Neurocrine shares ($NBIX) took a nasty drubbing after the biotech announced that its low-dose version of an experimental therapy for a rare movement disorder failed a Phase IIb study. The San Diego-based developer was upbeat, though, about the high-dose data, though investors nevertheless responded by carving out more than 30% of its share value.
Investigators said that the 50 mg dose of NBI-98854, a VMAT2 inhibitor in development for tardive dyskinesia, did not meet the primary endpoint. But the 100 mg dose, which had been expected to be the maximum amount tolerated by patients, was both well tolerated and effective in addressing the ailment, which is characterized by sudden and involuntary body movements. There are no therapies for this disease, which had helped spur some enthusiasm for the drug among some analysts, according to a report from Reuters.
"Although this was not the result we anticipated in the 50mg dose based on the data set from the earlier clinical studies, we were pleased to see a clear dose related response at the end of Week 2, with the 100 mg dose," said Neurocrine CEO Kevin Gorman. "The 100 mg dose was statistically superior to placebo as well as providing a clinically significant improvement in tardive dyskinesia symptoms and importantly a very good safety profile. We will now perform an additional Phase II study utilizing 100mg and higher doses."
Neurocrine's lead program is for elagolix, an endometriosis therapy that won a $575 million deal back in 2010 with Abbott, which later spun off its drug business as AbbVie ($ABBV). That's still the primary focus of the company and analysts. A Phase III study for that drug was launched in 2012 and the partners say they can file for an approval in 2016, provided the data are right.
In the meantime Neurocrine will regroup on NBI-98854, pursuing new mid-stage trials of the higher dose and other doses.
- here's the press release
- read the Reuters story