Investigator to Receive $1.5 Million Grant from the ALS Association to Help Fund Confirmatory Phase 2 Study of Neuraltus Pharmaceuticals’ NP001

Neuraltus Pharmaceuticals, Inc., a privately-held biopharmaceutical company focused on the development of groundbreaking drugs to treat neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), announced today that Robert G. Miller, M.D., Director of the Forbes Norris MDA/ALS Research Center at Sutter Health’s California Pacific Medical Center in San Francisco, has been awarded a grant from The ALS Association for $1.5 million to help fund a Phase 2 clinical study of the company’s investigational therapy for ALS, NP001. Inflammation is thought to be a contributing factor to ALS disease progression, and NP001 is believed to reduce neuroinflammation through the regulation of macrophage white blood cells within the central nervous system. The Phase 2 study is intended to help confirm the potential clinical benefits seen in the previous study of NP001, the results from which were published in the April 2015 issue of Neurology: Neuroimmunology & Neuroinflammation, an official journal of the American Academy of Neurology. Approximately 400,000 people worldwide are living with ALS, also known as Lou Gehrig’s disease, a progressive and fatal neurodegenerative disease that affects motor neurons in the brain and the spinal cord.[i] 

“ALS is a fatal condition with limited treatment options,” said Dr. Miller. “Clinical research of promising investigational drugs, like NP001, is critically important as we continue to learn more about this devastating disease. This Phase 2 study is expected to provide valuable insights, not only about NP001, but also on the role of inflammation in disease progression and the biomarkers identified by Neuraltus in a previous study.” 

The Phase 2 study, which is expected to begin before the end of the year, will be designed to confirm the potential impact of NP001 on two inflammation biomarkers: interleukin-18 (IL-18) and the presence of lipopolysaccharide (LPS) in the blood. The study will focus on patients who have had ALS symptoms for less than 3 years with a vital capacity (breathing test) greater than 65 percent. More specific details regarding the Phase 2 study of NP001 will be disclosed at the time of trial initiation. The $1.5 million grant, awarded by The ALS Association, will be combined with approximately $1.2 million in funding and support contributed by Neuraltus.

“The more we can learn from promising research, like that being conducted by Dr. Miller and the Neuraltus team, the more insight we have into the physiological profile of ALS,” said Lucie Bruijn, Ph.D. M.B.A, Chief Scientist, The ALS Association. “Awarding grants to fund research like the Phase 2 study of NP001 is vitally important in our pursuit of possible treatment options for ALS patients.”

“We appreciate the grant support from The ALS Association to help us advance the clinical development of NP001 as a potential treatment for patients with ALS,” said Rich Casey, Neuraltus Pharmaceuticals’ Chief Executive Officer. “We look forward to working with Dr. Miller, the ALS community and The ALS Association on this Phase 2 study as we work to advance NP001 as quickly as possible toward a pivotal clinical trial.”

About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) is a rare and fatal neurodegenerative disease characterized by the degeneration of motor neurons in the spinal cord and brain.

There are approximately 400,000 ALS patients worldwide, including approximately 30,000 patients in the United States and 50,000 patients in Europe. Most patients and physicians report only modest slowing of ALS progression from existing treatment for ALS, underscoring the need for new and effective drug therapy.

About NP001
The cause of ALS 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) macrophage white blood cells resulting in the release of factors in the central nervous system that lead to damage of motor neurons. NP001, a regulator of macrophage activation, exerts its effect by converting these activated inflammatory macrophages back to their normal state.

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at

About Neuraltus Pharmaceuticals, Inc.
Neuraltus Pharmaceuticals, Inc. is a privately-held biopharmaceutical company dedicated to developing and commercializing high-impact therapeutics that address critical unmet needs for patients and physicians, primarily in the treatment of neurodegenerative diseases. Neuraltus recognizes that ALS is a devastating condition, with only one approved therapy, and is focused on developing and bringing its investigational treatments to market as quickly as possible. 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. In addition to NP001, Neuraltus also has a pre-clinical program, NP003, for the treatment of lysosomal storage disorders such as Fabry’s disease and Gaucher’s disease.

For more information on Neuraltus, please visit

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