Neuvivo aims to join ALS field with immunotherapy candidate

Neuvivo has formally submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for its novel amyotrophic lateral sclerosis (ALS) immunotherapy, NP001. This drug, consisting of a sodium chlorite infusion, aims to broaden the range of available disease-modifying therapies for ALS. According to Neuvivo's CEO, Ari Azhir, NP001 has the potential to significantly preserve lung function and extend patient survival by up to a year.

The NDA submission is backed by a range of data sources, including biomarker analyses and data from two critical studies: a Phase IIa study (NCT01281631) and a Phase IIb study (NCT02794857). Additionally, overall survival studies have contributed to the application, according to Neuvivo's Chief Medical Officer, Dr. Matthew Davis, as mentioned in a press release dated October 7.

ALS is a severe and swiftly progressing neurodegenerative condition characterized by the damage and eventual death of nerve cells in the brain and spinal cord. This leads to muscle weakness, atrophy, and spasticity. Neuvivo argues that current ALS treatments do not adequately support lung function preservation in patients.

Presently, the ALS treatment market includes Sanofi's Rilutek (riluzole) and Mitsubishi Tanabe’s Radicava/Radicut (edaravone). Radicava aims to decelerate the progression of ALS by mitigating nerve damage, while Rilutek, first approved by the FDA in 1995, extends life expectancy by approximately three months. Sales figures from GlobalData reveal that Rilutek generated $105 million in 2001, whereas Radicava's sales reached $586 million in 2023.

NP001 is engineered to regulate the activation of macrophages and transition immune cells from a neurotoxic state to a neuroprotective one. This immunotherapy aims to reinstate the balance between pro- and anti-inflammatory processes within the immune system, thereby slowing ALS progression.

Interestingly, NP001 was initially investigated by Neuraltus Pharmaceuticals, which encountered negative outcomes in its Phase IIb clinical trial in April 2018. This randomized, double-blind, placebo-controlled study did not meet its primary endpoint of changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) or its secondary endpoint of evaluating pulmonary function. Following these results, Neuraltus Pharmaceuticals went out of business.

Neuvivo, however, conducted further analyses of the Phase IIb study and identified a subgroup of participants who exhibited a significantly slower decline in ALSFRS-R and vital capacity scores compared to those given a placebo.

Developing effective disease-modifying treatments for ALS has proven incredibly challenging, largely due to the disease's unclear causes and complex pathophysiology. Consequently, current treatment options only modestly extend life expectancy and do not effectively address the underlying mechanisms of ALS. This highlights a critical need for innovative therapies that go beyond merely treating the symptoms.

According to GlobalData's Amyotrophic Lateral Sclerosis Updated Market Analysis and Forecast to 2029 report, several pipeline candidates with varied mechanisms of action are in development. Nevertheless, the difficulty in successfully advancing these treatments remains high. For instance, Amylyx’s antisense therapy Relyvrio, initially deemed promising, failed to achieve its primary endpoints in a Phase III study (NCT05021536) and was subsequently withdrawn from the market.

Neuvivo's NP001 represents a potential new avenue in tackling ALS, offering hope for a more effective therapeutic option that could extend the survival and improve the quality of life for patients suffering from this devastating disease.