Positive PhIII leaves Neurocrine lining up for an FDA filing for tardive dyskinesia

Neurocrine CEO Kevin Gorman

Neurocrine Biosciences says its experimental drug for tardive dyskinesia hit the primary endpoint in a Phase III study, setting up a near-term application with the FDA and spurring a 28% spike in the biotech's share price in premarket trading.

NBI-98854 (valbenazine) is Neurocrine's ($NBIX) lead in-house program, listed one step down from the AbbVie ($ABBV)-partnered program for elagolix. The drug is a VMAT2 inhibitor and registered a significant improvement over placebo in reducing the involuntary muscle movements characteristic of tardive dyskinesia, an ailment that frequently afflicts patients taking older antipsychotic drugs.

Investigators reported that in the Phase III study, NBI-98854 reduced patients' Abnormal Involuntary Movement Scale (AIMS) score at week 6 in the 80-mg once-daily dosing group by 3.1 points more than the placebo arm.

The drug missed a secondary endpoint for the intent-to-treat crowd on a separate readout for the Clinical Global Impression of Change (CGI-TD) score, but hit among a per-protocol group that excluded patients who had no detectable levels of the drug in their blood.

San Diego-based Neurocrine has been on something of a roller coaster ride with this drug. A 50-mg dose disappointed the company in late 2013, but by titrating up to 75 mg they were able to get the response they were looking for in 2014. Elagolix, meanwhile, has been in a late-stage program for endometriosis and recently performed well in a Phase IIb trial for women with uterine fibroids who suffered from heavy menstrual bleeding.

"We will now turn our focus to completing the open-label safety portion of the studies in tardive dyskinesia patients and compiling the data for both doses of NBI-98854 to be included in the New Drug Application we intend to file with the FDA in 2016," noted CEO Kevin Gorman in a statement.

Neurocrine isn't working alone in this field. Back in the spring, Teva ($TEVA) agreed to pay $3.2 billion for Auspex Pharmaceuticals, another biotech that was working on a lead drug that was hunting pivotal data for tardive dyskinesia and related muscle-movement disorders.

"Generally speaking, I expect both products to compete in the market," noted Evercore/ISI analyst Umer Raffat. "Teva offers dose titration and good efficacy at lower doses (which means better tolerability on depression?). Neurocrine offers once daily dosing and with KINECT-3 in hand, can claim better pbo-adj efficacy on primary endpoint. I think both Teva and Neurocrine look good."

- here's the release

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