Marinus Pharmaceuticals Announces Four Presentations on Ganaxolone at AES2020 Virtual Annual Meeting

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 GABA_A 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 GABA_A 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 GABA_A 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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