AC Immune’s ACI-35.030 (now “JNJ-2056”) Granted FDA Fast Track Designation for Alzheimer’s Disease Lausanne, Switzerland, July 25, 2024 – AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, today announced that its active-immunotherapy candidate, ACI-35.030 (now called JNJ-2056), targeting the pathologic form of the Tau protein, phosphorylated Tau (pTau), has received Fast Track designation from the U.S.

2024年7月25日，AC Immune公司的ACI-35.030（现为“JNJ-2056”）获得FDA对瑞士洛桑阿尔茨海默氏病的快速通道指定–AC Immune SA（纳斯达克：ACIU）是一家临床阶段生物制药公司，开创了神经退行性疾病的精准医学，今天宣布其针对Tau蛋白磷酸化Tau（pTau）病理形式的主动免疫治疗候选药物ACI-35.030（现为JNJ-2056）已获得美国的快速通道指定。

Food and Drug Administration (FDA). The recently initiated Phase 2b clinical trial ReTain is currently recruiting participants with preclinical Alzheimer’s disease, where individuals have yet to show clinical symptoms.Dr. Andrea Pfeifer, CEO of AC Immune SA, commented: “Fast Track designation is an important recognition of the differentiation and potential value for patients of our anti-pTau active immunotherapy, ACI-35.030.

美国食品和药物管理局（FDA）。最近启动的2b期临床试验ReTain目前正在招募临床前阿尔茨海默病的参与者，其中个体尚未表现出临床症状。AC Immune SA首席执行官Andrea Pfeifer博士评论道：“快速通道指定是对抗pTau主动免疫疗法ACI-35.030患者分化和潜在价值的重要认可。

The Phase 2b ReTain study is the first time any active immunotherapy is being tested in a preclinical AD population. We believe this modality has the potential to offer therapeutic advantages, as well as benefits in terms of convenience and access. Fast Track designation offers opportunities for more efficient development and regulatory review.

2b期ReTain研究是首次在临床前AD人群中测试任何主动免疫疗法。我们相信这种方式有可能提供治疗优势，以及便利和获取方面的好处。快速通道设计为更有效的开发和监管审查提供了机会。

More importantly, this underscores and validates the potential therapeutic impact of an active immunotherapy specifically targeting pTau, the key pathologic species of Tau protein. In Phase 1b/2a clinical testing, ACI-35.030 was shown to specifically target this toxic form of Tau and spare normal endogenous forms of the protein.

更重要的是，这强调并验证了针对Tau蛋白关键病理物种pTau的主动免疫疗法的潜在治疗效果。在1b/2a期临床测试中，ACI-35.030被证明可以特异性靶向这种毒性形式的Tau蛋白，并保留正常的内源性蛋白质。

We and our partners continue to drive innovation in the treatment and potential prevention of Alzheimer’s disease, developing new mechanisms of action and first-in-class potential therapeutics that are s.

我们和我们的合作伙伴继续推动阿尔茨海默病治疗和潜在预防方面的创新，开发新的作用机制和一流的潜在治疗方法。

The Phase 2b ReTain trial is a potentially registration-enabling randomized, multicenter, double-blind, placebo-controlled clinical study in participants with preclinical AD to assess the clinical effect of active immunization with JNJ-64042056. It is designed to test the hypothesis that JNJ-2056 has a disease-modifying effect that can delay or prevent the onset of cognitive impairment or other clinical symptoms in individuals with preclinical AD through inhibition of seeding and spreading of pathological Tau.The study will include approximately 500 participants with preclinical AD (cognitively normal, Tau positive), who will be randomized in a 1:1 ratio to a single dose level of JNJ-2056 or placebo and administered as intramuscular injections for a maximum of 4 years.

2b期ReTain试验是一项潜在的注册研究，可用于临床前AD患者的随机，多中心，双盲，安慰剂对照临床研究，以评估JNJ-64042056主动免疫的临床效果。它旨在检验JNJ-2056具有疾病缓解作用的假设，该作用可以通过抑制病理性Tau的播种和扩散来延迟或预防临床前AD患者的认知障碍或其他临床症状的发作。该研究将包括大约500名临床前AD（认知正常，Tau阳性）的参与者，他们将以1:1的比例随机分配到JNJ-2056或安慰剂的单剂量水平，并作为肌内注射给药，最多4年。

The primary endpoint will measure cognitive decline as assessed by the Preclinical AD Cognitive Composite 5 (PACC-5) score. The key secondary efficacy endpoint will assess the effect of JNJ-2056 on the propagation and/or accumulation of Tau pathology compared with placebo, as measured by Tau PET imaging.About ACI-35.030 (JNJ-2056).

主要终点将通过临床前AD认知综合5（PACC-5）评分评估认知能力下降。通过Tau PET成像测量，与安慰剂相比，关键的次要疗效终点将评估JNJ-2056对Tau病理学传播和/或积累的影响。关于ACI-35.030（JNJ-2056）。

ACI-35.030, derived from AC Immune’s SupraAntigen® platform, has been shown in clinical studies to induce a strong polyclonal antibody response that matures and is maintained against key pathological forms of Tau believed to drive Tau aggregation and disease progression. ACI-35.030 is designed to enhance the formation of broad-spectrum protective antibodies against pTau.

来自AC Immune的SupraAntigen®平台的ACI-35.030已在临床研究中显示可诱导强烈的多克隆抗体反应，该反应成熟并针对据信驱动Tau聚集和疾病进展的Tau的关键病理形式进行维持。ACI-35.030旨在增强针对pTau的广谱保护性抗体的形成。

This investigational candidate has the potential to reduce pathological Tau spreading in the early stages of AD, and thereby may reduce or prevent disease progression..

这种研究性候选药物有可能减少 AD 早期阶段的病理性 Tau 扩散，从而可能减少或预防疾病进展。

About AC Immune SA

关于AC Immune SA

AC Immune SA is a clinical-stage biopharmaceutical company and a global leader in precision medicine for neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and NeuroOrphan indications driven by misfolded proteins. The Company’s two clinically validated technology platforms, SupraAntigen® and Morphomer®, fuel its broad and diversified pipeline of first- and best-in-class assets, which currently features sixteen therapeutic and diagnostic programs, five of which are currently in Phase 2 clinical trials and one of which is in Phase 3.

AC Immune SA是一家临床阶段的生物制药公司，是神经退行性疾病（包括阿尔茨海默氏病，帕金森氏病和由错误折叠的蛋白质驱动的神经孤儿适应症）精准医学的全球领导者。该公司的两个经过临床验证的技术平台SupraAntigen®和Morphomer®为其广泛多样的一流和一流资产提供了支持，目前拥有16个治疗和诊断计划，其中5个目前处于第二阶段临床试验，其中一个处于第三阶段。

AC Immune has a strong track record of securing strategic partnerships with leading global pharmaceutical companies, resulting in substantial non-dilutive funding in potential milestone payments plus royalties.SupraAntigen® is a registered trademark of AC Immune SA in the following territories: AU, EU, CH, GB, JP, RU, SG and USA.

AC Immune在与全球领先的制药公司建立战略伙伴关系方面有着良好的记录，因此在潜在的里程碑付款和版税方面获得了大量非稀释性资金。SupraAntigen®是AC Immune SA在以下地区的注册商标：AU，EU，CH，GB，JP，RU，SG和USA。