Neuraltus Pharmaceuticals Announces Plans for Second Phase 2 Study of NP001 in ALS Using Biomarker for Enriched Patient Selection

Phase 2 study, funded by newly secured capital, expected to begin enrolling during the summer of 2016

PALO ALTO, Calif. (April 4, 2016) – Neuraltus Pharmaceuticals, Inc., a privately-held biopharmaceutical company dedicated to developing innovative therapeutics for neurodegenerative diseases, announced plans to initiate a second Phase 2 study of its investigational treatment, NP001, in patients with amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease). This second Phase 2 study will be funded by a recent equity financing from existing investors. 

Neuraltus is developing this second Phase 2 study protocol to confirm the previous Phase 2 clinical study findings in patients with elevated levels of baseline inflammation. Neuraltus prospectively measured levels of C-reactive protein (CRP), a marker of systemic inflammation, from participating patients in the first Phase 2 study. Further analyses of these data, also announced today, suggest that increased levels of CRP, at baseline, may indicate which patients are more likely to have a positive response to NP001.  ALS progression is believed to be driven, in part, by systemic inflammation and NP001 is designed to regulate inflammatory cell activity, as measured by CRP. The Company believes this analysis, in conjunction with other independent research in ALS patients, makes CRP a credible biomarker to enrich patient selection in future studies of NP001. The Company expects to begin enrolling the second Phase 2 study in North America during the summer of 2016. Further information about the biomarker analysis can be found here.

“The recently secured equity financing allows us to advance the clinical development of NP001 in ALS,” said Rich Casey, chief executive officer, Neuraltus Pharmaceuticals. “Progressive neurological diseases and disorders of the brain are often complex, life-threatening conditions, many of which have few if any treatment options. This second Phase 2 study is expected to generate additional data about NP001’s safety and efficacy to provide a clear clinical and regulatory path forward for this potential ALS treatment.”

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 leads 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.

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