
    CONTACT:   
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

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  44. RADNOR, Pa., Feb. 03, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced the upcoming presentation of EEG data from its Phase 2 study at the American Clinical Neurophysiology Society (ACNS) 2020 Annual Meeting in New Orleans, Louisiana on February 7, 2020. The data from the study demonstrate a rapid, dose-dependent and sustained reduction of seizure burden in patients with status epilepticus (SE) treated with ganaxolone. In addition, an independent central review of EEG seizure data provided objective diagnostic confirmation in these patients of a clear dose effect, with the target…

    RADNOR, Pa., Feb. 03, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced the upcoming presentation of EEG data from its Phase 2 study at the American Clinical Neurophysiology Society (ACNS) 2020 Annual Meeting in New Orleans, Louisiana on February 7, 2020. The data from the study demonstrate a rapid, dose-dependent and sustained reduction of seizure burden in patients with status epilepticus (SE) treated with ganaxolone. In addition, an independent central review of EEG seizure data provided objective diagnostic confirmation in these patients of a clear dose effect, with the target dose level providing sustained reductions in seizure burden (greater than 80%) throughout the entire analysis window.

    Presentation details:    
         
    Title:   "Intravenous Ganaxolone Achieves Rapid and Dose-Dependent Sustained Improvement in EEG Seizure Burden in Patients with Refractory Status Epilepticus
    Authors:   A. M. Husain, et al.
    Abstract:   # 793677. Poster Number F53
    Date:   Friday, February 7
    Time:   7:30 pm to 9:00 pm Central Time


    About Marinus Pharmaceuticals

    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Based on results from a recent Phase 2 study in refractory SE and from biomarker analysis research, the Company intends to initiate later this year a Phase 3 study in SE and a Phase 2 study in Tuberous Sclerosis Complex (TSC), respectively. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical study testing schedule and milestones, the ability to complete enrollment in our clinical studies, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this 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, the uncertainties inherent in the conduct of future clinical studies, the timing of the clinical studies, enrollment in clinical studies, availability of data from ongoing clinical studies, expectations for regulatory approvals, the attainment of clinical study results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.

    CONTACT:
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

     

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  45. Enrollment on-track for CDKL5 Deficiency Disorder Phase 3 readout Q3 2020

    Two additional late-stage clinical trials planned to initiate in 2020

    Company presentation and live webcast at 38th Annual J.P. Morgan Global Healthcare Conference on Thursday, January 16, 2020 at 11:00am PT

    RADNOR, Pa., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) ("Marinus" or "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and other neuropsychiatric disorders, today provided a corporate update for investors, including its roadmap for advancing ganaxolone in 2020.

    Key 2020 Milestones:

    • Initiate Phase 2 clinical trial in patients with Tuberous Sclerosis Complex (TSC) – 1H…

    Enrollment on-track for CDKL5 Deficiency Disorder Phase 3 readout Q3 2020

    Two additional late-stage clinical trials planned to initiate in 2020

    Company presentation and live webcast at 38th Annual J.P. Morgan Global Healthcare Conference on Thursday, January 16, 2020 at 11:00am PT

    RADNOR, Pa., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) ("Marinus" or "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and other neuropsychiatric disorders, today provided a corporate update for investors, including its roadmap for advancing ganaxolone in 2020.

    Key 2020 Milestones:

    • Initiate Phase 2 clinical trial in patients with Tuberous Sclerosis Complex (TSC) – 1H 2020
    • Initiate U.S. pivotal Phase 3 clinical trial in patients with Status Epilepticus (SE) – mid-2020
    • Report top-line data from global, pivotal Phase 3 clinical trial in children with CDKL5 Deficiency Disorder (CDD) – Q3 2020

    "Our 2019 clinical progress, along with the bolstering of our executive leadership and balance sheet, now positions us to begin to unlock the value of our ganaxolone franchise," said Scott Braunstein, M.D., Chief Executive Officer of Marinus. "Our strategy is focused on mechanistically relevant disease states for ganaxolone that have the potential to significantly improve outcomes for patients. With our Phase 3 study reading out in CDKL5 deficiency disorder later this year, we are making preparations for our first NDA filing and commercial launch with oral ganaxolone.  In addition, we are advancing our hospital-directed intravenous dose form into a Phase 3, potentially NDA enabling, study in status epilepticus later this year."

