Netherlands biotech Azafaros is poised to start phase 3 trials of its lead drug for a trio of rare lysosomal storage disorders (LSDs), helped by €132 million ($147 million) in second-round financing from European venture capital firms.

荷兰生物技术公司 Azafaros 准备启动其主导药物针对三种罕见溶酶体贮积症 (LSD) 的第三阶段试验，得益于欧洲风险投资公司提供的 1.32 亿欧元（1.47 亿美元）的第二轮融资。

The Series B – led by Jeito Capital and Forbion Growth – will be used to fund two late-stage trials of Azafaros' nizubaglustat in Niemann-Pick disease Type C (NPC) disease and GM1/GM2 gangliosidoses, which lead to the accumulation of lipids in organs, including the brain, and cause neurological problems and developmental delays..

B轮融资由Jeito Capital和Forbion Growth领投，将用于资助Azafaros公司的nizubaglustat在C型尼曼-匹克病（NPC）以及GM1/GM2神经节苷脂沉积症的两项晚期试验，这些疾病会导致脂质在包括大脑在内的器官中积累，从而引发神经系统问题和发育迟缓。

The drug, a small-molecule inhibitor of ceramide glucosyltransferase and non-lysosomal neutral glucosylceramidase (NLGase), was discovered in the lab of Professor Hans Aerts of the University of Leiden and – according to the biotech – has the potential to modify the course of all three diseases with oral daily dosing..

该药物是神经酰胺葡萄糖基转移酶和非溶酶体中性葡萄糖神经酰胺酶（NLGase）的小分子抑制剂，由莱顿大学汉斯·艾茨教授的实验室发现，并且据这家生物技术公司称，该药物有望通过每日口服给药来改变这三种疾病的病程。

Azafaros has completed a first-in-human clinical study in healthy subjects, showing a favourable safety and tolerability profile and positive pharmacokinetics and pharmacodynamics data, and plans to start the phase 3 trials before the end of this year.

阿扎法罗斯已完成一项在健康受试者中进行的首次人体临床研究，显示出良好的安全性和耐受性，并获得积极的药代动力学和药效学数据，计划在今年年底前开始第三阶段试验。

LSDs

溶酶体贮积症

are a group of over 70 diseases that are characterised by dysfunction of lysosomes – organelles that clear toxic materials and defective cellular components from cells – and most are inherited as autosomal recessive traits. These disorders are individually rare but collectively affect 1 in 5,000 live births..

是超过70种疾病的组群，其特征为溶酶体功能障碍——溶酶体是清除细胞内有毒物质和缺陷细胞成分的细胞器——并且大多数为常染色体隐性遗传。这些疾病单独来看较为罕见，但总体上每5000例活产中便有1例受到影响。

Part of what is behind Azafaros' enthusiasm for nizubaglustat is the drug's potential to provide treatment for multiple LSDs, regardless of their underlying disease mechanism. Most current therapies for LSDs are specific for a particular condition and, for many of them, no treatment exists at all other than palliative care..

Azafaros 对尼祖巴格司他表现出热情的背后原因之一在于，该药物有可能为多种溶酶体贮积症（LSDs）提供治疗，而与其潜在的致病机制无关。目前大多数 LSDs 的疗法仅针对特定疾病，而其中许多疾病除了姑息治疗外，尚无任何治疗方法。

The drug has been awarded orphan drug designations in both the US and Europe, as well as fast-track status in the US.

该药物已在美国和欧洲获得孤儿药资格认定，并在美国获得快速通道资格。

'This successful Series B round marks a significant milestone for Azafaros, allowing us to accelerate the development of nizubaglustat and leverage our scientific understanding and competencies to bring additional candidates into development,' said Stefano Portolano, Azafaros' chief executive.

“这次成功的B轮融资对Azafaros来说是一个重要的里程碑，使我们能够加速尼祖巴格斯塔特的开发，并利用我们的科学理解和能力将更多候选药物引入研发。” Azafaros首席执行官斯特凡诺·波尔托拉诺表示。

'The fact that we have been able to attract leading life sciences investors to join our existing, strong group of specialist investors is a testament to the impressive accomplishments of the team and the large unmet medical need that currently exists for patients with these hugely debilitating neurological diseases,' he added.

“我们能够吸引领先的生物科学投资者加入我们现有的强大专业投资者团队，这证明了团队取得的令人瞩目的成就，以及目前这些严重致残的神经系统疾病患者所面临的巨大未满足医疗需求，”他补充道。

'We look forward to bringing nizubaglustat to patients.'.

“我们期待将尼祖巴格拉司他带给患者。”

The financing was supported by Seroba, Pictet Group and existing investors Forbion Ventures, Schroders Capital, and BioGeneration Ventures.

融资得到了Seroba、Pictet集团以及现有投资者Forbion Ventures、Schroders Capital和BioGeneration Ventures的支持。