Marinus execs herald the rebirth of the one-drug biotech as a positive PhIII readout arrives for a rare disease
After a long, neck-snapping ride full of spills, thrills and dismal chills, the execs now running small-cap biotech Marinus $MRNS say they have achieved a signal success in a Phase III study of ganaxolone.
Their sole drug, a GABA/A receptor modulator used as an anti-seizure drug — which has suffered some harsh failures in the past — hit the endpoint in a pivotal test for rare cases of CDKL5 deficiency disorder, called CDD in shorthand. Investigators say the drug achieved a 32.2% median reduction in 28-day major motor seizure frequency compared to a 4% bump in the placebo group.
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