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]

Core Insights - Neurocrine Biosciences announced significant findings from the Phase 3 CAHtalyst Pediatric study, demonstrating lasting reductions in glucocorticoid doses and improvements in clinical outcomes for pediatric patients with classic congenital adrenal hyperplasia (CAH) treated with CRENESSITY (crinecerfont) for up to one year [1][2][12] Group 1: Clinical Study Findings - The Phase 3 CAHtalyst Pediatric study included 103 pediatric patients aged 4 to 17 years, making it the largest interventional clinical trial program for classic CAH [2][10] - Patients receiving CRENESSITY showed a mean glucocorticoid dose reduction from 16.4 mg/m²/d at baseline to 13.5 mg/m²/d at Week 52, a decrease of 2.9 mg/m²/d [3] - Improvements in clinical outcomes included a reduction in mean body mass index (BMI) standard deviation scores by -0.09 in the CRENESSITY group compared to -0.02 in the placebo group [3] Group 2: Hormonal and Clinical Outcomes - Adrenocorticotropic hormone (ACTH), 17-hydroxyprogesterone (17-OHP), and androstenedione (A4) levels remained below baseline levels in patients treated with CRENESSITY, despite reduced glucocorticoid doses [1][4] - The proportion of patients achieving glucocorticoid doses within the physiologic range (≤11 mg/m²/d hydrocortisone equivalents) remained stable from Week 28 (30%) to Week 52 (32%) [4] Group 3: Safety and Tolerability - CRENESSITY was generally well tolerated, with no adrenal crises reported during the double-blind placebo-controlled (DBPC) period [5] - The most common adverse reactions included headache (25% vs. 6% for placebo), abdominal pain (13% vs. 0%), and fatigue (7% vs. 0%) [5] Group 4: Treatment Implications - CRENESSITY allows for a reduction in glucocorticoid doses while maintaining or improving hormonal levels, potentially transforming the treatment landscape for children and adolescents with classic CAH [2][14] - The study supports the ongoing use of CRENESSITY in conjunction with glucocorticoids for effective management of CAH [16]

Core Insights - Neurocrine Biosciences presented new analyses from a Phase 4 randomized withdrawal study showing that patients with tardive dyskinesia who continued treatment with INGREZZA® (valbenazine) capsules reported improvements in functional and health-related quality of life measures [1][2] - The findings were presented at the 2025 ISPOR Conference, highlighting the clinically meaningful improvements in patients' quality of life due to INGREZZA treatment [1][2] Study Details - The analyses were based on data from 127 patients in a Phase 4, double-blind, placebo-controlled study, where patients received up to 80 mg of INGREZZA for eight weeks before being randomized to continue treatment or receive a placebo for an additional eight weeks [2][3] - Significant improvements were observed in health-related quality of life (HRQoL) measures, including mobility, self-care, usual activities, and pain/discomfort, with placebo-adjusted differences noted in anxiety/depression and mobility [3][4] Health-Related Quality of Life Improvements - Patients receiving INGREZZA for eight weeks showed improvements in HRQoL dimensions, with changes from baseline reported as follows: mobility (-0.27), self-care (-0.28), usual activities (-0.36), and pain/discomfort (-0.34) [3] - Continued treatment for an additional eight weeks resulted in further improvements, including a placebo-adjusted difference in mobility of -0.34 and anxiety/depression of -0.38 compared to the placebo group [3] Functional Impairment Improvements - In the open-label portion of the study, patients reported significant improvements in work/school (-1.37), social life (-1.65), family/home life (-1.30), and total Sheehan Disability Scale (SDS) score (-4.28) after eight weeks of treatment [4] - Continued treatment for an additional eight weeks led to further improvements in social life (-0.95) and family/home life (-0.89) compared to those receiving placebo [4] About INGREZZA - INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for treating adults with tardive dyskinesia and chorea associated with Huntington's disease [12][13] - The drug is designed to inhibit dopamine release, potentially reducing uncontrollable movements associated with these conditions [13][14]

