- Marinus has a March PDUFA date - when the FDA will rule on whether to approve an oral formulation of Ganaxolone in CDKL5 Deficiency Disorder ("CDD").
- Ganaxolone is Marinus' lead and only product and it has a long and checkered history - having failed in depression, it may succeed in epilepsy.
- The data from the Phase trial of Ganaxolone in CDD is reasonably string, although the placebo arm's unexpectedly low efficacy may have helped achieve statistical significance.
- Marinus is targeting several other epilepsy indications - including Status Epilepticus in collaboration with BARDA - the drug may work best in an IV formulation.
- There's $100m in peak sales potentially on the table from an approval in CDD, but further approvals look years away and Marinus' high cash burn is problematic - I would stay on the sidelines investment-wise.
For further details see:
Marinus Stock Slides To 18-Month Low, Upcoming PDUFA May Offer Redemption