CINCINNATI--(BUSINESS WIRE)--Asha Therapeutics (Asha) (www.ashatherapeutics.com), a life sciences company designing de novo disease modifying medicines for neurodegenerative diseases with high unmet medical need, announced today the nomination of a development candidate, ASHA-624 targeting SARM1 as a potential disease modifying therapy for Amyotrophic Lateral Sclerosis (ALS) with additional indications in Chemotherapy-Induced Peripheral Neuropathy (CIPN), Glaucoma, and traumatic brain and spinal cord injuries.

辛辛那提--（商业新闻短讯）--Asha Therapeutics（www.ashatherapeutics.com）是一家生命科学公司，为未满足医疗需求的神经退行性疾病设计新的疾病缓解药物，今天宣布提名一名开发候选人Asha-624，靶向SARM1作为肌萎缩侧索硬化症（ALS）的潜在疾病缓解疗法，并在化疗引起的周围神经病变（CIPN），青光眼以及创伤性脑和脊髓损伤中有其他适应症。

This announcement follows the Company’s presentation of robust efficacy and safety data on its second lead neurology program, ASHA-091 in Alzheimer’s disease and Parkinson’s disease by Chief Scientific Officer & Scientific Co-founder Dr. Bradlee Heckmann earlier this month at the AD/PD 2024 International Conference in Lisbon, Portugal (see full release here)..

本月早些时候，在葡萄牙里斯本举行的AD/PD 2024国际会议上，首席科学官兼科学联合创始人布拉德利·赫克曼博士（BradleeHeckmann）介绍了该公司第二个主要神经病学项目ASHA-091在阿尔茨海默氏病和帕金森氏病中的强大疗效和安全性数据（请参阅此处的完整版本）。。

Dr. Michael Gold, MD, MS, a member of Asha’s Scientific Advisory Board noted, “SARM-1 is a well-validated therapeutic target that could yield novel therapies for patients suffering from a range of both central and peripheral nervous disorders. ASHA-624 prevents the activation of SARM-1 using a completely novel approach that has the potential to deliver a therapy with robust clinical efficacy and few, if any, off-target side effects.

Asha科学顾问委员会成员迈克尔·戈尔德博士（Michael Gold，MD，MS）指出：“SARM-1是一种经过充分验证的治疗靶点，可以为患有一系列中枢神经和周围神经疾病的患者提供新的治疗方法。Asha-624使用一种完全新颖的方法阻止SARM-1的激活，这种方法有可能提供具有强大临床疗效且几乎没有脱靶副作用的治疗方法。

As a veteran CNS drug developer, I am excited to see this compound continue its progress towards clinical trials.”.

作为一名经验丰富的中枢神经系统药物开发人员，我很高兴看到这种化合物继续朝着临床试验的方向发展。”。

“Nomination of ASHA-624 as a development candidate for clinical translation in ALS, alongside Asha’s previously nominated ASHA-091 compound with indications in Alzheimer’s disease, Parkinson’s disease, and ALS, is a key juncture for Asha in supporting our mission of providing novel disease modifying therapies for conditions with current treatment options limited largely to symptomatic standard of care.

“提名ASHA-624作为ALS临床翻译的发展候选者，以及ASHA先前提名的具有阿尔茨海默氏病，帕金森氏病和ALS适应症的ASHA-091化合物，是ASHA支持我们为目前治疗选择主要限于对症护理标准的疾病提供新型疾病缓解疗法的使命的关键时刻。

We are committed through our work to the transformation of patient outcomes, and believe ASHA-624 is a significant step forward towards achieving that goal,” commented Dr. Heckmann, PhD..

我们致力于通过我们的工作来改变患者的预后，并相信ASHA-624是朝着实现这一目标迈出的重要一步，”Heckmann博士评论道。。

About ASHA-624: ASHA-624 is a first-in-class, novel intra-molecular glue compound that exploits the normal biology of SARM1, a key protein that promotes axonal degeneration and neurodegeneration by selectively “gluing” activated SARM1 into an inactive conformation, leading to robust neuroprotection through the prevention of axon and neuron loss.

关于ASHA-624：ASHA-624是一种一流的新型分子内胶化合物，它利用了SARM1的正常生物学特性，SARM1是一种关键蛋白，通过选择性地将活化的SARM1“粘合”成无活性构象来促进轴突变性和神经变性，通过预防轴突和神经元丢失而产生强大的神经保护作用。

In ALS, the activation of SARM1 leads to axonal loss, neurodegeneration and ultimately motor dysfunction. ASHA-624 was designed to inhibit SARM1 with hyper selectivity, and in preclinical studies has demonstrated a robust safety profile as a functional cure in models of ALS. ASHA-624 therapeutic intervention in preclinical models of ALS reversed motor impairment and dysfunction to similar levels as healthy controls while placebo treated groups exhibited continual and exacerbated decline in motor function..

在ALS中，SARM1的激活会导致轴突丢失，神经变性并最终导致运动功能障碍。ASHA-624被设计用于以高选择性抑制SARM1，并且在临床前研究中已经证明在ALS模型中作为功能性治愈具有强大的安全性。ASHA-624在ALS临床前模型中的治疗干预将运动障碍和功能障碍逆转至与健康对照相似的水平，而安慰剂治疗组表现出持续且加剧的运动功能下降。。

About ALS: Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder that affects nerve cells (neurons) in the spinal cord and brain. Loss of motor neurons in ALS patients leads to loss of muscle control, resulting in impaired motor function.

关于肌萎缩侧索硬化症（ALS）：肌萎缩侧索硬化症（ALS），也称为Lou Gehrig病，是一种进行性神经退行性疾病，会影响脊髓和大脑中的神经细胞（神经元）。ALS患者运动神经元的丧失导致肌肉控制的丧失，导致运动功能受损。

ALS ultimately affects the regulation and control of muscles which are required for speaking, eating, and breathing. There is currently no available cure for ALS, and the disease is fatal..

ALS最终会影响说话，进食和呼吸所需肌肉的调节和控制。目前尚无治疗ALS的方法，这种疾病是致命的。。

Asha anticipates the advancement of ASHA-624 and ASHA-091 into clinical trials by late-2024 for their respective disease indications.

Asha预计Asha-624和Asha-091将在2024年底之前进入各自疾病适应症的临床试验。

About Asha Therapeutics: Asha Therapeutics (www.ashatherapeutics.com) is a life sciences company at the forefront of a new era of precision drug design, leveraging the power of its proprietary PRISM™ technology to design de novo compounds to create disease-modifying and curative therapeutics for neurological and infectious diseases with high unmet medical need..

关于Asha Therapeutics：Asha Therapeutics（www.ashatherapeutics.com）是一家生命科学公司，处于精确药物设计新时代的前沿，利用其专有PRISM™技术的力量设计从头化合物，为医疗需求未得到满足的神经和传染病创造疾病缓解和治疗疗法。。

Asha's lead therapeutic programs, ASHA-624 and ASHA-091 with indications in Amyotrophic Lateral Sclerosis, Parkinson's disease and Alzheimer’s disease, are anticipated to enter clinical trials by late-2024 and are disease modifying therapeutics.

Asha的主要治疗方案Asha-624和Asha-091具有肌萎缩侧索硬化症，帕金森病和阿尔茨海默氏病的适应症，预计将在2024年底进入临床试验，并且是改善疾病的疗法。