Osavampator

INGREZZA® (valbenazine) Fourth Quarter and Full Year 2024 Net Product Sales of $615 Million and $2.3 Billion, Representing Year-Over-Year Growth of 23% and 26% Respectively INGREZZA® (valbenazine) Full Year 2025 Net Product Sales Guidance of $2.5 - $2.6 Billion CRENESSITYTM (crinecerfont), a First-in-Class Treatment for Children and Adults with Classic Congenital Adrenal Hyperplasia, Approved and Launched in the United States Phase 3 Programs for Osavampator in Major Depressive Disorder and NBI-'568 in Schizophrenia Initiating in the First Half of 2025 SAN DIEGO, Feb. 6, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced its financial results for the fourth quarter and full year ended December 31, 2024 and provided financial guidance for 2025. "I'm proud of the tremendous progress we made last year with the continued growth of INGREZZA for patients living with tardive dyskinesia or Huntington disease chorea. With the approval and launch of CRENESSITY, we look forward to delivering the first new treatment for the congenital adrenal hyperplasia community in over 70 years, transforming the standard of care for patients," said Kyle W. Gano, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "With a rapidly advancing and growing pipeline and a strong financial profile, we are well positioned to build a leading neuroscience company." Financial Highlights Net Product Sales Highlights INGREZZA fourth quarter and fiscal 2024 net product sales were $615 million and $2.3 billion, respectively INGREZZA fourth quarter net product sales grew 23% compared to fourth quarter 2023, driven by strong underlying patient demand and improvement in gross-to-net dynamics CRENESSITY fourth quarter and fiscal 2024 net product sales were $2 million reflecting initial pharmacy orders following approval by the U.S. Food and Drug Administration (FDA) in December 2024 Other Key Financial Highlights Differences in fourth quarter 2024 GAAP and Non-GAAP operating expenses compared with fourth quarter 2023 were driven by: Increased R&D expense in support of an expanded and advancing portfolio including investments in osavampator in major depressive disorder, our muscarinic franchise and preclinical research and discovery activities. Increased SG&A expense includes incremental investment in CRENESSITY-related headcount, CRENESSITY-related pre-launch activities, and continued investment in INGREZZA, including the recent expansion of our psychiatry and long-term care sales teams in September 2024. Increased stock-based compensation expense (GAAP) of $28 million primarily related to performance-based awards achievement associated with CRENESSITY FDA approval and a charge associated with the retirement of our CEO in October 2024. Fourth quarter 2024 GAAP net income and earnings per share were $103 million and $1.00, respectively, compared with $148 million and $1.44, respectively, for fourth quarter 2023 Fourth quarter 2024 Non-GAAP net income and earnings per share were $173 million and $1.69, respectively, compared with $158 million and $1.54, respectively, for fourth quarter 2023 Differences in fourth quarter 2024 GAAP and Non-GAAP net income compared with fourth quarter 2023 driven by: Higher INGREZZA net sales Increased operating expenses to support expanding and advancing R&D portfolio, incremental investments for CRENESSITY pre-launch activities and INGREZZA recent sales force expansion Fourth quarter 2024 includes $66 million of stock-based compensation expense compared with $38 million for fourth quarter 2023 (Non-GAAP adjustment) Fourth quarter 2024 includes a $2 million loss from changes in fair values of equity investments compared with a $29 million gain for fourth quarter 2023 (Non-GAAP adjustment) As of December 31, 2024, repurchased and retired approximately 2.0 million shares of the Company's common stock valued at approximately $240.5 million