Vanda Announces Positive Results in the Second Phase III Study (RESET) of Tasimelteon for the Treatment of Non-24-Hour Disorder

  Vanda Announces Positive Results in the Second Phase III Study (RESET) of
            Tasimelteon for the Treatment of Non-24-Hour Disorder

-- Tasimelteon significantly maintains entrainment of circadian rhythms

-- Discontinuation of tasimelteon results in significant clinical

PR Newswire

WASHINGTON, Jan. 23, 2013

WASHINGTON, Jan. 23, 2013 /PRNewswire/ --Vanda Pharmaceuticals Inc. (NASDAQ:
VNDA), today announced positive results for the second Phase III study of
tasimelteon for the treatment of Non-24-Hour Disorder (Non-24). The RESET
study (Randomized-withdrawal study of the Efficacy and Safety of Tasimelteon
to treat Non-24-Hour Disorder), demonstrated the maintenance effect of 20mg of
tasimelteon to entrain melatonin and cortisol circadian rhythms in individuals
with Non-24. Tasimelteon treated patients maintained their clinical benefits
while placebo treated patients showed significant deterioration in measures of
nighttime sleep, daytime naps, and timing of sleep. 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.

"These results clearly demonstrate that tasimelteon can entrain the circadian
clock and continued treatment is necessary to maintain entrainment," said
Steven W. Lockley, Ph.D., Division of Sleep Medicine, Brigham and Women's
Hospital, a teaching affiliate of Harvard Medical School. "The study also
shows that entrainment is associated with meaningful clinical benefits and
that maintaining entrainment of the master body clock is critical to treating
the problems caused by Non-24."

"We are excited by these results as they move us one step closer towards
providing a treatment for blind individuals with Non-24," said Mihael H.
Polymeropoulos, M.D., President and CEO of Vanda. "These results also
highlight the importance of chronic therapy in treating Non-24. We are
confident that if approved, tasimelteon may significantly improve the quality
of life for individuals with Non-24."

RESET Study Results Summary

Primary Endpoint

The RESET study was a 20 patient randomized withdrawal study designed to
demonstrate the maintenance effect of 20mg of tasimelteon in the treatment of
blind individuals with Non-24.Patients were treated with tasimelteon for
three months during an open-label run-in phase. Patients who responded to
tasimelteon treatment during the run-in phase, as measured by entrainment of
the melatonin rhythm (aMT6s) to the 24-hour day, were then randomized to
receive either placebo or continue receiving tasimelteon 20mg for 2 months.
The primary endpoint of the study was the maintenance of effect as measured
by entrainment of the melatonin (aMT6s) rhythm.

Primary Endpoint Results

                                       Tasimelteon Placebo p-value

Maintenance of entrainment (aMT6s) (%) 90.0        20.0    0.0026

Secondary Endpoints

The RESET study also assessed a number of secondary endpoints including
maintenance of entrainment of the cortisol rhythm and a range of sleep and
wake parameters including LQ-nTST (total nighttime sleep in the worst 25% of
nights), UQ-dTSD (total daytime sleep duration in the worst 25% of days) and
MoST (midpoint of sleep timing from both nighttime and daytime sleep).

Secondary Endpoint Results

                                        Tasimelteon Placebo Difference p-value

Maintenance of entrainment (cortisol)   80.0        20.0    60.0       0.0118
LQ-nTST (LS mean minutes)^1             -6.6        -73.8   67.2       0.0233
UQ-dTSD (LS mean minutes)^2             -9.6        49.8    -59.4      0.0266
MoST (LS mean minutes)^1                19.8        -16.2   36.0       0.0108
1) Higher number indicates improvement
2) Lower number indicates improvement

From the run-in phase of the study, the rate of entrainment among tasimelteon
treated patients ranged from 50% to 85% based on individual patient
characteristics. In a time to relapse analysis (45 min decrement of weekly
average nighttime sleep), placebo treated patients relapsed in higher numbers
and at an earlier time than tasimelteon treated patients (P = 0.0907).

The RESET study demonstrates the efficacy of chronic treatment with
tasimelteon in Non-24 and further supports the results of the SET study, which
established the ability of tasimelteon to entrain the master body clock and
significantly improve the clinical symptoms of Non-24. Vanda plans to submit
a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in
mid-2013. Vanda will meet with the FDA in Q1 of 2013 for a pre-NDA meeting on
tasimelteon in the treatment of patients with 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 from 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. Individuals with
Non-24 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. This circadian disorder 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

About Tasimelteon

Tasimelteon is a circadian regulator in development for the treatment of
Non-24. Tasimelteon is a melatonin agonist of the human MT[1] and MT[2]
receptors, with greater specificity for MT[2]. ^ 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 Phase III development for Non-24 and Phase IIb/III for Major
Depressive Disorder (MDD).

Conference Call
Vanda has scheduled a conference call for today, Wednesday, January 23, 2013
at 9 AM ET to discuss the trial results. Investors can call 1-866-713-8565
(domestic) and 1-617-597-5324 (international) and use passcode 60037962. A
replay of the call will be available beginning Wednesday, January 23, 2013, at
11:00 AM ET and will be accessible until Wednesday, January 30, 2013, at 11:59
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 97367748.

The conference call will be broadcast simultaneously on Vanda's website, 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 February 21,

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 

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

Media Contact:
Cristina Murphy
Senior Communications Manager
Vanda Pharmaceuticals Inc.
(202) 734-3414

Laney Cohen
Account Supervisor
Makovsky & Company, Inc.


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; Vanda's failure to obtain regulatory
approval for tasimelteon for the treatment of Non-24-Hour Disorder or to
comply with ongoing regulatory requirements; the failure of Vanda's clinical
trials to demonstrate the safety and/or efficacy of tasimelteon in the
treatment of Major Depressive Disorder; 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 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.

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