EVIDENCE FOR MEANINGFUL PATIENT BENEFIT OF PEGCETACOPLAN IN C3G AND PRIMARY IC-MPGN FROM THE VALIANT TRIAL
Author(s)
Richard Lafayette, Prof., MD1, Mingyi Huang, PhD2, Carly Rich, BSc, MSc, PhD3, Tom Booth, PhD4, Meg Fluharty, PhD4, Caleb Dixon, MSc4, Sarah Acaster, MSc4, Sayna Norouzi, MD5, Fernando Caravaca, MD PhD6, Daniel Gale, PhD7;
1Stanford University, Stanford, CA, USA, 2Apellis Pharmaceuticals, Waltham, MA, USA, 3Sobi, Stockholm, Sweden, 4Acaster Lloyd, London, United Kingdom, 5Loma Linda University Medical Centre, Department of Nephrology, Loma Linda, CA, USA, 6Instituto de Investigación Hospital Universitario 12 de Octubre, Department of Nephrology, Madrid, Spain, 7University College London, London, United Kingdom
1Stanford University, Stanford, CA, USA, 2Apellis Pharmaceuticals, Waltham, MA, USA, 3Sobi, Stockholm, Sweden, 4Acaster Lloyd, London, United Kingdom, 5Loma Linda University Medical Centre, Department of Nephrology, Loma Linda, CA, USA, 6Instituto de Investigación Hospital Universitario 12 de Octubre, Department of Nephrology, Madrid, Spain, 7University College London, London, United Kingdom
OBJECTIVES: VALIANT, a Phase III trial of pegcetacoplan in C3G and primary IC-MPGN, met its primary endpoint of reduction in urine protein-to-creatine ratio (UPCR) at week 26. Evaluation of the patient relevance of biomarkers is important for regulatory and payor decision making regarding treatment benefit.
METHODS: This study explored the patient relevance of the UPCR treatment effect in VALIANT. Group differences in a Patient Global Impression of Change (PGI-C) by treatment (pegcetacoplan[N=42] versus placebo[N=41]) was assessed using Fisher’s Exact Test’s. Differences in patient-reported outcomes (PROs) at week 26 by UPCR reduction (≥50% reduction versus <50% reduction from baseline) were tested using Kruskal-Wallis tests. UPCR reduction groups were based on evidence of clinical relevance. PRO scores included the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue total score (range 0-52) and the total and subscale scores of the Kidney Disease Quality of Life (KDQoL-36; range 0-100) measure.
RESULTS: Significant differences in PGI-C response were observed (p=0.021) at week 26. A higher proportion of participants treated with pegcetacoplan (N=14; 33.3%) reported being ‘much improved’ compared to those on placebo (N=6;14.6%).At week 26, patients with ≥50% reduced UPCR were more likely to report less fatigue (H=6.90;p=0.009), with a median score of 47.5 on the FACIT-Fatigue compared to a median score of 40.0 for those with <50% reduction; with higher scores indicating better health-related quality of life (HRQoL). Patients with ≥50% reduced UPCR also reported less impact of their symptoms on daily activities (H=4.88;p=0.027) as assessed by the KDQoL-Effects scale, with median scores of 96.9 and 84.4 for the ≥50% reduced UPCR vs <50% reduced UPCR, respectively; with higher scores indicating better HRQoL. There were no other significant effects between reduced UPCR groups on other KDQoL domains.
CONCLUSIONS: The results suggest that the pegcetacoplan UPCR treatment effect reflects a patient-relevant benefit in the C3G and primary IC-MPGN VALIANT trial.
METHODS: This study explored the patient relevance of the UPCR treatment effect in VALIANT. Group differences in a Patient Global Impression of Change (PGI-C) by treatment (pegcetacoplan[N=42] versus placebo[N=41]) was assessed using Fisher’s Exact Test’s. Differences in patient-reported outcomes (PROs) at week 26 by UPCR reduction (≥50% reduction versus <50% reduction from baseline) were tested using Kruskal-Wallis tests. UPCR reduction groups were based on evidence of clinical relevance. PRO scores included the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue total score (range 0-52) and the total and subscale scores of the Kidney Disease Quality of Life (KDQoL-36; range 0-100) measure.
RESULTS: Significant differences in PGI-C response were observed (p=0.021) at week 26. A higher proportion of participants treated with pegcetacoplan (N=14; 33.3%) reported being ‘much improved’ compared to those on placebo (N=6;14.6%).At week 26, patients with ≥50% reduced UPCR were more likely to report less fatigue (H=6.90;p=0.009), with a median score of 47.5 on the FACIT-Fatigue compared to a median score of 40.0 for those with <50% reduction; with higher scores indicating better health-related quality of life (HRQoL). Patients with ≥50% reduced UPCR also reported less impact of their symptoms on daily activities (H=4.88;p=0.027) as assessed by the KDQoL-Effects scale, with median scores of 96.9 and 84.4 for the ≥50% reduced UPCR vs <50% reduced UPCR, respectively; with higher scores indicating better HRQoL. There were no other significant effects between reduced UPCR groups on other KDQoL domains.
CONCLUSIONS: The results suggest that the pegcetacoplan UPCR treatment effect reflects a patient-relevant benefit in the C3G and primary IC-MPGN VALIANT trial.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR198
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Urinary/Kidney Disorders