Chelsea Therapeutics Resubmits New Drug Application for NORTHERA(TM) (droxidopa) for the Treatment of Symptomatic NOH

Chelsea Therapeutics Resubmits New Drug Application for NORTHERA(TM)
(droxidopa) for the Treatment of Symptomatic NOH

         • Submission Includes Successful 300 mg Bioequivalence Study

               • Six Month Review Decision Expected Under PDUFA

CHARLOTTE, N.C., July 9, 2013 (GLOBE NEWSWIRE) -- Chelsea Therapeutics
International, Ltd. (Nasdaq:CHTP) today announced that it resubmitted a New
Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking
approval to market NORTHERA™ (droxidopa), an orally active synthetic precursor
of norepinephrine, 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 NDA
resubmission includes data from Study 306B, a large phase 3 study which
confirms findings from Study 301 demonstrating improvement in
dizziness/lightheadedness, the cardinal symptom of NOH.Under the Prescription
Drug User Fee Act, or PDUFA, the FDA is expected to review and act on the NDA
six months from the date of resubmission.

"The resubmission of our Northera NDA marks a critical step in our effort to
bring an important new therapeutic option to patients living with this
debilitating condition," said Joseph G. Oliveto, Interim Chief Executive
Officer of Chelsea. "We now have two large randomized placebo-controlled
clinical studies which show that treatment with Northera results in consistent
and statistically significant improvements in the symptoms of NOH, and a
greatly expanded database supporting the safety of Northera.We look forward
to working closely with the FDA as they review our application and, in
parallel, planning for commercialization."

In addition to Study 306B, the filing also includes three smaller supportive
randomized controlled trials, two long-term open-label extension studies, a
dedicated thorough QTc study, a 24-hour ambulatory blood pressure monitoring
study and a bioequivalence study for the 300 mg dose form of droxidopa.The
bioequivalence study showed comparable pharmacokinetics between the 300 mg
dose form of droxidopa and the 100 mg and 200 mg combination dose form.

As previously disclosed, FDA has indicated that the revised Northera NDA may
be reviewed by the Cardiovascular and Renal Drug Advisory Committee, or
CRDAC.The FDA has also raised the possibility of accelerated approval for
Northera, where it has indicated that endpoints measuring short-term clinical
benefits can be used as valid surrogates reasonably likely to predict
long-term clinical benefits under accelerated approval. FDA also noted that
the Company may be required to verify the durability of effect of Northera in
a post-marketing study. Chelsea continues to work with the FDA on the design
of such a study, which it intends to initiate in the fourth quarter of 2013.
The study is expected to include short-term clinical endpoints should the
Agency require an additional clinical trial for the approval of Northera.

Northera was previously granted Orphan Drug Designation and received Fast
Track designation from the FDA. 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.

About Symptomatic NOH

NOH is a chronic neurogenic disorder resulting from deficient release of
norepinephrine that predominantly affects patients with primary autonomic
failure, a group of diseases which includes Parkinson's disease (PD), multiple
system atrophy (MSA) and 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, often severely limiting a person's ability to perform routine daily
activities that require standing or walking for both short and long periods of
time.

About Northera

NORTHERA™ (droxidopa), the lead investigational agent in Chelsea Therapeutics'
pipeline, is currently in Phase III development for the treatment of
symptomatic neurogenic orthostatic hypotension (NOH) in patients with primary
autonomic failure — an indication that includes a significant number of
patients with Parkinson's disease, multiple system atrophy (MSA) and pure
autonomic failure (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 is
currently pursuing FDA approval in the U.S. for Northera™ (droxidopa), a
novel, late-stage, orally-active therapeutic agent for the treatment of
symptomatic neurogenic orthostatic hypotension in patients with primary
autonomic failure. 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 does not accept the resubmitted NDA for Northera for
filing and 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 any 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; risk that we
will not be able to obtain regulatory approvals of droxidopa or our other drug
candidates for additional indications; 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.

CONTACT: Investors:
         Fara Berkowitz / Susan Kim
         Argot Partners
         212-600-1902
         fara@argotpartners.com
         susan@argotpartners.com
        
         Media:
         David Pitts
         Argot Partners
         212-600-1902
         david@argotpartners.com

Chelsea Therapeutics Logo
 
Press spacebar to pause and continue. Press esc to stop.