Disclaimer: Epilepsy Research UK is completely neutral and is not affiliated with any pharmaceutical company, or any particular drug compound.Marinus Pharmaceuticals announced yesterday that their drug candidate, ganaxolone, developed for adults with drug-resistant focal onset epilepsy, did not meet the ‘primary endpoint’ (the outcome by which the effectiveness of treatments is evaluated) in a phase three clinical trial. In other words, there was no significant difference in seizure frequency seen between people taking ganaxolone and those taking a placebo.In a press release issued by Marinus Pharmaceuticals, CEO Christopher M. Cashman said: “We are disappointed with the outcome of this study, and the unfortunate impact on the epilepsy community and particularly the patients suffering from drug-resistant focal onset seizures who are benefiting from ganaxolone treatment.”The trial was conducted at 61 different sites in the US, Europe and Russia. It involved 359 participants who were randomly divided in two groups. Each group either received the drug or an identical-looking placebo. To avoid bias, neither the participants nor the clinicians knew who was taking the drug and who was taking the placebo (known as ‘double-blind’). Following a 12-week treatment period, the results showed that there was no significant difference in seizure frequency between the two groups.Ganaxolone was developed by Marinus Pharmaceuticals for the treatment of drug-resistant focal onset seizures. To date it has been trialled on more than 1,300 people with epilepsy. All trials conducted to date, including this one, showed that the drug candidate was generally safe and well-tolerated, with the most common side effect being sleepiness, dizziness, and fatigue.The company is expected to discontinue its programme in adults with drug-resistant focal onset seizures and instead focus its efforts in trialling the drug for status epilepticus, a condition in which seizures follow one another without recovery of consciousness in between.“We remain confident in the safety profile of ganaxolone and its ability to effectively reduce seizures in targeted patient populations. We are committed to building our ganaxolone franchise and are confident in the potential of ganaxolone in the treatment of status epilepticus and pediatric orphan (affecting fewer than 200,000 people nationwide) seizure and behavior disorders. We will provide an update in the upcoming weeks on our clinical programmes in these indications,” added Mr Cashman.Marinus Pharmaceuticals hosted a conference call for shareholders and other interested parties. The webcast of the call can be accessed via this link.Author: Dr Özge ÖzkayaNote: Negative outcomes generally under-reported, but they are an important part of research and the lead-up to breakthroughs.Click here for more articles about other treatments for epilepsy.