Lexicon rockets up after a positive PhIII study sets up an NDA

Lexicon CEO Lonnel Coats

Shares of Lexicon Pharmaceuticals ($LXRX) soared 35% this morning after The Woodlands, TX-based biotech claimed a win in Phase III for one of its lead drugs. The biotech said that telotristat etiprate proved effective against rare cases of carcinoid syndrome, a condition triggered by neuroendocrine tumor cells that causes severe diarrhea.

Lexicon's executive crew stuck to a top-line review of Phase III data for the oral drug, but specified positive data on secondary endpoints.

The primary endpoint was a reduction in bowel movements after 12 weeks of treatment. The secondary endpoints included a statistically significant rate of durability for the 250-mg and 500-mg doses; more than double the rate seen in the placebo arm,

Patients who received 250 mg of telotristat etiprate "experienced a 29% reduction in the average number of daily bowel movements during the final week (week 12) of the study period compared to baseline, and those in the 500-mg arm experienced a 35% reduction, while the placebo group showed a 17% reduction," the company reported. And the rates were consistent with what the company saw in Phase II.

"This morning marks a tremendous milestone for Lexicon," CEO Lonnel Coats told analysts in a call this morning, adding later that the company plans to file both doses for an approval at the FDA in Q1 2016. "We are well on our way toward commercialization."

A 36-week open-label study will track longer term responses to the 500-mg dose, which the company says should help demonstrate a significant improvement in reducing diarrhea compared to the current standard of care. More data from the Phase III study will be released at a later scientific conference.

Lexicon has had its ups and downs over the years. The biotech was forced to reorganize back in early 2014 as it circled its wagons around its other lead program, the SGLT1/2 inhibitor sotagliflozin (LX4211) for diabetes--a rare instance of a small biotech developing a new therapy for such a big and demanding field.

The drug telotristat, which Ipsen partnered on for the European rights, targets tryptophan hydroxylase, an enzyme that triggers the excess serotonin production within mNET cells that leads to carcinoid syndrome.

- here's the release