Shares of Marinus Pharmaceuticals Inc. (NASDAQ: MRNS) tumbled Monday morning after the company gave an update on top-line results from its Phase 3 clinical trial in adults with drug-resistant focal onset seizures. Unfortunately, ganaxolone did not meet the primary endpoint of demonstrating a statistically significant difference from placebo.
Considering previous studies, ganaxolone was consistent in being generally safe and well tolerated. Marinus plans to discontinue its program in adult focal onset seizures and focus its efforts on advancing ganaxolone in status epilepticus and pediatric orphan indications.
Overall, management was 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. Yet, the company does remain confident in the safety profile of ganaxolone and its ability to effectively reduce seizures in targeted patient populations.
The company said that it will provide an update in the upcoming weeks on its clinical programs in these indications.
Dr. Albena Patroneva, chief medical officer of Marinus, added:
The design, conduct, patient demographics and median separation versus placebo were similar to our positive Phase 2 trial and other studies conducted with recently approved antiepileptic drugs. While we did not see the incremental efficacy at a higher dose that we were hoping to achieve, ganaxolone continued to display a good safety profile – a key attribute for the product’s future development. We will leverage the findings from this study in the conduct of our ongoing and future studies in pediatric orphan indications and status epilepticus.
Shares of Marinus were down 70.2% at $1.59 early Monday morning, with a consensus analyst price target of $14.60 and a previous 52-week trading range of $4.00 to $20.72.