Neuraltus Pharmaceuticals has initiated the second Phase 2 clinical trial of NP001 for the treatment of amyotrophic lateral sclerosis (ALS), with the goal of confirming the clinical findings of the previous Phase 2 study in ALS patients with elevated levels of baseline inflammation. The study is currently recruiting patients.
Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neuromuscular disease of unknown cause for which no cure has been identified. 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.
The first Phase 2 clinical trial evaluated the preliminary efficacy, safety and tolerability of two dose levels of NP001 versus placebo in ALS patients. The ALS Functional Rating Score Revised (ALSFRS-R) was used to assess ALS patients’ daily living activities and function. Results from the first Phase 2 trial, published in Neurology: Neuroimmunology & Neuroinflammation, demonstrated that treatment with NP001 slowed the disease progression in patients with greater baseline inflammation. Based on these findings, Neuraltus conducted a secondary analysis in which the results showed that increased levels the C-reactive protein (a biomarker for systemic inflammation), may specify which patients are more likely to respond positively to the treatment.
The second Phase 2 study (NCT02794857) will further assess the clinical efficacy of NP001 and determine if ALS patients with elevated baseline inflammation might benefit from the treatment. This study is expected to generate additional data about NP001’s safety and tolerability to provide a clear clinical and regulatory path forward for this potential ALS treatment.
“NP001 is a very promising investigational therapy for ALS and other related neurodegenerative diseases,” Lawrence Steinman, MD, chairman of Neuraltus’ scientific advisory board and a professor of pediatrics, neurology and neurological sciences at Stanford University, said in a press release. “The drug’s anti-inflammatory mechanism of action is very attractive and capitalizes on numerous recent research insights about the importance of the immune system in driving the progression rates of many neurodegenerative disorders. The preliminary evidence from the Company’s first Phase 2 clinical trial of NP001 is quite compelling and should be confirmed by this next Phase 2 trial in patients with elevated levels of CRP,” Steinman said.
“The initiation of the second Phase 2 study of NP001 marks an important step in ALS research,” said Rich Casey, chief executive officer of Neuraltus Pharmaceuticals. “We are encouraged by the results of the first Phase 2 study of NP001 and pleased to continue our evaluation of this innovative, targeted investigational therapy.”