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Ganaxolone

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Philanthropic Funding Provided by: Epilepsy Therapy Project

Tx name Ganaxolone
Description Neurosteroid
Company Marinus
Status Phase 2
Overview Partial-Onset seizures: Synthetic Analogue of the Endogenous Neurosteroid Allopregnanolone, a Metabolite of Progesterone, that Lacks Hormonal Side Effects,High-Affinity, Stereoselective, Positive Allosteric Modulator of the GABAA Complex, Binds to Postsynaptic GABAA Receptor Binding Site Distinct from Benzodiazepines and Barbiturates (Alpha-4-Delta Subunit) ,  No Tolerance Expected from Preclinical Models or Clinical Data. Ganaxolone was safe and well tolerated at 1500 mg/day; Evidence of efficacy was observed in the first week of dosing, w/ no evidence of tolerance; efficacy was significantly different from placebo. BID dosing, IV possible
PK  
indications Partial-onset seizures; infantile spasms (?), mood disorders to be explored
preclin data  
preclin tox  
Phase 1  
Phase 2 Proportion >50% sz reduction: GNX (N98) -18%, Pla (N49) +2diff 20%, p<0.014
Phase 3  
Adverse effects Dizziness, fatigue/somnolence, NO weight gain
publications  
IP Unique Patent Estate Solves PK/COGs with coverage to 2025/2029
Potential problems Need new formulation, more PK.  Need partner to continue clinical development
Timeline  
Financial Status VC Funded
Partnerships  
ETP Support Investment

President
President
Created by President 07/9/2010 at 07:07am
admin
admin
Edited by admin 09/2/2010 at 01:08pm

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