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The first indication is paroxysmal nocturnal hemoglobinuria (PNH), a rare disease of the bone marrow. Apellis' drug candidate APL-2, an inhibitor of complement component C3, is designed to provide PNH patients with an alternative to the current standard-of-care. In its second program, Apellis is testing whether APL-1, another inhibitor of complement component C3, can affect the underlying disease mechanism in COPD and slow down disease progression. Finally, Apellis' third program aims to reduce the growth of retinal lesions through the intravitreal injection of APL-2 in patients suffering from geographic atrophy, the advanced form of dry age-related macular degeneration (AMD), for which no treatments exist. All three complement immunotherapy programs focus on the potential of complement inhibition to correct pathogenic Th17 immune responses.
Via Krishan Maggon
About APL-1
APL-1 is a derivative of compstatin, a small peptide inhibitor of complement factor C3. APL-1 binds tightly to complement component C3, preventing the activation of all three major pathways of the complement system and effectively shutting down a process that could otherwise lead to local inflammation, tissue damage and dysregulation of the adaptive immune system.
About APL-2
APL-2 is a next-generation inhibitor of the class of compstatin derivatives with improved physicochemical properties. APL-2 is currently being tested in a number of Phase I clinical trials and has received Orphan Drug Designation from the FDA to treat PNH. APL-2 inhibits complement at the levels of complement factor C3, thus blocking all downstream effector pathways of the complement cascade.