AGAZISIRAN

Advancing the Alector Brain Carrier (ABC) platform across multiple therapeutic modalities, including antibodies, enzymes, and siRNA, with continued progress across AL037/AL137 (ABC-enabled antibody for Alzheimer’s disease (AD)), AL050 (ABC-enabled GCase Enzyme Replacement Therapy for Parkinson’s disease (PD)), and AL064/AL164 (ABC-enabled Tau siRNA for AD and other tauopathies) $206.5 million in cash, cash equivalents and investments provide runway at least through 2027 SOUTH SAN FRANCISCO, Calif., May 07, 2026 (GLOBE NEWSWIRE) -- Alector, Inc. (Nasdaq: ALEC), a biotechnology company focused on developing therapies to counteract the devasting progression of neurodegeneration, today reported first quarter 2026 financial results and recent portfolio and business updates. As of March 31, 2026, Alector’s cash, cash equivalents, and investments totaled $206.5 million. “Over the past seven years, we have built a highly differentiated blood-brain barrier platform with the versatility to deliver antibodies, enzymes, proteins, and siRNA to the brain,” said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. “We have a rich and growing pipeline of ABC-enabled programs, and we remain deeply committed to developing therapies that can make a meaningful difference for patients living with neurodegenerative diseases.” Recent Program Updates Alector Brain Carrier (ABC): Preclinical and Research Pipeline At the core of Alector’s strategy is the Alector Brain Carrier (ABC), the company’s proprietary platform designed to enhance the delivery of therapeutics to the brain. ABC is intended for peripheral dosing and is adaptable across multiple drug modalities, including antibodies, enzymes, and siRNA. The platform is built on core design principles of versatility, optimized binding properties, and translatability, with the goal of achieving efficient and targeted brain delivery while maintaining a favorable safety profile. Across multiple ABC-enabled programs, preclinical studies have demonstrated robust brain penetration, supporting the advancement of a broad pipeline aimed at addressing the underlying drivers of neurodegenerative diseases. AL037/AL137 Alector continues to advance AL037/AL137, its ABC-enabled anti-amyloid beta (Aβ) antibody program for the treatment of AD, through investigational new drug (IND)-enabling studies. The company is targeting an IND submission in Q1 2027. Both of our AL037 and AL137 candidates are engineered for optimal brain uptake, potency, safety, and convenience. Both candidates comprise the same high-affinity, fully human antibody that selectively binds PyroGlu3, a validated epitope on toxic amyloid beta found in plaques and retain an active effector function intended to facilitate myeloid-mediated plaque clearance. AL037 and AL137 each incorporates Alector’s proprietary ABC technology with TfR binding domains that bind the same epitope on TfR, but with different affinities and binding kinetics, to balance brain penetration and plaque removal with minimized hematologic adverse effects. AL050 Alector continues to progress AL050, its ABC-enabled engineered glucocerebrosidase (GCase) enzyme replacement therapy (ERT) for PD, through preclinical development. AL050 is designed to address key challenges associated with enzyme delivery to the brain, featuring an engineered GCase with improved activity and stability and a silenced effector function to maximize safety, paired with Alector’s tunable ABC technology. Preclinical studies to date have demonstrated increased GCase activity and reduced toxic substrate accumulation, supporting its continued preclinical 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. We continue to evaluate our timeline to the clinic. ABC siRNA Platform Alector is advancing its ABC-enabled siRNA platform with the goal of making treatment more accessible by enabling peripheral dosing as a potential alternative to traditional intrathecal delivery. Alector continues to advance AL064/AL164, its ABC-enabled tau siRNA program for the treatment of AD and other tauopathies. AL064 demonstrated robust tau mRNA knockdown and durable reduction of phospho-Tau 217 in non-human primate studies, and it was subsequently modified to further optimize siRNA stability. This modified form is advancing into IND-enabling studies as AL164. In addition to AL164, the company is advancing early-stage siRNA programs toward lead candidate selection, including 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. Progranulin Program (nivisnebart (AL101/GSK4527226) The global, randomized, double-blind, placebo-controlled PROGRESS-AD Phase 2 clinical trial of nivisnebart (AL101/GSK4527226) in early AD has been discontinued following a pre-specified interim futility analysis. An independent data monitoring committee concluded that the trial was unlikely to meet its primary endpoint of slowing disease progression at completion. As the company’s platform and pipeline have continued to evolve, Alector remains focused on the significant opportunities ahead across its ABC-enabled programs. First Quarter 2026 Financial Results Revenue. Collaboration revenue for the quarter ended March 31, 2026, was $1.0 million, compared to $3.7 million for the same period in 2025. The decrease in year-over-year collaborative revenue was primarily due to lower manufacturing-related activity to support the Nivisnebart Phase 2 study in early Alzheimer’s disease. R&D Expenses. Total research and development expenses for the quarter ended March 31, 2026, were $17.9 million, compared to $33.6 million for the quarter ended March 31, 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 research and development expenses for the AL002 program. G&A Expenses. Total general and administrative expenses for the quarter ended March 31, 2026, were $8.1 million, compared to $14.7 million for the quarter ended March 31, 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 March 31, 2026, Alector reported a net loss of $22.9 million, or $0.21 per share, compared to a net loss of $40.5 million, or $0.41 net loss per share, for the same period in 2025. Cash Position. Cash, cash equivalents, and investments were $206.5 million as of March 31, 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, 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. Selected Consolidated Balance Sheet Data (in thousands) Condensed Consolidated Statement of Operations Data (in thousands, except share and per share data) Alector Contacts: Argot Partners (media) David Rosen (646) 461-6387 alector@argotpartners.com Argot Partners (investors) Laura Perry (212) 600-1902 alector@argotpartners.com Source: Alector, Inc.

