ANX005-Treated Patients Demonstrated Faster and More Complete Recovery from Week 1 through Week 26 on Primary and Multiple Pre-Specified Endpoints

ANX005治疗的患者在第一周至第26周的主要和多个预先指定的终点表现出更快，更完全的恢复

Two and a Half Times More ANX005-Treated Patients Returned to a Normal / Pre-Disease State of Health Over Placebo on GBS-DS by Week 26, Increasing Over Time

ANX005 Beneficial Impact Larger in Patients with North American and European Baseline Characteristics Across Key Measures of Disability and Muscle Strength

ANX005对北美和欧洲基线特征患者的残疾和肌肉力量的关键指标产生较大的有益影响

Single Infusion of ANX005 was Generally Well-Tolerated with Safety Profile Similar to Placebo

单次输注ANX005通常耐受性良好，安全性与安慰剂相似

Data Reinforce Potential of ANX005 to be First Targeted Immunotherapy Treatment for GBS

数据增强了ANX005成为GBS首次靶向免疫治疗的潜力

BRISBANE, Calif., June 25, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), today announced positive results from the completed pivotal Phase 3 trial of C1q-targeted immunotherapy, ANX005, in Guillain-Barré Syndrome (GBS) at the 2024 Peripheral Nerve Society (PNS) Annual Meeting in Montréal, Canada.

加利福尼亚州布里斯班，2024年6月25日（环球通讯社）--Annexon，Inc.（纳斯达克：ANNX）今天在加拿大蒙特勒尔举行的2024年周围神经学会（PNS）年会上宣布，C1q靶向免疫疗法ANX005在吉兰-巴雷综合征（GBS）中完成的关键性3期试验取得了积极结果。

Leading global experts in the GBS field highlighted the significant unmet need and opportunity to transform the GBS treatment landscape with a targeted immunotherapy approach, as well as additional Phase 3 analyses of early and durable treatment effects important to patients and the medical community..

GBS领域的全球领先专家强调了通过靶向免疫治疗方法改变GBS治疗格局的重大未满足需求和机会，以及对患者和医学界重要的早期和持久治疗效果的额外第三阶段分析。。

“ANX005 rapidly suppressed neuroinflammation and validated the role of C1q inhibition in GBS during the active phase of disease, leading to highly statistically significant improvements across multiple endpoints and over multiple timepoints versus placebo,” said Douglas Love, president and chief executive officer of Annexon.

Annexon总裁兼首席执行官道格拉斯·洛夫（DouglasLove）表示：“ANX005迅速抑制了神经炎症，并验证了C1q抑制在疾病活跃期GBS中的作用，从而在多个终点和多个时间点与安慰剂相比取得了高度统计学显着的改善。”。

“Having successfully completed the first placebo-controlled GBS trial in decades, we were honored to present the pivotal Phase 3 results showing accelerated and durable recovery of GBS patients treated with ANX005 compared to placebo in the plenary Symposium at PNS. With these favorable results, we are laser focused on bringing ANX005 to GBS patients worldwide as quickly as possible.”.

“成功完成了数十年来第一个安慰剂对照的GBS试验，我们很荣幸在PNS全体研讨会上展示了关键的3期结果，显示与安慰剂相比，ANX005治疗的GBS患者的恢复速度更快，持久性更强。有了这些有利的结果，我们专注于尽快将ANX005带给全球GBS患者。”。

Dr. Quazi Deen Mohammad, Principal Investigator of the trial and Founding Director of the National Institute of Neurosciences and Hospital (NINS), Bangladesh added, “This well-designed and well-executed study demonstrated that acute suppression of C1q with ANX005 enabled patients to get better sooner, which translated into continued long-term benefits and a significantly higher likelihood of full recovery versus placebo.

该试验的首席研究员兼孟加拉国国家神经科学与医院研究所（NINS）的创始主任Quazi Deen Mohammad博士补充说：“这项设计良好且执行良好的研究表明，ANX005对C1q的急性抑制使患者更快好转，这转化为持续的长期益处，与安慰剂相比，完全康复的可能性显着更高。

Notably, patients came off ventilation and walked one month earlier, regained their independence faster, and got back to a normal way of life sooner. In addition, Phase 3 patients with baseline characteristics consistent with North American and European GBS patients had a more pronounced treatment effect, being three times more likely versus placebo to be in a good state of health with ANX005 treatment.