pursuant to a previously announced $300 million accelerated share repurchase (ASR) program. The $300 million program was completed in early February 2025 repurchasing and retiring approximately 2.3 million shares. At December 31, 2024, the Company had cash, cash equivalents and marketable securities totaling approximately $1.8 billion A reconciliation of GAAP to Non-GAAP financial results can be found in Table 3 and Table 4 at the end of this news release. Recent Developments CRENESSITY was approved in December 2024 by the FDA as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients four years of age and older with classic congenital adrenal hyperplasia (CAH). Announced the initiation of the Phase 3 program for osavampator (formerly NBI-1065845 / TAK-653), a potential first-in-class AMPA positive allosteric modulator in development for patients with inadequate response to treatment of major depressive disorder (MDD). Announced amendment to strategic collaboration with Takeda to develop and commercialize osavampator. Under the amended agreement, Neurocrine will obtain exclusive rights for all indications to develop and commercialize osavampator in all territories worldwide except Japan, where Takeda will acquire exclusive rights. Under the terms of the updated agreement, each company is responsible for development costs in their respective region, and both companies are eligible to receive royalty payments. Received Centers for Medicare and Medicaid Services (CMS) notification in January that INGREZZA qualifies for the small biotech exemption under the Medicare Drug Price Negotiation Program. Announced the initiation of its Phase 1 clinical study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of investigational compound NBI-921355 in healthy adult participants. NBI-921355 is an investigational, selective inhibitor of voltage-gated sodium channels Nav1.2 and Nav1.6 and in development for the potential treatment of certain types of epilepsy. Presented subgroup analyses and data from the KINECT®-HD study showing the impact of INGREZZA capsules on emotional health and psychiatric stability in patients with chorea associated with Huntington's disease. The subgroup analysis showed consistent efficacy in reducing chorea compared to placebo across all identified subgroups, categorized by demographics and baseline assessment scores. A separate data analysis showed improvements in some aspects of emotional health with no worsening of psychiatric symptoms. Presented data from more than 300 patients diagnosed with tardive dyskinesia and treated with INGREZZA capsules. These data showed significant improvements in functional, social, emotional and health-related quality of life measures in Phase 3 and 4 studies and improvements in functional, social, independence, emotional and physical aspects of patients' lives and antipsychotic adherence in real-world practice. Full Year 2025 Financial Guidance Conference Call and Webcast Today at 4:30 PM Eastern Time Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 800-225-9448 (US) or 203-518-9708 (International) using the conference ID: NBIX. The webcast and accompanying slides can also be accessed at approximately 4:30 p.m. Eastern Time on Neurocrine Biosciences' website under Investors at . A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month. About Neurocrine Biosciences Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neuropsychiatric, neurological, and neuroendocrine disorders. The company's diverse portfolio includes U.S. FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia, endometriosis* and uterine fibroids*, as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, X (Formerly Twitter) and Facebook. (*in collaboration with AbbVie) NEUROCRINE, the NEUROCRINE BIOSCIENCES Logo, YOU DESERVE BRAVE SCIENCE, and INGREZZA are registered trademarks of Neurocrine Biosciences, Inc. CRENESSITY