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Marinus Pharmaceuticals shares surge on Nasdaq on successful clinical trial results

The candidate is being used to treat CDKL5 - a rare genetic disorder
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It mainly affects girls and is characterized by early-onset, difficult-to-control seizures and severe neuro developmental impairment

Shares in Marinus Pharmaceuticals (NASDAQ:MRNS) added over 46% on Nasdaq to $4.57 as it posted positive top-line data from a phase 2 study assessing orphan drug-tagged ganaxolone.

The candidate is being used to treat  CDKL5  - a rare genetic disorder, which is caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome.

It mainly affects girls and is characterized by early-onset, difficult-to-control seizures and severe neuro‑developmental impairment.

Dr Orrin Devinsky, the principal investigator in the current study, said: “I am impressed with the magnitude of seizure reduction and gain in seizure-free days seen with ganaxolone treatment in children with this highly refractory epilepsy.

"The durable anti-epileptic effect seen in several children distinguishes ganaxolone’s efficacy from the more than 20 currently available anti-epileptic drugs that provide limited seizure control lasting a few weeks to months.

"When treating children with the severest forms of epilepsy such as CDKL5 disorder, there is a desperate need for new drugs that are well-tolerated, efficacious and easy to administer with no need for special monitoring.

"With no other treatment showing this degree of promise, I am excited to lead the effort to advance ganaxolone into potentially the first, late-stage clinical trial in children with CDKL5 disorder.”



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