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Abide Therapeutics Announces Initiation of Phase 2 Clinical Trial of ABX-1431 in Tourette Syndrome

Abide Therapeutics Announces Initiation of Phase 2 Clinical Trial of ABX-1431 in Tourette Syndrome First patient dosed in Phase 2 trial to evaluate the efficacy of ABX-1431 in treating adult patients with Tourette Syndrome

PR Newswire

SAN DIEGO, Oct. 16, 2018

SAN DIEGO, Oct. 16, 2018 /PRNewswire/ -- Abide Therapeutics, a clinical-stage biopharmaceutical company focused on developing novel therapeutics targeting the serine hydrolase family of enzymes, announced today the initiation of Phase 2 clinical development of ABX-1431, a first-in-class investigational monoacylglycerol lipase (MGLL) inhibitor, in patients with Tourette Syndrome (TS). TS is a neurodevelopmental disorder characterized by sudden, involuntary movements and vocal tics.  

ABX-1431 is an oral, potent, and selective MGLL inhibitor being evaluated for its potential to treat a broad range of central nervous system (CNS) diseases by regulating neurotransmitter release. Inhibition of MGLL by ABX-1431 can modulate the endocannabinoid system selectively in areas where circuits are activated. In a Phase 1b TS trial conducted by Abide in 2017, patients receiving a single dose of ABX-1431 consistently showed a positive impact on key measures of TS symptoms. ABX-1431 was generally well-tolerated in this trial.

"We are excited to continue advancing our first-in-class MGLL inhibitor ABX-1431 in Tourette Syndrome," said Alan Ezekowitz, MBChB, DPhil, CEO and President of Abide Therapeutics. "The results of our initial Tourette trial indicated that selective modulation of endocannabinoid tone in overactive circuits by oral administration of ABX-1431 has the potential to treat the whole patient, beginning with blunting the urge that drives the need to tic, and may affect common co-morbid conditions like anxiety and obsessive-compulsive disorder. This, together with the tolerability profile of ABX-1431, supports the further development of ABX-1431 in Tourette Syndrome."

"New classes of medicines are greatly needed in Tourette Syndrome," commented Amanda Talty, President and CEO of the Tourette Association of America. "Currently, the two classes of medicines commonly used to treat Tourette can have a number of concerning side effects. Endocannabinoid modulation may represent a treatment alternative, and we are happy to see additional studies being conducted to test this potential treatment approach."

About the Phase 2 Trial

This Phase 2 multicenter clinical trial of ABX-1431, conducted in the European Union, is designed to evaluate the efficacy, safety, tolerability, and dosing regimen of ABX-1431 in treating up to 48 adult patients with TS or Chronic Motor Tic Disorder (CMTD). It is a double-blind, randomized, placebo-controlled trial that will evaluate ABX-1431, dosed once daily for up to 8 weeks, with an open-label extension arm for an additional 4 weeks. 

The primary endpoint of this trial is the change from baseline in Total Tic Score of the Yale Global Tic Severity Scale (YGTSS) compared with placebo. The study includes additional measures of tics from the patient and clinician perspective, and will measure the premonitory urge preceding tics. The study will also explore the potential impact of ABX-1431 on additional psychological problems that can accompany TS, such as attention-deficit hyperactivity disorder, obsessive-compulsive disorder, anxiety, and depression. 

About ABX-1431

ABX-1431 is a first-in-class, small-molecule inhibitor of monoacylglycerol lipase (MGLL) currently being investigated in clinical trials for the treatment of neurological disorders. MGLL is a serine hydrolase which regulates one of the body's key natural activators of the cannabinoid receptors, 2-arachidonoylglycerol (2-AG), which signals through the cannabinoid receptors CB1 and CB2 to modulate neurotransmission and inflammatory signaling, respectively. Potent and selective inhibition of MGLL by ABX-1431 prevents the breakdown of 2-AG and amplifies cannabinoid receptor signaling in neural circuits, which are often dysregulated in disease states.

Direct cannabinoid receptor activation by cannabis derivatives and synthetic agonists has demonstrated clinical benefits in a number of central nervous system (CNS) diseases associated with overactive neurotransmission, including spasticity associated with multiple sclerosis, chronic pain, and Tourette Syndrome. However, exocannabinoid use is limited by its broad activity and concomitant CNS adverse effects, and by challenges with administration and dosing precision. ABX-1431, as an investigational oral therapy, provides the ability to modulate the endocannabinoid receptors selectively in areas where circuits are activated. The ability to correct aberrant neurotransmission suggests that ABX-1431 may have broad utility in a wide range of neurological diseases.

About Abide Therapeutics

Abide Therapeutics is focused on developing first-in-class drugs for serious diseases with significant unmet medical need. An innovative discovery platform and a library of proprietary small molecules allow Abide to address biological pathways with potential therapeutics that enhance the body's normal physiological response to disease. The platform enables Abide to efficiently identify, modify, and validate small-molecule inhibitors that target serine hydrolases, a highly relevant but under-explored class of enzymes. Abide's initial area of focus is on addressing neurological disorders with limited treatment options through the endocannabinoid pathway.

Abide has offices in San Diego, California and Princeton, New Jersey. To learn more, visit

Media Contact:
Patty Pilon
Abide Therapeutics
Tel: +1 619.244.2679

Investor Contact:
John Graziano
Solebury Trout
Tel: +1 646.378.2942


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SOURCE Abide Therapeutics, Inc.

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Last Updated: 16-Oct-2018