Neuraltus Pharmaceuticals announced that it will initiate a second Phase 2 clinical trial of its investigational drug candidate NP001 for the treatment of amyotrophic lateral sclerosis (ALS), with the intent of confirming positive earlier findings in patients with elevated levels of baseline inflammation and further evaluating the drug’s efficacy.
Inflammation is thought to be a contributing factor to ALS disease progression, and NP001 is believed to reduce neuroinflammation by regulating macrophage white blood cells within the central nervous system.
Results from the first Phase 2 trial, published in Neurology: Neuroimmunology & Neuroinflammation, found that treatment with NP001 produced clinically meaningful slowing of ALS progression in patients with greater baseline inflammation. Based on these findings, Neuraltus is developing the second trial protocol to confirm these benefits, with baseline inflammation measured by the levels of C-reactive protein (CRP).
Neuraltus believes this analysis, in combination with other independent studies in ALS patients, makes CRP a reliable biomarker for the selection of patients in further NP001 trials. Patient enrollment in the second trial is expected to being this summer, and the study will be subsidized by a recent equity financing from existing stakeholders, the company said in a press release.
“The recently secured equity financing allows us to advance the clinical development of NP001 in ALS,” said Rich Casey, chief executive officer of Neuraltus. “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.”
Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, is a progressive and fatal neuromuscular disease for which no cure has been identified. Although ALS has no known definitive cause, familial ALS (a hereditary form) occurs in 5 percent to 10 percent of all cases.