    Clinical Development Overview
    Marinus is developing an oral and intravenous (IV) formulation of ganaxolone to treat adults and children suffering from acute and chronic rare neuropsychiatric conditions where there is a mechanistic rationale for ganaxolone to provide a benefit. Unlike benzodiazepines, ganaxolone exhibits anti-seizure, anti-anxiety, and antidepressant activity via its effects on synaptic and extrasynaptic GABAA receptors. 

    Status Epilepticus (SE)
    Following the successfully completed phase 2 study in patients with refractory SE, Marinus is preparing for an End-of-Phase 2 meeting with the FDA, which is expected in the first quarter of 2020.  Status epilepticus is a rare condition consisting of a prolonged state of continuous or near-continuous seizure activity that can cause permanent damage to the brain and even death if not quickly brought under control.  The Phase 2 dose-finding trial enrolled 17 medically heterogeneous patients who received an infusion of IV ganaxolone, added to second line standard of care antiepileptic drugs for the treatment of SE.  In the study, 100% of patients achieved the primary endpoint of preventing progression to IV anesthetics within 24 hours of treatment initiation. The median time to SE cessation was five minutes across all doses evaluated and a numerical dose response trend was observed in evaluable patients at clinically relevant early timepoints and the four-week long-term follow-up visit. Every patient receiving the identified target dose (713 mg/day) met the primary endpoint and did not have recurrence of SE up to the long-term follow-up visit.  This target dose will be further evaluated in a pivotal study that is planned to commence in mid-2020.  Additionally, the Company plans to request scientific advice later this year to discuss the approval pathway for ganaxolone in Europe.

    CDKL5 Deficiency Disorder (CDD)
    Marinus is currently in the final stages of recruitment in the Marigold Study, its pivotal Phase 3 study evaluating oral ganaxolone in children and young adults with CDD, a rare refractory form of pediatric epilepsy with no currently approved treatments. The global, double-blind, placebo-controlled, pivotal study will enroll approximately 100 patients between the ages of 2 and 21 with a confirmed disease-related CDKL5 gene variant. The Company remains on-track to report top-line data in Q3 2020. If the Phase 3 CDD study is successful, this could be the first approved indication for ganaxolone and the first approved treatment for CDD. Marinus has received orphan drug designation from both the US FDA and The European Medicines Agency (EMA) for ganaxolone for the treatment of CDD.

    PCDH19-related Epilepsy (PCDH19-RE)

    International site initiation and enrollment is continuing in the Violet Study, a biomarker stratified pivotal Phase 3 study evaluating oral ganaxolone in children with PCDH19-RE.  PCDH19-RE is a serious and rare epileptic disease characterized by highly variable early-onset cluster seizures with comorbid cognitive and behavioral disturbances with or without intellectual disability. Currently, there are no approved therapies for PCDH19-RE. The Violet Study will enroll up to 70 patients between the ages of 1 and 17 with a confirmed PCDH19 mutation.  Patients are stratified into biomarker positive and negative groups for Allo-S, which could potentially provide the epilepsy community with the first diagnostic blood test that predicts the likelihood of a treatment response. Top-line data from the Violet study are expected in 2021.

    Tuberous Sclerosis Complex (TSC)
    Marinus is planning to initiate a Phase 2, open label study to evaluate the safety and tolerability of adjunctive ganaxolone treatment in patients with seizures associated with TSC. TSC, a leading cause of genetic epilepsy, is a rare genetic disorder that affects many organs and causes non-malignant tumors in the brain, skin, kidney, heart, eyes, and lungs. The decision to expand the ganaxolone epilepsy program in TSC was strategically informed by the discovery of a potential new epilepsy biomarker, Allo-S, in the Phase 2 study in PCDH19-RE. This led to additional analyses that identified TSC as another rare genetic disorder that may be similarly impacted by Allo-S levels.

    The planned Phase 2 study will be conducted at approximately 4-6 sites in the United States and enroll 20-40 patients ages 2 to 65.  The primary endpoint for the study is percent change in 28-day primary seizure frequency through the end of the 12-week treatment period relative to the 4-week baseline period.

    Dr. Joe Hulihan, Chief Medical Officer stated, "Our clinical momentum in 2019 has further increased our confidence that ganaxolone has the potential to transform the treatment paradigm for a number of severe and life-threatening epilepsies with no currently approved treatments. We are proud of our progress and remain committed to the rapid advancement of ganaxolone for patients in need."

