NPT520-34 is a new small molecule oral treatment being developed by Neuropore Therapies for potential use against neurodegenerative disorders like amyotrophic lateral sclerosis (ALS) and Parkinson’s disease. The therapy is believed to reduce neuroinflammation in the brain.

What is ALS?

ALS is a progressive disease characterized by the death of motor neurons, the nerve cells that control the voluntary muscles in the body.  The symptoms of ALS include muscle stiffness, muscle twitching, and a gradual reduction in muscle strength. In the later stages of the disease, patients may become paralyzed, and experience difficulties in swallowing, speaking, and breathing.

One of the main reasons for the death of motor neurons is inflammation in the brain. It is thought that this is caused by the infiltration into the brain of immune cells such as lymphocytes and macrophages, as well as by the activation of brain-specific immune cells. Those brain-specific immune cells are microglia — macrophage-like cells in the brain — and the star-shaped astrocytes, the most common glial cells, which regulate immunity in the brain.

These cells generate reactive oxygen species (ROS), which are toxic to nerve cells.

How does NPT520-34 work?

NPT520-34 is a small molecule that can penetrate the blood-brain barrier.

Studies in animal models showed that NPT520-34 can significantly reduce the production of proteins associated with astrocytes and microglia. This suggests reduced inflammation in the brain.

These studies also showed reduced levels of misfolded superoxide dismutase 1 (SOD1) protein, which is one of the main causes of toxicity associated with ALS.

These findings suggest that NPT520-34 may slow down ALS disease progression by reducing neuroinflammation.

NPT520-34 in clinical trials

A Phase 1, randomized, double-blind, single and multiple ascending dose clinical trial (NCT03954600) was conducted to evaluate the safety, tolerability, and pharmacokinetics (movement in the body) of NPT520-34 in 49 healthy volunteers. The study was conducted in two phases: a single ascending dose phase and a multiple ascending dose phase. An additional arm of the trial examined the pharmacokinetics of a capsule formulation of NPT520-34, taken with and without food.

Neuropore reported the successful completion of the single-dose, safety, and food effect studies in August 2019. Based on those results, the U.S. Food and Drug Administration (FDA) granted NPT520-34 orphan drug designation for the treatment of patients with ALS.

In January 2020, Neuropore reported that NPT520-34 was safe and well-tolerated at all doses tested, including those thought to be therapeutically relevant. The company is currently evaluating the optimal study design and patient population to conduct a safety study in ALS patients.


Last updated: Jan. 20, 2020


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