SAN DIEGO, Jan. 16, 2015 /PRNewswire/ -- Neurocrine
Biosciences, Inc. (NASDAQ: NBIX) announced today that NBI-77860, a
proprietary corticotropin releasing factor 1 (CRF) receptor
antagonist, has been granted orphan drug status by the United
States Food and Drug Administration (FDA) for the treatment of
congenital adrenal hyperplasia (CAH) a disease that affects
approximately 20,000-30,000 people in the
United States.
"We are very pleased that the FDA has granted NBI-77860 orphan
status to treat congenital adrenal hyperplasia, a devastating
disease that is a significant challenge for both clinicians and
patients," said Malcolm Lloyd-Smith,
Chief Regulatory Officer of Neurocrine Biosciences. "This status
represents a significant regulatory milestone for the CAH program
and underscores the importance of bringing a safe and effective CAH
therapy to market. We look forward to the results from our recently
initiated 1401 Study of adolescents with classic CAH, in 2015."
About Orphan Drug Designation
Orphan Drug Designation is granted by the FDA Orphan Drug
Designation program for medicines intended for the safe and
effective treatment, diagnosis or prevention of rare diseases or
disorders that affect fewer than 200,000 people in the United States. Orphan Drug Designation
provides sponsors with development and commercial incentives for
such designated compounds and medicines.
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 classic 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.
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
day after dosing.
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, and 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.
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.