Completion of enrollment is expected in mid-2017, with Neuraltus shooting for results in early 2018. Eighteen clinical sites across North America are recruiting patients.
“The involvement of the patient and physician community is critically important to developing potential therapies, especially for conditions with limited treatment options,” Rich Casey, Neuraltus Pharmaceuticals’ chief executive officer, said in a press release.
“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,” Casey said. “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.”
The company started a second Phase 2 clinical trial of NP001 for ALS in September 2016. Its goal is to confirm the findings of the previous Phase 2 study in ALS patients with elevated levels of inflammation.
Patients in this randomized, double-blind, placebo-controlled, multicenter clinical trial (NCT02794857) will be treated with 2 mg/kg of NP001 or a placebo for six months. The primary objective is to evaluate whether the therapy improves patients’ ability to function. The measuring stick will be ALS Functional Rating Score Revised (ALSFRS-R), which assesses patients’ daily living activities and function.
A secondary endpoint, or goal, is whether NP001 improves pulmonary function as measured by vital capacity readings. The trial is also expected to generate additional data about NP001’s safety and tolerability. The hope is that, together, the results can provide a clear clinical and regulatory path forward for the treatment, Neuraltus said.
The company published the results of the first Phase 2 clinical trial of NP001 in Neurology: Neuroimmunology & Neuroinflammation. The trial (NCT01281631) evaluated the preliminary effectiveness, safety and tolerability of two dose levels of NP001 compared to placebo.
Results showed that NP001 slowed ALS progression in patients with high levels of inflammation when the treatment started.
The results prompted Neuraltus to perform a secondary analysis. It showed that increased levels of the C-reactive protein, a biomarker for systemic inflammation, may determine which patients are more likely to respond to NP001.
Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neuromuscular disease of unknown cause that has no cure. Inflammation is thought to be a contributing factor to ALS progression, and NP001 is believed to reduce neuro-inflammation by regulating macrophage white blood cells in the central nervous system.