Neurocrine Reports Positive Data from CAH Study
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 1 hour ago
0mins
Source: PRnewswire
- Significant Treatment Effects: In the CAHtalyst Pediatric study, after two years of CRENESSITY treatment, among 24 patients with advanced bone age, the mean bone age standard deviation score (SDS) decreased by 1.12, indicating slowed bone age progression and potential improvement in growth outcomes for children.
- Height Prediction Improvement: In the same cohort, 9 patients showed an average increase of 4.7 cm in predicted adult height, suggesting CRENESSITY's effectiveness in androgen control, which may positively influence pediatric growth.
- High Caregiver Satisfaction: In a preliminary survey of 29 participants, 100% of caregivers reported increased hope for their child's future, with 93% believing that CRENESSITY could effectively reduce the long-term impact of elevated adrenocorticotropic hormone and androgens, highlighting the treatment's positive social implications.
- Good Safety Profile: CRENESSITY demonstrated no new safety signals during the two-year treatment period, indicating good tolerability in pediatric patients and potentially reducing cardiometabolic risks associated with long-term glucocorticoid use.
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Analyst Views on NBIX
Wall Street analysts forecast NBIX stock price to rise
20 Analyst Rating
17 Buy
3 Hold
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Strong Buy
Current: 159.760
Low
143.00
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179.68
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Current: 159.760
Low
143.00
Averages
179.68
High
203.00
About NBIX
Neurocrine Biosciences, Inc. is a neuroscience-focused, biopharmaceutical company. It is engaged in discovering, developing, and commercializing life-changing treatments for patients with under-addressed neurological, psychiatric, endocrine, and immunological 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 first-in-class commercial portfolio includes INGREZZA (valbenazine) and CRENESSITY (crinecerfont). It also offers VYKAT XR (diazoxide choline) extended-release tablets, for the treatment of hyperphagia in adults and pediatric patients four years of age and older with Prader-Willi syndrome (PWS). Its pipeline includes direclidine / (M4 Agonist), osavampator॥ / (AMPA PAM) and others.
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.
Positive Growth Outcomes from CRENESSITY Treatment in Pediatric CAH Patients
- Slowed Bone Age Progression: Among pediatric patients treated with CRENESSITY, those with accelerated bone age at baseline showed a mean decrease of 1.12 in bone age standard deviation score (SDS) after two years, with some patients experiencing a predicted adult height increase of 4.7 cm, indicating the drug's effectiveness in hormonal control and potential to improve growth outcomes.
- Caregiver Satisfaction Survey: A survey of 29 caregivers revealed that 100% reported overall satisfaction with CRENESSITY and willingness to recommend the treatment, with 79% expressing increased optimism about the long-term impacts of high-dose steroid use, reflecting a significant boost in confidence regarding their children's future quality of life post-treatment.
- Safety and Tolerability: CRENESSITY demonstrated good tolerability over two years of treatment with no new safety signals observed, suggesting its long-term use in pediatric patients is safe and may reduce cardiometabolic risks associated with steroid use.
- Clinical Research Support: Results from the CAHtalyst studies support the FDA approval of CRENESSITY, showing its ability to effectively control hormone levels while reducing glucocorticoid doses, providing a new treatment option for patients with classic congenital adrenal hyperplasia, which holds significant clinical implications.
See More
Neurocrine Reports Positive Data from CAH Study
- Significant Treatment Effects: In the CAHtalyst Pediatric study, after two years of CRENESSITY treatment, among 24 patients with advanced bone age, the mean bone age standard deviation score (SDS) decreased by 1.12, indicating slowed bone age progression and potential improvement in growth outcomes for children.
- Height Prediction Improvement: In the same cohort, 9 patients showed an average increase of 4.7 cm in predicted adult height, suggesting CRENESSITY's effectiveness in androgen control, which may positively influence pediatric growth.
