Neuvivo, a biopharmaceutical company focused on neurodegenerative diseases, has announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for NP001, an investigational immunotherapy for amyotrophic lateral sclerosis (ALS). If approved, NP001 could become the first disease-modifying therapy for ALS that targets the underlying immune dysfunction associated with the disease.
Targeting Immune Dysfunction in ALS
ALS, a progressive neurodegenerative disease affecting approximately 30,000 adults in the U.S., leads to the gradual loss of muscle function, impacting the ability to walk, talk, eat, and breathe. Current treatments offer limited benefits, typically extending life by only a few months and failing to preserve lung function, a critical factor in patient survival. NP001 (sodium chlorite infusion) represents a novel approach by aiming to restore balance within the body's innate immune system, addressing the uncontrolled inflammation that contributes to ALS progression.
Clinical Evidence and Biomarker Analysis
NP001's NDA submission is supported by data from Phase 2a, Phase 2b, and overall survival studies, along with biomarker analyses. These studies suggest that NP001 may substantially preserve lung function and extend overall survival, particularly in patients identified through therapeutic biomarkers as having underlying, uncontrolled inflammation. Specifically, data indicated that plasma TGFB1 levels were significantly higher in patients with high C-reactive protein (CRP) levels treated with NP001, suggesting the therapy may help reset the innate immune system.
In a retrospective study analyzing up to 11 years of patient data, a subset of patients aged 65 and younger experienced an extended survival of 10.8 months (Hazard ratio: 0.69; 95% CI: 0.50, 0.95). Furthermore, in patients with moderate underlying inflammation and low baseline creatinine levels, median overall survival was extended by 17.1 months compared to placebo (45.5 months vs 28.4 months, respectively; p = 0.005).
Expert Commentary
Ari Azhir, PhD, Founder and CEO of Neuvivo, stated, "We are thrilled to have submitted NP001 for FDA approval as the treatment platform may substantially preserve lung function and extend overall survival by up to a year, especially in patients identified through therapeutic biomarkers as having underlying, uncontrolled inflammation as a result of ALS."
Matthew W Davis, MD, RPh, Chief Medical Officer of Neuvivo, added, "Based on the evidence shown across the Phase 2a, Phase 2b, and Overall Survival studies as well as the biomarker analysis, we believe submitting the New Drug Application to the FDA for NP001 offers great promise to the ALS community."
Regulatory Pathway and Future Directions
NP001 has been granted Orphan Drug and Fast Track Designations by the FDA, making it eligible for Accelerated Approval and Priority Review. Neuvivo plans to continue working closely with the FDA throughout the review process to bring NP001 to ALS patients as quickly and efficiently as possible. The company's ongoing research aims to further elucidate the mechanisms of action of NP001 and identify additional biomarkers that can predict treatment response.