is a trademark of Neurocrine Biosciences, Inc. Non-GAAP Financial Measures In addition to the financial results and financial guidance that are provided in accordance with accounting principles generally accepted in the United States (GAAP), this press release also contains the following Non-GAAP financial measures: Non-GAAP R&D expense, Non-GAAP SG&A expense, and Non-GAAP net income and net income per share. When preparing the Non-GAAP financial results and guidance, the Company excludes certain GAAP items that management does not consider to be normal, including recurring cash operating expenses that might not meet the definition of unusual or non-recurring items. In particular, these Non-GAAP financial measures exclude: non-cash stock-based compensation expense, charges associated with convertible senior notes, vacated legacy campus facility costs, net of sublease income, non-cash amortization expense related to acquired intangible assets, acquisition and integration costs, changes in fair value of equity investments, changes in foreign currency exchange rates and certain adjustments to income tax expense. These Non-GAAP financial measures are provided as a complement to results provided in accordance with GAAP as management believes these Non-GAAP financial measures help indicate underlying trends in the Company's business, are important in comparing current results with prior period results and provide additional information regarding the Company's financial position. Management also uses these Non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally and to manage the Company's business and evaluate its performance. The Company provides guidance regarding combined R&D and SG&A expenses on both a GAAP and a Non-GAAP basis. A reconciliation of these GAAP financial results to Non-GAAP financial results is included in the attached financial information. Forward-Looking Statements In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements related to: the benefits to be derived from our products and product candidates; the value our products and/or our product candidates may bring to patients; the continued success of INGREZZA; successfully launching CRENESSITY; our financial and operating performance, including our future revenues, expenses, or profits; our collaborative partnerships; expected future clinical and regulatory milestones; and the timing of the initiation and/or completion of our clinical, regulatory, and other development activities and those of our collaboration partners. Factors that could cause actual results to differ materially from those stated or implied in the forward-looking statements, include but are not limited to the following: risks and uncertainties associated with Neurocrine Biosciences' business and finances in general; risks and uncertainties associated with the commercialization of INGREZZA and CRENESSITY; risks related to the development of our product candidates; risks associated with our dependence on third parties for development, manufacturing, and commercialization activities for our products and product candidates, and our ability to manage these third parties; risks that the FDA or other regulatory authorities may make adverse decisions regarding our products or product candidates; risks that development activities may not be initiated or completed on time or at all, or may be delayed for regulatory, manufacturing, or other reasons, may not be successful or replicate previous clinical trial results, may fail to demonstrate that our product candidates are safe and effective, or may not be predictive of real-world results or of results in subsequent clinical trials; risks that the potential benefits of the agreements with our collaboration partners may never be realized; risks that our products, and/or our product candidates may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks associated