- High Caregiver Satisfaction: In a preliminary survey of 29 participants, 100% of caregivers reported increased hope for their child's future, with 93% believing that CRENESSITY could effectively reduce the long-term impact of elevated adrenocorticotropic hormone and androgens, highlighting the treatment's positive social implications.
- Good Safety Profile: CRENESSITY demonstrated no new safety signals during the two-year treatment period, indicating good tolerability in pediatric patients and potentially reducing cardiometabolic risks associated with long-term glucocorticoid use.
See More
Neurocrine Releases New Phase 3 Data on CRENESSITY for CAH
- Clinical Trial Results: Neurocrine Biosciences presented Phase 3 CAHtalyst adult study data at ENDO 2026, showing significant improvements in weight, BMI, and insulin resistance among 182 patients treated with CRENESSITY over two years, indicating the drug's potential in long-term health management.
- Dose Reduction: The study revealed a 37% to 38% reduction in average daily glucocorticoid doses over two years while maintaining baseline androgen control, demonstrating CRENESSITY's effectiveness in lowering hormone doses, alleviating side effects, and enhancing patient quality of life.
- Bone Health Improvement: Two years of CRENESSITY treatment showed favorable trends in bone density and turnover markers, highlighting the drug's potential benefits in improving bone health and possibly reducing future osteoporosis risk.
- High Patient Satisfaction: A survey of 48 patients indicated that 96% were satisfied with CRENESSITY treatment, with 93% reporting reduced side effects and 81% expressing less concern about weight gain, showcasing the drug's significant impact on improving patient quality of life.
See More
Neurocrine Releases New Ingrezza Data for Tardive Dyskinesia
- Clinical Trial Results: Neurocrine Biosciences announced new 48-week post-hoc data for Ingrezza from the KINECT 4 trial, revealing that 41% of patients failed to meet the symptomatic remission threshold from previous data, indicating the drug's potential in treating refractory patients.
- Significant Improvements: Among patients who did not achieve symptomatic remission, 86% (36/42) experienced more than a 30% reduction in the Abnormal Involuntary Movement Scale (AIMS) score, while 67% (28/42) achieved over a 50% reduction, demonstrating Ingrezza's efficacy in alleviating movement disorders.
- Drug Mechanism: Ingrezza (Valbenazine) selectively inhibits vesicular monoamine transporter 2 (VMAT2), reducing dopamine release to lower uncontrollable movements, highlighting its unique mechanism of action in treating TD.
- Market Outlook: With approximately 800,000 adults affected by TD in the U.S., Neurocrine's Ingrezza holds significant growth potential in the market, especially as the importance of evaluating hepatic risk factors in personalized treatment options becomes increasingly recognized.
See More
Neurocrine Biosciences Reveals New INGREZZA Data at Psych Congress
- Clinical Trial Results: In the KINECT®4 clinical trial, 59% of patients achieved significant symptom remission over 48 weeks, demonstrating the efficacy of INGREZZA in treating tardive dyskinesia, thereby enhancing the company's competitive position in the mental health drug market.
- Post-Hoc Analysis Findings: Even among patients who did not meet the stringent remission criteria, 86% achieved a ≥30% reduction in total AIMS score, indicating INGREZZA's broad clinical impact in improving movement severity, which may attract more patients to its use.
- Hepatic Risk Factors Analysis: A retrospective analysis of over 176,000 newly diagnosed patients revealed that 90% had at least one hepatic risk factor, underscoring the importance of evaluating liver health in treatment decisions, which could influence the market acceptance of INGREZZA.
- Long-Term Treatment Effects: In the KINECT 4 study, patients experienced an average AIMS total score reduction of 10.2 to 11.0 points over 48 weeks, indicating INGREZZA's good tolerability and effectiveness in long-term treatment, further solidifying its position in treating tardive dyskinesia.
