SAN DIEGO, July 15, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced results from a new analysis using real-world data from CAHtalog®, a comprehensive registry of patients with classic congenital adrenal hyperplasia in the United States. The analysis demonstrated significant variability in glucocorticoid treatment regimens and frequent changes in health status and outcomes over time in both pediatric and adult patients. These results were presented at the Endocrine Society's ...

Core Insights - Neurocrine Biosciences presented new data from the Phase 3 CAHtalyst studies showing that CRENESSITY® (crinecerfont) leads to significant improvements in weight-related outcomes for patients with classic congenital adrenal hyperplasia (CAH) [1][2][5] Group 1: Study Overview - The Phase 3 CAHtalyst program is the largest interventional clinical trial for classic CAH, involving 285 patients (103 pediatric and 182 adult) [2][12] - The studies included a double-blind, placebo-controlled period followed by an open-label period where all patients received CRENESSITY [2][12] Group 2: Weight-Related Outcomes - Adult patients treated with CRENESSITY showed a mean BMI reduction of 0.8 kg/m² compared to a 0.4 kg/m² reduction in the placebo group at Month 12 [4][6] - 39% of adult patients on CRENESSITY achieved over a 5% weight reduction, compared to 14% in the placebo group [4][6] - Pediatric patients on CRENESSITY maintained reductions in BMI standard deviation scores (SDS) through Week 52, while those on placebo saw an increase [6][4] Group 3: Insulin Resistance Improvements - Significant reductions in insulin resistance were observed in both adult and pediatric patients treated with CRENESSITY compared to placebo [5][6] - The mean HOMA-IR score decreased more significantly in the CRENESSITY group than in the placebo group [6][7] Group 4: Safety Profile - CRENESSITY demonstrated a favorable safety profile, with common side effects being mild to moderate and not leading to discontinuation of the drug [8][20] - Common side effects in children included headache and stomach pain, while adults reported fatigue and headache [8][22] Group 5: Regulatory and Market Context - Data from the CAHtalyst studies supported the FDA approval of CRENESSITY in December 2024 [15] - CRENESSITY is positioned to evolve the standard of care for classic CAH by allowing for reduced glucocorticoid doses while maintaining or improving androgen control [2][16]

Group 1 - Neurocrine Biosciences has scheduled its Q2 2025 financial results conference call and webcast for July 30, 2025, at 1:30 p.m. PT [1][4] - The conference call will be accessible via the company's website, and a replay will be available approximately one hour after the event [1][4] - The company focuses on developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders, with a diverse portfolio of FDA-approved treatments [2] Group 2 - Neurocrine's portfolio includes treatments for tardive dyskinesia, Huntington's disease-related chorea, congenital adrenal hyperplasia, endometriosis, and uterine fibroids, with some collaborations [2] - The company has a robust pipeline with multiple compounds in mid- to late-phase clinical development across its core therapeutic areas [2]

Group 1 - The article promotes a weekly newsletter focused on stocks in the biotech, pharma, and healthcare industries, aimed at both novice and experienced investors [1] - The newsletter provides insights on key trends, catalysts driving valuations, product sales forecasts, and integrated financial statements for major pharmaceutical companies [1] - The author, Edmund Ingham, has over 5 years of experience in the biotech sector and has compiled detailed reports on more than 1,000 companies [1]

Core Insights - Neurocrine Biosciences has initiated a Phase 1 first-in-human clinical study for the investigational compound NBIP-01435, aimed at treating congenital adrenal hyperplasia (CAH) [1][2] - NBIP-01435 is a long-acting corticotropin-releasing factor type 1 receptor antagonist, which may improve androgen control and allow for lower glucocorticoid dosing [3] - The company has a strong commitment to expanding treatment options for CAH, with NBIP-01435 being the first investigational peptide from its biologics pipeline to enter clinical trials [2][4] Company Overview - Neurocrine Biosciences is a leading biopharmaceutical company focused on neuroscience, dedicated to developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders [5] - The company has a diverse portfolio that includes FDA-approved treatments for various conditions, including CAH, and a robust pipeline with multiple compounds in mid- to late-phase clinical development [5] - In December 2024, Neurocrine received FDA approval for crinecerfont, marking the first new treatment for CAH in 70 years [3]

