The signal was its announcement that it had completed the last patient visit in the study.
The first study, completed in 2012, assessed the effectiveness of two intravenous doses of NP001 in 136 patients, compared with a placebo. The yardstick researchers used was the ALS Functional Rating Score Revised, which evaluates patients’ function and ability to carry out daily activities.
NP001 decreased levels of patients’ inflammatory markers, and was safe, researchers said. Importantly, the data suggested that patients with the highest levels of C-reactive protein, a biomarker for chronic inflammation, responded best to the drug.
Researchers published their study, “Randomized phase 2 trial of NP001, a novel immune regulator,” in the journal Neurology: Neuroimmunology & Neuroinflammation.
“The outcome of the confirmatory Phase 2 study of NP001 for the treatment of ALS is highly anticipated by researchers, patients and caregivers, as it has the potential to reaffirm the preliminary evidence from the company’s first Phase 2 clinical trial of this investigational compound,” Dr. Robert Miller, the study’s principal investigator, said in a press release. He is clinical professor of Neurology & Neurological Sciences at Stanford Medicine and director of the Forbes Norris MDA/ALS Research & Treatment Center at California Pacific Medical Center.
Although the cause of ALS remains unknown, growing evidence indicates that inflammation drives the disease’s progression. This includes findings that high levels of inflammation-generating, activated macrophages — a type of white blood cell — lead to the nerve cell damage in the disease. NP001 works by switching off macrophages’ activated state, preventing the immune cells from entering the brain, Neuraltus said.
The confirmation trial involved 138 ALS patients with chronic inflammation. It was held at a number of locations across North America. Patients received either intravenous NP001 or a placebo for six months. Doses were given over five consecutive days in the first month, and three consecutive days in each of the following months.
The trial’s main objective was to see whether NP001 improved patients’ ALS Functional Rating Scale-Revised score. Secondary objectives included seeing if it could show the progression of patients’ lung function and lower markers of inflammation in their blood.
Patient enrollment was completed in July 2017. Neuraltus said last year that it expected to announce key results of the study at the end of the first quarter of 2018.
“If the study results confirm the previous study’s findings, NP001 could be one step closer to regulatory review,” said Rich Casey, the company’s CEO.