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Expert Interview

Slingshot members are talking to an expert! The topic is:

Discussing investigational agents for C3 glomerulopathy (C3G) including pegcetacoplan and iptacopan

Ticker(s): NVS, APLS

Who's the expert?

Institution:  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.

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 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?

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Q3.

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?

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Q4.

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?

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Q5.

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?

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Q6.

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?

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Q7.

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
Q8.

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?

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