REYKJAVIK, Iceland and FORT WORTH, Texas, March 12, 2024 (GLOBE NEWSWIRE) -- Arctic Therapeutics ehf, an Icelandic drug development company and partner Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced approval by the European Medicines Agency (EMA) for initiation of the first clinical trial evaluating AT-001 (Nacuity’s NPI-001) for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA) in Iceland.

冰岛雷克雅未克和德克萨斯州沃思堡，2024年3月12日（环球通讯社）--冰岛药物开发公司Arctic Therapeutics ehf和合作伙伴Nacity Pharmaceuticals，Inc.，一家临床阶段的生物制药公司，开发治疗色素性视网膜炎、白内障和其他由氧化应激引起的疾病，今天宣布获得欧洲药品管理局（EMA）的批准启动第一项评估AT-001（Nacity的NPI-001）治疗冰岛遗传性胱抑素C淀粉样血管病（HCCAA）的临床试验。

HCCAA is an ultra rare, serious condition characterized by the accumulation of amyloid in the brain's blood vessels, which can lead to cerebral hemorrhage, stroke, and progressive neurological deterioration, including dementia. Current treatment options are severely limited, focusing primarily on symptom management rather than addressing the underlying cause of the disease.

HCCAA是一种极为罕见的严重疾病，其特征是大脑血管中淀粉样蛋白的积累，可导致脑出血，中风和进行性神经功能恶化，包括痴呆。目前的治疗选择受到严重限制，主要集中在症状管理上，而不是解决疾病的根本原因。

AT-001 is a differentiated, purified, prescription-quality, GMP-grade N-acetylcysteine amide (NACA). “EMA's approval paves the way for a comprehensive investigation into the safety and efficacy of a novel therapeutic approach designed to halt or reverse the progression of HCCAA. Our study aims to explore the potential of AT-001 in modifying the disease's course, improving patient outcomes, and ultimately, saving lives,” said Ivar Hakonarson, CEO and co-founder of Arctic.

AT-001是一种分化的，纯化的，处方质量的GMP级N-乙酰半胱氨酸酰胺（NACA）。“EMA的批准为全面调查旨在阻止或逆转HCCAA进展的新型治疗方法的安全性和有效性铺平了道路。我们的研究旨在探索AT-001在改变疾病进程，改善患者预后以及最终挽救生命方面的潜力，”Arctic首席执行官兼联合创始人伊瓦尔·哈科纳森（IvarHakonarson）说。

In September 2022, Nacuity and Arctic entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of Nacuity’s NPI-001 under the name AT-001 for HCCAA in Iceland and related forms of amyloid angiopathies (CAAs) worldwide. Nacuity is also currently evaluating NPI-001 in Phase 1/2 clinical trials in Australia for retinitis pigmentosa.

2022年9月，Nacity和Arctic签订了独家许可协议和战略合作伙伴关系，以开发和商业化Nacity的NPI-001，其名称为AT-001，用于冰岛HCCAA和全球相关形式的淀粉样血管病（CAA）。Nacity目前还在澳大利亚的1/2期视网膜色素变性临床试验中评估NPI-001。