值得注意的是，患者提前一个月停止通气并行走，更快地恢复了独立性，并更快地恢复了正常的生活方式。此外，基线特征与北美和欧洲GBS患者一致的3期患者的治疗效果更为明显，与安慰剂相比，ANX005治疗的健康状况良好的可能性是安慰剂的三倍。

These compelling data reinforce the therapeutic potential of ANX005 to be the first targeted immunotherapy treatment for GBS.”.

这些令人信服的数据增强了ANX005作为GBS的第一种靶向免疫治疗的治疗潜力。”。

Summary of Phase 3 Data with ANX005 30 mg/kg Treatment

ANX005 30 mg/kg治疗的3期数据摘要

GBS-Disability Scale (GBS-DS)

GBS残疾量表（GBS-DS）

Primary endpoint at Week 8: 2.41-fold higher likelihood of being in a better state of health with ANX005 vs. placebo (p = 0.0058)

第8周的主要终点：ANX005与安慰剂相比，健康状况更好的可能性高2.41倍（p=0.0058）

Week 1: 7.22-fold higher likelihood of being in a better state of health with ANX005 vs. placebo (*p < 0.0001)

第1周：ANX005与安慰剂相比，健康状况更好的可能性高7.22倍（*p<0.0001）

Week 4: 2.49-fold higher likelihood of being in a better state of health with ANX005 vs. placebo (*p = 0.0073)

第4周：ANX005与安慰剂相比，健康状况更好的可能性高2.49倍（*p=0.0073）

Week 26: 2.5 times more patients had fully recovered to a normal / pre-disease state of health (GBS-DS = 0) with ANX005 (21.5%) vs. placebo (8.6%) (OR 4.14, *p = 0.0092)

第26周：使用ANX005（21.5%）与安慰剂（8.6%）（OR 4.14，*p=0.0092）的患者完全恢复到正常/疾病前健康状态（GBS-DS=0）的人数是对照组的2.5倍

Week 8 responder analysis (pre-specified sensitivity analysis): 2-times more patients improved 3 points or more with ANX005 (28.2%) vs. placebo (13.6%) (*p = 0.0309)

第8周反应者分析（预先指定的敏感性分析）：与安慰剂（13.6%）相比，ANX005（28.2%）患者改善3分或更多2倍（*p=0.0309）

Week 8 dichotomy analysis (pre-specified sensitivity analysis): 2.5-times more patients were able to run or better ANX005 (29%) vs. placebo (12%) (OR 3.34, *p = 0.0065)

第8周的二分法分析（预先指定的敏感性分析）：与安慰剂（12%）相比，ANX005（29%）能够运行或更好的患者是2.5倍（or 3.34，*p=0.0065）

Functional Measures

功能性措施

Walking 31 days earlier with ANX005 treatment (56 days) vs. placebo (87 days) (*p = 0.0211)

ANX005治疗（56天）与安慰剂（87天）相比，步行提前31天（*p=0.0211）

Off ventilation 28 days earlier with ANX005 treatment (20 days) vs. placebo (48 days) (*p = 0.0356)

Patients with North American and European baseline characteristics

具有北美和欧洲基线特征的患者

Week 1: 8.8-point improvement in muscle strength measured by Medical Research Council (MRC) sumscore with ANX005 vs. placebo (*p <0.0001)

第1周：医学研究委员会（MRC）用ANX005与安慰剂相比，肌肉力量提高了8.8分（*p<0.0001）

Week 8: 3 times more likely to be in a better state of health on the GBS-DS scale with ANX005 vs. placebo (*p = 0.0102)

第8周：在GBS-DS量表上，ANX005与安慰剂相比，健康状况更好的可能性高3倍（*p=0.0102）

Key Findings from the Phase 3 GBS Trial of ANX005 30 mg/kg Treatment

ANX005 30 mg/kg治疗的3期GBS试验的主要发现

Phase 3 trial informed by dose-ranging Phase 1b trial, replicating earlier results

