Huntington Disease

SOMERVILLE, Mass., Oct. 30, 2024 /PRNewswire/ -- Aitia, a biotech company pioneering the use of causal AI and Digital Twins in drug discovery and development, is expanding its collaboration with Servier, a global and independent pharmaceutical company, to tackle gliomas, a broad group of brain cancers. This collaboration aims to use Aitia's breakthrough technology which is powered by millions of data points from patient cancer tissues to find new treatments for this deadly disease. Under the terms of the collaboration, Aitia and Servier will work together to discover and, validate novel drug targets and develop novel drug candidates for glioma using Aitia's Gemini Digital Twins and Servier's therapeutic discovery and clinical development expertise in oncology. This collaboration will include exploration of the heterogeneous biology of these brain cancers, mechanisms of resistance, and drug response at the individual patient level. In a recent breakthrough in another form of glioma, specifically Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery including biopsy, sub-total resection, or gross total resection in adult and pediatric patients 12 years and older, Servier recently announced approval of the brain cancer drug, VORANIGO, the first approved drug for this disease area in almost 25 years. Gliomas, tumors that originate in the brain or spinal cord from glial cells, affects more than 190,000 people worldwide annually. The most aggressive form of glioma, glioblastoma, has an average survival time after diagnosis of approximately 15 months, with less than 7% of patients surviving beyond five years, according to the National Brain Tumor Society. Despite standard treatments like surgery, radiation and chemotherapy, glioblastoma has a high rate of recurrence and there are currently no curative therapies. "We are excited to expand our collaboration with Aitia into gliomas as we continue to prioritize brain cancer after our recent positive results and approval of VORANIGO in the US and in growing number of territories," said Fabien Schmidlin, Global Head of Translational Medicine at Servier. "At Servier, we are committed to advancing therapies for cancer patients with high unmet medical needs, and we believe that integrating Aitia's Gemini Digital Twins with our expertise in oncology, particularly in targeting molecular mutations, could open new avenues for more effective treatments." "Glioma is the among the deadliest forms of cancer, and the lack of broadly effective treatments highlights the urgent need for new approaches that leverage the growth of genetic and multi-omic patient data and breakthroughs in AI," said Colin Hill, CEO and co-founder of Aitia. "We believe that our Gemini Digital Twins, along with Servier's drug discovery and clinical development expertise in hard-to-treat cancers can lead to new breakthrough therapies for this disease." This collaboration represents the fourth area of research Aitia and Servier are collaborating on. The companies began their partnership in 2022, to advance drug discovery, translational, and clinical development efforts in Multiple