First patient dosed in the VALIANT phase III study of pegcetacoplan for IC-MPGN and C3G, rare kidney diseases with high unmet medical need.
Sobi and Apellis Pharmaceuticals, Inc. announced that the first patient has been dosed in the VALIANT phase III study investigating pegcetacoplan, a targeted C3 therapy, in primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G) two rare and debilitating kidney diseases with similar underlying causes and no approved treatment.
“Individuals with IC-MPGN and C3G experience a high burden of disease, due to the lack of approved treatments to slow or stop disease progression. These diseases often lead to kidney failure, requiring dialysis or a kidney transplant, imposing further substantial burdens,” said Kristen Hood, MSN, RN, Executive Director of Research Engagement at Nephcure Kidney International. “We are excited that Apellis and Sobi are advancing a phase III study of pegcetacoplan in patients aged 12 and older, with primary IC-MPGN or C3G, including those with post-transplant recurrence.”
Uncontrolled activation of the complement cascade, a part of the body’s immune system, is believed to play a critical role in the progression of IC-MPGN and C3G where excessive accumulation of C3 breakdown products in the kidney causes inflammation and organ damage. It is estimated that up to 8,000 people in Europe and 5,000 in the United States are living with IC-MPGN or C3G5, and approximately 50 per cent ultimately suffer from kidney failure within five to ten years of diagnosis.
“Sobi and Apellis are the only companies actively pursuing a treatment for IC-MPGN," said Anders Ullman, MD, PhD, Head of Research and Development and Chief Medical Officer at Sobi. “We are pleased that the first patient has been dosed in this phase III study for IC-MPGN and C3G, both of which represent an extremely high unmet medical need. We are committed to bringing a meaningful treatment to patients affected by these severe diseases.”
About the VALIANT study: The VALIANT phase III study (NCT05067127) is a randomised, placebo-controlled, double-blinded, multi-centre study designed to evaluate pegcetacoplan efficacy and safety in approximately 90 patients who are 12 years of age and older with primary IC-MPGN or C3G. It is the only study to include both native kidney patients and patients who have recurrent disease after receiving a kidney transplant. Study participants will be randomised to receive 1080 mg of pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomised, controlled period, patients will proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study is the proportion of study participants with a reduction from baseline in urine protein-to-creatinine ratio (uPCR) of at least 50 per cent at week 26. uPCR is an important indicator of kidney function.
About immune-complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G); IC-MPGN and C3G are rare, debilitating kidney diseases that are estimated to affect up to 8,000 people in Europe and 5,000 in the United States. There are no approved therapies for the diseases, and symptoms include blood in the urine, dark foamy urine due to the presence of protein, swelling, and high blood pressure. Approximately 50 per cent of people living with IC-MPGN and C3G ultimately suffer from kidney failure within five to ten years of diagnosis. There are no treatments available that target the underlying complement-mediated mechanism of these diseases and prevent loss of kidney function, before or after renal transplant. Although IC-MPGN is considered a distinct disease from C3G, the underlying cause and progression of the two diseases are remarkably similar and include overactivation of the complement cascade, with excessive accumulation of C3 breakdown products in the kidney causing inflammation and damage to the organ.
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