Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) today announced positive results from a Phase 2 study of cemdisiran, an investigational RNAi therapeutic targeting the C5 component of the complement pathway, in development in collaboration with Regeneron Pharmaceuticals for the treatment of adult patients with immunoglobulin A nephropathy (IgAN). The results were presented at the 18th European Meeting on Complement in Human Disease (EMCHD) being held in Bern, Switzerland on August 26-28.
“We are pleased to present additional data from our Phase 2 study demonstrating that cemdisiran had favorable effects on different measures of proteinuria – a strong risk factor for disease progression in patients with IgAN,” said Sonalee Agarwal, Ph.D., Vice President and Program Leader for the Cemdisiran program at Alnylam. “IgAN is an inflammatory disease that can lead to severe loss of kidney function. Thus, given the persistent unmet need in the treatment landscape of this progressive disease, we, together with our partners at Regeneron, are working expeditiously to advance this investigational RNAi therapeutic into Phase 3 clinical development.”
The data showed that at Week 32, as previously reported, cemdisiran demonstrated a clinically meaningful reduction in 24-hour urine protein to creatinine ratio (UPCR) – the primary endpoint of this Phase 2 study – with a 37 percent (90 percent CI: -0.5, 61) reduction in 24-hour UPCR observed relative to placebo. New results that were presented demonstrated an equivalent (36 percent [90 percent CI: -6, 62]) reduction in 24-hour urine total protein and a higher proportion, 32 versus 13 percent, of patients treated with cemdisiran as compared to those on placebo, respectively, achieving greater than or equal to 50 percent reduction in 24-hour UPCR. Spot urine data were consistent with 24-hour urine data, with the initial onset of treatment effect emerging as early as Week 8 and remaining stable over time. Specifically, patients on cemdisiran achieved a 46 percent (90 percent CI: 26, 60) placebo-adjusted reduction from baseline in spot UPCR at 32 weeks.
Furthermore, the results showed that cemdisiran was generally well tolerated in patients with IgAN with no adverse events (AEs) leading to treatment or study discontinuation during the double-blind treatment period. One death occurred in the cemdisiran arm due to cardiorespiratory collapse; this was not considered related to study drug by the study investigator. AEs reported by greater than or equal to 10 percent of patients in the cemdisiran arm were injection site reactions (41 percent) and peripheral edema (14 percent). There were no drug-related serious or severe AEs.
To view all results presented by Alnylam and collaborators at EMCHD please visit Capella.
