Earlier today, Neuraltus Pharmaceuticals, Inc. announced that its Phase 2 study of NP001 in amyotrophic lateral sclerosis (ALS) patients has completed enrollment.
The study (NCT02794857
) has enlisted 138 ALS patients, and the final patient visit is anticipated for early 2018, with top-line results of the study to be released in the first quarter of the new year.
ALS, or “Lou Gehrig’s Disease,” is a classic motor neuron disease. Its etiology is not currently known, but there is increasing evidence that implicates neuroinflammation in its progression. Early symptoms include muscle weakness or stiffness, however, as the disease progresses, a patient’s abilities to move, speak, swallow, and eventually breath are rapidly lost. Life expectancy of an ALS patient after diagnosis is 2-5 years.
"Reaching the completion of enrollment in the confirmatory Phase 2 study of NP001 is a significant milestone for ALS patients, clinical investigators, and our team," said Rich Casey, chief executive officer, Neuraltus Pharmaceuticals in a press release
"Over the past 20 years, only two therapeutic agents have been approved to treat patients with ALS, a rare condition with a significant unmet need. We plan to request a meeting with the FDA to review all of the NP001 clinical and pharmacology data and, if the results are positive, discuss the required next steps to advance NP001 toward regulatory filing.”
With the intent of replicating the previous Phase 2 clinical study findings in ALS patients with elevated levels of baseline inflammation, the confirmatory Phase 2 study is a randomized, double-blind, placebo-controlled, multicenter trial spanning 22 different sites across North America.
In 2012, the company completed its first Phase 2 study assessing NP001 in ALS patients and the study showed a ‘positive trend’ in slowing disease progression. The second Phase 2 study that has completed its enrolment is is a randomized, double-blind, placebo-controlled, multicenter trial spanning 22 different sites across North America, Data from the study is expected to provide additional data about NP001’s safety and efficacy to give a better understanding of the clinical and regulatory path forward.
Patients in the study are either receiving NP001 2mg/kg or placebo over a 6 month period with the intention of evaluating the change from baseline of an individual's ALS Functional Rating Score Revised (ALSFRS-R). Additionally, the study will generate data as it pertains to the safety and tolerability of the drug.
Secondary end points include a change in pulmonary function as measured by vital capacity readings.
Neuraltus intends to meet with the U.S. Food and Drug Administration (FDA) after the results of the study are analyzed and the complete clinical data set can be reviewed, and the regulatory path forward for NP001 can be discussed.