NP001 Fails to Meet Primary, Secondary Endpoints in ALS Trial


The announcement was discouraging to many, as only 2 agents for ALS have been granted FDA approval in as many decades.

Rich Casey, BS, MBA

Topline results from a phase II trial of NP001, an investigational therapy for patients with amyotrophic lateral sclerosis (ALS) and elevated levels of systemic inflammation, did not achieve its primary or secondary endpoints a change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) score and in pulmonary function as measured by vital capacity readings.

The data from the randomized, double-blind, placebo-controlled, multicenter study were presented at the American Academy of Neurology’s (AAN) 70th Annual Meeting in Los Angeles, California. The announcement was discouraging to many, as only 2 agents for ALS have been granted US Food and Drug Administration (FDA) approval in as many decades.

"We recognize the desperate need for advances in treating ALS and are very disappointed with the findings in our confirmatory phase II study of NP001," said Rich Casey, BS, MBA, the chief executive officer of Neuraltus Pharmaceuticals, the therapy’s developer.1 "We are conducting additional analyses of the trial results to determine if and how we will proceed in developing the compound."

The study, which completed enrollment in July 2017, tallied a total of 138 patients, who were administered either NP001 or placebo. Those who received the trial drug were given it at a dose of 2 mg/kg intravenously for 5 consecutive days in month 1, then 3 consecutive days in months 2 through 6. Patients receiving placebo were administered treatment at the same frequency.

In addition to the secondary endpoint of pulmonary function, other secondary outcome measures included time-to-tracheotomy and a change in blood inflammatory biomarkers.

In January, the final patient visit was completed for the trial. At the time, Robert Miller, MD, the study’s principal investigator, a clinical professor of Neurosciences, Stanford University, and Director of the Forbes Norris ALS Research Center at California Pacific Medical Center in San Francisco, called the outcomes “highly anticipated by researchers, patients, and caregivers, as it has the potential to reaffirm the preliminary evidence from the company’s first phase II clinical trial of this investigational compound.”2

Casey had originally stated last July that Neuraltus planned to meet with the FDA to review clinical and pharmacology data and discuss the required next steps to advance NP001 toward regulatory filing—if the results were positive.

This news, while clearly disappointing, does not mark the end of the road for NP001. The company announced in September of last year that it had partnered with the John A. Burns School of Medicine at the University of Hawaii at Manoa to initiate a phase I-b, placebo-controlled biomarker trial of the therapy in individuals with Alzheimer disease (AD). The intention is to discover the effect of a single dose on blood markers of inflammation in those with mild-to-moderate AD.

"NP001 has shown promising results in reducing systemic inflammation levels in patients with other neurodegenerative diseases, and we hope that this study will show that it works similarly in Alzheimer's disease,” Casey said at the time.3 “This is the first clinical study of NP001 in Alzheimer's disease and we look forward to examining the results to see whether the compound impacts inflammation levels in this patient population."


1. Neuraltus Pharmaceuticals Reports Results from Phase 2 NP001 Study

in Amyotrophic Lateral Sclerosis (ALS). Los Angeles, CA: Neuraltus; April 26, 2018. Accessed April 26, 2018.

2. Neuraltus Pharmaceuticals Announces Completion of


Patient Visit in Confirmatory Phase 2 Study of NP001 for the Treatment of Amyotrophic Lateral Sclerosis. San Bruno, CA: Neuraltus; January 4, 2018. Accessed April 26, 2018.

3. Neuraltus Pharmaceuticals and the University of Hawaii at Manoa Announce Initiation of Phase 1b Study of NP001 in Patients with Mild-to-Moderate Alzheimer's Disease. San Bruno, CA, and Honolulu, HI: Neuraltus; Accessed April 26, 2018.

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