Vanda Announces Positive Results in the Second Phase III Study
(RESET) of Tasimelteon for the Treatment of Non-24-Hour Disorder
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 http://Non-24.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 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
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, 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 February 21, 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:
Cristina
Murphy
Senior Communications Manager
Vanda Pharmaceuticals Inc.
(202) 734-3414
cristina.murphy@vandapharma.com
Laney Cohen
Account Supervisor
Makovsky & Company, Inc.
(212)-508-9643
lcohen@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; 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 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.