Summary of Neurocrine Biosciences (NBIX) FY Conference Call - August 13, 2025 Company Overview - Neurocrine Biosciences specializes in neuro and endocrine disorders, with a strong focus on commercialization and a robust pipeline of products [2][5] Key Points on Commercialization and Product Launches - The company successfully launched KRONESTY, a treatment for congenital adrenal hyperplasia (CAH), which has exceeded internal expectations due to pre-launch disease state education efforts [6][10] - KRONESTY's uptake has been strong among healthcare providers, with a focus on patient identification and education to ensure ongoing treatment adherence [11][12] - The reimbursement rate for KRONESTY was reported at 76% in Q2, indicating strong market access compared to previous products [15][16] Financial Performance - Q2 revenue for INGREZZA reached $624 million, reflecting a 15% quarter-over-quarter growth and an 8% year-over-year increase [24] - The company narrowed its sales guidance for INGREZZA to between $2.5 billion and $2.55 billion for the year, anticipating double-digit growth [27][28] Pipeline and Research Development - Neurocrine aims to initiate four new Phase I trials and two new Phase II trials annually, with a goal of introducing one new medicine every two years [8] - The company is advancing its lead muscarinic program, dereclidine, in Phase III trials for schizophrenia and plans to explore bipolar mania later this year [39][46] - The company is also investigating opportunities in Alzheimer's disease psychosis, leveraging its diverse muscarinic portfolio [47] Competitive Landscape - Neurocrine acknowledges competition in the CAH space but emphasizes its history of being first in various disease states and its commitment to patient safety and tolerability [17][18] - The company is preparing for potential impacts from the Inflation Reduction Act (IRA) on its competitors, particularly regarding pricing negotiations starting in 2027 [30][33] Future Outlook - The focus remains on the U.S. market for KRONESTY, with plans for international expansion being evaluated but not imminent [23] - The company is optimistic about the long-term growth potential of INGREZZA, supported by a strong intellectual property position extending to 2038 [34] Additional Insights - Neurocrine's approach to patient engagement and education is critical for the success of its products, particularly in rare diseases where awareness is low [12][13] - The company is committed to maintaining high standards in its research and development efforts, ensuring a balanced portfolio of validated and unvalidated targets [7][8] This summary encapsulates the key insights from the Neurocrine Biosciences FY Conference Call, highlighting the company's strategic focus, financial performance, and future growth potential in the biopharmaceutical landscape.

Financial Data and Key Metrics Changes - Neurocrine Biosciences reported net product sales of $682 million for Q2 2025, reflecting a 17% year-over-year growth [11] - The company refined its net sales guidance for INGREZZA to a range of $2.5 billion to $2.55 billion, accounting for anticipated double-digit volume gains [14] - The company has $1.8 billion in cash and a strong balance sheet to support its commercial and clinical development strategies [15] Business Line Data and Key Metrics Changes - INGREZZA sales reached $624 million in Q2 2025, with a record number of new patient starts, representing 15% sequential growth and 8% year-over-year growth [16] - KRONESTIV achieved net sales of $53 million in Q2 2025, growing from $15 million in Q1 2025, indicating strong early adoption [12][19] Market Data and Key Metrics Changes - The company has expanded Medicare formulary coverage for INGREZZA to approximately 70% of Medicare beneficiary lives in the TD market, improving patient access [17] - Over 75% of all dispensed prescriptions for KRONESTIV in Q2 were reimbursed, indicating strong market acceptance [19] Company Strategy and Development Direction - Neurocrine aims to solidify its position as a leading neurology-focused enterprise by investing in advancing and expanding its pipeline [10] - The company is focused on enhancing payer access and market share for INGREZZA through strategic investments [6][18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth potential of both INGREZZA and KRONESTIV, driven by strong market access initiatives [6][10] - The company anticipates continued revenue contribution from both products, enabling further investment in R&D and patient support [10] Other Important Information - The company has initiated multiple Phase III programs within a single calendar year, showcasing a robust clinical pipeline [9] - The recent ENDO conference highlighted the company's progress and the positive reception of its products among clinicians [7][20] Q&A Session Summary Question: Guidance adjustment and pricing dynamics - Management explained that the adjustment in guidance was primarily due to a change in pricing assumptions, moving from flat pricing expectations to a projected 5% price decline for the year [30][32] Question: KRONESTIV launch dynamics - Management noted that the adoption of KRONESTIV has been steady and consistent, with no significant bolus effect from prior clinical trials [36][40] Question: Prescribing concentration and treatment dynamics - Management indicated that the patient population for CAH is not highly concentrated, allowing for broader adoption across various prescribers [44][46] Question: Transition to formulary-driven reimbursement - Management stated that while some health plans may conduct formulary reviews, many will continue to reimburse KRONESTIV as a non-formulary product [94] Question: Reauthorization and patient authorization patterns - Management reported that patients typically receive authorization for six or twelve prescriptions, with a pattern emerging for reauthorization [108]

