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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 |

| Title | Posted | |
|---|---|---|
| Nocturnal seizures | May 25, 2012 | |
| nocturnal11 | ||
| Lamictal & Seizure questions - help??? | May 25, 2012 | |
| PPlex | ||
| Severe Epilepsy And Pregnancy | May 25, 2012 | |
| AmyBoop88 | ||
| 7 yr old seizures and autism/hearing loss/adhd, etc | May 25, 2012 | |
| jackjack | ||
| epilepsy and hormonal effects | May 25, 2012 | |
| cindyloowho | ||
| Tried Potiga yet? | May 24, 2012 | |
| tcameron | ||
| Can exercise induce seizures? | May 24, 2012 | |
| MichaelK | ||
| Lamictal Nausea, Dizzy Spells | May 24, 2012 | |
| karyliin6 | ||
| Anyone on Lamictal AND Keppra? | May 24, 2012 | |
| Bloodyrose | ||
| Paying for your seizure pills with no insurance? | May 24, 2012 | |
| Tatianalee | ||
| View all Forums | ||
| Title | Page Views | |
|---|---|---|
| my.epilepsy.com Updates | 20,619 | |
| epi_help | ||
| topamax and weight loss | 19,330 | |
| alexia mom | ||
| kepra | 19,244 | |
| brian mattingly | ||
| Possible cure for absence seizures | 16,388 | |
| pdl1 | ||
| How exactly do aura's feel | 14,077 | |
| WendyBendy | ||
| Sexual Side Effects | 13,524 | |
| George R | ||
| MEDICAL ALERT I.D.'s | 12,453 | |
| picnupthepcs | ||
| Over 40 Different Types Of Seizures - Revised | 10,443 | |
| spiz | ||
| electrical shock in head? | 10,083 | |
| Maggie | ||
| Weight Gain and Depakote | 9,011 | |
| galinda | ||
| View all Forums | ||
| Title | Posted | |
|---|---|---|
| Update 2012 -- lots has happened! | May 25, 2012 | |
| ninabva68 | ||
| My Blog Disappeared! Can't my seizures too? | May 24, 2012 | |
| MichaelK | ||
| SEIZURES ARE CHANGING. WHY? WHAT KIND AM I HAVING NOW? BRAIN EXPLOSION I FELT IS A MYSTERY TO ME | May 24, 2012 | |
| ROCKNROLL | ||
| Hyperventilation & epilepsy, a way to help yourself. | May 23, 2012 | |
| buteykomike | ||
| Medicine adjustments | May 23, 2012 | |
| fashionlove45 | ||
| NOT YELLING, VISUALLY IMPAIRED. POSTICTAL SHOPPING. ANYONE ELSE WITH THIS PROBLEM? TBI AND SHOPPING | May 22, 2012 | |
| ROCKNROLL | ||
| Seizures and medical marijuana | May 21, 2012 | |
| seizureprone134 | ||
| being unigue and being hypocrite | May 20, 2012 | |
| futuer poet | ||
| Coming out? | May 19, 2012 | |
| fashionlove45 | ||
| RWAY40 | May 19, 2012 | |
| rway40 | ||
| View all Blogs | ||
| Title | Page Views | |
|---|---|---|
| my partial complex seizures | 4,540 | |
| Zanna1211 | ||
| Topomax... The Dreaded......... | 3,787 | |
| Dr Jason | ||
| Brain Zaps, tics & twitches | 3,586 | |
| JudiS | ||
| How can you tell if a sleep seizure happens? | 3,545 | |
| epl_controller | ||
| Feeling Sick | 3,308 | |
| JBJ1984 | ||
| side effects of phenobarb. | 3,215 | |
| pksmom | ||
| Tegretol XR and ANXIETY meds | 2,904 | |
| Butterflygrl | ||
| TYLENOL, AEDs & SEIZURES | 2,836 | |
| cmscribbles | ||
| Nonepileptic "Events" vs. "Seizures" | 2,711 | |
| teft | ||
| newborn seizure | 2,256 | |
| Sunny_80 | ||
| View all Blogs | ||
| Title | Posted | |
|---|---|---|
| Angela-48 | May 11, 2012 | |
| ANGELA-48 | ||
| Babies with seizures | May 5, 2012 | |
| gabrielsmommy121911 | ||
| Parent and Family Support Group Newton Kansas | Mar 13, 2012 | |
| Brown67114 | ||
| No more driving | Mar 9, 2012 | |
| Amy829 | ||
| Audubon, Pennsylvania (King of Prussia area) Parents of Kids with Epilepsy Looking to connect with others in thier area | Mar 7, 2012 | |
| ajssoscia | ||
| People who have epilepsy and are parents of young children, step children or pregnant. | Feb 27, 2012 | |
| kjsdestiny | ||
| Had the worse seizure of my life last night! Complex Partial | Feb 8, 2012 | |
| jlamont | ||
| Self-Management of Medication | Feb 7, 2012 | |
| columbia.epilepsy.study | ||
| Dravet Spectrum Disorder | Feb 4, 2012 | |
| mytboy | ||
| Diving with eplilepsy | Feb 1, 2012 | |
| p59 | ||
| View all Groups | ||
| Title | Posted | |
|---|---|---|
| Imagining the past | May 25, 2012 | |
| jrgeorge | ||
| Feeling Epilepsy as a part of Life....... | May 25, 2012 | |
| Avnish | ||
| My life with epilepsy | May 24, 2012 | |
| BigTom_85 | ||
| I thought there was nothing wrong | May 24, 2012 | |
| Mendoza87 | ||
| New to this | May 23, 2012 | |
| kitkat2503 | ||
| Is it Emotional | May 23, 2012 | |
| frustrated1965 | ||
| Adjusting with the thought of epilepsy after misdiagnosis for over 20 years | May 22, 2012 | |
| goofyheather | ||
| Continuing with life | May 21, 2012 | |
| nastaran | ||
| Working my life around having epilepsy | May 21, 2012 | |
| 1505loupylou | ||
| The frustration of not knowing exaclty what is wrong... | May 20, 2012 | |
| cag122 | ||
| View all Stories | ||
| Title | Page Views | |
|---|---|---|
| Jessica Roiz | 2,433 | |
| kroiz | ||
| my story | 562 | |
| snoby | ||
| Temporal Lobe Epilepsy | 561 | |
| Always Have On Clean Underwear | 508 | |
| crashllama | ||
| my brain has died a thousand deaths........... | 476 | |
| banffgirl | ||
| Kelly's Life With Epilepsy | 472 | |
| kjcanada1979 | ||
| What My Seizures Are Like...... | 421 | |
| javaman | ||
| Nocturnal grand mal seizures (primary generalized epilepsy) | 395 | |
| karalyeva | ||
| Life......... | 376 | |
| dizzygal | ||
| My Daily Philosophy..... | 366 | |
| KatieFerguson88 | ||
| View all Stories | ||