    Presentation at 38th Annual J.P. Morgan Global Healthcare Conference
    Dr. Scott Braunstein will present a corporate overview at the 38th Annual J.P. Morgan Global Healthcare Conference on Thursday, January 16, 2020 at 11:00am Pacific Time. The conference will take place at the Westin St. Francis Hotel in San Francisco, California. A live webcast of J.P. Morgan 2020 Global Healthcare presentation may be accessed on the "Investors" tab of the company's website, www.marinuspharma.com.  An archived version of the presentation will be available for 30 days.

    About Marinus Pharmaceuticals
    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Based on results from a recent Phase 2 study in refractory SE and from biomarker analysis research, the Company intends to initiate later this year a Phase 3 study in SE and a Phase 2 study in Tubular Sclerosis Complex (TSC), respectively. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical study testing schedule and milestones, the ability to complete enrollment in our clinical studies, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this 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, the uncertainties inherent in the conduct of future clinical studies, the timing of the clinical studies, enrollment in clinical studies, availability of data from ongoing clinical studies, expectations for regulatory approvals, the attainment of clinical study results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.          
               
    CONTACT:   
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674
     

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  46. RADNOR, Pa., Jan. 07, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), (NASDAQ:MRNS) ("Marinus" or "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and other neuropsychiatric disorders, today announced that Dr. Scott Braunstein, Chief Executive Officer, will present a corporate overview at the 38th Annual J.P. Morgan Global Healthcare Conference on Thursday, January 16, 2020 at 11:00am Pacific Time. The conference will take place at the Westin St. Francis Hotel in San Francisco, California.

    A live webcast of J.P. Morgan 2020 Global Healthcare presentation may be accessed on the "Investors" tab of the company's website, www.marinuspharma.com.  An archived version…

    RADNOR, Pa., Jan. 07, 2020 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), (NASDAQ:MRNS) ("Marinus" or "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and other neuropsychiatric disorders, today announced that Dr. Scott Braunstein, Chief Executive Officer, will present a corporate overview at the 38th Annual J.P. Morgan Global Healthcare Conference on Thursday, January 16, 2020 at 11:00am Pacific Time. The conference will take place at the Westin St. Francis Hotel in San Francisco, California.

    A live webcast of J.P. Morgan 2020 Global Healthcare presentation may be accessed on the "Investors" tab of the company's website, www.marinuspharma.com.  An archived version of the presentation will be available for 30 days.

    About Marinus Pharmaceuticals
    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy. Based on results from a recent Phase 2 study in refractory SE and from biomarker analysis research, the Company intends to initiate later this year a Phase 3 study in SE and a Phase 2 study in Tubular Sclerosis Complex (TSC), respectively. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.  

    CONTACT:   
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

    Primary Logo

    View Full Article Hide Full Article
  47. RADNOR, Pa., Dec. 13, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company", "Marinus"), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced that the Board of Directors of Marinus granted non-qualified stock options to purchase an aggregate of 121,000 shares of its common stock to new employees, with a grant date of December 11, 2019.

    The options have an exercise price of $1.78 per share, which is equal to the closing price of Marinus's common stock on December 11, 2019, the date of grant. The options will vest and become exercisable as to 25 percent of the shares on the one-year anniversary of the recipient's start…

    RADNOR, Pa., Dec. 13, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) (the "Company", "Marinus"), a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced that the Board of Directors of Marinus granted non-qualified stock options to purchase an aggregate of 121,000 shares of its common stock to new employees, with a grant date of December 11, 2019.

    The options have an exercise price of $1.78 per share, which is equal to the closing price of Marinus's common stock on December 11, 2019, the date of grant. The options will vest and become exercisable as to 25 percent of the shares on the one-year anniversary of the recipient's start date, and will vest and become exercisable as to the remaining 75 percent of the shares in 36 equal monthly installments at the end of each month following the anniversary, subject to the employee's continued employment with Marinus on such vesting dates.  The stock options were granted as inducements material to the new employees entering into employment with Marinus in accordance with NASDAQ Listing Rule 5635(c)(4), and are subject to the terms and conditions of a stock option agreement covering the grant.

    About Marinus Pharmaceuticals

    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy and has recently released top-line data from Phase 2 studies in women with postpartum depression and patients with refractory status epilepticus. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical study testing schedule and milestones, the ability to complete enrollment in our clinical studies, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this 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, the uncertainties inherent in the conduct of future clinical studies, the timing of the clinical studies, enrollment in clinical studies, availability of data from ongoing clinical studies, expectations for regulatory approvals, the attainment of clinical study results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.          