Core Insights - Neurocrine Biosciences announced significant reductions in cognitive and motor-related burdens in adults with Huntington's disease chorea treated with INGREZZA (valbenazine) capsules, based on new patient-reported post-hoc data from the KINECT-HD study [1][4] Group 1: Study Findings - The KINECT-HD study is the first clinical trial to show measurable changes in cognitive and motor disease burden with a vesicular monoamine transporter 2 inhibitor in Huntington's disease patients [1][2] - In the 12-week Phase 3 KINECT-HD clinical trial, INGREZZA showed a significant reduction in chorea severity, with a placebo-adjusted mean reduction in the Total Maximal Chorea (TMC) score of 3.2 units (p<0.0001) [11] - Patient-reported analyses indicated greater reductions in cognitive and motor-related disease burden for INGREZZA compared to placebo across various domains, including memory loss and mobility limitations [3][4] Group 2: Cognitive and Motor-Related Improvements - Statistically significant improvements were observed in cognition-related items, such as memory loss (-0.9 for INGREZZA vs -0.2 for placebo) and decision-making abilities (-1.0 for INGREZZA vs -0.3 for placebo) [3] - For motor-related items, INGREZZA demonstrated larger reductions in scores compared to placebo, including abnormal movements (-1.5 for INGREZZA vs -0.8 for placebo) and impaired coordination (-1.0 for INGREZZA vs -0.4 for placebo) [3][5] Group 3: Background on Huntington's Disease - Huntington's disease is a hereditary progressive neurodegenerative disorder affecting approximately 41,000 adults in the U.S., characterized by motor, cognitive, and psychiatric symptoms [7] - Chorea, a common symptom of Huntington's disease, involves irregular and unpredictable movements that can interfere with motor coordination and daily activities [7] Group 4: About INGREZZA - INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for treating chorea associated with Huntington's disease and tardive dyskinesia [14][15] - The drug is designed for once-daily administration without the need for titration, making it convenient for patients [16]

Core Insights - Neurocrine Biosciences appointed Lewis Choi as Chief Information Officer, effective June 9, 2025, to lead the company's technology initiatives [1][2] - Choi brings 25 years of experience in information technology and artificial intelligence, previously serving at Thermo Fisher Scientific [2] - The company is focused on advancing its technology to support the launch of CRENESSITY, growth of INGREZZA, and development of its pipeline [2] Company Overview - Neurocrine Biosciences is a biopharmaceutical company dedicated to developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders [3] - The company has a diverse portfolio that includes FDA-approved treatments for conditions such as tardive dyskinesia and Huntington's disease, along with a robust pipeline in mid- to late-phase clinical development [3] - The mission of the company is to relieve suffering for patients with significant unmet medical needs through innovative neuroscience [3]

Core Insights - Neurocrine Biosciences announced new data from the Phase 4 KINECT-PRO study showing significant improvements in physical, social, and emotional functioning in patients with tardive dyskinesia treated with INGREZZA [1][6][4] Group 1: Study Overview - KINECT-PRO is the first study to evaluate patient-reported outcomes with INGREZZA using multiple validated scales, including the Tardive Dyskinesia Impact Scale [1][6] - The study enrolled 59 patients who received once-daily INGREZZA (40 mg, 60 mg, or 80 mg) for up to 24 weeks, with 52 patients completing the Week 24 visit [2][4] Group 2: Study Objectives and Measures - The primary objective was to assess changes in patient-reported physical and socio-emotional impacts of tardive dyskinesia during treatment [3] - Outcomes were measured at Weeks 4, 8, 16, and 24 using the Tardive Dyskinesia Impact Scale, Sheehan Disability Scale, and EQ Visual Analogue Scale [3] Group 3: Results and Improvements - Improvements in TDIS, SDS, EQ-VAS, and AIMS were observed as early as Week 4 and sustained through Week 24, indicating robust and clinically meaningful enhancements in functioning [4][6] - The mean change from baseline at Week 24 for the overall population showed a reduction of -8.0 in TDIS, -2.3 in social life, -1.6 in family life, an increase of +13.1 in EQ-VAS, and a reduction of -6.8 in AIMS total score [5] Group 4: Subgroup Analysis - Both mild and moderate/severe tardive dyskinesia subgroups showed significant improvements, with mild TD subgroup reporting a mean change of -6.8 in TDIS and +12.8 in EQ-VAS [5][7] - The moderate/severe TD subgroup reported a mean change of -8.9 in TDIS and +13.3 in EQ-VAS [7] Group 5: Safety and Tolerability - The safety and tolerability profile of INGREZZA was consistent with known data, with no new safety concerns identified during the study [7][6]

