Phase 2 in ALS-Associated Inflammation Fails to Meet Endpoints

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This morning, top-line data from a confirmatory Phase 2 study of NP001 in ALS were reported, and showed that the trial did not meet its primary or secondary endpoints.

This morning at the American Academy of Neurology's (AAN) 70th Annual Meeting in Los Angeles, top-line data from a confirmatory Phase 2 study of NP001 in amyotrophic lateral sclerosis (ALS) were reported, and showed that the trial did not meet its primary or secondary endpoints.

The study completed enrollment in July 2017.

The drug, an investigational therapy in development from Neuraltis Pharmaceuticals, is designed specifically for individuals with ALS who suffer from elevated levels of systemic inflammation. The study enrolled 138 patients and did not meet its primary endpoint, a change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) score, or its secondary endpoint, a change from baseline in pulmonary function as measured by vital capacity readings.

"We recognize the desperate need for advances in treating ALS and are very disappointed with the findings in our confirmatory Phase 2 study of NP001," said Rich Casey, chief executive officer, Neuraltus Pharmaceuticals in a press release. "We are conducting additional analyses of the trial results to determine if and how we will proceed in developing the compound."

The Phase 2 clinical trial (NCT02794857) was a randomized, double-blind, placebo-controlled, multicenter study that enrolled subjects with ALS and evidence of systemic inflammation in North America. Patients were administered either NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6, or placebo at the same dosing frequency over a period of 6 months, with the hopes that a significant change from baseline in ALSFRS-R would be exhibited during the study period.

Additional secondary endpoints included time to tracheotomy and a change in levels of blood inflammatory biomarkers.

In a previous Phase 2 study of NP001 (NCT01281631), Neuraltus evaluated the safety, tolerability and preliminary efficacy of 2 different dose levels of NP001 compared to placebo using the same rating scale, and a secondary analysis of the study results implied that heightened levels of C-reactive protein (CRP), a biomarker for systemic inflammation, could potentially indicate which patients are more likely to respond to the therapy.

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.

"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,” Casey said in July.

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