Complement 3 glomerulopathy (C3G), a rare and progressive kidney disease, is seeing potential advancements with late-stage candidates from Novartis and Apellis nearing FDA submission. These therapies, already approved for other conditions, are showing promise in addressing the unmet needs of C3G patients who currently have limited long-term treatment options.
Urgent Need for Innovative C3G Therapies
C3G is characterized by inflammation and kidney damage, leading to symptoms such as high blood pressure, proteinuria, hematuria, swelling, fatigue, and recurrent infections. Current treatments like steroids and immunosuppressants offer only short-term relief and come with significant risks and side effects. A study by Spherix revealed that nephrologists are eager for new therapies that demonstrate slowed eGFR decline and proteinuria reduction, emphasizing the importance of targeting the complement system pathways.
Spherix’s analysis of 157 C3G patient records indicated that physicians would likely treat up to half of their C3G patients with new agents if approved. Projections suggest that a majority of these patients are likely to require dialysis within the next ten years, underscoring the critical need for treatments that can delay disease progression.
Novartis' Fabhalta (Iptacopan) Shows Promise
Novartis presented one-year data from its Phase III APPEAR-C3G trial of Fabhalta (iptacopan), an oral complement inhibitor, at ASN Kidney Week. The data demonstrated a significant and sustained reduction in proteinuria within 14 days of treatment, lasting up to 12 months. The drug met its primary endpoint at six months, and Novartis has already submitted applications for approval beyond the U.S., including in the EU, China, and Japan.
Apellis' Empaveli (Pegcetacoplan) Demonstrates Efficacy
Apellis, in collaboration with Sobi, presented Phase III VALIANT trial data for Empaveli (pegcetacoplan) at the same conference, showing a 68.1% reduction in proteinuria versus placebo. Additionally, 71.4% of treated patients achieved zero C3c staining intensity, signaling C3c deposit clearance. The VALIANT trial includes patients with both C3G and IC-MPGN, demonstrating early efficacy by week four.
Treatment Preferences and Market Dynamics
Spherix data indicates that treatment preferences are likely to be influenced by the route of administration, with Fabhalta taken orally twice daily and Empaveli administered subcutaneously twice weekly. This factor could play a significant role in the adoption of these therapies once they are approved.
As the C3G treatment landscape evolves, Spherix plans to continue providing in-depth insights into market developments through its Market Dynamix™, Patient Chart Dynamix™, and Launch Dynamix™ services.
