Apellis Pharmaceuticals, Inc. (APLS)

業務介紹

Apellis Pharmaceuticals, Inc.（Apellis製藥公司）是一家商業化階段的生物製藥公司，專注於通過抑制補體系統來發現、開發和商業化治療化合物，用於自身免疫和炎症性疾病。該公司的主要產品候選藥是pegcetacoplan，目前正在進行III期臨床試驗，用於治療與年齡相關性黃斑變性和夜間發作性血紅蛋白尿症（PNH）相關的地理性萎縮（GA）。它還開發了EMPAVELI（全身性pegcetacoplan），用於治療寒凝集素病（CAD）和血液學中的造血幹細胞移植相關性血栓性微血管病變（HSCT-TMA）；腎臟學中的C3腎小球病（C3G）和免疫複合物膜增生性腎小球腎炎（IC-MPGN）；以及神經學中的肌萎縮性脊髓側索硬化症（ALS）。此外，該公司還開發了APL-2006，一種雙特異性C3和VEGF抑制劑，用於治療補體介導的疾病；APL-1030，一種C3抑制劑，用於治療多種神經退行性疾病；以及EMPAVELI和小干擾RNA（siRNA）的組合，用於減少肝臟產生C3蛋白質的產量。該公司與瑞典奧菲倫生物制劑股份有限公司（Swedish Orphan Biovitrum AB）簽訂了合作和許可協議，共同開發pegcetacoplan；並與Beam Therapeutics Inc.進行研究合作，專注於使用Beam的基因編輯技術發現補體驅動疾病的新治療方法。Apellis Pharmaceuticals, Inc.成立於2009年，總部位於麻薩諸塞州沃爾瑟姆市。

Apellis Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases. The company's lead product candidate is pegcetacoplan that is in Phase III clinical trials for the treatment of geographic atrophy (GA) in age-related macular degeneration and paroxysmal nocturnal hemoglobinuria (PNH) diseases. It also develops EMPAVELI (systemic pegcetacoplan) for the treatment of cold agglutinin disease (CAD), and hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) in hematology; C3 glomerulopathy (C3G), and immune complex membranoproliferative glomerulonephritis (IC-MPGN) in nephrology; and amyotrophic lateral sclerosis (ALS) in neurology. In addition, the company develops APL-2006, a bispecific C3 and VEGF inhibitor for treating complement-mediated disorders; APL-1030, a C3 inhibitor for the treatment of multiple neurodegenerative diseases; and the combination of EMPAVELI and a small interfering RNA, or siRNA for reducing the production of C3 proteins by the liver. It has a collaboration and license agreement with Swedish Orphan Biovitrum AB (publ) to co-develop pegcetacoplan; and a research collaboration with Beam Therapeutics Inc. focused on the use of Beam's base editing technology to discover new treatments for complement-driven diseases. Apellis Pharmaceuticals, Inc. was incorporated in 2009 and is based in Waltham, Massachusetts.

公司網址

https://www.apellis.com