Ganaxolone Marinus Pharmaceuticals, Inc.
Ganaxolone Marinus Pharmaceuticals, Inc.
Ganaxolone Marinus Pharmaceuticals, Inc.
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<strong>Ganaxolone</strong><br />
<strong>Marinus</strong> <strong>Pharmaceuticals</strong>, <strong>Inc</strong>.<br />
Neurosteroid therapy for Epilepsy<br />
Epilepsy Pipeline Conference<br />
March 13, 2008
GABA A Receptor Binding and Site<br />
of Action of <strong>Ganaxolone</strong><br />
<strong>Ganaxolone</strong><br />
binding enhances<br />
chloride flux and<br />
promotes synaptic<br />
inhibition which<br />
accounts for the<br />
protective activity<br />
of the drug against<br />
seizures<br />
2
<strong>Ganaxolone</strong> –History and IP<br />
•<strong>Marinus</strong> <strong>Pharmaceuticals</strong> Licensed from<br />
Purdue Pharma<br />
•Historical Background<br />
–Neurosteroid with no hormonal activity<br />
–Efficacy in animal models of epilepsy<br />
–Good safety profile in animals and humans to<br />
date<br />
–Data from Inpatient DB study in Adults and OL<br />
Infantile Spasm study<br />
•IP Covering New Formulations Filed<br />
3
<strong>Ganaxolone</strong><br />
Development Challenges<br />
• Ongoing DBPCB controlled flexible dose trials:<br />
–Infantile Spasms<br />
–Refractory adult partial onset seizures<br />
• Infantile Spasms study, if positive, will need a<br />
second fixed dose pivotal trial<br />
• Pilot data in Catamenial epilepsy will need to be<br />
quantified and replicated in Phase III trials<br />
• BID dosing of solid dosage form needed for<br />
Phase III adult trials –work underway<br />
4
Timelines<br />
•Top line results for both IS and APS<br />
studies in Q3/Q4 2008<br />
•Solid dose formulation performance and<br />
BID dosing study results in Q3 2008<br />
•Open label Extension Studies now set at<br />
1 year and will continue into early 2009<br />
•Efficacy/Safety analysis will define the<br />
forward path<br />
5
Differentiation Opportunities<br />
•Basic claim for partial seizures with an<br />
advantageous safety profile<br />
•Indication for infantile spasms<br />
–Evaluating development for improvement<br />
•May have advantages for women with partial<br />
seizures<br />
–Lack of reproductive toxicity seen<br />
–Catamenial mechanism<br />
•Potential efficacy in other indications such as<br />
PTSD, TBI, anxiety/depression… .. 6