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Advancing the Alector Brain Carrier (ABC) blood-brain barrier platform across antibodies, enzymes, and siRNA, with AL137 advancing as Alector’s lead ABC-enabled anti-amyloid beta (Aβ) antibody program for Alzheimer’s disease (AD). Continued progress across AL064/AL164 (ABC-enabled Tau siRNA for AD and other tauopathies) and AL050 (ABC-enabled GCase Enzyme Replacement Therapy for Parkinson’s disease (PD)). $172.8 million in cash, cash equivalents and investments provide runway at least through 2027 SOUTH SAN FRANCISCO, Calif., Aug. 06, 2026 (GLOBE NEWSWIRE) -- Alector, Inc. (Nasdaq: ALEC), a biotechnology company focused on developing therapies to counteract the devastating progression of neurodegeneration, today reported second quarter 2026 financial results and recent portfolio and business updates. As of June 30, 2026, Alector’s cash, cash equivalents, and investments totaled $172.8 million. "We continue to make steady progress across our ABC-enabled preclinical and research pipeline,” said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. “Our work across antibodies, enzymes, and siRNA reflects the versatility of the ABC platform and our strategy to apply it to programs where enhanced brain delivery may address important limitations in the treatment of neurodegenerative diseases. We remain focused on disciplined execution as we advance our portfolio toward clinical development, with the goal of addressing important unmet needs for patients living with neurodegenerative diseases.” Recent Program Updates Alector Brain Carrier (ABC): Preclinical and Research Pipeline Alector’s proprietary Alector Brain Carrier (ABC) blood-brain barrier platform is central to the company’s strategy to enhance therapeutic delivery to the brain. ABC is designed to achieve efficient, enhanced brain delivery through differentiated binding to a distinct region of the transferrin receptor (TfR) and to enable peripheral dosing, while preserving a favorable safety profile. It is adaptable across multiple drug modalities, including antibodies, enzymes, and siRNA. Core to the platform's design are the principles of versatility, translatability, and differentiated binding. Preclinical studies across multiple ABC-enabled programs have shown robust brain penetration, supporting the advancement of a broad pipeline directed at the underlying drivers of neurodegenerative diseases. AL137 Following successful completion of IND-enabling studies evaluating both subcutaneous (SC) and intravenous (IV) administration, Alector selected AL137 as the lead molecule for its anti-amyloid beta (Aβ) antibody program for AD, with AL037 retained as a backup candidate. The