Apellis Pharmaceuticals, Inc. (NASDAQ: APLS)

Apellis Pharmaceuticals Inc is a commercial stage biopharmaceutical company focused on the discovery development and commercialization of novel therapeutic compounds to treat diseases with high unmet needs through inhibition of the complement system. The company believes that targeting C3 the central protein of the complement cascade provides a broad mechanism to control diseases driven by excessive complement activation. Its lead product pegcetacoplan is formulated for both intravitreal administration to treat eye diseases and subcutaneous injection for systemic indications.

The company generates revenue primarily from sales of its two approved products SYFOVRE and EMPAVELI. SYFOVRE is approved for geographic atrophy secondary to age related macular degeneration and generated US net product revenue of 586.9 million dollars in 2025 and 611.9 million dollars in 2024. EMPAVELI is approved for paroxysmal nocturnal hemoglobinuria and for C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis and generated US net product revenue of 102.4 million dollars in 2025 and 98.1 million dollars in 2024. In addition the company receives royalties from its collaboration partner Sobi amounting to 13.2 million dollars in 2025 and 18.4 million dollars in 2024. Apellis retains exclusive United States commercialization rights for both products while Sobi holds exclusive ex United States rights for systemic pegcetacoplan.

Apellis holds a leading position in the complement inhibition space with its proprietary molecule pegcetacoplan that targets C3 the central protein of the complement cascade. In geographic atrophy the company faces competition from Astellas Pharma's avacincaptad pegol a C5 inhibitor and from multiple early stage programs such as ANX007 from Annexon Biosciences and pozelimab plus cemdisiran from Regeneron and Alnylam. In paroxysmal nocturnal hemoglobinuria the main competitors are eculizumab and ravulizumab both C5 inhibitors marketed by AstraZeneca and newer oral agents including danicopan iptacopan and crovalimab. The company's competitive advantage stems from its ability to block complement at the C3 level providing broader pathway inhibition compared with upstream or downstream inhibitors. Additionally Apellis maintains a robust pipeline that includes a next generation combination of SYFOVRE with an siRNA aimed at comprehensively blocking complement activity in the retina and the choroid and early stage programs targeting the neonatal Fc receptor through its collaboration with Beam Therapeutics. The company also leverages its expertise in complement immunology to explore other targets within the cascade and to develop combination therapies that may enhance durability of response. Apellis benefits from strong intellectual property protection with numerous patents covering pegcetacoplan and related technologies that extend into the 2040s.

The company serves patients suffering from rare diseases such as geographic atrophy paroxysmal nocturnal hemoglobinuria C3 glomerulopathy primary immune complex membranoproliferative glomerulonephritis and other complement mediated conditions. Based on published estimates Apellis notes that approximately 1.5 million people in the United States live with geographic atrophy and about 4.7 thousand patients have paroxysmal nocturnal hemoglobinuria in the United States. It also cites an estimated 5 thousand individuals in the United States living with C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis and around 13 thousand primary FSGS patients in the United States. Its customers include retina specialists ophthalmologists hematologists nephrologists and other health care providers who prescribe its therapies. The company also works with specialty pharmacies distributors and payers to ensure patient access and reimbursement. Apellis provides patient support programs such as ApellisAssist to help with insurance navigation financial assistance and ongoing education.