NP001 is an injected therapy that Neuraltus is developing to slow or stop the progression of ALS.

How NP001 works

ALS is a progressive neurodegenerative disease. Its hallmark is loss of motor neurons, or nerve cells that control muscles. It leads to a decline in muscle control and eventually death.

In later stages of the disease, neuroiflammation can accelerate nerve damage.

The immune system regulates inflammation, and the neuroinflammation in ALS starts with the activation of a type of immune cell called a macrophage.

Activated pro-inflammatory macrophages associated with the disease’s progression are called M1s. NP001 contains chlorite, which can revert macrophages back to an inactive anti-inflammatory state called M2. By reducing neuroinflammation, NP001 aims to slow or stop the progression of ALS.

NP001 in clinical trials

Neuraltus completed a randomized, double-blind, placebo-controlled Phase 1 clinical trial  (NCT01091142) of NP001 in 2010. It tested its safety and participants’ ability to tolerate it.

NP001 reduced levels of an inflammation biomarker in ALS patients, according to an article in the journal Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration. Patients also tolerated it well, researchers said.

Neuraltus then tested NP001 in a randomized, double-blind, placebo-controlled Phase 2 clinical trial (NCT01281631) in 136 ALS patients. The study suggested that the compound might be able to slow or stop the progression of symptoms in patients with high levels of neuroinflammation.

Patients with elevated levels of C-reaction protein in particular may benefit from the treatment, researchers said. The protein is an inflammation biomarker.

Neuraltus published the results in the journal Neurology: Neuroimmunology & Neuroinflammation.

The company is conducting another Phase 2 clinical trial (NCT02794857) to confirm the results. Initial results of the study, whose enrollment was completed in July 2017, are expected in early 2018.

Neuraltus recruited 138 ALs patients with systemic inflammation for the randomized, double-blind, placebo-controlled study. Patients will receive NP001 injected into their bloodstream for five consecutive days in the first month of the study, and over three consecutive days in Months 2 to 6.

The primary goal of the trial is to see whether NP001 can slow the progression of ALS over six months. Researchers will measure progression with the ALS Functional Rating Scale-Revised, or ALSFRS-R.

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