company is targeting an IND submission in Q1 2027 and first-in-human dosing in Australia no later than April 2027. AL137 combines Alector’s proprietary high-affinity fully human anti-Aβ antibody which selectively binds pyroglutamate-3 (PyroGlu3-Aβ), a validated and pathogenic form of Aβ enriched in amyloid plaques, with the company’s proprietary ABC platform. Based on preclinical studies, AL137 is designed to be delivered SC and to combine several differentiated attributes intended to optimize efficacy, safety, convenience, and manufacturability, including: High brain exposure and efficient brain delivery through the ABC platform, supporting low projected efficacious dose. Preserved full IgG1 Fc effector function, designed to maximize recruitment of brain immune cells for efficient amyloid plaque clearance. A proprietary transferrin receptor (TfR)-binding epitope designed to maintain brain delivery while reducing hematologic adverse effects associated with targeting TfR with a full Fc effector function. A favorable preclinical safety profile to support the planned clinical study, with no observed adverse effects and only transient, reversible, non-adverse hematological findings with SC dosing. Supportive pharmacokinetic and formulation characteristics intended to enable convenient monthly subcutaneous administration using a high-concentration formulation. High-productivity manufacturability, with an identified stable, high-expressing cell line designed to support efficient large-scale manufacturing and commercial scalability. High brain exposure and efficient brain delivery through the ABC platform, supporting low projected efficacious dose. Preserved full IgG1 Fc effector function, designed to maximize recruitment of brain immune cells for efficient amyloid plaque clearance. A proprietary transferrin receptor (TfR)-binding epitope designed to maintain brain delivery while reducing hematologic adverse effects associated with targeting TfR with a full Fc effector function. A favorable preclinical safety profile to support the planned clinical study, with no observed adverse effects and only transient, reversible, non-adverse hematological findings with SC dosing. Supportive pharmacokinetic and formulation characteristics intended to enable convenient monthly subcutaneous administration using a high-concentration formulation. High-productivity manufacturability, with an identified stable, high-expressing cell line designed to support efficient large-scale manufacturing and commercial scalability. ABC-Enabled siRNA Platform Alector continues to advance its ABC-enabled siRNA platform, designed for peripheral dosing and offering the potential for more convenient administration