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Expert Interview

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A second look: Analyzing the Pivotal VALIANT Phase 3 Results for Pegcetacoplan in C3G and IC-MPGN from Apellis Pharmaceuticals (APLS)

Ticker(s): APLS

Who's the expert?

Institution: Montefiore

  • Medical Director, Pediatric Nephrology at Montefiore Health System, Inc., Associate Professor of Pediatrics at the Albert Einstein College of Medicine & Visiting Assistant Professor at Rockefeller University.
  • Clinical interests are polycystic kidney disease, lupus nephritis, genetic causes of stone disease, complement pathway defects, immune-mediated renal diseases (autoimmune and secondary immune disorders) and biomarkers of acute kidney injury with a research interest is in lupus nephritis.
  • Involved in clinical and translational research studying factors leading to progression to end-stage kidney disease, including fibrosis and complement-mediated pathways in lupus nephritis. 

Interview Questions
Q1.

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?

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Q2.

The 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_admin
Q3.

With 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_admin
Q4.

The 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_admin
Q5.

Given 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_admin
Q6.

Considering 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_admin
Q7.

With 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_admin
Q8.

The 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_admin

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