Neuvivo has submitted a New Drug Application (NDA) for its investigational amyotrophic lateral sclerosis (ALS) immunotherapy to the US Food and Drug Administration (FDA).
Neuvivo hopes its candidate, NP001 (sodium chlorite infusion), will diversify the disease-modifying therapy market for ALS, with CEO Ari Azhir adding the drug can “substantially preserve lung function and extend overall survival by up to a year”.
The NDA is supported by data from biomarker analyses and from a Phase IIa study (NCT01281631), a Phase IIb study (NCT02794857) and overall survival studies, said Neuvivo CMO Dr. Matthew Davis in the 7 October press release.
ALS is a rapidly progressing, fatal neurodegenerative disorder that causes damage and the eventual death of nerve cells in the brain and spinal cord. ALS patients suffer from muscle weakness, atrophy, and spasticity. Neuvivo claims the current treatment market lacks the means to preserve lung function in ALS patients.
Currently approved ALS treatments include Sanofi’s Rilutek (riluzole) and Mitsubishi Tanabe’s Radicava/Radicut (edaravone). Radicava is intended to slow the progression of ALS by preventing nerve damage. Rilutek was the first drug to gain FDA approval for the treatment of ALS in 1995 and prolongs life by approximately three months. According to GlobalData, Rilutek saw sales of $105m in 2001 while Radicava generated total sales of $586m in 2023.
GlobalData is the parent company of Pharmaceutical Technology.
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By GlobalDataNP001 is designed to regulate macrophage activation and restore immune cells from a neurotoxic state to a neuroprotective one. In doing so, the investigational immunotherapy re-establishes the balance of pro- and anti-inflammatory processes in a patient’s immune system to slow the progression of ALS.
The immunotherapy candidate was previously studied by Neuraltus Pharmaceuticals, which reported negative results for the Phase IIb study of NP001 in April 2018. The 138-patient, randomised, double-blind, placebo-controlled study failed to meet its primary endpoint of a change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R). The trial’s secondary endpoint evaluating pulmonary function was also missed. Neuraltus Pharmaceuticals went out of business after the failed study.
After conducting pre-specified and post hoc analyses of the Phase IIb study, Neuvivo identified a subgroup of participants whose decline in ALSFRS-R and vital capacity scores was significantly slower than placebo.
According to GlobalData Healthcare analysts, developing disease-modifying treatments for ALS has been notoriously challenging, with the high trial failure rates being attributed to the “disease’s unclear aetiology and complex pathophysiology”. As a result, the only available treatment options lack mechanistic efficacy and only marginally prolong life, leaving an urgent need to pivot to innovative mechanisms that can tackle more than the symptoms of ALS.
GlobalData’s Amyotrophic Lateral Sclerosis Updated Market Analysis and Forecast to 2029 report notes several pipeline candidates with diversified mechanisms of action (MOA) in development, but challenges remain with getting these across the finish line. Initially touted as an innovative by GlobalData’s analysts, Amylyx’s antisense therapy Relyvrio failed to achieve its endpoints in a Phase III study (NCT05021536) for ALS. The company formally announced the removal of Relyvrio from the market following the failure.