compared with traditional intrathecal delivery, as well as the potential for homogeneous drug distribution throughout the brain. AL064/AL164 Alector’s lead siRNA program, AL064/AL164, is a tau siRNA program for AD and other tauopathies and aims to reduce toxic tau protein expression and slowing of cognitive decline in AD. AL064 demonstrated robust tau mRNA knockdown and durable reduction of phospho-Tau 217 in non-human primate brains. AL064 was modified to incorporate a well-validated chemical modification intended to further optimize siRNA stability, and this modified form is advancing into IND-enabling studies as AL164. Early-Stage siRNA Programs Earlier-stage siRNA programs advancing toward lead candidate selection include ADP062-ABC, an alpha-synuclein siRNA for PD, and ADP065-ABC, an NLRP3 siRNA for multiple neurodegenerative conditions, reflecting the broad applicability of the ABC platform across disease mechanisms. Alector continues to evaluate development plans and timing for its ABC-enabled siRNA programs. AL050 AL050, Alector’s ABC-enabled engineered glucocerebrosidase (GCase) enzyme replacement therapy (ERT), is in preclinical development for PD. AL050 is engineered to overcome the central challenges of delivering enzyme therapy to the brain, combining an engineered GCase with enhanced activity and stability, a silenced effector function for safety, and Alector's tunable ABC technology. To date, preclinical data have demonstrated increased GCase activity in both rodents and NHPs and reduced toxic substrate accumulation in a rodent GBA disease model with no apparent hematologic findings, supporting continued development as a potential therapy for PD and Lewy body dementia (LBD) associated with GBA loss-of-function mutations and subsequently for idiopathic PD and LBD. Second Quarter 2026 Financial Results Revenue. Collaboration revenue for the quarter ended June 30, 2026, was $3.3 million, compared to $7.9 million for the same period in 2025. The decrease was primarily due to lower revenue recognized for the AL101 AD program. R&D Expenses. Total research and development expenses for the quarter ended June 30, 2026, were $19.5 million, compared to $27.6 million for the quarter ended June 30, 2025. The decrease was mainly due to a decrease in personnel-related costs as a result of the reductions in force as well as a decrease in facilities and other expenses. G&A Expenses. Total general and administrative expenses for the quarter ended June 30, 2026, were $8.3 million, compared to $14.4 million for the quarter ended June 30, 2025. The decrease was mainly driven by a decrease in personnel-related costs as a result of the reductions in force. Net Loss. For the quarter