编者按：近年来，寡核苷酸新药研发领域进展持续加速，在多个疾病领域得到广泛应用。例如，2026年第一季度在中国获批IND的1类新药中包括了近20款寡核苷酸疗法，数量是2025年同期约4倍。除此之外，当前，全球有数百项寡核苷酸疗法相关临床试验正在进行之中，彰显这一领域的发展潜力。作为全球医药创新的赋能者，药明康德一直以来依托“一体化、端到端”的CRDMO赋能平台，助力全球合作伙伴推进包括寡核苷酸药物在内的各类创新疗法开发，加速造福病患。寡核苷酸药物是全球新药开发的重要热点，近年来研发进展持续加速。例如，根据中国国家药监局药品审评中心（CDE）官网及公开资料，2026年第一季度，有至少16款寡核苷酸1类新药管线首次获批IND，进入新的研发阶段。从药物类型来看，这些寡核苷酸新药主要为小干扰RNA（siRNA），反义寡核苷酸（ASO），此外还包括小激活RNA（saRNA）等，靶点涵盖ANGPTL3、SMN2、ApoC3、CFB、INHBE、AGT/PCSK9、SCN1A、Tau、MASP-2等。从涉及适应症来看，心血管疾病、代谢性疾病、中枢神经系统（CNS）疾病适应症占多数，此外还包括肾病、癌症、感染性疾病、罕见遗传性病等。一季度近20款寡核苷酸1类新药在中国获批IND在全球范围内，基于寡核苷酸的降压疗法和降脂疗法倍受关注。这类疗法通常作用时间长，效果更持久，有望改善当前患者服药依从性不佳的现状，提高治疗依从性。2026年第一季度在中国获批IND的1类新药中就包括了多款寡核苷酸降压和降脂疗法，比如：礼来（Eli Lilly and Company）在研的靶向ANGPTL3的siRNA疗法solbinsiran注射液；瑞博泰克靶向ApoC3的siRNA疗法RBD5044注射液；必贝特医药AGT/PCSK9双靶siRNA药物BEBT-701注射液；民为生物在研的siRNA新药MWX401注射液；心正合医药siRNA药物LC-1016注射液，等等。图片来源：123RF近年来，寡核苷酸药物在神经系统疾病治疗领域也取得了诸多突破。这类疗法可通过直接调节mRNA水平而非蛋白功能，实现对疾病分子机制的有效干预。迄今为止，美国FDA已批准多款寡核苷酸药物用于神经系统疾病的治疗，显著改善了众多患者的生活质量。2026年第一季度，渤健（Biogen）用于脊髓性肌萎缩症（SMA）的ASO疗法BIIB115注射液；Stoke Therapeutics靶向SCN1A基因的ASO疗法zorevunersen注射液（用于Dravet综合征）；诺华（Novartis）Tau靶向ASO疗法NIO752注射液（用于进行性核上性麻痹）均在中国获批IND，进入临床研究阶段。除了上述疗法，近年来靶向INHBE基因的siRNA疗法治疗肥胖的方向也于近期迎来新进展。不同于GLP-1类药物的作用机制，INHBE功能丧失的人群拥有良好的体脂分布和有利的代谢特征，靶向抑制INHBE可以从基因水平减少内脏脂肪，改善代谢状态，更有利于长期体重管理。2026年第一季度，先衍生物靶向INHBE基因的siRNA疗法LDR2515注射液；康哲生物靶向INHBE的siRNA疗法CMS-D008注射液均在中国获批进入临床研究阶段。此外，信立泰用于治疗代谢相关脂肪性肝炎（MASH）的siRNA疗法SAL0145注射液也获批IND，意味着该寡核苷酸疗法有望将适应症拓展至MASH这类代谢类疾病。在肾病、感染性疾病、罕见遗传性疾病等领域，均看到寡核苷酸疗法的身影。2026年第一季度，元羿生物靶向CFB的siRNA疗法ADX-038注射液（用于补体介导的肾脏疾病，包括IgAN、C3G和IC-MPGN）；前沿生物靶向MASP-2的siRNA疗法FB7013注射液（用于原发性IgA肾病）；思合基因用于慢性乙型肝炎的ASO疗法SG12注射液；中美瑞康用于非肌层浸润性膀胱癌的小激活RNA（saRNA）疗法RAG-01注射液；元羿生物靶向PKK的siRNA疗法ADX-324注射液（用于预防遗传性血管性水肿发作）等均在中国迈入临床开发阶段。除了上述进展，在全球范围内，寡核苷酸创新疗法研发也在今年一季度迎来一系列新进展，包括多款寡核苷酸新药取得监管进展或公布新的临床数据，此外还有多家药企通过通过战略合作与并购加码寡核苷酸疗法，以及相关新锐公司获得融资等等。很高兴看到在产业共同努力下，更多创新成果加速造福患者。一体化CRDMO平台赋能寡核苷酸疗法研发凭借其高度靶向性、灵活可编程和相对较短的开发周期等特点，寡核苷酸疗法近年来受到产业界的广泛关注，而其研发生产也面临相应挑战。作为全球医药创新的赋能者，药明康德一直以来依托一体化、端到端的CRDMO赋能平台，助力全球合作伙伴推进包括寡核苷酸药物在内的各类创新疗法开发，加速造福病患。例如，药明康德药物代谢与动力学部（DMPK）针对寡核苷酸药物在体内代谢复杂、活性代谢产物难以区分与定量的关键挑战，构建了以高灵敏度生物分析与机制驱动解析为核心的一体化解决方案。与传统小分子不同，寡核苷酸在体内主要经核酸内切酶和外切酶降解，易产生具有活性的N-1、N-2等代谢产物，且这些代谢物在结构与理化性质上与原型药高度相似，分离与定量难度显著增加 。围绕这一难点，药明康德DMPK建立了基于液相色谱-串联质谱（LC-MS/MS）的高选择性分析平台，结合离子对反相色谱（IP-RPLC）、亲水作用色谱（HILIC）和离子交换色谱（IEC）等多种分离策略，实现对寡核苷酸及其多种活性代谢产物的高分辨分离与精准定量。同时，药明康德DMPK通过系统优化样品前处理、色谱条件及质谱参数，有效应对寡核苷酸高极性、多电荷状态及基质干扰等分析挑战 ，并可在复杂生物基质中实现原型药物与代谢物的同步检测与谱图解析。在此基础上，药明康德DMPK进一步整合代谢产物鉴定（MetID）、暴露定量及跨物种比较研究，支持全面描述寡核苷酸药物的体内代谢路径与活性贡献。依托这一“高灵敏分析+多策略分离+代谢机制解析”的综合能力，药明康德DMPK能够帮助客户提升寡核苷酸药物药代研究的准确性与可解释性，加速推动创新核酸疗法从早期研究向临床开发的高效转化。展望未来，药明康德将持续基于其一体化CRDMO平台，助力合作伙伴加快将科学创新转化为新药、好药，造福全球病患。免责声明：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：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「医药观澜」微信公众号回复“转载”，获取转载须知。