SAN
DIEGO, Sept. 21, 2023 /PRNewswire/ -- Neurocrine
Biosciences, Inc. (Nasdaq: NBIX), today announced that it will
present a new post hoc analysis of Phase 2 data of the
investigational drug crinecerfont in adolescent patients with
classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase
deficiency (21-OHD). The analysis suggests that adolescents with
classic CAH who have more elevated baseline hormone levels may have
the potential for a greater response to treatment with crinecerfont
and may experience a reduction in androgen levels across a broad
range of glucocorticoid doses. These new data will be presented at
the 61st Annual European Society for Pediatric
Endocrinology (ESPE) Meeting in The
Hague, Netherlands from Sept. 21–23, 2023.
![(PRNewsfoto/Neurocrine Biosciences, Inc.) (PRNewsfoto/Neurocrine Biosciences, Inc.)](https://mma.prnewswire.com/media/678850/Neurocrine_Biosciences_Logo.jpg)
In previously reported Phase 2 study data, crinecerfont
treatment for 14 days led to clinically meaningful reductions of
17-hydroxyprogesterone (17-OHP), adrenocorticotropic hormone (ACTH)
and androstenedione (A4) in adolescents (ages 14–16) with CAH due
to 21-OHD. Post hoc analysis of these data released today assessed
whether baseline hormone concentration and glucocorticoid (GC)
doses correlated with response to treatment. A strong correlation
was found between baseline hormone concentration and change from
baseline to Day 14 for 17-OHP, ACTH and A4, with the greatest
reductions observed in participants with the highest baseline
hormone levels. These data suggest adolescents with higher baseline
hormone concentrations may have the potential for a greater
response to treatment with crinecerfont. However, there was no
correlation between baseline GC dose and treatment response, which
suggests that androgen reduction might occur across a broad range
of GC doses in this population.
In the Phase 2 study, crinecerfont was generally well tolerated
in adolescents, with no serious adverse events or discontinuations
due to adverse events. There were no safety concerns with respect
to routine laboratory tests, vital signs, electrocardiograms or
neuropsychiatric assessments.
The results of the post hoc analysis in adolescents were
consistent with results from similar post hoc analyses of the Phase
2 study of crinecerfont in adults with classic CAH, suggesting that
higher baseline hormone concentrations might predict greater
response, and androgen reduction might occur across a broad range
of GC doses. See the following abstract for more information: Oral
Presentation # 97 FC1.4; Response to Crinecerfont
Treatment in Adolescents with Classic Congenital Adrenal
Hyperplasia Is Correlated with Elevated Baseline Hormone
Concentrations but Not Glucocorticoid Dose.
"Treating CAH in adolescents is an especially difficult task
because of the need to make treatment adjustments that will
minimize the risks associated with both androgen excess and
exposure to supraphysiologic glucocorticoid doses. Balance can be
particularly difficult to achieve in this population, where
glucocorticoid dose adjustment can be made more challenging by the
criticality of age-related physiological changes that occur as
children enter puberty," said Eiry W. Roberts, M.D., Chief Medical
Officer at Neurocrine Biosciences. "These data support the
potential of crinecerfont to provide improved androgen control
across a broad range of CAH patients. We plan to announce top-line
data from our Phase 3 CAHtalyst™ Pediatric Study in early Q4
2023."
About Classic Congenital Adrenal Hyperplasia
(CAH)
Congenital adrenal hyperplasia (CAH) refers to a
group of genetic conditions that result in an enzyme deficiency
that alters the production of adrenal hormones, which are essential
for life. Approximately 95 percent of CAH cases are caused by a
mutation that leads to deficiency of the enzyme 21-hydroxylase
(21-OHD). In classic CAH, severe deficiency of this enzyme leads to
an inability of the adrenal glands to produce cortisol and, in
approximately 75 percent of cases, aldosterone. If left untreated,
classic CAH can result in salt wasting, dehydration and even
death.
There are currently no non-glucocorticoid treatments approved by
the U.S. Food and Drug Administration (FDA) for classic CAH.
Glucocorticoids (GCs), the current standard of care, are used not
only to correct the endogenous cortisol deficiency but typically
used at greater than physiologic (supraphysiologic) doses to try to
suppress the high levels of corticotropin-releasing factor (CRF)
and adrenocorticotropic hormone (ACTH) that result in androgen
excess. However, glucocorticoid treatment at supraphysiologic doses
has been associated with serious and significant complications of
steroid excess, including metabolic issues such as weight gain and
diabetes, cardiovascular disease, and osteoporosis. Additionally,
long-term treatment with supraphysiologic GC doses may have
psychological and cognitive impact such as changes in mood and
memory. Androgen excess has been associated with abnormal bone
growth and development in pediatric patients, female health
problems such as acne, excess hair growth and menstrual
irregularities, testicular rest tumors in males and fertility
issues in both sexes. To learn more about CAH, click here.
