- Subset analysis of patients whose ALS did not progress from a long-term survival study showed NP001 saved lung function and extended life by 22 months vs. a control group (~70% on riluzole)
- NP001 significantly decreased inflammatory biomarkers traditionally associated with ALS progression (including Neurofilament light chain levels or NfL)
- Data consistently demonstrates that NP001 slows or halts ALS progression in a subset of patients with an inflammatory form of the disease after only six months of treatment
PALO ALTO, Calif.--(BUSINESS WIRE)--Neuvivo, a late-clinical stage biopharmaceutical company, today announced promising new results from a fresh analysis of a previous study of its investigational immunotherapy platform NP001 to treat Amyotrophic Lateral Sclerosis (ALS), which builds on evidence that NP001 spares lung function and substantially extends survival in ALS patients.
The new analysis was based on Neuvivo’s long-term survival study of NP001 to discern drug-related clinical benefits over the potential of an acute ALS reversal period. While there is no known cure or treatment that stops or reverses ALS, small reversals and plateaus may occur, especially over short periods of time. However, large, sustained reversals that last a long time are extremely rare.
Dr. Michael McGrath, MD, PhD, founder and Chief Scientific Officer at Neuvivo and Emeritus Professor of Medicine at University of California San Francisco, presented the results of this new analysis during the ALS Network Research Summit on January 16, 2025.
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
“One of the most confounding aspects of ALS research is that this disease does not uniformly progress,” Dr. McGrath said. “My analysis looked at patients whose progression slowed or halted using a standard test for functional change in ALS patients, the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R). When I compared patients treated with NP001 vs. a control group of patients whose disease was not progressing over six months, the results on respiratory function and on overall survival were striking.”
Results from this analysis targeting “non-progressors” showed that the NP001 treated group significantly (p=0.02) slowed or halted the loss of vital lung capacity compared with the control group (~70% on riluzole). Vital capacity is a quantitative but separate measure from the ALSFRS-R test that more accurately assesses a patient’s lung function. Changes in vital capacity have been clearly associated with survival in ALS.
A combined dataset from the phase 2A and 2B studies found that 31% of patients treated with NP001 (dosed at 2 mg/kg) vs. 14% of placebos who had risk characteristics defined in the natural history studies showed no disease progression over the six-month studies.
The median overall survival in the NP001 treated group was 58 months, as compared to 36 months in the control group (~70% on riluzole), meaning their life was extended by 22 months after a six-month clinical trial (p = 0.02), thereby supporting both the relationship between lung function and survival and the more promising drug-related benefit that NP001 offers in treating this complex disease.
Plasma biomarkers at baseline that corrected with NP001 but not with the placebo also showed a decrease in neurofilament light chain (NfL) levels in concordance with the correction of the inflammatory biomarkers. In this non progressor group, slowing of vital capacity loss and extension of survival was accompanied by the correction of inflammatory abnormalities and associated neuronal damage marker NfL.
Ari Azhir, PhD, founder and CEO of Neuvivo, said: “Dr. McGrath’s findings add to the body of evidence supporting the clinical benefits of NP001 to treat ALS. These results align well with the recent NP001 overall survival study and biomarker identification study, further supporting the consistency that this drug may offer substantial benefit to people living with ALS.”
Survival Study Results
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) when treated with NP001. 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 an 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). In this publication, patients treated with NP001 experienced a 50% slowing of vital capacity loss suggesting that sparing respiratory function contributed to this survival advantage.
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 drug 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 was previously granted Orphan Drug and Fast Track Designations by the FDA. 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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