剂量范围1b期试验通知的3期试验，复制了早期结果

Demonstrated a highly statistically significant effect on primary endpoint of GBS-DS, further supported by multiple prespecified sensitivity analyses

显示出对GBS-DS主要终点的高度统计学显着影响，并得到了多项预先指定的敏感性分析的进一步支持

Defined the effective treatment window during the active phase of GBS, an acute disease

确定了急性疾病GBS活动期的有效治疗窗口

Early, robust and durable treatment effects expedited patient recovery

早期、稳健和持久的治疗效果加速了患者的康复

Single dose administration of ANX005 was generally well-tolerated with mostly mild to moderate adverse events, no increased infection rate while not requiring vaccination or prophylactic antibiotics, and a profile similar to placebo

单剂量服用ANX005通常耐受性良好，大多数为轻度至中度不良事件，感染率没有增加，同时不需要接种疫苗或预防性抗生素，并且与安慰剂相似

ANX005 Comparability Analyses Flash Presentation and Poster Sessions

ANX005可比性分析Flash演示和海报会议

The indirect comparison of ANX005-treated patients from the Phase 1b GBS trial with a separate cohort of matched intravenous immunoglobulin (IVIg)-treated patients was presented as a flash oral and poster presentation at PNS, showing early and significant improvements in muscle strength and overall functional outcomes including reduced mechanical ventilation in patients treated with ANX005 versus IVIg.

来自1b期GBS试验的ANX005治疗患者与单独的匹配静脉注射免疫球蛋白（IVIg）治疗患者队列的间接比较在PNS上以快速口服和海报呈现，显示肌肉力量和整体功能的早期和显着改善结果，包括ANX005与IVIg治疗患者的机械通气减少。

The methodology for patient-matching based on prognostic factors for the real-world evidence (RWE) study was presented in a separate poster presentation at PNS. Data from the RWE study will compare the Phase 3 outcomes with patients from the International GBS Outcome Study (IGOS).

基于真实世界证据（RWE）研究的预后因素的患者匹配方法在PNS的单独海报演示中介绍。RWE研究的数据将与国际GBS结果研究（IGOS）的患者进行3期结果比较。

*Nominal p value

*标称p值

About ANX005

关于ANX005

Annexon’s lead investigational therapy, ANX005, is a first-of-its kind selective, targeted and rapid-acting agent designed to reduce inflammation and nerve damage by fully stopping C1q activity in the peripheral and central nervous systems. In GBS, ANX005 seeks out C1q and selectively blocks it from binding to its target on peripheral nerves.

Annexon的主要研究疗法ANX005是首创的选择性，靶向性和速效药物，旨在通过完全停止外周和中枢神经系统中的C1q活性来减轻炎症和神经损伤。。

ANX005 is administered intravenously and has been observed to act almost immediately. In GBS, the aim is to rapidly stop the autoimmune damage of nerve cells, allowing the patient to regain their muscle strength sooner to regain independence and return to pre-illness activities. ANX005 has received both fast track and orphan drug designations from the Food and Drug Administration as well as orphan drug designation by the European Medicines Agency for the treatment of GBS..

。在GBS中，目的是迅速停止神经细胞的自身免疫损伤，使患者更快地恢复肌肉力量，恢复独立性并恢复疾病前的活动。ANX005已获得食品和药物管理局的快速通道和孤儿药指定，以及欧洲药品管理局的孤儿药指定，用于治疗GBS。。

About Guillain-Barré Syndrome (GBS)

关于吉兰-巴雷综合征（GBS）

GBS is a severe disease resulting from an acute autoantibody attack on peripheral nerves that generally occurs post-infection in otherwise healthy persons following activation of C1q and the classical complement cascade. It is a rapid and acute neurological disease with a narrow therapeutic window that results in hospitalization of over 22,000 people annually in the U.S.

GBS是一种严重的疾病，由周围神经的急性自身抗体攻击引起，通常在C1q和经典补体级联激活后，在其他健康人感染后发生。它是一种快速而急性的神经系统疾病，治疗窗口狭窄，导致美国每年有22000多人住院。

and Europe. The peripheral nerve damage progresses rapidly, causing acute neuromuscular paralysis, and may lead to significant morbidity, disability and mortality. Currently, there are no approved treatments for GBS in the U.S. The long-term disease burden associated with GBS has led to a multi-billion-dollar annual economic cost to the U.S.

