- CAHtalyst™ Pediatric Study Baseline Characteristics Data
Highlight Need for Novel Treatments in Children and Adolescents
with Congenital Adrenal Hyperplasia (CAH)
- CAHtalog™ Registry Data of Glucocorticoid Treatment Patterns
in Pediatric and Adult Patients Illustrates the Challenges of
Long-Term CAH Management
SAN
DIEGO, May 3, 2024 /PRNewswire/ -- Neurocrine
Biosciences, Inc. (Nasdaq: NBIX), today presented the CAHtalyst™
Pediatric Phase 3 clinical study baseline characteristics data for
children and adolescents with congenital adrenal hyperplasia (CAH)
due to 21-hydroxylase deficiency enrolled in the study, along with
CAHtalog™ Registry data assessing glucocorticoid treatment patterns
in pediatric and adult patients with CAH.
These data demonstrate the limitations of current CAH treatment
approaches in pediatric patients, comorbidities associated with the
condition and/or current treatment, including obesity, advanced
bone age, and early puberty, and the difficulty in managing the
disease effectively as CAH patients age into adults. These new data
were presented at the Pediatric Endocrine Society 2024 Annual
Meeting in Chicago.
CAHtalyst Phase 3 Pediatric Study Baseline
Characteristics
Baseline characteristics of the subjects who enrolled in the
CAHtalyst Pediatric Phase 3 study were presented (Poster# 56). The
study enrolled 103 subjects 4 to 17 years of age with a medically
confirmed diagnosis of CAH due to 21-hydroxylase deficiency, with
52% male, mean age 12 years old, and majority in Tanner stages 3-5.
There was evidence indicating inadequate adrenal androgen control
in many of these patients despite supraphysiologic glucocorticoid
dosing. At baseline, more than a third of the participants reported
comorbidities of advanced bone age, early puberty, and obesity.
Hirsutism (excessive hair growth, 12%) and irregular menses (12%)
were reported in females, and testicular adrenal rest tumors were
identified in more than a third of males.
"At baseline, many participants in the CAHtalyst Pediatric study
showed clinical evidence of elevated glucocorticoid doses and
adrenal androgen excess. Many exhibited obesity, advanced bone age,
and early puberty, all of which can negatively impact development
in childhood and adolescence, and lead to further harm in
adulthood," said Eiry W. Roberts M.D., Chief Medical Officer
at Neurocrine Biosciences. "With adrenal androgen levels
elevated despite supraphysiologic glucocorticoid doses, it's clear
that there is a significant need for a new approach to treat this
condition."
In 2023, Neurocrine Biosciences announced positive top-line data
from the CAHtalyst Pediatric and CAHtalyst Adult Phase 3 clinical
studies evaluating the efficacy, safety, and tolerability of
crinecerfont in pediatric and adult patients with CAH due to
21-hydroxylase deficiency. The data from both studies supported two
New Drug Applications that were submitted to the U.S. Food and Drug
Administration in April 2024.
CAHtalog Patient Registry: Glucocorticoid Treatment Patterns
Neurocrine Biosciences also presented glucocorticoid treatment
patterns from a recent cohort of pediatric and adult patients
participating in the CAHtalog Registry (Poster# 51). Data from 42
pediatric and 32 adult patients with median duration of observation
between 10 to 13 years of age were analyzed.
Glucocorticoid treatment patterns seen in the CAHtalog Registry
data indicate patients are being treated with GC doses that are at
or above the upper end of the ranges recommended in the Endocrine
Society Guidelines.
"The CAHtalog Registry data showed that a vast majority of
patients had higher GCs and/or a loss of androstenedione control at
some point, or multiple points, in their journey living with CAH,"
said Oksana Lekarev, D.O., Associate Professor of Clinical
Pediatrics, Co-Medical Director of the Weill Cornell Medicine
Comprehensive Care Center for Congenital Adrenal Hyperplasia and an
Associate Attending Pediatrician at New York-Presbyterian/Weill
Cornell Medical Center and member of the CAHtalog Scientific
Advisory Board. "These analyses underscore that disease
control can vary widely and highlights the difficulty in achieving
optimal adrenal androgen control in many patients despite
supraphysiologic GC dosing."
