Ganaxolone Marinus Pharmaceuticals, Inc.

Ganaxolone
Marinus Pharmaceuticals, Inc.
Neurosteroid therapy for Epilepsy
Epilepsy Pipeline Conference
March 13, 2008

GABA A Receptor Binding and Site
of Action of Ganaxolone
Ganaxolone
binding enhances
chloride flux and
promotes synaptic
inhibition which
accounts for the
protective activity
of the drug against
seizures
2

Ganaxolone –History and IP
•Marinus Pharmaceuticals Licensed from
Purdue Pharma
•Historical Background
–Neurosteroid with no hormonal activity
–Efficacy in animal models of epilepsy
–Good safety profile in animals and humans to
date
–Data from Inpatient DB study in Adults and OL
Infantile Spasm study
•IP Covering New Formulations Filed
3

Ganaxolone
Development Challenges
• Ongoing DB­PCB controlled flexible dose trials:
–Infantile Spasms
–Refractory adult partial onset seizures
• Infantile Spasms study, if positive, will need a
second fixed dose pivotal trial
• Pilot data in Catamenial epilepsy will need to be
quantified and replicated in Phase III trials
• BID dosing of solid dosage form needed for
Phase III adult trials –work underway
4

Timelines
•Top line results for both IS and APS
studies in Q3/Q4 2008
•Solid dose formulation performance and
BID dosing study results in Q3 2008
•Open label Extension Studies now set at
1 year and will continue into early 2009
•Efficacy/Safety analysis will define the
forward path
5

Differentiation Opportunities
•Basic claim for partial seizures with an
advantageous safety profile
•Indication for infantile spasms
–Evaluating development for improvement
•May have advantages for women with partial
seizures
–Lack of reproductive toxicity seen
–Catamenial mechanism
•Potential efficacy in other indications such as
PTSD, TBI, anxiety/depression… .. 6