Investigational therapy NP001 failed to improve disease severity and pulmonary function in a Phase 2 confirmatory trial for amyotrophic lateral sclerosis (ALS) patients with elevated levels of systemic inflammation, Neuraltus Pharmaceuticals announced.
The findings, showing the trial failed to meet its primary and secondary goals, were recently shared during the American Academy of Neurology’s 70th Annual Meeting April 26 in Los Angeles. The presentation was titled “Randomized Phase 2B trial of NP001, a Novel Immune Regulator, in ALS.”
“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,” Rich Casey, CEO of Neuraltus Pharmaceuticals, said in a press release. “We are conducting additional analyses of the trial results to determine if and how we will proceed in developing the compound.”
Inflammation is thought to help drive ALS progression. NP001 is being investigated for its potential to reduce neuroinflammation by regulating the activity of immune cells in the central nervous system.
In a prior Phase 2 trial (NCT01281631), NP001 treatment was found to slow disease progression in ALS patients, compared with placebo. A later analysis, however, suggested that patients with high levels of inflammation were more likely to respond to the therapy.
This led Neuraltus to design a confirmatory Phase 2 trial (NCT02794857), testing the medicine in patients with evidence of systemic (whole body) inflammation.
The trial included 138 ALS patients, mean age of 58 years, who had had symptoms for a mean of 19 months.
Participants were randomly assigned NP001 or a placebo for six months. Treatment was given intravenously (directly into the blood) for five consecutive days in the first month, and for three consecutive days for the second through the sixth months.
The study’s primary goal was to determine if NP001 improves disease progression and severity, as measured by the ALS Functional Rating Score Revised. Additional objectives included improvements in lung function and change in the levels of blood inflammatory biomarkers.
To date, 86 patients have completed treatment, with a low incidence of infusion-related adverse events, and no discontinuations due to treatment-related adverse effects.
However, after six months of treatment, patients did not show improved ALS Functional Rating Scale Revised scores. Pulmonary function, measured by vital capacity readings, also had not improved.