ended June 30, 2026, Alector reported a net loss of $23.0 million, or $0.21 net loss per share, compared to a net loss of $30.5 million, or $0.30 net loss per share, for the same period in 2025. Cash Position. Cash, cash equivalents, and investments were $172.8 million as of June 30, 2026. Management anticipates that this will be sufficient to fund Alector’s operations at least through 2027. About Alector Alector is a biotechnology company focused on developing therapies to counteract the devastating progression of neurodegenerative diseases. Leveraging the principles of genetics, immunology, and neuroscience, the company is advancing a portfolio of programs that aim to remove toxic proteins, replace missing proteins, and restore immune and nerve cell function. Supported by biomarkers, Alector’s product candidates seek to treat a range of indications, such as Alzheimer’s disease and Parkinson's disease. The company is also developing Alector Brain Carrier (ABC), a proprietary blood-brain barrier platform, which is being applied to its preclinical and research pipeline. ABC aims to enhance the delivery of therapeutics, achieve deeper brain penetration and efficacy at lower doses, and ultimately improve patient outcomes while reducing costs. Alector is headquartered in South San Francisco, California. For more information, please visit www.alector.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements regarding our business plans, business strategy, product candidates, research and preclinical pipeline, blood-brain barrier technology platform, planned and ongoing preclinical studies, planned clinical trials, expected milestones, and financial and cash guidance. Such statements are subject to numerous risks and uncertainties, including but not limited to risks and uncertainties as set forth in Alector’s Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q, with the Securities and Exchange Commission (“SEC”), as well as the other documents Alector files from time to time with the SEC. These documents contain and identify important factors that could cause the actual results for Alector to differ materially from those contained in Alector’s forward-looking statements. Any forward-looking statements contained in this press release speak only as of the date hereof, and Alector specifically disclaims any obligation to update any forward-looking statement, except as required by law. Source: Alector, Inc.

