Neurocrine Biosciences公司推出WHAT THE C@H?!教育计划 - 旨在为先天性肾上腺增生症（CAH）社区提供有用信息[2] - 提供支持性和教育性的网站，让CAH患者和家长分享经验[5] - 进行了大量采访和市场研究，以制定教育计划，支持CAH社区[6] 先天性肾上腺增生症（CAH）介绍 - CAH是一种罕见的遗传疾病，导致酶缺乏改变肾上腺激素的产生[4] - CAH可能导致生命威胁性的肾上腺危机和雄激素过多[4]

For the transition period from __________to__________ Commission file number 0-22705 NEUROCRINE BIOSCIENCES, INC. (Exact name of registrant as specified in its charter) UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Delaware 33-0525145 (State or other jur ...

财务数据和关键指标变化 - 2023年Ingrezza销售额近18.4亿美元,同比增长超过4亿美元,增长近30% [16] - 2024年Ingrezza销售额预计为21亿-22亿美元,同比增长超过17% [19] - 2023年公司现金流超过6亿美元,主要受益于非GAAP净收益,但部分被175亿美元的业务发展投资所抵消 [17] 各条业务线数据和关键指标变化 - Ingrezza在精神病科、神经科和长期护理三大领域均实现了强劲增长 [29] - 尽管Ingrezza在TD和HD适应症的渗透率不断提高,但仍有大约三分之二的600,000名TD患者未被诊断,以及80%的20,000名中重度HD患者未接受VMAT2抑制剂治疗 [27] 各个市场数据和关键指标变化 - 尽管Ingrezza在TD和HD适应症的渗透率不断提高,但仍有大约三分之二的600,000名TD患者未被诊断,以及80%的20,000名中重度HD患者未接受VMAT2抑制剂治疗 [27] - Ingrezza的处方获得率超过80%,患者自付费用平均不到10美元,获得率和可及性保持很高 [30] 公司战略和发展方向及行业竞争 - 公司将继续投资Ingrezza销售团队和DTC营销,以提高患者诊断和治疗率 [195][196] - 公司正在为2025年推出crinecerfont做准备,包括开展广泛的教育培训,为CAH患者群体建立新的治疗范式 [41][44] - 公司内部研发管线不断丰富,未来几年将有更多小分子和生物大分子进入临床 [197] 管理层对经营环境和未来前景的评论 - 2023年和2022年都是公司发展历程中难得一遇的成功年份,业务表现出色 [11][24] - 公司有信心2024年也会取得同样出色的业绩 [17][18] - 公司对Ingrezza未来的增长前景充满信心,认为这个市场仍有巨大的未开发潜力 [195][196] - 公司对crinecerfont的上市前准备工作充满信心,认为这将为CAH患者群体带来新的治疗范式 [41][44] 问答环节重要的提问和回答 问题1 **Paul Matteis 提问** 对于即将在今年下半年读出的M4激动剂NBI-568的临床试验,公司如何选择剂量水平,对于达到统计学显著性和良好耐受性有何预期? [56][57] **Eiry Roberts 回答** 这是一项剂量探索性试验,公司根据前期临床前数据选择了多个剂量水平进行评估。公司期望看到与之前同类产品类似的疗效,同时也关注耐受性的差异。这个试验的目标不是单个剂量组的统计学显著性,而是综合评估疗效和安全性,为后续发展提供依据。 [58] 问题2 **Tazeen Ahmad 提问** 公司对Ingrezza在HD适应症的销售贡献有何预期?同时,为什么过去多年来Xenazine在该适应症市场渗透率较低? [61][62][63] **Eric Benevich 回答** HD适应症对Ingrezza销售的贡献相对较小,主要是因为这是一个罕见病适应症,患者群体较小。过去Xenazine在该市场渗透率较低,主要是由于其复杂的剂量滴定和潜在的不良反应担忧,以及一定的自付费用,而Ingrezza相比具有更简单的给药方案、良好的耐受性以及较低的自付费用。 [63][64][65] 问题3 **Brian Skorney 提问** 公司在2024年为crinecerfont准备工作的开支约为5000万美元,请问这是否为全部的启动成本,以及公司对CAH市场的渗透节奏有何预期? [70][71][73][74] **Eric Benevich 回答** 2024年5000万美元的投入主要用于教育培训等前期准备工作,并非crinecerfont全部的启动成本。对于CAH市场的渗透,公司计划在2025年正式上市前,先通过医疗团队和销售团队开展广泛的疾病教育,提高CAH患者群体和医生对新治疗方案的认知,为顺利推广奠定基础。 [73][74]

Brian Abrahams Second, what weÂ're doing, as you can see and as weÂ'll be discussing at some length in our R&D Day is the investment in ourselves from a research standpoint. And thatÂ's going to be our transition of a company into multiple therapeutic modalities in the areas that we concentrate on, which is neuroscience, neuropsychiatry, neuroendocrinology and at some point in the very near future, neuroimmunology. If you think about the dynamics that I described earlier, with only about 20% of the eligible ...

Open-Label Treatment (one year) OpenLabel Extension* Participants randomized to placebo will receive crinecerfont during open-label treatment Objective Evaluate the efficacy, safety and tolerability of crinecerfont in adults with classic CAH Crinecerfont is investigational and not approved in any country *The duration of participation in the study is approximately 20 months for the core study and will be a variable amount of time per participant for the open-label extension CAHtalystTM Adult and Pediatric S ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 0-22705 NEUROCRINE BIOSCIENCES, INC. (Exact name of registrant as specified in its charter) Delaware (State or other jurisdicti ...

Advancing Life-Changing Discoveries in Neuroscience Q2 2023 Corporate Presentation August 1, 2023 Nasdaq: NBIX Safe ...

Company Participants Conference Call Participants Good day, everyone, and welcome to today's Neurocrine Biosciences Reports Second Quarter Results. [Operator Instructions] It is now my pleasure to turn today's program over to Todd Tushla, Vice President of Investor Relations. During today's call, we will be making forward-looking statements. These statements are subject to certain risks and uncertainties, and our actual results may differ materially. I encourage you to review the risk factors discussed in o ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 0-22705 NEUROCRINE BIOSCIENCES, INC. (Exact name of registrant as specified in its charter) Delaware (State or other jurisdiction of ...

Great. Thanks for squeezing me in. With regards to your -- the performance of INGREZZA in the quarter was pretty strong and sort of leads me to think about the growth you see in the upcoming quarters and the growth that's baked into guidance. Can you comment on how you see that progressing? And at what point would you consider reevaluating your guidance there? And then secondly, with regards to Huntington's disease chorea, how quickly do you given the awareness level amongst physicians that do treat TD pati ...