Neuraltus Pharmaceuticals, Inc., a privately-held pharmaceutical company dedicated to developing innovative therapeutics for neurodegenerative diseases, today announced findings from a confirmatory Phase 2 study of its investigational therapy, NP001, in ALS patients with elevated levels of systemic inflammation. The study, which enrolled 138 patients, did not meet 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 top-line data will be presented during a podium presentation at the American Academy of Neurology’s 70th Annual Meeting in Los Angeles on April 26, 2017 at 8:00 a.m. PT.
“We recognize the desperate need for advances in treating ALS and are very disappointed with the findings in our confirmatory Phase 2 study of NP001,” said Rich Casey, chief executive officer, Neuraltus Pharmaceuticals. “We are conducting additional analyses of the trial results to determine if and how we will proceed in developing the compound.”
About the Confirmatory Phase 2 Study of NP001
The confirmatory Phase 2 study was a randomized, double-blind, placebo-controlled, multicenter study that enrolled 138 subjects in North America with ALS and evidence of systemic inflammation. Patients received either NP001 2 mg/kg or placebo over a period of 6 months. The study was designed to evaluate the change from baseline in ALSFRS-R during the study period. Secondary objectives include a change in pulmonary function as measured by vital capacity readings and inflammatory biomarkers. Further information about the study is available at https://clinicaltrials.gov.
In the first Phase 2 study of NP001, Neuraltus assessed the safety, tolerability and preliminary efficacy of two dose levels of NP001 versus placebo using the ALSFRS-R. A secondary analysis of the study results suggested that increased levels of a biomarker for systemic inflammation, C-reactive protein (CRP), may indicate which patients are more likely to respond to NP001.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) is a rare and fatal neurodegenerative disease affecting approximately 400,000 people worldwide, characterized by degeneration of motor neurons in the spinal cord and brain. The cause of the disease is currently unknown, but there is increasing evidence that implicates neuroinflammation with the progression of the disease. It is believed that in people with ALS, there are increased levels of inflammatory (activated) macrophages, a type of white blood cell, resulting in the release of factors in the central nervous system that damage motor neurons.
About Neuraltus Pharmaceuticals, Inc.
Neuraltus Pharmaceuticals, Inc. is a privately-held pharmaceutical company dedicated to developing and commercializing innovative therapeutics that address critical unmet needs for patients and physicians in the treatment of neurodegenerative diseases.
For more information, please visit www.neuraltus.com.