* Top-line Data From First Pivotal Trial Anticipated Later This Month
* Rolling NDA Submission to be Initiated in Q4 09
CHARLOTTE, N.C., Sept. 9, 2009 (GLOBE NEWSWIRE) -- Chelsea Therapeutics International, Ltd. (Nasdaq:CHTP) has successfully reached its target enrollment of 118 randomized patients for Study 301, the second of two pivotal Phase III clinical trials in Chelsea's registration program of Droxidopa for the treatment of symptomatic, neurogenic orthostatic hypotension (NOH).
"With top-line data from Study 302 expected within weeks, reaching our target enrollment in Study 301 marks a significant turning point in our Droxidopa development program as we conclude our clinical efficacy evaluations and move ahead with our regulatory submission and commercialization efforts," commented Dr. Simon Pedder, Chelsea's President and CEO. "Top-line data from Study 301 is expected late next quarter and we anticipate initiating a rolling NDA submission in the same quarter."
About the Trial and Droxidopa Registration Program
The Droxidopa Phase III registration program in NOH includes two highly similar, double-blind, placebo-controlled studies: Study 301 and Study 302. Both studies compare Droxidopa to placebo for the treatment of symptomatic NOH and are designed to demonstrate a mean improvement over placebo of 1.6 units on the Orthostatic Hypotension Symptom Assessment (OHSA) scale. The OHSA scale is a validated scale designed to rate symptoms occurring specifically because of low blood pressure and uses an 11-point scale (zero to 10), with more severe symptoms scoring higher. To date, both studies have demonstrated a greater than 4 unit improvement on the OHSA scale during their respective open-label dose titration phases. Given the relatively short duration of the study, top-line data from Study 302 is expected late in the third quarter of 2009.
Study 301was reviewed by the U.S. Food and Drug Administration (FDA) and awarded a Special Protocol Assessment (SPA) in February 2008. 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.
About Droxidopa and Symptomatic Neurogenic Orthostatic Hypotension (NOH)
Symptomatic NOH is a neurogenic disorder 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 decreased blood pressure when a person assumes a standing position and is characterized by lightheadedness, dizziness, blurred vision and syncope. Droxidopa, an orally active synthetic precursor of norepinephrine, increases the supply of norepinephrine available for delivery to its receptors to improve orthostatic blood pressure and alleviate symptoms of orthostatic hypotension.
Chelsea estimates that over 300,000 patients suffer from chronic symptomatic NOH in the U.S. and the E.U. In addition to creating significant health care costs, symptomatic NOH has a dramatic impact on the quality of patient life. Midodrine, currently the only FDA approved treatment for orthostatic hypotension, has not been shown to be effective in alleviating the symptoms of this condition. Moreover, it is limited in its use by a pronounced side-effect profile and black box warning for supine hypertension. Given the chronic nature of symptomatic NOH and the proven safety and tolerability of Droxidopa, Chelsea expects that daily oral treatment with Droxidopa should provide a significant improvement in the long-term treatment of symptomatic NOH.
About Chelsea Therapeutics
Chelsea Therapeutics is a biopharmaceutical development company that acquires and develops innovative products for the treatment of a variety of human diseases. Chelsea's most advanced drug candidate, Droxidopa, is an orally active synthetic precursor of norepinephrine initially being developed for the treatment of neurogenic orthostatic hypotension. Currently approved and marketed in Japan for the treatment of symptomatic orthostatic hypotension, freezing gait in Parkinson's disease and intradialytic hypotension, Droxidopa has accumulated over 15 years of proven safety and efficacy in Japan. In addition to Droxidopa, Chelsea is also developing a portfolio of metabolically inert oral antifolate molecules engineered to have potent anti-inflammatory and anti-tumor activity to treat a range of immunological disorders, including two clinical stage product candidates: CH-1504 and CH-4051. Preclinical and clinical data suggests superior safety and tolerability, as well as increased potency versus methotrexate (MTX), currently the leading antifolate treatment and standard of care for a broad range of abnormal cell proliferation diseases including RA.
This press release contains forward-looking statements regarding future events. These statements are just predictions and are subject to risks and uncertainties that could cause the actual events or results to differ materially. These risks and uncertainties include our need to raise operating capital, our history of losses, risks and costs of drug development, risk of regulatory approvals, our reliance on our lead drug candidates droxidopa and CH-1504, reliance on collaborations and licenses, intellectual property risks, competition, market acceptance for our products if any are approved for marketing, reliance on key personnel including specifically Dr. Pedder.