    CONTACT:   
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

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  48. RADNOR, Pa., Dec. 13, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), ("Marinus" or the "Company") today announced the closing of an underwritten public offering of 32,200,000 shares of common stock at a public offering price of $1.25 per share, including the exercise in full by the underwriters of their option to purchase up to 4,200,000 additional shares of common stock. The offering included participation from new and existing investors, and together with the concurrent private placement raised gross proceeds of approximately $70 million, before deducting underwriting discounts and commissions, placement agent fees and estimated offering expenses payable by the Company.

    Oppenheimer & Co. Inc. and Ladenburg Thalmann…

    RADNOR, Pa., Dec. 13, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), ("Marinus" or the "Company") today announced the closing of an underwritten public offering of 32,200,000 shares of common stock at a public offering price of $1.25 per share, including the exercise in full by the underwriters of their option to purchase up to 4,200,000 additional shares of common stock. The offering included participation from new and existing investors, and together with the concurrent private placement raised gross proceeds of approximately $70 million, before deducting underwriting discounts and commissions, placement agent fees and estimated offering expenses payable by the Company.

    Oppenheimer & Co. Inc. and Ladenburg Thalmann & Co. Inc., a subsidiary of Ladenburg Thalmann Financial Services Inc., (NYSE:LTS), acted as joint bookrunning managers for the offering. Craig-Hallum Capital Group LLC acted as co-manager for the offering.

    Concurrently with the completion of the public offering, the Company sold to certain investors in a private placement, a number of shares of convertible preferred stock equal to an aggregate purchase price of up to $30 million, at a conversion price of $1.25 per share. Marinus intends to use the net proceeds of this offering to advance the clinical development of ganaxolone, including trials for its rare seizure disorder programs and for working capital and general corporate purposes.

    The public offering was made pursuant to an effective shelf registration statement on Form S-3 (No. 333-221243) previously filed with the U.S. Securities and Exchange Commission (the "SEC") and declared effective by the SEC on December 1, 2017. A final prospectus supplement and accompanying prospectus relating to the public offering were filed with the SEC and are available on the SEC's website located at http://www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying prospectus may be obtained by contacting Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, NY, 10004, by telephone at (212) 667-8055, or by email at ; or from Ladenburg Thalmann & Co. Inc., Prospectus Department, 277 Park Avenue, 26th Floor, New York, New York 10172 or by email at .

    The shares of convertible preferred stock sold in the concurrent private placement have not been registered under the Securities Act or under any state securities laws and, unless so registered, may not be offered or sold in the United States except pursuant to an exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and applicable state securities laws. Marinus has agreed to file a resale registration statement with the SEC for purposes of registering the resale of the shares of common stock issuable upon conversion of the preferred shares issued in the concurrent private placement.

    This press release does not constitute an offer to sell or the solicitation of offers to buy any securities of Marinus being offered, and shall not constitute an offer, solicitation or sale of any security in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Marinus Pharmaceuticals

    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy and has recently released top-line data from Phase 2 studies in women with postpartum depression and patients with refractory status epilepticus.

    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 intended use of proceeds from the public offering and concurrent private placement. Forward-looking statements in this release involve substantial risks and uncertainties. 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.

    CONTACT:
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

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  49. RADNOR, Pa., Dec. 12, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) ("Marinus" or "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy, depression and other neuropsychiatric disorders, announced today that Elan Ezickson, has joined the Company's Board of Directors.  Mr. Ezickson is a highly accomplished executive with over 30 years of experience in the life sciences industry across commercial, operational and strategic leadership roles.

    "We are thrilled to welcome Elan to our board of directors," said Scott Braunstein, M.D., Chief Executive Officer of Marinus.  "His diverse leadership experience across the entire value chain of pharmaceutical operations, from…

    RADNOR, Pa., Dec. 12, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS) ("Marinus" or "Company"), a pharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy, depression and other neuropsychiatric disorders, announced today that Elan Ezickson, has joined the Company's Board of Directors.  Mr. Ezickson is a highly accomplished executive with over 30 years of experience in the life sciences industry across commercial, operational and strategic leadership roles.

    "We are thrilled to welcome Elan to our board of directors," said Scott Braunstein, M.D., Chief Executive Officer of Marinus.  "His diverse leadership experience across the entire value chain of pharmaceutical operations, from capital raising to program development, will be invaluable to Marinus as we continue to execute on the clinical development of ganaxolone across multiple registrational Phase 3 studies. We look forward to leveraging his valuable insight as we prepare for significant regulatory and clinical milestones in 2020."

