Apellis initiates mid-stage pegcetacoplan studies in rare kidney, neurological diseases

• Apellis Pharmaceuticals (NASDAQ:APLS) has initiated registrational programs of pegcetacoplan for C3 glomerulopathy (C3G) or immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare kidney diseases, and amyotrophic lateral sclerosis (ALS), a rare neurodegenerative disease that leads to progressive muscle weakness and paralysis.
• The company will commence Phase 2 NOBLE study in 12 C3G / IC-MPGN patients, with first patients to be dosed by the end of this year. Primary endpoint is the proportion of patients with reduction in C3c staining on renal biopsy after 12 weeks of treatment. It also plans to begin a Phase 3 study in 1H of 2021 with reduction in proteinuria (excess proteins in the urine) as its primary endpoint.
• Apellis is expanding pegcetacoplan development into neurology with a potentially Phase 2 study in ALS. MERIDIAN study has been initiated in ~200 patients, with first patient to be dosed by end of this year. Primary endpoint is the Combined Assessment of Function and Survival rank scores at week 52.
• Last month, the company submitted a marketing application for pegcetacoplan for rare blood disease, in the U.S. and European Union.
• https://seekingalpha.com/news/3619738-apellis-initiates-mid-stage-pegcetacoplan-studies-in-rare-kidney-and-neurological-diseases