Expert Interview
Analyzing the Phase 3 VALIANT Study Results for Pegcetacoplan in C3G and IC-MPGN from Apellis and Sobi
Ticker(s): APLS, SOBI.XSTOInstitution: UPENN
- Associate Professor of Clinical Medicine and the Clinical Director, Glomerular Diseases Program at University of Pennsylvania.
- Currently manages 100 patients with IgAN, 1000 patients with CKD, and 15 patients with C3 glomerulopathy.
- Research focuses on Glomerular diseases, Onconephrology, and kidney biopsy.
The VALIANT study demonstrated a significant 68% reduction in proteinuria (p<0.0001) compared to placebo at Week 26. How meaningful is this reduction in proteinuria for C3G and IC-MPGN patients in terms of delaying disease progression and potentially improving long-term outcomes?
Added By: catalin_adminBeyond proteinuria reduction, the study also achieved significance on key secondary endpoints related to estimated glomerular filtration rate (eGFR) stabilization. Can you discuss the importance of these findings, particularly in preventing or slowing the decline in kidney function in patients with these rare diseases?
Added By: catalin_adminThe reduction in C3c staining observed in kidney biopsies was another positive outcome in the study. How significant is this histological improvement in indicating reduced disease activity, and what role does it play in supporting the long-term efficacy of pegcetacoplan?
Added By: catalin_adminThe results were consistent across different subgroups, including adolescent and adult patients, as well as native and post-transplant kidneys. How crucial is this broad applicability in addressing the unmet needs of diverse patient populations with C3G and IC-MPGN?
Added By: catalin_adminPegcetacoplan demonstrated a favorable safety profile, consistent with its established use in PNH. Given the chronic nature of C3G and IC-MPGN, how does this safety and tolerability profile position pegcetacoplan as a long-term therapy option compared to off-label or supportive care currently available?
Added By: catalin_adminConsidering that two-thirds of patients with a kidney transplant experience disease recurrence, what do these findings mean for post-transplant patients specifically, and could pegcetacoplan offer a viable strategy to preserve transplant function?
Added By: catalin_adminWith plans to submit regulatory filings in both the U.S. and EU in 2025, what are the key considerations for securing approval given the rarity of these diseases, and how could pegcetacoplan potentially reshape the treatment landscape for C3G and IC-MPGN?
Added By: catalin_adminAll patients who completed the VALIANT study have now enrolled in the VALE long-term extension study. What are your expectations for the long-term data, and how critical will these findings be in understanding the sustained benefits and durability of pegcetacoplan's effects over time?
Added By: catalin_adminAre You Interested In These Questions?
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