Apellis Pharmaceuticals develops first‑in‑class C3‑targeted inhibitors to treat diseases caused by overactivation of the complement system. Its approved drug pegcetacoplan (SYFOVRE®/EMPAVELI®) blocks all three complement pathways and is used for geographic atrophy, paroxysmal nocturnal hemoglobinuria, and complement‑mediated kidney disorders, while its pipeline extends C3 inhibition to additional rare and renal diseases.
Funding
$275M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
SOFounders
Product
Problem
Overactivation of the complement cascade, particularly the central protein C3, leads to tissue damage in a range of serious and rare diseases—including geographic atrophy, paroxysmal nocturnal hemoglobinuria, and complement-mediated kidney disorders—where few or no effective therapies exist.
Solution
Apellis focuses on controlling complement activation by developing first‑in‑class C3‑targeted inhibitors. Its lead products, pegcetacoplan (marketed as SYFOVRE® and EMPAVELI®), are approved for geographic atrophy and paroxysmal nocturnal hemoglobinuria, and have received approval for C3 glomerulopathy and primary IC‑MPGN. By inhibiting C3, these agents block all three activation pathways, providing comprehensive disease modulation. The company’s pipeline extends the C3‑targeting approach to additional indications, including focal segmental glomerulosclerosis, delayed graft function, and novel oral and gene‑edited complement therapies. Clinical development is supported by a deep‑science platform that integrates complement biology, protein engineering, and advanced delivery modalities to deliver safe and effective treatments for high‑unmet‑need patients.
Target Audience
Primary customers are patients with complement‑driven diseases such as geographic atrophy, paroxysmal nocturnal hemoglobinuria, and rare kidney disorders, as well as ophthalmologists, hematologists, nephrologists, and healthcare systems that prescribe these therapies.
Features
- Pegcetacoplan, a C3‑targeted peptide inhibitor, approved for multiple indications (geographic atrophy, PNH, C3G, IC‑MPGN)
- Mechanistic coverage of all three complement activation pathways through central C3 inhibition
- Ongoing pipeline of C3‑based therapies in pivotal Phase II/III trials for kidney disease (FSGS, delayed graft function) and other rare disorders
- Development of next‑generation modalities, including oral complement inhibitors and gene‑edited FcRn or complement therapies (Beam platform)
- Robust clinical trial infrastructure with compassionate use and early‑access programs for patients lacking alternatives