Neuraltus Announces Completion of Enrollment in Confirmatory Phase 2 Study of NP001 in ALS Patients with Systemic Inflammation
SAN BRUNO, Calif., July 12, 2017 -- Neuraltus Pharmaceuticals, Inc., a privately-held biopharmaceutical company dedicated to developing innovative therapeutics for neurodegenerative diseases, announced that it has completed enrollment of 138 patients with amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) in the confirmatory Phase 2 study (NCT02794857) of NP001. The last patient visit is expected to occur in early 2018, with top line results anticipated in the first quarter of 2018. The Company plans to meet with the U.S. Food and Drug Administration (FDA) after the study results are analyzed to review the complete clinical data set and discuss the regulatory path forward for NP001.
"Reaching the completion of enrollment in the confirmatory Phase 2 study of NP001 is a significant milestone for ALS patients, clinical investigators, and our team," said Rich Casey, chief executive officer, Neuraltus Pharmaceuticals. "Over the past 20 years, only two therapeutic agents have been approved to treat patients with ALS, a rare condition with a significant unmet need. We plan to request a meeting with the FDA to review all of the NP001 clinical and pharmacology data and, if the results are positive, discuss the required next steps to advance NP001 toward regulatory filing."
The confirmatory Phase 2 study is being conducted with the intent of replicating the previous Phase 2 clinical study findings in ALS patients with elevated levels of baseline inflammation. This second Phase 2 study of NP001 is a randomized, double-blind, placebo-controlled, multicenter trial which has enrolled 138 ALS patients with evidence of systemic inflammation at 22 sites across North America. Patients are receiving 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, and generate additional data about NP001’s safety and tolerability. Secondary end points include a change in pulmonary function as measured by vital capacity readings. 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.
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.