Chelsea Therapeutics Announces Northera(TM) (Droxidopa) Study 306B Meets Primary Endpoint

Chelsea Therapeutics Announces Northera(TM) (Droxidopa) Study 306B Meets
Primary Endpoint

  oNorthera™ Demonstrated Statistically Significant Improvement Over Placebo
    in Reduction of Dizziness/Lightheadedness at Week 1 (p=0.018), the Primary
    Endpoint, and Standing Blood Pressure at Week 1 (p=0.032), a Key Secondary
  oNorthera Patients Experienced Fewer Falls and Fall-related Injuries,
    Although Results Were Not Statistically Significant
  oNorthera was Well Tolerated with Safety Results Consistent with Those from
    Previous Studies
  oFDA Previously Advised Chelsea Study 306B is Unlikely to Provide
    Sufficient Confirmatory Evidence to Support a Northera NDA
  oManagement to Host Conference Call with More Detailed Data Presentation
    Today at 5:00 PM ET to Discuss Results

CHARLOTTE, N.C., Dec. 4, 2012 (GLOBE NEWSWIRE) -- Chelsea Therapeutics
International, Ltd. (Nasdaq:CHTP) today announced positive preliminary results
for Study 306B, a Phase III trial evaluating the safety and efficacy of
investigational drug Northera™ (droxidopa) for the treatment of symptomatic
neurogenic orthostatic hypotension (NOH) associated with Parkinson's Disease
(PD), including achievement of the study's pre-defined, pre-specified primary

The results showed that treatment with Northera provided clinically meaningful
and statistically significant improvements compared to placebo in
dizziness/lightheadedness at week 1 (1.0 unit change; p=0.018), the primary
endpoint.In addition, compared to placebo, a statistically significantly
greater number of patients were observed to experience 2, 3 or 4 unit
improvements at week 1 compared to baseline (all p-values<0.05).Study results
also demonstrated a statistically significant increase in standing systolic
blood pressure (SBP) at week 1 (5.6 mmHg; p=0.032), a key secondary endpoint
of the study.At time points beyond week 1, dizziness/lightheadedness and
standing blood pressure predominantly favored Northera-treated patients,
although the results were not statistically significant.

Treatment with Northera also resulted in a reduction in the rate of patient
falls over the course of the study, although these results were not
statistically significant. Patients receiving placebo experienced a rate of
falls per patient per week of 2.0 vs. 0.4 for those on Northera, an 80%
reduction.Because several patients on placebo experienced a very large number
of falls, Chelsea performed multiple sensitivity analyses on this
outcome.These analyses showed that the beneficial effect of Northera on falls
was evident even if the top 2, 5 or 10 fallers from each treatment group were
removed (34%, 36% and 29% reduction, respectively, p=NS).Importantly, the
falls data were supported by additional safety data showing that 34% fewer
patients receiving Northera experienced fall-related injuries (e.g.,
contusions, lacerations, fractures) than patients receiving placebo
(placebo=25.6% vs. Northera=16.9%, p=NS).Both the reduction in falls and
fall-related injuries associated with Northera are consistent with results
observed in Study 306A.

Patient falls are the leading cause of death in the elderly, and for PD
patients, injuries sustained due to falls are the primary reason for being
admitted to the hospital.

Preliminary safety data show Northera was well tolerated at all dosages
tested. Adverse events occurring in at least 5% of Northera patients with at
least a 1% difference in incidence between arms were headache (Northera=13.5%
vs. placebo=7.3%), an event of dizziness (10.1% v. 4.9%), nausea (7.9% v.
2.4%) and hypertension (7.9% v. 1.2%). Adverse events were predominately mild
to moderate in severity and generally consistent with previous studies.

As in prior studies, the incidence of supine hypertension was low; 3% of
patients on Northera and 1% of patients on placebo had measurements of supine
SBP of >200mmHg. Seven percent of patients treated with Northera vs. 5%
receiving placebo had measurements of supine SBP of >180mmHg. Four
Northera-treated patients (4.5%) and 5 placebo-treated patients (6.1%)
discontinued treatment due to hypertension.

