Alterity Therapeutics has completed a Phase 1 human clinical trial of ATH434, a chemical that inhibits the aggregation of α-synuclein and tau proteins, which lead to neurodegenerative diseases. While the company is currently placing its focus on multiple system atrophy, which causes loss of function in multiple areas and is similar to Parkinson’s disease, such a drug could potentially be useful for other proteostasis-related neurodegenerative diseases, including Alzheimer’s.
The Phase 1 safety trial reported side effects similar to that of placebo, showing that this drug is clinically safe, and it accumulates in cerebrospinal fluid in greater concentrations than in animal models. Its efficacy, however, remains untried in human beings. Alterity plans to launch a Phase II trial in 2021 and has received orphan drug designation for multiple system atrophy from the FDA and EU.