with government and third-party regulatory and/or policy efforts which may, among other things, impose sales and pharmaceutical pricing controls on our products or limit coverage and/or reimbursement for our products; risks associated with competition from other therapies or products, including potential generic entrants for our products; and other risks described in our periodic reports filed with the Securities and Exchange Commission, including our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than as required by law. SOURCE Neurocrine Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

▎药明康德内容团队编辑 突破RNAi疗法大脑递送瓶颈，新锐完成9700万美元B轮融资 Atalanta Therapeutics今日宣布完成9700万美元的B轮融资，以支持公司针对KCNT1相关癫痫和亨廷顿病的RNAi药物的1期临床试验。该公司独有的创新di-siRNA药物设计在临床前研究中显示出在大脑和脊髓中的广泛分布以及在大脑中的显著持久性。单次给药di-siRNA显示出6个月或更长时间的基因沉默潜力，这可能在临床环境中允许每年一次或两次给药。 Atalanta Therapeutics的研发管线中包含多款治疗中枢神经系统疾病的RNAi疗法。其中，ATL-201是用于治疗KCNT1相关癫痫的在研疗法。KCNT1相关癫痫是一种由KCNT1基因出现功能获得性突变引起的早发性癫痫。患儿通常会经历严重且频繁的癫痫发作，现有的抗癫痫药物无法有效控制，并且常常伴有发育迟缓和智力障碍。ATL-201旨在降低KCNT1水平并正常化神经元的兴奋性。临床前研究表明，ATL-201能够显著减少癫痫发作，并改善行为，且具有良好的持久性和耐受性。 ▲Atalanta Therapeutics的di-siRNA设计改善在大脑中的药物分布（图片来源：Atalanta Therapeutics公司官网） 另一款候选药物ATL-101旨在通过沉默HTT基因来治疗亨廷顿病。亨廷顿病是一种进行性神经退行性疾病，由HTT基因中特定序列扩增引起，导致患者的身体、认知和精神能力逐渐恶化。临床前研究表明，单次给药的ATL-101能够在深部脑区显著降低HTT基因表达，效果可持续六个月，并且具有出色的耐受性。 开发潜在“first-in-class”减肥疗法，新锐完成6500万美元A轮融资 日前，Helicore Biopharma公司宣布走出隐匿模式，并完成6500万美元A轮融资。本轮融资由Versant Ventures和OrbiMed共同领投，Longitude Capital和Wellington Management也参与了本轮融资。 Helicore公司聚焦于开发潜在“first-in-class”葡萄糖依赖性促胰岛素多肽（GIP）拮抗剂，用于治疗肥胖症和相关健康问题。其研发进度最快的候选疗法HCR-188是一款处于临床开发阶段的抗GIP单克隆抗体。该公司的研发管线中还包括靶向GIP的抗体偶联药物，GLP-1药物以及组合疗法，旨在针对特定肥胖患者亚群。 Helicore公司的研发策略借助从人类遗传学、专有临床前数据、以及第三方临床研究获得的洞见。携带GIP或GIP受体失活性突变的个体可能因为脂肪细胞中GIP信号通路介导的甘油三酯摄取降低而不易出现肥胖。HCR-188与GLP-1药物联用，在临床前研究中显示可让动物优先减少脂肪而不是瘦体质量（lean mass）。与GLP-1药物相比，可能带来更高质量的体重减轻。 ▲Helicore Biopharma公司研发管线（图片来源：Helicore Biopharma公司官网） 此外，通过与GIP在外周结合，HCR-188可能阻断GIP进入中枢系统，防止大脑中的GIP信号传导，潜在恢复瘦素（leptin）敏感性，在降低食物摄入和脂肪积累的同时，支持饱足感提高。抑制GIP还具有心血管代谢益处，特别是在与GLP-1药物联用的情况下。 HCR-188的首个人体临床试验预计在2025年下半年获得顶线数据。Helicore的研发活动得到Versant发现引擎Ridgeline在蛋白工程学方面的支持。 潜在“first-in-class”抑郁症疗法挺进3期临床 Neurocrine Biosciences公司日前宣布，启动注册性3期临床试验，评估在研疗法osavampator（此前名为NBI-1065845）作为抗抑郁药物的辅助疗法，治疗抑郁症的疗效和安全性。该公司还宣布更新了与武田（Takeda）公司合作开发osavampator的研发协议，获得osavampator在日本以外全球其它地区的独家开发权益。 Osavampator是一款潜在“first-in-class”的AMPA受体正向别构调节剂（PAM）。2024年公布的2期临床试验SAVITRI的顶线数据显示，接受osavampator治疗的患者在第28天（主要终点）和第56天（次要终点）的蒙哥马利-阿斯伯格抑郁评分量表（MADRS）总分上均显示出统计学上显著的改善。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Neurocrine Biosciences Announces Initiation of Phase 3 Registrational Program for Osavampator as an Adjunctive Therapy for the Treatment of Major Depressive Disorder in Adults. Retrieved January 29, 2025, from https://neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-announces-initiation-phase-3 [2] Neurocrine Biosciences Announces Amendment to Strategic Collaboration with Takeda to Develop and Commercialize Osavampator (formerly NBI-1065845/TAK-653). Retrieved January 29, 2025, from https://neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-announces-amendment-strategic [3] Atalanta Therapeutics Closes Oversubscribed $97 Million Series B Financing to Advance Two RNAi Therapies for CNS Diseases to Clinical Trials. Retrieved January 29, 2025, from https://www.businesswire.com/news/home/20250128583264/en/Atalanta-Therapeutics-Closes-Oversubscribed-97-Million-Series-B-Financing-to-Advance-Two-RNAi-Therapies-for-CNS-Diseases-to-Clinical-Trials [4] Helicore Biopharma Emerges from Stealth to Continue Advancing Portfolio of First-in-Class GIP Antagonists for Obesity and Related Conditions. Retrieved January 28, 2025, from https://www.globenewswire.com/news-release/2025/01/28/3016188/0/en/Helicore-Biopharma-Emerges-from-Stealth-to-Continue-Advancing-Portfolio-of-First-in-Class-GIP-Antagonists-for-Obesity-and-Related-Conditions.html 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新