Alterity Therapeutics has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for its lead candidate, ATH434.

Alterity Therapeutics 的主要候选药物 ATH434 已获得美国食品药品监督管理局 (FDA) 的快速通道资格。

ATH434 is an orally administered compound that targets the abnormal build-up of proteins linked to neurodegeneration. In preclinical studies, the drug has shown the ability to reduce α-synuclein pathology and maintain neuronal function by restoring normal iron regulation in the brain. Clinical trials have also demonstrated its ability to reach effective levels in the brain while maintaining a favourable safety profile..

ATH434 是一种口服化合物，靶向与神经退行性疾病相关的异常蛋白质积累。在临床前研究中，该药物已显示出通过恢复大脑正常铁调节来减少 α-突触核蛋白病理并维持神经元功能的能力。临床试验还表明，它能够在大脑中达到有效水平，同时保持良好的安全性。

MSA is a progressive disorder affecting the autonomic nervous system and movement control. Common symptoms include difficulty with coordination, posture, and bladder function, as well as blood pressure regulation issues. The disease is marked by the accumulation of α-synuclein in glial cells and neuronal loss in various parts of the brain.

多系统萎缩症（MSA）是一种影响自主神经系统和运动控制的进行性疾病。常见症状包括协调、姿势和膀胱功能障碍，以及血压调节问题。该疾病的特点是α-突触核蛋白在胶质细胞中积累，并且大脑不同部位出现神经元损失。

An estimated 15,000 people in the United States are affected by the condition..

据估计，美国有15,000人受到这种情况的影响。

The designation applies to the treatment of Multiple System Atrophy (MSA), a rare and debilitating neurodegenerative disease for which no approved therapy currently exists.

该指定适用于多系统萎缩症（MSA）的治疗，这是一种罕见且致残的神经退行性疾病，目前尚无获批的疗法。

The Fast Track designation is designed to speed up the development and regulatory review process for drugs that address serious medical conditions with high unmet needs.

快速通道资格旨在加速针对高未满足需求的严重医疗状况的药物的开发和监管审查过程。

Biomarker analysis demonstrated that ATH434 was associated with stabilisation or reduction of iron accumulation in affected areas of the brain and potential preservation of brain volume. The treatment was generally well tolerated, with adverse events comparable to those seen in the placebo group and no treatment-related serious adverse events reported..

生物标志物分析表明，ATH434与稳定或减少大脑受影响区域的铁积累以及潜在的脑容量保留有关。该治疗总体耐受性良好，不良事件与安慰剂组相当，且未报告与治疗相关的严重不良事件。

In addition to the Fast Track designation, ATH434 has already received Orphan Drug Designation from both the U.S. FDA and the European Commission for the treatment of MSA. A second open-label Phase 2 trial focusing on patients with more advanced MSA is currently ongoing.

除了快速通道资格认定外，ATH434 已经获得美国 FDA 和欧洲委员会授予的孤儿药资格认定，用于治疗 MSA。目前，一项针对病情更严重的 MSA 患者的第二项开放标签 2 期试验正在进行中。