Rare Disease Report

First Patients with NMO Dosed in ABX 1431 Study

OCTOBER 10, 2017
Mathew Shanley
Earlier today, it was announced that dosing was initiated in a Phase 1b study of ABX-1431 in patients with neuromyelitis optica (NMO) and other similar neuroinflammatory disorders associated with central pain.

The study is being conducted at two clinical centers in the United Kingdom, and is intended to evaluate the effect of ABX-1431 on chronic central pain.

NMO, also known as Devic’s disease or Devic’s syndrome because of related studies conducted by Eugene Divic in the late 1890’s, is a heterogenous condition, characterized by severe autoimmune disorders in the central nervous system. The disease selectively attacks astrocytes of the optic nerves and spinal cord, and presents with progressive loss of sight and spinal cord function.

The drug, developed by Abide Therapeutics, is a first-in-class, small molecule inhibitor of monoacylglycerol lipase (MGLL) to treat neurological disorders, pain, and neuroinflammation. ABX-1431 is designed to treat both pain and neuroinflammation, and does so by raising the levels of 2-Arachidonoylglycerol (2-AG) in the brain and simulating the positive analgesic effects of marijuana. Through this method, the therapy has the potential to provide clinical benefit for a variety of neurological diseases, including multiple sclerosis and Alzheimer’s disease.

In addition to the modulation of 2AG levels, MGLL inhibition by ABX-1431 depletes the supply of the inflammatory signaling molecule arachidonic acid, thus providing another potential mechanism to assuage pain and swelling.

"Restoring neurotransmitter balance represents a common thread of the mode of action of ABX-1431, and this study in NMO patients provides an opportunity to test the ability of the drug to provide relief to patients with a painful central demyelinating condition," said Alan Ezekowitz, MBChB, DPhil, CEO and President of Abide Therapeutics in a press release.

The Phase 1b study is a placebo-controlled crossover design, and ABX14-31 has already successfully completed dosing in a first-in-human, placebo-controlled, Phase 1a study. The Phase 1a began dosing in July 2015, and throughout, the drug was well-tolerated and displayed no serious adverse events (AEs).

ABX-1431 is also currently undergoing evaluation in Tourette Syndrome and functional dyspepsia.

In September 2016, Celegene Corporation implemented its opportunity to acquire ex-US rights to ABX-1431. The company will be held accountable for development costs for all indications from Phase 2 clinical trials and beyond. Abide will retain the drug’s US rights and is conducting several Phase 1b studies to investigate indications where endocannabinoid modulation may affect disease progression.

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