See More
Neurocrine Reports New INGREZZA Data for Tardive Dyskinesia
- Clinical Data Highlights: Neurocrine Biosciences presented new data at a conference in Dallas showing that INGREZZA (valbenazine) significantly improves tardive dyskinesia (TD) severity in patients with intellectual and developmental disabilities, with 89% of patients demonstrating symptom improvement within four weeks, indicating the drug's potential to enhance quality of life.
- Functional Status Improvement: The study revealed that 96% of patients treated with INGREZZA experienced improvements in overall functional status, with 90% negatively impacted by TD symptoms at baseline, showcasing significant enhancements in independence and social skills post-treatment, reflecting the drug's importance in daily life.
- Patient Population Characteristics: The analysis was based on clinician-reported data from 30 patients with intellectual and developmental disabilities, averaging 47.3 years old, with 70% diagnosed with schizophrenia, highlighting the high risk and treatment needs of this population for TD.
- Research Commitment: Neurocrine emphasizes its ongoing commitment to advancing studies that better represent and serve diverse populations affected by TD, underscoring INGREZZA's potential in improving patient functionality and emotional health, further promoting awareness and treatment of the condition.
See More
Positive Growth Outcomes from CRENESSITY Treatment in Pediatric CAH Patients
- Slowed Bone Age Progression: Among pediatric patients treated with CRENESSITY, those with accelerated bone age at baseline showed a mean decrease of 1.12 in bone age standard deviation score (SDS) after two years, with some patients experiencing a predicted adult height increase of 4.7 cm, indicating the drug's effectiveness in hormonal control and potential to improve growth outcomes.
- Caregiver Satisfaction Survey: A survey of 29 caregivers revealed that 100% reported overall satisfaction with CRENESSITY and willingness to recommend the treatment, with 79% expressing increased optimism about the long-term impacts of high-dose steroid use, reflecting a significant boost in confidence regarding their children's future quality of life post-treatment.
- Safety and Tolerability: CRENESSITY demonstrated good tolerability over two years of treatment with no new safety signals observed, suggesting its long-term use in pediatric patients is safe and may reduce cardiometabolic risks associated with steroid use.
- Clinical Research Support: Results from the CAHtalyst studies support the FDA approval of CRENESSITY, showing its ability to effectively control hormone levels while reducing glucocorticoid doses, providing a new treatment option for patients with classic congenital adrenal hyperplasia, which holds significant clinical implications.
See More
Neurocrine Reports Positive Data from CAH Study
- Significant Treatment Effects: In the CAHtalyst Pediatric study, after two years of CRENESSITY treatment, among 24 patients with advanced bone age, the mean bone age standard deviation score (SDS) decreased by 1.12, indicating slowed bone age progression and potential improvement in growth outcomes for children.
- Height Prediction Improvement: In the same cohort, 9 patients showed an average increase of 4.7 cm in predicted adult height, suggesting CRENESSITY's effectiveness in androgen control, which may positively influence pediatric growth.
- High Caregiver Satisfaction: In a preliminary survey of 29 participants, 100% of caregivers reported increased hope for their child's future, with 93% believing that CRENESSITY could effectively reduce the long-term impact of elevated adrenocorticotropic hormone and androgens, highlighting the treatment's positive social implications.
- Good Safety Profile: CRENESSITY demonstrated no new safety signals during the two-year treatment period, indicating good tolerability in pediatric patients and potentially reducing cardiometabolic risks associated with long-term glucocorticoid use.
See More
Neurocrine Releases New Phase 3 Data on CRENESSITY for CAH
- Clinical Trial Results: Neurocrine Biosciences presented Phase 3 CAHtalyst adult study data at ENDO 2026, showing significant improvements in weight, BMI, and insulin resistance among 182 patients treated with CRENESSITY over two years, indicating the drug's potential in long-term health management.
- Dose Reduction: The study revealed a 37% to 38% reduction in average daily glucocorticoid doses over two years while maintaining baseline androgen control, demonstrating CRENESSITY's effectiveness in lowering hormone doses, alleviating side effects, and enhancing patient quality of life.