About Crinecerfont
Crinecerfont is an investigational,
oral, selective corticotropin-releasing factor type 1 receptor
(CRF1) antagonist being developed to reduce and control
excess adrenal androgens through a steroid-independent mechanism
for the treatment of CAH due to 21-hydroxylase deficiency (21-OHD).
Antagonism of CRF type 1 receptors in the pituitary has been shown
to decrease adrenocorticotropic hormone (ACTH) levels, which in
turn decreases the production of adrenal androgens and potentially
the symptoms associated with classic CAH. Our data demonstrates
that lowering androgen levels enables lower, more physiologic
dosing of glucocorticoids and thus could potentially reduce the
complications associated with exposure to greater than normal
glucocorticoid doses in patients with classic CAH.
Learn more about crinecerfont.
About CAHtalyst™ Studies
The randomized,
double-blind, placebo-controlled CAHtalyst™ Phase 3 global
registrational studies were designed to evaluate the safety,
efficacy and tolerability of crinecerfont in children and
adolescents (ages 2–17 years of age) as well as adults (18 years of
age and older) with classic congenital adrenal hyperplasia (CAH).
As part of the CAHtalyst clinical trial program, participants who
completed these studies were able to continue to receive
crinecerfont as part of an open-label extension. On September 12, 2023, Neurocrine Biosciences
announced positive top-line data from the Phase 3 CAHtalyst Adult
study. Neurocrine Biosciences plans to announce top-line data from
its Phase 3 CAHtalyst Pediatric study in early Q4 2023.
For more information about the CAHtalyst Adult and CAHtalyst
Pediatric Phase 3 studies, please visit
ClinicalTrials.gov (Adult) and
ClinicalTrials.gov (Pediatric).
About Neurocrine Biosciences
Neurocrine Biosciences is a leading neuroscience-focused,
biopharmaceutical company with a simple purpose: to relieve
suffering for people with great needs but few options. We are
dedicated to discovering and developing life-changing treatments
for patients with under-addressed neurological, neuroendocrine and
neuropsychiatric disorders. The company's diverse portfolio
includes FDA-approved treatments for tardive dyskinesia, chorea
associated with Huntington's disease, Parkinson's disease,
endometriosis* and uterine fibroids*, as well as a robust pipeline
including multiple compounds in mid- to late-phase clinical
development across our core therapeutic areas. For three decades,
we have applied our unique insight into neuroscience and the
interconnections between brain and body systems to treat complex
conditions. We relentlessly pursue medicines to ease the burden of
debilitating diseases and disorders, because you deserve brave
science. For more information, visit neurocrine.com, and
follow the company on LinkedIn, Twitter and Facebook. (*in
collaboration with AbbVie)
NEUROCRINE, the Neurocrine logo, and CAHtalyst are registered
trademarks of Neurocrine Biosciences, Inc.
Forward-Looking Statement
In addition to historical
facts, this press release contains forward-looking statements that
involve a number of risks and uncertainties. These statements
include statements regarding the potential benefits of crinecerfont
to patients and future clinical development plans. Among the
factors that could cause actual results to differ materially from
those indicated in the forward-looking statements include the risk
that crinecerfont will not be found to be safe and/or effective or
may not prove to be beneficial to patients; that development
activities for crinecerfont may not be completed on time or at all;
risks that clinical development activities may be delayed for
regulatory or other reasons, may not be successful or replicate
previous and/or interim clinical trial results, or may not be
predictive of real-world results or of results in subsequent
clinical trials; risks that regulatory submissions for crinecerfont
may not occur or be submitted in a timely manner; risks that
crinecerfont may not obtain regulatory approvals; or that the U.S.
Food and Drug Administration or regulatory authorities outside the
U.S. may make adverse decisions regarding crinecerfont; and other
risks described in the Company's periodic reports filed with the
Securities and Exchange Commission, including without limitation
the Company's quarterly report on Form 10-Q for the quarter ended
June 30, 2023. Neurocrine disclaims
any obligation to update the statements contained in this press
release after the date hereof.
View original content to download
multimedia:https://www.prnewswire.com/news-releases/neurocrine-biosciences-presents-post-hoc-data-analysis-in-congenital-adrenal-hyperplasia-at-espe-2023-301932611.html
SOURCE Neurocrine Biosciences, Inc.