Apellis Pharmaceuticals has reported positive results for pegcetacoplan from the phase 2 DISCOVERY clinical trial in patients with C3 glomerulopathy (C3G), a type of kidney disease.
According to the US biopharma company, the 48-week results demonstrated that patients subjected to pegcetacoplan had sustained improvements across key clinical measures. These include a greater than 65% mean reduction in proteinuria, which is a key marker of renal damage.
Considered to be a rare, complement-driven kidney disease, C3 glomerulopathy currently has no approved drugs.
Apellis Pharmaceuticals will present the phase 2 DISCOVERY clinical trial results at the American Society of Nephrology (ASN) Kidney Week on 22 October 2020.
Pegcetacoplan is an investigational, targeted C3 therapy. It has been designed by the biopharma company to control surplus complement activation, which can result in the onset and progression of several serious diseases.
The investigational drug candidate, which is a synthetic cyclic peptide that is conjugated to a polyethylene glycol polymer, specifically binds to C3 and C3b.
The phase 2 DISCOVERY clinical trial saw a reduction in mean (SE) proteinuria from 3.48 (0.82) mg/mg at baseline to 0.93 (0.27) mg/mg at week 48.
The patients also had a 73.3% reduction in mean (SE) proteinuria as measured by 24-hour urine protein-to-creatinine ratio (uPCR).
Apellis Pharmaceuticals said that what was important is that the reduction in proteinuria came along with a corresponding increase in mean serum albumin.
The biopharma company said that there was improvement across other biomarkers. These include an increase in mean serum C3 and stabilization of renal function, as measured by mean serum creatinine.
There were also no serious or severe adverse events reported in the phase 2 DISCOVERY clinical trial and pegcetacoplan was well tolerated overall, said Apellis Pharmaceuticals.
Federico Grossi – Chief Medical Officer of Apellis Pharmaceuticals said: “There is an urgent need for treatments for C3G, a disease that ultimately leads to kidney failure for about half of people living with the disease. These positive results highlight the potential of pegcetacoplan to make a meaningful difference for the C3G community and further strengthen our confidence in targeting C3 across multiple complement-driven diseases.
“We are working quickly to progress a registrational program of our targeted C3 therapy for people living with C3G.”
Earlier this week, Apellis Pharmaceuticals said that it was advancing phase 2 and phase 3 studies of the C3 therapy in patients having C3 glomerulopathy or immune complex membranoproliferative glomerulonephritis (IC-MPGN), which is a rare, complement-driven kidney disease as well with no approved medicines.