和欧洲。周围神经损伤进展迅速，导致急性神经肌肉麻痹，并可能导致严重的发病率，残疾和死亡率。目前，美国还没有批准的GBS治疗方法。与GBS相关的长期疾病负担已导致美国每年数十亿美元的经济成本。

healthcare system alone..

仅医疗保健系统。。

About Annexon

关于Annion

Annexon Biosciences (Nasdaq: ANNX) is a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye. Annexon’s novel scientific approach targets upstream C1q to block the classical complement inflammatory cascade before it starts, and its therapeutic candidates are designed to provide meaningful benefits across multiple autoimmune, neurodegenerative and ophthalmic diseases.

Annexon Biosciences（Nasdaq:ANNX）是一家生物制药公司，为患有破坏性经典补体介导的身体，大脑和眼睛神经炎性疾病的人提供新型治疗的晚期临床平台。。

With proof-of concept data in Guillain-Barré syndrome, Huntington’s disease and geographic atrophy, Annexon is rigorously advancing its mid-to late-stage clinical trials to bring their potential treatments to patients as quickly as possible. To learn more visit annexonbio.com..

凭借格林-巴利综合征，亨廷顿舞蹈病和地理萎缩的概念验证数据，Annexon正在严格推进其中晚期临床试验，以尽快为患者带来潜在的治疗方法。。。

Forward Looking Statements

前瞻性声明

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “suggest,” “target,” “on track,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology.

本新闻稿包含经修订的《1933年证券法》第27A节和经修订的《1934年证券交易法》第21E节所指的前瞻性声明。在某些情况下，您可以通过术语来识别前瞻性陈述，例如“目标”，“预期”，“假设”，“相信”，“沉思”，“继续”，“可能”，“设计”，“到期”，“估计”，“预期”，“目标”，“打算”，“可能”，“目标”，“计划”，“定位”，“潜力”，“预测”，“寻求”，“应该”，“建议”，“目标”，“正在轨道上”，“将”，“将会”以及其他类似的表达，这些表达是对未来事件和未来趋势的预测或指示，或者这些术语或其他类似术语的负面影响。

All statements other than statements of historical facts contained in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, statements about: ability of ANX005 to stop C1q activity; ability to bring ANX005 to patients worldwide as soon as possible; the clinical and regulatory status of ANX005; the potential of ANX005 to be the first approved C1q-targeted treatment for GBS; the timing of completion of RWE study and potential submission of a BLA with the FDA; the potential therapeutic benefit of ANX005 or any other product candidates on GBS, Huntington’s disease or geographic atrophy; potential benefit of ANX005, if approved, compared to existing therapies; market size; the potential benefits from treatment with anti-C1q therapy; and Annexon’s ability to rigorously advance mid-to late-stage clinical trials and continue development of the company’s portfolio.

除本新闻稿中包含的历史事实声明外，所有声明均为前瞻性声明。这些前瞻性声明包括但不限于以下声明：ANX005停止C1q活动的能力；能够尽快将ANX005带给世界各地的患者；ANX005的临床和监管状况；；RWE研究的完成时间以及向FDA提交BLA的可能性；ANX005或任何其他候选产品对GBS，亨廷顿舞蹈病或地理萎缩的潜在治疗益处；与现有疗法相比，如果获得批准，ANX005的潜在益处；市场规模；抗C1q治疗的潜在益处；以及Annexon严格推进中晚期临床试验并继续开发公司投资组合的能力。

Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events t.

前瞻性陈述并不能保证未来的表现，并且会受到可能导致实际结果和事件的风险和不确定性的影响。

Investor Contact:

投资者联系人：

Joyce Allaire

乔伊斯·阿莱尔

LifeSci Advisors, LLC

jallaire@lifesciadvisors.com

jallaire@lifesciadvisors.com

Media Contact:

媒体联系人：

Sheryl Seapy

谢丽尔·西皮。

Real Chemistry

真正的化学

949-903-4750

949-903-4750

sseapy@realchemistry.com

sseapy@realchemistry.com