Neurocrine Biosciences abstracts presented at the meeting
included:
- Baseline Characteristics of Children and Adolescents with
Classic Congenital Adrenal Hyperplasia Enrolled in CAHtalyst
Pediatric, a Phase 3 Study of Crinecerfont, a
Corticotropin-Releasing Factor Type 1 Receptor Antagonist,
May 3; 12:15–1:45pm (Poster# 56)
- Glucocorticoid Treatment Patterns in Pediatric and Adult
Patients with Classic Congenital Adrenal Hyperplasia: Results from
the CAHtalog Registry, May 3;
12:15–1:45pm (Poster# 51)
- Natural History of Classic Congenital Adrenal Hyperplasia:
Results from Pediatric and Adult Patients in the CAHtalog Registry,
May 3; 12:15–1:45pm (Poster# 60)
About Congenital Adrenal Hyperplasia
Congenital
adrenal hyperplasia (CAH) is a rare genetic condition that results
in an enzyme deficiency that alters the production of adrenal
hormones which are essential for life. Approximately 95% of CAH
cases are caused by a mutation that leads to deficiency of the
enzyme 21-hydroxylase. Severe deficiency of this enzyme can lead to
an inability of the adrenal glands to produce cortisol and, in
approximately 75% of cases, aldosterone. If left untreated, CAH can
result in salt wasting, dehydration and even death.
Glucocorticoids (GCs) are currently used not only to correct the
endogenous cortisol deficiency, but doses used are higher than
cortisol replacement needed (supraphysiologic) to lower the levels
of adrenocorticotropic hormone (ACTH) and adrenal androgens.
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. Adrenal 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 glucocorticoid-independent
mechanism for the treatment of congenital adrenal hyperplasia due
to 21-hydroxylase deficiency. Antagonism of CRF1
receptors in the pituitary has been shown to decrease
adrenocorticotropic hormone levels, which in turn decreases the
production of adrenal androgens and potentially the symptoms
associated with CAH. Our data demonstrate that lowering adrenal
androgen levels enables lower, more physiologic dosing of
glucocorticoids and thus could potentially reduce the complications
associated with exposure to greater than replacement glucocorticoid
doses in patients with CAH.
To learn more about crinecerfont, click here.
About the CAHtalyst™ Phase 3
Studies
The CAHtalyst™ Pediatric and Adult Phase 3
global studies are the largest registrational studies conducted to
date to evaluate the safety, efficacy, and tolerability of
crinecerfont in children and adolescents, and adults respectively,
with congenital adrenal hyperplasia due to 21-hydroxylase
deficiency. The double-blind portions of the CAHtalyst Phase 3
studies have completed enrollment and are closed, while open-label
treatment in both studies is ongoing.
For more information about the CAHtalyst Pediatric Phase 3
study, please visit ClinicalTrialsPediatric.gov.
For more information about the CAHtalyst Phase 3 study in adults
(ages 18 years of age and older), please visit
ClinicalTrialsAdult.gov.
About the CAHtalog™
Registry
In 2021, the CARES Foundation, Neurocrine
Biosciences and PicnicHealth partnered to establish the CAHtalog
(Congenital Adrenal Hyperplasia:
Patient and Clinical Outcomes in Real-World
Practice Settings) Registry. The CAHtalog Registry is a U.S.
based CAH patient registry, or collection of clinical patient data,
for patients with CAH due to 21-hydroxylase deficiency. The
database was developed to support patient-centered clinical
research to enhance the scientific community's foundational
knowledge about CAH, and ultimately help patients who live with the
condition. For more information about the CAHtalog Registry, please
visit CAHtalog.com.
About Neurocrine Biosciences, Inc.
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, 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, X
(formerly Twitter), and Facebook.
(*in collaboration with AbbVie)
The NEUROCRINE BIOSCIENCES Logo Lockup and YOU DESERVE BRAVE
SCIENCE are registered trademarks of Neurocrine Biosciences,
Inc. The Neurocrine logo, CAHtalyst and CAHtalog are trademarks of
Neurocrine Biosciences, Inc.
Forward-Looking Statements
In addition to historical
facts, this press release contains forward-looking statements that
involve a number of risks and uncertainties. These statements
include, but are not limited to, statements regarding the potential
benefits to be derived from crinecerfont, as well as the
therapeutic potential and clinical benefits or safety profile of
crinecerfont. Among the factors that could cause actual results to
differ materially from those indicated in the forward-looking
statements include: the crinecerfont NDAs may not be accepted for
filing by the FDA or may not obtain regulatory approval or such
approval may be delayed; additional regulatory submissions may not
occur or be submitted in a timely manner; the FDA may make adverse
decisions regarding crinecerfont; crinecerfont may not be
found to be safe and/or effective or may not prove to be beneficial
to patients; development activities for crinecerfont may not be
completed on time or at all; 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; competitive products and technological changes
that may limit demand for our products; uncertainties relating to
patent protection and intellectual property rights of third
parties; our dependence on third parties for development and
manufacturing activities related to crinecerfont, and our ability
to manage these third parties; our future financial and operating
performance; risks and uncertainties associated with the
commercialization of our products; 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 March 31, 2024. Neurocrine Biosciences disclaims
any obligation to update the statements contained in this press
release after the date hereof.
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