- New CAHtalyst™ Pediatric and CAHtalyst™ Adult Phase 3 Clinical
Study Data in Congenital Adrenal Hyperplasia
- CAHtalog™ Registry Data Highlighting Impact of Supraphysiologic
Glucocorticoid Dosing
- Phase 2 (CHAMPAIN) Study Data for MRHC vs. Plenadren in Primary
Adrenal Insufficiency
- Long-Term Study Data for MRHC in Adults with CAH
SAN
DIEGO, May 28, 2024 /PRNewswire/
-- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced
that it will present key information from its neuroendocrinology
pipeline, including new Phase 3 clinical study data from its
CAHtalyst™ registrational studies of crinecerfont in pediatric and
adult patients with classic congenital adrenal hyperplasia
(CAH) due to 21-hydroxylase deficiency and data from its
modified-release hydrocortisone (MRHC) studies in primary adrenal
insufficiency and CAH. These data, along with several additional
posters, will be presented at the Endocrine Society Annual Meeting
(ENDO 2024) in Boston,
June 1-4.
Neurocrine Biosciences posters that will be presented at ENDO
2024 include:
- June 1, 3:00-3:15 p.m.: Oral Presentation, Richard Auchus, M.D. (OR20-05): CAHtalyst™:
Results from the Randomized, Double-Blind, Placebo-Controlled
Period of a Phase 3 Trial of Crinecerfont, a
Corticotropin-Releasing Factor Type 1 Receptor (CRF1)
Antagonist, in Adults with Classic Congenital Adrenal
Hyperplasia
- June 2, 12:00-1:30 p.m. (Poster #SUN-441):
CAHtalyst™: Results from the Randomized, Double-Blind,
Placebo-Controlled Period of a Phase 3 Trial of Crinecerfont, a
Corticotropin-Releasing Factor Type 1 Receptor (CRF1)
Antagonist, in Children and Adolescents with Classic Congenital
Adrenal Hyperplasia
- June 2, 12:00-1:30 p.m. (Poster #SUN-685): Negative
Impacts of Supraphysiologic Glucocorticoid Dosing in Patients with
Classic Congenital Adrenal Hyperplasia: An Analysis of Data from
the CAHtalog™ Registry
- June 1, 12:15-1:45 p.m. (Poster #SAT-437): Disease-
and Glucocorticoid-related Comorbidities in Classic Congenital
Adrenal Hyperplasia: A Claims-Based Retrospective Cohort
Analysis
- June 2, 12:00-1:30 p.m. (Poster #SUN-417): Natural
History of Classic Congenital Adrenal Hyperplasia: Results from
Pediatric and Adult Patients in the CAHtalog Registry
- June 3, 12:00-1:30 p.m. (Poster #MON-676), Rapid-Fire
Session, 1:45-1:50 p.m. (RF36-01): 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
- June 1, 12:15-1:45 p.m. (Poster #SAT-427): Incidence
of Adrenal Crisis in Congenital Adrenal Hyperplasia (CAH) Patients
During a Prospective Monitored Long-Term Study of Modified-Release
Hydrocortisone (MRHC) Capsules, (Efmody)
- June 1, 12:15-1:45 p.m. (Poster #SAT-412): Morning
Cortisol Levels in Patients with Established Primary Adrenal
Insufficiency
In 2023, Neurocrine Biosciences announced top-line data from the
CAHtalyst Pediatric and CAHtalyst Adult Phase 3 clinical studies
evaluating the efficacy, safety and tolerability of crinecerfont in
adult and pediatric patients with CAH due to 21-hydroxylase
deficiency. Data from both studies supported two New Drug
Application submissions to the U.S. Food and Drug Administration in
April 2024.
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 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 CHAMPAIN Study in Adrenal Insufficiency
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 and YOU DESERVE BRAVE
SCIENCE are registered trademarks of Neurocrine Biosciences, Inc.
CHRONOCORT and EFMODY are registered trademarks of Diurnal Limited.
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 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 or 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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