CHARLOTTE, N.C., Dec. 9, 2013 (GLOBE NEWSWIRE) -- Chelsea Therapeutics International, Ltd. (Nasdaq:CHTP) today announced that the first patient has been dosed in Study 401, a multi-center, multi-national, randomized, parallel-group, placebo-controlled, double-blind study of investigational drug NORTHERA™ (droxidopa), an orally active synthetic precursor of norepinephrine, being studied for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with primary autonomic failure (Parkinson's disease, multiple system atrophy and pure autonomic failure), dopamine beta hydroxylase deficiency and non-diabetic autonomic neuropathy.
The Company expects Study 401 to enroll approximately 450 patients. Study 401 will evaluate the clinical efficacy and safety of Northera versus placebo over a 17 week (maximum) treatment period consisting of an initial, open-label dose titration (up to 2 weeks), followed by a washout period (up to 3 weeks), followed by a 12 week treatment period on a stable dose. The primary outcome measure of the study is to evaluate the duration of clinical benefit of Northera as demonstrated by the change in the Orthostatic Hypotension Symptom Assessment (OHSA) Item 1, which includes symptoms of dizziness and lightheadedness. Secondary outcome measures include patient reported falls, standing blood pressure change, and other efficacy measures, including the orthostatic hypotension questionnaire (OHQ) composite and individual item scores and the clinical global impression scales.
"Chelsea is committed to understanding the long-term safety and durability of Northera in symptomatic nOH, a goal we hope to achieve through Study 401, the largest randomized, placebo-controlled study undertaken in this orphan indication," said Joseph G. Oliveto, Interim Chief Executive Officer of Chelsea Therapeutics. "We thank the many investigators who have committed to participating in this global study, as well as the patients and their families who will take part in it. There exists a significant unmet need in nOH, and it remains Chelsea's goal to bring new treatment options to those suffering from this debilitating condition."
Chelsea Therapeutics is currently seeking approval from the U.S. Food and Drug Administration (FDA) to market Northera for the treatment of symptomatic nOH in adult patients with primary autonomic failure, non-diabetic autonomic neuropathy and dopamine-beta-hydroxylase deficiency. The company's New Drug Application is scheduled for review by the Cardiovascular and Renal Drug Advisory Committee on January 14, 2014, with a Prescription Drug User Fee Act target date of February 14, 2014.
About Symptomatic nOH
It is estimated that nearly 300,000 patients suffer from chronic symptomatic nOH in the U.S. and EU combined. Symptomatic nOH is a chronic disorder that is caused by an underlying neurogenic disorder, such as Parkinson's disease, multiple system atrophy (MSA) or pure autonomic failure (PAF). Symptoms of nOH include dizziness, lightheadedness, blurred vision, fatigue, poor concentration, and fainting episodes when a person assumes a standing position. These symptoms often severely limit a person's ability to perform routine daily activities that require standing or walking for both short and long periods of time.
NORTHERA™ (droxidopa), the lead investigational agent in Chelsea Therapeutics' pipeline, is currently in Phase III development for the treatment of symptomatic nOH in patients with primary autonomic failure — an indication that includes a significant number of patients with Parkinson's disease, MSA and PAF. Droxidopa is a synthetic catecholamine that is directly converted to norepinephrine (NE) via decarboxylation, resulting in increased levels of NE in the nervous system, both centrally and peripherally.
Droxidopa, developed by and licensed from Dainippon Sumitomo Pharma Co., Ltd. (DSP), initially received Japanese approval in 1989 for the treatment of frozen gait and dizziness on standing associated with Parkinson's Disease and for the treatment of orthostatic hypotension, syncope or dizziness on standing associated with Shy-Drager syndrome and Familial Amyloidotic Polyneuropathy. In 2000, Droxidopa received expanded marketing approval to include prevention of vertigo, dizziness and weakness associated with orthostatic hypotension in hemodialysis patients.
About Chelsea Therapeutics
Chelsea Therapeutics (Nasdaq:CHTP) is a biopharmaceutical development company that acquires and develops innovative products for the treatment of a variety of human diseases, including central nervous system disorders. Chelsea acquired global development and commercialization rights to droxidopa (L-DOPS), or NORTHERA, from Dainippon Sumitomo Pharma Co., Ltd. in 2006, excluding Japan, Korea, China and Taiwan. For more information about the Company, visit www.chelseatherapeutics.com.
This press release contains forward-looking statements regarding future events including our intention to pursue the development of NORTHERA . These statements are subject to risks and uncertainties that could cause the actual events or results to differ materially. These include reliance on key personnel and our ability to attract and/or retain key personnel; the risk that FDA will not agree that our clinical trial results demonstrate the safety and effectiveness of droxidopa; the risk that the FDA will not accept our proposal regarding any trial or other data to support a new drug application; the risk that the FDA will not approve the resubmitted NDA; the risk that our resources will not be sufficient to conduct any study of Northera that will be acceptable to the FDA; the risk that we cannot complete Study 401 or any other additional study for Northera without the need for additional capital; the risks and costs of drug development and that such development may take longer or be more expensive than anticipated; our need to raise additional operating capital in the future; our reliance on our lead drug candidate droxidopa; the risk that we will not be able to obtain regulatory approvals of droxidopa or our other drug candidates for additional indications; the risk of volatility in our stock price, related litigation, and analyst coverage of our stock; reliance on collaborations and licenses; intellectual property risks; our history of losses; competition; market acceptance for our products if any are approved for marketing.
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