Myeloma, then expanding in 2023 to include drug discovery and development for pancreatic cancer, and again in January 2024 to discover patient responders for Servier's LRRK2 inhibitor in Parkinson's disease. Aitia's approach centers on two key innovations: causal AI and Gemini Digital Twins. Causal AI goes beyond correlative associations to identify which genetic or molecular changes drive diseases and patient response to therapies. Gemini Digital Twins are virtual models that are reverse-engineered from huge quantities of genetic, multi-omic, and clinical data from patients that reveal how genes, proteins, and other molecules interact within cells and tissues to drive clinical outcomes. These Gemini Digital Twins of a specific disease allow researchers to rapidly test billions of experiments such as gene and protein knockdowns or application of a drug candidate in a human patient context versus animal models or cells in petri dishes. These high-throughput "virtual experiments" in patient-derived Gemini Digital Twins leads to discovering and selecting drug candidates that are more likely to succeed in clinical trials in specific patient populations. About Aitia Aitia is an AI-enabled biotechnology company deploying causal AI and creating Gemini Digital Twins to discover the next generation of breakthrough drugs in neurodegenerative diseases and oncology, including a lead program in Huntington's disease. By leveraging the convergence of multi-omic patient data, high-performance computing, and causal AI, Aitia's Gemini Digital Twins reveal the hidden biological mechanisms of disease to transform the discovery and development of new therapeutics. To date, Gemini Digital Twins have been used to accelerate R&D in oncology, neurodegeneration, cardiometabolic, and immunology. Media Contact: Isabelle Caffry Senior Director, Strategy & Corporate Development [email protected] +1-617-374-2300 Ryan Flinn In Like Flinn Communications [email protected] +1 510-207-7616 SOURCE Aitia WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

INGREZZA® (valbenazine) Third Quarter Net Product Sales of $613 Million Representing 26% Year-Over-Year Growth INGREZZA ® (valbenazine) 2024 Net Product Sales Guidance Raised to $2.30 - $2.32 Billion Board Authorizes $300 Million Share Repurchase Plan SAN DIEGO, Oct. 30, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced its financial results for the third quarter ended September 30, 2024 and provided an update on its 2024 financial guidance. "With continued INGREZZA growth across the tardive dyskinesia and Huntington's disease chorea indications, FDA Priority Review for crinecerfont in congenital adrenal hyperplasia, a deep neuroscience focused pipeline and a strong balance sheet, we are confident in our ability to help more patients than ever before," said Kyle W. Gano, Ph.D., Chief Executive Officer of Neurocrine Biosciences. William Rastetter, Chairman of the Board of Directors of Neurocrine Biosciences, said, "The share repurchase authorization reflects the Board's confidence in Neurocrine's significant value creation potential. Importantly, the new share repurchase