Significant Breakthroughs in Preserving ALS Lung Function and Survival Unveiled in Peer-Reviewed Studies of Novel Immunotherapy Platform NP001

• New data from an Overall Survival study, demonstrate that NP001, an investigational immunotherapy platform, may help adults living with ALS survive up to a year longer
• Biomarker Identification study shows with practical biomarkers, NP001 significantly preserved lung function and extended survival by up to 17 months
• First time that a treatment affecting the immune system is linked to long term survival in ALS
• Peer-reviewed studies support New Drug Application (NDA) Neuvivo recently submitted to the FDA for approval

PALO ALTO, Calif.--(BUSINESS WIRE)--Neuvivo, a late-clinical stage biopharmaceutical company, today announced promising results from two groundbreaking publications on its investigational immunotherapy platform, NP001. The Overall Survival study demonstrated substantially extended survival in people living with ALS who received the investigational treatment. The second analysis identified the importance of underlying inflammation and the use of practical biomarkers to predict greater responses with preserving lung function and extending survival. The results of the overall survival study and the biomarker identification study were published in Biomedicines.

Neuvivo submitted a New Drug Application (NDA) for NP001 in October to the U.S. Food and Drug Administration (FDA) for approval.

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.

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 two to five years after symptoms appear, with death usually resulting from respiratory failure. 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.

Michael McGrath, MD, PhD, co-founder and Chief Scientific Officer at Neuvivo and Emeritus Professor of Medicine at University of California, San Francisco, said: “By restoring balance to the innate immune system of ALS patients, NP001 could be the first treatment that zeros in on a root cause of disease progression to slow loss of muscle function. In addition to the new data suggesting an extended survival benefit for people aged 21-65, NP001 is also the first ALS treatment that has been shown to preserve lung function, a major cause of morbidity and mortality in ALS patients.”¹

Overall Survival Study

Neuvivo clinical investigators conducted a blinded, placebo-controlled, retrospective study looking at up to 11 years of patient data that demonstrated a pronounced extension of survival in people living with ALS (n=268). The study followed the original participants who were initially treated with NP001 for six months during its Phase 2a and 2b trials to determine the therapy’s long-term impact on survival. The total population (ITT), which included adults 80 years old and under, resulted in extended survival of 4.8 months (Hazard Ratio: 0.77; 95% CI: 0.57, 1.03). However, a subset of people 65 years of age and younger had an extended survival of 10.8 months (Hazard ratio: 0.69; 95% CI: 0.50, 0.95).

Identifying Practical Biomarkers Predicting Lung Function and Survival

Neuvivo’s biomarker identification study showed results from an analytical review of specific patients with moderate levels of inflammation (plasma C-reactive protein (hsCRP) level of > 1.13 mg/L) who participated in the 11-year Overall Survival study. Serum creatinine was also used in this analysis as an indicator of underlying inflammation, since muscle is a major source of amino acids required to produce C-reactive protein (CRP), which is a protein produced by the liver that can indicate inflammation in the body.

In the biomarker study, median overall survival was extended by 17.1 months compared with a placebo (45.5 months vs 28.4 months, respectively; p = 0.005) in patients with moderate underlying inflammation and low baseline creatinine levels treated with six cycles of NP001. This result is consistent with the greatest drug effect demonstrated in patients with ongoing innate immune activation correlated with muscle consumption.

CRP production in ALS patients consumes muscle, thereby lowering creatinine — the blood marker for muscle mass.² It is worth noting that the same ALS patient group leveraging both baseline CRP and creatinine biomarkers had a 46.7% slower rate of lung capacity loss compared with a placebo (p = 0.02), further supporting the importance of respiratory function on survival.¹

Matthew Davis, MD, RPh, Chief Medical Officer of Neuvivo, said: “We will continue to work closely with the FDA throughout the review process to move NP001 toward a potential regulatory approval as quickly and efficiently as possible. We are so inspired by the people who participated in our trials and their families, the trial investigators and advisory committees, the ALS community and all our partners and team. You are truly what continues to drive our effort to make this substantiative therapy accessible to ALS patients.”

Ari Azhir, PhD, founder and CEO of Neuvivo, said: “We’re thrilled to share the results of these two studies of our investigational immunotherapy platform, NP001, which show great promise for treating ALS. Adding another year of life — to continue enjoying favorite activities and spending time with loved ones — could be tremendously impactful for people living with ALS and their families. I want to extend my deepest gratitude to the study participants and investigators. Their contributions have been invaluable in helping us understand who responds best to the compound we developed and the potential long-term benefits of NP001 to treat people with ALS.”

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

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 two to five years after symptoms appear, with death usually resulting from respiratory failure. 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 U.S. are living with ALS, and 1 in 300 people will be diagnosed in their lifetime.

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.

A New Drug Application (NDA) for the use of NP001 as a disease modifying treatment for ALS was recently filed with the FDA in October 2024. 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.

Important Supportive Information

Recent multinational ALS natural history incidence studies suggest an imbalance of inflammatory responses resulting in the destruction of motor neurons and muscle. Normally, in response to infection/activation, a subset of macrophages become inflammatory, and another subset become wound healing to maintain immune activation equilibrium. In ALS, the process is compromised, resulting in unchecked inflammation. This study used vital capacity as a quantitative test for respiratory function to highlight the relationship between a compromised innate immune system and ALS disease activity. NP001 restores the innate immune activation balance.

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. Andrews JA, et al. Association between decline in slow vital capacity and respiratory insufficiency, use of assisted ventilation, tracheostomy, or death in patients with amyotrophic lateral sclerosis. JAMA Neurol. 2018 Jan 1;75(1):58-64.

2. Cui C, et al. Creatinine and C-reactive protein in amyotrophic lateral sclerosis, multiple sclerosis and Parkinson’s disease. Brain Communications, Volume 2, Issue 2, 2020, fcaa152, https://doi.org/10.1093/braincomms/fcaa152