Vanda Announces Positive Phase III Results For Tasimelteon In The Treatment Of Non-24-Hour Disorder

Vanda Announces Positive Phase III Results For Tasimelteon In The Treatment Of
                             Non-24-Hour Disorder

-- Tasimelteon is shown to entrain the master body clock as measured by
melatonin and cortisol circadian rhythms

-- Tasimelteon is shown to significantly improve clinical symptoms across a
number of sleep and wake measures

PR Newswire

WASHINGTON, Dec. 18, 2012

WASHINGTON, Dec. 18, 2012 /PRNewswire/ -- Vanda Pharmaceuticals Inc.
(NASDAQ:VNDA) today announced positive results from the SET (Safety and
Efficacy of Tasimelteon) Phase III study, evaluating tasimelteon, a circadian
regulator for the treatment of Non-24-Hour Disorder (Non-24). Tasimelteon
succeeded in the primary endpoint of Entrainment of the melatonin (aMT6s)
rhythm as compared to placebo. 

Additionally, tasimelteon demonstrated significant improvements across a
number of sleep and wake parameters including measures of total sleep time,
nap duration, and timing of sleep. Tasimelteon also showed significant
improvements over placebo in the Non-24 Clinical Response Scale (N24CRS) as
well as in the Clinical Global Impression of Change (CGI-C), an overall global
functioning scale. These results provide robust evidence of a direct and
clinically meaningful benefit to patients with Non-24.

Non-24 is a serious, rare circadian rhythm disorder that affects a majority of
totally blind individuals who lack light perception and cannot entrain (reset)
their master body clock to the 24-hour day. Currently there is no approved
treatment for Non-24.

"Today's results confirm tasimelteon as a strong circadian regulator capable
of entraining the master body clock in patients with Non-24. We are
particularly impressed and excited by the magnitude and robustness of the
direct clinical benefits to patients," said Mihael H. Polymeropoulos, M.D.,
President and CEO of Vanda. "We believe that tasimelteon can be an effective
and clinically meaningful treatment for patients suffering with this
debilitating disorder."

"As a person who regularly experiences the debilitating symptoms of Non-24,
these findings are important to me and I think they are important to the blind
community as a whole, because they give us hope that a potential new treatment
approach is on the horizon," said Melanie Brunson, Executive Director of the
American Council of the Blind.

Primary Endpoints

The SET study was an 84 patient randomized, double-masked, placebo-controlled
study in patients with Non-24. The primary endpoints for this study were
Entrainment of the melatonin (aMT6s) rhythm to the 24-hour clock and Clinical
Response as measured by Entrainment plus a score of greater than or equal to 3
on N24CRS.

Primary Endpoint Results

Table 1

                                 Tasimelteon (%) Placebo (%) p-value
Entrainment (aMT6s)              20.0            2.6         0.0171
Clinical Response
                                 23.7            0.0         0.0028
(Entrainment^1+ N24CRS >=3)^
 Clinical Response^2
                                 28.9            0.0         0.0006
 (Entrainment^1+ N24CRS >=2)
 N24CRS >=3^2                28.9            2.9         0.0031
 N24CRS >=2^2                57.9            20.6        0.0014

1) Entrainment status from the randomized portion of the SET study and/or the
screening portion of the RESET study
2) Sensitivity Analysis

Secondary Endpoints

The SET study also assessed a number of secondary endpoints including
Entrainment of cortisol rhythm and a broad range of clinical sleep and wake
parameters. These parameters included improvement in the total nighttime
sleep in the worst 25% of nights (LQ-nTST), decrease in the total daytime
sleep duration in the worst 25% of days (UQ-dTSD) and midpoint of sleep timing
(MoST) which is derived from a combination of the sleep reported for both
nighttime and daytime. CGI-C is a seven-point rating scale of global
functioning with lower scores indicating larger improvements.

