Marinus Pharmaceuticals Provides Pipeline Update - Seite 2

• 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.             

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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 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.