    Mr. Ezickson brings extensive biopharmaceutical operational, strategic and capital formation expertise to Marinus's board, most recently having served as the Chief Operating Officer and head of corporate development at Scholar Rock Holding Corporation, a clinical stage biotechnology company focused on novel oncology and rare disease treatments.  At Scholar Rock, Mr. Ezickson directed corporate development, operations and strategy, and played a significant role in corporate finance, leading the company's initial public offering.  Prior to joining Scholar Rock, Mr. Ezickson served as Executive Vice President and Chief Operating Officer of Aveo Pharmaceuticals where he built and oversaw corporate development, commercial operations, technical operations, medical affairs, program management and legal functions and led capital raising and licensing efforts with multinational pharmaceutical companies.  Prior to Aveo, he was at Biogen in roles that included President of Biogen Canada, Program Executive and Associate General Counsel.  Mr. Ezickson holds a Bachelor of Arts degree in political science from Yale University and a Juris Doctorate from the Columbia University School of Law.

    Mr. Ezickson also currently serves on the Boards of Directors of three clinical stage biotechnology firms including Carmine Therapeutics, Tvardi Therapeutics and Ziopharm Oncology.

    About Marinus Pharmaceuticals
    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy and has recently released top-line data from Phase 2 studies in women with postpartum depression and patients with refractory status epilepticus. For more information visit www.marinuspharma.com. Please follow us on Twitter: @MarinusPharma.

    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 interpretation of preclinical studies, development plans for our product candidate, including the development of dose forms, the clinical study testing schedule and milestones, the ability to complete enrollment in our clinical studies, interpretation of scientific basis for ganaxolone use, timing for availability and release of data, the safety, potential efficacy and therapeutic potential of our product candidate and our expectation regarding the sufficiency of our working capital. Forward-looking statements in this 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, the uncertainties inherent in the conduct of future clinical studies, the timing of the clinical studies, enrollment in clinical studies, availability of data from ongoing clinical studies, expectations for regulatory approvals, the attainment of clinical study results that will be supportive of regulatory approvals, and other matters, including the development of formulations of ganaxolone, and the availability or potential availability of alternative products or treatments for conditions targeted by the Company that could affect the availability or commercial potential of our drug candidates. 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.

        
    CONTACT: 

    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

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  50. RADNOR, Pa., Dec. 11, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), ("Marinus" or the "Company") today announced the pricing of an underwritten public offering of 28,000,000 shares of common stock at a public offering price of $1.25 per share. The gross proceeds to the Company from the public offering and concurrent private placement are expected to be approximately $65 million before deducting underwriting discounts and commissions, placement agent fees, and other estimated offering expenses. The Company has granted to the underwriters participating in the offering a 30-day option to purchase up to an additional 4,200,000 shares of common stock. The offering is expected to close on or about December 13, 2019, subject…

    RADNOR, Pa., Dec. 11, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), ("Marinus" or the "Company") today announced the pricing of an underwritten public offering of 28,000,000 shares of common stock at a public offering price of $1.25 per share. The gross proceeds to the Company from the public offering and concurrent private placement are expected to be approximately $65 million before deducting underwriting discounts and commissions, placement agent fees, and other estimated offering expenses. The Company has granted to the underwriters participating in the offering a 30-day option to purchase up to an additional 4,200,000 shares of common stock. The offering is expected to close on or about December 13, 2019, subject to customary closing conditions.  

    Oppenheimer & Co. Inc. and Ladenburg Thalmann & Co. Inc., a subsidiary of Ladenburg Thalmann Financial Services Inc., (NYSE:LTS), acted as joint bookrunning managers for the offering. H.C. Wainwright & Co., LLC acted as co-manager for the offering.

    Concurrently, with the completion of the public offering, the Company has agreed to sell to certain investors in a private placement, a number of shares of convertible preferred stock equal to an aggregate purchase price of up to $30 million, at a conversion price of $1.25 per share, which is at least equal to the lower of (i) the closing price per share of our common stock as of the close of the trading day immediately prior to the execution of the purchase agreement; or (ii) the average closing price per share of our common stock for the five trading days immediately prior to the execution of the purchase agreement.

    Marinus intends to use the net proceeds received from the sale of its common and preferred stock to advance the preclinical and clinical development of ganaxolone, including expenses for clinical trials for our seizure disorder programs, and regulatory, research and development, pre-commercial, general and administrative and manufacturing expenses and for working capital and general corporate purposes. 