Financial Data and Key Metrics Changes - The company reported net product sales of $682 million for Q2 2025, representing a 17% year-over-year growth [9] - INGREZZA sales reached $624 million in Q2, showing a 15% sequential growth and an 8% year-over-year increase [14] - KRONESTIV sales grew from $15 million in Q1 2025 to $53 million in Q2 2025, reflecting strong early adoption [10] - The company has refined its net sales guidance for INGREZZA to a range of $2.5 billion to $2.55 billion, accounting for anticipated double-digit volume gains [12] Business Line Data and Key Metrics Changes - The INGREZZA franchise continues to show solid growth, with record new patient starts and total prescriptions [14] - KRONESTIV has been well received, with over 75% of all dispensed prescriptions reimbursed in Q2 [18] - The company has seen a significant increase in Medicare formulary coverage for INGREZZA, now covering approximately 70% of Medicare beneficiary lives in the TD market [15] Market Data and Key Metrics Changes - The company noted that less than half of the estimated 800,000 TD patients have been diagnosed, indicating a substantial market opportunity [16] - The adoption of KRONESTIV is widespread across both pediatric and adult patients, with a slight trend towards pediatric patients [19] Company Strategy and Development Direction - The company aims to solidify its position as a leading neurology-focused enterprise by investing in advancing and expanding its pipeline [9] - The strategic investments in market access and sales force expansion are expected to drive long-term growth for both INGREZZA and KRONESTIV [11][14] - The company is focused on diversifying its neuroscience pipeline, with multiple Phase III programs initiated within a single calendar year [8] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued growth of the INGREZZA franchise due to market access initiatives [5] - The company anticipates that the strong performance in the first half of the year will enable further investments in R&D and commercial strategies [9] - Management acknowledged the dynamic nature of the business and the need to adjust guidance based on pricing and volume expectations [30] Other Important Information - The company has a strong balance sheet with $1.8 billion in cash, positioning it well for continued growth [13] - The clinical pipeline includes multiple Phase III programs, with anticipated data readouts in 2027 and 2028 [22] Q&A Session Summary Question: How is the company triangulating the guidance adjustments? - The company adjusted the top end of guidance due to a change in pricing assumptions, moving from flat pricing expectations to a negative 5% price decline for the year [30] Question: Can you discuss the dynamics at clinics regarding KRONESTIV? - The company noted steady and consistent adoption of KRONESTIV, with no significant bolus effect from prior clinical trials [36][40] Question: What metrics can be shared regarding prescribing concentration for KRONESTIV? - The company estimates that around 15% of the total CAH patient population flows through a small number of accredited centers, indicating room for growth in community endocrinologists [44][46] Question: What is the reimbursement process for KRONESTIV? - The company reported a high rate of reimbursement for KRONESTIV, with over 75% of dispensed prescriptions reimbursed, and noted that many patients do not need to go on free goods programs [93] Question: How does the company view the implications of the IRA? - The company aims to maximize patient access to INGREZZA and is preparing for the new pricing environment that will come with the IRA [82][84]

