Neuvivo, a late-clinical stage biopharmaceutical company, today announced it submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for NP001 (sodium chlorite infusion) to treat amyotrophic lateral sclerosis (ALS). NP001 is an investigational treatment that, if approved, could be the first disease-modifying therapy for ALS that restores balance over uncontrolled inflammation within the body’s own innate immune system. The NP001 treatment is unique as it targets ALS from a more immunologic mechanism than neurologic.

There are currently no medications available for ALS that preserve breathing function or extend life by more than 2-3 months. Approximately 30,000 adults in the U.S. are living with ALS and 1 in 300 people will be diagnosed in their lifetime.

“ALS is a devastating disease and patients who are fighting ALS need much better treatment options,” said Ari Azhir, PhD, Founder and CEO of Neuvivo. “Our organization was built on our passion and commitment to help those struggling with ALS by finding new treatments that can make a meaningful difference in their quality of life. 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.”

Dr. Namita Goyal, MD, Clinical Professor of Neurology at University of California, Irvine, and Neuromuscular Medicine specialist, said: “The submission of NP001 for FDA approval brings new hope to people living with ALS, their families and caregivers. We are truly honored to have collaborated with such a distinguished investigator team and thankful for Neuvivo’s resilience with continuing to develop NP001 to reach this critical milestone.”

About ALS

ALS is a relentlessly progressive neurodegenerative disease without cure that, over time, takes away a person’s muscle function and impacts their ability to walk, talk, eat and — ultimately — breathe. Even with available treatments, the current life expectancy of a person with ALS is about 2-5 years after symptoms appear, with death usually resulting from respiratory failure.¹ There are currently no medicines available for ALS that preserve breathing function or extend life by more than 2-3 months. Approximately 30,000 adults in the US are living with ALS,² and 1 in 300 people will be diagnosed in their lifetime.³

About NP001

NP001 is a transformative, investigational therapy that could become the first immunotherapy for ALS designed to restore balance within a dysfunctional innate immune system where pro- and anti-inflammatory processes are no longer in equilibrium. By regaining balance in this natural process, NP001 may help slow the progression of ALS and preserve skeletal muscle function, including the diaphragm. To date, no other therapy has been able to preserve lung function. If approved, NP001 would be the first disease-modifying treatment with this novel mechanism of action and potentially have a meaningful effect on the lives of patients with ALS.

NP001 has been granted Orphan Drug and Fast Track Designations by the US Food and Drug Administration (FDA) and is eligible for Accelerated Approval and Priority Review, which could shorten the time to a potential approval. A New Drug Application (NDA) for NP001 in ALS was filed in October 2024.

Prior studies established that NP001 is generally safe and well tolerated.

About Neuvivo

Neuvivo is a private, late-clinical stage biopharmaceutical company dedicated to creating and delivering advanced treatments for ALS and other neurodegenerative diseases. Neuvivo has developed a proprietary platform that includes a patented formulation for NP001 and its manufacture. For more information, please visit www.Neuvivo.com.

References

1. Masrori P, Van Damme P. Amyotrophic lateral sclerosis: a clinical review. European journal of neurology. 2020 Oct;27(10):1918-29

2. Mehta P, et al. Prevalence of amyotrophic lateral sclerosis in the United States, 2018. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 2023; 24(7–8), 702–708. https://doi.org/10.1080/21678421.2023.2245858

3. Martin, S., Al Khleifat, A., & Al-Chalabi, A. (2017). What causes amyotrophic lateral sclerosis?. F1000Research, 6, 371. https://doi.org/10.12688/f1000research.10476.1

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