Apellis Pharmaceuticals Inc.

APLS

Held by 13 specialist biotech funds

High Convergence

13 funds opened new positions. Next phase3 readout (Pegcetacoplan): Nov 2025. Short interest: 14.6% of float.

BiotechnologyCEO: Cedric FrancoisIPO: 2017Website ↗

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.

Recent News & Research

PubMed

Pegcetacoplan in idiopathic and familial pediatric C3 glomerulopathy.

# Apellis Investment Summary Pegcetacoplan (Apellis's complement C3 inhibitor) demonstrated efficacy in pediatric C3 glomerulopathy patients, expanding the drug's addressable market beyond adult populations and supporting label extension potential. This pediatric data strengthens Apellis's competitive position in rare complement-mediated kidney diseases, though the market size for pediatric C3 glomerulopathy remains limited compared to larger indications like C3 glomerulonephritis. The results could support regulatory submissions for pediatric indications and improve long-term revenue visibility if adopted into standard-of-care treatment pathways.

May 1, 2026

PubMed

Pegcetacoplan Prophylaxis for Recurrent C3 Glomerulopathy with Multifactorial Genetic Background.

# Investment Summary: Apellis Pegcetacoplan in C3 Glomerulopathy This case study demonstrates pegcetacoplan's potential utility in C3 glomerulopathy (C3GN), a rare complement-mediated kidney disease where genetic factors drive disease recurrence—a population where existing therapies have limited efficacy. The prophylactic use case could expand pegcetacoplan's addressable market beyond its approved indications (PNH, geographic atrophy) and support label expansion arguments, particularly valuable given the orphan disease designation and high unmet need in complement-driven kidney disease. However, this represents a single case report rather than controlled trial data, so commercial impact depends on whether Apellis pursues formal development in C3GN and achieves positive Phase 2/3 results to support reimbursement.

Apr 28, 2026

PubMed

Indirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment.

# Investment Summary: APLS Iptacopan vs. Pegcetacoplan **Clinical Finding:** This indirect treatment comparison suggests iptacopan (Factor B inhibitor) may offer superior efficacy to pegcetacoplan (C3 inhibitor) in PNH patients with residual anemia despite C5 inhibitor therapy, a key patient population where treatment switching or combination approaches are clinically relevant. **Commercial Implication:** The data supports iptacopan's competitive positioning in the PNH market against Apellis' own pegcetacoplan, potentially validating a differentiated mechanism for patients with inadequate response to current C5-based regimens, though it highlights direct competitive pressure within Apellis' own portfolio and from Factor B inhibitors. **Investment Relevance:** For APLS, this underscores both market opportunity (large unmet need in C

Apr 1, 2026

Apellis Pharmaceuticals Inc.

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