1) What are your thoughts on the ph.2 RSE data? (cover pt baseline, magnitude of effect, any bias). SAGE's allosteric GABA receptor failed SRSE. Are there any negative read-throughs between RSE and SRSE? 

 

 

2) Differences in ph.3 trial include lower baseline seizure burden of 20% (vs 50% in ph2). Do you think this will reduce efficacy delta?

3) IV ganaxolone's commercial success will require being added to formulary via the hospital DRG (reimbursement system). This requires cost-savings (eg. reduced hospital stay from IV anesthesia avoidance). Do you think anesthesia avoidance will reduce hospital stay? or, do you think hospital stay is driven by underlying disease (eg. brain cancer) since seizure is just a symptom.

4) Given hospitals are very financially incentive driven, is there a conflict of interest by administering ganaxolone to avoid IV anesthesia (which may be a hospital profit center).

Background Questions:
How important is IV anesthesia avoidance in RSE?
How much does Ganaxolone need to reduce IV anesthesia use for uptake?
Is there a clinical risk in trying Ganaxolone before IV anesthesia? (if patient fail ganaxolone, will clinical harm be done vs. going stright to IV anesthesia where seizure cessation will be crtain)

Given Seizure is a symptom of the underlying disease (driving seizures), do you think ganaxolone will work for all pts? ( was seizure etiology in ph.II data representative of typical and diverse patient populations?