Neurocrine Presents New Two-Year Data on CAH Treatment
Written by Emily J. Thompson, Senior Investment Analyst
Updated: May 01 2026
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Should l Buy NBIX?
Source: PRnewswire
- Durable Efficacy: The CAHtalyst® Pediatric study demonstrated that children and adolescents treated with CRENESSITY® achieved significant androgen control over two years, with mean reductions of 157 pg/mL in ACTH and 1924 ng/dL in 17-OHP, indicating the drug's long-lasting effectiveness in hormone regulation.
- Reduced GC Dosing: Patients experienced substantial decreases in daily glucocorticoid (GC) doses, with reductions of 2.9 mg/m² and 3.2 mg/m² at 12 and 24 months respectively, thereby mitigating the risks associated with long-term GC use.
- Improved Clinical Outcomes: Participants showed significant improvements in clinical outcomes, with average BMI SDS decreasing by 0.30 and 61% of participants no longer being insulin resistant, highlighting CRENESSITY's positive impact on patient health.
- Positive Participant Feedback: Among 86 participants, over 80% retained participation over two years with no new safety signals observed, indicating good tolerability of CRENESSITY and supporting its potential in CAH treatment applications.
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Analyst Views on NBIX
Wall Street analysts forecast NBIX stock price to rise
20 Analyst Rating
17 Buy
3 Hold
0 Sell
Strong Buy
Current: 149.590
Low
143.00
Averages
179.68
High
203.00
Current: 149.590
Low
143.00
Averages
179.68
High
203.00
About NBIX
Neurocrine Biosciences, Inc. is a neuroscience-focused, biopharmaceutical company. The Company is engaged in discovering and developing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. Its diverse portfolio includes the United States Food and Drug Administration-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 its core therapeutic areas. Its commercial products include INGREZZA, ALKINDI, EFMODY, Orilissa, and Oriahnn. INGREZZA is marketed as DYSVAL (valbenazine) in Japan and REMLEAS (valbenazine) in other select Asian markets, where Mitsubishi Tanabe Pharma Corporation retains commercialization rights. ALKINDI is marketed as ALKINDI SPRINKLE (hydrocortisone) in the United States, where Eton Pharmaceuticals, Inc. retains commercialization rights.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
FDA Grants Orphan Drug Designation to Neurocrine's NBIB-223
- Orphan Drug Status: The FDA has granted orphan drug designation to Neurocrine Biosciences' NBIB-223 (frataxin), which allows for up to seven years of market exclusivity post-approval, significantly enhancing its commercial potential.
- Tax Incentives: With orphan drug status, NBIB-223 will benefit from tax credits for clinical trial expenses, reducing R&D costs and thereby strengthening Neurocrine's competitive position in the Friedreich ataxia treatment landscape.
- Gene Therapy Innovation: NBIB-223 is a gene therapy that employs Voyager Therapeutics' modified AAV capsid to deliver the frataxin gene throughout the body, showcasing Neurocrine's commitment to cutting-edge medical technology and innovation.
- Market Competition: The only approved treatment for Friedreich ataxia is Biogen's Skyclarys (omaveloxolone), and the introduction of NBIB-223 will provide patients with more options while potentially challenging Biogen's market share.
See More
First Expert Recommendations for Glucocorticoid Dose Reduction Published
- Expert Recommendations Released: Neurocrine Biosciences has published the first expert recommendations for glucocorticoid dose reduction in patients with classic congenital adrenal hyperplasia, marking a significant advancement in the field.
- Expansion of CRENESSITY® Use: As the real-world application of CRENESSITY® (crinecerfont) expands among pediatric and adult patients, these recommendations address a critical gap in clinical practice, meeting urgent patient needs.
- Peer-Reviewed Recognition: The guidelines have been published in The Journal of Clinical Endocrinology & Metabolism, indicating rigorous peer review and enhancing their authority and credibility within the medical community.
- Far-Reaching Clinical Impact: By providing systematic dose reduction recommendations, the guidelines are expected to improve treatment outcomes, reduce the risk of side effects, and ultimately enhance patient quality of life and treatment satisfaction.
See More
New Algorithms Aid in Reducing Hormone Doses for CAH Patients
- Clinical Recommendations Released: Neurocrine Biosciences has published the first expert recommendations for glucocorticoid dose reduction in patients with classic congenital adrenal hyperplasia (CAH), aimed at guiding healthcare providers in effectively lowering supraphysiologic glucocorticoid doses while using CRENESSITY, addressing a critical clinical need.
- Pediatric and Adult Protocols: The recommendations are divided into two parts for pediatric patients aged 4 to 17 and adults aged 18 and older, providing systematic algorithms for adjusting glucocorticoid doses that help clinicians reduce long-term risks associated with supraphysiologic doses while maintaining necessary cortisol replacement.
- Clear Treatment Goals: The recommendations for pediatric patients emphasize supporting normal growth and bone age maturation while reducing long-term complications from excess glucocorticoids, whereas the adult recommendations focus on minimizing metabolic, cardiovascular, and skeletal complications while ensuring androgen control.
