Expert Interview
Discussing investigational agents for C3 glomerulopathy (C3G) including pegcetacoplan and iptacopan
Ticker(s): NVS, APLSInstitution: Mass General | Harvard
- Clinical researcher & clinician in academic nephrology practice; Nephrologist at Massachusetts General Hospital and Instructor in Medicine at Harvard Medical School.
- Currently manages 8-10 patients with CKD associated pruritus, and 6 patients with C3 glomerulopathy.
- Conducts patient-oriented research in the areas of calciphylaxis, vascular calcification and hyponatremia, has authored over 50 peer-reviewed research articles, and research is funded by organizations including the American Kidney Fund & the National Kidney Foundation.
The VALIANT study reported a statistically significant 68% reduction in proteinuria for pegcetacoplan-treated patients compared to placebo. How meaningful is this level of reduction in delaying disease progression and improving outcomes for C3G and IC-MPGN patients?
Added By: wilson_adminThe VALIANT study results provide an optimistic outlook, but what are your expectations for the long-term efficacy and durability of pegcetacoplan as patients transition into the open-label phase and the long-term VALE study?
Added By: wilson_adminThe stabilization of eGFR observed in pegcetacoplan-treated patients is a critical marker of kidney function. Could you discuss the implications of this finding for long-term kidney health in C3G and IC-MPGN patients?
Added By: wilson_adminWith 71% of pegcetacoplan-treated patients achieving zero C3c staining intensity, what does this level of deposit clearance suggest about the treatment’s ability to reduce disease activity and inflammation?
Added By: wilson_adminThe results were consistent across C3G and IC-MPGN patients, as well as adolescents, adults, and both native and post-transplant kidneys. How significant is this broad applicability for meeting the treatment needs of diverse patient groups?
Added By: wilson_adminGiven the favorable safety profile demonstrated in VALIANT, how does pegcetacoplan’s safety and tolerability compare to current options, and how critical is this for patients requiring long-term treatment?
Added By: wilson_adminConsidering the high recurrence rate of disease in post-transplant patients, what does this study indicate about pegcetacoplan’s potential to support transplant longevity and reduce recurrence risk?
Added By: wilson_adminWith plans to submit regulatory filings in the U.S. and EU in early 2025, what are the main considerations for pegcetacoplan’s approval, especially given the rarity and severity of C3G and IC-MPGN?
Added By: wilson_adminAre You Interested In These Questions?
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