What is ALS?
ALS is a neurological disease characterized by the progressive damage and loss of motor neurons, which are the nerve cells that relay instructions to move from the brain to the muscles. As motor neurons are damaged or lost, muscles cannot receive the necessary instructions, and so cannot move. Over time the neurons start to die.
As ALS progresses, many patients have difficulty controlling their jaw and throat muscles. Without control over these muscles, patients have difficulty swallowing, which means that saliva builds up in the mouth, resulting in drooling.
How does NT0502 work?
NT0502 uses a micro-particle delivery system, which contains an inhibitor that acts on the nerve cells controlling activity of the salivary glands.
Humans have three pairs of salivary glands in the mouth and throat. These glands are controlled autonomically or involuntarily. When nerve signals from the brain instruct the salivary glands to secrete saliva, a neurotransmitter called acetylcholine is released from the nerve cell endings. This neurotransmitter binds to receptors found on the salivary glands, increasing the production and secretion of saliva.
The small molecule contained in NT0502 is an inhibitor that binds to the receptor to which acetylcholine would normally bind By blocking this receptor (called a muscarinic receptor), NT0502 reduces the amount of saliva that is produced and secreted by the salivary glands.
NT0502 in clinical trials
Neos Therapeutics conducted a Phase 1 clinical trial to evaluate the safety and tolerability of NT0502 in 30 healthy volunteers. Participants received a single dose of NT0502 and oxybutynin, another medication that inhibits saliva production, in one of four modified-release oral disintegrating tablet formulations.
Top-line data from the study are expected in early 2020 and will provide insight into the pharmacokinetics (movement in the body) of the four formulations. This information will be used to guide the design of both the final formulation and dosages for future clinical trials testing the treatment.
Last updated: Jan. 14, 2020
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