authorization preserves our flexibility to drive sustained growth through investments in INGREZZA and the anticipated launch of crinecerfont, while also advancing our diverse pipeline and maintaining our strong balance sheet." Financial Highlights INGREZZA Net Product Sales Highlights INGREZZA third quarter 2024 net product sales were $613 million and grew 26% compared to the third quarter 2023 Year-over-year growth driven by strong underlying patient demand and improvement in gross-to-net dynamics Other Key Financial Highlights Differences in third quarter 2024 GAAP and Non-GAAP operating expenses compared with third quarter 2023 were driven by: Increased R&D expense in support of an expanded and advancing portfolio including investments in muscarinic compounds, gene therapy programs, and second generation VMAT2 inhibitors. Third quarter 2024 R&D expense includes $39 million for development milestones achieved under collaborations with Nxera Pharma UK Limited (Nxera, formerly known as Sosei Heptares) and Voyager Therapeutics, Inc. (Voyager). Increased SG&A expense includes incremental investment in crinecerfont-related headcount, crinecerfont-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. Third quarter 2024 GAAP net income and earnings per share were $130 million and $1.24, respectively, compared with $83 million and $0.82, respectively, for third quarter 2023 Third quarter 2024 Non-GAAP net income and earnings per share were $189 million and $1.81, respectively, compared with $156 million and $1.54, respectively, for third quarter 2023 Differences in third quarter 2024 GAAP and Non-GAAP net income compared with third quarter 2023 driven by: Higher INGREZZA net sales and improved operating margin Third quarter 2024 includes $17 million loss from changes in fair values of equity investments compared with $40 million loss for third quarter 2023 (Non-GAAP adjustment) Third quarter 2024 includes $39 million of expense for development milestones achieved under collaborations with Nxera and Voyager At September 30, 2024, the Company had cash, cash equivalents and marketable securities totaling approximately $1.9 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 Kyle W. Gano, Ph.D. appointed Chief Executive Officer effective October 11, 2024. Announced the Company's Board of Directors has authorized a $300 million share repurchase plan. The Company subsequently intends to enter into a $300 million accelerated share repurchase transaction in the coming days, subject to market conditions, which will constitute the entirety of the authorized share repurchase plan. Announced positive topline data for the Phase 2 study of NBI-1117568, a first-in-class, orally active, highly selective investigational M4 agonist, in development as a potential treatment for schizophrenia. The successful completion of the Phase 2 study triggered a $35 million milestone payment to Nxera in the third quarter of 2024. We expect to advance NBI-1117568 into Phase 3 development in the first half of 2025, which would trigger an additional $15 million milestone payment to Nxera upon initiation of the Phase 3 study. Presented KINECT ®-HD2 interim data at the 2024 MDS International Congress of Parkinson's Disease and Movement Disorders demonstrating robust and sustained improvements in chorea associated with Huntington's Disease through week 104 irrespective of antipsychotic use. Announced the ERUDITE™ Phase 2 study of luvadaxistat (NBI-1065844) in cognitive impairment associated with schizophrenia (CIAS) did not meet its primary endpoint. In addition, we provided Takeda Pharmaceutical Company Limited with written notice of termination of the license agreement to develop and commercialize luvadaxistat and NBI-1065846. The termination is anticipated to be effective in April 2025. Provided Idorsia Pharmaceuticals Ltd. with written notice of termination of the license agreement to develop and commercialize NBI-827104 in epileptic encephalopathy with continuous spike and wave during sleep. The termination is anticipated to be effective in January 2025. Raised 2024 Net Sales Guidance and Updated Expense Guidance Conference Call and Webcast Today at 8:00 AM Eastern Time Neurocrine Biosciences will hold a live conference call and webcast today at 8:00 a.m. Eastern Time (5:00 a.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 8:00 a.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, but few options. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine, and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, 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 BIOSCIENCES, NEUROCRINE and YOU DESERVE BRAVE SCIENCE are registered trademarks of Neurocrine Biosciences, Inc. The Neurocrine logo 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 security 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; our financial and operating performance, including our future revenues, expenses, or profits; our collaborative partnerships; expected future clinical and regulatory milestones; the timing of the initiation and/or completion of our clinical, regulatory, and other development activities and those of our collaboration partners; and our intention to enter into an accelerated share repurchase transaction, including the expected dollar amounts and the timing of the transaction. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are: our future financial and operating performance; risks and uncertainties associated with the commercialization of INGREZZA; risks that the crinecerfont New Drug Applications (NDAs) may not obtain regulatory approval, such approval may be delayed, or may not receive the benefits associated with priority review; 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 clinical 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; constraints, volatility, or disruptions in the capital markets or other factors affecting our ability to enter into or complete an accelerated share repurchase transaction; and other risks described in our periodic reports filed with the SEC, 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

