New preclinical data presented at CTAD demonstrates exciting potential for a new drug approach for Alzheimer's disease, reducing brain tau pathology by ~70%

CTAD提供的新临床前数据显示，针对阿尔茨海默病的新药方法具有令人兴奋的潜力，可将脑tau病理学减少约70%

CAMBRIDGE, Mass., Oct. 30, 2023 /PRNewswire/ -- Aquinnah Pharmaceuticals announced preclinical research findings for a novel therapeutic designed to slow or stop the progression of Alzheimer's disease and related disorders. Aquinnah is a leader in pharmaceutical approaches targeting stress granule biology, which underlies the pathology of a wide range of neurodegenerative disorders.

马萨诸塞州剑桥市，2023年10月30日/PRNewswire/-Aquinnah Pharmaceuticals宣布了一种旨在减缓或阻止阿尔茨海默病和相关疾病进展的新型治疗药物的临床前研究结果。Aquinnah是针对压力颗粒生物学的药物方法的领导者，这是各种神经退行性疾病病理学的基础。

The Aquinnah team announced today that their lead compound targets the interaction of tau with stress granules and removes ~70% of tau pathology in an Alzheimer's animal model with advanced stage disease, measured using three different markers of pathological tau that also increase in patients as their disease progresses.

Aquinnah团队今天宣布，他们的主要化合物针对tau与应激颗粒的相互作用，并在患有晚期疾病的阿尔茨海默病动物模型中去除约70%的tau病理学，使用三种不同的病理学tau标记物测量，这些标记物在患者中也随着患者的增加而增加他们的疾病进展。

Importantly, the Aquinnah compound is expected to be administered orally as a pill, which is generally preferred by patients over injections that are required currently for approved Alzheimer's immunotherapies. .

重要的是，Aquinnah化合物预计将作为丸剂口服给药，患者通常更喜欢目前批准的阿尔茨海默氏症免疫疗法所需的注射剂。

Administration of the compound once daily in preclinical models was sufficient to achieve a strong reduction of abnormal tau protein, which is the key protein contributing to loss of memory and cognition in Alzheimer's disease. Because Aquinnah's compound acts through a unique approach, this new compound also has the potential to be used in combination with anti-amyloid drugs, recently approved by the FDA.

在临床前模型中每天一次施用化合物足以实现异常tau蛋白的强烈减少，异常tau蛋白是导致阿尔茨海默病中记忆和认知丧失的关键蛋白。由于Aquinnah的化合物通过独特的方法起作用，这种新化合物也有可能与FDA最近批准的抗淀粉样蛋白药物联合使用。

If successful in clinical trials, Aquinnah's treatment could benefit patients suffering from Alzheimer's disease by reducing brain damage, restoring memory capabilities, improving brain function and slowing disease progression..

如果在临床试验中取得成功，Aquinnah的治疗可以通过减少脑损伤，恢复记忆能力，改善脑功能和减缓疾病进展来使患有阿尔茨海默病的患者受益。。

The company's Co-Founder and Chief Scientific Officer, Dr. Benjamin Wolozin MD, Ph.D., presented the research – 'Development of Orally Available, Brain Penetrant Compound Reducing Tau Pathology' – at the 2023 Clinical Trials on Alzheimer's Disease (CTAD) conference last week in Boston.

该公司的联合创始人兼首席科学官Benjamin Wolozin博士博士在上周的2023年阿尔茨海默病（CTAD）临床试验会议上介绍了这项研究-“开发口服，脑渗透化合物减少Tau病理学”。

'As the number of patients and families impacted by Alzheimer's disease continues to grow worldwide, new medical approaches to target tau pathology, alone or in combination with anti-amyloid therapeutics, will benefit these patients greatly. Research from around the world, including the numerous presentations at CTAD, show that decreasing pathological tau, correlates strongly with improved cognitive function,' said Dr.

“随着受阿尔茨海默病影响的患者和家庭数量在全球范围内持续增长，单独或与抗淀粉样蛋白治疗相结合的针对tau病理学的新医学方法将使这些患者受益匪浅。来自世界各地的研究，包括CTAD的大量介绍，表明减少病理性tau，与改善认知功能密切相关。

Wolozin. 'We're excited about these results and the potential to give Alzheimer's patients a therapeutic agent that will not just delay the disease's progression, but potentially improve their cognitive functioning. Our success with animal models, equivalent to advanced stage Alzheimer's patients, suggests that our compound has exactly this ability to turn the tide for patients suffering from cognitive loss and have or are facing impending dementia caused by Alzheimer's disease.' .

