Neuraltus Pharmaceuticals has announced the confirmatory phase 2 study of the company’s lead investigational treatment NP001 for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) has enrolled 50 of the study’s 120 participants. The study is enrolling patients at 18 sites in North America. The company anticipates completing enrollment of the study during the second quarter of 2017, with data analysis expected in the first quarter of 2018.
“The involvement of the patient and physician community is critically important to developing potential therapies, especially for conditions with limited treatment options,” said Rich Casey, Neuraltus Pharmaceuticals’ chief executive officer. “Our team is working closely with study investigators to quickly identify and screen potential patients for enrollment in the confirmatory phase 2 study of NP001 in ALS. The results of this study will help determine the regulatory path forward for NP001, and we are thankful to all of our previous, current, and future study participants. We could not advance the clinical development of NP001 without their support.”
In September 2016, Neuraltus announced a 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 during the study of an individual’s ALS Functional Rating Score Revised (ALSFRS-R), a measurement of key activities of daily living and function for patients with ALS. Secondary objectives include a change in pulmonary function as measured by vital capacity readings.
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.
Neuraltus Pharmaceuticals, 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.
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