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Pipeline Drug: ABX-1431

October 10, 2016

Abide Therapeutics announced that Celgene has opted to obtain the rights, outside of the United States, to ABX-1431, Abide’s endocannabinoid system modulator being developed to treat neurological diseases, including multiple sclerosis (MS), by reproducing within the body the physical benefits (minus the psychotropic effects) of cannabis.

ABX-1431 is an oral, selective, small molecule inhibitor of monoacylglycerol lipase (MGLL) — an enzyme that catalyzes the breakdown of the endogenous cannabinoid, 2-arachidonoylglycerol (2-AG) and serves the major regulator of its levels. In turn, 2-AG is thought to regulate neurotransmitter balance and inflammation. Immune attacks, like those that characterize MS, lead to neurotransmitter imbalance.

The cannabinoid 2-AG binds to the cannabinoid receptors CB1 and CB2 — the molecular targets of the psychoactive component of cannabis: delta-9 tetrahydrocannabinol or THC. CB1 is the primary cannabinoid receptor in the nervous system, and its activation accounts for most of the neurobehavioral effects of THC. CB2 is found primarily on immune cells and mediates the immunosuppressive effects of cannabinoids.

Direct activation of cannabinoid receptors by medicinal cannabis is thought to offer therapeutic benefits to conditions like spasticity, pain, disrupted sleep, nausea, and poor appetite. ABX-1431 has the potential to produce similar therapeutic benefits.

Celgene will pay $20 million to Abide for these rights, part of an agreement between the two companies reached in 2014, and be in charge of development costs of all indications from Phase 2 clinical trials onward. Abide will retain U.S. rights and will lead Phase 1b studies.

We plan to perform a number of Phase 1b studies to evaluate the therapeutic potential of what appear to be cannabinoid-sensitive diseases. We plan to study neuromyelitis optica (NMO) and movement disorders as follow-on indications,” Alan Ezekowitz, MDChB, DPhil, Abide Therapeutics’ chief executive officer and president, said in a press release.