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Immunoglobulin A (IgA) nephropathy (IgAN) is a chronic immune-mediated disease with significant patient burden, underscoring the need for disease-modifying therapies that prevent kidney function decline. Nefecon is a novel, oral, targeted-release capsule formulation of budesonide, designed to treat IgAN by downregulating the production of pathogenic IgA1 antibody production at the source (i.e., the Peyer’s patch–rich distal ileum). Nefecon is approved by the FDA and EMA for adult patients with primary IgAN at risk of rapid disease progression. Previously published results from the 2-year global NefIgArd trial demonstrated that Nefecon 16 mg/day treatment for 9 months was associated with a significant reduction in time-weighted average change in estimated glomerular filtration rate (eGFR) over 2 years of 5.1 mL/min/1.73 m2 vs placebo. It also demonstrated a significant 30% reduction in urine protein-creatinine ratio (UPCR) at 9 months and a 50% reduction at 12 months vs placebo. Here, we present the 2-year results of quality of life (QoL) analyses based on 36-Item Short Form Survey (SF-36) assessments at 9 and 24 months.
NefIgArd was a global, double-blind, randomized, placebo-controlled Phase 3 trial in patients with primary IgAN despite optimized renin–angiotensin system inhibition. Eligibility criteria included biopsy-confirmed primary IgAN, persistent proteinuria (UPCR ≥0.8 g/g or proteinuria ≥1 g/24 h), and an eGFR of 35-90 mL/min/1.73 m2. Patients received a 9-month treatment course of Nefecon 16 mg/day or placebo in addition to optimized supportive care, followed by a 15-month off-drug observational period. The SF-36 QoL questionnaire contains 36 questions, each of which are grouped into one of eight subscales: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems, and mental health. Final scores for each subscale range from 0 to 100, with a higher score indicating better health and QoL. The mean re-coded scores for each of the eight subscales and the overall physical and mental health scores were summarized by treatment group at baseline, 9 months, and 24 months.
The full analysis set comprised 364 patients. SF-36 scores were similar between groups at baseline across all QoL domains. As shown in the Table, no meaningful differences in any QoL domain were observed between Nefecon and placebo groups after 9 months of treatment. SF-36 scores remained consistent after 15 months of off-drug observational follow-up.
These findings further support the benefit/risk profile of Nefecon, demonstrating that 9 months of treatment with Nefecon 16 mg/daily did not result in changes in QoL at any measure. This was achieved while also providing significant benefit in preserving kidney function.