NPT520-34 is an experimental therapy being developed by Neuropore Therapies to treat Parkinson’s disease and amyotrophic lateral sclerosis (ALS). The treatment was granted orphan drug designation by the U.S. Food and Drug Administration for ALS.

What is Parkinson’s disease?

Parkinson’s disease is a progressive neurological disorder characterized by the degeneration of nerve cells in the brain. The symptoms of the disease include tremors, muscle stiffness, and difficulty walking.

The causes of Parkinson’s disease are unknown, but researchers have found neuroinflammation or inflammation around the nerve cells, as well as protein aggregation in the brains of patients. Neuroinflammation can speed up the loss of nerve cells in the brain of Parkinson’s disease patients so targeting neuroinflammation is an area of therapeutic interest to researchers.

How does NPT520-34 work?

NPT520-34 is a small molecule therapy that has been shown to reduce neuroinflammation in preclinical studies of Parkinson’s disease. Mice treated with NPT520-34 showed decreases in Lewy bodies (abnormal protein deposits in the brain that interfere with nerve cell function), reductions in a protein called TSPO (a measure of immune cell activation), and improvement in dopamine levels. The treated animals also showed higher grip strength, indicating that their motor function was improved.

The compound is administered orally and is small enough to be able to cross the blood-brain barrier, a highly selective, semipermeable membrane that isolates the brain from the blood circulating in the body.

NPT520-34 in clinical trials

A Phase 1 clinical trial (NCT03954600) assessed the safety, tolerability, and pharmacokinetics (movement in the body) of NPT520-34 in 49 healthy volunteers.

The trial had two phases: an initial single-dose ascending phase, in which NPT520-34 was given at a single dose to all participants, with the possibility of incremental adjustments, and a second multiple-ascending dose phase, in which the compound was administered at different doses, again with the possibility of small adjustments.

Neuropore Therapeutics announced results of the trial which found NPT520-34 to be safe and well-tolerated. Based on these findings, the company is designing a clinical trial that will assess the safety and tolerability of NPT520-34 in Parkinson’s disease patients.


Last updated: Jan. 16, 2020


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