Progress in neurology for Celgene and GW

It’s been a week notable for developments in neurology medicine, with Celgene exercising an option to buy a potential multiple sclerosis drug, as GW Pharma prepares a filing for a phase 3 epilepsy drug derived from the cannabis plant.

Celgene has exercised a $20 million option to obtain ex-US rights to Abide’s first-in-class endocannabinoid system modulator, ABX-1431, which could treat neurological diseases such as multiple sclerosis (MS) by producing similar effects to cannabinoid drugs.

Celgene will pay for development for all indications from phase 2 clinical trials onward, while Abide retains US rights and will conduct phase 1b studies.

San Diego-based biotech Abide said it had completed a first-in-human phase 1a study, showing the drug is well tolerated, and with no adverse events, with data from PET scans showing the drug had penetrated the brain.

ABX-1431 is a first-in-class, orally active, selective, small molecule inhibitor of monoacylglycerol lipase (MGLL) that modulates the levels of the endogenous cannabinoid, 2-arachidonoylglycerol (2-AG).

This in turn is believed to regulate neurotransmitter balance and inflammation. Neurotransmitter imbalance as a result of immune attack is a feature of MS.

Abide said ABX-1431 elicits a similar beneficial effect to medicinal cannabis on pain, spasticity, sleep, appetite, and nausea – but by amplifying signalling in the body’s cannabinoid signalling system.

Meanwhile GW Pharma announced a second set of positive phase 3 trial results for Epidiolex (cannabidiol) for the rare childhood epilepsy, Lennox-Gastaut syndrome.

Patients taking the oral, cannabis-derived drug daily at 40mg dose, showed a 42% reduction in monthly seizures, compared with 17% who took placebo.

This is the second phase 3 success for Epidiolex in Lennox-Gastaut after a first set of positive results announced in June, and the company plans a US filing in the first half of 2017.

The filing will cover Lennox-Gastaut syndrome and Dravet syndrome – the drug produced positive results in the latter in March in a separate phase 3 trial.

The dossier will contain data from one Dravet trial, and two Lennox Gastaut trials, with the US Food and Drug Administration giving a simultaneous decision on both indications, without waiting for results from a second Dravet trial.

Also being developed in Tuberous Sclerosis Complex and infantile spasms, analysts have predicted the drug could become a blockbuster if it is approved in all indications.

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