Apellis Pharmaceuticals, Inc.
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- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) is a commercial-stage global biopharmaceutical company focused on developing and commercializing targeted C3 therapies for complement-driven diseases in areas including hematology, ophthalmology, nephrology, and neurology; its core products comprise EMPAVELI (pegcetacoplan injection for subcutaneous use), approved for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) and for adult and pediatric patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) to reduce proteinuria, Aspaveli (pegcetacoplan for subcutaneous use), the ex-U.S. brand equivalent of EMPAVELI approved for PNH, and SYFOVRE (pegcetacoplan injection for intravitreal use), indicated for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Founded in 2009 and headquartered in Waltham, Massachusetts, the company operates in the United States, Europe, Switzerland, and other international markets through commercialization rights and partnerships; it maintains systemic pegcetacoplan rights worldwide and worldwide intravitreal rights for its GA program. Recent developments include the U.S. FDA approval of EMPAVELI label expansion for C3G and IC-MPGN in 2025 with supportive Phase 3 VALIANT data demonstrating proteinuria reduction, a capped royalty purchase agreement with Sobi providing up to $300 million upfront ($275 million cash plus milestones) in exchange for 90% of ex-U.S. Aspaveli royalties to bolster its balance sheet amid pursuit of profitability, ongoing SYFOVRE market leadership at 60% share in GA treatment, and collaborations such as with Beam Therapeutics for gene editing technologies targeting neonatal Fc receptor and with Genpharm for Middle East/North Africa commercialization of intravitreal pegcetacoplan.
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