    The public offering is being made pursuant to an effective shelf registration statement on Form S-3 (No. 333-221243) previously filed with the U.S. Securities and Exchange Commission (the "SEC") and declared effective by the SEC on December 1, 2017. A final prospectus supplement and accompanying prospectus relating to the public offering will be filed with the SEC and will be available on the SEC's website located at http://www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying prospectus, when available, may be obtained by contacting Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, NY, 10004, by telephone at (212) 667-8055, or by email at ; or from Ladenburg Thalmann & Co. Inc., Prospectus Department, 277 Park Avenue, 26th Floor, New York, New York 10172 or by email at .

    The shares of convertible preferred stock to be sold in the concurrent private placement have not been registered under the Securities Act or under any state securities laws and, unless so registered, may not be offered or sold in the United States except pursuant to an exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and applicable state securities laws. Marinus has agreed to file a resale registration statement with the SEC for purposes of registering the resale of the shares of common stock issuable upon conversion of the preferred shares issued in the concurrent private placement.

    This press release does not constitute an offer to sell or the solicitation of offers to buy any securities of Marinus being offered, and shall not constitute an offer, solicitation or sale of any security in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Marinus Pharmaceuticals

    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy and has recently released top-line data from Phase 2 studies in women with postpartum depression and patients with refractory status epilepticus.

    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 intention, completion, and timing relating to the proposed public offering and concurrent private placement. Forward-looking statements in this release involve substantial risks and uncertainties, including, without limitation, risks and uncertainties related to market conditions and the satisfaction of customary closing conditions related to the proposed public offering and concurrent private placement. 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.   

    CONTACT:   
    Lisa M. Caperelli
    Executive Director, Investor & Strategic Relations
    Marinus Pharmaceuticals, Inc.
    484-801-4674

    Primary Logo

    View Full Article Hide Full Article
  51. RADNOR, Pa., Dec. 10, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), ("Marinus" or the "Company") today announced that it intends to offer and sell shares of its common stock in an underwritten public offering.  All of the shares in the offering are to be sold by Marinus.  Marinus also expects to grant the underwriters a 30-day option to purchase up to an additional 15 percent of the number of shares to be issued and sold in the public offering.  The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    Concurrently with the completion of the public offering, the Company expects to sell to certain…

    RADNOR, Pa., Dec. 10, 2019 (GLOBE NEWSWIRE) -- Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), ("Marinus" or the "Company") today announced that it intends to offer and sell shares of its common stock in an underwritten public offering.  All of the shares in the offering are to be sold by Marinus.  Marinus also expects to grant the underwriters a 30-day option to purchase up to an additional 15 percent of the number of shares to be issued and sold in the public offering.  The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    Concurrently with the completion of the public offering, the Company expects to sell to certain investors in a private placement, a number of shares of convertible preferred stock equal to an aggregate purchase price of up to approximately $30 million, at a price equal to the public offering price.

    Oppenheimer & Co. Inc. and Ladenburg Thalmann & Co. Inc., a subsidiary of Ladenburg Thalmann Financial Services Inc., (NYSE:LTS), are acting as joint bookrunning managers for the offering.

    Marinus intends to use the net proceeds received from the  public offering and concurrent private placement to advance the preclinical and clinical development of ganaxolone, including expenses for clinical trials for our seizure disorder programs, and regulatory, research and development, pre-commercial, general and administrative and manufacturing expenses and for working capital and general corporate purposes. 

    The shares of common stock in the public offering will be issued by Marinus pursuant to a shelf registration statement that was previously filed with, and declared effective by, the Securities and Exchange Commission (SEC) on December 1, 2017.  The public offering will be made only by means of the written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement  and the accompanying prospectus relating to the public offering will be filed by the Company with the SEC.  Copies of the preliminary prospectus supplement and the accompanying prospectus relating to the  public offering may also be obtained from Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, NY, 10004, by telephone at (212) 667-8055, or by email at ; or from Ladenburg Thalmann & Co. Inc., Prospectus Department, 277 Park Avenue, 26th Floor, New York, New York 10172 or by email at .

    This press release does not constitute an offer to sell or the solicitation of offers to buy any securities of Marinus being offered in the public offering or concurrent private placement, and shall not constitute an offer, solicitation or sale of any security in the public offering or concurrent private placement in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About Marinus Pharmaceuticals

    Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of ganaxolone, which offers a new mechanism of action, demonstrated efficacy and safety, and convenient dosing to improve the lives of patients suffering from epilepsy and depression. Ganaxolone is a positive allosteric modulator of GABAA 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 is conducting the first ever pivotal studies in children with CDKL5 deficiency disorder and PCDH19-related epilepsy and has recently released top-line data from Phase 2 studies in w