Abide Therapeutics Announces Celgene Option Exercise to Develop ABX-1431, an Investigational Endocannabinoid System Modulator for Patients with Neurological Diseases

SAN DIEGO, Sept. 29, 2016 /PRNewswire/ -- Abide Therapeutics announced today that Celgene has exercised its option to obtain ex-US rights to Abide's first-in-class endocannabinoid system modulator, ABX-1431, for the potential treatment of neurological diseases. Celgene will be responsible for development costs for all indications from Phase 2 clinical trials and beyond. Abide retains US rights and will conduct a number of Phase 1b studies. With the exercise of this option, Abide will receive a $20 million exercise fee.

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 multiple sclerosis (MS).

"We look forward to partnering with Celgene to explore whether we can bring symptomatic improvement to these long-suffering patients," said Alan Ezekowitz MBChB, DPhil, CEO and President of Abide Therapeutics. "In addition, at Abide 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."

ABX-1431 has successfully completed dosing in a first-in-human, placebo-controlled, Phase 1a study.  The drug was well tolerated and there were no serious adverse events. Preliminary data from an ongoing PET occupancy study indicate dose-dependent brain penetrance of orally-administered ABX-1431 using [¹⁸F]ABX-1488, an Abide proprietary, MGLL-specific PET ligand.  Furthermore, an fMRI study aimed at assessing the patterns of neural activity in the brain associated with ABX-1431 administration is scheduled to start early in the fourth quarter of 2016.

"We are delighted with the rapid progress that the Abide team has made in bringing forward ABX-1431. We look forward to evaluating the therapeutic potential of this exciting molecule in MS. We also recognize that this mechanism has broad therapeutic potential for a range of neurological diseases," said Rupert Vessey, BMBCh, DPhil, President of Research and Early Development, Celgene Corporation.

About the Endocannabinoid System

MGLL is an enzyme that catalyzes the breakdown of the endocannabinoid 2-arachidonoylglycerol (2-AG), an endogenous ligand of the cannabinoid receptors CB1 and CB2, which are the molecular targets of the psychoactive component of Cannabis, delta-9 tetrahydrocannabinol (THC). MGLL is the major regulator of 2-AG levels available to signal through CB1 and CB2. 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 preparations elicits therapeutically beneficial effects on pain, spasticity, sleep, appetite, and nausea. ABX-1431 has the potential to produce similar therapeutic effects through amplification of endogenous cannabinoid signaling.

About Abide Therapeutics

Abide Therapeutics is focused on developing innovative medicines that target serine hydrolases, one of the largest enzyme classes in nature with validated but mostly untapped therapeutic potential. Serine hydrolases play important regulatory roles in human physiology and disease. Abide has created a proprietary platform, based on technology developed at The Scripps Research Institute by Professors Ben Cravatt and Dale Boger, that specifically targets serine hydrolases with selective small molecules. The ability to target and modulate serine hydrolases has potential to develop new medicines in many therapeutic areas. Abide is located in San Diego. To learn more, visit www.abidetx.com.

SOURCE Abide Therapeutics

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