（来源：中国医药报） 转自：中国医药报 近年来，与中枢神经系统（CNS）疾病相关的全球疾病负担持续上升，造成巨大的经济影响。预计到2030年，仅心理健康问题造成的全球经济负担将达到6万亿美元，而阿尔茨海默病及相关痴呆症造成的经济负担将增至4.7万亿美元 CNS疾病创新疗法开发迫在眉睫。神经炎症、肠脑轴、迷幻疗法、数字健康成为业界关注的焦点，并取得了突破性进展，为CNS疾病治疗带来了新希望 临床需求存在缺口 如今，全球心理健康和神经疾病造成的负担持续加重。据世界卫生组织（WHO）最新统计，2021年，全球非传染性心理健康疾病和神经疾病对健康相关生活质量的长期综合影响为3.04亿失能调整生命年（DALY），较2010年增长26%；相比之下，2021年全球恶性肿瘤造成的疾病负担为2.7亿DALY。 阿尔茨海默病是造成全球CNS疾病负担加重的主要因素。WHO数据显示，2010—2021年，阿尔茨海默病相关DALY增加了54%。同时，多种CNS疾病负担显著加重，其中帕金森病全球患病率从35%上升至47%，抑郁症从16%上升至36%，焦虑症从11%上升至34%,药物滥用从33%上升至54%。 80%的精神疾病患者生活在低收入和中等收入国家（LMIC）。WHO等机构的最新统计显示，2021年，心理健康疾病负担排名前10的国家中，有9个是LMIC。在中上等收入国家的前20种非传染性疾病中，抑郁症和焦虑症分别排在第12位和第15位，而在中低收入国家和低收入国家中，抑郁症分别排在第17位和第18位。同时，WHO估计，在LMIC，超过75%的心理疾病患者无法获得适当的治疗，心理健康服务的提供受到医疗政策、资金、资源等多种因素制约，同时还存在文化障碍和社会歧视现象。 尽管CNS领域的临床需求持续扩大、市场潜力巨大，但新型CNS疗法的研发和商业化仍面临严峻挑战。第一，由于动物模型转化的局限性以及CNS药物靶点验证不足，CNS药物研发的失败率高于其他治疗领域。第二，临床试验中，患者筛选和招募难度大，尤其是在无症状、疾病早期阶段。根据艾昆纬的统计数据，2019—2024年，CNS临床试验的中位招募时间从9.5个月增加到13.4个月，增幅超过40%。第三，CNS药物临床试验缺乏具有临床意义的客观终点指标，主观依赖较重。第四，医疗系统负荷过重，包括专业人员短缺、治疗路径不清晰、基础设施瓶颈等问题，影响新技术落地。第五，医疗预算压力不断加大，导致新型疗法的市场准入和医保支付要求愈发严格。 前沿创新助力突破 近年来，对疾病生物学理解的日渐深入、基因组分层工具的应用等都为CNS药物研发带来切实进展；人工智能（AI）正在加速药物发现和再利用，人脑类器官模型则重新定义了人类相关系统中研究疾病机制和药物反应的方式。这些进展共同推动以治疗模式为驱动的CNS医疗创新，涵盖神经炎症、肠脑轴、迷幻疗法和数字疗法等领域。 神经炎症：病理机制研究提供干预靶点 神经炎症被认为是许多脑部疾病发病和发展的关键因素。神经炎症是大脑的免疫反应，涉及小胶质细胞和星形胶质细胞等支持细胞，以及从血液中进入的免疫细胞。虽然这种反应最初可能有助于清除损伤或毒素，但如果持续时间过长，就会加剧进一步损伤和长期退化。近期，针对阿尔茨海默病、帕金森病、多发性硬化症和中风等病症的研究表明，持续的炎症与组织损伤可形成恶性循环，从而加剧疾病进展。上述发现促使人们转变思路，控制大脑内的炎症被视为改变疾病进程的重要策略，能够进入中枢神经系统的治疗方法获得重点关注。 在帕金森病领域，数据较为复杂。尽管一些早期临床试验结果表明，GLP-1类药物对运动和认知功能有潜在益处，但相关结果在更大规模的研究中并未得到一致证实。有的药物虽然耐受性良好，但安慰剂组和治疗组在运动评分方面并无显著差异，临床试验中仅观察到轻微的体重减轻。对于帕金森病患者而言，体重减轻，尤其是瘦肌肉量的减少，会使临床结果恶化。尽管如此，GLP-1类药物的生物学原理依然成立，未来的研究可能会为下一代药物研发开辟道路，包括既能保持肌肉量又具有更强脑部渗透性的药物。 除了GLP-1类药物，其他不同机制的针对神经炎症的创新疗法研究也在推进。 TREM2激动剂旨在激活小胶质细胞的保护功能。近期，AL002在阿尔茨海默病的INVOKE-2 Ⅱ期临床试验失败。该TREM2激动剂虽然具有较强机制合理性，但未能显示出临床效益，反映出靶点选择策略仍需优化。目前还有多个TREM2的临床项目正在进行中，未来有望通过更好的化合物设计或患者选择，验证该通路的临床价值。 多发性硬化症领域数十年的临床经验表明，精准的免疫靶向治疗能够改变疾病进展。相关经验正在影响对阿尔茨海默病和帕金森病等疾病的治疗方式。尽管上述疾病的根本病理机制不同，但多发性硬化症的治疗经验为免疫调节疗法在神经退行性疾病中的应用提供了宝贵的经验，尤其是在时机和靶点选择得当的前提下。 肠脑轴：微生态撬动神经性疾病新知 越来越多的研究发现，肠道内的状况会影响大脑。肠道微生物群不仅是神经退行性疾病风险的促成因素之一，还可能成为疾病早期标志物。在阿尔茨海默病和帕金森病中，微生物群落的变化已被证实与炎症和认知能力下降有关，包括链球菌、放线菌、牙龈卟啉单胞菌等病原体水平的升高。此外，某些微生物代谢产物（如胆汁酸）也可能影响CNS疾病的发展进程，凸显了肠道与大脑之间存在功能性联系。相关发现不仅为新型疗法指明了方向，还为CNS疾病的早期诊断和风险分层提供了可能。 目前，多家医药企业正在积极研发基于微生物组的疗法，包括活性生物治疗、定制益生菌组合（工程微生物群落），以及能中和肠源性神经毒素的口服制剂。上述疗法旨在纠正肠道内微生物失衡，减轻全身性炎症，并通过免疫和神经通路影响大脑功能，适应证覆盖自闭症谱系障碍、阿尔茨海默病、帕金森病等。虽然相关研究还处于早期阶段，但近年来得到转化医学和临床观察越来越多的支持，肠道靶向干预能够改变脑部疾病的观点已不只是推测，而是有望成为神经科学的前沿领域。 迷幻疗法：“神经可塑性”作用受关注 近年来，业界对迷幻剂疗法的关注明显增加，将其视为治疗CNS疾病尤其是心理健康障碍的一种新颖且可能具有疾病修正作用的方法。迷幻剂诱导或增强神经可塑性的潜力被认为是其能够带来持久临床改善的核心所在，包括改善难治性抑郁症或创伤后应激障碍。截至2025年底，迷幻剂相关治疗药物研发管线共有125款，其中54%处于临床前阶段，18%处于Ⅰ期临床试验阶段，22%处于Ⅱ期临床试验阶段，6%处于Ⅲ期临床试验阶段。相关适应证涵盖多种心理健康疾病，包括抑郁症（24%）、物质/酒精使用障碍（16%）、其他神经精神疾病（10%）、焦虑症（8%）、疼痛（7%）和创伤后应激障碍（5%）等。 目前，55%的迷幻疗法研发管线集中在7种分子及其衍生物上，分别为裸盖菇素、5-甲氧基二甲基色胺、亚甲基二氧基甲基苯丙胺、氯胺酮、二甲基色胺、伊博加因和麦角酸二乙酰胺。其中，裸盖菇素是研究最多的致幻剂，占比17%。此外，研究人员还积极探索其他方法，以克服传统致幻剂面临的挑战，如产生幻觉、成瘾可能性、治疗过程耗时且需要配合心理治疗和密切监测，以及恶心、癫痫发作等。 Psilera、Seaport、Gilgamesh等企业正在开发新型非致幻性神经可塑性促进剂，其中一些公司利用AI辅助药物设计来优化候选药物的药理特性。