NP001 was an injected therapy that was being developed by Neuraltus to slow or stop the progression of ALS. There is currently no evidence of this investigative therapy still being considered a treatment candidate for ALS due to failed results from a Phase 2 trial.

How NP001 works

ALS is a progressive neurodegenerative disease. Its hallmark is loss of motor neurons, or nerve cells that control muscles. In later stages of the disease, neuroinflammation can accelerate nerve damage, leading to a decline in muscle control and eventually muscle cell death.

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 was thought to revert macrophages back to an inactive anti-inflammatory state called M2. By reducing neuroinflammation, NP001 aimed 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, which tested its safety and participants’ ability to tolerate it.

The trial showed that NP001 reduced levels of an inflammation biomarker in ALS patients. These results were published in the journal Amyotrophic Lateral Sclerosis and Frontotemporal DegenerationPatients 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 an inflammation biomarker called C-reaction protein might particularly benefit from the treatment, researchers said. 

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

The company then conducted another Phase 2 clinical trial (NCT02794857to confirm the results. The randomized, double-blind, placebo-controlled study recruited 138 ALs patients with systemic inflammation. Participants received NP001 injected into their bloodstream for five consecutive days in the first month of the study, and over three consecutive days in months two to six.

The primary goal of this trial was to see whether NP001 could slow the progression of ALS over six months. Researchers measured disease progression using the ALS functional rating scale-revised (ALSFRS-R). However, the results were negative and the trial failed to meet both the primary and secondary endpoints. 

There is currently no evidence suggesting that NP001 is still being considered a treatment candidate for ALS.

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