"Data from Study 306B are consistent with the findings of 306A and other
Northera clinical studies. They demonstrate an improvement in dizziness and
lightheadedness and suggest a reduction in falls for patients taking Northera.
In addition, Northera was well tolerated, with a relatively low incidence of
supine hypertension," commented Dr. Robert A. Hauser, Professor of Neurology,
Molecular Pharmacology, and Physiology, and Director of the Parkinson's
Disease Movement Disorders Center, University of South Florida and principal
investigator for the trial."There is an urgent need for new therapies that
help alleviate the potentially disabling symptoms of NOH and keep patients

Following an interim analysis, Study 306 was divided into two parts, 306A and
306B. 306A enrolled 51 patients, and 306B enrolled 174 patients. Study 306B is
the largest randomized, placebo-controlled Phase III trial ever conducted in
NOH, and was performed entirely in the U.S. Patients were treated for up to 10
weeks, including an initial blinded dosage titration period that lasted up to
two weeks. The study used a pre-specified modified intent-to-treat analysis on
the primary analysis population (n=147) which was defined as all randomized
patients who received at least one dose of study drug and completed an
orthostatic hypotension questionnaire (OHQ) assessment at visit 4 (week 1).A
total of 27 patients dropped out prior to visit 4 (6 placebo, 18 Northera, 3
prior to dosing) and were therefore not available for OHQ assessment. The mean
dose (TID) at week 1 was 436 mg for Northera and 468 mg for placebo.

A meta-analysis of the combined studies 306A and 306B (n=225, mITT: n=197)
showed highly statistically significant improvements in
dizziness/lightheadedness at week 1 (1.2 unit difference vs. placebo;
p=0.008), and a strong trend toward improvement in dizziness/lightheadedness
at week 8 (0.8 unit difference vs. placebo; p=0.077). These results also
showed a statistically significant improvement in standing blood pressure
compared to placebo after week 1 (6.8 mmHg; p=0.014), and a numerical
improvement in standing blood pressure at week 8 (2.2 mmHg difference vs.
placebo; p=0.414).

"We are pleased with these results from Study 306B as they are consistent with
the safety and efficacy of Northera observed in our previous randomized,
controlled trials.Furthermore, we now have studied over 650 NOH patients,
constituting a large safety and efficacy database in this orphan indication,"
said Joseph G. Oliveto, Interim Chief Executive Officer of Chelsea. "We look
forward to more fully evaluating this rich dataset and working with key
opinion leaders and health authorities to further define our path to secure
marketing approval."

The U.S. Food and Drug Administration previously advised Chelsea that Study
306B is unlikely to provide sufficient confirmatory evidence to support a
Northera New Drug Application.

Conference Call and Presentation Today at 5:00 PM ET

Chelsea will host a conference call to discuss the Northera Phase III clinical
trial results today, December 4, 2012, at 5:00 PM Eastern Time. Interested
investors may participate in the conference call by dialing (877) 638-9567
(domestic) or (720) 545-0009 (international) and referencing conference ID
number: 76835721.A presentation, which will include Study 306B results in
more detail, will accompany the call and be available at 15 minutes prior to the start of the call. A
replay will be available for one week following the call by dialing (855)
859-2056 for domestic participants or (404) 537-3406 for international
participants and referencing conference ID number: 76835721 when prompted.
Participants may also access both the live and archived webcast of the
conference call and presentation on Chelsea's web site at The detailed data presentation will also be
available through a Securities and Exchange Commission filing on Form 8K.

About Neurogenic Orthostatic Hypotension Associated with Parkinson's Disease

Parkinson's disease (PD) is the second most common neurodegenerative disorder
in America. As a result of decreased levels of norepinephrine associated with
PD, approximately 20% of PD patients may experience symptomatic NOH. NOH is a
neurogenic disorder resulting from deficient release of norepinephrine, the
neurotransmitter used by sympathetic autonomic nerves to send signals to the
blood vessels and the heart to regulate blood pressure. This deficiency
results in decreased blood pressure when a person assumes a standing position
and is characterized by lightheadedness, dizziness, and falls. Symptoms of
chronic NOH can be incapacitating, not only putting patients at high risk for
falls and generating significant health care costs, but also severely
affecting the quality of life for patients and their loved ones.

About Northera

NORTHERA™ (droxidopa), the lead investigational agent in Chelsea Therapeutics'
pipeline, is currently in Phase III clinical trials for the treatment of
symptomatic neurogenic orthostatic hypotension (NOH) in patients with primary
autonomic failure – a group of diseases that includes 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.

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

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; risks of
distraction of the Board and management at this critical time; the risk that
the FDA will not accept our proposal regarding any trial or other data to
support a NDA for Northera; the risk that we will not be able to resubmit the
NDA for Northera and that the FDA will not approve a 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 and ability to raise
additional operating capital in the future; our reliance on our lead drug
candidate Northera; risk of regulatory approvals of Northera 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; and competition.

CONTACT: Investors:
         Michelle Carroll / Susan Kim
         Argot Partners
         David Pitts
         Argot Partners

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