Neurocrine’s Experimental Therapy for EE Fails to Meet Endpoint in Phase 2 Study

Neurocrine Biosciences said its experimental therapy NBI-827104 failed to meet its primary endpoint in the phase 2 STEAMBOAT study evaluating it compared to placebo in pediatric patients with epileptic encephalopathy with continuous spike-and-wave during sleep.

Photo: Eiry Roberts, chief medical officer

Neurocrine paid Idorsia $5 million for an option on the drug in 2019 and in May 2020 acquired the global rights to NBI-827104 from Idorsia for an additional $45 million.

Epileptic encephalopathy with continuous spike-and-wave during sleep (EE-CSWS) is a rare pediatric developmental and/or epileptic encephalopathy that is characterized by a continuous or nearly continuous spike and wave electroencephalogram pattern during the non-rapid eye movement phase of sleep, and regression or stagnation in cognitive, language, behavioral, or motor functions.

The company said NBI-827104 was generally well tolerated.

The STEAMBOAT phase 2 trial is a randomized, double-blind, placebo-controlled study that evaluated the efficacy, safety, tolerability, and pharmacokinetics of NBI-827104 when administered once daily up to 13 weeks in pediatric patients with EE-CSWS.

The primary endpoint was a reduction from baseline as compared to placebo in the ratio of spike-wave index when measured after six weeks of study treatment. The SWI, a measure of the percentage of sleep affected by epileptic activity, was measured during the first hour of non-rapid eye movement sleep by independent and centralized readings of overnight video-electroencephalograms.

“While we did not meet the primary endpoint for this phase 2 study, we remain committed to advancing care for patients living with epilepsy, including rare pediatric forms,” said Eiry Roberts, chief medical officer of Neurocine. “We will continue to analyze the rich data set generated from this study to determine next steps.”

Author: Rare Daily Staff