- Advantages of CRENESSITY: By reducing adrenocorticotropic hormone (ACTH) and androgen production, CRENESSITY enables patients to transition toward more physiologic glucocorticoid dosing, thereby improving CAH management and lowering the risks associated with long-term exposure to supraphysiologic doses.
See More
Neurocrine Biosciences Reports Record Q1 2026 Earnings Driven by INGREZZA
- Sales Milestone: Neurocrine Biosciences achieved a historic quarterly net product sales exceeding $800 million in Q1 2026, representing a 44% year-over-year growth, primarily driven by strong performance from INGREZZA, marking a significant breakthrough in the market and expected to propel future growth.
- Financial Performance: Total revenue surpassed $800 million, exceeding analysts' expectations of $767.88 million, with net income around $200 million, showcasing robust growth driven by demand and further solidifying the company's position in the biopharmaceutical industry.
- Product Outlook: Management reaffirmed its 2026 sales guidance for INGREZZA at $2.7 billion to $2.8 billion, while noting that CRENESSITY is now annualizing over $600 million, indicating the potential and future growth opportunities for the company in the new drug market.
- Acquisition Progress: CEO Gano emphasized that the acquisition of Soleno Therapeutics is on track to close in the second quarter, and while detailed financial guidance cannot be provided during this period, this strategic move is expected to bring new growth momentum to the company.
See More
Neurocrine Biosciences Q1 Earnings Exceed Expectations
- Strong Earnings Performance: Neurocrine Biosciences reported a Q1 non-GAAP EPS of $1.94, beating expectations by $0.24, which reflects a significant improvement in profitability and boosts investor confidence.
- Robust Revenue Growth: The company achieved revenues of $814.5 million, a 42.2% year-over-year increase, exceeding expectations by $46.62 million, indicating sustained demand for its products and enhancing overall performance.
- Financial Guidance Reaffirmed: Neurocrine reaffirmed its 2026 financial guidance, projecting net product sales for INGREZZA between $2.7 billion and $2.8 billion, demonstrating confidence in future growth and providing clear performance expectations for investors.
- R&D and SG&A Expense Planning: GAAP R&D expenses are expected to range from $1.2 billion to $1.25 billion, while non-GAAP R&D expenses are projected between $1.11 billion and $1.16 billion, indicating ongoing investment in innovation and marketing aimed at driving long-term growth.
See More
Investigation Alerts for Multiple Company Mergers
- XOMA Acquisition Deal: XOMA Royalty Corporation is set to be acquired by Ligand Pharmaceuticals for $39.00 per share, totaling approximately $739 million; however, this price is below its 52-week high of $42.38, raising concerns among shareholders about the fairness of the transaction.
- Organon Merger Case: Organon & Co. will be acquired by Sun Pharmaceutical for $14.00 per share, with an enterprise valuation of $11.75 billion, and the investigation focuses on whether the Organon Board failed to uphold fiduciary duties to ensure a fair process for shareholders.
- RE/MAX Merger Investigation: RE/MAX Holdings, Inc. is being acquired by The Real Brokerage Inc., allowing shareholders to choose between 5.152 shares of the new company or $13.80 in cash, with investigations looking into whether the Board adequately handled the transaction to protect shareholder interests.
- Soleno Acquisition Case: Soleno Therapeutics, Inc. will be acquired by Neurocrine Biosciences for $53.00 per share, representing a total equity value of approximately $2.9 billion, while the investigation questions whether the Board ensured the deal was fair to shareholders, given that the price is below its 52-week high of $90.32.
See More
FDA Grants Orphan Drug Designation to Neurocrine's NBIB-223
- Orphan Drug Status: The FDA has granted orphan drug designation to Neurocrine Biosciences' NBIB-223 (frataxin), which allows for up to seven years of market exclusivity post-approval, significantly enhancing its commercial potential.
- Tax Incentives: With orphan drug status, NBIB-223 will benefit from tax credits for clinical trial expenses, reducing R&D costs and thereby strengthening Neurocrine's competitive position in the Friedreich ataxia treatment landscape.
- Gene Therapy Innovation: NBIB-223 is a gene therapy that employs Voyager Therapeutics' modified AAV capsid to deliver the frataxin gene throughout the body, showcasing Neurocrine's commitment to cutting-edge medical technology and innovation.
- Market Competition: The only approved treatment for Friedreich ataxia is Biogen's Skyclarys (omaveloxolone), and the introduction of NBIB-223 will provide patients with more options while potentially challenging Biogen's market share.
See More
First Expert Recommendations for Glucocorticoid Dose Reduction Published
- Expert Recommendations Released: Neurocrine Biosciences has published the first expert recommendations for glucocorticoid dose reduction in patients with classic congenital adrenal hyperplasia, marking a significant advancement in the field.
- Expansion of CRENESSITY® Use: As the real-world application of CRENESSITY® (crinecerfont) expands among pediatric and adult patients, these recommendations address a critical gap in clinical practice, meeting urgent patient needs.
