Marinus sinks as seizure drug fails PhIII, jettisons program


Marinus Pharmaceuticals ($MRNS) has pulled the plug on its late-stage epilepsy treatment ganaxolone after it failed to best placebo in a Phase III study.

The trial missed its primary endpoint of percent change in the 28-day seizure frequency from baseline as its median percent reduction of focal onset seizures in the ganaxolone group was 21.28% compared to 10.25% with placebo during the titration and the 12-week treatment period.

This did not reach statistical significance and Marinus now plans to “discontinue its program” in adult focal onset seizures. It will now instead focus its efforts on testing ganaxolone in status epilepticus and pediatric orphan indications, the Branford, CT-based biotech said in a statement.


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Christopher Cashman, CEO of Marinus, 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. 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 seizure and behavior disorders. We will provide an update in the upcoming weeks on our clinical programs in these indications.”

Ganaxolone works as a CNS-selective GABAA modulator and has been developed in 3 different dose forms: IV, capsule, and liquid as it looks to provide options for patients who do not react to presently available antiepileptics.

Its shares were down 70% this morning on the news.

- here’s the release

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