Lennox-Gastaut Syndrome is a rare form of epilepsy that occurs in childhood and is characterized by seizures and intellectual disability. Adverse conditions such as injury to brain and its malformation, lack of oxygen to the new-born infants, infections of central nervous system and inherited metabolic or degenerative disorders are responsible for occurrence of Lennox-Gastaut Syndrome. Anti-epileptic medications are the main treatment option for Lennox-Gastaut Syndrome. The medication is often complicated in case of paediatric disorders as there is risk in development of new-born, due to which various research organizations are focussing on development of therapeutics of Lennox-Gastaut Syndrome.
The growth in therapeutic pipeline of Lennox-Gastaut Syndrome can also be attributed to the upsurge in technical advancements in research, increase in funding by non-profit organization such as National Organization for Rare Disorders as well as availability of limited number of treatments.
In August 2017, INSYS Therapeutics Inc. started a Phase III study of an oral solution of cannabidiol, to assess its safety and efficacy as an adjunctive therapy for treatment in subjects with uncontrolled Lennox-Gastaut Syndrome. The trial is expected to be completed in June 2018. In December 2016, Eisai Inc. conducted a Phase III study on its drug candidate, perampanel, for reducing the drop seizures in patients with uncontrolled seizures and Lennox-Gastaut Syndrome. The trial is expected to be completed in February 2020. In January 2016, Zogenix, Inc. started a Phase II clinical study of its drug candidate, fenfluramine, in subjects having epilepsy and Lennox Gastaut Syndrome. The purpose of the study was to evaluate the anti-epileptic effects of low dose fenfluramine in these patients. The trial was expected to be completed in March 2017, however, the study is still ongoing and recruiting participants. In June 2011, Eisai Inc. strated a Phase III study of drug candidate, rufinamide, to evaluate its safety, efficacy and pharmacokinetic activity in subjects of less than four years of age and having uncontrolled Lennox-Gastaut Syndrome. The trial was completed in November 2015.
In January 2017, Takeda Pharmaceutical Company Limited signed a collaborative agreement with Ovid Therapeutics Inc. for the development and commercialization of its investigational new drug candidate, TAK-935, which is selective inhibitor of Cholesterol 24-hydroxylase (CH24H). Currently, the drug candidate is in Phase I/II clinical trial. Under the terms of agreement, Takeda Pharmaceutical Company Limited was to receive an equity in Ovid Therapeutics Inc. for the development milestones of TAK-935. The two companies agreed to share the development and commercialisation cost equally. Takeda Pharmaceutical Company Limited got the rights to lead commercialization in Japan whereas, Ovid Therapeutics Inc. got the commercialization rights in the U.S., Europe, Canada and Israel. In June 2016, Eisai Inc. organized a hackathon called as #HackLGS, in association with Lennox-Gastaut Syndrome (LGS) Foundation, in which technology developers, LGS caregivers, students and creative specialists participated. The purpose of the event was to increase the awareness about the Lennox-Gastaut Syndrome and sharing information about technical innovation that can help to overcome unmet needs for the treatment of the disease.
Some of the companies having a pipeline of Lennox-Gastaut Syndrome therapeutics include, INSYS Therapeutics Inc., Eisai Inc., GW Research Ltd., Zogenix, Inc., Johnson & Johnson Pharmaceutical Research & Development, LLC, H. Lundbeck A/S, GlaxoSmithKline plc, Valeant Pharmaceuticals International, Inc., Ortho-McNeil Pharmaceutical Ltd.