(APLS) Apellis Pharmaceuticals - Ratings and Ratios

Apellis Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company that specializes in targeting the complement system—a critical part of the immune system—to treat autoimmune and inflammatory diseases. Their approach focuses on inhibiting this system to address conditions where it plays a central role in driving pathology.

Their lead product, EMPAVELI (pegcetacoplan), is approved for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder, and two types of kidney diseases: C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Additionally, EMPAVELI is used to treat thrombotic microangiopathy (TMA) associated with hematopoietic stem cell transplantation (HSCT), which is a serious complication in stem cell transplant patients.

SYFOVRE (pegcetacoplan) is their second approved drug, targeting geographic atrophy (GA), a leading cause of vision loss in older adults. GA is a condition where the macula, the part of the eye responsible for central vision, deteriorates. This is a significant area of unmet need, and SYFOVRE represents a first-in-class therapy for this indication.

Beyond their approved products, Apellis has a robust pipeline. APL-3007, a small interfering RNA (siRNA), is in Phase 1 clinical trials. This molecule is designed to target the complement system at a different point in the pathway, offering potential for new therapeutic applications. Additionally, they are developing an oral complement inhibitor, which is still in preclinical development—a promising candidate given the convenience oral administration offers over injectable therapies.

The company has formed strategic collaborations to enhance its capabilities. Their partnership with Swedish Orphan Biovitrum (Sobi) focuses on the development and commercialization of pegcetacoplan, expanding its reach and distribution network. Another notable collaboration is with Beam Therapeutics, leveraging Beams base editing technology to explore novel treatments for complement-driven diseases. This partnership underscores Apellis commitment to innovation and accessing cutting-edge technologies to stay ahead in the competitive biotech landscape.

Founded in 2009 and headquartered in Waltham, Massachusetts, Apellis has established itself as a leader in the complement system space. Their focus on a specific biological pathway has allowed them to build