Core Insights - Neurocrine Biosciences announced positive Phase 3 data for CRENESSITY (crinecerfont) in treating congenital adrenal hyperplasia (CAH), showing significant improvements in hormone levels and glucocorticoid dose reductions in both pediatric and adult patients [1][3][16] Group 1: Study Results - In the pediatric study, 90% of participants on CRENESSITY achieved at least one threshold for androstenedione reduction or glucocorticoid reduction, compared to 21% on placebo [7][8] - At Week 28, 30% of pediatric participants on CRENESSITY reached a physiologic glucocorticoid dose while maintaining or improving androstenedione levels, whereas no placebo participants achieved this [8] - In the adult study, at Week 24, 47% of male patients on CRENESSITY normalized luteinizing hormone (LH) levels compared to 22% on placebo [10] Group 2: Treatment Mechanism - CRENESSITY is a novel oral corticotropin-releasing factor type 1 receptor antagonist that aims to reduce excess adrenal androgens and allow for lower glucocorticoid doses, addressing complications from high-dose steroid use [3][17] - The drug works by decreasing adrenocorticotropic hormone (ACTH) levels, which in turn reduces the production of adrenal androgens, potentially alleviating symptoms associated with CAH [17] Group 3: Safety and Tolerability - CRENESSITY was generally well tolerated in pediatric patients, with no adrenal crises reported during the double-blind treatment period [5] - The most common adverse reactions in pediatric patients included headache (25% vs. 6% for placebo) and abdominal pain (13% vs. 0%) [5] - In adult patients, 1.6% experienced adrenal crisis while on CRENESSITY, with common side effects including fatigue (25% vs. 15% for placebo) and headache (16% vs. 15%) [10]

Core Viewpoint - Grifols (GRFS) is currently positioned as a better value opportunity compared to Neurocrine Biosciences (NBIX) based on various valuation metrics and earnings outlook [1][3]. Valuation Metrics - GRFS has a forward P/E ratio of 7.65, significantly lower than NBIX's forward P/E of 31.35, indicating that GRFS may be undervalued [5]. - The PEG ratio for GRFS is 0.26, while NBIX's PEG ratio is 1.22, suggesting that GRFS has a more favorable earnings growth outlook relative to its price [5]. - GRFS's P/B ratio stands at 0.60, compared to NBIX's P/B of 4.63, further highlighting GRFS's relative undervaluation [6]. Earnings Outlook - GRFS has a Zacks Rank of 2 (Buy), indicating a stronger improvement in its earnings outlook compared to NBIX, which has a Zacks Rank of 3 (Hold) [3][6]. - The overall valuation metrics and solid earnings outlook position GRFS as the superior value option in the current market [6].

Summary of Neurocrine's Conference Call Company Overview - **Company**: Neurocrine Biosciences - **CEO**: Kyle Gano, appointed in October 2023 Macro Issues - **Executive Order on Most Favored Nation**: - Uncertainty regarding the impact on Neurocrine due to lack of details on the executive order [3][4][5][6] - Focus on building a resilient business amidst evolving regulations [6][7] - **Tariffs**: - Tariffs are not expected to significantly impact the cost of goods for Neurocrine's products [9][10] - **FDA Interactions**: - No current NDA reviews; recent interactions focused on Phase II discussions for upcoming Phase III programs [11][12] Product Launch: Crinesity (Canestrafont) - **Sales Performance**: - Recognized $14.5 million in sales with over 400 new patient starts in the last quarter [16] - Impressive reimbursement rate of approximately 70% [16][18] - **Launch Strategy**: - The launch is described as "measured" due to the need for physician education and patient awareness [18][20] - Patients must navigate insurance processes, which may delay prescriptions [20][21] - **Patient Demographics**: - Initial patient population is skewed towards pediatrics due to more frequent physician visits [27][32] - Adult women are expected to show more motivation for treatment compared to men [49] - **Direct-to-Consumer Advertising**: - Traditional advertising may be less effective due to the smaller patient population for congenital adrenal hyperplasia (CAH) [34][36] - **Market Potential**: - Crinesity is viewed as a potential blockbuster, with expectations of significant market uptake [38][39] Competitive Landscape - **Differentiation**: - Neurocrine's approach with Crinesity is based on efficacy, safety, and tolerability, which are critical for patient adoption [43][46] - The company aims to treat the majority of patients with CAH before competitors enter the market [45][46] Pipeline Developments - **Upcoming Data Readouts**: - Phase III trials for osavapitur and MBI-568 are anticipated [52] - Studies on valbenazine for dyskinesia associated with cerebral palsy and adjunctive treatment for schizophrenia are ongoing [56][60] - **Muscarinic Agonists**: - Neurocrine is developing multiple muscarinic agonists, focusing on selective activation to improve efficacy and safety [70][72] Financial Outlook - **Investment in Growth**: - The company is prioritizing growth over immediate profitability, with significant investments in sales and marketing for Crinesity and INGREZZA [77][78] - R&D spending is projected to be in the mid-30% range of net revenue due to ongoing Phase III trials [79] Conclusion - Neurocrine is navigating a complex regulatory environment while launching Crinesity, which shows promising early sales. The company is focused on educating physicians and patients, with a strong pipeline and strategic investments aimed at long-term growth and market leadership in the treatment of CAH and other conditions.