- Peer-Reviewed Recognition: The guidelines have been published in The Journal of Clinical Endocrinology & Metabolism, indicating rigorous peer review and enhancing their authority and credibility within the medical community.
- Far-Reaching Clinical Impact: By providing systematic dose reduction recommendations, the guidelines are expected to improve treatment outcomes, reduce the risk of side effects, and ultimately enhance patient quality of life and treatment satisfaction.
See More
New Algorithms Aid in Reducing Hormone Doses for CAH Patients
- Clinical Recommendations Released: Neurocrine Biosciences has published the first expert recommendations for glucocorticoid dose reduction in patients with classic congenital adrenal hyperplasia (CAH), aimed at guiding healthcare providers in effectively lowering supraphysiologic glucocorticoid doses while using CRENESSITY, addressing a critical clinical need.
- Pediatric and Adult Protocols: The recommendations are divided into two parts for pediatric patients aged 4 to 17 and adults aged 18 and older, providing systematic algorithms for adjusting glucocorticoid doses that help clinicians reduce long-term risks associated with supraphysiologic doses while maintaining necessary cortisol replacement.
- Clear Treatment Goals: The recommendations for pediatric patients emphasize supporting normal growth and bone age maturation while reducing long-term complications from excess glucocorticoids, whereas the adult recommendations focus on minimizing metabolic, cardiovascular, and skeletal complications while ensuring androgen control.
- Advantages of CRENESSITY: By reducing adrenocorticotropic hormone (ACTH) and androgen production, CRENESSITY enables patients to transition toward more physiologic glucocorticoid dosing, thereby improving CAH management and lowering the risks associated with long-term exposure to supraphysiologic doses.
See More
Neurocrine Biosciences Reports Record Q1 2026 Earnings Driven by INGREZZA
- Sales Milestone: Neurocrine Biosciences achieved a historic quarterly net product sales exceeding $800 million in Q1 2026, representing a 44% year-over-year growth, primarily driven by strong performance from INGREZZA, marking a significant breakthrough in the market and expected to propel future growth.
- Financial Performance: Total revenue surpassed $800 million, exceeding analysts' expectations of $767.88 million, with net income around $200 million, showcasing robust growth driven by demand and further solidifying the company's position in the biopharmaceutical industry.
- Product Outlook: Management reaffirmed its 2026 sales guidance for INGREZZA at $2.7 billion to $2.8 billion, while noting that CRENESSITY is now annualizing over $600 million, indicating the potential and future growth opportunities for the company in the new drug market.
- Acquisition Progress: CEO Gano emphasized that the acquisition of Soleno Therapeutics is on track to close in the second quarter, and while detailed financial guidance cannot be provided during this period, this strategic move is expected to bring new growth momentum to the company.
See More
Neurocrine Biosciences Q1 Earnings Exceed Expectations
- Strong Earnings Performance: Neurocrine Biosciences reported a Q1 non-GAAP EPS of $1.94, beating expectations by $0.24, which reflects a significant improvement in profitability and boosts investor confidence.
- Robust Revenue Growth: The company achieved revenues of $814.5 million, a 42.2% year-over-year increase, exceeding expectations by $46.62 million, indicating sustained demand for its products and enhancing overall performance.
- Financial Guidance Reaffirmed: Neurocrine reaffirmed its 2026 financial guidance, projecting net product sales for INGREZZA between $2.7 billion and $2.8 billion, demonstrating confidence in future growth and providing clear performance expectations for investors.
- R&D and SG&A Expense Planning: GAAP R&D expenses are expected to range from $1.2 billion to $1.25 billion, while non-GAAP R&D expenses are projected between $1.11 billion and $1.16 billion, indicating ongoing investment in innovation and marketing aimed at driving long-term growth.
See More
Investigation Alerts for Multiple Company Mergers
- XOMA Acquisition Deal: XOMA Royalty Corporation is set to be acquired by Ligand Pharmaceuticals for $39.00 per share, totaling approximately $739 million; however, this price is below its 52-week high of $42.38, raising concerns among shareholders about the fairness of the transaction.
- Organon Merger Case: Organon & Co. will be acquired by Sun Pharmaceutical for $14.00 per share, with an enterprise valuation of $11.75 billion, and the investigation focuses on whether the Organon Board failed to uphold fiduciary duties to ensure a fair process for shareholders.
- RE/MAX Merger Investigation: RE/MAX Holdings, Inc. is being acquired by The Real Brokerage Inc., allowing shareholders to choose between 5.152 shares of the new company or $13.80 in cash, with investigations looking into whether the Board adequately handled the transaction to protect shareholder interests.
- Soleno Acquisition Case: Soleno Therapeutics, Inc. will be acquired by Neurocrine Biosciences for $53.00 per share, representing a total equity value of approximately $2.9 billion, while the investigation questions whether the Board ensured the deal was fair to shareholders, given that the price is below its 52-week high of $90.32.
See More
