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Marinus Pharmaceuticals Provides Pipeline Update

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

Benzinga · -
  • 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 Phase3 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 Phase2 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.