Financial Performance - Total Neurocrine net product sales reached $682 million in Q2 2025, representing a 21% quarter-over-quarter (QoQ) growth and a 17% year-over-year (YoY) growth compared to Q1 2025 and Q2 2024, respectively [11] - INGREZZA net product sales were $624 million in Q2 2025, reflecting a 15% QoQ growth and an 8% YoY growth compared to Q1 2025 and Q2 2024, respectively [11] - CRENESSITY net product sales amounted to $53 million in Q2 2025, including 664 total new patient enrollment forms [11] - The company narrowed INGREZZA net sales guidance for 2025 to $2.50 - $2.55 billion, reflecting double-digit volume growth partially offset by a lower net price due to expanded access [7, 11] - Cash and investments totaled approximately $1.8 billion as of June 30, 2025 [7] Research and Development - The company initiated all Phase 3 registrational programs for osavampator (AMPA PAM) for MDD and NBI-'568 (Selective M4 Agonist) for schizophrenia [11] - A Phase 1 study was initiated for NBIP-'1435, a long-acting CRF-1 receptor antagonist, for the potential treatment of classic CAH [11] - Phase 2 studies are expected to be initiated for NBI-'568 in bipolar mania and NBI-'570 (Selective Dual M1 / M4 Agonist) for schizophrenia [11] - Top-line data is expected to be reported in Q4 2025 for the Phase 3 study of valbenazine for dyskinetic cerebral palsy and the Phase 2 study of NBI-'770 (NMDA NR2B NAM) for the treatment of MDD [11] Pipeline and Portfolio - The company has a pipeline of 12 programs, including the first biologic program NBIP-'1435 [8] - The company expects Phase 1 muscarinic agonist results for NBI-'567 (Selective M1 Agonist), NBI-'569 (Selective M4 Agonist), and NBI-'570 [11]

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]

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]

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.

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]

Financial Data and Key Metrics Changes - Neurocrine Biosciences reported $545 million in product sales for INGREZZA in Q1 2025, impacted by one less order week and patient reauthorization processes [9] - The company reaffirmed its 2025 sales guidance range for INGREZZA at $2.5 billion to $2.6 billion, factoring in expected acceleration of new patient additions [10][11] - The company has approximately $1.8 billion in cash to support its commercial and clinical development strategies [12] Business Line Data and Key Metrics Changes - INGREZZA achieved record new patient starts in Q1 2025, indicating strong demand despite market challenges [10][14] - KRONESITY generated $15 million in net revenue in its first full quarter of launch, with 70% of dispensers receiving reimbursement [11][19] - The company expanded formulary coverage in Medicare Part D, increasing patient access for INGREZZA [10][16] Market Data and Key Metrics Changes - The payer environment has been influenced by the Inflation Reduction Act, affecting prior authorization processes for both new and continuing patients [16] - Approximately 40% of patients with tardive dyskinesia (TD) have been diagnosed, with less than 10% receiving standard care treatment [15] Company Strategy and Development Direction - The company aims to evolve from a single blockbuster to a multiple blockbuster neuroscience company, focusing on both near and long-term revenue growth [6] - Neurocrine is advancing its R&D portfolio, with ongoing Phase III studies for osevampatore and MBI-568, and plans to expand its muscarinic portfolio into new Phase II studies [7][23] - The leadership transition includes welcoming Dr. Sanjay Keswani as the new Chief Medical Officer [7][27] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the momentum from record new patient starts and the potential for growth in Q2 and beyond [35][36] - The company is optimistic about the early adoption of KRONESITY and expects continued growth in the pediatric and adolescent segments [19][70] Other Important Information - The company has retired 3.6 million shares over the past two quarters, reflecting a commitment to returning capital to shareholders [12] - The transition of Dr. Eiry Roberts to a strategic advisory role marks a significant change in leadership, with a focus on maintaining the momentum in the company's pipeline [27] Q&A Session Summary Question: What are the expectations for INGREZZA in Q2? - Management noted that Q1 challenges were anticipated, but record new patient starts provide confidence for Q2 growth [34][35] Question: How should KRONESITY's initial numbers be interpreted? - Management indicated that it is too early to define a trend for KRONESITY, but initial adoption has exceeded expectations [37][38] Question: What are the reimbursement dynamics for KRONESITY moving forward? - The company expects some health plans to conduct formal reviews of KRONESITY, but initial reimbursement metrics are favorable [41][44] Question: Can you provide insights on the share split between INGREZZA and Teva? - Management stated that INGREZZA continues to hold the majority share in TD prescriptions, with trends aligning with expectations [48] Question: What is the expected growth for INGREZZA in Q2? - Management anticipates a nice step up in sales due to one additional order week and record new patient starts, despite some gross to net impacts [53][54] Question: What is the timeline for KRONESITY data from ongoing studies? - Management plans to release one-year data on KRONESITY at upcoming medical meetings, with a focus on various clinical endpoints [99]