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Chelsea Therapeutics Reports Clinical Trial Data on NORTHERA

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Chelsea Therapeutics
International, Ltd. (Nasdaq: CHTP) today announced the publication in Neurology
of its pivotal, Phase 3 study 301, a multicenter, multinational, double-blind,
randomized, placebo-controlled, parallel-group study of NORTHERA^TM
(droxidopa) that details how NORTHERA demonstrated a statistically significant
difference in efficacy compared to placebo for improving the symptoms of
neurogenic orthostatic hypotension (NOH).

NORTHERA was approved by the U.S. Food and Drug Administration on February 18,
2014, for the treatment of orthostatic dizziness, lightheadedness, or the
"feeling that you are about to black out" in adult patients with symptomatic
neurogenic orthostatic hypotension caused by primary autonomic failure
(Parkinson's disease, multiple system atrophy, and pure autonomic failure),
dopamine beta-hydroxylase deficiency, and non-diabetic autonomic neuropathy.
Effectiveness beyond 2 weeks of treatment has not been demonstrated. The
continued effectiveness of NORTHERA should be assessed periodically.

Data from pivotal study 301, published online ahead of print in Neurology, was
used to support the safety and efficacy of NORTHERA as part of its new drug
application.

"Droxidopa, a norepinephrine prodrug, is the first treatment approved in 20
years for symptomatic neurogenic orthostatic hypotension, a syndrome
characterized by blunted noradrenergic response to standing," said lead author
Horacio Kaufmann, M.D., Professor of Neurology and Medicine at New York
University and Director of the Dysautonomia Center at NYU Langone Medical
Center. "In a double-blind randomized trial, 7-day treatment with droxidopa
was superior to placebo in relieving symptoms and was associated with an
increase in standing systolic blood pressure."

"Neurogenic orthostatic hypotension is a rare and debilitating condition
associated with neurogenic disorders such as Parkinson's Disease and multiple
system atrophy, that is often overlooked and underdiagnosed," said Joseph G.
Oliveto, President and CEO of Chelsea. "The publication of our 301 study in
Neurology, a highly respected medical journal, adds to the peer-reviewed
literature on NOH and will help increase understanding of NOH among
physicians."

The trial examined the efficacy and safety of droxidopa versus placebo. The
primary endpoint was the relative improvement in mean Orthostatic Hypotension
Questionnaire (OHQ) composite score following 1 week of treatment. The OHQ is
a validated, NOH-specific tool assessing symptom severity and symptom impact
on daily activities as reported by patients. When evaluating the OHQ
composite, it was found that droxidopa-treated patients improved by 0.90 units
(p=0.003), compared to placebo.

The OHQ may be divided into two independently validated composite sub scores.
The orthostatic hypotension symptom assessment composite (OHSA), which
examines a variety of symptoms, and the orthostatic hypotension daily
activities assessment composite (OHDAS), which examines a variety of symptom
impacts. Improvement in the OHSA composite score favored droxidopa by 0.73
units (p=0.010). The largest improvement in an individual symptom item was
recorded for item 1 (dizziness/lightheadedness) which favored droxidopa by
1.30 units (p<0.001). Improvement in OHDAS composite favored droxidopa by 1.06
units (p=0.003), with the largest individual item change recorded for "ability
to conduct activities that require standing a long time" which favored
droxidopa by 1.30 units (p=0.001)

A biologically relevant correlate for efficacy, the mean change in standing
systolic blood pressure (BP) increased by 11.2 vs 3.9 mmHg (p<0.001). An
important safety observation was the change in the mean supine systolic BP by
7.6 vs 0.8 mmHg (p<0.001) study. There were relatively few patients who
experienced BP increases above180 mmHg: 4.9 percent of droxidopa and 2.5
percent of placebo recipients.

Overall, this short-term multicenter trial showed that droxidopa treatment was
associated with significant improvement in multiple symptoms of NOH and of NOH
impact on activities requiring standing or walking as well as an associated
increase in standing systolic BP. Furthermore, these benefits were associated
with an acceptable safety profile.

Posted-In: News FDA Press Releases

 

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