SAN DIEGO, Dec. 10, 2014 /PRNewswire/ -- Neurocrine
Biosciences, Inc. (NASDAQ: NBIX) announced today that NBI-77860, a
proprietary corticotropin releasing factor 1 (CRF) receptor
antagonist, has entered clinical trials for the treatment of
classic congenital adrenal hyperplasia (CAH) a disease that affects
approximately 20,000-30,000 people in the
United States. The Company has successfully completed a
pilot clinical trial in adults with classic CAH and is initiating
an open-label, single ascending dose trial in approximately fifteen
adolescent females with classic CAH, the 1401 Study.
"We are very pleased with the results from our pilot study of
NBI-77860 in patients that was conducted earlier this year and
excited to add another significant program to our clinical
development pipeline," said Christopher F.
O'Brien, Chief Medical Officer of Neurocrine Biosciences.
"We have requested orphan drug status from the FDA while we
continue to expand our preclinical and clinical efforts around
NBI-77860 in classic CAH."
"I am very pleased with the data generated from this initial
exploratory study; it looks very promising," said Dr. Richard Auchus, Professor of Internal Medicine,
Division of Metabolism, Endocrinology & Diabetes at
University of Michigan Health System.
"This treatment strategy, if validated in future clinical trials,
has the potential to revolutionize the treatment for CAH patients
and make an important difference in their lives."
Results from the Initial Pilot Clinical Study
The pilot clinical trial was a blinded, single-site,
pharmacokinetic/pharmacodynamic study assessing two single,
ascending doses of NBI-77860 against placebo in adult females with
refractory CAH. The eight study participants visited the
investigative site for three separate overnight visits consisting
of bedtime dosing with placebo or one of two active doses of
NBI-77860. Each of the visits was separated by a three-week washout
period. Key biomarker measurements included adrenocorticotropic
hormone (ACTH), 17-hydroxyprogesterone (17-OHP), androgen, and
cortisol levels collected in the morning after dosing. Data from
this initial single dose exploratory study demonstrated a robust
decrease in ACTH and 17-OHP.
A full description of the study results and related data will be
presented at an upcoming scientific meeting.
1401 Study Design
The 1401 study is a Phase I/II open-label, sequential cohort,
single ascending dose pharmacokinetic/pharmacodynamic study
assessing three doses of NBI-77860. The fifteen adolescent females
with classic CAH will be split into three cohorts and each will
receive one dose of NBI-77860 at bedtime. Biomarker measurements
include ACTH, 17-OHP, androgen, and cortisol levels collected the
morning after dosing.
About Classic Congenital Adrenal Hyperplasia (CAH)
Classic CAH is a genetic disorder that results in an enzyme
deficiency altering the production of adrenal steroids. Because of
this deficiency, the adrenal glands have little to no cortisol
biosynthesis resulting in a potentially life-threatening condition.
If left untreated, classic CAH can result in salt wasting,
dehydration and eventually death. Even with cortisol replacement,
persistent elevation of ACTH from the pituitary gland results in
excessive androgen levels leading to virilization of females
including precocious puberty, menstrual irregularity, short
stature, hirsutism, acne and fertility problems.
Corticosteroids are the current standard of care for classic CAH
which are used to both correct the endogenous cortisol deficiency
and reduce the excessive ACTH levels and androgen excess. However,
the dose and duration of steroid use required to suppress ACTH is
well above the normal physiological level of cortisol; resulting in
metabolic syndrome, bone loss, growth impairment, and Cushing's
syndrome as common and serious side effects.
Additional information on CAH can be obtained from the National
Institutes of Health http://www.nlm.nih.gov, and patient advocacy
groups such as CARES http://www.caresfoundation.org.
About NBI-77860
NBI‑77860 is a potent, selective non-peptide CRF receptor
antagonist as demonstrated in a range of in vitro/in vivo assays
and human clinical studies. Blockade of CRF receptors at the
pituitary has been shown to decrease the release of ACTH, which in
turn decreases the production of adrenal steroids including
androgens, and potentially the symptoms associated with classic
CAH. Lower ACTH levels would also reduce the amount of exogenous
corticosteroid necessary for classic CAH patients to thrive
avoiding the side-effects currently associated with excessive
steroid therapy.
About Neurocrine Biosciences
Neurocrine Biosciences, Inc. discovers and develops innovative
and life-changing pharmaceuticals, in diseases with high unmet
medical needs, through its novel R&D platform, focused on
neurological and endocrine based diseases and disorders. The
Company's two lead late-stage clinical programs are elagolix, a
gonadotropin-releasing hormone antagonist for women's health that
is partnered with AbbVie Inc., and a wholly owned vesicular
monoamine transporter 2 inhibitor for the treatment of movement
disorders. Neurocrine intends to maintain certain commercial
rights to its VMAT2 inhibitor for evolution into a fully-integrated
pharmaceutical company. Neurocrine Biosciences, Inc. news
releases are available through the Company's website via the
internet at http://www.neurocrine.com.
In addition to historical facts, this press release may
contain forward-looking statements that involve a number of risks
and uncertainties. Among the factors that could cause actual
results to differ materially from those indicated in the
forward-looking statements are risks and uncertainties associated
with Neurocrine's business and finances in general, as well as
risks and uncertainties associated with the Company's CRF program
and Company overall. Specifically, the risks and uncertainties the
Company faces with respect to the Company's CRF program include,
but are not limited to; risk that NBI-77860 will not proceed to
later stage clinical trials and risk that the Company's clinical
trials will fail to demonstrate that NBI-77860 is safe and
effective, risk that NBI-77860 may not replicate the results
observed in our prior clinical development, risk that NBI-77860 may
cause other unanticipated adverse effects, in the 1401 study
or subsequent clinical trials, the risk of cessation or delay of
the 1401 study or any ongoing or planned preclinical or
clinical development activities for a variety of reasons, including
additional information that may be requested or additional
obligations that may be imposed by the FDA as a condition to our
commencement and continuation of clinical trials with NBI-77860,
and risk that NBI-77860 will not receive Orphan Drug designation.
With respect to its pipeline overall, the Company faces risk that
it will be unable to raise additional funding required to complete
development of its product candidates; risk relating to the
Company's dependence on contract manufacturers for clinical drug
supply; risks associated with the Company's dependence on corporate
partners for development, commercial manufacturing and marketing
and sales activities for the Company's partnered programs;
uncertainties relating to patent protection and intellectual
property rights of third parties; risks and uncertainties relating
to competitive products and technological changes that may limit
demand for the Company's products; and overall risks associated
with the process of discovering, developing and commercializing
drug candidates that are safe and effective for use as human
therapeutics and the other risks described in the Company's reports
on Form 10-K for the year ended December 31,
2013 and on Form 10-Q for each of the quarters ended
March 31, 2014, June 30, 2014 and September 30, 2014. Neurocrine undertakes no
obligation to update the statements contained in this press release
after the date hereof.
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SOURCE Neurocrine Biosciences, Inc.