Secondary Endpoint Results

Table 2

                                    Tasimelteon Placebo p-value
Entrainment (cortisol) (%)          17.5         2.6      0.0313
N24CRS(LS mean)                    1.77         0.67     0.0004
CGI-C^1 (LS mean)                  2.6          3.4      0.0093
LQ-nTST and UQ-dTSD >=90 min^2 (%) 23.8         4.5      0.0767
LQ-nTST and UQ-dTSD >= 45 min^3(%)  31.6         8.8      0.0177
LQ-nTST(LS mean minutes)           57.0         16.8     0.0055
UQ-dTSD^1 (LS mean minutes)        -46.2        -18.0    0.0050
MoST(LS mean minutes)              34.8         14.4     0.0123

1) For CGI-C and UQ-dTSD smaller numbers indicate improvement.
2) For this endpoint, only subjects with significant sleep and nap problems at
baseline were included.
3) Sensitivity Analysis

The results of the SET study represent the initial data from the tasimelteon
Non-24 Phase III development program and demonstrate the multiple benefits of
this novel therapy in treating patients suffering from this rare circadian
rhythm disorder. In the SET study, tasimelteon was demonstrated to be safe
and well tolerated. Vanda expects to report top-line results from the second
Phase III study (RESET) for tasimelteon in Non-24 in the first quarter of
2013. Vanda plans to submit a New Drug Application (NDA) to the U.S. Food and
Drug Administration (FDA) in mid-2013.

"We would like to thank our patients, their advocates, investigators, advisors
and colleagues for making this study possible," said Mihael H. Polymeropoulos,
M.D., President and CEO of Vanda. "We look forward to the successful
completion of the Non-24 clinical program."

Non-24 Scale of Clinical Response (N24CRS)

Assessment Threshold of response
LQ-nTST    >=45 minutes increase in average nighttime sleep duration
UQ-dTSD    >=45 minutes decrease in average daytime sleep duration
MoST       >=30 minutes increase and a standard deviation <=2 hours during
           double-masked phase
CGI-C      <=2.0 from the average of Day 112 and Day 183 compared to baseline

Tasimelteon Development Program for Non-24

The SET study is the first of four clinical studies conducted as part of
Vanda's Phase III development program for tasimelteon in the treatment of
Non-24. Data from the RESET study, a Phase III study evaluating the
maintenance of entrainment effect of tasimelteon, is expected in the first
quarter of 2013. In addition, two safety studies are ongoing to support an
NDA filing in the U.S. The tasimelteon Non-24 development program is the
largest conducted to date for any investigational therapy for the treatment of
Non-24.

About Non-24-Hour Disorder

Non-24-Hour Disorder (Non-24) is a serious, rare and chronic circadian rhythm
disorder that affects a majority of totally blind individuals in the U.S., or
between 65,000 and 95,000 people. Tasimelteon has been granted orphan drug
designation for the treatment of Non-24 in both the U.S. and the European
Union. Non-24 occurs almost entirely in individuals who lack the light
sensitivity necessary to entrain, or synchronize, the master body clock in the
brain with the 24-hour day-night cycle. Most people have a master body clock
that naturally runs longer than 24-hours, and light is the primary
environmental cue that resets it to 24-hours each day. Non-24 sufferers have
a master body clock that continually delays, putting them to sleep later and
later each day, turning night into day and day into night, until the cycle
starts all over again. The sleep condition is highly disruptive, making it
difficult to do well in school, hold down a job or maintain relationships.
For more information on Non-24, please visit http://24sleepwake.com/.

About Tasimelteon

Tasimelteon is a circadian regulator in development for the treatment of
Non-24. Tasimelteon is a melatonin agonist of the human MT1 and MT2 receptors,
with a 2-4 times greater specificity for MT2. Tasimelteon's ability to reset
the master body clock in the suprachiasmatic nucleus (SCN), located in the
hypothalamus, results in the entrainment of the body's melatonin and cortisol
rhythms to align to the 24-hour day-night cycle. Tasimelteon is currently in
development for both Non-24 and Major Depressive Disorder (MDD).

