Pipeline

STL303

STL303 is an oral, once-daily small molecule and a potent, highly selective inhibitor of complement factor B.

PNH

Paroxysmal nocturnal hemoglobinuria, or PNH, is a rare, acquired blood disorder in which red blood cells are vulnerable to destruction by the complement system. This can lead to hemolysis, anemia, fatigue, blood clots and other serious complications.

In PNH, blood cells lack key protective proteins that normally prevent complement mediated attack. As a result, the complement system can become overactive and destroy healthy red blood cells, driving ongoing inflammation and disease burden.

STL303 is an oral small molecule designed to inhibit complement factor B, a key driver of the alternative complement pathway, with the goal of reducing complement-mediated red blood cell destruction and improving outcomes for patients with PNH.

IgAN

IgA nephropathy, or IgAN, is an autoimmune kidney disease that causes glomerulonephritis and can lead to progressive loss of kidney function.

In IgAN, B cells produce abnormally glycosylated IgA1, known as Gd-IgA1. The immune system recognizes Gd-IgA1 as abnormal and produces autoantibodies against it, leading to the formation of immune complexes. These immune complexes deposit in the kidney, triggering inflammation, complement activation and kidney damage.

STL303 is designed to inhibit complement factor B, a key regulator of the alternative complement pathway implicated in IgAN, with the aim of reducing immune complex-mediated damage and preserving kidney function.

GA

Age-related macular degeneration, or AMD, is classified as early, intermediate or late-stage disease. Late-stage AMD includes two subtypes: geographic atrophy, or GA, and choroidal neovascularisation, or CNV.

GA causes the loss of retinal cells, leading to blurred areas or blind spots in central vision. Genetics, inflammation and complement activation have all been linked to the development and progression of AMD.

By inhibiting complement factor B, STL303 is designed to reduce harmful inflammation, with the aim of preserving retinal cells and ultimately protecting vision.