Wolozin我们对这些结果以及为阿尔茨海默病患者提供治疗剂的潜力感到兴奋，这种治疗剂不仅可以延缓疾病的进展，还可以改善他们的认知功能。我们在相当于晚期阿尔茨海默病患者的动物模型方面取得的成功表明，我们的化合物正具有这种能力，可以为患有认知丧失并且患有或即将面临由阿尔茨海默病引起的痴呆症的患者扭转局势。

In addition to its application to Alzheimer's disease, Aquinnah's therapeutic is a potential treatment for other diseases with tauopathies, including Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), Frontotemporal Lobar Dementia (FTD) and Traumatic Brain Injury (TBI).

除了应用于阿尔茨海默病外，Aquinnah的治疗方法还可用于治疗其他患有tau蛋白病的疾病，包括进行性核上性麻痹（PSP），皮质基底变性（CBD），额颞叶痴呆（FTD）和创伤性脑损伤（TBI）。

Aquinnah's CEO, Glenn Larsen, Ph.D., added, 'Alzheimer's disease affects millions of patients and their families each year, so these results and their potential impact are a significant and promising step forward in the fight against this pervasive disease. More importantly, our approach contrasts to existing and developing therapies which are administered through injections.

Aquinnah的首席执行官Glenn Larsen博士补充说，“阿尔茨海默病每年影响数百万患者及其家属，因此这些结果及其潜在影响是在抗击这种普遍性疾病方面迈出的重要而有希望的一步。更重要的是，我们的方法与通过注射给药的现有和开发疗法形成对比。

As a once-a-day pill, it will be a preferred method for patients, easier to take early in disease to prevent progression and be less costly compared to injectable drugs. Our strong results have attracted significant interest from the industry to collaborate with us in advancing the development of this promising therapeutic.'  .

作为一种每日一次的药丸，它将是患者的首选方法，与注射药物相比，更容易在疾病早期服用以预防进展并且成本更低。我们的强大成果吸引了业界的极大兴趣，与我们合作推动这一有希望的治疗方法的发展  .

Focused on innovative neurodegenerative research, Aquinnah Pharmaceuticals signed a collaboration agreement with Roche in 2022 to advance oral small molecules for ALS and other neurodegenerative diseases, by modulating TDP-43 pathology, which is the hallmark pathology in more than 95% of ALS patients.

专注于创新性神经退行性研究，Aquinnah Pharmaceuticals于2022年与罗氏签署了合作协议，通过调节TDP-43病理学推进ALS和其他神经退行性疾病的口服小分子，这是95%以上ALS患者的标志性病理学。

Aquinnah has received funding from Pfizer, AbbVie and Takeda, with additional grant funding from the National Institute of Health, the Alzheimer's Association, The Rainwater Foundation and the Mass Life Sciences Center. .

Aquinnah已获得辉瑞，AbbVie和武田制药的资助，并获得了美国国立卫生研究院，阿尔茨海默氏症协会，雨水基金会和大众生命科学中心的额外资助。

About Aquinnah Pharmaceuticals

关于Aquinnah制药公司

Aquinnah Pharmaceuticals is harnessing the power of stress granule biology to develop oral small molecule therapeutics in Neurodegenerative Diseases, focusing on Alzheimer's disease and other tauopathy diseases such as Progressive Supranuclear Palsy and Frontotemporal dementia and ALS, also known as Lou Gehrig's disease, by developing chemical modulators of stress granule pathology.

Aquinnah Pharmaceuticals利用应激颗粒生物学的力量开发神经退行性疾病的口服小分子疗法，重点研究阿尔茨海默病和其他tau蛋白病，如进行性核上性麻痹和额颞叶痴呆和ALS，也称为Lou Gehrig病，通过开发化学调节剂的压力颗粒病理学。

More information is available at www.aquinnahpharma.com..

更多信息请访问www.aquinnahpharma.com。。

SOURCE Aquinnah Pharmaceuticals

来源Aquinnah制药