Concert Pharmaceuticals is developing CTP-354, a novel, potentially first-in-class, non-sedating, once-daily oral formulation aimed at treating conditions like multiple sclerosis (MS), cerebral palsy, amyotropic lateral sclerosis (ALS), spinal cord injury, and hereditary paraplegia, all of which contribute to spasticity in humans.

As part of developing CTP-354, Concert is utilizing its deuterated chemical entity (DCE) platform, which involves the addition and modification of existing, approved drugs or those studied clinically beforehand with deuterium to improve safety and clinical efficacy.

How CTP-354 Works

Chemically, CTP-354 is a subtype-selective GABAA receptor modulator. The GABAA receptors are abundant in the nervous system, and their main ligand, gamma-aminobutyric acid (GABA), is a major inhibitory neurotransmitter. These are involved in the reduction or inhibition of conduction of nerve impulses. GABA inhibitory neurotransmission is important for normal functioning of the nervous system and conduction of nerve impulses.

Antigen-presenting cells (APC, immune cells) in autoimmune disorders possess these GABAA receptors, and GABA treatment has previously been shown to decrease inflammatory cytokine production in peripheral macrophages. T-cell mediated autoimmunity and development of inflammatory responses have also been shown to be reduced with GABA treatment. As a result, these GABAA receptor modulators help bind the GABA neurotransmitter and reduce synaptic action potential, diminishing autoimmune responses over time.

History of CTP-354

Preclinical testing of the safety and efficacy of the drug in animal models was successful, as the observed pharmacological activity and serum levels of GABA were shown to be similar to previous generations of modulators, such as benzodiazepines. However, treatment with deuterium proved beneficial in reducing side effects compared to benzodiazepines.

In Phase 1 clinical trials in healthy volunteers with a multiple ascending dose to check for safety and tolerability of the drug, no sedation or ataxia was reported, and the drug had a healthy pharmacokinetic and pharmacodynamic safety profile in all cohort of the study.

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