文章核心观点 - SYFOVRE®(pegcetacoplan注射液)在GALE长期延长研究中的预设终点上展示了视觉功能获益 [1][3] - 这些数据进一步证实了SYFOVRE在治疗地理性萎缩(GA)方面的强大疗效和良好的安全性 [2] - 公司期待分享数据,进一步证明SYFOVRE是视网膜专科医生最常选择的GA治疗药物,强调其随时间推移不断增强的疾病进展减缓能力 [3] GALE长期延长研究 - GALE研究(n=792)是一项评估SYFOVRE长期安全性和疗效的III期、多中心、开放标签延长研究 [3] - 研究目标包括评估治疗相关不良事件的长期发生率和严重程度,以及GA病灶总面积的变化 [3] - 超过80%完成OAKS和DERBY研究的患者进入了GALE研究,此外还包括10名之前参与pegcetacoplan GA I期研究的患者 [3] - OAKS研究中的假手术转换组在前24个月接受假手术,之后24-36个月接受SYFOVRE治疗,这部分患者纳入了微视网膜测量分析 [4] OAKS和DERBY III期研究 - OAKS(n=637)和DERBY(n=621)是两项III期、多中心、随机、双盲、假手术对照研究,比较SYFOVRE每月和隔月给药与假手术在GA患者中的疗效和安全性 [5] - 研究评估了SYFOVRE每月和隔月给药在24个月时对GA病灶总面积的影响,结果显示两种给药方式均能减缓GA病灶进展,且随时间效果增强,安全性良好 [5] SYFOVRE®(pegcetacoplan注射液) - SYFOVRE是首个获批治疗地理性萎缩(GA)的药物,通过靶向C3发挥对补体级联反应的全面调控作用 [6][7] - SYFOVRE已在美国获批用于治疗由年龄相关性黄斑变性引起的GA [7] 地理性萎缩(GA) - GA是年龄相关性黄斑变性的一种进展型形式,是全球主要致盲原因之一,在美国影响超过100万人,全球影响500万人 [8] - GA是一种进行性、不可逆的疾病,病灶的扩大会破坏视网膜负责视觉的细胞,导致视力严重下降,严重影响患者的独立生活和生活质量 [8] - 平均只需2.5年,GA病灶就会开始影响中心视力负责的视网膜中心凹区 [8]

Apellis Pharmaceuticals (APLS) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a negative opinion for the second time on the marketing authorization application (MAA) for intravitreal pegcetacoplan to treat geographic atrophy (GA) in the EU. Despite this decision, there were several dissenting votes among CHMP members. Apellis plans to request a re-examination of the opinion and anticipates a final decision in the fourth quarter of 2 ...

文章核心观点 - 欧洲药品管理局(EMA)的药品委员会(CHMP)对Apellis公司的地理性萎缩(GA)治疗药物pegcetacoplan的上市申请(MAA)发表了否定意见 [1][2] - 但CHMP委员中有多名委员投票持反对意见,Apellis公司计划申请重新审查,预计在2024年第四季度获得最终意见 [2] - Apellis公司表示将继续致力于为欧洲GA患者提供这一重要治疗方法 [4] 地理性萎缩(GA)相关信息 - GA是一种严重的老年性黄斑变性,是全球主要致盲原因之一,在美国影响超过100万人,全球影响超过500万人 [5] - GA是一种进行性不可逆的疾病,由视网膜细胞损害导致的病灶扩大造成 [5] - GA导致的视力损失严重影响患者的独立生活和生活质量,平均只需2.5年病灶就会影响中央视力 [5] 关于pegcetacoplan - pegcetacoplan是一种针对补体C3的靶向疗法,旨在调节补体级联反应过度激活,这是多种严重疾病发生和进展的原因 [5] - 在美国,pegcetacoplan已获批用于治疗老年性黄斑变性引起的GA [5]

WALTHAM, Mass., June 28, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the marketing authorization application (MAA) of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA). Notably, there were multiple dissenting votes by CHMP members to this opinion. Apellis plans to seek re- examination and expects a final opinio ...

Apellis Pharmaceuticals (APLS) announced positive data from the late-stage GALE long-term extension study evaluating its marketed drug, Syfovre (pegcetacoplan injection), to treat patients with geographic atrophy (GA) secondary to age-related macular degeneration. Per the data readout, presented at a recent medical conference, treatment with Syfovre in the GALE study preserved visual function at 36 months in the GA patients. Per Apellis, Syfovre is the only approved GA treatment to show a benefit on visual ...

SYFOVRE is the only approved GA treatment to demonstrate a visual function benefit in a prespecified endpoint Data presented at the Clinical Trials at the Summit Meeting WALTHAM, Mass., June 10, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) today announced that SYFOVRE® (pegcetacoplan injection) preserved visual function at 36 months in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). These positive data from the GALE long-term extension study ...

It has been about a month since the last earnings report for Apellis Pharmaceuticals, Inc. (APLS) . Shares have lost about 1.9% in that time frame, underperforming the S&P 500. Will the recent negative trend continue leading up to its next earnings release, or is Apellis Pharmaceuticals due for a breakout? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at its most recent earnings report in order to get a better handle on the important catalysts. Apellis’ Q1 E ...

Apellis Pharmaceuticals (APLS) , in collaboration with Sobi, reported positive one-year results from the mid- stage study evaluating systemic pegcetacoplan to treat post-transplant recurrence of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Pegcetacoplan is being developed as a targeted C3 therapy that addresses the cause of these rare and debilitating kidney diseases. Per the data readout, seven patients in the phase II NOBLE study, representing 64% ...

Rapid reduction in disease activity seen at 12 weeks was sustained at one year 55% of patients showed zero C3c staining intensity, indicating that C3c deposits were cleared Highlighted as late-breaking oral presentation at the European Renal Association (ERA) Congress WALTHAM, Mass. and STOCKHOLM, Sweden, May 24, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Sobi® (STO:SOBI) today announced positive one-year results from the Phase 2 NOBLE study investigating systemic pegcetacoplan ...

Rapid reduction in disease activity seen at 12 weeks was sustained at one year 55% of patients showed zero C3c staining intensity, indicating that C3c deposits were cleared Highlighted as late-breaking oral presentation at the European Renal Association (ERA) Congress WALTHAM, Mass. and STOCKHOLM, Sweden, May 24, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Sobi® (STO:SOBI) today announced positive one-year results from the Phase 2 NOBLE study investigating systemic pegcetacoplan ...