Aquinox's bladder pain drug misses the mark in Phase II

Aquinox's ($AQXP) lead drug failed its main goal in a Phase II trial on bladder pain syndrome, but the company believes its oral treatment showed enough to promise to merit further study.

The drug, AQX-1125, didn't significantly beat placebo in reducing patient-reported pain in a 6-week study on 69 women with chronic bladder pain. Patients taking 200 mg of AQX-1125 per day reported an average 2.4-point reduction in pain on an 11-point scale, the company said, while placebo led to a mean 1.3-point reduction, a difference that fell short of statistical significance.

The miss sent Aquinox's shares down as much as 24% on Thursday, but the company recovered by late afternoon, closing 7.8% off of its Wednesday value.

And despite the Phase II failure, Aquinox says it sees enough of an efficacy signal to press forward. About 49% of AQX-1125 patients reported a pain reduction of at least 2 points, which CEO David Main said is affirmation of the drug's potential in relieving bladder pain.

"While we did not meet our statistical endpoint, we are encouraged to see in our first BPS/IC trial a positive trend in reduction of pain with AQX-1125 compared to placebo," Main said in a statement. "... We believe today's top line data support further development of AQX-1125 for BPS/IC patients."

The company is releasing only top-line data for now, still analyzing its secondary endpoints with plans to disclose full results at a later medical meeting.

AQX-1125 works by boosting SHIP1, a component of the PI3K cellular signaling pathway that modulates inflammation. Beyond bladder pain syndrome, Aquinox is developing the drug for COPD flare-ups and eczema, working through concurrent Phase II trials.

- read the statement (PDF)