Single Therapy for Wet AMD & Geographic Atrophy

描述（由申请人提供）：大约85%的总年龄相关性黄斑变性（AMD）患者具有干性形式。地图状萎缩（GA）是干性AMD的晚期形式，由视网膜上皮细胞变性引起。虽然GA不像湿性AMD那么常见，但在美国，它造成了10-20%的法律的失明。目前没有FDA批准的GA治疗方法。然而，一项评估药物Lampalizumab（一种靶向替代补体途径的抗因子D治疗性抗体）的试验的最新结果提供了令人信服的证据，证明替代补体途径在GA患者视力丧失的发展中起着关键作用。补体介导的RPE、感光细胞和布鲁赫膜的破坏是GA的标志。Lampalizumab对干性AMD患者的获益率接近44%。NovelMed的主要候选药物NM 5072是一种上级AP抑制剂，与拉帕珠单抗相比，预计在较低剂量下更有效，持续时间更长。我们期望NM 5072在可以知道患者稍后可能发展（如果不治疗）这两种病理中的哪一种之前抑制CNV和GA。NovelMed的主要候选药物是一种选择性补体旁路抑制剂。分子; a）以高亲和力和选择性结合靶点，B）以有效剂量水平防止新血管的形成，c）防止炎症，和d）对于改进的给药方案具有更长的药代动力学。考虑到旁路补体途径在GA中的作用，我们期望我们的药物防止CNV和GA的发作和进展而不影响组织修复。目前还没有治疗方法可以同时控制导致视力丧失的湿性和干性AMD。该药物已按照FDA规格生产，并准备进行GLP毒性研究和人体临床试验。我们打算开发NM 5072作为湿性和干性AMD的单一疗法。我们期望NM 5072在AMD的早期阶段抑制CNV和GA两者，然后才能知道患者稍后可能发展两种病理中的哪一种（如果不治疗）。通过在CNV和GA发作之前治疗AMD，可以完全预防AMD驱动的视力丧失。

DESCRIPTION (provided by applicant): Approximately 85% of the total age-related macular degeneration (AMD) patients have the Dry form. Geographic atrophy (GA) is the advanced form of Dry AMD and is caused by the degeneration of the retinal epithelial cells. While GA is less common than Wet AMD, it is responsible for 10-20% of legal blindness in the US. There is currently no FDA approved treatment for GA. However, the latest results from a trial evaluating the drug Lampalizumab, an anti-Factor D therapeutic antibody targeted to the alternative complement pathway, provide convincing evidence that the alternative complement pathway plays a key role in the development of vision loss in patients with GA. Complement-mediated destruction of RPE, photoreceptor cells, and Bruch's membrane are the hallmarks of GA. Lampalizumab demonstrated nearly 44% benefit for patients with Dry AMD. NovelMed's lead drug candidate, NM5072, is a superior AP inhibitor and is expected to be more effective and longer lasting, at lower dosages, when compared to Lapalizumab. We expect NM5072 to inhibit both the CNV and the GA before it can be known which of the two pathologies a patient might develop later on (if not treated). NovelMed's lead drug candidate is a selective inhibitor of the alternative complement pathway. The molecule; a) binds the target with high affinity and selectivity, b) prevents formation of new blood vessels at potent dose levels, c) prevents inflammation, and d) has longer pharmacokinetics for improved dosing schedule, Given the role of the alternative complement pathway in GA, we expect our drug to prevent the onset and progression of CNV and GA without affecting tissue repair. There is no therapy available today that could control both the Wet and the Dry form of AMD that leads to vision loss. The drug has been manufactured to FDA specifications and ready for GLP toxicity studies and for human clinical trial. We intend to develop NM5072 as a single therapy for both Wet and Dry AMD. We expect NM5072 to inhibit both CNV and GA at an early stage of AMD, before it can be known which of the two pathologies a patient might develop later on (if left untreated). By treating AMD prior to onset of CNV and GA, AMD driven vision loss could be prevented entirely.