Drug Development

Two flops in two weeks for Marinus Pharmaceuticals’ ganaxolone 

Days after Marinus Pharmaceutical’s ganaxolone failed on an epilepsy study, the same drug failed a Fragile X syndrome trial, further bruising the biotech’s already badly damaged market value.

Rather incredibly, the biotech ($MRNS) claims it didn’t expect to hit the primary endpoint: overall improvement across the Fragile X syndrome. Instead the biotech hailed data on ganaxolone’s effectiveness in treating anxiety for patients and intends to use that to find some path forward for the GABAA modulator.

Christopher Cashman, CEO

Christopher Cashman, CEO of Marinus

“Our goal for this study was to measure improvement in these behaviors based upon the anxiolytic activity of ganaxolone and use the results from this exploratory study to inform enrollment criteria and endpoint selection in future studies. The data we observed in several analyses related to patient anxiety and attention are encouraging and clearly warrant further investigation,” offered Marinus CEO Christopher Cashman in a prepared statement.

Its shares are down under $2.00 this morning on the news. The company traded as high as $17.00 this time last year.


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RAPS Regulatory Convergence 2017