RogCon and its collaborators from The Florey Institute of Neuroscience presented preclinical data on the company’s lead program, RC-222, an antisense oligonucleotide designed to down-regulate SCN2A, which is being developed with Ionis Pharmaceuticals, Inc. (Ionis), the leader in RNA-targeted drug discovery and development.

In preclinical studies evaluating the antisense oligonucleotide in an SCN2A mouse model, efficacy was evaluated by survival, seizure number, electroencephalography (EEG), behavioral test batteries and whole cell recording in brain slices. Results demonstrated that:

• The therapeutic effect of a single peri-natal dose of the antisense oligonucleotide was significant and long lasting, with nearly 70% of treated mice surviving to 80 days compared to 0% survival at 30 days in untreated mice or those receiving current standard of care.
• Treatment with the antisense oligonucleotide mitigated spontaneous seizures and largely restored neuronal excitability, EEG activity and behavioral characteristics of treated mice to wildtype levels.
• These results demonstrate the remarkable efficacy of SCN2A down-regulation and has laid an important foundation for the accelerated clinical development of the RC-222 program.

This study aims to evaluate the efficacy and tolerability of Perampanel (PER) used as first add on in non-refractory epilepsy in everyday clinical practice and to study possible synergistic combinations with PER.

This is a multicenter, observational, consecutive and retrospective study, performed in 22 Spanish Hospitals. Inclusion criteria were: (1)patients =12 years old, (2)definite history of focal or idiopathic generalized epilepsy, (3)being treated previously with one or two antiepileptic drugs on monotherapy, (4)patients treated with PER as first-add on therapy.

This real world study demonstrates that PER at low doses and as a first add-on treatment is a good option for patients with focal and generalized epilepsy. PER retention rate at 12 months is high and nearly half of the patients achieve seizure freedom. Most of the AE were mild/moderate and the association with LEV was safe and effective.

Fenfluramine HCl has demonstrated superior efficacy compared with placebo for the reduction in frequency of convulsive seizures in children and young adults (2-18 years old) with Dravet syndrome (DS) in two recently completed Phase 3 double-blind, placebo-controlled clinical trials. Patients with epileptic encephalopathies, such as DS, also have impairment in cognition and neurodevelopment, which is believed to be in part due to the frequency of poorly treated seizures. Here we provide an analysis of the impact of fenfluramine on caregiver-reported everyday executive functioning of their children with DS in a Phase 3 placebo-controlled trial.

Patients in the fenfluramine 0.2 mg/kg and 0.8 mg/kg groups showed significant and clinically meaningful improvements in behavior regulation, emotion regulation, and planning and organization. Theoretical and empirical models support that improved emotional control and behavioral regulation serve as “building blocks” to then enable higher level cognitive regulation functions. Children and young adults must be able to inhibit impulses, modulate emotions, and adapt flexibly to changes before they can demonstrate or develop cognitive regulatory functions, including working memory, planning, and organization. The impact of behavior and emotion regulation might be expected to enable improvements in the higher level cognitive regulation with longer-term treatment and will be examined in the long-term extension study of fenfluramine.

Nayzilam™ (USL261; midazolam nasal spray, MDZ NS) is a novel nasal spray formulation of MDZ specifically developed as an acute rescue treatment for seizures in the outpatient setting for patients who require control of intermittent episodes of increased seizure activity (ie, acute repetitive seizures [ARS], seizure clusters [SC]).

This novel nasal spray formulation of MDZ provides convenient, non-invasive drug administration without active inhalation and can easily be given by caregivers when patients are experiencing an episode. The safety and efficacy of MDZ NS was evaluated in a randomized, double-blind, placebo-controlled, Phase 3 study (ARTEMIS-1; NCT01390220). This open label extension to ARTEMIS-1 was conducted to evaluate long-term safety and efficacy of repeated treatment of SC episodes (ARTEMIS-2, NCT01529034). Efficacy data are reported in a separate abstract (Sequeira et al, AES 2018).

The open-label ARTEMIS-2 study supports the long-term safety of repeated usage of MDZ NS in the outpatient setting to treat SCs. MDZ NS potentially offers caregivers a novel and reliable means of rescue treatment for patients experiencing SCs.

Beginning Monday, patients with epilepsy will have a new option to reduce the number and severity of life-limiting seizures, avoiding radical surgery that removes a part of the brain.

Called deep-brain stimulation, the treatment uses electrodes implanted in the thalamus, a structure located near the center of the brain that receives information from the senses and sends signals to the cerebral cortex.

The electrodes are controlled by a device implanted in the chest, similar to a pacemaker, and patients have a remote-control device that can adjust the amount of stimulation or even turn it off for periods of time.

Deep-brain stimulation is much less aggressive than traditional surgery. The neurostimulator device is implanted in the chest and two leads are brought under the skin to the top of the head, with one being inserted into each thalamus on either side of the brain. Each lead has four contacts. “You can stimulate any or all of them in any combination,” Gerrard said.

After the stimulator is implanted, programming it is done over time, with “adjustments made depending on any potential side effects patients are having” and how well it is working to reduce the number and severity of seizures. Each patient is given a programmer “that allows them to interact with the device,” Gerrard said. “It’s kind of like a parental programmer for the television. In some patients, as they become more familiar with the device and their stimulator . . . they may change their programs at home.”