Neuraltus Pharmaceuticals 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.
"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."
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.
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.