Chelsea completes enrollment in Northera Phase III Study 301 for neurogenic orthostatic hypotension

Jun 30 2010

• Top-line Data Expected in Q3 10

Chelsea Therapeutics International, Ltd. (Nasdaq:CHTP) has successfully reached its target enrollment of 150 patients in its Northera™ (Droxidopa) pivotal Phase III Study 301 for the treatment of symptomatic, neurogenic orthostatic hypotension (NOH). Results from the trial are expected in September 2010 and the full registration program is on track to initiate a new drug application (NDA) in the first half of 2011.

NOH is a disorder of the nervous system resulting from a deficient release of norepinephrine, the neurotransmitter used by sympathetic autonomic nerves to send signals to the blood vessels and the heart. This deficiency results in lightheadedness, dizziness, blurred vision and fainting episodes when a person assumes a standing position. Northera, an oral compound which the body converts directly to norepinephrine, is the only therapeutic treatment to specifically target the underlying cause of NOH: norepinephrine deficiency.

"We are delighted to have reached our target enrollment in Study 301 and are confident the trial will capture the full therapeutic benefit of Northera in symptomatic NOH," commented Dr. Simon Pedder, president and CEO of Chelsea Therapeutics. "We are grateful to all the clinicians and patients for their continued strong support and participation in this trial. We look forward to seeing the top-line results from this trial in September and continuing to work with the clinical investigators and their staff throughout the year to complete our other on-going Phase III trial and file a new drug application next year."

Study 301 was reviewed by the U.S. Food and Drug Administration (FDA) and awarded a Special Protocol Assessment (SPA) in February 2008. The FDA subsequently confirmed that the SPA remained in effect following the protocol amendments approved by the FDA late last year. An SPA provides a binding agreement that the study design, including trial size, clinical endpoints and/or data analyses is acceptable to support regulatory approval.

In addition to the SPA, the FDA has awarded Chelsea Fast Track designation for its pivotal program in NOH. Fast Track designation is designed to facilitate the review of products that address serious or potentially life-threatening conditions for which there is an unmet medical need and provides the option to file a New Drug Application (NDA) on a rolling basis. This permits the FDA to review the filing as it is received, expediting the review process.