IgA Nephropathy Primary Endpoint Met at a Planned Interim Analysis
NEFIGAN Trial Hits Efficacy Target Based on Highly Statistically Significant Reduction of Proteinuria
Stockholm, Sweden – 14 April 2015. Pharmalink AB, a specialty pharma company, today announces that the NEFIGAN Trial of Nefecon® for the treatment of primary IgA nephropathy has fully met its primary efficacy endpoint at a planned interim analysis and been stopped early with respect to statistical analysis of the endpoint.
The NEFIGAN Trial is a Phase 2b randomized double-blinded, placebo-controlled clinical trial assessing the safety and efficacy of two different doses of Nefecon – a new oral modified-release capsule of the corticosteroid, budesonide – administered daily over a nine-month treatment period to primary IgA nephropathy patients with persistent proteinuria despite optimized standard-of-care therapy. The trial has been conducted in 62 centers in 10 European countries and was originally intended to recruit 90 patients. Over-recruitment has increased this to more than 150 patients.
“Meeting the primary endpoint of the NEFIGAN Trial with such high significance and at this interim analysis is a tremendous result and major milestone for Pharmalink,” said Johan Häggblad, Ph.D., Managing Director of Pharmalink. “The NEFIGAN Trial was conducted with excellent efficiency, and the fact that the patient recruitment target was also exceeded so rapidly indicates the clear unmet medical need for new treatments for IgA nephropathy patients. We would like to thank all the investigators and patients who contributed to this clinical trial and look forward to the further development of Nefecon.”
Bengt Fellström, MD, PhD, Professor of Nephrology at Uppsala University Hospital, Sweden and Principal Investigator of the NEFIGAN Trial, added: “IgA nephropathy is the most common inflammatory renal disease and in real need of new treatment options. Existing options are insufficient to prevent a significant proportion of patients progressing to renal failure, with a devastating impact of patients’ quality of life. No current therapies address the root cause of the disease which makes the results from the NEFIGAN Trial all the more encouraging. A new medicine for early treatment with the potential to stop disease progression and minimize any further loss of renal function would be very welcome news indeed to patients and clinicians alike.”
The interim analysis, conducted by the Data & Safety Monitoring Board (DSMB), demonstrated that Nefecon treatment (both treatment groups combined) resulted in a highly significant improvement [p=0.0066 on an intention-to-treat (ITT) basis] in the primary endpoint, defined as the mean reduction in urine protein creatinine ratio (UPCR) during the nine-month treatment period, as compared to placebo. The result will enable an optimal dose of Nefecon to be selected for a Phase 3 registration trial.
Enrolled patients will conclude the treatment phase by the end of April and complete the three-month follow-up phase of the study, with results expected in Q3 2015. Results from the study will be presented at upcoming scientific meetings and submitted for publication in a peer-reviewed journal.