Core Insights - Neurocrine Biosciences, Inc. will participate in two investor conferences in May 2025, showcasing its commitment to engaging with investors and stakeholders [1][5]. Company Overview - Neurocrine Biosciences is a leading biopharmaceutical company focused on neuroscience, 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, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia, endometriosis, and uterine fibroids, along with a robust pipeline of compounds in mid- to late-phase clinical development [3]. Upcoming Events - CEO Kyle Gano and VP of Investor Relations Todd Tushla will present at the BofA Securities 2025 Health Care Conference on May 13, 2025, at 11:20 AM Pacific Time in Las Vegas [5]. - CFO Matt Abernethy and CCO Eric Benevich will present at the RBCCM Global Healthcare Conference on May 20, 2025, at 11:30 AM Eastern Time in New York [5].

Core Insights - Neurocrine Biosciences Inc. reported better-than-expected first-quarter 2025 earnings, with adjusted EPS of 70 cents, surpassing the consensus estimate of 54 cents, but lower than the $1.20 reported a year ago [1] - The company reported sales of $572.6 million, an increase from $515.3 million a year ago, but missed the consensus of $559.3 million [1] Ingrezza Performance - Ingrezza's first-quarter 2025 net product sales were $545 million, reflecting an 8% year-over-year growth driven by strong patient demand and improved gross-to-net dynamics [2] - Neurocrine reaffirmed its 2025 Ingrezza sales guidance of $2.5 billion to $2.6 billion [3] Analyst Commentary - Analysts view Ingrezza as a leading treatment for tardive dyskinesia (TD), highlighting its once-daily dosing and lack of severe FDA warnings, along with 14 years of potential patent protection [4] - The positive performance of Crenessity indicates strong sales efforts, with early metrics showing significant interest in the orphan disease therapy [5] - Stock price for Neurocrine Biosciences increased by 14% to $125.07 following the earnings report [5] Analyst Ratings and Price Targets - Needham maintains a Buy rating, raising the price target from $138 to $139 [6] - Canaccord Genuity also maintains a Buy rating, increasing the price target from $158 to $160 [6] - UBS keeps a Buy rating, raising the price target from $137 to $152 [6] - BMO Capital maintains a Market Perform rating, increasing the price target from $96 to $115 [6] - Guggenheim maintains a Buy rating, raising the price target from $155 to $165 [6]

New multimedia awareness campaign empowers people living with tardive dyskinesia to stand up to stigma and speak openly with their healthcare provider about symptoms and treatment SAN DIEGO, May 6, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the launch of ConnectING with Carnie™, a multimedia awareness campaign in partnership with Grammy- nominated singer-songwriter and mental health advocate, Carnie Wilson. The campaign aims to support people struggling with the uncontr ...

Financial Performance & Guidance - INGREZZA net product sales reached $545 million in Q1 2025, representing an 8% year-over-year growth[12] - CRENESSITY net product sales were $145 million in Q1 2025, including 413 total patient enrollment forms[11] - The company reaffirms INGREZZA net sales guidance of $250 billion to $260 billion for 2025[6, 11] - Cash and investments totaled approximately $18 billion as of March 31, 2025[6] - Non-GAAP R&D expense was $2402 million in Q1 2025, an increase from $1424 million in Q1 2024[12] - Non-GAAP SG&A expense was $245 million in Q1 2025, compared to $216 million in Q1 2024[12] Pipeline Development - Phase 3 registrational program initiated for osavampator as an adjunctive therapy for the treatment of MDD in adults[11] - Phase 1 studies initiated for NBI-'355 (Nav12 / 16 Inhibitor) for Epilepsy and NBI-'675 (VMAT2 Inhibitor) for Movement Disorders[11] Market & Product - Approximately 800000 people in the US are estimated to be affected by Tardive Dyskinesia[21] - Chorea affects approximately 90% of the 40000 patients with Huntington's Disease in the US[24] - CRENESSITY is the first new treatment available for Classic CAH in 70 years, targeting approximately 20000 pediatric and adult patients in the US[30, 31]