Core Insights - Neurocrine Biosciences announced significant improvements in symptoms and overall severity of schizophrenia in adults from the Phase 2 study of NBI-1117568, the first oral muscarinic M4 selective orthosteric agonist in clinical development for this condition [1][2][6] Company Overview - Neurocrine Biosciences is focused on developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders, with a diverse portfolio that includes FDA-approved treatments and a robust pipeline of compounds in clinical development [13] Study Details - The Phase 2 study involved 210 adults aged 18 to 55 with schizophrenia, randomized to receive either NBI-1117568 or placebo over a six-week period, followed by a two-week safety follow-up [2][9] - The primary endpoint was the change in total Positive and Negative Syndrome Scale (PANSS) score from baseline to Week 6, showing statistically significant improvements with the 20 mg dose of NBI-1117568 by Week 3 [4][6] Safety and Tolerability - NBI-1117568 was generally safe and well tolerated, with treatment discontinuation rates due to adverse events similar to placebo; common adverse events included somnolence (10.7% vs 2.9%) and dizziness (9.3% vs 1.4%) [3][6] Future Development - Based on positive Phase 2 results, a Phase 3 registrational program has been initiated to further evaluate the efficacy, safety, and tolerability of NBI-1117568, expected to enroll approximately 280 patients [6][9]

Summary of Neurocrine's Conference Call Company Overview - **Company**: Neurocrine Biosciences - **Key Products**: Pranesity, INGREZZA - **Financial Guidance**: Expected revenue of $2.5 to $2.6 billion for the year [5][41] Core Insights and Arguments Product Launch and Performance - **Pranesity Launch**: Launched in December, first treatment for congenital adrenal hyperplasia in over 70 years; exceeded expectations with over 400 treatment forms submitted in Q1 [6][16] - **INGREZZA Performance**: Despite a challenging environment, INGREZZA showed better-than-expected performance in Q1, with a record number of new patient starts [33][41] Market Dynamics - **Diagnosis Rates**: For INGREZZA, diagnosis rates have increased from 2% at launch to 40% currently, but only 10% of diagnosed patients are being treated [5][34] - **Patient Population**: Estimated 20,000 classic congenital adrenal hyperplasia patients in the U.S., primarily treated by endocrinologists [14][15] Financial Position - **Cash Reserves**: Neurocrine has $1.8 billion in cash, reflecting financial flexibility despite using $750 million to retire convertible debt [9] - **Reimbursement Success**: 70% of Pranesity prescriptions were commercially reimbursed in Q1, exceeding expectations [31] Additional Important Points Clinical Trials and Pipeline - **Osvampir and M4 Agonist Programs**: Two major phase three programs underway, focusing on major depressive disorder and schizophrenia [7][9] - **Patient Engagement**: Early patient engagement and education efforts contributed to the successful launch of Pranesity [13][21] Market Challenges - **Competitive Dynamics**: The market for INGREZZA faced challenges due to competitive pressures and payer dynamics, leading to a slowdown in new patient starts in late 2022 [34][36] - **Reauthorization Process**: Many existing patients require reauthorization at the beginning of the year, impacting Q1 performance [37] Future Outlook - **Growth Expectations**: Anticipated continued growth in new patient starts and overall revenue, with confidence in the sales team's productivity [41][42] - **Contracting Strategy**: Entering into contracts with payers to ensure patient retention and facilitate new patient additions [39][40]