Conference Call
Vanda has scheduled a conference call for today, Tuesday, December 18, 2012 at
9 AM ET to discuss the trial results. Investors can call 1-800-901-5231
(domestic) and 1-617-786-2961 (international) and use passcode 51793839.A
replay of the call will be available beginning Tuesday, December 18, 2012, at
11:00 AM ET and will be accessible until Tuesday, December 25, 2012, at 12:00
PM ET.The replay call-in number is 1-888-286-8010 for domestic callers and
1-617-801-6888 for international callers. The access number is 17591426.

The conference call will be broadcast simultaneously on Vanda's website,
http://www.vandapharma.com. Investors should click on the Investor Relations
tab and are advised to go to the website at least 15 minutes early to
register, download and install any necessary software. The call will also be
archived on Vanda's website for a period of 30 days, through January 17, 2013.

About Vanda Pharmaceuticals Inc.:
Vanda Pharmaceuticals Inc. is a biopharmaceutical company focused on the
development and commercialization of products for the treatment of central
nervous system disorders. For more on Vanda, please visit
http://www.vandapharma.com. 

Company Contact:
Jim Kelly
Senior Vice President and Chief Financial Officer
Vanda Pharmaceuticals Inc.
(202) 734-3428
jim.kelly@vandapharma.com

Media Contact:
Kristie Kuhl
Senior Vice President
Makovsky & Company, Inc.
(212)-508-9642
kkuhl@makovsky.com

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
Various statements in this release are "forward-looking statements" under the
securities laws. Words such as, but not limited to, "believe," "expect,"
"anticipate," "estimate," "intend," "plan," "project," "target," "goal,"
"likely," "will," "would," and "could," or the negative of these terms and
similar expressions or words, identify forward-looking statements.
Forward-looking statements are based upon current expectations that involve
risks, changes in circumstances, assumptions and uncertainties. Important
factors that could cause actual results to differ materially from those
reflected in the company's forward-looking statements include, among others:
the inability to reach agreement with the FDA regarding Vanda's regulatory
approval strategy or proposed path to approval for tasimelteon for the
treatment of Non-24-Hour Disorder; the failure of Vanda's clinical trials to
demonstrate the safety and/or efficacy of tasimelteon in the treatment of
Non-24-Hour Disorder or Major Depressive Disorder; Vanda's failure to obtain
regulatory approval for tasimelteon for the treatment of Non-24-Hour Disorder
or to comply with ongoing regulatory requirements; delays in the completion of
Vanda's RESET clinical trial for tasimelteon; and other factors that are
described in the "Risk Factors" and "Management's Discussion and Analysis of
Financial Condition and Results of Operations" sections of Vanda's annual
report on Form 10-K for the fiscal year ended December 31, 2011 which is on
file with the SEC and available on the SEC's website at www.sec.gov. In
addition to the risks described above and in Vanda's annual report on Form
10-K and quarterly reports on Form 10-Q, other unknown or unpredictable
factors also could affect Vanda's results. There can be no assurance that the
actual results or developments anticipated by Vanda will be realized or, even
if substantially realized, that they will have the expected consequences to,
or effects on, Vanda. Therefore, no assurance can be given that the outcomes
stated in such forward-looking statements and estimates will be achieved.

All written and verbal forward-looking statements attributable to Vanda or any
person acting on its behalf are expressly qualified in their entirety by the
cautionary statements contained or referred to herein. Vanda cautions
investors not to rely too heavily on the forward-looking statements Vanda
makes or that are made on its behalf. The information in this release is
provided only as of the date of this release, and Vanda undertakes no
obligation, and specifically declines any obligation, to update or revise
publicly any forward-looking statements, whether as a result of new
information, future events or otherwise.

SOURCE Vanda Pharmaceuticals Inc.

Website: http://www.vandapharma.com
 
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