Neuraltus Pharmaceuticals Announces Completion of Last Patient Visit in Confirmatory Phase 2 Study of NP001 for the Treatment of Amyotrophic Lateral Sclerosis
Top-line data to be reported in late Q1 2018
SAN BRUNO, Calif. – Jan. 4, 2018 - Neuraltus Pharmaceuticals, Inc., a privately held biopharmaceutical company dedicated to developing innovative therapeutics for neurodegenerative diseases announced today the completion of the last patient visit in the confirmatory Phase 2 study of its investigational treatment, NP001, in patients with amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) in North America. The study (NCT02794857) was conducted to confirm previous Phase 2 findings in ALS patients with elevated levels of systemic inflammation. Neuraltus expects to share top-line results from the confirmatory study at the end of first quarter 2018.
“The outcome of the confirmatory Phase 2 study of NP001 for the treatment of ALS is highly anticipated by researchers, patients and caregivers, as it has the potential to reaffirm the preliminary evidence from the Company’s first Phase 2 clinical trial of this investigational compound,” said Robert Miller, M.D., principal investigator, and Clinical Professor of Neurosciences, Stanford University, and Director of the Forbes Norris ALS Research Center at California Pacific Medical Center in San Francisco. “Progression of ALS is believed to be driven by systemic inflammation and NP001 regulates cells associated with the body’s response to inflammation.”
ALS is a rare and fatal neurodegenerative disease affecting approximately 400,000 individuals worldwide. In the first Phase 2 study of NP001, Neuraltus assessed the safety, tolerability, and preliminary efficacy of two dose levels of NP001 versus placebo using the ALS Functional Rating Score Revised (ALSFRS-R), which measures key activities of daily living and function for patients with ALS. A secondary analysis of the study results suggested that increased levels of a biomarker for systemic inflammation, C-reactive protein (CRP), may indicate which patients are more likely to respond to NP001.
“If the study results confirm the previous study’s findings, NP001 could be one step closer to regulatory review,” said Rich Casey, chief executive officer, Neuraltus Pharmaceuticals. “We look forward to sharing the top-line results and continuing to explore this innovative, investigational therapy for people living with ALS, a progressive and life-threatening condition.”
About the Confirmatory Phase 2 Study of NP001
The second Phase 2 study is a randomized, double-blind, placebo-controlled, multicenter study that enrolled 138 subjects in North America with ALS and evidence of systemic inflammation. Patients received either NP001 2 mg/kg or placebo over a period of 6 months. The study was designed to evaluate the change from baseline in ALSFRS-R during the study period. Secondary objectives include a change in pulmonary function as measured by vital capacity readings. Top-line results are planned for the end of Q1 2018. Further information about the study 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 two approved drugs that slow the progression of the ALS, underscoring the need for a 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. NP001, the Company’s lead product candidate, is also being studied in patients with amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease).
For more information, please visit www.neuraltus.com.