Apellis Initiates Registrational Programs of Pegcetacoplan in Patients with C3G / IC-MPGN and ALS, Rare Diseases with High Unmet Need
Advancing Phase 2 and Phase 3 studies of the company’s targeted C3 therapy in C3G / IC-MPGN, rare kidney diseases with no approved
medicines. First patient in Phase 2 study expected to be dosed by the end of the year
- Potentially registrational Phase 2 study in ALS to enroll ~200 adults globally. First patient expected to be dosed by the end of the year
WALTHAM, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that it has initiated registrational programs of pegcetacoplan, a targeted C3 therapy, for people living with C3 glomerulopathy (C3G) or immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare diseases that can lead to kidney failure within five to 10 years, and amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease that leads to progressive muscle weakness and paralysis. Uncontrolled activation of the complement cascade, a part of the body’s immune system, is believed to play a role in the progression of these serious diseases.
“People and families living with C3G, IC-MPGN, and ALS are in significant need of new medicines. We believe that controlling complement activation centrally, at the level of C3, has the potential to offer an important new therapeutic approach for each of these rare diseases,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. “Our data in C3G support continued advancement of pegcetacoplan, and numerous studies suggest that elevated levels of C3 may play a role in the progression of ALS. These new trials build on the broad platform potential of targeting C3, and we are working urgently to advance these programs for patients.”
Nephrology: Registrational Program for C3G / IC-MPGN
C3G / IC-MPGN are rare kidney diseases that impact ~18,000 patients in the United States and Europe1 and currently have no approved therapies. C3 deposition in the kidneys is present in both C3G and IC-MPGN; however, substantial immunoglobulin deposition is also seen in IC-MPGN.2,3
Apellis is advancing a registrational program in C3G / IC-MPGN beginning with the Phase 2 NOBLE study, a randomized, controlled trial in 12 patients with post-transplant disease recurrence that will focus on the histopathology of the kidneys. The first patient is expected to be dosed in this study by the end of 2020. Apellis also plans to begin a Phase 3 study in the first half of 2021 with reduction in proteinuria as its primary endpoint.
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