A new analysis of Phase 2 clinical trial data reveals that NP001, an investigational therapy developed by Neuvivo, provided a significant survival benefit of 22 months in a subset of amyotrophic lateral sclerosis (ALS) patients. The findings, presented at the recent ALS Network Research Summit in San Francisco, specifically focused on patients who showed no disease progression during the initial treatment period.
The data showed that among non-progressor patients, those treated with NP001 achieved a median overall survival of 58 months, compared to 36 months in the control group. This substantial survival benefit appears to be linked to the therapy's ability to slow respiratory decline, a crucial factor in ALS progression.
Mechanism of Action and Trial Design
NP001, administered intravenously, works by reprogramming immune cells called macrophages from a proinflammatory state to a noninflammatory state. This mechanism aims to suppress uncontrolled immune responses that contribute to nerve cell damage in ALS.
The analysis drew from two previous placebo-controlled Phase 2 trials that tested NP001 in ALS patients aged 21-80 whose symptom onset occurred within three years prior to enrollment. Participants received either NP001 (at doses of 1 mg/kg or 2 mg/kg) or placebo in monthly cycles over a six-month treatment period.
Key Findings in Non-Progressor Population
Dr. Michael McGrath, Neuvivo's founder and chief scientific officer, presented compelling data showing that:
- 31% of NP001-treated patients showed no disease progression over six months, compared to 14% in the placebo group
- Patients receiving the 2 mg/kg dose experienced significant slowing of lung function decline
- Treatment led to corrections in inflammatory biomarkers and reductions in neurofilament light chain, indicating decreased nerve damage
"One of the most confounding aspects of ALS research is that this disease does not uniformly progress," noted Dr. McGrath, who is also an emeritus professor of medicine at the University of California San Francisco. "My analysis looked at patients whose progression slowed or halted using a standard test for functional change in ALS patients."
Implications for ALS Treatment
These findings carry particular significance as no currently available therapies can preserve breathing function in ALS patients. If approved, NP001 would become the first disease-modifying treatment with this capability, according to Neuvivo.
Dr. Ari Azhir, CEO and co-founder of Neuvivo, emphasized that these results "align well" with previously published data and "add to the body of evidence supporting the clinical benefits of NP001 to treat ALS."
The results are especially promising for patients with inflammatory forms of ALS, as the therapy has consistently demonstrated its ability to slow or halt disease progression in this subset after just six months of treatment. These findings have supported Neuvivo's application to the U.S. Food and Drug Administration (FDA) for NP001's approval in ALS treatment.