- Bone Health Improvement: Two years of CRENESSITY treatment showed favorable trends in bone density and turnover markers, highlighting the drug's potential benefits in improving bone health and possibly reducing future osteoporosis risk.
- High Patient Satisfaction: A survey of 48 patients indicated that 96% were satisfied with CRENESSITY treatment, with 93% reporting reduced side effects and 81% expressing less concern about weight gain, showcasing the drug's significant impact on improving patient quality of life.
See More
Neurocrine Releases New Ingrezza Data for Tardive Dyskinesia
- Clinical Trial Results: Neurocrine Biosciences announced new 48-week post-hoc data for Ingrezza from the KINECT 4 trial, revealing that 41% of patients failed to meet the symptomatic remission threshold from previous data, indicating the drug's potential in treating refractory patients.
- Significant Improvements: Among patients who did not achieve symptomatic remission, 86% (36/42) experienced more than a 30% reduction in the Abnormal Involuntary Movement Scale (AIMS) score, while 67% (28/42) achieved over a 50% reduction, demonstrating Ingrezza's efficacy in alleviating movement disorders.
- Drug Mechanism: Ingrezza (Valbenazine) selectively inhibits vesicular monoamine transporter 2 (VMAT2), reducing dopamine release to lower uncontrollable movements, highlighting its unique mechanism of action in treating TD.
- Market Outlook: With approximately 800,000 adults affected by TD in the U.S., Neurocrine's Ingrezza holds significant growth potential in the market, especially as the importance of evaluating hepatic risk factors in personalized treatment options becomes increasingly recognized.
See More
Neurocrine Biosciences Reveals New INGREZZA Data at Psych Congress
- Clinical Trial Results: In the KINECT®4 clinical trial, 59% of patients achieved significant symptom remission over 48 weeks, demonstrating the efficacy of INGREZZA in treating tardive dyskinesia, thereby enhancing the company's competitive position in the mental health drug market.
- Post-Hoc Analysis Findings: Even among patients who did not meet the stringent remission criteria, 86% achieved a ≥30% reduction in total AIMS score, indicating INGREZZA's broad clinical impact in improving movement severity, which may attract more patients to its use.
- Hepatic Risk Factors Analysis: A retrospective analysis of over 176,000 newly diagnosed patients revealed that 90% had at least one hepatic risk factor, underscoring the importance of evaluating liver health in treatment decisions, which could influence the market acceptance of INGREZZA.
- Long-Term Treatment Effects: In the KINECT 4 study, patients experienced an average AIMS total score reduction of 10.2 to 11.0 points over 48 weeks, indicating INGREZZA's good tolerability and effectiveness in long-term treatment, further solidifying its position in treating tardive dyskinesia.
See More
Neurocrine Reports New INGREZZA Data for Tardive Dyskinesia
- Clinical Data Highlights: Neurocrine Biosciences presented new data at a conference in Dallas showing that INGREZZA (valbenazine) significantly improves tardive dyskinesia (TD) severity in patients with intellectual and developmental disabilities, with 89% of patients demonstrating symptom improvement within four weeks, indicating the drug's potential to enhance quality of life.
- Functional Status Improvement: The study revealed that 96% of patients treated with INGREZZA experienced improvements in overall functional status, with 90% negatively impacted by TD symptoms at baseline, showcasing significant enhancements in independence and social skills post-treatment, reflecting the drug's importance in daily life.
- Patient Population Characteristics: The analysis was based on clinician-reported data from 30 patients with intellectual and developmental disabilities, averaging 47.3 years old, with 70% diagnosed with schizophrenia, highlighting the high risk and treatment needs of this population for TD.
- Research Commitment: Neurocrine emphasizes its ongoing commitment to advancing studies that better represent and serve diverse populations affected by TD, underscoring INGREZZA's potential in improving patient functionality and emotional health, further promoting awareness and treatment of the condition.
See More
