Chelsea Therapeutics' Phase III NORTHERA Study 301 meets primary endpoint

Sep 20 2010

• Northera Provided Statistically Significant Improvement Over Placebo on Composite Orthostatic Questionnaire Score.
• Study Showed Statistically Significant Benefits in all 3 Composite Scores and 8 out of 10 Individual Items of the Orthostatic Hypotension Questionnaire
• Northera Treatment Resulted in Significant Improvement in Standing BP (P<0.001)
• Management to Host Conference Call to Discuss Results at 8:30 AM ET

Chelsea Therapeutics International, Ltd. (Nasdaq:CHTP) announced that preliminary analyses of its Phase III NORTHERA™ Study 301 met its primary endpoint. Treatment with Northera provided clinically meaningful and statistically significant improvement.

"We are extremely excited by these top-line results which provide validation of the safety and efficacy of Northera as a novel treatment for symptomatic neurogenic orthostatic hypotension, a serious condition for which there is an urgent need for improved treatments," said Dr. Simon Pedder, president and CEO of Chelsea Therapeutics. "Symptoms of chronic neurogenic orthostatic hypotension are severe, not only putting patients at high risk for falls and associated injuries but also severely impacting their quality of life and generating significant added health care costs. Northera is the first and only drug to repeatedly demonstrate clinical improvement in these patients by both alleviating symptoms of neurogenic orthostatic hypotension and improving their ability to perform daily activities."

Northera Trial Meets Primary and Multiple Secondary Endpoints

Patients randomized into this double-blind, placebo controlled study were evaluated for symptomatic and functional improvement using the orthostatic hypotension questionnaire (OHQ), which is specifically designed to rate the severity of symptoms resulting from low blood pressure and the degree those symptoms interfere with a patient's ability to perform activities of daily living.

The OHQ is a two-part questionnaire that uses an 11-point scale (zero to 10) to assess the severity of six symptoms on the orthostatic hypotension symptom assessment scale (OHSA) and four patient function criteria on the orthostatic hypotension daily activities scale (OHDAS). The trial's primary outcome measure, the composite OHQ score, reflects the average improvement (decrease) in mean OHSA and OHDAS scores.

Notably, in addition to the symptomatic and functional benefits registered on the OHQ, an analysis of blood pressure findings validate Northera's unique mechanism of action and confirmed the preferential effect of Northera on standing systolic blood pressure versus supine systolic blood pressure. Northera demonstrated a statistically significant improvement in standing systolic blood pressure (SBP) (p< 0.001) relative to placebo with Northera patients reporting a mean increase of 11.2 mmHg compared to a mean change of 3.9 mmHg in standing SBP for the placebo group.This significant improvement was achieved with a relatively minor impact on supine blood pressure, with mean standing SBP for patients on Northera increasing 12% compared to only a 6% increase in mean supine SBP. Furthermore, no patients receiving Northera had SBP>200 mmHg during the comparative phase of the trial.

No Significant Adverse Events

As anticipated, Northera proved to be safe and well tolerated at all dose levels. Headache (7.4%) was the most common adverse event reported, with all cases considered mild. Consistent with prior study results, treatment with Northera was associated with fewer patient reported falls. In Study 301, all 3 patient-reported falls were in the placebo arm while no patients on Northera arm reported falling during the one-week treatment period.