LEHI, Utah, Oct. 7, 2024. Halia Therapeutics, a clinical-stage biopharmaceutical company at the forefront of developing innovative treatments for inflammatory and neurodegenerative diseases, today announced that the first healthy volunteer was dosed in its Phase 1 clinical trial evaluating its LRRK2 inhibitor, HT-4253, for a potential treatment for Alzheimer's Disease.'Dosing the first subject in the Phase 1 trial of HT-4253 marks a significant step in the fight against neurodegenerative diseases such as Alzheimer's disease,' said Dr.

犹他州莱希，2024年10月7日。Halia Therapeutics是一家临床阶段的生物制药公司，处于开发炎症和神经退行性疾病创新治疗方法的前沿，今天宣布，第一名健康志愿者在其第一阶段临床试验中服用了LRRK2抑制剂HT-4253，以评估其治疗阿尔茨海默氏病的潜力在HT-4253的第一阶段试验中给第一个受试者服用药物，标志着在对抗阿尔茨海默病等神经退行性疾病方面迈出了重要的一步。

David Bearss, CEO of Halia Therapeutics. 'We believe that HT-4253 will provide a groundbreaking treatment option for patients who currently have few choices to prevent or delay Alzheimer's onset, as the drug targets the underlying neuroinflammation linked to causing neurocognitive disease.'The Phase 1 clinical trial (ClinicalTrials.gov ID: NCT06537817) is a first-in-human, randomized, placebo-controlled, double-blind study evaluating the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of HT-4253.

Halia Therapeutics首席执行官戴维·贝尔斯（DavidBearss）我们相信，HT-4253将为目前几乎没有预防或延缓阿尔茨海默病发作选择的患者提供开创性的治疗选择，因为该药物针对与引起神经认知疾病有关的潜在神经炎症。”第一阶段临床试验（ClinicalTrials.gov ID:NCT06537817）是首次评估HT-4253的安全性，耐受性，药代动力学（PK）和药效学（PD）的人体，随机，安慰剂对照，双盲研究。

This study will employ a single ascending dose (SAD) and multiple ascending dose (MAD) format, designed to explore the potential of HT-4253 to inhibit LRRK2—a critical enzyme designed to explore the potential of HT-4253 to inhibit LRRK2. Recent human genetic studies have revealed specific variants of the Rab10 gene protect individuals with the APOE4 genotype—a known risk factor for Alzheimer's disease.

本研究将采用单次递增剂量（SAD）和多次递增剂量（MAD）格式，旨在探索HT-4253抑制LRRK2的潜力，LRRK2是一种旨在探索HT-4253抑制LRRK2潜力的关键酶。最近的人类遗传学研究表明，Rab10基因的特定变异可以保护APOE4基因型的个体，APOE4基因型是阿尔茨海默病的已知危险因素。

These variants inhibit Rab10, a key protein activated by LRRK2, thus reducing neuroinflammation and offering neuroprotection. Halia's LRRK2 inhibitor, HT-4253, mimics these Rab10 variants by blocking LRRK2, which in turn prevents Rab10 activation. This mechanism shows promise for preventing or delaying Alzheimer's onset in APO.

这些变体抑制由LRRK2激活的关键蛋白Rab10，从而减少神经炎症并提供神经保护。Halia的LRRK2抑制剂HT-4253通过阻断LRRK2来模拟这些Rab10变体，从而阻止Rab10的激活。这种机制有望预防或延缓载脂蛋白阿尔茨海默病的发作。