Retrospective Analysis Finds Amyotrophic Lateral Sclerosis Patients with High Serum C-Reactive Protein (CRP) Levels May Respond to Immune Regulators Like NP001
-JAMA Neurology manuscript examines potential effect of immune regulators in ALS patients with evidence of systemic inflammation-
SAN BRUNO, Calif., April 3, 2017 -- Neuraltus Pharmaceuticals, Inc., a privately-held biopharmaceutical company dedicated to developing innovative therapeutics for neurodegenerative diseases, announced that results from an independent retrospective analysis of three clinical data sets in amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease), including NP001 Phase 2 data, have been published in the April online edition of JAMA Neurology. The analysis found a correlation between CRP serum levels and the rate of disease progression in ALS patients. CRP is a biomarker for systemic inflammation and a higher inflammatory state is believed to contribute to the progression of ALS. These findings, which were replicated in an independent cohort of ALS patients identified through a regional population registry, suggest that increased levels of CRP may indicate which patients are more likely to respond to drugs that focus on inflammation, like NP001.
"Our findings mark an important next step in ALS research, as they suggest that ALS patients with elevated systemic inflammation as identified through CRP levels, may progress more rapidly," said Christian Lunetta, MD, lead author, Centro Clinico NeMO (NeuroMuscular Omnicenter), Fondazione Serena Onlu. "Understanding the role of inflammation in disease progression is critical as researchers continue to work on potential treatments for ALS. While additional research is necessary, our retrospective analysis of three separate data sets will help guide the development of investigational therapies, especially those, like NP001, focused on systemic inflammation."
The findings from the retrospective data analysis reinforce the use of CRP as a biomarker and potential indicator of disease progression in individuals with ALS. NP001, an investigative therapy being developed by Neuraltus, is currently enrolling a confirmatory Phase 2 study of ALS patients with elevated levels of inflammation as measured by CRP serum levels.
"NP001 is a very promising investigational therapy for ALS and other related neurodegenerative diseases," said Rich Casey, chief executive officer, Neuraltus Pharmaceuticals. "We are assessing CRP levels in ALS patients as an indicator of increased inflammation in our ongoing Phase 2 study, and will identify any change in baseline of an individual's function and daily living as measured by the ALSFRS-R. This approach appears to be supported by the results of the retrospective analysis published in JAMA Neurology, especially the correlation between CRP levels and a patient's ALSFRS-R in the two independent cohorts of ALS patients. We anticipate completing enrollment of our confirmatory Phase 2 study this year with the expectation that the collective data from both Phase 2 studies will inform the regulatory path forward for NP001."
NP001's Ongoing Phase 2 Study Design
Neuraltus is currently enrolling patients in the confirmatory second Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in ALS patients who have evidence of systemic inflammation. Patients will receive either NP001 2mg/kg or placebo over a period of 6 months. The study is designed to evaluate the change from baseline of an individual's ALS Functional Rating Score Revised (ALSFRS-R) during the study. Secondary objectives include a change in pulmonary function as measured by vital capacity readings.
The confirmatory Phase 2 study is being conducted at 20 sites in North America and has enrolled 87 of the study's 120 participants. The Company anticipates completing enrollment of the study during the second quarter of 2017, with data analysis expected in the first quarter of 2018. Further information about the study and clinical centers is available at https://clinicaltrials.gov.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) is a rare and fatal neurodegenerative disease characterized by degeneration of motor neurons in the spinal cord and brain. The cause of the disease is currently unknown, but there is increasing evidence that implicates neuroinflammation with the progression of the disease. It is believed that in people with ALS, there are increased levels of inflammatory (activated) macrophages, a type of white blood cell, resulting in the release of factors in the central nervous system that damage motor neurons. NP001, a regulator of macrophage activation, exerts its effect by converting these activated inflammatory macrophages back to their normal state.
There are approximately 400,000 ALS patients worldwide. There are no existing treatments that meaningfully slow the progression of or stabilize the disease, underscoring the need for new and effective drug therapy.
About Neuraltus Pharmaceuticals, Inc.
Neuraltus Pharmaceuticals, Inc. is a privately-held biopharmaceutical company dedicated to developing and commercializing innovative therapeutics that address critical unmet needs for patients and physicians in the treatment of neurodegenerative diseases. The Company is collaborating with global ALS specialists and seeking input from patient advocacy organizations to help inform the clinical development path and better understand the needs of people affected by the disease.
For more information, please visit www.neuraltus.com.
About Centro Clinico NeMO
NeMO (NEuroMuscular Omnicentre) Clinical Center is a highly specialized Center devoted to take care of people affected by neuromuscular diseases, including Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA) and Muscular Dystrophies. These diseases cause severe disability with a high social impact. They need long and complex therapeutic and clinical care paths and there are not cures for them at the moment. Nowadays in Italy neuromuscular diseases affect 40.000 patients. NeMO Clinical Center is sited in four Italian cities: Milan, Rome, Messina and Arenzano (Genoa).
For more information, please visit www.centrocliniconemo.it