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.
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.
Lisa M. Caperelli
Executive Director, Investor & Strategic Relations
Marinus Pharmaceuticals, Inc.