Apellis and Novartis both target rare kidney diseases with new drugs

Apellis Pharmaceuticals and Novartis recently shared data for their rare kidney disease drugs, pegcetacoplan and iptacopan. Both have performed well in treating C3 glomerulopathy (C3G) and improving estimated glomerular filtration rate (eGFR).
Apellis, which partnered with Sobi for its C3 therapy pegcetacoplan, showed that in phase 3 trial results, the treatment successfully met primary and secondary endpoints in patients with primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) or debilitating kidney diseases, including C3 glomerulopathy.
In the late-stage VALIANT study, at the 24-week mark, pegcetacoplan showed a statistically significant and clinically meaningful reduction in proteinuria. The trials tested Apellis’ C3 med in combination with standard of care against placebo and standard of care treatment.

The reductions in proteinuria were first observed around the 4-week mark and then continued for the entire 6-month duration of the trial. Reductions were consistent in patients across the two diseases and across age demographics and transplant status. Children and adults, both those who had and had not received kidney transplants, experienced consistent reductions.
Apellis and Sobi revealed topline data on pegcetacoplan in early August, and phase 3 trial results have been in line with those. A new data package on estimated glomerular filtration rate (eGFR), however, has been added to the mix now. Pegcetacoplan has been seen to achieve stabilization of estimated glomerular filtration rate (eGFR) compared to placebo over 6 months. eGFR is considered an important category to investigate because it is a key measure of kidney function.
Implicated in the body’s immune system, C3c is a fragment of the protein complement component 3. If present in excess in the body, this fragment can lead to kidney inflammation, damage, or failure and is considered a marker for disease activity. When it comes to achieving a reduction in C3c staining intensity, only a little over 11% of the participants in the control cohort fell by two or more orders of magnitude from baseline, while the figure went over 73% for patients on pegcetacoplan.
Only two days after Apellis, Novartis also shared data for its drug iptacopan. It was observed that as early as within the first two weeks of the study, two daily doses of the oral drug could lead to clinically meaningful proteinuria reductions. These results were sustained over a year.
At the trial’s six-month mark, compared to a placebo on top of supportive care, Novartis’s iptacopan resulted in over 35% reduction in proteinuria.
APPEAR-C3G, an open-label period of the phase 3 study, showed that even when switched over to Novartis’ drug from another treatment, a reduction in proteinuria was observed in patients. At the start of the treatment, iptacopan also caused an improvement in eGFR.
Cross-trial comparisons, although generally considered inherently flawed, in the case of Apellis’s and Novartis’s drugs show that the former might be taking the lead over the latter on key kidney disease metrics. Novartis, however, is able to make up for this with a head start in potential approvals.

Already the Swiss pharma has sent out regulatory submissions for the new indications of iptacopan in China, Japan, and Europe. In the case of the U.S., the company plans to submit a file by the end of the year. In contrast, Apellis plans to file an application for its drug with the FDA sometime in early 2025.

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