Neurocrine Biosciences to Present New Phase 3 CAHtalyst™ Data in Adult and Pediatric Patients with CAH and Study Data for Modified-Release Hydrocortisone in Primary Adrenal Insufficiency and CAH at ENDO 2024

Rhea-AI Summary

Neurocrine Biosciences announced the presentation of new Phase 3 CAHtalyst™ data at ENDO 2024, focusing on congenital adrenal hyperplasia (CAH) in both adults and children. The data includes results from randomized, double-blind, placebo-controlled trials evaluating crinecerfont's efficacy, safety, and tolerability. Additional presentations cover modified-release hydrocortisone (MRHC) studies for primary adrenal insufficiency and CAH, analyzing the impact of supraphysiologic glucocorticoid dosing and disease-related comorbidities. These findings have supported New Drug Application submissions to the FDA in April 2024.

The presentations will take place from June 1-4 in Boston, featuring key data and several posters highlighting the company's neuroendocrinology pipeline.

Positive

• Presentation of Phase 3 clinical study data for CAHtalyst™ at ENDO 2024.
• New Drug Application submissions to the FDA supported by Phase 3 data.
• Data showcases crinecerfont's efficacy, safety, and tolerability in treating CAH.
• Analysis of negative impacts of supraphysiologic glucocorticoid dosing.
• Study on disease-related comorbidities in CAH patients.

Negative

• No specific financial data provided to gauge immediate impact on revenue.
• Focus on clinical data might not immediately translate to stock performance.
• Potential risks if FDA does not approve crinecerfont based on presented data.
• Impact of negative findings on supraphysiologic glucocorticoid dosing not fully clarified.

Insights

Medical Research Analyst

The presentation of new Phase 3 clinical study data for Neurocrine Biosciences' crinecerfont in both adult and pediatric patients with classic congenital adrenal hyperplasia (CAH) is very significant. Crinecerfont acts as a Corticotropin-Releasing Factor Type 1 Receptor (CRF1) antagonist, which has the potential to be a groundbreaking treatment for CAH. Current treatments often rely on supraphysiologic glucocorticoid dosing, which can lead to severe side effects over time. If crinecerfont proves effective with fewer adverse effects, it could shift the treatment paradigm for CAH patients, improving their quality of life substantially. This data supports Neurocrine's New Drug Application (NDA) submissions to the FDA, which introduces a possible next phase of market approval.

Additionally, the data from the modified-release hydrocortisone (MRHC) studies in primary adrenal insufficiency and CAH addresses a critical need for more stable and effective treatments in these conditions. The presentation of these data at the ENDO 2024 conference could also increase visibility and generate further interest from the medical community, highlighting the company's commitment to innovative therapies in endocrinology.

Note: Terms like 'Phase 3 clinical study' refer to late-stage trials involving large patient groups to confirm effectiveness, monitor side effects and collect information that will allow the drug to be used safely. 'New Drug Application (NDA)' is a formal proposal for the FDA to approve a new pharmaceutical for sale and marketing in the U.S. These steps are important for bringing new treatments to market, but they also represent a substantial financial investment and regulatory scrutiny.

Financial Analyst

From a financial perspective, Neurocrine Biosciences providing new Phase 3 data and pursuing NDA submissions for crinecerfont signifies a potential near-term catalyst for the company's stock. Clinical trial progression into Phase 3 and successful outcomes are often inflection points for biotech companies because they can lead to FDA approval and subsequent commercialization. The NDA submissions based on the positive study results could translate into market approval and revenue generation, significantly impacting the company's financial health.

Moreover, the modified-release hydrocortisone (MRHC) studies, showing potentially improved treatment options, could also broaden Neurocrine's product portfolio and diversify revenue streams. Such diversification can help mitigate risks typically associated with biotech companies where multiple products in the pipeline offer a buffer against individual drug failures.

Investors should also monitor the company's cash burn rate and the financial implications of continued clinical trials and regulatory submissions. The potential for generating revenue from these new treatments must be weighed against the ongoing costs associated with bringing them to market.

• 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. 

View original content to download multimedia:https://www.prnewswire.com/news-releases/neurocrine-biosciences-to-present-new-phase-3-cahtalyst-data-in-adult-and-pediatric-patients-with-cah-and-study-data-for-modified-release-hydrocortisone-in-primary-adrenal-insufficiency-and-cah-at-endo-2024-302155663.html

SOURCE Neurocrine Biosciences, Inc.

FAQ

What new data did Neurocrine Biosciences present at ENDO 2024?

Neurocrine Biosciences presented Phase 3 clinical study data for CAHtalyst™ in treating congenital adrenal hyperplasia (CAH) in adults and children, along with studies on modified-release hydrocortisone (MRHC) for primary adrenal insufficiency and CAH.

How does crinecerfont benefit CAH patients according to the ENDO 2024 data?

Crinecerfont showed efficacy, safety, and tolerability in treating CAH in both adults and children, supporting New Drug Application submissions to the FDA.

What are the potential risks mentioned in Neurocrine Biosciences' latest data presentation?

Potential risks include the possibility of the FDA not approving crinecerfont and the impact of negative findings on supraphysiologic glucocorticoid dosing in CAH patients.

What is the significance of the CAHtalog™ registry data presented by Neurocrine Biosciences?

The CAHtalog™ registry data highlighted the negative impacts of supraphysiologic glucocorticoid dosing and provided insights into disease-related comorbidities in CAH patients.

When were the New Drug Application submissions to the FDA made by Neurocrine Biosciences?

The New Drug Application submissions to the FDA were made in April 2024, supported by data from the CAHtalyst Pediatric and CAHtalyst Adult Phase 3 clinical studies.