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Re: kmastra post# 213143

Tuesday, 08/15/2017 6:43:53 PM

Tuesday, August 15, 2017 6:43:53 PM

Post# of 251620
Initially SAGE-547 is an IV formulation that goes after Super-Refractory Status Epilepticus which is the acute form of seizure while MRNS ganaxolone is
an oral formulation for focal seizure which is a chronic condition.

While SAGE-547 was successful in SRSE and later on Post Partum Depression (PPD), ganaxolone failed in various indications of seizure including pediatric application.

Then MRNS copied SAGE and reformulated ganaxolone as IV to go after PPD and SRSE but they have not started any trial for SRSE yet, only PPD first.
The only trial that completed is CDKL5 pediatric epilepsy which is an open label trial. There is no POC that ganaxolone would work in IV for SRSE or PPD, look like a hail Mary to me IMO.

In the mean time SAGE developed the 2nd generation SAGE-217 which is an
oral formulation much more potent than SAGE-547 for PPD and SRSE.
MRNS has 20M in cash as of June 30 17 vs 285.9M in cash for SAGE, too poor
to do any meaningful development.

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