以下内容转自“澎湃新闻”。 “从2018年到现在，在六年的时间里，我们已经有6种品牌药以及1款健康消费品进入中国市场。实实在在看到了中国日益多元的市场需求。”第七届中国国际进口博览会即将开幕，梯瓦大中华区总经理黄迪仁在接受采访时表示，梯瓦将继续深化“在中国，为中国”战略，希望能把更多的优质医药产品带给中国患者。 近120多年历史的梯瓦，是全球领先的生物医药企业，因其在仿制药领域上的强劲实力，而被外界熟知。发展至今，梯瓦拥有全面多元的产品线，覆盖仿制药、创新药、生物类似药、非处方药等领域。2018年，梯瓦进入中国，用百年历史积淀形成的多元化产品线，为中国市场提供着覆盖中枢神经系统、肿瘤、皮肤管理、抗感染等领域产品资源。 黄迪仁提到，梯瓦受益于进博会的平台优势，已然成为中国市场的深耕者。今年进博会，梯瓦将带来两款中国“首展”、“首秀”的产品，并将进一步深化和拓展与本土伙伴的合作，携手推动医疗行业的高质量发展。 01 赋能健康未来 进博老友再上新 第七届进博会，梯瓦以全新主题“全力以赴，共筑健康未来”参展。 黄迪仁表示，“‘全力以赴，共筑健康未来’是梯瓦在去年发布的全新企业使命，代表着梯瓦创立120多年来的不变初心，和对于改善各国人民健康的坚定决心。以全新主题参展，不仅是对梯瓦企业使命的重申，也展现了我们对中国市场的长期承诺，以及全面助力健康中国2030实现的决心。” 对于很多跨国药企来说，进博会可以让大众了解到他们的最新药品以及能为中国患者提供的价值。在过去两年的进博旅程中，梯瓦不断展示着医药产品方面的深厚积淀，多款产品受益于不断释放的溢出效应，更快、更深入地为中国患者带来福音。 其中，创新药安泰坦®（氘丁苯那嗪片）是一个生动的例子。作为中国首个获批的氘代药物，安泰坦®在治疗成人与亨廷顿病有关的舞蹈病（Huntington's Disease, HD）及迟发性运动障碍（Tardive Dyskinesia, TD）方面表现出色，填补了相关治疗领域的空白。2020年，它被纳入医保目录，提升患者治疗的可负担性。 在2022年的进博会上，安泰坦®和罕见病亨廷顿病都受到了更广泛的关注。而在2023年，安泰坦®更是成功续约医保目录，长期降低了患者及其家庭的经济负担。到了2024年，梯瓦与江苏恩华药业股份有限公司就安泰坦®达成了在华经销协议，进一步扩大了患者的可及性。可以说，安泰坦®之所以每年都能传来好消息，离不开进博会溢出效应的助力。 今年进博会，安泰坦®缓释片剂Austedo XR®将在中国“首秀”，会进一步提高患者的依从性。此外，梯瓦外用药物密丝高（5%米诺地尔溶液）也将迎来中国“首展”，将丰富中国脱发患者的治疗选择。 依托进博会这一重要桥梁，梯瓦还不断探索数字化生态机会及深化本土合作的机遇。去年，梯瓦积极推动罕见病的治疗信息普及工作，与多方合作提升大众对疾病的认知。通过与阿里、京东、腾讯等平台合作，且联动专家学者完善了HD罕见病地图，上线了TD及HD疾病医典词条，让患者在查询相关信息时得到权威的了解，同时与京东健康开展了提升药物可及性方面的合作。 今年进博会期间，梯瓦还将举行一系列合作签约仪式和医学论坛活动，不断扩大本土“朋友圈”，共同推动行业的高质量发展，打造创新生态。 02 助力健康中国 释放医药“向新力” “进博会强大的溢出效应远超我们的预期。进博会的强‘磁吸力’让梯瓦能够和全球同行、本土伙伴等深入交流，对于我们深化在华发展具有强大助益。”黄迪仁说，“每年赴约进博会代表着梯瓦重视和深耕中国市场的诚意和决心。” 进入中国以来，梯瓦的中国战略是基于对市场需求的洞察而确定的。过去六年，梯瓦基于市场需求以创新特药为切口进入中国市场，为患者提供创新治疗方案，在中国市场上逐步建立起自己的竞争优势。 创新被梯瓦视为是高质量发展的动力之源。作为百年企业，创新其实根植于梯瓦的品牌基因之中。“梯瓦的仿制药业务备受行业和大众的认可，但大家可能不太了解的是，从1990年代开始，梯瓦已经开始加大全球创新布局，聚焦国际前沿创新领域。例如在帕金森疾病、复发型多发性硬化疾病等方面，我们研发了很多领先且有突破性的治疗方案，为患者带来治疗新选择。” 与此同时，梯瓦一直关注患者所需，助力健康中国发展。近期，进口原研药品的可及性这一问题受到了广泛关注。为了满足中国患者对原研药的需求，今年9月，梯瓦携手海南华蓝医药健康产业有限公司在国内市场上市了舒美特®（阿奇霉素片）。 随着更多创新药物的引进和研发成果的落地，梯瓦希望在中国市场上实现更大的突破和发展。黄迪仁表示，未来，梯瓦将在其长期深耕的中枢神经系统治疗领域向中国加速引进多款创新药。例如，梯瓦计划于2028年引进治疗成人精神分裂症的改良型新药——奥氮平长效针剂，将增加患者治疗的便利度，提升患者的依从性，进一步满足中国患者的健康需求。 03 聚焦创新 深化“在中国，为中国”战略 梯瓦进入中国的六年，正是中国医药行业进入高质量发展新阶段的时间。正如黄迪仁所言，真切感受到了进入中国后的每一步都是机遇。 2018年，梯瓦的肿瘤用药存达®（注射用盐酸苯达莫司汀）国家药监局的批准，这是梯瓦在中国市场迈出的坚实一步。仅仅两年后，治疗舞蹈病及成人迟发性运动障碍的创新药安泰坦®便在华获批，同年更是纳入国家医保目录，极大地提升了药物的可及性，让更多患者受益。随后，梯瓦在进博会的助力下，成功获批神经系统罕见病药物固派松®（醋酸格拉替雷注射液），进一步丰富了产品线。 去年，偏头痛创新药物AJOVY®（瑞玛奈珠单抗）在粤港澳大湾区的落地，得益于“港澳药械通”政策的支持，再次展现了梯瓦在中国市场的敏锐洞察力和强大执行力。对梯瓦而言，除了业务增长和营收提升，最欣慰的是在中国服务患者数量的增加。面对不断变化的市场环境和国家政策的调整，如何在这样一个充满机遇的市场与时俱进，是梯瓦愿意持续探索的问题。 去年，梯瓦提出“Pivot to Growth”全球战略，目的在于“聚焦创新，重启增长之路”。黄迪仁表示，“‘Pivot to Growth’全球战略的推出，是梯瓦主动顺应市场变革，持续巩固市场地位的必然选择。” 值得关注的是，“Pivot to Growth”战略中“创新”不仅体现在创新药业务布局上，还体现在结构、合作方式的创新布局。这一战略由四大增长支柱——发挥增长引擎潜力、夯实创新能力、巩固优势地位、聚焦核心业务，共同构成了梯瓦在中国市场的未来发展蓝图。 作为梯瓦三大国际市场中增长最快的地区之一，梯瓦大中华区将沿袭“Pivot to Growth”战略。黄迪仁强调，“我们注重的是行稳致远的长期效果，希望将更多优质药品及消费品引入中国，并不断提高药品可及性和可负担性，帮助更多患者改善健康状况。”在梯瓦看来，中国医药行业始终展示着巨大的发展潜力。与此同时，日益增长的新型消费趋势也促使梯瓦将好口碑的消费品逐个引入中国市场。 去年，梯瓦引进中国首个健康消费品——Sudocrem®（舒多可蕾）。作为一款源自爱尔兰的经典护臀霜，Sudocrem®畅销全球40多个国家。自去年在京东、天猫海外旗舰店上线以来，该产品的销量与口碑并驾齐驱。在本届进博会上，梯瓦将进一步与浙江英特集团深化合作，扩大该产品的可及性。这是梯瓦在中国多元化战略布局的实施，也是梯瓦深入洞察中国健康消费新趋势和前沿风向的快速落地。 作为一家在华业务迅速增长的中小型跨国药企，梯瓦除了加速全球优质产品的引入，还不断探索最适合自身的发展模式。黄迪仁介绍，梯瓦目前在中国展开了合作销售模式、授权许可模式等多元合作模式，借此来深耕产品与渠道。此外，在团队打造上，旨在构建一个高效敏捷的扁平化组织结构，为业务发展提供了有力保障。  “ 对于未来，黄迪仁表示 “梯瓦将会把‘Pivot to Growth’的全球战略与中国处境结合起来，让战略本土化。一方面，梯瓦将会加快创新药物的引进和产品管线布局，丰富患者治疗用药的选择；另一方面，梯瓦将积极与本土企业构建合作关系，加速在企业中国的本土化进展以及影响力。” 审批编号：AUST-CN-00479