Chelsea Therapeutics reports positive efficacy results in PhII fibromyalgia study

Chelsea Therapeutics ($CHTP) has racked up some more positive efficacy data for its lead drug droxidopa, which is now up for review at the FDA. Investigators reported the drug spurred a statistically significant response on some key measures of fibromyalgia in the Phase II study, which was designed to test a range of doses as well as a combo approach with carbidopa. The biotech recruited 120 patients for the study, ultimately zeroing in on 7 separate arms.

Charlotte, NC-based Chelsea designed the trial with the understanding that droxidopa, a synthetic amino acid, is converted by the body into norepinephrine. By boosting levels of norepinephrine, Chelsea believes it can directly address some of the disease's severe side effects. Shares of Chelsea were up slightly this morning.

"Since norepinephrine is a key neurotransmitter involved with the modulation of chronic pain, we were not surprised to see evidence of droxidopa's therapeutic benefit in treating patients with fibromyalgia," stated Dr. Art Hewitt, Chelsea's chief scientific officer. "As we continue to evaluate potential indications for droxidopa such as fibromyalgia and adult attention deficit disorder, trials like this provide insight into how to optimize dosing for more robust future clinical evaluations. Given the broad biologic activity of norepinephrine, we continue to believe there are wide-ranging therapeutic applications for a first-in-class oral prodrug of norepinephrine. We also continue to be encouraged by the remarkable safety profile of droxidopa which, even at its highest dose, again proved not to be associated with any serious or significant adverse events."

A few weeks ago the FDA accepted Chelsea's application for droxidopa as a new treatment for hypotension. The drug, in-licensed from Japan's Dainippon Sumitomo 5 years ago, flunked its first Phase III, but Chelsea regrouped and announced positive data in 2010. Regulators are expected to make its final decision on the program by March 28 under a priority review.

- here's the press release
- read the Reuters story