- CAHtalyst™ Pediatric and CAHtalyst™ Adult Baseline
Characteristics Data in Congenital Adrenal Hyperplasia
- Phase 2 (CHAMPAIN) Clinical Study Data for Modified-Release
Hydrocortisone (Chronocort®/Efmody®) in
Adrenal Insufficiency
- Phase 3 Extension Study Data for Modified-Release
Hydrocortisone in Congenital Adrenal Hyperplasia
SAN
DIEGO, May 7, 2024 /PRNewswire/
-- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced
that it will present key information from its neuroendocrinology
pipeline, including baseline characteristics data from its
CAHtalyst™ Program of crinecerfont in congenital adrenal
hyperplasia (CAH), as well as data from its modified-release
hydrocortisone studies in primary adrenal insufficiency and CAH, at
the European Congress of Endocrinology 2024 meeting in Sweden, May 11–14.
Neurocrine Biosciences will be presenting several abstracts and
posters at ECE 2024, including:
- 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 13; 5:30–6:30pm (Poster#
P225)
- Baseline Characteristics of Adults with Classic Congenital
Adrenal Hyperplasia Enrolled in CAHtalyst Adult, a Phase 3 Study of
Crinecerfont, a Corticotropin-Releasing Factor Type 1 Receptor
Antagonist
-
- May 14; 1:40–3:10pm (Poster# P423)
- CHAMPAIN study: Initial Results from a Phase II Study of
Efficacy, Safety and Tolerability of Modified-Release
Hydrocortisones: Chronocort® (Efmody®) versus
Plenadren, in Primary Adrenal Insufficiency,
-
- May 12; 2:00–2:40pm (Rapid
Communication #RC3.4)
- Biochemical Control with Dose Reduction in Chronic
Glucocorticoid Therapy over 4 Years: A Phase III Extension Study of
Chronocort (Efmody®) in the Treatment of Congenital
Adrenal Hyperplasia (CAH)
-
- May 12; 2:00–2:40pm (Rapid
Communication #RC3.1)
- Incidence of Adrenal Crisis in Congenital Adrena Hyperplasia
(CAH) Patients During a Prospective Monitored Long-Term study of
Modified-Release Hydrocortisone (MRHC) Capsules, (Efmody)
-
- May 12; 4:20–6:00pm (Poster
#P215)
- Morning Cortisol Levels in Patients with Established Primary
Adrenal Insufficiency
-
- May 13; 5:30– 6:30pm (Poster #P13)
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 (21-OHD). Severe deficiency of this enzyme
leads 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 and the CAHtalyst
Studies
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 normal glucocorticoid
doses in patients with CAH.
The CAHtalyst™ Pediatric and Adult Phase 3 global registrational
studies are designed 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 primary portions of the CAHtalyst
Phase 3 studies have completed and enrollment is closed, while the
open-label treatment portions of both studies are ongoing.
To learn more about crinecerfont and the CAHtalyst studies,
click here.
About Primary Adrenal Insufficiency
Primary adrenal
insufficiency is a chronic endocrine condition that occurs when the
body does not make enough of certain adrenal hormones, including
cortisol and often aldosterone. Glucocorticoids such as
hydrocortisone are used to replace the missing cortisol, but
typical dosing regimens do not match the natural diurnal rhythm of
the body's cortisol production.
About Modified-Release Hydrocortisone
Diurnal Ltd.
developed modified-release hydrocortisone, a preparation of
hydrocortisone that has been specifically designed to replicate the
natural circadian rhythm of cortisol, when given in a twice-a-day
"toothbrush" regimen, (administered last thing at night before
sleep and first thing in the morning on waking). In 2021,
modified-release hydrocortisone (EFMODY®) received
marketing authorization for the treatment of congenital adrenal
hyperplasia from the Medicines and Healthcare products Regulatory
Agency (MHRA) in Great Britain
(England, Wales, and Scotland) and from the European Commission in
the European Union. Neurocrine Biosciences acquired Diurnal Group
plc. in November 2022. A new drug
application for the modified-release hydrocortisone formulation has
not been submitted to the U.S. Food and Drug Administration.
About MRHC Phase 2 Study in Adrenal Insufficiency
(CHAMPAIN)
The CHAMPAIN Phase 2 clinical study compared the
efficacy, safety and tolerability of twice daily DNL0200
(Chronocort), a modified-release hydrocortisone, with once daily
Plenadren, a combination of immediate- and delayed-release
hydrocortisone (authorized for use in the European Union), over a
treatment period of up to 2 months in participants ≥18 years of age
and diagnosed with primary adrenal insufficiency.
About the Phase 3 Extension Study for MRHC in CAH
(DIUR-006)
The DIUR-006 Phase 3 open-label extension study
assessed the long-term efficacy, safety and tolerability of
twice-daily DNL0200 (Chronocort®), a modified-release
hydrocortisone in adults with CAH. The study was performed to build
on the results of feeder studies DIUR-003 (Phase 2 in adults with
CAH) and DIUR-005 (EU Phase 3 Registrational Open-Label Study of
Chronocort compared to standard of care in adults with CAH) and
evaluate the long-term safety of Chronocort, and also its long-term
efficacy in improving control of serum androgen levels (using
17-OHP and A4 as biomarkers).
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, YOU DESERVE BRAVE
SCIENCE, CHRONOCORT and EFMODY are registered trademarks of
Neurocrine Biosciences, Inc. CAHtalyst is a trademark 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 certain of our products. Among the
factors that could cause actual results to differ materially from
those indicated in the forward-looking statements include: risks
that regulatory submissions for our products and/or product
candidates may not occur be submitted in a timely manner, or
accepted for filing; our products and/or product candidates 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 our products and/or product candidates;
our products and/or product candidates will not be found to be safe
and/or effective or may not prove to be beneficial to patients;
that development activities for our products and/or product
candidates may not be completed on time or at all; 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; 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 our products and our product candidates, 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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