2025年5月，Gilgamesh公布其GM-2505的Ⅱa期临床试验顶线数据，受到业界关注。GM-2505是一种5-HT2A受体激动剂和5-HT释放剂，用于治疗重度抑郁症。该药物的临床试验结果显示出快速且持久的抗抑郁效果。 不过，迷幻疗法领域并非一帆风顺。2024年8月，美国食品药品管理局（FDA）拒绝了Lykos的亚甲基二氧基甲基苯丙胺辅助治疗创伤后应激障碍疗法。这一监管决定凸显了稳健的临床试验设计的重要性，尤其是要控制“功能揭盲”。例如，在Ⅲ期临床试验中可采用多剂量对照设计，如设置低剂量组，以降低因患者识别治疗分组而影响疗效评估的风险。 数字健康：提供全天候全程治疗支持 医疗健康领域人力短缺日益严重，促使人们寻求可扩展的、技术驱动的解决方案。在此背景下，CNS领域数字干预措施不断拓展，涵盖从新兴的聊天机器人到更成熟的处方数字疗法等，旨在辅助临床医生，为患者提供全天候、全程治疗支持。 聊天机器人是由AI驱动的对话机器人，近年来逐渐在心理健康领域探索应用。由于相关互动通常缺乏医疗监督、临床验证，并存在隐私泄露风险，对聊天机器人的评估开始出现。在2025年3月发表的一项临床试验结果中，使用达特茅斯大学开发的一款AI聊天机器人后，重度抑郁症症状平均减轻了51%，广泛性焦虑症症状平均减轻了31%，饮食失调症状平均减轻了19%。参与者认为，使用该AI聊天机器人的体验感接近与心理治疗师交流。然而，AI聊天机器人尚处于起步阶段，进一步的验证和长期结局研究仍在进行。 处方数字疗法是能够提供基于证据的软件型医疗干预手段，正受到越来越广泛的关注。Click Therapeutics与大冢、勃林格殷格翰分别合作开发用于抑郁症和精神分裂症的处方数字疗法，探索处方数字疗法与药物治疗的联合方案。数字疗法的可扩展性和临床价值日益受到业界关注，根据艾昆纬发布的《2024年数字健康趋势》，与2021年相比，2024年全球市场可获得的处方数字疗法产品数量猛增560%，达到141种；处于商业可用或开发阶段的处方数字疗法数量从2021年的137种增加至2024年的256种，研发管线中有62%集中在心理健康和神经领域。 近期，Click Therapeutics的CT-132获得美国FDA批准上市，进一步激发了业界对处方数字疗法的热情。该产品是FDA批准上市的首款用于预防发作性偏头痛的处方数字疗法。此外，用于治疗广泛性焦虑障碍的Daylight Rx、用于治疗失眠的SleepioRx等新型心理健康应用程序的上市，也凸显了处方数字疗法在临床验证方面取得的成果。这些数字解决方案提供结构化的认知和行为疗法，不再只是辅助手段，而是增强现代医疗健康、填补医疗可及性和连续性方面空白的重要组成部分。 研发活跃市场多元 2024年，全球共启动了605项CNS临床试验，占医药行业临床试验总数的11%，与2020年持平。CNS在临床试验活跃度排名中位居第三，仅次于肿瘤和免疫领域。 2020—2024年，阿尔茨海默病、抑郁症、帕金森病是CNS疾病临床试验的前三大适应证，启动临床试验数量均超过200项；精神分裂症、癫痫、多发性硬化症、肌萎缩侧索硬化症、偏头痛、焦虑症和肌肉萎缩症分列第四至十位，启动临床试验数量均超过100项。 对于新型CNS疗法的临床开发，业界正积极采用远程、虚拟或去中心化（RVD）等技术赋能的试验模式。2020—2024年，CNS疗法占RVD试验总数的27%，在所有治疗领域中占比最高。 新兴生物制药企业在CNS创新领域占据主导地位，许多企业正在开发前沿的治疗方法。例如，Neurona Therapeutics正在探索利用多能干细胞衍生疗法来替换耐药性癫痫患者体内功能失调的神经元，以长期减少癫痫发作；Kyverna正在部署CAR-T细胞疗法，通过深度B细胞耗竭，致力于在多发性硬化症或重症肌无力等神经炎症性疾病中，帮助患者缓解病情，并实现长期稳定、无须持续用药。 新兴生物制药企业作为CNS创新的主要推动者，正成为合作、授权和收购的热门目标。值得关注的CNS交易案例包括：强生以146亿美元收购Intra-Cellular Therapies，通过此次收购，Caplyta（2019年12月首次获得美国FDA批准用于治疗成人精神分裂症）被纳入强生的神经科学产品线；礼来以14亿美元从Sangamo Therapeutics获得神经趋向腺相关病毒衣壳技术的许可，用于开发CNS疾病的基因疗法；艾伯维以14亿美元收购Aliada Therapeutics，以获得其主打产品ALIA-1758（一种针对淀粉样蛋白斑块的抗体），从而进军阿尔茨海默病领域；百时美施贵宝以127亿美元收购Karuna、艾伯维以87亿美元收购Cerevel Therapeutics，分别获得精神分裂症药物KarXT（已经以Cobenfy品牌上市）和emraclidine。 在CNS领域活跃的中型和大型制药企业，根据不同的战略方向，可分为三种类型。 一是深耕CNS领域。灵北、渤健、大冢等企业在神经学和心理健康领域有着深厚积累，其营收中有很大一部分来自CNS市场，且不断投资CNS药物研发管线，以保持竞争力。渤健围绕神经学领域构建了强大的在研产品线，涵盖10个项目；灵北积极探索不同治疗途径，包括神经肽信号传导、神经生物学、蛋白质聚集和清除机制、神经炎症等，拥有7个CNS相关资产。二是强化CNS布局。罗氏、艾伯维、诺华、强生和礼来等企业正加大CNS领域投入，打造庞大的CNS产品线，力求成为该领域的领军企业。罗氏已构建起业内领先的CNS产品线，涵盖20余个项目，重点关注神经退行性疾病和罕见神经系统疾病。艾伯维拥有十多个项目，涉及心理健康、神经退行性疾病和罕见神经系统疾病等领域。 三是谨慎探索。一些企业对CNS疗法采取了探索性策略，通常在该领域投资不多，研发管线较为轻量，有时仅包含一项资产。勃林格殷格翰在心理健康领域的研发管线涵盖药物治疗和数字疗法相结合的产品。拜耳探索细胞和基因疗法在神经学领域的潜力，尤其关注罕见病。诺和诺德在EVOKE和EVOKE+试验中研究GLP-1受体激动剂司美格鲁肽的潜在抗神经炎症特性，用于治疗阿尔茨海默病，并研究针对帕金森病的细胞疗法。葛兰素史克与Alector合作，分别在额颞叶痴呆和阿尔茨海默病中研究靶向sortilin受体的抗体药物latozinemab和AL101。总体来看，这类药企期望通过外部创新资源来推进其在CNS领域的发展。 （艾昆纬供稿）