Marinus Pharmaceuticals
Marinus Pharmaceuticals is focused on developing innovative therapies for severe, rare forms of epilepsy, including status epilepticus, tuberous sclerosis complex, and CDKL5 deficiency disorder, with its lead product ganaxolone in both intravenous and oral formulations.
Services
Marinus Pharmaceuticals provides innovative treatment options tailored for both acute and chronic care settings. They focus on breakthrough therapies for conditions such as status epilepticus, tuberous sclerosis complex, and CDKL5 deficiency disorder. The company's primary drug, ganaxolone, is developed in both intravenous (IV) and oral formulations to cater to different patient needs. Marinus conducts high-quality in-house research, collaborates with external consultants, and operates under strong scientific guidelines to ensure safety and efficacy in its treatments.
Products
The primary product of Marinus Pharmaceuticals is ganaxolone, which is available in intravenous (IV) and oral formulations. These formulations aim to provide rapid cessation of status epilepticus and reduce seizure frequency in children with refractory epilepsy. Ganaxolone has shown promise in treating severe forms of epilepsy, such as tuberous sclerosis complex and CDKL5 deficiency disorder. Additionally, ZTALMY—a commercial product based on ganaxolone—generated $7.5 million in net product revenue for the first quarter of 2024. The company is also developing a second-generation oral formulation and a prodrug of ganaxolone.
Clinical Trials and Research
Marinus Pharmaceuticals is actively engaged in various clinical trials to expand the therapeutic applications of ganaxolone. The company is conducting the TrustTSC Phase 3 trial to assess the efficacy of ganaxolone for treating seizures associated with tuberous sclerosis complex. A new method of use patent for ganaxolone in this area extends until 2040. The company plans to submit a supplemental New Drug Application to the U.S. FDA by April 2025 for this use. Phase 3 RAISE II trial in refractory status epilepticus was halted, pending further assessment. A proof-of-concept study with ZTALMY is also planned for developmental and epileptic encephalopathies, including Lennox-Gastaut syndrome, in early 2025.
Collaborations and Partnerships
Marinus Pharmaceuticals collaborates with various groups to support patients and families affected by seizure disorders. The company holds an agreement with the Biomedical Advanced Research and Development Authority (BARDA) to explore ganaxolone’s use in response to nerve agent exposure threats. Internationally, Marinus is targeting achievement milestones in Europe that could yield a €10 million payment for their CDKL5 deficiency disorder (CDD) launch. Such collaborations and strategic partnerships are central to Marinus' mission of providing innovative and effective treatment options.
Financial and Strategic Planning
Marinus Pharmaceuticals has implemented cost reduction plans to extend its cash runway into late Q1 2025. Net product revenue from ZTALMY recorded $7.5 million for the first quarter of 2024, which is a 125% increase from the previous year's first quarter. The company is eligible for a €10 million payment upon achieving specific launch milestones in Europe for CDD. Marinus also ceased the Phase 3 RAISE II trial to reassess further development efforts based on topline data. These financial strategies align to support their ongoing and future developmental plans.