Neurocrine’s $50M bet on Idorsia drug flames out in second PhII

The drug that Neurocrine Biosciences licensed from Idorsia for $50 million has flunked a second Phase II study, failing to help pediatric patients with a rare type of epilepsy.

Just a few months ago, Neurocrine revealed that NBI-827104, a calcium channel blocker, did not meet the endpoint in a separate mid-stage study for essential tremor.

“We think expectations for the trial were low,” wrote Cowen analyst Phil Nadeau. And he added that the negative readout may put the program to bed: “After two failed studies, we suspect there is no path forward for ‘104.”

The study, dubbed STEAMBOAT, enrolled children with epileptic encephalopathy with continuous spike-and-wave during sleep — a condition tied to seizures and neurocognitive decline. The 24 patients in the trial were given daily oral doses of either NBI-827104 or placebo.

For the primary endpoint, investigators tracked the ratio of spike-wave index, which measures the percentage of sleep affected by epileptic activity using video-electroencephalograms, after six weeks of treatment. The drug didn’t beat placebo. The company added it’s well-tolerated.

Eiry Roberts

NBI-827104 is designed to be a brain-penetrating T-type calcium channel blocker, hitting Cav 3.1, Cav 3.2 and Cav 3.3. Neurocrine secured an option to the drug for $5 million in 2019 and paid Idorsia $45 million the year after to license it.

While Neurocrine announced almost immediately in the wake of the essential tremor data that it will drop that indication, this time it is holding back a decision.

“We will continue to analyze the rich data set generated from this study to determine next steps,” said Eiry Roberts, Neurocrine’s chief medical officer.

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